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...
03:02 , Jun 30, 2018 |  BioCentury  |  Finance

Smitten by SMID-caps

Risk is pervasive heading into the third quarter and choppy waters are expected, but 13 buysiders told BioCentury they’re not afraid to stay the course. A politically charged environment is likely to intensify as the...
18:27 , Jun 22, 2018 |  BC Week In Review  |  Clinical News

Sarepta's DMD candidate leads to microdystrophin expression of 76.2% in Phase I/IIa

Sarepta Therapeutics Inc. (NASDAQ:SRPT) reported on June 19 preliminary data from a Phase I/IIa trial evaluating AAVrh74.MHCK7.micro-Dystrophin to treat Duchenne muscular dystrophy (DMD). In three DMD patients in the open-label, U.S. trial, the microdystrophin gene...
23:58 , Jun 21, 2018 |  BC Extra  |  Company News

Sarepta says no to right to try for its DMD therapies

Following data from a small trial of a Duchenne muscular dystrophy gene therapy, Sarepta Therapeutics Inc. (NASDAQ:SRPT) told BioCentury that it will not offer pre-approval access under right-to-try legislation or FDA’s expanded access program for...