Eleven COVID-19 patients discharged after receiving Ascletis drug  Ascletis Pharma Inc. (HKEX:1672) said all 11 COVID-19 patients in a cohort receiving Ganovo danoprevir plus ritonavir in a Chinese Phase IV trial have been discharged from...

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...

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...

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...

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...

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%...

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....

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...

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...

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...