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CRISPR, Capsida team up on gene-edited therapies for ALS, Friedreich's ataxia firstwordpharma
June 16, 2021
CRISPR Therapeutics has inked a partnership with newly launched Capsida Biotherapeutics to develop and commercialise in vivo gene-editing therapies delivered with engineered adeno-associated virus (AAV) vectors for the treatment of familial amyotrophic
Retrotope Announces First Dosing of RT001 in Friedreich’s Ataxia Study americanpharmaceuticalreview
January 15, 2020
Retrotope has dosed its first patient in a Phase 2/3 clinical trial of RT001 in Friedreich’s ataxia, the most common of the inherited ataxias. RT001, a stabilized fatty acid drug, has been shown to reduce lipid peroxidation leading to cell death in ...