19:39 , Feb 9, 2018 |  BC Week In Review  |  Clinical News

Abeona reports early data for AAV9 MPS IIIB gene therapy

Abeona Therapeutics Inc. (NASDAQ:ABEO) reported that its adeno-associated viral serotype 9 (AAV9)-based gene therapy ABO-101 was well tolerated with no treatment-related serious adverse events reported through day 30 in the first patient dosed in a...
21:43 , Feb 7, 2018 |  BC Extra  |  Clinical News

Abeona gains on early data for AAV9 MPS IIIB gene therapy

Abeona Therapeutics Inc. (NASDAQ:ABEO) was up $2.25 (14%) to $18.20 on Wednesday after reporting that its adeno-associated viral serotype 9 (AAV9)-based gene therapy ABO-101 was well tolerated with no treatment-related serious adverse events reported through...
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...
07:00 , Sep 28, 2015 |  BioCentury  |  Product Development

Sailing on in Sanfilippo

uniQure N.V. says Phase I/II data on AMT-110 support both the start of a pivotal trial for Sanfilippo type B syndrome, and the company's plans to use its AAV5 vector in other rare lysosomal storage...
07:00 , Jun 15, 2015 |  BC Week In Review  |  Company News

Abeona, PlasmaTech Biopharmaceuticals deal

PlasmaTech acquired Abeona for 4 million PlasmaTech shares, or about $31.9 million based on PlasmaTech’s close of $7.98 on May 15, the last trading day before the deal closed. In mid-2015, the company plans to...
07:00 , Mar 23, 2015 |  BioCentury  |  Emerging Company Profile

Complementary edge

The neurological manifestations of many lysosomal storage disorders are poorly treated because it hasn't been possible to get enough of an enzyme or gene therapy vector across the blood-brain barrier to replace dysfunctional proteins. Abeona...
07:00 , May 19, 2014 |  BC Week In Review  |  Clinical News

ABX-A regulatory update

Abeona said FDA granted Orphan Drug designation to ABX-A to treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo A syndrome) and to ABX-B to treat mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo B syndrome). Both programs are gene...