Capsida Biotherapeutics Inc. has gained IND clearance from the FDA for CAP-002, its first-in-class, intravenously administered gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). Dosing in the phase I/IIa SYNRGY trial will begin in the third quarter of this year.
Following last fall’s $1 billion development deal with Eli Lilly and Co., precision medicine company Haya Therapeutics SA has raised $65 million in a series A. It’s all part of increased validation from big pharmas that long noncoding RNAs, such as those being developed by Haya, have a strong future.
Genprex Inc. and University of Pittsburgh have entered into a new sponsored research agreement (SRA) to study GPX-002, Genprex’s gene therapy for diabetes in animal models of type 1 and type 2 diabetes.
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
The appointment May 6 of Vinay Prasad as the head of the U.S. FDA’s Center for Biologics Evaluation and Research (CBER) “bodes poorly” for Sarepta Therapeutics Inc.’s development-stage pipeline, said Wainwright analyst Mitchell Kapoor – and Wall Street reflected as much, as the stock (NASDAQ:SRPT) ended that day down 26.6% vs. an XBI drop of 6.6% – this ahead of the after-hours earnings disclosure that pushed the Cambridge, Mass.-based firm down even farther by more than another 20%, with the XBI unchanged.
Nuevocor Pte. Ltd. has closed a $45 million series B, enabling it to move lead gene therapy NVC-001 into the clinic in the treatment of an inherited form of cardiomyopathy.
Nuevocor Pte. Ltd. has closed a $45 million series B, enabling it to move lead gene therapy NVC-001 into the clinic in the treatment of an inherited form of cardiomyopathy.
Odylia Therapeutics Inc. has announced it is working under a codevelopment partnership with the NPHP1 Family Foundation to create an AAV-based gene replacement therapy for retinal dystrophy caused by mutations in the NPHP1 (nephrocystin-1) gene.
U.S. FDA Commissioner Marty Makary is starting to fill the vacancies at the agency that’s seen its senior leadership ravaged by retirements and terminations. Makary’s first pick is Vinay Prasad as the new head of the FDA’s Center for Biologics Evaluation & Research (CBER), the center that oversees vaccines, blood products, allergenics and cellular, tissue and gene therapies.
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