Latest news with #STXBP1-DEE
Business Wire
5 days ago
- Health
- Business Wire
Capsida Receives FDA Fast Track Designation for Its Potential First-in-Class IV-Administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its CAP-002 program. CAP-002 is the company's investigational IV-administered gene therapy for the treatment of STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), and is the first IV-delivered, AAV blood brain barrier-crossing genetic medicine program entering a human clinical trial. The FDA's Fast Track designation is designed to facilitate the development and expedite the review of drugs that are intended to treat serious or life-threatening conditions and have the potential to address an unmet medical need. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of application for marketing approval. A Fast Track-designated drug also may be eligible for Priority Review if relevant criteria are met. 'FDA's granting of Fast Track designation for CAP-002 highlights the significant unmet need in the treatment of STXBP1-DEE and the potential of CAP-002 to be a first-in-class treatment,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'We are currently initiating study start-up activities for our Phase 1/2a SYNRGY clinical trial with the aim of providing the first potential disease-modifying treatment for STXBP1-DEE patients and their families.' About CAP-002 and the SYNRGY Clinical Trial CAP-002 is enabled by one of Capsida's proprietary engineered capsids and optimized cargo. In non-human primate (NHP) studies to date, CAP-002 has established transduction of more than 70% of neurons across critical brain areas, while simultaneously detargeting the liver and dorsal root ganglia. This brain-wide expression of STXBP1 has the potential to correct seizures, developmental disabilities, and motor abnormalities, after a single IV infusion. The NHP Good Laboratory Practice (GLP) toxicology study demonstrated a well-tolerated safety profile with no adverse histopathology. CAP-002 is manufactured in Capsida's state-of-the-art wholly owned facility using a proprietary manufacturing process. CAP-002 received FDA Orphan Drug Designation in October 2024 and Investigational New Drug (IND) clearance in May 2025. Capsida expects to dose the first patient in the SYNRGY Phase 1/2a clinical trial in the third quarter of this year. For more information about the SYNRGY clinical trial, please visit (NCT06983158). About STXBP1-DEE Syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE) is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in the U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. Mutations in the STXBP1 gene are associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments for STXBP1-DEE. About Capsida Biotherapeutics Capsida Biotherapeutics is a clinical-stage, fully integrated next-generation genetic medicines company. It has 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 its first-in-class investigational treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received U.S. Food and Drug Administration Investigational New Drug (IND) clearance to initiate the SYNRGY Ph1/2a clinical trial. Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development in the second quarter 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
Business Wire
14-05-2025
- Business
- Business Wire
Capsida Presents New GLP Toxicology Data Supporting Recent FDA IND Clearance of Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced the presentation of new non-human primate (NHP) GLP toxicology data for CAP-002, its wholly owned first-in-class, intravenously (IV) administered investigational gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). These data were the foundation for the Investigational New Drug (IND) application for CAP-002 that was recently cleared by the U.S. Food and Drug Administration (FDA). Capsida will deliver these data in an oral presentation today at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 13-17, 2025, in New Orleans and virtually. The three-month cohort data from the NHP GLP toxicology study demonstrate dose-dependent brain-wide expression of STXBP1 and simultaneous detargeting of the liver and dorsal root ganglia (DRGs). CAP-002 was delivered as a single dose via IV infusion at 3.8E13 vg/kg, 5.9E13 vg/kg, and 7.4E13 vg/kg. There was a dose-dependent increase in average cargo RNA levels in the brain, with the 3.8E13 vg/kg dose of CAP-002 demonstrating a 207-fold increase over cargo RNA levels previously shown for 2.5E13 vg/kg of IV-delivered adeno-associated virus 9 (AAV9). Together with pharmacology data in a mouse model of STXBP1-DEE, these NHP GLP toxicology results show that all doses of CAP-002 tested have the potential to fully correct seizures and meaningfully correct cognitive and motor dysfunction in patients. The 3.8E13 vg/kg dose of CAP-002 also demonstrated robust 10x and 13x detargeting of liver and DRGs respectively, compared to a 2.5E13 vg/kg dose of AAV9. CAP-002 was well tolerated with no adverse clinical pathology or histopathology findings throughout the central nervous system, DRGs, or peripheral organs, including the liver. CAP-002 is the first IV-administered, AAV blood brain barrier-crossing genetic medicine program entering a human clinical trial. CAP-002 is manufactured in Capsida's state-of-the-art wholly owned facility using a proprietary manufacturing process. Capsida received FDA Orphan Drug Designation in October 2024 and is now initiating study start-up activities for the SYNRGY Phase 1/2a clinical trial, with the first patient expected to be dosed in the third quarter of this year. 'These GLP toxicology data established the potential of CAP-002 to safely treat STXBP1-DEE and were a critical part of the data package to enable the FDA IND clearance of this first-in-class IV investigational gene therapy,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'Our focus is on initiating our Phase 1/2a SYNRGY clinical trial with the aim of providing a safe, disease-modifying treatment for STXBP1-DEE patients and their families.' Presentation Details Oral Presentation: Title: 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 Date and Time: Session: Viral Vectors in Large Animal Models Location: New Orleans Theater B Presenter: Nicholas Flytzanis, Ph.D., Founder, Chief Research and Innovation Officer, Capsida Abstracts can be found at and the presentation will be available under the 'Publications' section of the Capsida website. About STXBP1-DEE Syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE) is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in the U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. Mutations in the STXBP1 gene are associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments for STXBP1-DEE. 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 its first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received Investigational New Drug (IND) clearance to initiate clinical trials from the U.S. Food and Drug Administration (FDA). Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development this quarter. 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
Associated Press
14-05-2025
- Business
- Associated Press
Capsida Presents New GLP Toxicology Data Supporting Recent FDA IND Clearance of Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--May 14, 2025-- Capsida Biotherapeutics ('Capsida') today announced the presentation of new non-human primate (NHP) GLP toxicology data for CAP-002, its wholly owned first-in-class, intravenously (IV) administered investigational gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). These data were the foundation for the Investigational New Drug (IND) application for CAP-002 that was recently cleared by the U.S. Food and Drug Administration (FDA). Capsida will deliver these data in an oral presentation today at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 13-17, 2025, in New Orleans and virtually. The three-month cohort data from the NHP GLP toxicology study demonstrate dose-dependent brain-wide expression of STXBP1 and simultaneous detargeting of the liver and dorsal root ganglia (DRGs). CAP-002 was delivered as a single dose via IV infusion at 3.8E13 vg/kg, 5.9E13 vg/kg, and 7.4E13 vg/kg. There was a dose-dependent increase in average cargo RNA levels in the brain, with the 3.8E13 vg/kg dose of CAP-002 demonstrating a 207-fold increase over cargo RNA levels previously shown for 2.5E13 vg/kg of IV-delivered adeno-associated virus 9 (AAV9). Together with pharmacology data in a mouse model of STXBP1-DEE, these NHP GLP toxicology results show that all doses of CAP-002 tested have the potential to fully correct seizures and meaningfully correct cognitive and motor dysfunction in patients. The 3.8E13 vg/kg dose of CAP-002 also demonstrated robust 10x and 13x detargeting of liver and DRGs respectively, compared to a 2.5E13 vg/kg dose of AAV9. CAP-002 was well tolerated with no adverse clinical pathology or histopathology findings throughout the central nervous system, DRGs, or peripheral organs, including the liver. CAP-002 is the first IV-administered, AAV blood brain barrier-crossing genetic medicine program entering a human clinical trial. CAP-002 is manufactured in Capsida's state-of-the-art wholly owned facility using a proprietary manufacturing process. Capsida received FDA Orphan Drug Designation in October 2024 and is now initiating study start-up activities for the SYNRGY Phase 1/2a clinical trial, with the first patient expected to be dosed in the third quarter of this year. 'These GLP toxicology data established the potential of CAP-002 to safely treat STXBP1-DEE and were a critical part of the data package to enable the FDA IND clearance of this first-in-class IV investigational gene therapy,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'Our focus is on initiating our Phase 1/2a SYNRGY clinical trial with the aim of providing a safe, disease-modifying treatment for STXBP1-DEE patients and their families.' Presentation Details Oral Presentation: Abstracts can be found at and the presentation will be available under the 'Publications' section of the Capsida website. About STXBP1-DEE Syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE) is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in the U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. Mutations in the STXBP1 gene are associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments for STXBP1-DEE. 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 its first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received Investigational New Drug (IND) clearance to initiate clinical trials from the U.S. Food and Drug Administration (FDA). Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development this quarter. 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 View source version on CONTACT: Media Contact Greig Communications, Inc. Kathy Vincent [email protected] KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA LOUISIANA INDUSTRY KEYWORD: HEALTH GENETICS CLINICAL TRIALS RESEARCH SCIENCE PHARMACEUTICAL BIOTECHNOLOGY SOURCE: Capsida Biotherapeutics Copyright Business Wire 2025. PUB: 05/14/2025 07:45 AM/DISC: 05/14/2025 07:44 AM
Yahoo
12-05-2025
- Health
- Yahoo
Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
CAP-002 is the first engineered IV-delivered gene therapy that crosses the blood-brain-barrier and detargets the liver and dorsal root ganglia to enter the clinic THOUSAND OAKS, Calif., May 12, 2025--(BUSINESS WIRE)--Capsida Biotherapeutics ("Capsida") today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for CAP-002, its wholly owned first-in-class, intravenously (IV) administered gene therapy to enter clinical trials for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). This is the first program entering a human clinical trial utilizing an IV-administered, blood brain barrier-crossing engineered capsid that also is detargeted from off-target tissues, like liver and dorsal root ganglia (DRG). CAP-002 is enabled by one of Capsida's proprietary engineered capsids and optimized cargo. In addition, Capsida uses a proprietary manufacturing process and CAP-002 is manufactured in Capsida's state-of-the-art wholly owned Good Manufacturing Practice (GMP) facility. STXBP1-DEE is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. A mutation in the STXBP1 gene is associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments. "STXBP1-related disorders present devastating challenges in communication, development, motor function and seizures. We are in dire need of targeted therapies that can improve the lives and functioning of our children and families," said Charlene Son Rigby, STXBP1 Foundation President and Cofounder. Gene therapy for STXBP1-DEE had not been possible because wild-type adeno-associated viruses (AAVs), such as AAV9, cannot achieve the level of widespread neuronal transduction required to modify the disease. In non-human primate (NHP) studies to date, CAP-002 has established transduction of more than 70% of neurons across critical brain areas, while simultaneously detargeting the liver and DRGs. This brain-wide expression of STXBP1 has the potential to correct seizures, developmental disabilities, and motor abnormalities, after a single IV infusion. The NHP Good Laboratory Practice (GLP) toxicology study has shown a well-tolerated safety profile with no adverse histopathology. CAP-002 received Orphan Drug Designation (ODD) from the FDA in October 2024. Capsida is now initiating study start-up activities for the SYNRGY Phase 1/2a clinical trial, with the first patient expected to be dosed in the third quarter of this year. "This is the first potentially disease-modifying treatment for STXBP1-DEE, and we are excited to be part of the SYNRGY clinical trial," said Ingo Helbig, M.D., Pediatric Neurologist and Clinical Director of Clinical Research at the Center for Epilepsy and Neurodevelopmental Disorders (ENDD) at Children's Hospital of Philadelphia (CHOP), and paid consultant to Capsida. "The FDA clearance of the CAP-002 IND is a significant milestone for Capsida, the STXBP1 community, and the field of genetic medicine," said Swati Tole, M.D., Chief Medical Officer of Capsida. "We look forward to initiating the SYNRGY clinical trial and dosing patients starting in the third quarter of this year with this potential first targeted therapy for STXBP1-DEE." 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 its first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received Investigational New Drug clearance (IND) to initiate clinical trials from the U.S. Food and Drug Administration (FDA). Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development this quarter. 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 View source version on Contacts Media Contact Greig Communications, Vincentkathy@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
12-05-2025
- Health
- Business Wire
Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for CAP-002, its wholly owned first-in-class, intravenously (IV) administered gene therapy to enter clinical trials for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). This is the first program entering a human clinical trial utilizing an IV-administered, blood brain barrier-crossing engineered capsid that also is detargeted from off-target tissues, like liver and dorsal root ganglia (DRG). CAP-002 is enabled by one of Capsida's proprietary engineered capsids and optimized cargo. In addition, Capsida uses a proprietary manufacturing process and CAP-002 is manufactured in Capsida's state-of-the-art wholly owned Good Manufacturing Practice (GMP) facility. STXBP1-DEE is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. A mutation in the STXBP1 gene is associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments. "STXBP1-related disorders present devastating challenges in communication, development, motor function and seizures. We are in dire need of targeted therapies that can improve the lives and functioning of our children and families," said Charlene Son Rigby, STXBP1 Foundation President and Cofounder. Gene therapy for STXBP1-DEE had not been possible because wild-type adeno-associated viruses (AAVs), such as AAV9, cannot achieve the level of widespread neuronal transduction required to modify the disease. In non-human primate (NHP) studies to date, CAP-002 has established transduction of more than 70% of neurons across critical brain areas, while simultaneously detargeting the liver and DRGs. This brain-wide expression of STXBP1 has the potential to correct seizures, developmental disabilities, and motor abnormalities, after a single IV infusion. The NHP Good Laboratory Practice (GLP) toxicology study has shown a well-tolerated safety profile with no adverse histopathology. CAP-002 received Orphan Drug Designation (ODD) from the FDA in October 2024. Capsida is now initiating study start-up activities for the SYNRGY Phase 1/2a clinical trial, with the first patient expected to be dosed in the third quarter of this year. "This is the first potentially disease-modifying treatment for STXBP1-DEE, and we are excited to be part of the SYNRGY clinical trial," said Ingo Helbig, M.D., Pediatric Neurologist and Clinical Director of Clinical Research at the Center for Epilepsy and Neurodevelopmental Disorders (ENDD) at Children's Hospital of Philadelphia (CHOP), and paid consultant to Capsida. 'The FDA clearance of the CAP-002 IND is a significant milestone for Capsida, the STXBP1 community, and the field of genetic medicine,' said Swati Tole, M.D., Chief Medical Officer of Capsida. "We look forward to initiating the SYNRGY clinical trial and dosing patients starting in the third quarter of this year with this potential first targeted therapy for STXBP1-DEE.' 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 its first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received Investigational New Drug clearance (IND) to initiate clinical trials from the U.S. Food and Drug Administration (FDA). Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development this quarter. 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
