This document provides information on investment opportunities in pediatric health at a leading pediatric academic medical center. It summarizes the center's patient population and annual statistics, lists several interdisciplinary centers focused on critical pediatric health issues, describes the research institute including NIH funding and clinical trials, and provides information on the center for innovation and several startup companies with pediatric health technologies.
Cancer immunotherapy market & clinical pipelineKuicK Research
This document discusses cancer immunotherapy and provides an overview of the market and clinical pipeline. It notes that cancer immunotherapy utilizes the body's immune system to target cancerous cells and has advantages over conventional treatments like chemotherapy. The market for cancer immunotherapy has grown significantly in recent years and is expected to continue expanding due to the development of new immunotherapies. The document also outlines the large number of immunotherapies currently in clinical trials, including monoclonal antibodies, vaccines, oncolytic viruses, cytokines, and cell therapies. It analyzes the competitive landscape and clinical pipelines of major pharmaceutical companies developing cancer immunotherapies.
This document provides information about Dr. Paul Cornes and his work in oncology. It discloses that Dr. Cornes receives a salary from the UK National Health Service and has received honoraria from several pharmaceutical companies. The bulk of the document discusses the costs of cancer to individuals and societies and highlights both challenges and areas of progress in cancer treatment, including improved survival rates due to innovations in targeted therapies and monoclonal antibodies.
Medicines and vaccines have helped deliver improvements in patient health. History shows us the great advances we have made - today we continue to see the potential to eradicate disease and improve health outcomes when we invest in science and adopt and use new medicines.
Ομιλία - Παρουσίαση: “The Value of Innovation to Patients & Health Systems”
Clare Hague PhD, Therapy Area Market Access Leader for Hematology, Janssen EMEA Region
The document discusses the importance of manufacturing to the UK pharmaceutical industry and the life sciences sector. It outlines the goals of the Medicines Manufacturing Industry Partnership (MMIP) in making the UK an attractive location for medicines manufacturing through initiatives like skills development programs. The document also notes industry's significant financial contributions to the NHS through the Pharmaceutical Price Regulation Scheme, and the importance of ensuring a regulatory framework that supports innovation as the UK leaves the EU.
The UK is at the forefront of the global pharmaceutical industry. As well as developing new medicines for many diseases, the pharmaceutical industry in the UK provides many other benefits to the British economy, including income, employment, expertise and major investment.
di Pier Giuseppe Pelicci, MD-PhD, Istituto Europeo di Oncologia IEO, Università degli Studi di Milano.
Slide per l'intervento tenuto in Fondazione Giannino Bassetti in occasione del primo incontro del ciclo "La medicina di precisione", primo progetto dalla convenzione tra Università di Pavia e Fondazione Bassetti.
12 marzo 2018
Cancer immunotherapy market & clinical pipelineKuicK Research
This document discusses cancer immunotherapy and provides an overview of the market and clinical pipeline. It notes that cancer immunotherapy utilizes the body's immune system to target cancerous cells and has advantages over conventional treatments like chemotherapy. The market for cancer immunotherapy has grown significantly in recent years and is expected to continue expanding due to the development of new immunotherapies. The document also outlines the large number of immunotherapies currently in clinical trials, including monoclonal antibodies, vaccines, oncolytic viruses, cytokines, and cell therapies. It analyzes the competitive landscape and clinical pipelines of major pharmaceutical companies developing cancer immunotherapies.
This document provides information about Dr. Paul Cornes and his work in oncology. It discloses that Dr. Cornes receives a salary from the UK National Health Service and has received honoraria from several pharmaceutical companies. The bulk of the document discusses the costs of cancer to individuals and societies and highlights both challenges and areas of progress in cancer treatment, including improved survival rates due to innovations in targeted therapies and monoclonal antibodies.
Medicines and vaccines have helped deliver improvements in patient health. History shows us the great advances we have made - today we continue to see the potential to eradicate disease and improve health outcomes when we invest in science and adopt and use new medicines.
Ομιλία - Παρουσίαση: “The Value of Innovation to Patients & Health Systems”
Clare Hague PhD, Therapy Area Market Access Leader for Hematology, Janssen EMEA Region
The document discusses the importance of manufacturing to the UK pharmaceutical industry and the life sciences sector. It outlines the goals of the Medicines Manufacturing Industry Partnership (MMIP) in making the UK an attractive location for medicines manufacturing through initiatives like skills development programs. The document also notes industry's significant financial contributions to the NHS through the Pharmaceutical Price Regulation Scheme, and the importance of ensuring a regulatory framework that supports innovation as the UK leaves the EU.
The UK is at the forefront of the global pharmaceutical industry. As well as developing new medicines for many diseases, the pharmaceutical industry in the UK provides many other benefits to the British economy, including income, employment, expertise and major investment.
di Pier Giuseppe Pelicci, MD-PhD, Istituto Europeo di Oncologia IEO, Università degli Studi di Milano.
Slide per l'intervento tenuto in Fondazione Giannino Bassetti in occasione del primo incontro del ciclo "La medicina di precisione", primo progetto dalla convenzione tra Università di Pavia e Fondazione Bassetti.
12 marzo 2018
The UK is at the forefront of the global pharmaceutical industry. As well as developing new medicines for many diseases, the pharmaceutical industry in the UK provides many other benefits to the British economy, including income, employment, expertise and major investment.
These slides offer a useful, referenced resource for members and visitors to our website who wish to share the story about the value of medicines. They complement existing resources available on the ABPI website and will be updated regularly as the ABPI updates other data and content.
OxfordSM's pharma case studies - providing a call to actionOxfordSM
Brand teams have to be increasingly innovative when finding ways to prompt patients and physicians to intervene at the right time.
Campaigns such as GSK’s Greatest Season Ever for FLONASE®, implemented last year in the United States which made the decision to prepare for the allergy season easier for patients by linking the proactive purchase of the brand to the start of the baseball season.
Providing A Call To Action:
We find that examples from within and outside of healthcare can often prompt this innovation. They act as a way of bringing in new perspectives and allowing teams to explore new avenues and new ideas.
So, in the spirit of hoping this will prompt some new ideas in your brand team, here are our favourite case studies that speak to the need to provide a call to action.
The document discusses the UK's commitment to becoming a global leader in stratified medicine. Some key points:
- Stratified medicine aims to optimize treatment for individual patients rather than broad disease groups, improving outcomes and unlocking business opportunities.
- The UK offers world-leading research expertise, data and infrastructure like large patient datasets and biobanks to help businesses develop and deliver stratified medicine products.
- There are significant market opportunities for stratified medicine and companion diagnostics globally. The UK can help businesses prepare for and access this growing market.
The future of healthcare is an exciting one. With innovations in genomics, healthcare data, advanced therapies and innovative technologies, our industry will continue to progress and provide hope to people so they can live longer, healthier and productive lives.
This document discusses various topics related to drug development including:
- The high costs of drug development which average $1.2 billion per approved drug.
- Success rates for drugs entering clinical trials are low, around 10-11% make it from Phase I to approval.
- Biomarkers and pharmacogenomics can help improve efficiency by identifying patients most likely to respond to treatments. Enrichment strategies focus trials on these responsive patient groups.
- Adaptive trial designs and learning versus confirming approaches also aim to make trials more efficient and lower costs.
Trailblazing scientists who are the backbone of our industry. These are the people that discover the molecules and develop the medicines to tackle the toughest diseases we face in society.
The document discusses the key role of diagnostics in addressing the challenge of antimicrobial resistance (AMR). It makes three key points:
1) AMR is a major threat as many infections are becoming increasingly difficult to treat due to drug-resistant bacteria. Without effective antibiotics, common infections and surgeries could become life-threatening.
2) Diagnostics are critical to appropriate antibiotic use by enabling rapid and precise diagnosis of whether a bacterial or viral infection is present and, if bacterial, determining the best antibiotic to use or avoid. This can help reduce unnecessary antibiotic prescribing.
3) The document outlines a vision for AMR diagnostics in the UK to ensure rapid diagnostic technologies are universally available to guide antibiotic
Download Global breast cancer vaccine market & clinical pipeline outlook ...KuicK Research
“Global Breast Cancer Vaccine Market and Clinical Pipeline Outlook 2022” report analyzes ongoing clinical and non-clinical trends in the global breast cancer vaccine development market. Currently there is no single breast cancer vaccine commercially available in the market. This report analyzes the ongoing clinical trial of 36 breast cancer vaccines in clinical pipeline and gives comprehensive clinical insight on various parameters associated with the development of the vaccine. Most of the breast cancer vaccines in clinical trials are in preclinical phase followed by Phase-I trials. The advance stage of development of breast cancer vaccine in clinical trials across the globe is in Phase-III.
The conventional cancer treatment methods have proved their importance in last decade. They are widely accepted among patients and physicians (oncologists) because of familiarity and large amount of statistically significant data supporting their efficacy. Despite their benefits, they sometimes fall short in meeting the patient’s personal requirements. For instance, efficacy is a major requirement, but different patients display different response to the same medication, this is attributed to their unique genetic makeup. To meet the individual’s necessities, high efficacy and safety, concept of cancer vaccine was formulated.
This document summarizes presentations and discussions from a working group meeting between the EU and US to propose standards for measuring antimicrobial use in hospitals. It was noted that while surveillance of hospital antimicrobial consumption is becoming more widespread, challenges remain in harmonizing measurement methods between regions. Key challenges include determining the best data sources, units of measurement, and ways to account for differences in patient case mix between hospitals. The group discussed various approaches used in the US and EU and potential standardized indicators, but recognized further work is still needed to develop truly comparable data across multiple healthcare settings and jurisdictions.
‘Orphan drugs’ future growth potential for indian pharmaceutical marketNitin Patel
This document discusses orphan drugs and their potential in the Indian pharmaceutical market. It defines orphan drugs as those developed for rare diseases affecting less than 8% of the population. The US was the first to pass orphan drug legislation in 1983 to incentivize development of these drugs. Similar laws now exist in other countries and provide benefits like 7 years of market exclusivity. While initially developed for small populations, some orphan drugs like Rituxan have become very profitable blockbusters. The global orphan drug market was worth $50 billion in 2011 and is growing faster than other drug markets. Several Indian companies are now developing or manufacturing orphan drugs for rare diseases.
This document provides an overview of Cancer Genetics, Inc. It discusses the company's focus on empowering personalized cancer treatment through molecular diagnostics. The summary highlights Cancer Genetics' proprietary diagnostic products for various cancers, partnerships with biopharma companies, growth in revenue and testing volume in 2013, and pipeline of diagnostic tests in development. It also outlines the company's business model and strategy to commercialize its products to target markets globally.
EuroBioForum 2013 - Day 1 | Emmanuelle BenzimraEuroBioForum
EuroBioForum 2013 2nd Annual Conference
27-28 May 2013 - Hilton Munich City, Munich, Germany
http://www.eurobioforum.eu/2013
=======================================
# MARKET PERSPECTIVES #
Navigating the Challenges of Personalised Medicine Access in Europe
Emmanuelle Benzimra
General Delegate at EPEMED, The European Personalised Medicine Association
=======================================
http://www.eurobioforum.eu
The document discusses innovation in cancer treatment in California. It notes that California companies have hundreds of oncology products in development that could lead to new lifesaving therapies. Continued investment is critical to ongoing research and development of new treatments. The pace of innovation has quickened in recent years, with 29 new cancer treatments approved nationally since 2008 alone.
This document discusses the use of domain specific languages (DSLs) in Ruby. It provides examples of DSLs for tasks like defining database tables, sending emails, and testing code. It also covers concepts like fluent interfaces, method missing, and declarative programming that are commonly used to build internal DSLs in Ruby.
TDC 2014 - JavaScript de qualidade: hoje, amanhã e sempre!Guilherme Carreiro
The document discusses JavaScript quality, past, present and future. It covers topics like variable declarations, prototypes, inheritance, design patterns like Factory and Strategy. It discusses good practices like separating concerns between HTML, CSS and JavaScript code. It also discusses code smells like duplicated code, long methods and lists common JavaScript patterns.
Os tópicos abordados nessa palestra vão desde o conceito abstrato da metaprogramação, até o comportamento da máquina virtual do Ruby, quando executamos determinadas operações. Primeiramente, os conceitos de reflection e metaprogramação são introduzidos no contexto do mundo Ruby. Em seguida, abordarei as APIs que a linguagem oferece para metaprogramação: definição de métodos, conceito de refinements, escopo léxico e exemplos de como código pode gerar código. Após introduzir a API da linguagem, mostrarei como as técnicas abordadas podem ser portadas para escrever códigos que implementam lógicas de negócio, em um nível mais alto de abstração e desacoplamento. A palestra é concluída comentando sobre a importância de entender como o Ruby funciona internamente e o que ocorre quando utilizamos suas APIs. Mais do que invocar métodos, metaprogramação é um recurso poderoso da linguagem que deve ser utilizado com parcimônia e responsabilidade.
RLTG by OYO - The Great Ladakh Road TripDisha Devnani
Dates: 6th - 15th August
Cities: Manali, Jispa, Leh, Nubra Valley, Pangong Lake
Inclusions: Leh - Delhi Air fare, Hotel Stay in Leh, Manali and Jispa, Camps in Nubra Valley & Pangong Lake, Sight seeing in Innova, All Transfers.
Cost per person on twin sharing basis travelling in Innova (basis 5 persons in 01 car) Rs 41,800 PP
Cost per person on triple sharing Rs 40,800 PP
Single Traveller Rs 53,200 PP
The Southern California Consortium for Technology and Innovation in Pediatrics (CTIP) is one of seven FDA-funded pediatric device consortia working to develop medical devices for children. CTIP engages clinicians, engineers, entrepreneurs and others in developing pediatric technologies through events like Med-Innovation Rounds and symposiums. They provide support to 35 distinct pediatric device projects in their first year. To convince industry partners of opportunities in pediatrics, CTIP clearly defines projects' value propositions and market potential through due diligence. Their strategy pairs entrepreneurs with domain experts to advance technologies for children.
The UK is at the forefront of the global pharmaceutical industry. As well as developing new medicines for many diseases, the pharmaceutical industry in the UK provides many other benefits to the British economy, including income, employment, expertise and major investment.
These slides offer a useful, referenced resource for members and visitors to our website who wish to share the story about the value of medicines. They complement existing resources available on the ABPI website and will be updated regularly as the ABPI updates other data and content.
OxfordSM's pharma case studies - providing a call to actionOxfordSM
Brand teams have to be increasingly innovative when finding ways to prompt patients and physicians to intervene at the right time.
Campaigns such as GSK’s Greatest Season Ever for FLONASE®, implemented last year in the United States which made the decision to prepare for the allergy season easier for patients by linking the proactive purchase of the brand to the start of the baseball season.
Providing A Call To Action:
We find that examples from within and outside of healthcare can often prompt this innovation. They act as a way of bringing in new perspectives and allowing teams to explore new avenues and new ideas.
So, in the spirit of hoping this will prompt some new ideas in your brand team, here are our favourite case studies that speak to the need to provide a call to action.
The document discusses the UK's commitment to becoming a global leader in stratified medicine. Some key points:
- Stratified medicine aims to optimize treatment for individual patients rather than broad disease groups, improving outcomes and unlocking business opportunities.
- The UK offers world-leading research expertise, data and infrastructure like large patient datasets and biobanks to help businesses develop and deliver stratified medicine products.
- There are significant market opportunities for stratified medicine and companion diagnostics globally. The UK can help businesses prepare for and access this growing market.
The future of healthcare is an exciting one. With innovations in genomics, healthcare data, advanced therapies and innovative technologies, our industry will continue to progress and provide hope to people so they can live longer, healthier and productive lives.
This document discusses various topics related to drug development including:
- The high costs of drug development which average $1.2 billion per approved drug.
- Success rates for drugs entering clinical trials are low, around 10-11% make it from Phase I to approval.
- Biomarkers and pharmacogenomics can help improve efficiency by identifying patients most likely to respond to treatments. Enrichment strategies focus trials on these responsive patient groups.
- Adaptive trial designs and learning versus confirming approaches also aim to make trials more efficient and lower costs.
Trailblazing scientists who are the backbone of our industry. These are the people that discover the molecules and develop the medicines to tackle the toughest diseases we face in society.
The document discusses the key role of diagnostics in addressing the challenge of antimicrobial resistance (AMR). It makes three key points:
1) AMR is a major threat as many infections are becoming increasingly difficult to treat due to drug-resistant bacteria. Without effective antibiotics, common infections and surgeries could become life-threatening.
2) Diagnostics are critical to appropriate antibiotic use by enabling rapid and precise diagnosis of whether a bacterial or viral infection is present and, if bacterial, determining the best antibiotic to use or avoid. This can help reduce unnecessary antibiotic prescribing.
3) The document outlines a vision for AMR diagnostics in the UK to ensure rapid diagnostic technologies are universally available to guide antibiotic
Download Global breast cancer vaccine market & clinical pipeline outlook ...KuicK Research
“Global Breast Cancer Vaccine Market and Clinical Pipeline Outlook 2022” report analyzes ongoing clinical and non-clinical trends in the global breast cancer vaccine development market. Currently there is no single breast cancer vaccine commercially available in the market. This report analyzes the ongoing clinical trial of 36 breast cancer vaccines in clinical pipeline and gives comprehensive clinical insight on various parameters associated with the development of the vaccine. Most of the breast cancer vaccines in clinical trials are in preclinical phase followed by Phase-I trials. The advance stage of development of breast cancer vaccine in clinical trials across the globe is in Phase-III.
The conventional cancer treatment methods have proved their importance in last decade. They are widely accepted among patients and physicians (oncologists) because of familiarity and large amount of statistically significant data supporting their efficacy. Despite their benefits, they sometimes fall short in meeting the patient’s personal requirements. For instance, efficacy is a major requirement, but different patients display different response to the same medication, this is attributed to their unique genetic makeup. To meet the individual’s necessities, high efficacy and safety, concept of cancer vaccine was formulated.
This document summarizes presentations and discussions from a working group meeting between the EU and US to propose standards for measuring antimicrobial use in hospitals. It was noted that while surveillance of hospital antimicrobial consumption is becoming more widespread, challenges remain in harmonizing measurement methods between regions. Key challenges include determining the best data sources, units of measurement, and ways to account for differences in patient case mix between hospitals. The group discussed various approaches used in the US and EU and potential standardized indicators, but recognized further work is still needed to develop truly comparable data across multiple healthcare settings and jurisdictions.
‘Orphan drugs’ future growth potential for indian pharmaceutical marketNitin Patel
This document discusses orphan drugs and their potential in the Indian pharmaceutical market. It defines orphan drugs as those developed for rare diseases affecting less than 8% of the population. The US was the first to pass orphan drug legislation in 1983 to incentivize development of these drugs. Similar laws now exist in other countries and provide benefits like 7 years of market exclusivity. While initially developed for small populations, some orphan drugs like Rituxan have become very profitable blockbusters. The global orphan drug market was worth $50 billion in 2011 and is growing faster than other drug markets. Several Indian companies are now developing or manufacturing orphan drugs for rare diseases.
This document provides an overview of Cancer Genetics, Inc. It discusses the company's focus on empowering personalized cancer treatment through molecular diagnostics. The summary highlights Cancer Genetics' proprietary diagnostic products for various cancers, partnerships with biopharma companies, growth in revenue and testing volume in 2013, and pipeline of diagnostic tests in development. It also outlines the company's business model and strategy to commercialize its products to target markets globally.
EuroBioForum 2013 - Day 1 | Emmanuelle BenzimraEuroBioForum
EuroBioForum 2013 2nd Annual Conference
27-28 May 2013 - Hilton Munich City, Munich, Germany
http://www.eurobioforum.eu/2013
=======================================
# MARKET PERSPECTIVES #
Navigating the Challenges of Personalised Medicine Access in Europe
Emmanuelle Benzimra
General Delegate at EPEMED, The European Personalised Medicine Association
=======================================
http://www.eurobioforum.eu
The document discusses innovation in cancer treatment in California. It notes that California companies have hundreds of oncology products in development that could lead to new lifesaving therapies. Continued investment is critical to ongoing research and development of new treatments. The pace of innovation has quickened in recent years, with 29 new cancer treatments approved nationally since 2008 alone.
This document discusses the use of domain specific languages (DSLs) in Ruby. It provides examples of DSLs for tasks like defining database tables, sending emails, and testing code. It also covers concepts like fluent interfaces, method missing, and declarative programming that are commonly used to build internal DSLs in Ruby.
TDC 2014 - JavaScript de qualidade: hoje, amanhã e sempre!Guilherme Carreiro
The document discusses JavaScript quality, past, present and future. It covers topics like variable declarations, prototypes, inheritance, design patterns like Factory and Strategy. It discusses good practices like separating concerns between HTML, CSS and JavaScript code. It also discusses code smells like duplicated code, long methods and lists common JavaScript patterns.
Os tópicos abordados nessa palestra vão desde o conceito abstrato da metaprogramação, até o comportamento da máquina virtual do Ruby, quando executamos determinadas operações. Primeiramente, os conceitos de reflection e metaprogramação são introduzidos no contexto do mundo Ruby. Em seguida, abordarei as APIs que a linguagem oferece para metaprogramação: definição de métodos, conceito de refinements, escopo léxico e exemplos de como código pode gerar código. Após introduzir a API da linguagem, mostrarei como as técnicas abordadas podem ser portadas para escrever códigos que implementam lógicas de negócio, em um nível mais alto de abstração e desacoplamento. A palestra é concluída comentando sobre a importância de entender como o Ruby funciona internamente e o que ocorre quando utilizamos suas APIs. Mais do que invocar métodos, metaprogramação é um recurso poderoso da linguagem que deve ser utilizado com parcimônia e responsabilidade.
RLTG by OYO - The Great Ladakh Road TripDisha Devnani
Dates: 6th - 15th August
Cities: Manali, Jispa, Leh, Nubra Valley, Pangong Lake
Inclusions: Leh - Delhi Air fare, Hotel Stay in Leh, Manali and Jispa, Camps in Nubra Valley & Pangong Lake, Sight seeing in Innova, All Transfers.
Cost per person on twin sharing basis travelling in Innova (basis 5 persons in 01 car) Rs 41,800 PP
Cost per person on triple sharing Rs 40,800 PP
Single Traveller Rs 53,200 PP
The Southern California Consortium for Technology and Innovation in Pediatrics (CTIP) is one of seven FDA-funded pediatric device consortia working to develop medical devices for children. CTIP engages clinicians, engineers, entrepreneurs and others in developing pediatric technologies through events like Med-Innovation Rounds and symposiums. They provide support to 35 distinct pediatric device projects in their first year. To convince industry partners of opportunities in pediatrics, CTIP clearly defines projects' value propositions and market potential through due diligence. Their strategy pairs entrepreneurs with domain experts to advance technologies for children.
The itinerary includes visits to landmarks in and around Leh such as Shanti Stupa, Leh Palace, monasteries in Ladakh, excursions to Nubra Valley and Pangong Lake, with overnight stays in camps and hotels. The pricing ranges from Rs. 38,450-46,900 depending on room type and number of travelers. The package includes transportation, accommodations, some meals, and sightseeing as per the detailed daily itinerary provided.
This document outlines a marketing campaign plan for Snapple beverages. The goals are to increase purchases in Snapple's core "Heartland" region, drive purchases nationwide, build brand awareness, and engage consumers through shareable experiences. Research found consumers see Snapple as a sugary treat rather than healthy option. The campaign's big idea is to position Snapple as embracing quirks and not taking oneself too seriously by encouraging consumers to "Live a Little." Elements include print and video ads showing fun moments, in-store promotions, social media campaigns, product placements, and experiential out-of-home promotions. The media plan aims to create interactions and build the Snapple brand across digital, social, television, and
Презентація досвіду роботи класного керівника.
Автор: Миколенко Аліна Володимирівна,
класний керівник 11 класу
Плетеноташлицької ЗШ І-ІІІ ступенів
Маловисківської районної ради
Кіровоградської області
The document provides a summary of a job applicant's qualifications and experience for an operations manager position. It summarizes over 15 years of experience in banking operations, commercial lending, and managing staff across various roles and banks. The applicant has extensive experience managing all aspects of banking operations including loans, accounts, compliance, and staff. She demonstrates strong leadership, customer service, and regulatory compliance skills.
The marketing research report summarizes research conducted to help eHarmony increase its market share among millennials aged 18-24. In-depth interviews were conducted with 10 individuals in this age group, and 5 hypotheses were developed based on their feedback. A survey of 54 millennials then tested these hypotheses. Key findings included that millennials preferred a more affordable service, being able to meet matches sooner, and a separate site for casual dating. The interviews suggested hosting youth-focused events and adjusting advertising to appeal more to millennials.
Subhiksha was a retail chain established in 1997 in Chennai with a theme of offering products at lower prices. It implemented strategies like critical cost control, convenience of one-stop shopping, no frills small stores, everyday low pricing and discounts on multiple products. However, the company rapidly expanded through debt financing to 1600 stores by 2008 without having sufficient working capital. It defaulted on vendor payments, resulting in empty shelves. Stores also faced vandalism as salaries and statutory dues went unpaid. Ultimately, overly aggressive expansion without regard for financial constraints or competitors led to Subhiksha's downfall.
The Nicholas Conor Institute aims to improve treatments for pediatric cancer by developing personalized therapies tailored to each child's unique cancer characteristics. It plans to do this through translating medical discoveries into integrated treatment programs, developing predictive tests to guide therapy, and bringing new child-friendly treatments to clinics. The Institute was founded in memory of Nicholas "Conor" Boddy to address the lack of treatment options and high costs faced by children with cancer.
Cell Therapy Expansion and Challenges in Autoimmune DiseaseHealth Advances
There is increasing confidence that cell therapies will soon play a role in the treatment of autoimmune disorders, but the extent of this impact remains to be seen. Early readouts on autologous CAR-Ts in lupus are encouraging, but manufacturing and cost limitations are likely to restrict access to highly refractory patients. Allogeneic CAR-Ts have the potential to broaden access to earlier lines of treatment due to their inherent cost benefits, however they will need to demonstrate comparable or improved efficacy to established modalities.
In addition to infrastructure and capacity constraints, CAR-Ts face a very different risk-benefit dynamic in autoimmune compared to oncology, highlighting the need for tolerable therapies with low adverse event risk. CAR-NK and Treg-based therapies are also being developed in certain autoimmune disorders and may demonstrate favorable safety profiles. Several novel non-cell therapies such as bispecific antibodies, nanobodies, and RNAi drugs, may also offer future alternative competitive solutions with variable value propositions.
Widespread adoption of cell therapies will not only require strong efficacy and safety data, but also adapted pricing and access strategies. At oncology-based price points, CAR-Ts are unlikely to achieve broad market access in autoimmune disorders, with eligible patient populations that are potentially orders of magnitude greater than the number of currently addressable cancer patients. Developers have made strides towards reducing cell therapy COGS while improving manufacturing efficiency, but payors will inevitably restrict access until more sustainable pricing is achieved.
Despite these headwinds, industry leaders and investors remain confident that cell therapies are poised to address significant unmet need in patients suffering from autoimmune disorders. However, the extent of this impact on the treatment landscape remains to be seen, as the industry rapidly approaches an inflection point.
1) The non-profit health organization Clalit Health Services in Israel will fund the Oncotype DX test for breast cancer patients to determine who would benefit from chemotherapy and who could avoid it.
2) Oncotype DX testing changed the treatment recommendation in 40% of cases, avoiding chemotherapy in 84% deemed at low risk of recurrence by the test.
3) The added cost of Oncotype DX testing was partially offset by savings from avoiding chemotherapy and resulted in a cost-effectiveness ratio of $10,770 per quality-adjusted life year gained.
The document summarizes recent news and events in oncology, including:
1) Daiichi Sankyo's acquisition of Plexxikon for its promising drug PLX4032 that targets the BRAF mutation in melanoma.
2) Positive results from Roche's phase 2 trial of its hedgehog pathway inhibitor vismodegib for advanced basal cell carcinoma.
3) How next-generation sequencing is enabling more personalized cancer treatment by matching drugs to patients based on their tumor's molecular profile.
The maturation of genomic technologies has enabled new
discoveries in disease pathogenesis as well as new approaches to patient care.
In pediatric oncology, patients may now receive individualized genomic analysis to identify molecular aberrations of relevance for diagnosis and/or treatment.
Several recent clinical studies have begun to explore the feasibility and utility of genomics-driven precision medicine.
SMi Group's 5th annual Cancer Vaccines 2016 conferenceDale Butler
This document provides information about a cancer vaccines conference, including two pre-conference workshops, being held on September 20-22, 2016 in London. The conference will focus on the latest advances in cancer vaccine development and preparation for commercialization of immunotherapy breakthroughs. The workshops on September 20th will address cancer vaccines and combination therapies as well as active immune therapy strategies for cancer. The two-day main conference on September 21-22nd will include sessions on biomarkers, dendritic cell vaccines, personalized therapies, RNA vaccines, and targeting strategies. Speakers will represent academic institutions and companies developing cancer immunotherapy technologies and therapies.
This document provides information about a cancer vaccines conference, including two pre-conference workshops on cancer vaccines and combination therapies. The conference will take place September 20-22, 2016 in London and focus on the latest advances in cancer vaccine development and commercialization. It lists the agenda and speakers for the two days of the main conference, which will cover topics like RNA-based vaccines, biomarkers, clinical trials, and combination therapies. Registration discounts are available before certain dates. The workshops on September 20th will address how cancer vaccines can work with and without other treatments to maximize efficacy against cancer.
Vaxeal is a biopharmaceutical company developing therapeutic cancer vaccines and treatments for infectious diseases. It has several vaccine candidates in pre-clinical development targeting cancers and hepatitis C that are expected to begin clinical trials in Europe in 2014-2015. Vaxeal takes promising early-stage research from academic partners and advances it through clinical proof-of-concept, with the goal of improving patient access to new treatments.
World Cancer Day, established by the Union for International Cancer Control (UICC) in 2000, is observed every year on February 4th. Over 10 million people die each year from cancer, more than HIV/AIDS, malaria, and tuberculosis combined. This makes cancer a leading cause of death with a global impact; cancer is not specific to any one geographic region or any one demographic. Cancer can affect anyone of any age, but we are moving towards extraordinary medical breakthroughs in the fight against cancer...
The economics of the oncology industry are changing in ways that threaten future progress:
1) Advances in diagnostics and treatments are reducing the pool of late-stage cancer patients where new therapies are introduced, limiting the volume opportunity for products.
2) Increasing competition from similar mechanisms of action divides the market further and puts pressure on prices to maintain revenues.
3) Payers are responding by restricting access and demanding greater evidence of meaningful improvements in outcomes to justify prices, especially in Europe. Unless product developers can demonstrate significantly better survival or quality of life benefits, the future of investment in oncology is at risk.
Neonatal mortality remains a significant global challenge, accounting for nearly half of all under-5 deaths in 2016. Group B Streptococcus (GBS) is a leading cause of neonatal infections and deaths. While intrapartum antibiotic prophylaxis (IAP) reduces early-onset GBS disease, it does not prevent late-onset disease and universal implementation is difficult. A maternal GBS vaccine could help address this unmet need by providing broader protection against both early and late-onset GBS disease compared to IAP alone. Standardized immunological assays will be important to establish a correlate of protection and validate potential GBS vaccines.
Get the right cancer drug, at right TimeSubin Suresh
Mitra Biotech is a Boston and Bengaluru-based startup that is developing personalized cancer therapies. It focuses on testing drugs on recreated tumor microenvironments in the lab before human trials. This approach has higher success rates and lower toxicity than conventional trials. Mitra Biotech has raised over $27 million to develop these personalized therapies and diagnostics. Major challenges include high costs, ensuring data quality, and coordinating information between different treatment centers.
ZOOM ON: Breast cancer: what’s new?
• NEWS: Big pharma acquire a new collection of PDX
• IMODI around the world: Meet the experts!
• FOCUS on: The Leon Berard Clinical Center
• ADVERT: A new web catalogue for IMODI products
Global cancer vaccine market & clinical trial insightKuicK Research
The document discusses global cancer vaccine market and clinical trials. It provides an overview of the history and development of cancer vaccines. Currently, there are a range of cancer therapeutics available but they have limitations like low effectiveness and specificity. Researchers have developed cancer vaccines to alleviate patient suffering and they work by activating the immune system to attack cancer cells. There are now numerous cancer vaccines in clinical trials for different cancer types. Several have also been approved and are available commercially. The market for cancer vaccines is growing due to factors like fewer side effects, specificity, and innovations in delivery methods. Many companies are actively investing in developing new cancer vaccines.
Global breast cancer vaccine clinical trial insightKuicK Research
The document discusses breast cancer vaccines that are currently in clinical trials. It notes that there are 35 breast cancer vaccines in clinical trials, with most (12) in Phase I. The highest phase reached is Phase III, occupied by two vaccines (NeuVax and OBI-822). The document outlines the global breast cancer vaccine market and pipeline. It explores factors driving development as well as challenges, and predicts that the first breast cancer vaccines may reach the market within the next 3-5 years.
This investor presentation by Cancer Genetics, Inc provides an overview of the company and its proprietary genomic testing programs. CGI has locations in the US, India, and China serving biopharma, clinical, and research clients. It has launched 7 proprietary diagnostic products focused on hematologic and urogenital cancers, and is working to validate additional next generation sequencing panels. CGI's tests provide information to aid in diagnosis, prognosis, and therapeutic decision making for cancers like CLL, cervical cancer, and kidney cancer. The company is collaborating with academic centers to further develop and validate its genomic tests.
Nature medicine top ten advancements in biomedicine in 2020DoriaFang
In the field of biomedicine, which is closely related to human health, many innovative therapies have matured this year and promoted medical progress. Recently, the authoritative academic journal "Nature Medicine" published a series of articles reviewing ten remarkable advancements in 2020.
Global cancer vaccines market & pipeline analysisKuicK Research
“Global Cancer Vaccine Market & Pipeline Analysis” Report Highlights:
Global Cancer Market Overview
Personalized Cancer Vaccines: Progress & Possibilities
Platforms for Cancer Vaccines Delivery
Cancer Vaccines: Mechanism & Innovations
Global Cancer Vaccines Clinical Pipeline by Phase, Indication, Company & Country
Global Cancer Vaccine Clinical Pipeline: 289 Vaccines
Marketed Cancer Vaccines: 12 Vaccines
Regulatory Framework for Cancer Vaccines Development & Marketing
The document discusses a potential collaboration between LifeArc and Biocartis to develop a circulating tumor ESR1 Mutation Test using Biocartis' Idylla platform. The test would detect mutations in the ESR1 gene indicative of resistance to endocrine hormone therapy in metastatic breast cancer. LifeArc has expertise in diagnostic development and Biocartis has an automated molecular testing platform. The test could help guide treatment decisions for breast cancer patients by monitoring their risk of resistance to hormone therapies.
Similar to CHLA Pediatric Health Investment Opportunities_Sept 16 (20)
2. Leading Pediatric Academic Medical Center
Patient diversity
African-American 6%
Asian 6%
Latino 62%
Caucasian 25%
Native American 0.06%
Other 1%
Capacity
357 active beds
5,500 employees
802 medical staff
Annual Statistics
16,000
inpatients admissions
350,000
outpatient visits
>110,000
individual patients
78,000
Emergency
Department visits
2,300 patients transported by
helicopter, learjet and ambulance
U.S. News & World report
ranked No.1
Children’s Hospital in California
3. Interdisciplinary Centers in Areas Critical to Pediatric Health
Center for Cancer and Blood Diseases
One of the nation’s largest pediatric hematology-
oncology programs. Includes the only federally
funded basic and translational research programs
studying sickle cell disease on the west coast. Large,
integrated clinical trials program(167 active studies).
Home to 3 international phase I clinical trial
consortia. 5,200 unique patients.
Center for Fetal and Neonatal Medicine
Treats the most critically ill preemies, newborns and
infants in Southern California.
Center for Endocrinology, Diabetes and
Metabolism
Provides care to nearly 2,000 children with diabetes
and more than 5,000 children with other endocrine
disorders.
Children’s Orthopaedic Center
One of the nation’s most comprehensive
programs seeing 23,500 patients and
performing 1,880 orthopaedic surgeries
annually.
Vision Center
The largest pediatric ophthalmology
program in the U.S.
Cardiac Center
One of the busiest cardiac centers in the
country, and serves as a global patient
reference center.
Neurosciences Center
Provides a comprehensive continuum of
care bringing together services from
neurology, neurosurgery, psychiatry,
neuro-psychology, neuro-radiology, physical
therapy, occupational therapy, dietetics and
social work
Complete range of pediatric specialties in 5
departments and 32 divisions all devoted to children
4. The Saban Research Institute
The Saban Research Institute at Children’s
Hospital Los Angeles comprises basic,
translational and clinical research and is one
of the few freestanding pediatric hospitals in
the country where scientific inquiry is
combined with clinical care devoted
exclusively to children.
Among U.S. children’s hospitals, ranks
eighth for NIH funding
• $32.7 million in NIH funding (FY 2015)
• $74.8 million total research funding (FY
2015)
The Institute includes 198,000 square feet of
wet and dry lab space
• Core facilities fully equipped with state of
the art instrumentation
• Cutting edge biomedical informatics and
imaging technologies.
Home to leading international pediatric
cancer consortia: New Approaches to
Neuroblastoma Therapy (NANT),
Therapeutic Advances in Childhood
Leukemia/Lymphoma (TACL), Pediatric
Blood and Marrow Transplant Consortium
(PBMTC)
• 200 current clinical trials
• 1,625 square foot Clinical Trials Unit
5. The Center for Innovation raises standards in pediatric care through the
development, commercialization and implementation of novel products and
services in partnership with industry, venture capitalists, investors,
entrepreneurs and philanthropists.
The Center for Innovation operates the Consortium for Technology and
Innovation in Pediatrics (CTIP) a pediatric health accelerator serving
innovators and start-ups nationwide and funded in part by the US Food and
Drug Administration.
We evaluate over 80 technologies each year that seek to significantly
improve pediatric standards of care while offering broader market
opportunities.
The Center for Innovation
6. OUR PROCESS
We have a deep understanding of
and access to pediatric markets
Pediatric clinical needs
Requirements for translating
medical technologies to the
bedside
Regulatory strategies needed to
bring pediatric technologies to
market
Reimbursement strategies
Drivers of clinical adoption and
appropriate commercial strategies
Unmet need
validation
Business
model/case
Product
development
Regulatory
approval
Clinical
acceptance
Reimbursement
and
implementation
7. OUR ADVANTAGES
We have access to unique resources to
support development & commercialization
A clinical advisory network in all
areas of pediatric specialty with
KOLs and domain area experts
Clinical and translational research
collaborators
Hospital administrators and leaders
7 FDA-funded sites accelerating
the commercialization of pediatric
medical devices
150+ children’s hospitals
participating in the International
Society for Pediatric Innovation
(iSPI)
8. Avexegen
A preclinical stage company developing Neuregulin-4 (NRG-4), a first-in-class oral therapy
consisting of a naturally occurring peptide that functions to promote gut mucosal healing in
patients suffering from Inflammatory Bowel Disease (IBD), as well as the orphan neonate
condition necrotizing enterocolitis (NEC). Given its novel mechanism of action (MOA),
NRG-4 is well positioned to make significant inroads into the IBD and NEC market segments,
which combined are projected to exceed $10 billion in total market size by 2021.
Nanovalent
A preclinical stage oncology company developing a targeted drug delivery platform that uses
polymerized liposomal nanoparticles that are highly robust, customizable, stable, cheap, and
easy to manufacture. Nanovalent will pursue orphan designation for expedited commercialization
of its first therapeutic candidate in Ewing Sarcoma, a rare pediatric cancer.
CerRx
A clinical stage oncology company seeking to improve the lives of cancer patients by
bringing to the marketplace unique therapeutics that target the ceramide pathway. CerRx is
developing a portfolio of lead compounds including an oral formulation of fenretinide to treat
pediatric cancers, including neuroblastoma.
Therapeutics
9. TheraPEDS
A preclinical stage drug delivery company developing proprietary mucoadhesive oral drug
formulations combined with taste masking chemistry to reformulate existing drugs for
improved compliance and dosing in pediatric (and elderly) patients. The pediatric medicines
market is projected to reach over $100B by 2019.
Hillhurst Biopharmaceuticals
A preclinical stage company developing HBI-002, an oral therapeutic targeting the
cytoprotective Heme Oxygenase metabolic pathway for patients suffering from conditions
associated with inflammation and apoptosis, such as sickle cell disease, transplantation,
and cerebral injury. This therapeutic enables the use of a gasotransmitter for which seven
completed Phase 1 and 2 clinical studies have demonstrated safety and tolerability, albeit with
other delivery modalities that are associated with toxicity and other issues. These indications
combined represent a greater than $6.8B market opportunity.
Therapeutics
10. Antibody-Dependent NK Exosome Therapeutic for Neuroblastoma, Leukemia and Other Cancers*
Antibody-Dependent Exosome Cytotoxicity (ADEC) represents a completely new approach to cancer
immunotherapy with applicability to a wide array of malignancies in children and adults. It’s advantages include
an allogeneic source, expanded from readily available PBMCs, a distinct mechanism of action from cytotoxic
chemotherapies and targeted therapies, synergy with antibody therapy and their small size to penetrate
sanctuary sites. *Available for licensing from CHLA
AM80, An oral therapy to reduce incidence of infections and related mortality in patients with
chemotherapy-induced neutropenia when used in combination with GCSF*
For 20 years, G-CSF (Neupogen®, filgrastim) daily injection has been an effective treatment for increasing
numbers of neutrophils and white blood count. However, despite this therapy, chemotherapy induced
neutropenia continues to be a problem with infections, febrile neutropenia and associated deaths.
Am80 is an oral therapy that increases the bactericidal activity of neutrophils. Increased bactericidal activity
reduces the risk of infection. Patients have a therapy that reduces their risk of infection and associated
mortality while on chemotherapy. *Available for licensing from CHLA
Antibody Blockade of Integrin Alpha6 for the Treatment of Relapse Leukemia and Other Cancers*
Current therapies address tumor shrinkage, but not the tumor microenvironment in metastatic disease (bone
marrow). Relapse of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) remain a
significant problem with a high rate of bone marrow metastasis. Invasive breast cancer remains a significant
problem (200,000 new cases diagnosed per year) in the US with a 20% mortality (relapse) rate. Bone marrow
metastasis remains a problem for all 3.
P5G10 – A first in class blocking antibody to integrin a6 prevents tumor cell adhesion to bone marrow stroma.
Reduced adhesion to bone marrow sensitizes tumor cells to chemotherapy. Patients have a therapy that
reduces their risk of relapse disease and mortality associated with metastatic disease. *Available for licensing
from CHLA
Therapeutics
11. Tourmaline Medical
A device company commercializing the STARFISH Luminal Expansion System (LXS), an innovative, patented device
solution for short bowel syndrome (SBS). SBS is a devastating condition with no current cure that afflicts both
pediatrics and adults. Currently, patient care and associated drugs to facilitate this care can impart costs of over $1M
per year per patient. Unique regulatory pathways will allow Tourmaline to gain rapid and relatively low cost market
access with a unique value proposition for the payor community. Patient care centers are highly concentrated allowing
for an efficient commercial model that will drive rapid revenue growth and break-even. The STARFISH LXS System
has been proven in small and large animals and published in over a dozen peer reviewed publications to date. The
total addressable market for this device is $3B.
Epipace*
An injectable micropacemaker for fetuses, and for children and adults, to control abnormal heart rhythms. Epipace is
developing an alternative approach to the new leadless pacemakers by implanting its micropacemaker on the exterior
of the heart, or epicardially. This safer minimally-invasive approach enables for the first time, the pacing of fetuses, and
offers significant benefits for children and adults. The addressable market for replacing epicardial systems and fetal
pacing is $800M. The total pacemaker market is close to $10B.
*Available for licensing from CHLA
LifeFlow by 410 Medical, A Rapid Infusion System
Pediatric sepsis is responsible for over 75,000 hospitalizations, 10,000 deaths, and $4.8 billion in cost in the US each
year. Multiple studies have shown that implementation of ACCM/PALS guidelines early in the course of sepsis,
particularly antibiotics and aggressive fluid resuscitation, saves lives. Unfortunately, fluid delivery guidelines are rarely
achieved in clinical practice. Numerous studies, and the experience of the Children’s Hospital Associated sepsis
collaborative, have shown that barriers to fluid delivery remain one of the most common reasons for ineffective
resuscitation in pediatric sepsis.The LifeFlow™ rapid infuser is a single-use, hand-operated, disposable device that
allows fast and efficient fluid delivery to critically ill patients with shock and sepsis. Designed to help overcome
common barriers to fluid resuscitation, the device is simple and intuitive, minimizes provider fatigue, and frees
providers to address other patient care issues simultaneously. This solution is particularly useful in pre-hospital and
emergency department settings where speed is important and personnel resources may be limited, and is also well
suited for inpatient, critical care, and operating room environments.
Therapeutic Devices
12. NeoLight
Jaundice is one of the most common and deadliest afflictions facing babies. It occurs in 6 out of 10 newborn
babies globally and affects over 2.4 million infants in the US alone. Jaundice phototherapy devices are a sub-set
of the neonatal equipment market, which is projected to reach $4B by 2019. NeoLight has developed an innovative
light-channeling technique which produces high-intensity light that will measurably reduce treatment time and
eliminate complications associated with current phototherapy equipment on the market. This technology can
potentially reduce treatment time by 40% thus saving the hospital $8000 per procedure.
Hypothermia Devices
Therapeutic hypothermia has been used as a treatment of choice for the management of inflammatory responses
associated with soft tissue injuries and spinal cord injury, to ameliorate the effects of traumatic brain injuries
and strokes, to improve neurologic outcomes after cardiac arrest and neonatal hypoxic-ischemic
encephalopathy. However, existing cooling devices are not designed to address the requirements of stability,
transportability and efficiency for treatment. Hypothermia Devices has developed flexible and ergonomic
cooling/heating pads to be placed in contact with the skin and can be electronically controlled in order to produce
rapid and efficient temperature regulation. The total addressable global market for this technology is $3.9B.
Polyvascular, A Stent Mounted Heart Valve
Congenital heart defects are the most common of all birth defects and remain amongst the leading causes of infant
mortality in the developed world. Multiple types of congenital heart defects require valve replacement surgery.
Pulmonary valves are usually replaced using cadaveric homograft valves, since mechanical valves have increased
risk of thrombosis in the pulmonary position. Homograft pulmonary valves are not commonly available, particularly
in sizes suitable for use in children. Moreover, these valves do not grow with the patient, becoming too small as the
child grows. All these factors result in the need for repeated valve replacement surgeries. Therefore, there is an
urgent need of solutions to limit the frequency of repeated valve replacement surgeries for these children.
Polyvascular is developing an innovative stent mounted heart valve that does not contain any biologic tissue, limiting
any immune sensitization and that can be delivered via cardiac catheterization, eliminating the need for open heart
surgery. This heart valve is also expandable over time and will follow the child's growth, eliminating the need for
multiple surgeries.
Therapeutic Devices
13. TESI, A point of care cell processing device for treating small bowel syndrome*
Necrotizing enterocolitis (NEC) is a gastrointestinal disease that affects premature infants. The current
standard of care requires surgical removal of necrotized tissue, which requires life-long management of
nutritional insufficiencies (transplant and/or arterial line feeding) associated with short bowel syndrome.
TESI is a device-based kit for processing salvaged small intestinal tissue during surgery to create bowel
segments for implantation. A subset of cells is seeded on a biodegradable scaffold, which is implanted into
the patient resulting in autologous tissue-engineered intestines. A proof of concept has been obtained in
non-clinical models and pediatric scalability is being pursued in non-clinical studies. *Available for licensing
from CHLA
Lully Sleep
According to the National Sleep Foundation, 36 Million children experience sleep disorders on a weekly
basis. Night terrors are one of the leading causes of children not sleeping well at young ages. It is
estimated that nearly 25% of children age 2-12 will experience at least one night terror. The Lully Sleep
Guardian is designed to bring this clinically validated, effective technique to the hands of parents. Based
on a child's specific sleep patterns the Lully system uses vibrations, prior to a transition into unhealthy
deep sleep. The Sleep Guardian is based on a tried-and-true technique called scheduled awakenings.
The Sleep Guardian's once-a-night vibrations partially wake your child, bringing him or her out of the night
terror causing sleep patterns. Because children have high sleep pressure, they almost immediately return
to deep sleep. This interrupts the unhealthy sleep cycle and prevents night terrors. Clinical studies have
shown that this method has reduced 90% of night terrors.
Therapeutic Devices
14. Vaximmune
A device company focused on preventing the transmission of the most common infectious agents from
mothers to their babies during labor, Group B Strep (GBS), Ureaplasma (UP) and E. Coli by developing
a proprietary, disposable culture device that allows for the collection, stabilization and concentration of the
relevant sample enabling the use of a rapid diagnostic to read out the test result. The kit contains a sample
collection swab, a disposable culture device and a lateral flow assay. The sample can be collected by the
patient or the physician and the test can be performed by the physician or the laboratory. This product
supports a $700MM market opportunity in the US and a combined opportunity of $2.8BB in the developed
world.
MIND Dx
Neuropsychiatric disorders are devastating illnesses where approximately one-fourth of adults have a
mental illness and nearly half will develop one mental illness during their lifetime. The economic cost of care
for mental illnesses is roughly $100 billion in year 2003 in the US and $193 billion are lost in the in earnings
and wages. The gold standard for diagnoses is clinical interviews and history, resulting in misdiagnoses as
high as 50% at initial presentation and time to proper diagnosis can take years. MindDx has developed
machine-learning based diagnostic algorithms from MR images of the brain to diagnose neuropsychiatric
disorders with high sensitivity and specificity. The market opportunity for ADHD is $800M and $40B for
ADHD, bipolar, schizophrenia, depression and other psychiatric disorders combined.
Deton Corp
Deton is a company committed to eliminating the need for collection of sputum (the phlegm that resides deep
in the airway) as a sample for the diagnosis of lower respiratory tract infections. Sputum sampling has a
50% failure rate resulting from the inability of the patient to produce a sample or sample contamination from
the mouth. For these patients, results cannot be provided, leading to empirical treatment which, in turn, leads
to the proliferation of antibiotic resistant bacteria. Failure rates are highest for pediatric and geriatric patients.
A cough sample is a simple and effective replacement for sputum. There are approximately 10M sputum
samples requested annually in the developed world, supporting a $1B market cap.
SOFTWARE
Diagnostic Devices
15. CrestaBio
There are approximately 1,200,000 people living with or in remission from blood cancer in the US, with an estimated
150,000 new cases diagnosed annually. Minimal residual disease (MRD), defined as the number of cancerous cells
found in the blood or bone marrow during or after treatment, is used to determine if a patient is free of cancer. MRD is
used to monitor the effectiveness of therapy, determine when a patient is in remission and for early detection of
relapse. When MRD is undetected and therefore untreated, it is the primary cause of relapse. Since the limit of
detection of the diagnostic technology defines when a patient is free of cancer, improving the limit of detection directly
results in an improvement in overall survival. Today flow cytometry has a 1 in 10,000 limit of detection and PCR can
detect 1 in 100,000 for some subsets of leukemia. CrestaBio has developed a technology that combines the benefits
of both methods into one test, while improving the sensitivity by three orders of magnitude. The technology allows for
the rapid and cost-effective detection followed by capture and genetic characterization of 1 in 100,000,000 cells.
AZA Technology, A Portable Ammonemia Detector (PAD)
Hyperammonemia is a metabolic condition characterized by elevated levels of ammonia in the blood. Increased entry
of ammonia to the brain is a primary cause of neurologic disorders, such as congenital deficiencies of urea cycle
enzymes, hepatic encephalopathies, Reye syndrome, several other metabolic disorders, and some toxic
encephalopathies. Early diagnosis and improved monitoring are important because hyperammonemia can be treated
by effective medical interventions. In children, 400 infants are born annually in the U.S. with mutations in one of 44
genes that affect several metabolic pathways, and 400,000 infants who are born before 37 weeks of gestation are at
risk for temporarily compromised urea cycles due to immature liver function. The current standard of practice requires
at least 3 ml of blood that must be transported to a central laboratory for immediate processing. The child must endure
intravenous puncture for blood near a central laboratory. Aza Technology, Inc has developed an innovative portable
device that measures ammonia levels in blood obtained by finger or heel stick. This Portable Ammonemia Detector
(PAD) uses a novel technology that adapts an inexpensive commercially available ammonia detector for use with
blood. PAD measures blood ammonia within seconds from 0.01 ml (10 µL) of blood obtained by a simple finger or heel
stick. PAD will allow screening for early diagnosis and thus save lives and protect cognitive function in many children.
In addition, subsequent management of these children will improve when PAD is deployed to the bedside and
outpatient clinics.
Diagnostic Devices
16. Hangar Hustle, A Game-Based Cognitive Assessment Tool
Attention deficits affect millions of people in the US; the percent of children 3-17 years of age with ADHD is 9.5%
and the prevalence of ADHD in the U.S. adult population is approximately 4.4%. Beyond the personal costs to
relationships, education, employment, is the economic cost to the nation in accidents, lost productivity, and
additional healthcare services. Expeditious assessment means earlier access to treatment that will reduce the
personal and economic impact of cognitive impairments. Blue Marble has developed an innovative game-based
assessment tool designed to measure various forms of attention, working memory and executive functions in the
course of video game play. This tool is intended to replace current paper-based or computerized assessment that
are cumbersome, time consuming and non-engaging for the patient. Using this new technology will reduce the
per-patient cost for assessment and will therefore stretch resources farther to serve more people.
Medulloblastoma Diagnostic Assay*
Medulloblastoma is the most common malignant pediatric brain tumor. Approximately 30% of patients remain
incurable and current radiation therapy containing treatment protocols cause significant adverse long-term
neurocognitive effects and endocrine dysfunction. In order to increase survival rates and reduce adverse
sequelae, there is a need of early detection methods and refined patient stratification. This 31-gene diagnostic
assay allows accurate identification of medulloblastoma subgroups and examines the expression of inflammation-
related genes with respect to each subgroup. Treatment for medulloblastoma is currently conducted with a multi-
modality approach (surgery, radiation therapy and chemotherapy) and the modality is heavily dependent on
tumor staging and risk group categorization. The molecular classification of medulloblastoma using this platform
will greatly improve current clinical risk stratification and therefore help with choosing the more efficient treatment
for the patient. *Available for licensing from CHLA
Neuroblastoma Prognostic Assay*
Neuroblastoma, an embryonal tumor of the peripheral sympathetic nervous system, is one of the most common
solid tumors in children, with approximately 45% of patients presenting with metastatic disease at diagnosis. Only
45% of those with high-risk metastatic neuroblastoma are cured with current treatments that include intensive
chemotherapy and immunotherapy. Having a new means to identify individuals at extremely high-risk for disease
progression and to assess response to therapy would be valuable for disease management.This 14-gene
prognostic assay identifies high and ultra-high risk groups and a highly sensitive and specific 5-gene response
assay to quantify circulating and bone marrow tumor cells in patients with neuroblastoma. These tests represent
an integrated disease management platform that may be particularly useful in adapting and evaluating
treatments, and in predicting outcome in children with neuroblastoma. A novel method for recovering high quality
RNA from viable fresh and frozen specimens was established for use in these tests. This platform, with
modifications, may also be useful in adults with cancer. *Available for licensing from CHLA
SOFTWARE
Diagnostic Devices
17. Next Generation Cystic Fibrosis Diagnostic Tool*
Cystic fibrosis (CF) is a life-threatening genetic disease that primarily affects the lungs and digestive system. An
estimated 30,000 children and adults in the United States (70,000 worldwide) have CF. CF is caused by mutations in a
gene that produces a protein, called CFTR. The CFTR protein controls the flow of salt and water in and out of the cells
of organs like the lungs and pancreas. CF is usually diagnosed by conducting a QPIT sweat test, which measures the
amount of chloride in a person’s sweat. However, this test is only valid for patient with severe disease and does not
take into account the spectrum of pathologies associated with variable degrees of CFTR dysfunction. This is
particularly the case with adolescents, adults, and asymptomatic infants identified as being at risk for CF and
presenting with milder symptoms of the disease. This new method is based on beta adrenergically induced sweat
secretion. The rate of b-adrenergically stimulated sweating is used of diagnose CF disease and reflect the probability
of underlying genotypes. B-adrenergic sweat secretion rates are assessed with an evaporimeter to provide an
accurate, reliable and practical real-time assay over a range of CFTR functional activity in vivo. This new test will be a
very valuable asset to diagnose CF disease and a possible biomarker for CF drug efficacy testing. *Available for
licensing from CHLA
Diagnostic Devices
18. Prospiria, an optoaccountic guidance system for endotracheal tube placement
Over 25 million endotracheal tubes (ETs) are placed in patients each year. However, there is not an expedient
solution to identify the 20 to 50 percent of ETs that are initially malpositioned, or that move during intubation.
Incorrectly positioned ETs pose serious risk to patients, especially infants.
Prospiria has developed a patent-pending technology that uses optoacoustics (ultrasound signals generated by
pulsed laser light) to rapidly, precisely, and noninvasively identify the position of endotracheal tubes that have been
placed within the trachea. This technology will increase effectiveness and reduce complications by allowing
clinicians to quickly confirm and monitor both initial and subsequent ET placement.
Inceptio Medical Technologies
An estimated, 2.5 million peripherally inserted central catheters (PICC) line placements are performed in the U.S.
annually. Although a common procedure, PICC placements are particularly difficult in neonates because their
arteries have such small diameters and low pressure for palpitation. Complications that can arise from
misplacement include catheter induced perforation, thrombosis, and catheter induced arrhythmia. Inceptio has
developed a novel technology for precisely locating the distal tip of a cardiac catheter. The system provides
clinicians with real-time visualization during the procedure which greatly increasing the accuracy of placement and
reduces complications. The U.S. PICC market is project to reach over $583M by 2017.
Lifebubble, an Umbilical Catheter Protective Device
Critically ill neonates in the neonatal intensive care unit (NICU) receive umbilical catheters (UCs) because the
umbilical vein and artery in the umbilical cord are an easy and painless way to access the central circulation system.
Many of these babies are premature and have immature immune systems, leaving them susceptible to bloodstream
infections that are attributable to the presence of the umbilical catheter. The rate of UC central-line associated
bloodstream infections (CLABSIs) in neonates is at least five times greater than CLABSIs in the adult population.
Studies estimate that 5-15% of neonates with UCs develop CLABSIs, with rates highest for infants <1,250 grams.
For adult central lines, an occlusive bandage can be applied to the site of catheter insertion that is relatively flat to
the patient’s skin to keep it clean and dry. This standard cannot be applied to the UC that exits the vessel
perpendicular to the skin through a piece of tissue (umbilical stump) that has a unique shape and that must dry out
over the course of a few weeks. LIFEbubble is a innovative device that is tailored to the baby’s unique anatomy. It
replaces the non-sterile tape configuration to reduce the migration of bacteria along the skin, protect the umbilical
stump-catheter interface from accidental touch, and secure the UC to prevent entry of the non-clean catheter into
the bloodstream. LIFEbubble provides a streamlined solution to improve the current standard of care and save the
lives of vulnerable infants.
Positioning Devices
19. NonInvasix
NonInvasix is dedicated to the measurement and monitoring of venous oxygenation, delivering valuable
information regarding the adequacy of oxygen perfusion to the brain. A platform technology with a wide
clinical utility, the technology will first be applied to the monitoring of neonates in the NICU to predict
hypoxic ischemic encephalopathy (HIE). The inability to predict poor perfusion to the brain leads to
long term neurological deficit and can be avoided by monitoring brain venous oxygenation. HIE occurs at
a rate of 1 per 1,000 live births, costing $900,000 per patient per lifetime and is responsible for $3.6B in
annual economic burden.
GI Logic
Millions of adults, children and neonates struggle with digestive disorders, both acute and chronic.
Diagnosing digestive problems requires a hospital visit, use of expensive imaging equipment or expensive
invasive procedures. Current diagnostics monitor “one point in time episodes”, are expensive and not
always immediately available. GI Logic is commercializing “AbStats”, a non-invasive monitoring technology
that accurately measures intestinal activity “continuously” or a “snap shot”, providing an immediate
measurement of digestive health. AbStats can be used in a hospital setting, a clinic setting or a
residential environment. Disposable sensors prevent transfer of bacteria and web services interface
provides for easy storing of information in electronic health records.
BestDose*
Dosing errors make up 37% of all preventable medication errors at a cost of $8B to the healthcare system.
BestDose is developing an enterprise solution for individualized dosing, addressing a >$1B total market.
Its peer-reviewed software platform that combines critical patient data and important drug characteristics
to optimize dosing for any drug. Its solutions can be integrated into the EMR as part of the computerized
physician ordering process (CPOE) and improve prescriber and pharmacist efficiency by accurately
calculating dosages with a button click.
*Available for licensing from CHLA
SOFTWARE
SOFTWARE
Monitoring/CDS Devices
20. Respivice*
Over 800,000 US children are intubated and receive mechanical ventilation each year. Current
strategies to manage mechanical ventilators in children are inefficient, leading to prolonged intubation in
50% of patients and extubation failure in another 10% of patients. Respivce is a stand alone system that
provides quantitative measures to (1) optimize ventilator weaning, (2) easily identify the most common
reasons for extubation failure and (3) manage noninvasive ventilation after extubation. By avoiding these
complications Respivice can reduce healthcare costs by $6.75B in an addressable market of $600M.
*Available for licensing from CHLA
E-Vent, A Digital Ventilator Management Protocol*
Ventilation management is the accumulation of multiple decisions. This type of iterative decision making is
amenable to guidance using a medical treatment protocol. Medical treatment protocols facilitate
consistent, evidence-based decisions for equivalent patient states and digital protocols improve adherence
over paper protocols. E-Vent is an electronic decision support tool translated from an adult ventilator
protocol that improves management of pediatric and adult patients with acute hypoxemic respiratory
failure.
*Available for licensing from CHLA
4DX
A medical technology company established to commercialize four-dimensional lung imaging technology to
provide the spatial and functional information needed to optimize treatment for each patient with conditions
such as cystic fibrosis, asthma, emphysema and COPD. 4DX offers the resolution of CT with doses
comparable to X-Ray and functional insights of a pulmonary function test in a single platform. The software
has been successfully proven and patented and the company is now seeking to raise funds to scale their
SAAS business and commercialize custom designed and dedicated scanning hardware for increased
patients per hour of operation, reduced radiation dose and enhanced fidelity of scans. Enhanced scanning
rates will make each dedicated scanner a valuable additional source of revenue for the analysis service.
SOFTWARE
SOFTWARE
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Monitoring/CDS Devices
21. Click Brain
The current standard of care using pediatric brain MRI for assessment of developmental disorders,
neurological trauma and diseases is time-consuming, qualitative, and requires visual assessment of a
multitude of 2D images. ClickBrain™ is a reimbursable, quantitative translational clinical decision support
software that compares patients’ brain MRI morphometry to healthy normal subjects. ClickBrain™ is
intended to significantly impact patient care by 1) increasing efficiency of interpretation and reporting, 2)
promoting uniformity of interpretation regardless of radiologist experience level, thus reducing risk of
misdiagnosis, and 3) enabling earlier diagnosis and quantitative longitudinal studies for treatment
monitoring.
TimeStamp
Cerebrospinal fluid (CSF) flow can de obstructed due to a variety of medical conditions such as infections,
meningitis, brain tumors, traumatic injuries etc. Because CSF is made continuously, medical conditions
that block its normal flow will result in an over-accumulation of CSF. The resulting pressure of the fluid
against brain tissue causes hydrocephalus. The incidence of pediatric hydrocephalous is about 1/1,000
births and early detection of CSF flow obstruction is a key component for proper treatment selection and
diminution of downstream effects. Current techniques generate images that lose tissue/CSF contrast over
the image capture process thereby complicating the radiologist's ability to identify CSF flow obstruction.
TimeStamp is a new MRI-based image acquisition software that allows MRI images to maintain high image
contrast for easier and faster interpretation by the radiologist.
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SOFTWARE
Monitoring/CDS Devices
22. Qidza
CDC reports that 1 out 6 children have developmental or behavioral problems and 1 in 9 US births are
premature (Boyle et al.,2011), costing healthcare $30+ billion annually. Extensive research proves early
detection can improve outcomes and reduce costly interventions (Glascoe et al, 1997; WHO, 2007). Yet
52% of pediatricians don’t have time or expertise to screen for developmental issues (American Academy
of Pediatrics, 2014). To close the screening gap and offer parents peace of mind, Qidza is a mobile
platform that enables parents work seamlessly with their physicians to track their children’s developmental
milestones and improve their health and well‐being. Quizda has developed BabyNoggin, a preventative
online tool to monitor and track your baby’s brain development from birth to age two. Within minutes
you can screen your baby in the comfort of your home. The app offers an easy and fun way to interact with
your baby and track his or her development. Data gathered from BabyNogin will improve efficiency of well-
child visits to pediatrician and potentially improve clinical outcomes.
Neuromuscular Dynamics
It is well known that both sensory and motor systems contribute to the ability to maintain balance. Sensory
inputs are necessary to detect unstable conditions and motor contributions are vital to initiate timely and
appropriate responses to counteract these perturbations. The current clinical measures quantify the
functional domains of strength and multi-join coordination but they do not quantify the functional domain of
dexterity. This new strength-dexterity test device becomes unstable when compressed. Therefore, the
maximal level of sustained compression is informative of the maximal ability of the neuromuscular system
to stabilize unstable interactions. For the fingers, it is a metric of dexterous manipulation ability. For the
legs, it is a metric of leg dexterity and the ability of the leg to regulate dynamical interactions with the
ground.
SOFTWARE
Monitoring/CDS Devices
23. LifeSpeed
Lifespeed is a health and wellness platform that allows families, medical professionals, and caretakers to
safely store and share data. The tool provides deeper consumer engagement by offering a visually
compelling experience to enable users (starting with chronically ill and mums) to manage their health
profile efficiently. The consumer-centric platform/dashboard allows users to securely organize, archive and
share medical data such as x-rays, data from wearables, lab data, forms and appointments (analogous to
Pinterest + Dropbox). Glimpse allows users to invite doctors, caretakers or loved ones to view their data
securely. For providers, Lifespeed offers easy-to-use tools for digital content distribution, communication
and analysis of patient engagement. The integration of consumer populated data with existing medical
records creates an ecosystem that offers comprehensive collaboration between consumers and providers.
The platform facilitates a marketplace where stakeholders and third party developers bring apps to the
dashboard. The Lifespeed dashboard also provides a medium where healthcare organizations can
advertise to targeted communities through the consumer centered portal for health data management.
NICOlette
NICOlette’s first offering is a forthcoming parent-facing, HIPAA-compliant app called NicoBoard. It shares
NICU patients’ electronic health record (EHR) data in visually intuitive ways designed to allow parents to
quickly grasp the information. It extracts the EHR raw data and synthesizes it into a consumable format for
parents so that they can feel competent and confident in participating in their child’s journey to wellness. In
addition to parsing out easily digestible EHR data visualizations (such as color-coded graphs of a patient’s
oxygen saturation, carbon dioxide levels and apnea episode history, for example), NicoBoard will also
furnish parents with curated research relevant to newborn health. The idea is for parents to use the
research to make more informed healthcare decisions on behalf of their babies.
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Patient Engagement