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Avidity Biosciences, Inc. (Nasdaq: RNA) Avidity Biosciences, Inc., a biopharmaceutical company pioneering RNA therapeutics, shared positive initial data from the Phase 1/2 FORTITUDE? trial of AOC 1020 (delpacibart braxlosiran, del-brax). The data show unprecedented and consistent reductions of over 50% in DUX4 regulated genes, functional improvement trends, and favorable safety in people with facioscapulohumeral muscular dystrophy (FSHD). This has led Avidity to expedite the initiation of registrational cohorts in the FORTITUDE? study.
FSHD, a rare hereditary disorder, causes progressive muscle function loss, significant pain, fatigue, and disability. Del-brax is the first investigational therapy targeting DUX4, the abnormal gene expression causing FSHD. Currently, no approved therapies exist for FSHD treatment.
Key findings from the FORTITUDE trial include:
Over 50% mean reductions in DUX4 regulated genes.
Mean reductions of 25% or more in circulating biomarkers and creatine kinase.
Trends of functional improvements in muscle strength and function.
Favorable safety with mild or moderate adverse events and no serious adverse events.
Dr. Jeffrey M. Statland from the University of Kansas Medical Center highlighted the potential of del-brax to change the course of FSHD by improving muscle strength and function. The data will be presented at the 31st Annual FSHD Society International Research Congress in June 2024.
Sarah Boyce Sarah Boyce, CEO of Avidity, emphasized the urgency of developing del-brax as a first-in-class therapy for FSHD, showcasing the potential of their AOC platform to revolutionize RNA therapeutics.
Mark Stone, CEO of FSHD Society, expressed hope that del-brax could offer significant relief for those affected by FSHD.
#Biotech #RNAtherapeutics #FSHD #ClinicalTrials #AvidityBiosciences #MedicalResearch #HealthcareInnovation #RareDisease #DrugDevelopment #Pharmaceuticals #RNA #AntibodyOligonucleotideConjugates
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