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ProQR Therapeutics NV is a biopharmaceuticals company. It discovers and develops RNA therapeutics for patients with severe genetic rare diseases such as cystic fibrosis, Leber's congenital amaurosis type 10 and dyastrophic epidermolysis bullosa.
Taysha Gene Therapies Inc is a patient-centric gene therapy company to eradicate monogenic CNS disease. It is focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. The company develops and commercializes transformative gene therapy treatments. Its product pipeline includes TSHA-102 which is a is a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome.