SRP-1006

SRP-1006 is an investigational small interfering RNA (siRNA) therapy being developed by Sarepta Therapeutics for the treatment of Spinocerebellar Ataxia Type 3 (SCA3), also known as Machado-Joseph disease. SCA3 is a progressive, hereditary neurodegenerative disorder caused by a CAG repeat expansion in the *ATXN3* gene, which leads to the accumulation of toxic, misfolded ataxin-3 protein in the brain. SRP-1006 is designed to utilize the RNA interference (RNAi) pathway to specifically target and degrade disease-associated *ATXN3* messenger RNA (mRNA) transcripts. By reducing the expression of the *ATXN3* gene, the therapy aims to decrease the production of the mutant protein and potentially slow or halt the progression of the disease. SRP-1006 is currently in the preclinical stage of development.