Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC)
Streamlining Adeno-Associated Virus (AAV) manufacturing and regulatory frameworks to increase accessibility of gene therapies for rare diseases
The Disease Nomination review process is nearing completion and a formal announcement of candidate diseases eligible for Clinical Trial proposals through an RFP will be provided in July 2022.
Overview
The Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (BGTC) is the sixth AMP program and the first to focus on a therapeutic platform. The 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 four-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.
To address this unmet medical need the NIH, the FDA, and a wide range of industry and nonprofit partners have joined together to launch the BGTC, which is coordinated by the FNIH. The 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.
Goals:
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Make adeno-associated virus (AAV) technology more accessible to a broader range of diseases;
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Accelerate the potential to streamline preclinical and product testing;
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Facilitate scientific and regulatory advances that will ultimately benefit the entire field;
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Bring gene therapies to all individuals in need sooner
Design Phase Documents
- Concept plan
- Design Phase Working Group participants
- Program Plan and Partnering Webinar video
- Program Plan and Partnering Webinar slides
Guidance Documents
Media
Interview with Courtney Silverthorn (April 27, 2022): Foundation for the National Institutes of Health Accelerates Biomedical Research and Strategies Around the World, AscellaHealth Developments in Specialty Pharmacy
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
Scientific Publications
After losing her daughter to a rare disease, Sharon King is looking to rewrite the playbook of drug development - PharmaVoice
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
Related Resources
- Accelerating Medicines Partnership® Program
- Accelerating Medicines Partnership® Program - Alzheimer's Disease
- Accelerating Medicines Partnership® Program Bespoke Gene Therapy Consortium (AMP® BGTC)
- Accelerating Medicines Partnership® Program—Common Metabolic Diseases
- Accelerating Medicines Partnership® Program - Parkinson's Disease
- Accelerating Medicines Partnership® Program: Rheumatoid Arthritis, Systematic Lupus Erythematosus & Related Autoimmune Disorders
- Accelerating Medicines Partnership® Program – Schizophrenia (AMP® SCZ)
- Accelerating Medicines Partnership® Program – Type 2 Diabetes Project
AAV Biology and Clinical Opportunities
The Disease Nomination review process is nearing completion and a formal announcement of candidate diseases eligible for Clinical Trial proposals through an RFP will be provided in July 2022.
Click here for a description of the review process and expected timelines. We have also prepared this resource page to assist patient groups in their advocacy journey.
The Bespoke Gene Therapy Consortium has reissued new AAV biology research opportunities. View and download the RFPs below. Proposals are due by 11:59 PM EDT, August 28, 2022.
AAV Biology Gene Expression RFP
AAV Biology Vector Production RFP

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.
Committed Partners
Public-Sector Partners:
- 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)
Private-Sector Partners:
- Alliance for Regenerative Medicine (ARM)*
- American Society of Gene & Cell Therapy*
- ASC Therapeutics Inc*
- Biogen Inc.*
- CureDuchenne*
- Danaher Corporation*
- Foundation Fighting Blindness*
- GENETHON*
- 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 support for this program.
FNIH Contact
- Courtney Silverthorn, Associate Vice President, Research Partnerships, csilverthorn@fnih.org
- Brad Garrison, Senior Project Manager, bgarrison@fnih.org
Related Videos
Accelerating Medicines Partnership® Program - Alzheimer's Disease
Accelerating Medicines Partnership® Program - RA/Lupus Project
Accelerating Medicines Partnership® Program—Common Metabolic Diseases
Accelerating Medicines Partnership® Program – Type 2 Diabetes Project
Support
For more information about supporting or becoming a partner in this program, please contact Heidi Blythe, Director of Development, hblythe@fnih.org.
ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.