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news.The US Food and Drug Administration (FDA) has approved Repligen to commence double-blind RG3039 Phase 1 clinical trial for spinal muscular atrophy (SMA).
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RG3039, an orally bioavailable compound, is an inhibitor of an RNA processing enzyme which targets SMN2.
The trial is designed to investigate the safety and pharmacokinetic of escalating doses of RG3039 in 40 healthy subjects.
In prior studies, RG3039 has demonstrated increase in production of SMN protein in cells derived from patients and has improved mobility and lifespan.
Repligen president and CEO Walter Herlihy said RG3039 targets the core deficit of SMA and if this therapeutic approach is successful, it has the potential to arrest or slow disease progression and significantly improve patients’ lives.