NIH, FDA, 15 Orgs Boost Gene Therapy Development for Rare Diseases

The National Institutes of Health (NIH), FDA, ten pharmaceutical companies, and five non-profit organizations recently partnered to boost the development of gene therapies for 30 million Americans living with a rare disease. 

The organizations will leverage the newly launched Bespoke Gene Therapy Consortium (BGTC), part of the NIH Accelerating Medicines Partnership (AMP) Program and managed by the Foundation for the National Institutes of Health (FNIH). 

BGTC aims to improve understanding of the biology of a common gene delivery vector, known as the adeno-associated virus (AAV). Researchers examine the steps involved in AAV vector product, vector delivery into cells, and how therapeutic genes are activated in target cells. 

The findings will provide vital information to improve the efficacy of vector manufacturing and boost the overall therapeutic benefit of AAV gene therapy, an NIH spokesperson explained. 

“Most rare diseases are caused by a defect in a single gene that could potentially be targeted with a customized or ‘bespoke’ therapy that corrects or replaces the defective gene,” Francis S. Collins, MD, PhD, director of the NIH, said in the announcement. 

“There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies,” Collins continued. 

Nearly 7,000 rare diseases are affecting about 25 to 30 million Americans. But currently, just two heritable diseases have FDA-approved gene therapies. 

Most rare inherited diseases stem from a specific gene mutation already known, making gene therapy a promising therapeutic approach. But gene therapy development can be complex, time-consuming, and expensive. 

According to experts, a standardized therapeutic development model that includes a common gene delivery technology could advance the approach to specific gene therapies, saving time and cost. 

A clinical component of BGTC-funded research will support between four and six clinical trials that focus on a different rare disease. The diseases in the trial will have the groundwork in place to initiate preclinical and clinical studies and have no approved therapies available.

The BGTC program also aims to develop a standard set of analytic tests to apply to the manufacture of viral vectors. The tests could apply to various manufacturing methods and make the gene therapy development process more efficient. 

Additionally, the program will streamline regulatory requirements and processes for the FDA approval of safe and effective gene therapies.

Over a total of five years, NIH and private partners will contribute about $76 million to support BGTC-funded projects, including $39.5 million from the participating NIH institutes and centers. 

And NCATS, the NIH lead institute for BGTC, expects to contribute nearly $8 million.