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Rocket Pharmaceuticals Inc is a late-stage biopharmaceutical company. It is focused on the development of gene therapies for rare and devastating pediatric diseases. The pipeline products of the company include RP-A501 for Danaon Disease, RP-L102 for Fanconi Anemia, RP-L201 for Leukocyte Adhesion, RP-L301 for Pyruvate Kinase Deficiency, and RP-L401 for Infantile Malignant Osteopetrosis. The Company has one reportable segment related to R&D and commercial readiness of its gene therapies.
Mereo BioPharma Group PLC is a United Kingdom (U.K.) based biopharmaceutical company focused on the development of therapeutics for rare diseases. The company has developed a portfolio of late-stage clinical product candidates, and its two rare disease product candidates are setrusumab for the treatment of osteogenesis imperfecta (OI) and alvelestat primarily for the treatment of severe alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD).