Repligen Initiates Phase 1b Trial of RG3039 for Spinal Muscular Atrophy Licensed from Families of SMA
Elk Grove Village, IL. September 26, 2012. Repligen Corporation has initiated a Phase 1b clinical study of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA). The primary objectives of the study are to further evaluate the safety and plasma pharmacokinetics (PK) of multiple doses of RG3039 in healthy volunteers. Repligen licensed RG3039 in 2009 from Families of Spinal Muscular Atrophy (FSMA). FSMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million. This was the first drug