20:46 , Apr 15, 2019 |  BC Extra  |  Company News

$1.2B Catalent-Paragon deal latest in race to lock up gene therapy manufacturing

The 6x multiple Catalent is paying for gene therapy manufacturer Paragon Bioservices suggests buyers believe gene therapy companies will remain heavily dependent on outsourcing as new therapies are approved in the next few years. Catalent...
18:20 , Feb 15, 2019 |  BC Week In Review  |  Clinical News

Solid reports another DMD gene therapy setback

Solid Biosciences Inc. (NASDAQ:SLDB) reported lower-than-expected microdystrophin protein levels in liver biopsies from the first three patients treated with its microdystrophin gene transfer therapy, SGT-001, in the Phase I/II IGNITE DMD trial to treat Duchenne...
21:02 , Feb 7, 2019 |  BC Extra  |  Clinical News

Solid tumbles after latest DMD gene therapy setback

Solid Biosciences Inc. (NASDAQ:SLDB) lost $15.15 (68%) to $7.19 Thursday after reporting lower than expected microdystrophin protein levels in liver biopsies from the first three patients treated with its microdystrophin gene transfer therapy, SGT-001, in...
20:46 , Oct 5, 2018 |  BC Week In Review  |  Clinical News

Sarepta reports microdystrophin expression data for fourth patient in DMD gene therapy trial

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said AAVrh74.MHCK7.micro-Dystrophin led to mean sarcolemma-localized transduced microdystrophin expression, as measured by immunohistochemistry, of 81.2% and a mean fiber intensity of 96% compared with normal controls in four Duchenne muscular dystrophy...
17:27 , Sep 28, 2018 |  BC Week In Review  |  Clinical News

FDA lifts holds on Epizyme, Sarepta programs

FDA lifted separate clinical holds on tazemetostat from Epizyme Inc. (NASDAQ:EPZM) and a gene therapy program targeting micro-dystrophin from Sarepta Therapeutics Inc. (NASDAQ:SRPT), the companies said Sept. 24. Epizyme is reopening enrollment in all of...
22:32 , Sep 24, 2018 |  BC Extra  |  Clinical News

FDA lifts holds on Epizyme, Sarepta programs

FDA lifted separate clinical holds on tazemetostat from Epizyme Inc. (NASDAQ:EPZM) and a gene therapy program targeting micro-dystrophin from Sarepta Therapeutics Inc. (NASDAQ:SRPT), the companies said Monday. Epizyme is reopening enrollment in all of its...
16:28 , Aug 10, 2018 |  BC Week In Review  |  Company News

Sarepta bolsters gene therapy pipeline through deal with Lacerta

Sarepta Therapeutics Inc. (NASDAQ:SRPT) gained exclusive rights to an undisclosed preclinical gene therapy to treat Pompe disease from Lacerta Therapeutics Inc. (Alachua, Fla.), as well as options to two additional CNS gene therapy candidates. Lacerta...
23:25 , Aug 8, 2018 |  BC Extra  |  Company News

Sarepta bolsters gene therapy pipeline through deal with Lacerta

Sarepta Therapeutics Inc. (NASDAQ:SRPT) gained exclusive rights to an undisclosed preclinical gene therapy to treat Pompe disease from Lacerta Therapeutics Inc. (Alachua, Fla.), as well as options to two additional CNS gene therapy candidates. Lacerta...
18:19 , Jul 27, 2018 |  BC Week In Review  |  Clinical News

FDA places clinical hold on Sarepta's DMD gene therapy

Sarepta Therapeutics Inc. (NASDAQ:SRPT) and partner Nationwide Children’s Hospital said FDA placed a clinical hold on a Phase I/IIa trial of their Duchenne muscular dystrophy gene therapy, AAVrh74.MHCK7.micro-Dystrophin. The partners said the hold is due...
03:05 , Jun 30, 2018 |  BioCentury  |  Finance

Hidden in the rough

Judicious stock picking was especially important in the second quarter, as 57% of global biotech stocks saw their equity values decline. Volatility continued to roil global markets during the period and additional uncertainty looms with...