STXBP1-related epileptic encephalopathy

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 . 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 . 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THOUSAND OAKS, Calif., Nov. 25, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida"), a leading fully integrated next-generation gene therapy company developing treatments for rare and common diseases across all ages, today announced that the company will present at the 7th Annual Evercore HealthCONx Conference being held December 3-5, 2024 in Coral Gables, FL. Management will highlight progress with Capsida's wholly owned IV-administered gene therapies utilizing the company's proprietary engineered capsids, which enable high transduction levels of neurons, while limiting tropism to non-target organs, such as the liver. The lead programs are for STXBP1 developmental and epileptic encephalopathy (CAP-002) and Parkinson's disease associated with GBA mutations (CAP-003), which are both on track for IND filings in the first half of 2025. A third wholly owned program for Friedreich's ataxia (CAP-004) is in IND-enabling studies. Capsida's Chief Executive Officer Peter Anastasiou will participate in a fireside chat on Wednesday, December 4, 2024 at 12:55 PM ET. Management will also participate in one-on-one investor meetings. The Capsida corporate presentation can be accessed by visiting the News section of the Company's website at . 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 wholly owned pipeline includes a potential first-in-class treatment for STXBP1 developmental and epileptic encephalopathy, best-in-class treatment for Parkinson's disease associated with GBA mutations, and best-in-class therapy for Friedreich's ataxia. 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 . SOURCE Capsida Biotherapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NHP data demonstrate potential for CAP-004 to treat CNS, cardiac, and sensory manifestations of Friedreich's ataxia THOUSAND OAKS, Calif., Nov. 12, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced new preclinical data for CAP-004, a potential best-in-class systemically administered next-generation gene therapy for the treatment of Friedreich's ataxia. CAP-004 is Capsida's third wholly owned program and utilizes one of the company's proprietary engineered capsids to target central nervous system (CNS), cardiac, and sensory manifestations of the disease while limiting effects in off-target tissues, especially the liver. CAP-004 is now entering Investigational New Drug (IND) application enabling studies. Data highlighting the therapeutic potential of CAP-004 will be presented in a late-breaking poster presentation at the International Congress for Ataxia Research (ICAR), being held November 12-15, 2024 in London, UK. Friedreich's ataxia is a rare hereditary disease with limited treatment options. It is caused by a mutation that reduces frataxin (FXN) protein expression, leading to progressive neurodegeneration, cardiac dysfunction, and sensory deterioration. CAP-004 is designed to target and address the key pathophysiological features of the disease by delivering therapeutically meaningful FXN expression in the CNS, cardiac tissues, and sensory regions. In the study conducted in non-human primates (NHPs), results showed that CAP-004 demonstrated: Brain-wide biodistribution, achieving more than 80% transduction in key cell types, such as Purkinje cells in the cerebellum, neurons in the dentate nucleus, and motor neurons in the cortex and spinal cord. 100 times higher RNA expression compared with AAV9 in the cerebellum, motor cortex, and spinal cord. Significant cardiac tissue transduction, covering nearly 30% of the left ventricle tissue area. Bulk hFXN protein levels in treated NHPs were up to 8.2 times higher than endogenous levels in the motor cortex and 1.7 times higher in the left ventricle, highlighting the therapeutic potential in both CNS and cardiac regions. Potential in treating sensory vision loss, with significant RNA expression detected in the retina. Significant detargeting of the liver and other non-target tissues contributed to the favorable safety profile characterized by no adverse immunogenicity, clinical pathology, and histopathology findings. "These preclinical data demonstrate CAP-004's potential to be a best-in-class gene therapy for Friedreich's ataxia, a devastating disease with few treatment options," said Peter Anastasiou, CEO of Capsida Biotherapeutics. "With CAP-004 delivered as a single IV injection, Capsida has the potential to address neurodegenerative, cardiovascular, and sensory components of this disease." Details about the CAP-004 ICAR poster presentation: Systemic AAV Gene Therapy with Next Generation Engineered Capsids for Treatment of CNS and Cardiac Symptoms in Friedreich's Ataxia Date: Wednesday, November 13, 2024, 6:00 PM GMT Session: Emerging and existing therapeutics – preclinical research Poster Number: 477 Location: Leonardo Royal Hotel London Tower Bridge, Minories Suite Presenter: Celeste Stephany, Ph.D., Director of CNS and Ophthalmology Preclinical Research, Capsida 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 intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including a potential first-in-class treatment for STXBP1 developmental and epileptic encephalopathy and a best-in-class treatment Parkinson's disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. Capsida's third program is a potential best-in-class therapy for Friedreich's ataxia and is in IND-enabling studies. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. 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 . SOURCE Capsida Biotherapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED