Repligen claims that RG3039 is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.
The goal of this grant is to support the advancement of RG3039 into human clinical testing, including continued evaluation in preclinical models of SMA, GMP manufacturing for human clinical trials and early clinical evaluation in healthy volunteers and patients.
Repligen president and CEO Walter Herlihy said they plan to complete preclinical studies and advance RG3039 into human clinical testing in 2011.