Gene therapy cell culture media c onsists of important nutrients that provide ample energy to regulate healthy cell cycle. Moreover, it contains bovine serum, animal-derived nutrients, and growth ...

A new kind of cancer gene therapy can be remotely activated at a specific part of the body. The team developed a version of CRISPR that responds to ultrasound, and demonstrated how it can be used ...

Atsena Therapeutics and Nippon Shinyaku have formed an exclusive licensing agreement for ATSN-101, a gene therapy for Leber congenital amaurosis (LCA1). Nippon Shinyaku will commercialize ATSN-101 in ...

An experimental gene therapy raised levels of the key protein protocadherin-15, shown in green, in light-sensing cells in the retinas of nonhuman primates. Credit: Maryna Ivanchenko ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models. Gene therapy can effectively treat various ...

In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutting down its U.S. R&D hub for cell and gene therapy in Pasadena, California ...

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often too large to fit inside the viral vector used for delivery. Researchers ...

Kebilidi is a one-time gene therapy administered directly to the brain in patients with AADC deficiency, rare genetic disorder. The FDA has granted accelerated approval for the gene therapy Kebilidi ...

Summary: Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness. By splitting the large PCDH15 gene into ...

After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells investors that it’s evaluating options for the program. In addition to a ...

An experimental gene therapy raised levels of the key protein protocadherin-15, shown in green, in light-sensing cells in the retinas of nonhuman primates. Image: Maryna Ivanchenko Harvard Medical ...

The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy ...