FasterCures Presentation at LifeFest 2008
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FasterCures' Kristin Schneeman provides an overview of the organization's TRAIN initiative at the Life Raft Group's annual meeting.
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Valley of Death CAN Briefing Slides
The document discusses a forum on infusing life into the "Valley of Death" in the development pipeline. The forum featured speakers from Senator Specter's office, Parkinson's Action Network, and FasterCures. It describes the development pipeline, noting inconsistent funding in the prototype design/discovery and preclinical development stages, known as the "Valley of Death". It also lists some opportunities to provide funding in these stages, such as private foundations, NIH programs, and the Cures Acceleration Network.
BIO 2010 Partnering with Patients
The document discusses various ways that nonprofit organizations partner with patients and the medical research community to advance research and drug development. It provides examples of how organizations fund research, develop tools and resources, facilitate clinical trials, advocate for policy changes, and build communities to accelerate the development of new treatments. Specific organizations highlighted include the Alzheimer's Drug Discovery Foundation, Juvenile Diabetes Research Foundation, Multiple Myeloma Research Foundation, ALS Therapy Development Institute, and Genetic Alliance.
BIO 2010 Partnering with Patients
The document discusses various ways that nonprofit organizations partner with patients and the medical research community to advance research and drug development. It provides examples of how organizations fund research, develop tools and resources, facilitate clinical trials, advocate for policy changes, and build communities to accelerate the development of new treatments. Specific organizations highlighted include the Alzheimer's Drug Discovery Foundation, Juvenile Diabetes Research Foundation, Multiple Myeloma Research Foundation, ALS Therapy Development Institute, and Genetic Alliance.
Pharma Assets Portal
The document summarizes a webinar presentation about accessing shelved pharmaceutical compounds through the CTSA Pharmaceutical Assets Portal. It provides background on the goals of the portal, which is to improve information exchange between pharmaceutical companies and academic researchers regarding drugs discontinued in clinical trials that could be candidates for drug repositioning. It describes some initial achievements and collaborations of the portal project. It also discusses the Clinical and Translational Science Awards program and how the portal was inspired by examples of drug repositioning.
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A panel at the inaugural Partnering for Cure conference spotlights the translational funding and management gap in biomedical research and development.
Moderator: Sharon Begley, Science Editor, Newsweek
Panelists:
Robi Blumenstein, President, CHDI Management/CHDI Foundation
Randall Carpenter, Co-Founder, President and Chief Executive Officer, Seaside Therapeutics
Dennis Purcell, Managing Partner, Aisling Capital
Amy Comstock Rick, Chief Executive Officer, Parkinson's Action Network
Christian Loucq, PATH Malaria Vaccine InitiativeProgeria Research Foundation at Partnering for Cures
The document summarizes research on progeria, a rare premature aging disease. It discusses how the Progeria Research Foundation was established in 1999 to address the lack of research, information, and treatment options. Through the foundation's efforts like establishing a patient registry and funding research, the understanding of progeria increased and a gene was identified. This led to the first-ever progeria treatment trial in 2007 testing drugs that target the abnormal protein produced in progeria cells. The research on progeria also provides insights into normal aging and age-related diseases.
PATH Malaria Vaccine Initiative
MVI's mission is to accelerate malaria vaccine development and ensure availability in developing countries. Its goal is an 80% effective vaccine lasting 4+ years by 2025. Malaria infects 250 million people annually and kills 900,000, mostly children in Africa. Vaccine development faces challenges due to the parasite's complexity and limited commercial markets. MVI partners with organizations to systematically develop promising approaches through clinical trials. Its current strategy focuses on pre-erythrocytic, transmission-blocking, and P. vivax vaccines. With a phase 3 trial underway, MVI's public-private model brings malaria vaccines closer to reality.
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Leukemia Lymphoma Society: An Invitation to Innovation
Leukemia Lymphoma Society: An Invitation to Innovation - as presented at the Partnering for Cures meeting, December 2, 2009, New York
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Susan G. Komen for the Cure presents its research and development strategies at the 2009 Partnering for Cures conference in New York.
JDRF at the Partnering for Cures Conference
The Juvenile Diabetes Research Foundation (JDRF) aims to cure, treat, and prevent diabetes through funding research. It has funded over $1 billion in research over 39 years to find disease-modifying drugs and devices. JDRF partners with industry, academia, patients, and other organizations to fund early-stage, translational, and clinical research. Its highest priorities include beta cell therapies, immune therapies, artificial pancreas devices, and therapies to prevent complications.
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the International AIDS Vaccine Initiative's presents its R&D strategies and approaches at the Partnering for Cures 2009 conference in New York.
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Jana Armstrong, Executive Director, DNDi North America, presents DNDi's research strategy at the 2009 Partnering for Cures meeting in New York.
TB Alliance: Improving Treatments Against TB
The TB Alliance is a nonprofit organization developing new treatments for tuberculosis (TB). It has built the largest portfolio of TB drug candidates in history, with three in late-stage clinical trials. Current TB treatments are long and ineffective against drug-resistant strains. The TB Alliance aims to develop shorter, simpler, and more effective TB therapies to address this urgent global health need. However, increased funding is needed to advance its pipeline of potential new TB drugs and treatments.
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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.
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Learning objectives:
1. Describe the organisation of respiratory center
2. Describe the nervous control of inspiration and respiratory rhythm
3. Describe the functions of the dorsal and respiratory groups of neurons
4. Describe the influences of the Pneumotaxic and Apneustic centers
5. Explain the role of Hering-Breur inflation reflex in regulation of inspiration
6. Explain the role of central chemoreceptors in regulation of respiration
7. Explain the role of peripheral chemoreceptors in regulation of respiration
8. Explain the regulation of respiration during exercise
9. Integrate the respiratory regulatory mechanisms
10. Describe the Cheyne-Stokes breathing
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FasterCures Presentation at LifeFest 2008
- 1. Kristin Schneeman Program Director September 12, 2008 “ What You Seed Is What You Get” Making Faster Cures the Central Organizing Principle of the Research Enterprise
- 5. “Entrepreneurs for Cures” What would your business model be if your bottom line were curing a disease?
- 6. The High Costs of Business as Usual We’re spending a lot on treating disease: $2.1 trillion on healthcare in 2006 – more than $7000 per person We’re not spending much on curing disease: And productivity is falling: Sources: CMS, Research!America
- 9. Separating what we need to do in science and what we need to do in the culture of science to accelerate curing diseases
- 10. TRAIN: You Are Not Alone The Redstone Acceleration and Innovation Network Collaborative, mission-driven, results-oriented, and strategic in their use of capital, these groups are motivated solely by moving promising therapies from the laboratory bench to the patient’s bedside as rapidly as possible – even those that do not directly fund therapy development.
- 16. Instead of following the money, lead the money.