Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR Alnylam Pharmaceuticals, Inc. , a leading RNAi therapeutics company, announced today that it offers initiated a pilot Stage II study with ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin gene in advancement for the treatment of TTR-mediated amyloidosis . The Phase II trial, which is open for enrollment now, is aimed at evaluating the tolerability and preliminary medical activity of ALN-TTRsc in TTR cardiac amyloidosis sufferers with familial amyloidotic cardiomyopathy – which is caused by autosomal dominant mutations in the TTR gene, or senile systemic amyloidosis – which is caused by idiopathic accumulation of wild-type TTR in the heart orgasme .
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