Capsida Biotherapeutics Presents Data Demonstrating Unique Industry-Leading Technology and Capabilities With Potential to Treat Both Rare and Common Diseases at the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Platform utilizes combinatorial engineering and automation to screen in high-throughput, generating capsids greatly improved over AAV9 in CNS efficiency and specificity

Process development and manufacturing data validate in-house capability to produce highly productive and scalable processes up to 200L

THOUSAND OAKS, Calif., May 18, 2022 – Capsida Biotherapeutics (“Capsida”), the only fully integrated gene therapy platform company that through intravenous (IV) delivery of a single, engineered capsid can target single or multiple organs simultaneously while limiting exposure to non-targeted organs, today announced four posters delivered at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. Capsida’s approach unlocks the potential to treat both rare and common diseases across all ages.

Capsida’s presentations detail advancements from the Company’s biologically driven, high-throughput, automated adeno-associated virus (AAV) engineering platform, and innovative process development and manufacturing capabilities. The ASGCT Annual Meeting is being held virtually and in person May 16-19, 2022, in Washington, D.C. at the Walter E. Washington Convention Center.

“Capsida’s high-throughput method for simultaneously enriching target tissues and avoiding off-target tissues demonstrates organ-level and cell-type specificity,” said Nick Goeden, Ph.D., co-founder and chief technology officer, Capsida. “By leveraging our automated capsid engineering platform, which is capable of processing >20,000 samples annually, we have greatly expanded our ability to interrogate tissues and rapidly engineer improved capsids across therapeutic areas. The totality of these data, along with achievements in our process development and manufacturing processes, will allow us to rapidly advance gene therapies into the clinic.”

Poster Presentations:

Combinatorial Engineering Across Multiple Surface Exposed Loops of AAV2 and AAV9 Yields Capsids With High Degrees of Enrichment and Specificity for Target Tissues
Date: Wednesday, May 18, 2022, 5:30-6:30 PM ET
Session: AAV Vectors – Virology and Vectorology III
Abstract Number: 905
Location: Hall D
Presenter: Xiaojing Shi, Ph.D., scientist II, AAV engineering, Capsida

Many central nervous system (CNS) indications require broad delivery of genetic therapies throughout the brain and/or spinal cord. Intravenous delivery of engineered capsids capable of crossing the blood–brain-barrier is well-suited to achieve broad and uniform transduction of the CNS. However, there are several ongoing clinical trials focused on CNS or muscle therapeutic areas that have observed severe adverse events related to liver toxicity due to the high viral burden of wildtype AAV serotypes in the liver. This research employed Capsida’s combinatorial engineering strategy to evolve novel AAV9- and AAV2-based capsids that are simultaneously enriched in the CNS and detargeted in the liver following IV administration in non-human primates (NHPs), thereby significantly improving therapeutic potential. Following multiple rounds of screening in NHPs, Capsida identified lead candidates with >100-fold enrichment in the CNS and >10-fold detargeting in the liver relative to the parent capsid.

Next-Generation Automated AAV Engineering Platform for Rapid Identification of Efficient and Specific Capsids in Non-Human Primates
Date: Wednesday, May 18, 2022, 5:30-6:30 PM ET
Session: AAV Vectors – Virology and Vectorology III
Abstract Number: 900
Location: Hall D
Presenter: Troy Sandberg, Ph.D., principal scientist, AAV engineering and automation, Capsida

To bypass the challenges of translating capsids engineered in vitro or in lower-order species while still leveraging the success of directed evolution in a high-throughput and high-capacity manner, Capsida has developed an automated AAV engineering platform capable of interrogating targets throughout the body, directly in NHPs and human cell lines at a rapid pace. This platform has generated several iterations of improved capsids for targets in the CNS and is now being leveraged for targets throughout the body. In addition to increased capacity and throughput, the automated engineering platform has simultaneously improved data quality by increasing the volume of interrogated primate tissues by >400 percent year-over-year, enabling greater predictive analysis in the bioinformatics platform and more rapid identification of improved capsids.

Manufacturability and Product Quality of Novel AAV Capsids Optimized for Specific Targeting Using Capsid Library Selection in Non-Human Primates
Date: Wednesday, May 18, 2022, 5:30-6:30 PM ET
Session: Vector Product Engineering, Development, or Manufacturing III
Abstract Number: 1147
Location: Hall D
Presenter: Laura Adamson, Ph.D., executive director, process and analytical development, Capsida

In this research, Capsida evaluated engineered capsids with multiple capsid insertions during single variant production and demonstrated improved productivity in a pooled library. The Company compared productivity across multiple production platforms, demonstrated scalability of these novel engineered capsids containing a therapeutic gene in Capsida’s suspension-based manufacturing platform, and determined product quality of therapeutic transgenes packaged in novel capsids. Capsid variants selected from Capsida’s high-throughput AAV engineering screening platform not only show improved brain enrichment, but also are capable of generating high titer and high-quality products in a suspension-based scalable production platform. The interface between Capsida’s in-house process development and capsid engineering platform allows for seamless identification of manufacturable, high-performing AAV capsids.

Optimization of a Scalable Upstream Process for Novel AAV Capsids
Date: Wednesday, May 18, 2022, 5:30-6:30 PM ET
Session: Vector Product Engineering, Development, or Manufacturing III
Abstract Number: 1153
Location: Hall D
Presenter: Hari Acharya, Ph.D., associate director, upstream development, Capsida

The development of novel capsids possessing more efficient gene delivery capabilities is the next evolution in recombinant AAV (rAAV)-based therapeutics. The objective of this work was to develop a scalable transient transfection process for producing our novel capsids in suspension culture. To meet clinical demands, scalable production processes are needed. The work presented here focuses on developing a scalable transient transfection process using HEK293 cells in suspension and scaling to 200L. These results demonstrate that shaker flasks are a viable method for screening transfection parameters and establishing ranges, that screening work can identify conditions and reagents yielding higher titers, and finally, that productivity is similar all the way across from shaker flasks to 200L bioreactors. Capsida has a scalable production process for our novel capsids and performs as expected at 200L, preparing us for future clinical demand.

Abstracts can be accessed via the conference website at annualmeeting.asgct.org.

About Capsida Biotherapeutics

Capsida Biotherapeutics is the only fully integrated gene therapy platform company that through IV delivery of a single engineered capsid can target single or multiple organs simultaneously, while limiting exposure to non-targeted organs. Capsida’s approach unlocks the potential to treat both rare and common diseases across all ages. Capsida’s technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of rare and more common genetic and sporadic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida’s technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development expertise. Capsida’s biologically driven, high-throughput AAV engineering and cargo optimization 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.

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