Last edition
News about "rare genetic disorders"
AskBio Doses First Participant in Phase 1/2 Trial for Pompe Disease Gene Therapy
AskBio has dosed the first participant in its Phase 1/2 PROGRESS-GT LOPD trial evaluating investigational gene therapy AB-1009 for late-onset Pompe disease, advancing research into potential long-term treatment solutions for the rare genetic disorder.
Rare Genetic Disorders | 14/05/2026 | By News Bureau
THX Pharma and Biocodex Sign Licensing Deal for Rare Disease Drug Candidates
Biocodex secures global rights to Batten-1 and North American rights to TX01 from THX Pharma in a deal worth up to Euro 173 million to advance treatments for juvenile Batten disease, Gaucher disease and Niemann-Pick type C.
Rare Genetic Disorders | 13/02/2026 | By News Bureau | 176
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