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 is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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