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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.
Palvella Therapeutics Inc is a late clinical-stage biopharmaceutical company committed to serving individuals suffering from serious, rare genetic skin diseases without approved therapies. The Company's lead product candidate, QTORIN 3.9 percent rapamycin anhydrous gel (QTORIN rapamycin), is based on the Company's patented QTORIN platform. QTORIN rapamycin is in clinical development for two rare genetic skin disorders.