BC Extra | Dec 23, 2019
Company News

Roche’s ex-U.S. deal for Sarepta DMD programs could extend beyond micro-dystrophin gene therapy

After weathering one clinical failure in DMD this fall, Roche broadened its commitment to gene therapy by obtaining ex-U.S. rights to Sarepta’s micro-dystrophin-targeting program in a deal worth more than $1.1 billion up front. Roche...
BC Extra | Dec 18, 2019
Financial News

With new name, Epirium snags $85M series A to develop hormonal therapy for muscular dystrophies

Ten-year-old Cardero Therapeutics Inc. relaunched Wednesday as Epirium Bio with a tranched $85 million series A led by Longitude Capital and Arch Venture Partners, IP around a new hormonal pathway as well as human proof-of-concept...
BC Extra | Nov 8, 2019
Clinical News

Roche’s RG6206 becomes second myostatin inhibitor to fail in DMD in past year

The failure of Roche’s RG6206 marks another blow to the hypothesis that blocking myostatin can improve muscle growth and function in ambulatory boys with Duchenne muscular dystrophy. Roche (SIX:ROG; OTCQX:RHHBY) sent a letter Wednesday to...
BC Extra | Sep 17, 2019
Company News

Sept. 17 Company Quick Takes: Priority Review for Merck Ebola vaccine; plus Edgewise’s debut, Adaptive, AskBio and Acceleron

Merck Ebola vaccine granted Priority Review  FDA accepted and granted Priority Review to a BLA from Merck & Co. Inc. (NYSE:MRK) for its Ebola vaccine V920 (rVSV-ZEBOV). The candidate has a PDUFA date of March...
BC Extra | May 9, 2019
Clinical News

May 9 Clinical Quick Takes: Polyphor, BMS, Cel-Sci, University of Pittsburgh, Merck, Scholar Rock, Promethera

Polyphor halts Phase III enrollment of pneumonia therapy  Polyphor Ltd. (SIX:POLN) said late Thursday it will suspend enrollment in the Phase III PRISM-MDR and PRISM-UDR trials of pneumonia antibiotic murepavadin (POL7080) after observing a 56%...
BC Week In Review | Apr 12, 2019
Clinical News

Acceleron discontinues ACE-2494 due to antidrug antibodies

Acceleron discontinued development of ACE-2494 due to the frequency of antidrug antibodies observed among healthy volunteers in a Phase I trial. Acceleron Pharma Inc. (NASDAQ:XLRN) was developing ACE-2494 as a potential treatment of neuromuscular disorders....
BioCentury | Mar 15, 2019
Emerging Company Profile

Neurogene: Genes over ERT for monogenic CNS diseases

Neurogene is developing disease-modifying gene therapies for two monogenic, pediatric disorders in which enzyme replacement is not feasible or hasn't been explored. Aspartylglucosaminuria (AGU) is a lysosomal storage disorder caused by autosomal recessive AGA mutations...
BC Innovations | Sep 28, 2018
Emerging Company Profile

GDF11 against aging

Harvard University spinout Elevian Inc. is developing a regenerative platform around GDF11 to treat age-related diseases, including Alzheimer's disease. The company, which emerged from stealth on Sept. 6, aims to modulate the protein’s pathway with...
BioCentury | Sep 14, 2018
Regulation

Closing NICE’s Orphan gap

As the list of Orphan drugs rejected by NICE grows, industry and patient groups are pressuring the agency to make broader use of tools that currently apply only to selected ultra-Orphan therapies. These tools allow...
BC Week In Review | Aug 31, 2018
Clinical News

Pfizer ends Phase II trials of DMD candidate

Pfizer Inc. (NYSE:PFE) terminated two Phase II trials of domagrozumab (PF-06252616) to treat Duchenne muscular dystrophy after one of the studies missed its primary endpoint. The company will evaluate the data to determine next steps...
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