22:30 , Feb 2, 2018 |  BioCentury  |  Product Development

AA Viral meme

Gene therapy pioneer James Wilson’s revelation of two new types of toxicity in animals receiving high systemic doses of adeno-associated viral vectors is unlikely to herald a major disruption of the AAV space, though it...
21:12 , Jan 27, 2017 |  BC Week In Review  |  Company News

Sarepta, Nationwide Children's Hospital deal

The hospital granted Sarepta exclusive, worldwide rights to a preclinical gene therapy program that delivers the beta-1,4-N-acetyl-galactosaminyltransferase 2 ( GALGT2 ; B4GALNT2) gene, which expresses a protein that performs a function similar to dystrophin. Sarepta...
00:00 , Jan 11, 2017 |  BC Extra  |  Company News

Sarepta discusses Exondys 51 sales, DMD pipeline

Sarepta Therapeutics Inc. (NASDAQ:SRPT) soared $6.63 (21%) to $37.89 on Tuesday following a presentation in which President, CEO and CMO Edward Kaye reported initial sales of Exondys 51 eteplirsen and provided an update on the...