Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium
Streamlining regulatory frameworks to accelerate gene therapies for rare diseases
The Accelerating Medicines Partnership® (AMP®) Program Bespoke Gene Therapy Consortium (AMP BGTC) is the first AMP project to focus on a therapeutic platform. AMP BGTC brings together partners from the public, private, and non-profit sectors to foster development of gene therapies intended to treat rare genetic diseases, which affect populations too small for viable commercial development.
Building on the successful AMP model, this program focuses on generating a standard operational playbook for developing such gene therapies. This playbook, which will be established and piloted using up to six clinical trial test cases, will include streamlined templates, master regulatory files, and uniform manufacturing processes to create a pathway toward the commercial viability and sustainability of gene therapies for very rare diseases. This approach could have substantial positive impacts on the larger gene therapy field, especially as the field moves into the era of genome editing.
Increased demand for gene therapy manufacturing, and limitations in the development and regulatory approval process, have led to a bottleneck in delivering these important new therapies to patients. Unfortunately, there is a lack of scalability, reproducibility, or regulatory generalizability in the current environment, which makes access to these therapies inefficient for most patients.
The issues include determining the quantity of supportive preclinical evidence needed prior to patient treatment, understanding the clinical information that should be captured, and producing a quality product that is fit-for-purpose – all in the context of an appropriate regulatory paradigm that facilitates safe patient treatment with a high likelihood of effectiveness.
Out of over 7,000 rare diseases affecting a total of 25-30 million Americans, approximately 4,000 are single-gene diseases that have the potential to be treated by gene therapy. However, to date only two gene therapy products for rare diseases have received FDA approval. To address this unmet medical need the NIH, the FDA, and a wide range of industry and nonprofit partners have joined together to launch AMP BGTC, which is coordinated by the FNIH. AMP BGTC focuses on a single gene delivery technology, adeno-associated virus (AAV), one of the safest platforms for gene delivery to address a variety of human diseases. The program’s primary aims include pursuing an in-depth understanding of basic AAV life cycle biology, which will facilitate optimization of vector generation and delivery, as well as standardizing and streamlining regulatory requirements for approval of gene therapies for ultra-rare and bespoke diseases.
Read more about AMP BGTC on the National Center for Advancing Translational Therapeutics’ website.
- Make adeno-associated virus (AAV) technology more accessible to a broader range of diseases.
- Accelerate the potential to streamline preclinical and product testing.
- Facilitate scientific and regulatory advances that will ultimately benefit the entire field.
- Bring gene therapies to all individuals in need sooner.
Group Therapy – PharmaTimes
`Bespoke Gene Therapy Consortium’ sets out to enable gene therapies for ultra-rare diseases - Nature Reviews Drug Discovery
Creating drug master files for AAV vectors – BioCentury
NIH Director's Blog (January 24, 2023): A Rare Public Health Challenge
MedPage Today (January 19, 2023): NIH Project Aims to Make Gene Therapy 'Playbook' Public
MedPage Today (January 10, 2023): Accelerated Approval Not the Finish Line, Says FDA Commissioner
RareDiseaseAdvisor (December 29, 2022): Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases
CheckRare (November 30, 2022): The Bespoke Gene Therapy Consortium
Science Advisory Board (October 13, 2022): FDA biologics chief outlines ways to remove barriers to cell and gene therapies
FNIH Announcement (July 12, 2022): AMP® BGTC Announces Rare Disease Selection Candidates, Issues Clinical Trial RFP
AscellaHealth Developments in Specialty Pharmacy interview with Courtney Silverthorn, Ph.D. (April 27, 2022): Foundation for the National Institutes of Health Accelerates Biomedical Research and Strategies Around the World
FNIH Press Release (October 27, 2021): FNIH Launches a Transformative Partnership to Streamline Development of Treatments for Rare Diseases
NIH Press Release (October 27, 2021): NIH, FDA and 15 private organizations join forces to increase effective gene therapies for rare diseases
BGTC Funding Opportunities
Addressing an Unmet Need
Approximately 25–30 million people in the United States live with the devastating effects of rare diseases. There are over 5,000 rare diseases that are caused by genetic defects. These patients frequently lack access to effective treatment, as knowledge about many rare diseases, as well as funding for research, often lag behind more common diseases.
- California Institute for Regenerative Medicine (CIRM)
- National Center for Advancing Translational Sciences (NCATS)
- National Institute for Neurological Disorders and Stroke (NINDS)
- National Institute of Mental Health (NIMH)
- National Human Genome Research Institute (NHGRI)
- National Heart, Lung and Blood Institute (NHLBI)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- National Eye Institute (NEI)
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- National Institute of Dental and Craniofacial Research (NIDCR)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- The Brain Research Through Advancing Innovative Neurotechnologies® (BRAIN) Initiative, a trans-NIH initiative involving 10 NIH Institutes and Centers
- U.S. Food and Drug Administration (FDA)
- Alliance for Regenerative Medicine (ARM)*
- American Society of Gene & Cell Therapy*
- ASC Therapeutics Inc*
- Biogen Inc.*
- Danaher Corporation*
- Foundation Fighting Blindness*
- Janssen Research & Development, LLC*
- National Organization for Rare Disorders (NORD)*
- Novartis Institutes for BioMedical Research*
- Ovid Therapeutics Inc.*
- Pfizer Inc.*
- REGENXBIO Inc.*
- Rett Syndrome Research Trust*
- RTW Charitable Foundation*
- Spark Therapeutics*
- Takeda Pharmaceutical Company Limited*
- Taysha Gene Therapies*
- The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)*
- Thermo Fisher Scientific*
- Ultragenyx Pharmaceutical*
*Provided financial and/or support for this project.
ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.