The FDA is setting out to streamline international regulations of gene therapies with a pilot program to review applications alongside global regulatory partners, an agency spokesperson told Endpoints News.

The spokesperson said that the program, called the Collaboration on Gene Therapies Global Pilot, is part of the agency’s work toward global regulatory harmonization of these products. The FDA is working with international partners, global regulators and the World Health Organization on these efforts, they said.

While it’s still early on in development, the pilot will explore the possibility for “concurrent collaborative” review of applications alongside global regulatory partners.

“While countries around the world have their own regulatory authorities, there are no uniform global standards for the evaluation and regulation of cell and gene therapy products,” the spokesperson said. “We believe that harmonization efforts in this area can help facilitate more efficient clinical development.”

The FDA has also used this approach for Project Orbis, which it established in 2019 to provide a pathway for concurrent submission and review of oncology products.

The pilot for gene therapy products comes as the agency has tightened its focus on gene therapies and rare disease in recent years.

This past fall, the FDA launched an Operation Warp Speed-like pilot program for rare disease to speed the approval process. FDA’s Peter Marks has also previously said that the agency is pushing the accelerated approval pathway for rare disease products.

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Related Services

Plasmids GMP Services

Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.

AAV GMP Services

Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
aav icon

Technology Platforms

PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.