Cell & Gene Therapy Review

For many CGT companies, the U.S. has long been the default starting point for new ventures. Biotech and pharma companies have traditionally built commercial strategies around the primacy of the American market, prioritizing the FDA pathway and U.S. funding streams, and treating other regions as secondary opportunities once a product had clinical and commercial momentum. In recent years, that model appears to be weakening, as more companies look beyond the U.S. earlier in their development.

RNA-based gene therapy developer Rznomics will join Lilly Gateway Labs (LGL) in San Diego, California, tightening its ties with the U.S. drugmaker. LGL is designed to support biotech innovation by providing access to strategic capital, lab space and technology, and R&D capabilities.

Valerio Therapeutics has agreed to buy Etherna Immunotherapies for an enterprise value of $35 million (€30 million). The acquisition marks a major milestone in Valerio's strategy to build a leading platform in the development of next-generation precision-guided RNA therapeutics.

The FDA has placed a second clinical hold on Regenxbio’s investigational gene therapy, RGX-121, for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter Syndrome, following the discovery of asymptomatic spine MRI findings in five participants in the CAMPSIITE study.

The U.S. FDA has extended the PDUFA target action date for Capricor Therapeutics’ BLA for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy, from August 22, 2026 to November 22, 2026. In a closely watched adcomm meeting in July, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 9-3 that available evidence did not support the effectiveness of Capricor’s deramiocel for the treatment of cardiomyopathy in patients with DMD.

Cellares is resizing its organization after a large pharmaceutical customer ended its partnership with the integrated development and manufacturing organization, according to a LinkedIn post from Cellares CEO and co-founder, Fabian Gerlinghaus. According to Gerlinghaus, the customer’s decision was specific to that partnership and the loss requires the company to reduce its headcount. While the customer was not identified, industry insiders are speculating that it is Bristol Myers Squibb.

The U.S. FDA has granted accelerated approval to Ultragenyx Pharmaceutical’s gene therapy, Genglycos (pariglasgene brecaparvovec-opnr), in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream.

Dr. Heidi Overton, a White House domestic policy aide, has been selected to lead the FDA, President Trump announced today. Overton received her medical degree from the University of New Mexico School of Medicine, before attending Johns Hopkins University Bloomberg School of Public Health. Prior to joining Trump's second administration, she was the chief policy officer at the America First Policy Institute, a conservative think tank.

For decades, scientists dreamed about the seemingly limitless therapeutic potential of RNA. Part of what made RNA so compelling was its versatility. Across its various forms, RNA could target previously undruggable disease drivers, instruct cells to produce therapeutic proteins, silence harmful mutations, rewrite faulty RNA transcripts before they were translated into dysfunctional proteins, or train the immune system to fight disease.

AbelZeta Pharma has received U.S. FDA clearance of its IND application for prizlon-cel, an anti-CD20/CD19 bispecific CAR-T, for the treatment of relapsed or refractory large B-cell lymphoma. In the same press release, AbelZeta revealed that it had regained all development, regulatory, manufacturing, commercialization, out-licensing and other collaboration rights relating to prizlon-cel back in July.