AAV gene therapy

Tuberous sclerosis (TSC) 1/2

estimated prevalence



Tuberous sclerosis (TSC) 1/2

Genetic Source


Clinical Phase



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Tuberous sclerosis complex (TSC) is a genetic disease caused by a mutation in either the TSC1 or TSC2 gene. TSC encompasses significant symptoms across multiple organs, including refractory seizures. BBP-821 and BBP-822 are investigational adeno-associated virus (AAV) gene therapies to treat TSC due to a mutation in the TSC1 gene or the TSC2 gene. The AAV gene therapy is designed to deliver, through systemic administration, a functional copy of the TSC1 or TSC2 gene, and has shown through multiple animal studies to result in efficient and persistent delivery to the brain. If successful, the goal is to restore a working copy of the aberrant gene to the central nervous system, enabling people with mutations in those genes to make functional proteins, something that is not possible with any treatment on the market or currently in clinical trials for TSC1 or TSC2. IND submission is expected in 2025.

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