Aranca's Report on Gene Therapy - a promising tool for Cancer, Parkinson's, HIV, severe combined immuno-deficiencies, hemophilia etc. In this report, you will discover the challenges associated with Gene Therapy as well as its expected future.
Research Ethics Forum: Ethical Challenges in Trials of Human Genome Editing a...SC CTSI at USC and CHLA
In her 60-minute presentation, Professor Charo addressed Ethical Challenges in Trials of Human Genome Editing and Gene Therapy, as gene therapy and genome editing clinical trials involve ethical challenges not always found in other areas of research.
Crowdsourcing applied to knowledge management in translational research: the ...SC CTSI at USC and CHLA
Date: November 8th, 2018
Speaker: Andrew Su, PhD, Professor, Department of Integrative, Structural and Computational Biology, The Scripps Research Institute
Overview: Crowdsourcing involves the engagement of large communities of individuals to collaboratively accomplish tasks at massive scale. These tasks could be online or offline, paid or for free. But how can crowdsourcing science help your research? This webinar will describe two crowdsourcing projects for translational research, both of which aim to better organize biomedical information so that it can be more easily accessed, integrated, and queried:
First, the goal of the Gene Wiki project is to create a community-maintained knowledge base of all relationships between biological entities, including genes, diseases, drugs, pathways, and variants. This project draws on the collective efforts of informatics researchers from a wide range of disciplines, including bioinformatics, cheminformatics, and medical informatics.
Second, the Mark2Cure project partners with the citizen scientist community to extract structured content from biomedical abstracts with an emphasis on rare disease. Although citizen scientists do not have any specialized expertise, after receiving proper training, Mark2Cure has shown that in aggregate they perform bio-curation at an accuracy comparable to professional scientists.
INTRODUCTION
DNA VACCINES
GENE THERAPY
TIME LINE OF DEVELOPING GENE THERAPY
GENE THERAPY STRATEGIES
TECHNOLOGY OF CLASSICAL GENE THERAPY
PRINCIPLES OF GENE TRANSFER
VECTORS
VIRAL VECTORS
NON-VIRAL VECTORS
APPLICATIONS OF GENE THERAPY
ETHICAL IMPLICATIONS
THE FUTURE
CONCLUSION
REFERENCES
Gene therapy involves the insertion of a functioning gene into cells to correct a cellular dysfunction
KEY WORDS : GENETICS, MUTATION , GENETIC ENGINEERING.
London & Partners MedCity Cell & Gene Therapy Report londonandpartners
The golden triangle & Japan, natural collaborators in cell and gene therapy. Discover Cambridge, London and Oxford’s thriving life sciences ecosystem and cell and gene therapy expertise. The golden triangle offers clinical trials capabilities, a diverse population and single healthcare system, all supported by a committed government. Collaborate with us and help develop the next generation of cell and gene therapy treatments.
Research Ethics Forum: Ethical Challenges in Trials of Human Genome Editing a...SC CTSI at USC and CHLA
In her 60-minute presentation, Professor Charo addressed Ethical Challenges in Trials of Human Genome Editing and Gene Therapy, as gene therapy and genome editing clinical trials involve ethical challenges not always found in other areas of research.
Crowdsourcing applied to knowledge management in translational research: the ...SC CTSI at USC and CHLA
Date: November 8th, 2018
Speaker: Andrew Su, PhD, Professor, Department of Integrative, Structural and Computational Biology, The Scripps Research Institute
Overview: Crowdsourcing involves the engagement of large communities of individuals to collaboratively accomplish tasks at massive scale. These tasks could be online or offline, paid or for free. But how can crowdsourcing science help your research? This webinar will describe two crowdsourcing projects for translational research, both of which aim to better organize biomedical information so that it can be more easily accessed, integrated, and queried:
First, the goal of the Gene Wiki project is to create a community-maintained knowledge base of all relationships between biological entities, including genes, diseases, drugs, pathways, and variants. This project draws on the collective efforts of informatics researchers from a wide range of disciplines, including bioinformatics, cheminformatics, and medical informatics.
Second, the Mark2Cure project partners with the citizen scientist community to extract structured content from biomedical abstracts with an emphasis on rare disease. Although citizen scientists do not have any specialized expertise, after receiving proper training, Mark2Cure has shown that in aggregate they perform bio-curation at an accuracy comparable to professional scientists.
INTRODUCTION
DNA VACCINES
GENE THERAPY
TIME LINE OF DEVELOPING GENE THERAPY
GENE THERAPY STRATEGIES
TECHNOLOGY OF CLASSICAL GENE THERAPY
PRINCIPLES OF GENE TRANSFER
VECTORS
VIRAL VECTORS
NON-VIRAL VECTORS
APPLICATIONS OF GENE THERAPY
ETHICAL IMPLICATIONS
THE FUTURE
CONCLUSION
REFERENCES
Gene therapy involves the insertion of a functioning gene into cells to correct a cellular dysfunction
KEY WORDS : GENETICS, MUTATION , GENETIC ENGINEERING.
London & Partners MedCity Cell & Gene Therapy Report londonandpartners
The golden triangle & Japan, natural collaborators in cell and gene therapy. Discover Cambridge, London and Oxford’s thriving life sciences ecosystem and cell and gene therapy expertise. The golden triangle offers clinical trials capabilities, a diverse population and single healthcare system, all supported by a committed government. Collaborate with us and help develop the next generation of cell and gene therapy treatments.
Dr. Leroy Hood lectured to a group of Ohio State University College of Medicine students and faculty on May 13, 2010 in advance of an announcement of a partnership between the Ohio State University Medical Center and the Institute for Systems Biology. The partnership will be known as
Death prompts a review of gene therapy vectorLindsay Meyer
Case study and analysis of Targeted Genetics' adeno-associated virus, tgAAC94. Includes overview of clinical trial design, FDA action, NIH investigation, and outcomes surrounding the death of a patient enrolled in the investigational trial.
These slides discusses on cellular and gene therapy: the use of cells and genes to treat disease. These therapies can be effective on a wide range of previously untreated diseases, such as hematological, ocular, neurodegenerative diseases, and several types of cancers.
Gene therapy is a new tool used in combating different diseases. The majority of gene therapy clinical trials are focused on cancer and so it was no coincidence that the first commercial treatment in 2003 was for neoplasia. Currently there are a wide variety of gene therapy proposals involving a large number of anti tumour molecular mechanisms that will conceivably pave the way for highly effective a treatment options. Despite the significant advances that how been made in gene therapy in the fight against cancer, its efficacy,safety and commercial availability are still limited. Ms. Chetana D. Patil | Ms. Siddhi Chavan | Mr. Ritesh Kadam "Gene Therapy for Cancer Treatment" Published in International Journal of Trend in Scientific Research and Development (ijtsrd), ISSN: 2456-6470, Volume-3 | Issue-5 , August 2019, URL: https://www.ijtsrd.com/papers/ijtsrd26537.pdfPaper URL: https://www.ijtsrd.com/pharmacy/biotechnology-/26537/gene-therapy-for-cancer-treatment/ms-chetana-d-patil
A Rare International Dialogue (Sunday, May 12, 2019)
Theme One: Diagnosis and Beyond
WORKSHOP G: Cell and Gene Therapy from Laboratory to Market - Mark Lundie, Pfizer Canada
HD Insights recognized three papers from 2016 with awards.
Flavia Niccolini of King's College London won for "Altered PDE10A Expression Detectable Early Before Symptomatic Onset in Huntington's Disease."
Jong-Min Lee of the GeM-HD Consortium, won for "Genetic Modifiers of HD"
Dr. Leroy Hood lectured to a group of Ohio State University College of Medicine students and faculty on May 13, 2010 in advance of an announcement of a partnership between the Ohio State University Medical Center and the Institute for Systems Biology. The partnership will be known as
Death prompts a review of gene therapy vectorLindsay Meyer
Case study and analysis of Targeted Genetics' adeno-associated virus, tgAAC94. Includes overview of clinical trial design, FDA action, NIH investigation, and outcomes surrounding the death of a patient enrolled in the investigational trial.
These slides discusses on cellular and gene therapy: the use of cells and genes to treat disease. These therapies can be effective on a wide range of previously untreated diseases, such as hematological, ocular, neurodegenerative diseases, and several types of cancers.
Gene therapy is a new tool used in combating different diseases. The majority of gene therapy clinical trials are focused on cancer and so it was no coincidence that the first commercial treatment in 2003 was for neoplasia. Currently there are a wide variety of gene therapy proposals involving a large number of anti tumour molecular mechanisms that will conceivably pave the way for highly effective a treatment options. Despite the significant advances that how been made in gene therapy in the fight against cancer, its efficacy,safety and commercial availability are still limited. Ms. Chetana D. Patil | Ms. Siddhi Chavan | Mr. Ritesh Kadam "Gene Therapy for Cancer Treatment" Published in International Journal of Trend in Scientific Research and Development (ijtsrd), ISSN: 2456-6470, Volume-3 | Issue-5 , August 2019, URL: https://www.ijtsrd.com/papers/ijtsrd26537.pdfPaper URL: https://www.ijtsrd.com/pharmacy/biotechnology-/26537/gene-therapy-for-cancer-treatment/ms-chetana-d-patil
A Rare International Dialogue (Sunday, May 12, 2019)
Theme One: Diagnosis and Beyond
WORKSHOP G: Cell and Gene Therapy from Laboratory to Market - Mark Lundie, Pfizer Canada
HD Insights recognized three papers from 2016 with awards.
Flavia Niccolini of King's College London won for "Altered PDE10A Expression Detectable Early Before Symptomatic Onset in Huntington's Disease."
Jong-Min Lee of the GeM-HD Consortium, won for "Genetic Modifiers of HD"
Nucleic Acid Based Therapeutic Delivery System.pptxRAHUL PAL
Therapeutic nucleic acids (TNAs) are nucleic acids themselves or closely related compounds used to treat disease. Although various types of TNAs exist, they share a common mechanism of action that is mediated by sequence‐specific recognition of endogenous nucleic acids through Watson–Crick base pairing 7.
What are the advantages of nucleic acid based therapeutics?
The major advantage of nucleic acid-based therapeutics lies in the fact that they can be used to accurately target a tumor or tissue, then have a specific therapeutic protein, biologic, or immune engager expressed only at the site of interest.
Nucleic Acid Based Therapeutic Delivery System.pptxPrachi Pandey
The delivery of nucleic acid molecules into cells to alter physiological functions at the genetic level is a powerful approach to treat a wide range of inherited and acquired disorder.
This technique has been a common research tool in laboratory for decades to study gene functions.
The therapeutic potential of this approach was not fully realized due to lack of reliable and practical methods to transfer and express recombinant DNA in mammalian cells.
Gene Therapy: Central concept of gene therapy, basic molecular mechanism of gene transfer, prerequisite of human gene therapy, biological basis of gene therapy strategies, vehicles for gene transfer, Antisence oligonucleotides and RNAi, clinical gene therapy studies, gene therapy for hereditary disease, gene therapy for cancer, gene therapy for HIV.
Gene Therapy: Central concept of gene therapy, basic molecular mechanism of gene transfer, prerequisite of human gene therapy, biological basis of gene therapy strategies, vehicles for gene transfer, Antisence oligonucleotides and RNAi, clinical gene therapy studies, gene therapy for hereditary disease, gene therapy for cancer, gene therapy for HIV.
Gene therapy is the process of inserting therapeutic genes into cells to prevent or cure wide range of diseases. The newly introduced genes will encode proteins and correct the deficiencies that occur in genetic diseases. Gene therapy primarily involves genetic manipulations in animals or humans to correct a disease, and keep organism in good health. It is a technique for correcting defective genes responsible for disease and development.
The French Animal Healthcare Market is on the upswing boosted by growing disposable income, a rising number of pets, growing animal healthcare awareness, etc.Get detailed Business Analysis Infographic from Aranca.
Owing to IoT and increased customer preference for wearable gadgets, the Global Wearable Devices Market is expected to grow at a 21% CAGR during 2015-18. Get detailed market infographics on wearable devices market here.
The global market potential for Commercial Drones could be worth 2.1 billion USD by 2022. Know details on the future of Commercial Drone from Aranca's Market Research Experts.
The GCC Facilities Management Market - ArancaAranca
The rising demand of multi-billion construction projects is expected to fuel the growth of FM Market in the GCC region. Read on to know details from Aranca's Business Research Experts.
Brazil’s continuous and sustainable agricultural growth represents tremendous growth opportunity for the Brazilian Pesticide Market . Read on to know more from Aranca's Market Research Experts.
With the increased adoption of Cryogenic Equipment, a growth of 8% CAGR in the Global Cryogenic Equipment Market is anticipated. Read on to know details from Aranca's Business Research Experts.
With increase in urban population & investment in infrastructure sector, China's elevator & escalator market is expected to grow at a CAGR of 14%. Read on to know detailed forecast from Aranca's Business Research Experts.
Growing regulations regarding emissions, fuel efficiency and longing for light weight vehicle has increased the demand of Aluminum Automotive Sheets globally. Read on to know details from Aranca's Business Research Experts.
Indian Agrochemical Industry is expected to grow by a rate of 12 to 14% CAGR owing to the increased demand of food grains & supportive Government initiatives. Read on to know more from Aranca's Business Research Experts.
Weak currency, strong anti-corruption reforms, and stiff luxury taxes are forcing global luxury brands to rethink their expansion plans in one of the world's fastest growing consumer economies. Read more from Aranca's Investment Research Experts here.
Saudi Aramco IPO - A Reality of Mythical ProportionsAranca
Aranca's special report on Saudi Aramco IPO highlights its impact on industry, global investment banks and discusses the political and economic ramifications a large IPO may have. Download the report here!
Fatca high cost initiative to curb tax evasionAranca
Enacted by the United States Congress in March of 2010, the Foreign Account Tax Compliance Act (FATCA) is a federal law meant to deter tax evasion. Read details from Aranca's Business Research Experts here.
Winning shelf space private labels or fmcg brandsAranca
Consumer preference for Fast Moving Consumer Goods based on quality and affordability, in the high inflationary markets led to the emergence of private labels across geographies such as Europe, China, India, and the Americas.
Virtual currency has been a debated concept within the technology community in the past few years, as transactions through this medium do not require any third party’s involvement. Know more details from Aranca's Business Research Experts here.
Here we discuss the sectoral winners and losers for Patient Protection and Affordable Healthcare Act I (Obamacare) along with the key factors driving these trends due to Obamacare. Check out the Special Report on Obamacare from Aranca's Business Research Experts!
Therapeutic Vaccines for Alzheimer’s — Are We Close Enough?Aranca
Could a vaccine for Alzheimer's be a reality any sooner? Find more on Alzheimer Therapy Market Challenges from Aranca's Technology Intelligence & IP Research Experts.
Indian Telecom Tower Industry - Aranca ResearchAranca
India's telecom tower industry is expected to grow significantly owing to the Government's plans of providing high speed internet throughout the country. Read on to know more Aranca's Business Research experts.
Virtual Reality (VR) Industry has been through Ups & downs for around 3 decades but due to adaption of connected devices, it is expected to grow. Read on to know more details here.
Insurance Industry is Going to Soar in the GCCAranca
A positive economic outlook and several regulatory reforms are likely to drive the insurance industry in the GCC region. Know more from Aranca's Research Experts here.
ICH Guidelines for Pharmacovigilance.pdfNEHA GUPTA
The "ICH Guidelines for Pharmacovigilance" PDF provides a comprehensive overview of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guidelines related to pharmacovigilance. These guidelines aim to ensure that drugs are safe and effective for patients by monitoring and assessing adverse effects, ensuring proper reporting systems, and improving risk management practices. The document is essential for professionals in the pharmaceutical industry, regulatory authorities, and healthcare providers, offering detailed procedures and standards for pharmacovigilance activities to enhance drug safety and protect public health.
Deep Leg Vein Thrombosis (DVT): Meaning, Causes, Symptoms, Treatment, and Mor...The Lifesciences Magazine
Deep Leg Vein Thrombosis occurs when a blood clot forms in one or more of the deep veins in the legs. These clots can impede blood flow, leading to severe complications.
The dimensions of healthcare quality refer to various attributes or aspects that define the standard of healthcare services. These dimensions are used to evaluate, measure, and improve the quality of care provided to patients. A comprehensive understanding of these dimensions ensures that healthcare systems can address various aspects of patient care effectively and holistically. Dimensions of Healthcare Quality and Performance of care include the following; Appropriateness, Availability, Competence, Continuity, Effectiveness, Efficiency, Efficacy, Prevention, Respect and Care, Safety as well as Timeliness.
Telehealth Psychology Building Trust with Clients.pptxThe Harvest Clinic
Telehealth psychology is a digital approach that offers psychological services and mental health care to clients remotely, using technologies like video conferencing, phone calls, text messaging, and mobile apps for communication.
Antibiotic Stewardship by Anushri Srivastava.pptxAnushriSrivastav
Stewardship is the act of taking good care of something.
Antimicrobial stewardship is a coordinated program that promotes the appropriate use of antimicrobials (including antibiotics), improves patient outcomes, reduces microbial resistance, and decreases the spread of infections caused by multidrug-resistant organisms.
WHO launched the Global Antimicrobial Resistance and Use Surveillance System (GLASS) in 2015 to fill knowledge gaps and inform strategies at all levels.
ACCORDING TO apic.org,
Antimicrobial stewardship is a coordinated program that promotes the appropriate use of antimicrobials (including antibiotics), improves patient outcomes, reduces microbial resistance, and decreases the spread of infections caused by multidrug-resistant organisms.
ACCORDING TO pewtrusts.org,
Antibiotic stewardship refers to efforts in doctors’ offices, hospitals, long term care facilities, and other health care settings to ensure that antibiotics are used only when necessary and appropriate
According to WHO,
Antimicrobial stewardship is a systematic approach to educate and support health care professionals to follow evidence-based guidelines for prescribing and administering antimicrobials
In 1996, John McGowan and Dale Gerding first applied the term antimicrobial stewardship, where they suggested a causal association between antimicrobial agent use and resistance. They also focused on the urgency of large-scale controlled trials of antimicrobial-use regulation employing sophisticated epidemiologic methods, molecular typing, and precise resistance mechanism analysis.
Antimicrobial Stewardship(AMS) refers to the optimal selection, dosing, and duration of antimicrobial treatment resulting in the best clinical outcome with minimal side effects to the patients and minimal impact on subsequent resistance.
According to the 2019 report, in the US, more than 2.8 million antibiotic-resistant infections occur each year, and more than 35000 people die. In addition to this, it also mentioned that 223,900 cases of Clostridoides difficile occurred in 2017, of which 12800 people died. The report did not include viruses or parasites
VISION
Being proactive
Supporting optimal animal and human health
Exploring ways to reduce overall use of antimicrobials
Using the drugs that prevent and treat disease by killing microscopic organisms in a responsible way
GOAL
to prevent the generation and spread of antimicrobial resistance (AMR). Doing so will preserve the effectiveness of these drugs in animals and humans for years to come.
being to preserve human and animal health and the effectiveness of antimicrobial medications.
to implement a multidisciplinary approach in assembling a stewardship team to include an infectious disease physician, a clinical pharmacist with infectious diseases training, infection preventionist, and a close collaboration with the staff in the clinical microbiology laboratory
to prevent antimicrobial overuse, misuse and abuse.
to minimize the developme
CRISPR-Cas9, a revolutionary gene-editing tool, holds immense potential to reshape medicine, agriculture, and our understanding of life. But like any powerful tool, it comes with ethical considerations.
Unveiling CRISPR: This naturally occurring bacterial defense system (crRNA & Cas9 protein) fights viruses. Scientists repurposed it for precise gene editing (correction, deletion, insertion) by targeting specific DNA sequences.
The Promise: CRISPR offers exciting possibilities:
Gene Therapy: Correcting genetic diseases like cystic fibrosis.
Agriculture: Engineering crops resistant to pests and harsh environments.
Research: Studying gene function to unlock new knowledge.
The Peril: Ethical concerns demand attention:
Off-target Effects: Unintended DNA edits can have unforeseen consequences.
Eugenics: Misusing CRISPR for designer babies raises social and ethical questions.
Equity: High costs could limit access to this potentially life-saving technology.
The Path Forward: Responsible development is crucial:
International Collaboration: Clear guidelines are needed for research and human trials.
Public Education: Open discussions ensure informed decisions about CRISPR.
Prioritize Safety and Ethics: Safety and ethical principles must be paramount.
CRISPR offers a powerful tool for a better future, but responsible development and addressing ethical concerns are essential. By prioritizing safety, fostering open dialogue, and ensuring equitable access, we can harness CRISPR's power for the benefit of all. (2998 characters)
Medical Technology Tackles New Health Care Demand - Research Report - March 2...pchutichetpong
M Capital Group (“MCG”) predicts that with, against, despite, and even without the global pandemic, the medical technology (MedTech) industry shows signs of continuous healthy growth, driven by smaller, faster, and cheaper devices, growing demand for home-based applications, technological innovation, strategic acquisitions, investments, and SPAC listings. MCG predicts that this should reflects itself in annual growth of over 6%, well beyond 2028.
According to Chris Mouchabhani, Managing Partner at M Capital Group, “Despite all economic scenarios that one may consider, beyond overall economic shocks, medical technology should remain one of the most promising and robust sectors over the short to medium term and well beyond 2028.”
There is a movement towards home-based care for the elderly, next generation scanning and MRI devices, wearable technology, artificial intelligence incorporation, and online connectivity. Experts also see a focus on predictive, preventive, personalized, participatory, and precision medicine, with rising levels of integration of home care and technological innovation.
The average cost of treatment has been rising across the board, creating additional financial burdens to governments, healthcare providers and insurance companies. According to MCG, cost-per-inpatient-stay in the United States alone rose on average annually by over 13% between 2014 to 2021, leading MedTech to focus research efforts on optimized medical equipment at lower price points, whilst emphasizing portability and ease of use. Namely, 46% of the 1,008 medical technology companies in the 2021 MedTech Innovator (“MTI”) database are focusing on prevention, wellness, detection, or diagnosis, signaling a clear push for preventive care to also tackle costs.
In addition, there has also been a lasting impact on consumer and medical demand for home care, supported by the pandemic. Lockdowns, closure of care facilities, and healthcare systems subjected to capacity pressure, accelerated demand away from traditional inpatient care. Now, outpatient care solutions are driving industry production, with nearly 70% of recent diagnostics start-up companies producing products in areas such as ambulatory clinics, at-home care, and self-administered diagnostics.
How many patients does case series should have In comparison to case reports.pdfpubrica101
Pubrica’s team of researchers and writers create scientific and medical research articles, which may be important resources for authors and practitioners. Pubrica medical writers assist you in creating and revising the introduction by alerting the reader to gaps in the chosen study subject. Our professionals understand the order in which the hypothesis topic is followed by the broad subject, the issue, and the backdrop.
https://pubrica.com/academy/case-study-or-series/how-many-patients-does-case-series-should-have-in-comparison-to-case-reports/
2. 2
Challenges Associated With Developing Genetic Treatments 2
Administering Gene Therapy 3
Viral Vectors 4
Non-viral Vectors 5
Possible Strategies to Improve Current Viral/Non-viral Vector Systems 5
Patent Filing Trend in the Domain of “Delivery Vehicles Used in Gene Therapy” 5
Gene Therapy Considered a Path-changing Treatment for the Coming Era 6
Fat Metabolism Disorder 6
Adenosine Deaminase (ADA) Deficiency 6
Severe Combined Immune Deficiency (SCID) 6
Hemophilia 6
Cystic Fibrosis (CF) 7
β-thalassemia 7
Hereditary Blindness 7
Parkinson's Disease 7
Top Players in Gene Therapy 8
FDA Approvals Granted 8
The Future of Gene Therapy 9
Table of Contents
3. Considerable research has been conducted in genomics
in the past two decades. Extensive research in the gene
therapy domain began in 2001, when two separate
versions of human genome sequences were published.
These drafts contained 30,000 genes, which were used
to decipher gene function, gene abnormality, and
malignant alternations at the gene levels.
James Watson was quoted as stating, “We used to think
that our fate was in our stars, but now we know, in
large measures, our fate is in our genes.” Genes are the
functional unit of heredity. When altered, the proteins
that they encode are unable to carry out their normal
functions. Gene therapy (the use of genes as medicine)
is basically used to correct defective genes.
Gene therapy involves inserting/deleting/correcting
genetic material into human cells to fight or prevent
diseases. It is a promising tool not only for cancer but for
several other diseases, such as Parkinson's, HIV, severe
combined immuno-deficiencies and hemophilia, to
name a few.
Gene therapy initially encountered a lot of problems
as people considered it unethical to use humans as
subjects for clinical trials. However, as time passed, gene
therapy has proved to be a useful tool to cure several
forms of disease.
In this report, we’ll cover the challenges associated
with gene therapy, various modes of administration of
gene based therapies, expected future of gene therapy,
companies that are investing heavily in gene therapy
research, as well as FDA approved gene-based drugs
that are currently available in the market or undergoing
clinical trials.
Executive Summary
4. 2
Challenges Associated
with Developing Genetic Treatments
Dealing with diseases at a genetic level is not easy. While gene therapy isn’t a new field, it has
witnessed very limited success despite over half a century research and development.
Researchers are constantly working on overcoming several challenges being faced by gene therapy.
There is a risk that the
new gene will insert itself
into the path of another
gene, disrupting
its activity.
This could have
damaging effects, for
example, if it interferes
with an important gene
involved in regulating
cell division, it could
result in cancer.
Delivering a gene into
the wrong cell wouldbe
inefficient and cause
health problems for
the patient
Even once the right cell
has been targeted the
gene has to be
turned ON
Cells sometimes obstruct
this process by shutting
down genes that are
showing unusual activity
New considered
genes introduced by
gene therapy may be
potentially-harmful
intruders.
This can spark an
immune response in the
patient, that could be
harmful to them.
Scientists therefore have
the challenge of finding
a way to deliver genes
without the immune
system ‘noticing’.
Many genetic disorders
that can be targeted
with gene therapy are
extremely rare.
Gene therapy therefore
often requires an
individual, case-by-case
approach.
This may be effective, but
may also be
very expensive
Challenges
in Gene Therapy
Disruption Caused by
New Gene
Delivering to the Right
Place and Switching
it ON
Avoiding Immune
Response
Cost
Source : Aranca Analysis
5. 3
Administering Gene Therapy
Gene therapy may be defined as the introduction of genetic material into defective cells for a
therapeutic purpose.
While gene therapy holds great potential as an effective means for selective targeting and treatment
of disease, the field has seen relatively slow progress in the development of effective clinical protocols.
Although identifying genetic factors that cause a physiological defect is pretty straightforward,
successful targeted correction techniques are proving continually elusive. Creating an ideal delivery
vector to target diseased — and only diseased — tissue has proved difficult for those researchers
toiling away tirelessly in their search for the safe treatments of tomorrow.
6. 4
Viral Vectors
These are virus-based vectors. Examples include the retrovirus vector, adeno virus vector system,
adeno associated virus vector, and herpes simplex virus. Extensive research is being conducted on the
various viral vectors used in gene delivery. Here’s a snapshot depicting some of the research being
conducted on various viral vectors.
Viral Vector Type Advantages Disadvantages
Retrovirus
Integrates with host
chromatin
Effective over long
periods
Effective transfection
ex vivo
Low immune response
in host
Small insert size
Effective transfection
in vivo
Safety concerns
Lentivirus
Integrates with host
chromatin
New generations self-
inactivate for safety
purposes
Transfects proliferating,
non-proliferating host
Small insert size
Safety concerns,
immuno-deficiency
origin
Needs active transport in
the cell
Adeno-Associated
Virus
Integrates with host
chromatin
Good length of
expression in vivo
Effective transfection
ex vivo
Low immune response
in host
Safety problems owing
to potential insertional
mutagenesis
Small insert size
Technologically
challenging
Adeno Virus
Extrachromosomal
DNA
Highly efficient
transfection in vivo/ex
vivo
Transfects proliferating,
non-proliferating host
Repeat treatments
ineffective due to strong
immune response
Small insert size
Technologically
challenging
Herpes Simplex
Virus
Extrachromosomal
DNA
Good length of
expression in vivo
Safe for use in immune-
compromised patients
Large insert size up to
30kb
Effective on various cell
types
Difficult to produce in
large quantities
7. 5
Non-viral Vectors
Examples of non-viral vector systems include pure DNA constructs, lipoplexes, DNA molecular
conjugates, and human artificial chromosomes. Owing to the following advantages, non-viral vectors
have gained significant importance in the past few years:
1. Less immunotoxic
2. Risk-free repeat administration
3. Relative ease of large-scale production
A major disadvantage is that the corrected gene needs to be unloaded into the target cell, and the
vector has to be made to reach the required treatment site.
Possible Strategies to Improve Current Viral/Non-viral Vector Systems
Here’s a list of likely strategies that can be employed to improve current vector systems:
1. Making the systems correctly target the defective cell
2. Rendering them capable of transcription
3. Making the systems efficiently penetrate the cell membrane barrier
4. Making them appropriate bio distribution vectors
5. Improving their circulation time in the body
6. Making the systems efficiently interact with the serum component’s therapeutic material so
the therapeutic material is not lost
7. Ensuring that the systems do not react with the immune system and macrophages
8. Rendering them capable to interact with a defective cell surface
9. Making the systems competent to escape degradation by nucleases
10. Maintaining the correct gene expression over a longer time
Patent filing in gene therapy has picked up in the last decade, primarily due to unmet requirements in
life-threatening diseases.
Source : Aranca Analysis Note: The data post 2012 is not reliable, as the patents that are filed in this period are yet to be published.
Patent Filing Trend in the Domain of “Delivery Vehicles Used in Gene Therapy”
8. 6
Gene Therapy Considered a Path-changing Treatment for the Coming Era
Gene therapy has transitioned from the conceptual, technology-driven, laboratory research, to
clinical trial stages for a wide variety of diseases. In addition to curing several genetic disorders, such
as Hemophilia, Chronic Granulomatous Disorder (CGD) and Severe Combined Immune Deficiency
(ADA-SCID), it is also being tested to cure acquired diseases acquired diseases such as cancer,
neurodegenerative diseases, influenza and hepatitis, to name a few.
Gene therapy is not limited to any particular disease. It is also proving to be a promising treatment for
rare diseases such as X-linked adrenoleukodystrophy.
This therapy has proved effective in research conducted for the following diseases:
Fat Metabolism Disorder
Gene therapy is used to correct rare genetic diseases caused due to lipoprotein lipase
deficiency (LPLD). This deficiency leads to fat molecules clogging the blood stream. An
adeno-associated virus vector is used to deliver the corrected copy of the LPL to the
muscle cells. This corrected copy prevents the excess accumulation of fat in the blood
by breaking down the fat molecules. In 2012, the European Union approved Glybera, the
first viral gene therapy treatment for LPLD, manufactured by UniQure. Glybera is likely to
be approved for the American market by 2018.
Adenosine Deaminase (ADA) Deficiency
Gene therapy has successfully been used to treat another inherited immune disorder:
ADA deficiency. More importantly, none of the patients undergoing this treatment
developed any other disorder. The retroviral vector is used in multiple small trials to
deliver the functional copy of the ADA gene. Primarily, all the patients involved in
these trials did not require any injection of ADA enzyme as their immune functions had
immensely improved.
Severe Combined Immune Deficiency (SCID)
A lot of documented work is already available regarding treating this immunodeficiency
with gene therapy; however, clinical trials have not shown promising results. The viral
vectors used during the trials triggered leukaemia in patients. Since then, the focus of
the research and trials has been on preparing new vectors that are safe and do not cause
cancer.
Hemophilia
Patients with haemophilia suffer excessive blood loss as the blood clotting protein
(Factor IX) is absent. Researchers have successfully inserted the missing gene in the liver
cells using an adeno-associated viral vector. After undergoing this treatment, patients
experienced less bleeding as their body was able to create some of the Factor IX protein.
9. 7
Cystic Fibrosis (CF)
CF is a chronic lung disease caused due to a faulty CFTR gene. Genes are injected into
cells using a virus. Recent studies also include testing the cationic liposome (a fatty
container) to deliver DNA to the faulty CFTR gene, thus making the use of the non-viral
gene carrier more successful. Phase II trials using this therapy were published in early
2015, which promised a novel therapeutic approach to CF.
β-thalassemia
Clinical trials on gene therapy for β-thalassemia (the faulty beta-globin gene, which
codes for an oxygen-carrying protein in RBC) can be tracked back to 2007. Blood stem
cells were taken from the patient’s bone marrow and a retrovirus was used to transfer a
working copy of the faulty gene. The modified stem cells were re-injected into the body
to supply functional red blood cells. This treatment, once conducted, lasted over seven
years, even if the patient did not undergo blood transfusion during this time.
Hereditary Blindness
Currently, gene therapy is being tested to treat the degenerative form of inherited
blindness, where patients lose the light-sensing cells in their eyes with time.
Experimental data suggests that the animal models of a mouse, rat and dog show slow
or even reverse vision loss using gene therapy.
The most important advantage associated with gene therapy for eye disorders is that
AAV (adeno-associated virus) cannot shift from the eye to other body parts and hence
does not cause an immune reaction.
In one of the trials, gene therapy was used to improve the vision of a patient with a
degenerative blindness named Leber Congenital Amaurosis (LCA). However, although
their vision improved, the retina kept degenerating with time.
Another trial reported that the vision of six of nine patients who suffered from
degenerative blindness (choroideremia) had improved when the corrected form of REP1
was delivered to them using a viral vector.
Parkinson's Disease
Patients with Parkinson's disease lose the ability to control their movement as their
brain cells stop producing the dopamine molecule used for signaling. A small group of
patients showed improved muscle control when a small area of their brain was treated
with a retroviral vector that contained dopamine-producing genes.
10. 8
Top Players in Gene Therapy
A majority of the big pharmaceutical and biotech companies are actively researching the various
aspects of gene therapy. A lot of research is also being conducted by universities. Players with high
patenting activity include:
Despite the high number of patents being filed, very few gene therapy products have been
commercialized, and some are in the clinical trial stage. A few examples are listed below:
Source : Aranca Analysis
Source : Aranca Analysis
FDA Approval Status for Key Gene Therapy Players
Company/Institution Collaborator Disease Targeted Status
Amgen -- Metastatic melanoma Late-stage human trials
Advantagene -- Prostate cancer
Expected to complete last stage
clinical trials in September 2015
UniQure -- Fat metabolism disorder Approved for sales in Europe
Bluebird Bio -- Neurodegenerative disease
Mid- and late-combination trials
completed in 2013
Glybera Europe -- LPDL
EU commercial plan launched
for 2015
Glybera US -- LPDL IND filing initiated in 2014
AnGesMG -- Artery failure in limbs
To commence late-stage global
testing
Children’s Hospital of
Philadelphia
-- Hereditary blindness Human trials ended in April 2015
Human Stem Cell Institute Neovasculogen Critical limb ischemia
Marketing in Russia since 7
December 2011
11. The Future of Gene Therapy
According to consulting firm Global Data, “The total number of deals in
the global gene therapy market more than doubled from 16 in 2013 to
36 in 2014, with their combined value rising spectacularly from $122.8
million to $4.9 billion over the same period, representing a forty-fold
increase.”
The focus areas for gene therapy include:
1. The genetic manipulation of viral vectors for efficient delivery
2. New viral vectors for delivery
3. Various routes of administration, and the dosage range for
new diseases
4. New approaches of gene therapy such as Spliceosome-
mediated RNA Trans-splicing (SMaRT), hybrid vectors,
gene splicing using ribozymes, triple helix forming
oligonucleotides, antisense, zinc finger nucleases and nano-
robotics are also picking up
Documented data shows that gene therapy involves substantially low
mergers and acquisitions; this is due to the technology being highly
experimental and the majority products being in the early stage of
clinical development. A few famous mergers and acquisitions include:
1. The acquisition of CFR Pharmaceuticals by Abbott
Laboratories to develop gene therapy for chronic pain and
alcoholism
2. The licensing agreement with UniQure and Bristol-Myers
Squibb to develop UniQure’s phase I candidate for congestive
heart failure; this deal is forecast to have the greatest
financial impact in the current fiscal year
3. Voyager Therapeutics signed a multi-million dollar agreement
with Genzyme to develop three phase I programs to develop
therapies for central nervous system disorders
Aranca considers gene therapy a promising technology of the present
times. The future of gene therapy is expected to revolutionize the
medical world.