PART

Gene therapy products, namely, biopharmaceutical preparations primarily composed of adeno-associated virus (AAV) vectors comprising of a transgene or other nucleic acid payload for delivery to a cell or tissue; biopharmaceutical preparations, namely, biopharmaceutical preparations primarily composed of adeno-associated virus (AAV) vectors comprising a transgene or other nucleic acid payload for delivery to a cell or tissue; biological preparations; therapeutic biopharmaceutical products for medical and clinical gene therapy; all of the above for the treatment of genetic diseases, conditions and disorders primarily related to the muscle or central nervous system

Scientific and medical research and development services in the field of treatment of rare genetic diseases, conditions and disorders primarily related to the muscle or central nervous system