Capsida Previews Its Oral and Poster Presentations for ASGCT 2025
The company’s exhibitions will be focused in the areas of its pipeline, proprietary capsid engineering, and manufacturing.
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Thousand Oaks, Calif.-based Capsida Biotherapeutics (Capsida) has announced its slate of seven scientific presentations, three oral and four in poster form, that have been accepted for the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), which will be held in New Orleans from May 13–17, 2025 (1).
In a press release, Capsida said the presentations would show new data demonstrating the company’s positive progress across its wholly owned pipeline, proprietary capsid engineering, and manufacturing (1).
More specifically, the three oral presentations will highlight advances in Capsida’s intravenously (IV)-delivered genetic medicines—including its first-in-class IV administered gene therapy CAP-002, designed to achieve brain-wide neuronal expression while simultaneously detargeting the liver—which are enabled by the company’s engineered capsids and cargo (1,2).
The first, to be presented by Celeste Stephany, PhD, Capsida director of CNS (central nervous system) and Ophthalmology Preclinical Research, is entitled “Systemic AAV (adeno-associated virus) Gene Therapy with Next Generation Engineered Capsid Demonstrates Expression Levels Supporting Potential Therapeutic Benefit for CNS, Cardiac, and Sensory Symptoms in Friedreich’s Ataxia” (1). The second is closely related: “Systemic Gene Therapy CAP-002 Demonstrates Potential for Disease-Modifying Treatment of Seizures and Motor and Cognitive Deficits of STXBP1-DEE Using an Engineered, CNS-Targeted AAV,” to be presented by one of Capsida’s founders and Chief Research and Innovation Officer Nick Flytzanis, PhD.
A third oral presentation will be made by founder and Chief Technology Officer, Nick Goeden, PhD, entitled “Identification of Multiple Novel Blood-Brain-Barrier Receptors for CNS Gene Therapy and Other Drug Modalities via an Integrated AAV Capsid Engineering Platform” (1).
All oral presentations will be made on Wednesday, May 14, as will two of the four poster presentations; the other posters will be exhibited during evening sessions on Tuesday, May 13 and Thursday, May 15 (1). Capsida said the data contained in each presentation will be embargoed until the morning of May 13 for posters, and the morning of May 14 (the presentation day) for oral sessions.
“These data reflect the significant progress we are making in translating Capsida’s innovative capabilities into differentiated clinical therapies,” said Peter Anastasiou, Capsida chief executive officer, in an April 28, 2025 press release (1). “We are on track to enter the clinic with our STXBP1-DEE and PD-GBA programs this quarter, with the potential to bring disease-modifying and possibly curative treatments to these communities who so desperately need them.”
Abstracts can be accessed through the ASGCT website (3).
Capsida presented preclinical evidence for CAP-002 at ASGCT 2024 in Baltimore, in May 2024 (2).
“This study built upon our previous proof-of-concept study and represents a significant advancement in our understanding of the therapeutic potential of engineered AAV gene supplementation therapy in the treatment of genetic epilepsy and developmental disorders due to STXBP1 mutations. These data are encouraging and emphasize the potential for CAP-002 to meaningfully improve outcomes in patients with this disease,” Mingshan Xue, PhD, associate professor, Department of Neuroscience, Department of Molecular and Human Genetics at Baylor College of Medicine and the Cain Foundation Laboratories, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and an ASGCT 2024 presentation co-author, said at the time (2).
References
1. Capsida Biotherapeutics. Capsida to Present Progress Updates at the ASGCT Annual Meeting, Including NHP GLP Toxicology Study Results for its Potential First-in-Class STXBP1 Developmental and Epileptic Encephalopathy Program (CAP-002 STXBP1-DEE). Press Release. April 28, 2025.
2. Capsida Biotherapeutics. Capsida Biotherapeutics Presents New Preclinical Evidence Indicating Novel First-in-Class IV-Administered Gene Therapy Effectively Treats Genetic Epilepsy Due to STXBP1 Mutations. Press Release. May 7, 2024.
3. American Society of Gene & Cell Therapy. ASGCT Annual Meeting. annualmeeting.asgct.org (accessed April 29, 2025).
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