The US Food and Drug Administration (FDA), the National Institutes of Health (NIH), 10 bio/pharmaceutical companies, and five non-profit organizations have partnered to accelerate development of gene therapies for rare diseases.

The newly launched Bespoke Gene Therapy Consortium (BGTC), part of the NIH Accelerating Medicines Partnership program and project-managed by the Foundation for the National Institutes of Health, aims to optimize and streamline the gene-therapy development process for rare diseases.

The NIH and private partners will contribute approximately $76 million over five years to support BGTC-funded projects. Private partners include: Pfizer; Takeda; Biogen; Janssen Research & Development; Novartis Institutes for BioMedical Research; Regenxbio, a Rockville, Maryland-based bio/pharmaceutical company; Spark Therapeutics, a Philadelphia-based gene-therapy company and part of Roche; Taysha, a Dallas, Texas-based company developing adeno-associated viruses-based gene therapies; Thermo Fisher Scientific; and Ultragenyx Pharmaceutical, a bio/pharmaceutical company developing drugs for rare and ultra-rare genetic diseases.

The primary aim of BGTC is to improve understanding of the basic biology of a common gene-delivery vector, the adeno-associated virus (AAV). To improve and accelerate gene and vector manufacturing and production processes, the BGTC program plans to develop a standard set of analytic tests to apply to the manufacture of viral vectors made by consortium researchers. Such tests could be broadly applicable to different manufacturing methods and make the process of developing gene therapies for very rare conditions much more efficient.

A clinical component of BGTC-funded research will support between four and six clinical trials, each focused on a different rare disease. These diseases are expected to be rare, single-gene diseases with no gene therapies or commercial programs in development and that already have substantial groundwork in place to rapidly initiate preclinical and clinical studies. The trials will employ different types of AAV vectors that have been used before in clinical trials. For these trials, the BGTC will aim to shorten the path from studies in animal models of disease to human clinical trials.

In addition, the BGTC also will 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 (e.g., toxicology studies).

Of the $76 million in funding, the NIH will contribute $39.5 million from participating NIH institutes and centers, pending availability of funds. The NIH’s National Center for Advancing Translational Sciences, which developed the related Platform Vector Gene Therapy program and is the NIH lead institute for BGTC, expects to contribute approximately $8 million over five years.

Several non-profit partners also are involved, including: the Alliance for Regenerative Medicine; the American Society of Gene and Cell Therapy; CureDuchenne, an organization which is focused on a cure for Duchenne muscular dystrophy; the National Organization for Rare Disorders; and The National Institute for Innovation in Manufacturing Biopharmaceuticals.

Source: US Food and Drug Administration