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- myTomorrows and Rare Disease Research announced a partnership to improve patient identification and referrals into rare disease clinical trials through structured pathways. - The collaboration will initially support RDR sites in Georgia, Florida, and North Carolina, using the myTomorrows platform to enable structured referrals from community and specialist physicians. - The partnership aims to reduce administrative burden for trial sites while creating smoother experiences for families and referring providers through centralized workflows. - myTomorrows has previously helped more than 17,700 patients and 3,000 physicians across 440+ sites in over 135 countries access pre-approval treatment options.
- Epicrispr Biotechnologies has dosed the first patient in a global first-in-human clinical trial of EPI-321, an investigational one-time epigenetic editing therapy for facioscapulohumeral muscular dystrophy (FSHD). - EPI-321 is the first investigational therapy designed to silence DUX4 expression via epigenetic modulation, targeting the root cause of FSHD which affects approximately 1 in 8,000 individuals worldwide. - The therapy has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations, with initial data expected in early 2026. - FSHD is a progressive genetic disease leading to skeletal muscle degeneration and severe loss of function, with currently no approved disease-modifying therapies available.
