ASGCT Joins National Partnership to Accelerate Gene Therapy Development for Rare Diseases

The American Society of Gene and Cell Therapy (ASGCT) is joining forces with FDA, 11 NIH institutes and centers, and more than a dozen other nonprofit groups and private companies to help accelerate the delivery of gene therapies for rare disease patients through the newly launched Bespoke Gene Therapy Consortium (BGTC).

This public-private collaboration, managed by the Foundation for the National Institutes of Health (FNIH), aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with many rare diseases, in particular those diseases that are so rare that they challenge the typical commercial development model.

Most rare inherited diseases stem from a specific gene mutation that is already known, making gene therapy a promising therapeutic approach. However, the gene therapy development process is also complex and hampered by a lack of common biologic, manufacturing, and regulatory standards. The new effort aims to make gene therapy more accessible by creating a development template that can be used repeatedly to expedite the delivery of novel therapies for many different genetic disorders. As the primary professional membership organization for gene and cell therapy, ASGCT is well positioned to aid in this effort.

“We are thrilled to be a part of such a meaningful and necessary partnership,” said ASGCT CEO David Barrett, JD. “Our goal as an organization has always been to leverage the science to make gene therapies more accessible, and this effort will speed up that process in order to bring therapeutic treatments to rare disease patients.”

A primary goal of the BGTC is to improve understanding of the basic biology of AAV vectors, one of the safest platforms and most common tools for gene delivery, so they can be optimized for treating rare diseases. The partnership will also support a series of clinical trials conducted by NIH for rare, monogenic diseases with no gene therapies in development using small batch manufacture of vector, standardization of clinical protocols, a standard gene delivery system, and production and purification methods with open access to data. It will also explore methods to streamline regulatory requirements and processes for the FDA approval of safe and effective gene therapies, including developing standardized approaches to preclinical testing.

The BGTC is the latest initiative to emerge from the Accelerating Medicines Partnership® (AMP®) Program, a public-private collaboration among the NIH, the FDA, the pharmaceutical industry, patient organizations, and other stakeholders to speed drug development across different diseases. At a cost of $76.5 million over five years, the BGTC brings together the resources of 27 partner organizations spanning the public, private, and nonprofit sectors.