The gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) is gearing up to unveil initial data on its engineered adeno-associated virus (AAV) capsids at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16–20 in Los Angeles.
In developing these capsids, Capsida Chief Scientific Officer Susan Catalano explained that the company employed directed evolution and a high-throughput screening platform to optimize capsid performance across key domains. “We start with a disease and aim to create the best possible therapy, as opposed to trying to mold a disease profile around the limitations of wildtype capsids,” Catalano said.
Preliminary results suggest these engineered AAV capsids can reach up to 68% of neurons across multiple brain regions when delivered intravenously. That finding could translate to new possibilities in gene therapies targeting common neurological diseases that have…