AD/PD 2023 | Novel brain penetrant capsids for Parkinson’s disease gene therapy

Gene therapies hold great potential in neurology, but the limitations of current viral vectors are hampering progress. Todd Carter, PhD, Chief Scientific Officer at Voyager Therapeutics, Inc., Cambridge, MA, discusses ongoing work at Voyager Therapeutics to develop novel adeno-associated virus (AAV)-based vectors that penetrate the blood-brain-barrier, achieve widespread transgene expression, and limit toxicity attributable to off-target effects. The team have identified capsids that can be delivered intravenously with enhanced tropism for the central nervous system, leading to substantial increases in transgene expression across a range of brain regions, compared to conventional AAV serotypes. It is hoped that these novel capsids could be used for gene replacement, gene silencing, and delivery of vectorized antibody payloads. Dr Carter highlights ongoing work to develop gene therapy targeting GBA1 using the novel capsid technology. This interview took place at the AD/PD™ 2023 congress in Gothenburg, Sweden.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.