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- Novartis' RNA drug del-brax hits primary endpoint in early-stage FSHD trialNovartis reported that its antisense oligonucleotide conjugate, del-brax, achieved the primary biomarker endpoint in a Phase 1/2 study of patients with facioscapulohumeral muscular dystrophy (FSHD)…
- Sensorion drops OTOF gene therapy to focus on GJB2-targeted hearing loss candidate after Regeneron approvalSensorion said it is ending development of its gene therapy SENS-501 for OTOF related hearing loss after a strategic review. The review concluded that the competitive landscape had changed signific…