Capsida Biotherapeutics (“Capsida”) today announced it will present preliminary data on the company’s next-generation engineered adeno-associated virus (AAV) capsids in non-human primates.
Preliminary data show next-generation engineered capsids in non-human primates delivered systemically resulted in widespread transduction in up to 68% of neurons across multiple brain regions Data to be showcased during industry-sponsored symposium about unlocking the potential of gene therapy with panel featuring academic and industry luminaries in gene therapy Poster presentations highlight optimization of process and analytical methods for engineered AAV capsids THOUSAND OAKS, Calif., May 10, 2023 /PRNewswire/ -- Capsida Biotherapeutics (“Capsida”) today announced it will present preliminary data on the company’s next-generation engineered adeno-associated virus (AAV) capsids in non-human primates. The initial data showed that Capsida’s engineered AAV capsids, when delivered intravenously at a clinically relevant dose, resulted in widespread transduction including protein production across multiple brain regions including cortex, thalamus, and putamen, in up to 68% of neurons. Importantly, liver transduction was reduced five-fold compared to wild-type AAVs. Capsida will present these data at an industry-sponsored symposium on Friday, May 19, 2023 at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023 in Los Angeles. In addition, Capsida will present several posters that highlight optimization of process and analytical methods for engineered AAV capsids as part of the ASGCT meeting. “These data demonstrate the potential of our gene therapy platform to deliver targeted non-invasive therapies to the central nervous system to potentially treat more common diseases beyond the ultra-rare indications to which wild-type AAVs have been largely relegated,” said Peter Anastasiou, Capsida’s Chief Executive Officer. “This potential was demonstrated through broad neuronal transduction and protein expression with significantly less exposure to the liver.” “This capsid performance represents the potential to achieve a significantly increased therapeutic index,” said Susan Catalano, Chief Scientific Officer of Capsida. “It also showcases Capsida’s multi-dimensional capsid engineering platform that optimizes the entire drug product, including specifically engineered tissue tropism and de-targeting, immune evasion, and manufacturability.” Industry Symposium—Unlocking the Potential of Gene Therapy: A Targeted Next-Generation Platform In addition to presentation of the preliminary data on Capsida’s capsids delivered systemically in non-human primates, topics will examine the promise of next-generation AAV gene therapy; noninvasive administration of AAV to treat central nervous system (CNS) disorders; and advancing gene therapies for age-related neurodegenerative diseases. Posters Development and Comparative Assessment of a Rapid, High-Throughput Method for Accurate Quantification of Host Cell DNA Impurities in Final Drug Product and Process Intermediates Optimization of a Versatile Downstream Process for Multiple Novel AAV Capsids That Demonstrates Improved Recovery Exploring Upstream Process Conditions to Improve Productivity of Novel AAV Capsids Find out more information about the posters here. Career Fair About Capsida Biotherapeutics
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