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 Problem

With the current commercial drug development model, companies cannot recover the costs required to develop gene therapies to treat rare and ultra-rare genetic diseases, because these diseases affect relatively few patients.

The Solution

This project will create tools to streamline the gene therapy development process, aiming to reduce associated costs and encourage companies to pursue gene therapies for rare genetic 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:

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

Design Phase Documents

Concept plan
Design Phase Working Group participants
Program Plan and Partnering Webinar video
Program Plan and Partnering Webinar slides

Guidance Documents

Current FDA CBER Cellular & Gene Therapy Guidances

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

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:

Private-Sector Partners:

*Provided financial support for this program.

Read what the partners are saying

FNIH Contact

Courtney Silverthorn, Associate Vice President, Research Partnerships, csilverthorn@fnih.org
Brad Garrison, Senior Project Manager, bgarrison@fnih.org

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.