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...
21:21 , Nov 10, 2017 |  BC Week In Review  |  Clinical News

BioMarin reports Phase I/II data for BMN 250 in mucopolysaccharidosis IIIB

In September, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported interim data from 3 patients with mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo type B syndrome) in the dose-escalation portion of the Phase I/II BMN 250-201 trial showing that intracerebroventricular...
19:39 , Mar 10, 2017 |  BC Week In Review  |  Company News

Alexion SBC-103 news

Alexion said it will "reduce its investment" in SBC-103 (rhNAGLU). The company said that in 4Q16 it recognized an $85 million impairment charge related to SBC-103. Alexion said no additional trials are planned and that...
21:19 , Nov 18, 2016 |  BC Week In Review  |  Company News

uniQure restructuring news

uniQure said it will reduce headcount by 50-60 (20-25%) to about 190 by YE17 to focus on key pipeline projects. The company expects the job cuts will save €5-€6 million ($5.4-$6.4 million) annually, while reprioritizing...
23:50 , Nov 15, 2016 |  BC Extra  |  Company News

uniQure paring down gene therapy portfolio

Gene therapy company uniQure N.V. (NASDAQ:QURE) said it will reduce headcount by 20-25%, eliminating 50-60 positions by YE17, in order to focus on key pipeline projects. The company expects the job cuts will save EUR...
07:00 , May 2, 2016 |  BC Week In Review  |  Clinical News

BMN 250: Phase I/II started

BioMarin began the open-label, dose-escalation, international Phase I/II BMN 250-201 trial to evaluate BMN 250 given via intracerebroventricular infusion. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), San Rafael, Calif.   Product: BMN 250   Business: Endocrine/Metabolic   Molecular...
08:00 , Mar 7, 2016 |  BC Week In Review  |  Clinical News

RhNAGLU: Phase I/II data

Top-line data from an open-label, U.S. and U.K. Phase I/II trial in 11 patients ages 2-10 with MPS IIIB showed that 1 and 3 mg/kg IV SBC-103 every other week reduced mean CSF heparan sulfate...
07:00 , Sep 28, 2015 |  BC Week In Review  |  Clinical News

AMT-110: Phase I/II data

Top-line data from an open-label, French Phase I/II trial in 4 patients ages 20-53 months with Sanfilippo type B syndrome showed that a single administration of intracerebral AMT-110 plus continuous immune suppression therapy with mycophenolate...