Date: 11/02/2021

October 27, 2021 11:47 AM EDT Cell/Gene TxFDA+ Endpoints News

Zachary Brennan, Senior Editor

Ten top drugmakers are joining forces with the NIH, the FDA and other nonprofits to streamline the development of new gene therapies for those with rare diseases.

Known as the Bespoke Gene Therapy Consortium, companies including Pfizer, Novartis, Janssen, Biogen, Takeda, Spark Therapeutics, Regenxbio, Thermo Fisher Scientific, and Ultragenyx are teaming up to better understand the basic biology of a common gene delivery vector, known as the adeno-associated virus. The consortium will seek to improve the efficiency of vector manufacturing and enhance the overall therapeutic benefit of AAV-based gene therapies.

According to NIH, the 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 NIH said. “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.”

The NIH and private partners will contribute approximately $76 million over five years to support BGTC-funded projects, including about $39.5 million from the participating NIH institutes and centers, pending the availability of funds. NCATS, which developed the related Platform Vector Gene Therapy (PaVe-GT) program and is the lead NIH institute for BGTC, expects to contribute approximately $8 million over five years.

The BGTC program also will develop a standard set of analytic tests to apply to the manufacture of viral vectors made by consortium researchers.

“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,” the NIH said.

CBER Director Peter Marks has long sought to harness the power of AAV vectors, calling for such a consortium back in April 2020.

“In the case of AAV, current manufacturing constraints present a sort of goldilocks phenomenon, in which viable commercial solutions are not available for very small or large populations but do exist for mid-size indications,” Marks wrote in Nature. “The mission of a public–private partnership applied to AAV-vectored gene therapy would be to provide access to bespoke gene therapy products addressing serious unmet medical needs.”

AUTHOR Zachary Brennan, Senior Editor