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 ...

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 ...

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 ...

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 ...

Regenxbio reported Monday the first clinical evidence showing improved muscle function in boys with Duchenne muscular dystrophy following treatment with its experimental gene therapy. The positive ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. Now, seven years and many approvals later, innovation in the space has hit new heights with ...

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 followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene ...