Capsida Biotherapeutics to Present New Data Showing Increased Capsid Expression in CNS at American Society of Gene & Cell Therapy 26th Annual Meeting

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) 26^th 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 
The company is hosting an industry-sponsored symposium entitled, "Unlocking the Potential of Gene Therapy: A Targeted Next Generation Platform." The event will include presentations by senior leaders from Capsida, AbbVie, and Lilly's Prevail Therapeutics, and a panel featuring industry and academic luminaries Aravind Asokan, Ph.D., Viviana Gradinaru, Ph.D., and Katherine High, M.D. The symposium will take place on Friday, May 19, 2023, from 12:00 p.m. – 1:30 p.m. PT, Room 511.

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 
Capsida will present three posters that highlight the optimization of process and analytical development methods for engineered AAV capsids.

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 
Abstract Number: 787 
Session: Wednesday Poster Session 
Presenter: Jesse Granados, Capsida Biotherapeutics 
Date/Time: Wednesday, May 17, 2023; 12:00 p.m. – 2:00 p.m. PT

Optimization of a Versatile Downstream Process for Multiple Novel AAV Capsids That Demonstrates Improved Recovery 
Abstract Number: 1169
Session: Thursday Poster Session
Presenter: Sophia Petraki, Capsida Biotherapeutics 
Date/Time: Thursday, May 18, 2023; 12:00 p.m. – 2:00 p.m. PT

Exploring Upstream Process Conditions to Improve Productivity of Novel AAV Capsids 
Abstract Number: 1673 
Session: Friday Poster Session
Presenter: Hari Acharya, Ph.D., Capsida Biotherapeutics 
Date/Time: Friday, May 19, 2023; 12:00 p.m. – 2:00 p.m. PT

Find out more information about the posters here.

Career Fair 
Capsida will also participate in the ASGCT career fair for those interested in learning more about the company and potential job opportunities. Visit our table and learn about exciting opportunities to be a part of our DNA. The fair will be held from May 17-19 between 3:15–5:15 p.m. on the Concourse Foyer.

About Capsida Biotherapeutics 
Capsida Biotherapeutics is a fully integrated next-generation gene therapy platform company. Capsida's approach unlocks the potential to treat both rare and common diseases across all ages. We create customized therapies that selectively target specific organ systems and simultaneously limit exposure to non-targeted organs. The company has wholly owned programs in central nervous system (CNS) and strategic collaborations with AbbVie (CNS and eye care), Lilly (CNS), and CRISPR Therapeutics (CNS), providing independent validation of Capsida's capabilities. Capsida is backed by Versant Ventures and Westlake Village BioPartners. Its platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. Visit us at www.capsida.com to learn more.

SOURCE Capsida Biotherapeutics