A new engineered virus can deliver gene therapy to the brain 2,000 times more effectively than current methods.
April 1, 2026
Original Paper
Novel Engineered AAV Variants Demonstrate Superior Blood-Brain Barrier Penetration and Safety in Non-Human Primates
bioRxiv · 10.64898/2026.03.29.713052
The Takeaway
Delivery to the brain is the biggest bottleneck in treating neurological diseases. This study reports a massive leap in performance, achieving unprecedented brain penetration while simultaneously reducing toxic side effects in the liver by 50-fold, potentially solving the delivery problem for brain gene therapy.
From the abstract
Systemic delivery of adeno-associated virus (AAV) for gene therapy of central nervous system (CNS) disorders is limited by inefficient blood-brain barrier (BBB) penetration and dose-limiting toxicity in peripheral organs, notably the liver and dorsal root ganglia (DRG) . Here, we report the development of novel AAV variants via a proprietary capsid engineering platform (REACH). In non-human primates (NHPs), intravenous administration of lead variants resulted in transgene expression levels in th