Bespoke Gene Therapy Consortium

Overview

The Accelerating Medicines Partnership® (AMP®) program is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private organizations. Managed through the Foundation for the NIH (FNIH), the AMP program aims to improve our understanding of therapeutically relevant biological pathways and validate information that could be relevant for the development of multiple therapeutics.

The AMP Bespoke Gene Therapy Consortium (BGTC) aims to develop platforms and standards that will speed the development and delivery of customized or ‘bespoke’ gene therapies that could treat the millions of people affected by rare diseases.

Launched in October 2021, BGTC is the first AMP initiative focused on rare diseases and the sixth AMP initiative overall. The effort aims to overcome major obstacles related to developing gene therapies and will create a gene therapy protocol book that the research community can use to make the process of developing gene therapies for rare conditions much more efficient.

Need for New Therapies

Because most rare diseases are caused by a defect in a single gene, gene therapy is a potentially powerful treatment approach. Gene therapy treats disease by replacing a malfunctioning gene causing the disease, with a working version of the gene using a delivery system often called a “vector.” For many rare diseases, the limiting factor for developing a gene therapy is not scientific knowledge, but rather operational and financial hurdles. In the current environment, research and testing focus on one disease at a time. In addition, companies often are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring a single gene therapy for a limited number of people to market.

AMP Approach

The BGTC aims to overcome major obstacles related to developing gene therapies.

Basic Research: BGTC-funded scientists will focus on developing a better understanding of the basic biology of a common gene delivery vehicle, or vector, known as the adeno-associated virus (AAV). They will examine the biological and mechanistic steps of delivering vectors and the genes they carry into human cells, as well as activation of the genes in those cells. These results will provide important information for improving the efficiency of vector manufacturing and enhancing the therapeutic benefit of AAV gene therapy. 

Clinical Research: The BGTC program plans to conduct between three and six clinical trials for different rare diseases. These trials will employ different types of AAVs that have been used before in clinical trials. For these trials, the BGTC will focus on streamlining the path from studies in animal models of disease to human clinical trials.

Manufacturing and Production: The BGTC program will develop a standard set of analytic tests to apply to the manufacture of viral vectors made by consortium researchers. Analytics that are broadly applicable to different manufacturing methods could improve and accelerate gene and vector manufacturing and production processes.

Regulatory Requirements: The BGTC program plans to test ways to streamline regulatory requirements and processes for the FDA approval of gene therapies, including developing standardized approaches to preclinical testing (e.g., toxicology studies).

Governance

The BGTC steering committee (SC) is organized by FNIH and includes representatives from each of the partner organizations. The SC operates under the direction of the overall AMP Executive Committee and is responsible for defining and maintaining the research plan, reviewing progress of the project, and providing detailed assessment of milestones for BGTC.

About Rare Diseases

About 7,000 known rare diseases affect an estimated 30 million people in the United States, or nearly 10% of the population. In this country, a rare disease – sometimes called an orphan disease – is a condition that affects fewer than 200,000 people. While the occurrence of a particular rare disease is infrequent, taken altogether, rare diseases are significant public health problem. It currently takes many years for people with a rare disease to receive an accurate diagnosis, and only a few hundred rare diseases have an FDA-approved treatment.

Eighty percent of rare diseases can be traced to mutations or changes in a single gene. In principle, gene therapy could represent a therapeutic strategy for the majority of single gene disorders. At present however, only two FDA-approved treatments for single gene disorders are gene therapies.

Budget

5 Years ($76 Million Total Project Funding)

Disease area Total NIH funding ($M) Total Industry funding ($M) Total non-profit and other organziations funding ($M) Total project funding ($M)
Rare Diseases 39.5 29.9 7.1 76.5

This page last reviewed on October 29, 2021