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Abeona Therapeutics Inc is a clinical-stage biopharmaceutical company. The firm is focused on developing gene therapies and plasma-based products for life-threatening rare genetic diseases. Its clinical programs consist of pz-cel, an autologous, engineered cell therapy currently in development for recessive dystrophic epidermolysis bullosa (RDEB). The Company's development portfolio also features adeno-associated virus (AAV)-based gene therapies designed to treat unmet, medically needed ophthalmic diseases using the novel.
Capricor Therapeutics Inc is a clinical-stage biotechnology company focused on the development of transformative cell and exosome-based therapeutics for treating Duchenne muscular dystrophy (DMD), a rare form of muscular dystrophy which results in muscle degeneration and premature death, and other diseases with high unmet medical needs. The company's product pipeline includes CAP-1002, deramiocel an allogeneic cardiac-derived cell therapy. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Through its proprietary StealthXTM platform, the group uses its exosome technology to conduct preclinical research focused on vaccinology, delivering oligonucleotides, proteins, and small molecules to treat.