Capsida Biotherapeutics Advances Novel Gene Therapies for CNS Disorders Ahead of ASGCT 2025
Key Insights
Capsida Biotherapeutics (search) will present seven scientific presentations at ASGCT 2025, showcasing progress on their engineered AAV gene therapies targeting neurological disorders.
The company's lead candidate CAP-002 for STXBP1-DEE (search) demonstrated widespread brain expression in non-human primate studies, with potential to correct seizures, motor abnormalities, and developmental disabilities.
Capsida is on track to initiate clinical trials for both CAP-002 (STXBP1-DEE (search)) and CAP-003 (Parkinson's disease (search) with GBA (search) mutations) programs in the first half of 2025.
Capsida Biotherapeutics (search) has announced an extensive lineup of scientific presentations for the upcoming 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), scheduled for May 13-17, 2025, in New Orleans. The Thousand Oaks, California-based company will deliver seven presentations, including three oral sessions and four posters, highlighting significant advances in their proprietary gene therapy platform.
Breakthrough CNS-Targeted Gene Therapies
At the core of Capsida's presentations are promising updates on their intravenously (IV) administered gene therapies designed to cross the blood-brain barrier (search) while minimizing off-target effects in peripheral tissues. The company's engineered adeno-associated virus (AAV) capsids enable targeted delivery to the central nervous system (CNS), potentially overcoming a major challenge in treating neurological disorders.
Nick Flytzanis, PhD, Capsida's founder and Chief Research and Innovation Officer, will present data on CAP-002, the company's lead candidate for STXBP1 (search) Developmental and Epileptic Encephalopathy (STXBP1-DEE (search)). The presentation will showcase non-human primate GLP toxicology results demonstrating that CAP-002 achieves widespread STXBP1 expression throughout the brain at levels exceeding thresholds needed to correct seizures, motor abnormalities, and developmental disabilities.
"These data reflect the significant progress we are making in translating Capsida's innovative capabilities into differentiated clinical therapies," said Peter Anastasiou, Capsida's Chief Executive Officer, in a press release. "We are on track to enter the clinic with our STXBP1-DEE (search) and PD-GBA (search) programs this quarter, with the potential to bring disease-modifying and possibly curative treatments to these communities who so desperately need them."
Expanding Pipeline for Rare Neurological Disorders
Celeste Stephany, PhD, Director of CNS and Ophthalmology Preclinical Research at Capsida, will present data on CAP-004, the company's gene therapy for Friedreich's Ataxia (search) (FA). This single IV-delivered therapy has demonstrated high expression rates across CNS, cardiac, and sensory tissues in preclinical studies, suggesting potential to address multiple manifestations of this debilitating disorder.
Additionally, Capsida will present data on CAP-003 for Parkinson's disease (search) associated with GBA (search) mutations (PD-GBA). According to the poster abstract, this CNS-targeted IV-delivered AAV gene therapy safely increases brain glucocerebrosidase (search) (GCase) in non-human primates to levels that could potentially normalize enzyme activity in PD-GBA patients.
Novel Blood-Brain Barrier Targeting Technology
A particularly noteworthy presentation will come from Nick Goeden, PhD, Capsida's founder and Chief Technology Officer, who will discuss the "Identification of Multiple Novel Blood-Brain-Barrier Receptors for CNS Gene Therapy and Other Drug Modalities via an Integrated AAV Capsid Engineering Platform." This research could have far-reaching implications beyond Capsida's current pipeline, potentially enabling more effective delivery of various therapeutic modalities to the brain.
Manufacturing and Analytical Innovations
Beyond therapeutic candidates, Capsida will showcase advancements in manufacturing and analytical methods critical for gene therapy development. Presentations will cover strategic designs of engineered rAAV two-plasmid systems for cost-effective scaling, comprehensive characterization of vector integrity using next-generation sequencing, and a novel automated approach for enriching full AAV capsids.
These manufacturing innovations could address key challenges in the gene therapy field, including production scalability, quality control, and cost-effectiveness—factors that significantly impact the commercial viability of advanced genetic medicines.
Clinical Development Timeline
Capsida confirmed that both CAP-002 for STXBP1-DEE (search) and CAP-003 for PD-GBA (search) are on track to enter clinical development in the first half of 2025. These programs represent potential first-in-class and best-in-class treatments, respectively, for conditions with significant unmet medical needs.
STXBP1-DEE (search) is a rare genetic disorder characterized by seizures, developmental delay, and intellectual disability, with no approved disease-modifying treatments currently available. PD-GBA (search) affects approximately 10% of Parkinson's disease (search) patients who carry mutations in the GBA gene, typically resulting in earlier disease onset and more rapid progression.
James Goss, Scientific Director of the STXBP1 (search) patient advocacy organization, will represent the STXBP1 community at the conference, underscoring the importance of these developments for patients and families affected by this rare disorder.
Company Background
Founded in 2019 with backing from Versant Ventures and Westlake Village BioPartners, Capsida originated from groundbreaking research in the laboratory of Viviana Gradinaru, PhD, a neuroscience professor at Caltech. The company has established partnerships with pharmaceutical giants including AbbVie (search), Eli Lilly, and CRISPR Therapeutics, validating its proprietary platform technology.
Capsida's approach combines engineered AAV capsids with optimized genetic cargo, potentially enabling more targeted, effective, and safer gene therapies for both rare and common neurological disorders.
Data from the ASGCT presentations will be embargoed until the morning of May 13 for posters and May 14 for oral sessions, with abstracts currently available through the ASGCT website.