AAV.SPR

AAV.SPR is an investigational adeno-associated virus (AAV) gene therapy developed by Asklepios BioPharmaceutical (AskBio), a subsidiary of Bayer, for the treatment of Sepiapterin Reductase (SPR) deficiency. SPR deficiency is a rare autosomal recessive neurometabolic disorder caused by mutations in the *SPR* gene, leading to a shortage of the SPR enzyme. This enzyme is critical for the final step of tetrahydrobiopterin (BH4) biosynthesis, which is a mandatory cofactor for the enzymes that produce dopamine and serotonin. AAV.SPR delivers a functional copy of the human *SPR* gene to the brain, aiming to restore endogenous enzyme production and normalize neurotransmitter levels, thereby improving motor function and developmental outcomes in affected individuals.

Other names

Sepiapterin reductase gene therapy