Data to be featured as oral and poster presentations at American Epilepsy Society 2024 Annual Meeting
THOUSAND OAKS, Calif., Dec. 6, 2024 /PRNewswire/ -- Capsida Biotherapeutics (“Capsida”) today announced new preclinical data on CAP-002, its first-in-class IV-administered next-generation gene therapy candidate for developmental and epileptic encephalopathy (DEE) due to syntaxin-binding protein 1 (STXBP1) mutations. The new data in non-human primates (NHPs) and human cells establish the potential for CAP-002 to significantly correct seizures, motor abnormalities, and developmental/intellectual disabilities in STXBP1-DEE patients. CAP-002 is on track to enter the clinic in the first half of 2025. Capsida recently completed a pre-IND meeting with the U.S. Food and Drug Administration (FDA) and received Orphan Drug Designation from the FDA for CAP-002.
In diseased human neurons in vitro, CAP-002 restored STXBP1 protein to normal levels and fully corrected neuronal network activity. In NHPs, CAP-002 administered intravenously (IV) demonstrated dose-dependent brain-wide distribution and expression, while limiting effects to off-target tissue, such as liver and dorsal root ganglia (DRGs). The dose-dependent increases of STXBP1 significantly correlate with preclinical mouse pharmacology studies that have established the potential to treat and even fully correct the disease through gene supplementation. CAP-002 was safe and well-tolerated, with 20x lower targeting to liver and 143x lower DRG expression compared to AAV9, with no adverse histopathological findings.
“STXBP1-DEE is a life-altering condition for patients and their families, and there are no approved treatments,” said Peter Anastasiou, Chief Executive Officer of Capsida Biotherapeutics. “These new data demonstrate the potential of CAP-002 to safely address all of the manifestations of the disease, and we look forward to progressing this program into the clinic in the first half of 2025.”
These data will be featured in oral and poster presentations at the American Epilepsy Society (AES) 2024 Annual Meeting being held December 6-10, 2024 in Los Angeles, CA.
Presentation Details
Oral Presentation:
- Title: Systemic Gene Therapy with Engineered AAV Demonstrates Preclinical Efficacy and Safety Supporting a Disease-Modifying Treatment for STXBP1 Developmental and Epileptic Encephalopathy
- Date and Time: Saturday, December 7, 2024, 2:45 – 5:00 PM PT
- Session: Platform B | Translational Research
- Session Number: B.06
- Location: Los Angeles Convention Center, Room 404, Level 2
- Presenter: Allison Knoll, Ph.D., Director of Preclinical Research, Capsida
Poster Presentation:
- Title: Systemic Gene Therapy with Engineered AAV Demonstrates Preclinical Efficacy and Safety Supporting a Disease-Modifying Treatment for STXBP1 Developmental and Epileptic Encephalopathy
- Date and Time: Saturday, December 7, 2024, 12:00 – 2:00 PM PT
- Poster Session: Poster Session 1
- Poster Number: 237
- Location: Los Angeles Convention Center, South Hall H, Level 1
- Presenter: Allison Knoll, Ph.D., Director of Preclinical Research, Capsida
Abstracts can be found on the AES website at www.aesnet.org.
About STXBP1 Developmental and Epileptic Encephalopathy (STXBP1-DEE)
The STXBP1 protein is present in every neuron in the brain and is essential for normal neurotransmission. A mutation in the STXBP1 gene is associated with treatment-resistant seizures, severe developmental delay and intellectual disability, motor abnormalities, and sudden unexpected death in epilepsy (SUDEP). STXBP1-DEE is estimated to affect up to one in 26,000 children globally. There are no approved treatments.About Capsida Biotherapeutics
Capsida Biotherapeutics is a fully integrated gene therapy 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 proprietary engineered capsids enable high levels of neuron transduction while limiting tropism to non-target organs, such as the liver. Capsida’s wholly owned pipeline includes a potential first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), best-in-class treatment for Parkinson’s disease associated with GBA mutations (PD-GBA), and best-in-class therapy for Friedreich’s ataxia. The STXBP1-DEE and PD-GBA programs are on track to enter the clinic in the first half of 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. Visit us at www.capsida.com.
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