Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium

Overview

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.

Goals:

• 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.

Scientific Publications

Group Therapy – PharmaTimes

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

Media

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

BGTC One-Year Anniversary Newsletter (November 30, 2022)

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

Regulatory Resources

FDA CBER Cellular & Gene Therapy Guidances

FDA releases Guidance on Human Gene Therapy for Neurodegenerative Diseases

Interactions with the FDA Office of Tissues and Advanced Therapies

FNIH Contacts

• Courtney Silverthorn, Ph.D., Associate Vice President, Research Partnerships, csilverthorn@fnih.org
• Brad Garrison, MBA, PMP, Senior Project Manager, bgarrison@fnih.org
• Juan Esparza-Trujillo, M.S., GLPCP, GMPCP, Project Manager, jesparza@fnih.org