Core Insights - Design Therapeutics, Inc. has appointed Justin Gover to its Board of Directors, effective immediately, bringing over 25 years of biotechnology leadership experience [1][2] - Gover's previous role as founding CEO of GW Pharmaceuticals included guiding the company through a $7 billion acquisition by Jazz Pharmaceuticals and the successful commercialization of Epidiolex® [2] - Dr. Arsani William has stepped down from the Board, having provided strategic counsel since 2021, including support for the company's IPO and clinical pipeline advancement [3] Company Overview - Design Therapeutics is a clinical-stage biotechnology company focused on developing GeneTAC gene-targeted therapies aimed at addressing serious degenerative genetic diseases [5] - The company's GeneTAC molecules are designed to modulate the expression of disease-causing genes, with ongoing clinical programs for Friedreich ataxia (DT-216P2), Fuchs endothelial corneal dystrophy (DT-168), myotonic dystrophy type-1, and Huntington's disease [5] - Discovery efforts are also underway for multiple genomic medicines, indicating a broad pipeline potential [5] Leadership Insights - Justin Gover's expertise in translating innovative science into medicines is expected to be invaluable for advancing Design's clinical pipeline [4] - Dr. William expressed pride in the progress made during his tenure, particularly in advancing the GeneTAC platform and establishing a transformative pipeline for genetic diseases [4] - Gover's involvement with other biotech boards and nonprofit organizations highlights his commitment to the industry and patient advocacy [4]

Core Insights - Jazz Pharmaceuticals announced improved outcomes for adults with narcolepsy or idiopathic hypersomnia treated with Xywav, as demonstrated in the Phase 4 DUET study [1][2] - The company is committed to addressing neurological disorders with limited treatment options, emphasizing the importance of ongoing data generation for Xywav and Epidiolex [3] Xywav Treatment Insights - The Phase 4 DUET trial showed statistically significant improvements in Epworth Sleepiness Scale (ESS) scores, reduced sleep stage shifts, increased deep sleep, and fewer awakenings in adults with narcolepsy treated with Xywav [2] - In adults with idiopathic hypersomnia, Xywav treatment also resulted in improvements in ESS and Idiopathic Hypersomnia Severity Scale scores [2] - Patient-reported data indicated that Xywav treatment was associated with improvements in daytime symptoms and cognitive complaints among adults with narcolepsy or idiopathic hypersomnia [2] Epidiolex Treatment Insights - A novel analysis of real-world Epidiolex treatment patterns revealed a nearly 70% (69.9%) probability of persistence at one year among new patients, highlighting the importance of dose optimization [5] - The analysis indicated that 52% of patients were taking dosages greater than 15 mg/kg/day at 12 months, with those on average dosages greater than 20 mg/kg/day having the lowest likelihood of discontinuation [5] Upcoming Presentations - Jazz Pharmaceuticals will present seven abstracts at the 2025 AAN Annual Meeting, focusing on the effectiveness and safety of Xywav and real-world data on Epidiolex [1][4]