News & Media

News
July 11, 2022
Optimizing Process Development Early – A critical factor in successful viral vector scale up and large scale manufacture
Blog
July 20, 2020
Current Good Manufacturing Processes (cGMP) are outlined by the FDA, which defines them as the “minimum requirements for the methods,...
Blog
April 15, 2020
Gene therapy is the process of replacing damaged or unhealthy genetic material with new material for treating various diseases and...
Research
May 28, 2019
Researchers identify a key regulatory protein implicated in Galgt2 overexpression and begin to elucidate its protective mechanism against muscular dystrophy.
Research
April 19, 2019
Researchers recently published the long-term outcomes of patients who received the investigational drug AVXS-101 – an adeno-associated virus serotype 9 mediated gene replacement therapy.
Research
November 16, 2018
The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.
Research
July 24, 2018
Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.
Research
April 2, 2018
Recent applications of adeno-associated virus (AAV) mediated gene therapy have focused mainly on correcting recessively inherited diseases. But what about dominantly inherited disorders? It looks like AAV could be a delivery mechanism for treating those genetic disorders, too.
News
September 20, 2016
Sarepta Therapeutics announced yesterday that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy...