02:23 , May 18, 2019 |  BioCentury  |  Product Development

AAVs on the brink: Charting the clinical landscape of in vivo gene therapies

Gene therapy is finally teetering on the edge of becoming a drug development mainstay, with at least nine AAV-based therapies in Phase III or II/III testing. An analysis of the AAV therapies in the clinic...
11:10 , May 8, 2019 |  BC Extra  |  Company News

Why dwarfism start-up Therachon picked Pfizer takeout over IPO

Rather than file for an IPO as it had been planning to do, Therachon has elected to take a buyout offer from Pfizer, handing the pharma a clinical program to treat achondroplasia. Pfizer will pay...
19:37 , May 6, 2019 |  BC Extra  |  Company News

May 6 Company Quick Takes: Esperion, BioMarin, WuXi AppTec, Axsome, Harbour BioMed/CTTQ, GW, Adynxx

FDA accepts Esperion NDAs for LDL-C-lowering indications  Esperion Therapeutics Inc. (NASDAQ:ESPR) gained $5 (12%) to $48.24 on Monday after FDA accepted for review a pair of NDAs for bempedoic acid (ETC-1002) and a bempedoic acid/ezetimibe...
21:34 , Apr 16, 2019 |  BC Extra  |  Clinical News

Wave down on Phase I safety data for DMD therapy

Following a setback last week for its Huntington disease program, Wave lost $9.55 (28%) to $24.47 on Tuesday after the company announced plans to test two lower doses of suvodirsen in a Phase II/III trial...
20:23 , Apr 2, 2019 |  BC Extra  |  Clinical News

Sangamo gains on Phase I/II readout for hemophilia A gene therapy

Interim Phase I/II data for Sangamo’s hemophilia A gene therapy Tuesday helped shares of the company complete a round-trip from its 31% tumble on a February readout for its mucopolysaccharidosis therapies. Sangamo Therapeutics Inc. (NASDAQ:SGMO)...
17:36 , Mar 8, 2019 |  BC Week In Review  |  Company News

CHMP recommendations include Ondexxya, Waylivra

EMA's CHMP recommended a basket of approvals and label extensions on March 1, including Ondexxya andexanet alfa and Waylivra volanesorsen. The agency recommended conditional approval of Ondexxya from Portola Pharmaceuticals Inc. (NASDAQ:PTLA) as the first...
01:10 , Mar 2, 2019 |  BioCentury  |  Product Development

Roche broadens its BD scope

Roche’s proposed acquisition of Spark Therapeutics Inc. is the pharma’s first major deal since James Sabry took over partnering, and could be a glimpse of what’s to come as the pharma broadens its horizons on...
22:10 , Mar 1, 2019 |  BC Extra  |  Company News

CHMP recommendations include Ondexxya, Waylivra

EMA's CHMP recommended a basket of approvals and label extensions Friday, including Ondexxya andexanet alfa and Waylivra volanesorsen. The agency recommended conditional approval of Ondexxya from Portola Pharmaceuticals Inc. (NASDAQ:PTLA) as the first approved antidote...
20:02 , Mar 1, 2019 |  BC Week In Review  |  Company News

NICE rebuffs BioMarin's ultra-orphan therapy

The U.K.'s NICE did not recommend Brineura cerliponase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) due to uncertainty around the therapy's long-term efficacy and concerns NHS would be unable to implement the rare disease biotech's proposed...
21:09 , Feb 22, 2019 |  BC Extra  |  Company News

NICE nixes BioMarin's ultra-orphan therapy for lack of long-term data

The U.K.'s NICE did not recommend Brineura cerliponase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) due to uncertainty around the therapy's long-term efficacy and concerns NHS would be unable to implement the rare disease biotech's proposed...