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Repligen Reports Positive Results From Phase 1 Clinical Trial Of RG3039 For Spinal Muscular Atrophy

In addition to the presentation at AAN, Dr. David Jacoby, Medical Director at Repligen Corporation, will present the RG3039 Phase 1 study outcomes during the 2012 International SMA Research Group Meeting. This FSMA organized meeting is being held in Minneapolis from June 21-23, 2012. Dr. Jacoby’s presentation titled, “A Phase I Study in Healthy Volunteers to Assess the Safety, Pharmacokinetics and Pharmacodynamics of the DcpS inhibitor RG3039,” is scheduled for Friday, June 22, 2012 at 3:40 p.m.

About Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive neuromuscular 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. It is the leading cause of infant mortality and the second most common inherited neuromuscular disease, with symptoms that typically emerge before the age of two. SMA is characterized by progressive muscle weakness leading to severe physical disability and often, loss of life due to respiratory insufficiency. The prevalence of SMA in the U.S. and EU is approximately 20,000 patients.

Orphan Drug and Fast Track Designations

Repligen has previously received U.S. Orphan Drug and Fast Track designations for RG3039 for the treatment of SMA, as well as Orphan Medicinal Product designation in the EU. Fast Track is a process designed to facilitate the development and expedite the review of drugs that have the potential to treat serious disease and fill an unmet medical need. The orphan programs provide incentives for the research, development and marketing of products intended to diagnose, prevent or treat rare conditions and/or serious or debilitating diseases with unmet medical needs. Orphan designation grants the sponsor exclusive marketing rights for seven years in the U.S. and ten years in the EU following regulatory approval of the designated product. In the U.S., rare diseases are defined as those affecting fewer than 200,000 Americans. In the U.S., rare diseases are defined as those affecting fewer than 200,000 Americans. In the EU, rare diseases are considered those that affect no more than five per ten thousand persons in the community.

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