17:50 , Jul 20, 2018 |  BC Week In Review  |  Company News

PTC acquiring CNS gene therapy play Agilis

PTC Therapeutics Inc. (NASDAQ:PTCT) will acquire gene therapy company Agilis Biotherapeutics Inc. (Cambridge, Mass.), giving the rare disease company four products in development for CNS disorders. PTC will pay Agilis shareholders about $200 million up front,...
22:43 , Jul 19, 2018 |  BC Extra  |  Company News

PTC acquiring CNS gene therapy play Agilis

PTC Therapeutics Inc. (NASDAQ:PTCT) will acquire gene therapy company Agilis Biotherapeutics Inc. (Cambridge, Mass.), giving the rare disease company four products in development for CNS disorders. PTC will pay Agilis shareholders about $200 million up...
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...
00:39 , Nov 22, 2016 |  BC Week In Review  |  Clinical News

AGIL-AADC regulatory update

FDA granted rare pediatric disease designation to Agilis’ AGIL-AADC to treat Aromatic L-Amino Acid Decarboxylase (AADC) deficiency. The product is an adeno-associated viral (AAV) vector encoding the dopa decarboxylase (DDC; AADC) gene. Agilis Biotherapeutics LLC, Cambridge,...
07:00 , Jun 18, 2012 |  BC Week In Review  |  Clinical News

AADC gene therapy: Phase I data

Researchers at the university reported data from a Phase I trial in 4 patients aged 4-6 with mutations in the AADC gene showing that AAV vector-mediated gene transfer of the AADC gene injected bilaterally into...