WALTHAM, Mass.--(BUSINESS WIRE)--Repligen Corporation (NASDAQ: RGEN) announced today that it has received approval from the Food and Drug Administration (FDA) to initiate a Phase 1 clinical trial of RG3039, a potential treatment for Spinal Muscular Atrophy (SMA). SMA is an inherited neurodegenerative disease in which a defect in the SMN1 (“survival motor neuron”) gene results in low levels of the protein SMN and leads to progressive damage to motor neurons, loss of muscle function and, in many patients, early death. This is a double-blind study to evaluate the pharmacokinetic and safety profile of escalating doses of RG3039 in up to 40 healthy volunteers. This will be the first clinical trial of a novel drug specifically designed to treat SMA and the first treatment approach which seeks to increase levels of the protein SMN.
