Capsida Biotherapeutics to Present Broad Pipeline and Platform Advances at ASGCT 2025 Annual Meeting
Capsida Biotherapeutics (“Capsida”), a biotechnology company focused on developing targeted gene therapies for serious neurological and systemic disorders, announced today that it will present significant new data highlighting the breadth of its scientific and clinical progress at the upcoming 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The conference will be held May 13–17, 2025, both in New Orleans and virtually, and will feature seven Capsida presentations across oral and poster sessions.
The presentations span advances in Capsida’s wholly owned pipeline, proprietary capsid engineering platform, and manufacturing capabilities. Together, the data showcase the company’s momentum in translating its cutting-edge science into differentiated, intravenously (IV) delivered genetic medicines designed to precisely target the central nervous system (CNS) and other difficult-to-reach tissues.
“Our upcoming presentations at ASGCT reflect the significant strides we have made in turning our proprietary capsid engineering and gene therapy platform into a powerful pipeline of differentiated clinical programs,” said Peter Anastasiou, Chief Executive Officer of Capsida. “We are on track to initiate clinical trials for our STXBP1-DEE and PD-GBA programs this quarter, marking a major milestone in our mission to bring disease-modifying and potentially curative therapies to patients who currently have few or no treatment options.”
Highlights of Capsida’s ASGCT 2025 Presentations
Capsida will deliver three oral presentations and four poster presentations at ASGCT 2025. The three oral presentations focus on:
- CAP-002 for STXBP1-DEE: NHP GLP Toxicology Results
Capsida will present non-human primate (NHP) Good Laboratory Practice (GLP) toxicology study results for its lead gene therapy candidate, CAP-002, targeting STXBP1-related developmental and epileptic encephalopathy (STXBP1-DEE). The data demonstrate widespread, safe, and therapeutically meaningful expression of STXBP1 throughout the brain after a single IV administration. The levels of gene expression achieved exceed the thresholds believed necessary to correct the seizures, motor abnormalities, and cognitive deficits associated with the disease. These findings strongly support the advancement of CAP-002 into human clinical trials later this year. - CAP-004 for Friedreich’s Ataxia (FA): Preclinical Proof of Concept
A second oral presentation will showcase preclinical efficacy data for CAP-004, Capsida’s investigational gene therapy for Friedreich’s ataxia (FA), a rare inherited neurodegenerative disease. CAP-004, delivered via a single IV infusion, demonstrated high rates of frataxin expression across CNS tissues, cardiac muscle, and dorsal root ganglia — the major sites of pathology in FA. These results suggest the potential for CAP-004 to address not only the neurological symptoms but also the cardiac and sensory manifestations of the disease, offering a comprehensive, systemic therapeutic solution. - Discovery of Novel Blood-Brain Barrier Receptors and Next-Generation CNS-Tropic Capsids
In a third oral presentation, Capsida scientists will reveal discoveries from their ongoing capsid engineering work, including the identification of novel receptors at the blood-brain barrier that facilitate highly efficient CNS gene delivery. The company will also introduce a new class of engineered AAV capsids that achieve enhanced CNS tropism while minimizing peripheral organ exposure, a major advance in the safety and effectiveness of IV-administered gene therapies.
The four poster presentations will provide additional insights into Capsida’s broader research, including manufacturing advances enabling consistent large-scale production of its proprietary vectors.
CAP-002: Leading the Way in STXBP1-DEE
STXBP1-DEE is a severe, early-onset neurodevelopmental disorder characterized by intractable epilepsy, profound developmental delay, and significant motor dysfunction. Mutations in the STXBP1 gene impair neurotransmitter release at synapses, leading to widespread neuronal dysfunction. There are currently no approved disease-modifying treatments for this disorder, and existing therapies are limited to seizure management with varying degrees of success.
CAP-002 is designed to deliver a healthy copy of the STXBP1 gene directly to affected neurons via a minimally invasive IV infusion. By restoring normal synaptic function, Capsida aims to fundamentally alter the trajectory of the disease. The NHP GLP toxicology data being presented at ASGCT show that CAP-002 not only achieves widespread brain distribution but does so safely, without significant off-target effects.
“These findings are critical as we prepare to enter the clinic,” said Dr. Susan Catalano, Chief Scientific Officer of Capsida. “They validate the translational potential of our platform and support our belief that CAP-002 could become the first effective disease-modifying therapy for STXBP1-DEE.”
CAP-004: A Comprehensive Approach to Friedreich’s Ataxia
Friedreich’s ataxia is a progressive genetic disease caused by insufficient expression of the frataxin protein, leading to degeneration of the nervous system and heart failure. Because the disease affects multiple tissues — the brain, spinal cord, heart, and peripheral nerves an effective therapy must achieve widespread systemic delivery.
CAP-004’s preclinical results demonstrate that Capsida’s engineered capsids can cross the blood-brain barrier efficiently and simultaneously transduce critical non-CNS tissues. In animal models, a single IV dose led to robust frataxin expression in all key disease-relevant tissues, raising hopes that CAP-004 could offer broad clinical benefits to patients.
“There is a clear need for a systemic gene therapy approach in FA, and CAP-004 represents an elegant solution,” said Catalano. “Our data suggest that we can address the neurological, cardiac, and sensory impairments simultaneously, which could dramatically improve quality of life for individuals living with this debilitating condition.”
Advancing Capsid Engineering and Manufacturing Excellence
At the heart of Capsida’s success is its proprietary capsid engineering platform, which combines high-throughput screening, machine learning, and structural biology to develop novel AAV vectors with improved characteristics. The ability to engineer capsids with high CNS selectivity and reduced peripheral exposure has the potential to transform the treatment landscape for a wide range of neurological diseases.
In parallel, Capsida has built state-of-the-art manufacturing capabilities to produce its customized vectors at clinical and commercial scales. Its manufacturing platform enables efficient, high-yield production while maintaining vector quality and consistency, a critical requirement as the company advances into late-stage clinical development and eventual commercialization.
On Track for Clinical Entry
Following the ASGCT presentations, Capsida remains on track to submit Investigational New Drug (IND) applications for CAP-002 in STXBP1-DEE and for its PD-GBA program, targeting a genetic subset of Parkinson’s disease, within the current quarter. Initiation of first-in-human clinical trials for both programs is anticipated shortly thereafter.
“These upcoming clinical trials represent the culmination of years of groundbreaking science and hard work by the Capsida team,” said Anastasiou. “We are eager to begin testing our therapies in patients and to move closer to our goal of delivering transformative genetic medicines to those who need them most”.
About Capsida Biotherapeutics
Capsida Biotherapeutics is a fully integrated next-generation genetic medicines company with a central nervous system (CNS) pipeline consisting of disease-modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida’s wholly owned pipeline includes a potential first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), best-in-class treatment for Parkinson’s disease associated with GBA mutations (PD-GBA), and best-in-class therapy for Friedreich’s ataxia (FA). The STXBP1-DEE and PD-GBA programs are on track to enter the clinic in 1H 2025. In addition to its wholly owned programs, the Company has validating partnerships with AbbVie, Lilly, and CRISPR Therapeutics. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech.
