Obtained ex-US Regulatory Clearance for DT-818, a Potentially Best-in-Disease Treatment for Myotonic Dystrophy Type-1 (DM1) Trials of DT-216P2 in Friedreich Ataxia (FA) and DT-168 in Fuchs Endothelial ...
Almost a year after buying Kate Therapeutics, Novartis has scooped up another San Diego-based muscle dystrophy biotech in one of the biggest acquisitions of 2025 so far. The Swiss pharma is paying $12 ...
Dyne Therapeutics received positive FDA feedback enabling a registrational cohort for z-basivarsen in DM1, with Accelerated Approval possible using vHOT as intermediate efficacy endpoint. DYN expects ...
– 53.7% mean splicing correction observed following a single 15 mg/kg dose of PGN-EDODM1, with all patients showing an improvement in splicing – – PGN-EDODM1 was generally well-tolerated at 15 mg/kg, ...
SAR446268 is a one-time adeno-associated viral gene therapy that delivers a vectorized RNA interference to silence DMPK expression. The Food and Drug Administration (FDA) has granted Fast Track ...
Designation earned for one-time AAV gene therapy SAR446268, designed to silence DMPK expression Myotonic dystrophy type 1 (DM1) is a rare, genetic disorder that causes progressive muscle weakness and ...
- Extension financing led by new investor Bpifrance, with renewed participation from all existing shareholders, AdBio Partners, CDTI Innovación, Columbus Venture Partners, European Innovation Council ...
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