STXBP1

The therapy restored syntaxin-binding protein 1 (STXBP1) protein levels in primate neurons and in lab-cultured human neurons. STXBP1 is one of the genes that can cause DEE when mutated.\n Capsida Biotherapeutics has unveiled data showing that its AAV-delivered gene therapy for developmental and epileptic encephalopathy (DEE), the most severe form of epilepsy, corrected cognitive and motor deficits and reduced seizures in mice up to one year after treatment.The California-based biotech presented the results on Dec. 7 at the annual meeting of the American Epilepsy Society in Los Angeles.Capsida recently met with the FDA for a pre-Investigational New Drug meeting and received an Orphan Drug Designation for the therapy, called CAP-002, the company said in a Dec. 6 release. Capsida plans to bring CAP-002 into the clinic in the first half of 2025. The therapy restored syntaxin-binding protein 1 (STXBP1) protein levels in 70% of primate neurons, according to the abstract. STXBP1 is one of the genes that can cause DEE when mutated. CAP-002 also restored STXBP1 protein levels and neuronal network activity in human neurons cultured in the lab, Capsida said.“STXBP1-DEE is a life-altering condition for patients and their families, and there are no approved treatments,” Capsida CEO Peter Anastasiou said in the release. “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.”DEE often manifests in infancy, with children experiencing frequent seizures that are difficult to control with typical anti-seizure medications. Patients commonly have other conditions alongside DEE, like sleep problems, movement disorders and autism spectrum disorder.

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 . 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

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