The financing round was jointly led by new investor Regeneron Ventures and current investor Deerfield Management.

The trial aims to assess the safety, pharmacodynamics, efficacy, and pharmacokinetics of the therapy in nearly 255 subjects.

The CW News at 10pm ...

Ocugen has a valuable IP portfolio, which should give it funding options. OCGN’s cash runway remains the main risk. Check out ...

Senate Republicans released their version of the Trump administration’s “One Big Beautiful Bill Act” on Monday with a few ...

Global Phase 3 BRAVE study will evaluate the efficacy and safety of omaveloxolone in children 2 to <16 years old with Friedreich ataxia, a rare neurodegenerative disorder BRAVE study will explore the ...

Medicovestor Inc.’s Adobind MC-001 has been awarded orphan drug designation by the FDA for the treatment of pancreatic cancer. Adobind MC-001 is a chemoimmunotherapy antibody-drug conjugate that ...

Eli Lilly and Co. is buying Verve Therapeutics Inc. and its gene-editing program for about $1.3 billion. Two of the one-time treatments are in the clinic. Lead candidate VERVE-102, a gene-editing ...

The biopharma industry has hit a setback in its ambition to leverage President Donald Trump’s “big, beautiful bill” to amend ...

Capricor Therapeutics (NASDAQ:CAPR) received the Orphan Drug designation from the U.S. FDA for its lead cell therapy candidate, Deramiocel, to treat becker muscular dystrophy. With its orphan drug ...

ABOUT ORPHAN DRUG DESIGNATION The FDA grants Orphan Drug Designation to investigational drugs intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. Benefits ...