SRP-1007

SRP-1007 is an investigational small interfering RNA (siRNA) therapeutic being developed by Sarepta Therapeutics for the treatment of Spinocerebellar Ataxia Type 1 (SCA1). SCA1 is a progressive, autosomal dominant neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the *ATXN1* gene, which leads to the production of a toxic mutant ataxin-1 protein. SRP-1007 is designed to utilize the RNA interference (RNAi) pathway to specifically target and degrade *ATXN1* messenger RNA (mRNA) transcripts. By reducing the expression of the disease-associated transcript, the therapy aims to lower the levels of the toxic protein and potentially slow or halt the progression of the disease. The program is currently in the preclinical stage of development.