Latest news with #PD-GBA
Business Wire
2 days ago
- Health
- Business Wire
Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson's Disease Associated With GBA Mutations
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-003, its potential best-in-class intravenously (IV) administered gene therapy, to enter clinical trials for Parkinson's disease associated with GBA mutations (PD-GBA). This is the second wholly owned clinical program developed by Capsida with a cleared IND. Both programs utilize a proprietary IV-delivered, blood brain barrier-crossing engineered capsid and proprietary cargo that is detargeted from off-target tissues, like liver and dorsal root ganglia (DRG). In addition, Capsida uses a proprietary manufacturing process and CAP-003 is manufactured in Capsida's state-of-the-art wholly owned Good Manufacturing Practice (GMP) facility. 'PD-GBA is an area of substantial unmet need given the lack of approved treatments that target GCase, which is the protein encoded by the GBA gene, and provide meaningful slowing or halting of disease progression,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'We recognize the urgency for new treatment approaches, so we are working diligently to initiate the Phase 1/2 clinical trial for CAP-003 with the aim of dosing the first patient in the third quarter of this year.' About CAP-003 and the Phase 1/2 Clinical Trial In non-human primate (NHP) studies to date, a single IV infusion of CAP-003 resulted in dose-dependent increases in GCase activity in critical brain regions including the substantia nigra, frontal cortex, caudate nucleus, and putamen substantially above the established 30% efficacy threshold expected to restore enzyme activity levels back to normal in patients with PD-GBA. The NHP Good Laboratory Practices (GLP) toxicology study demonstrated a well-tolerated safety profile with no adverse histopathology. Capsida expects to dose the first patient in the Phase 1/2 clinical trial in the third quarter of this year. For more information about the Phase 1/2 clinical trial, please visit (NCT07011771). About Parkinson's disease associated with GBA mutations (PD-GBA) Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of Parkinson's patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients compared to healthy individuals 1. There are no approved treatments that target GCase and there are no approved disease modifying treatments for PD. Other investigational treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, those treatments have required invasive direct brain or cerebrospinal fluid (CSF) administration, with limited results, and a significant burden for patients. 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), CAP-002; and potential best-in-class investigational treatment for Parkinson's disease associated with GBA mutations (PD-GBA) CAP-003. Both have received FDA Investigational New Drug (IND) clearance to initiate clinical trials. Capsida's pipeline also includes a potential best-in-class treatment for Friedreich's ataxia (FA). 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 1 Leyns, C. E, G. et al (2023). npj Parkinson's Disease, 74(9).
Yahoo
2 days ago
- Business
- Yahoo
Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson's Disease Associated With GBA Mutations
Capsida is initiating the Phase 1/2 study for CAP-003, with the first patient expected to be dosed in the third quarter of this year THOUSAND OAKS, Calif., June 11, 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-003, its potential best-in-class intravenously (IV) administered gene therapy, to enter clinical trials for Parkinson's disease associated with GBA mutations (PD-GBA). This is the second wholly owned clinical program developed by Capsida with a cleared IND. Both programs utilize a proprietary IV-delivered, blood brain barrier-crossing engineered capsid and proprietary cargo that is detargeted from off-target tissues, like liver and dorsal root ganglia (DRG). In addition, Capsida uses a proprietary manufacturing process and CAP-003 is manufactured in Capsida's state-of-the-art wholly owned Good Manufacturing Practice (GMP) facility. "PD-GBA is an area of substantial unmet need given the lack of approved treatments that target GCase, which is the protein encoded by the GBA gene, and provide meaningful slowing or halting of disease progression," said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. "We recognize the urgency for new treatment approaches, so we are working diligently to initiate the Phase 1/2 clinical trial for CAP-003 with the aim of dosing the first patient in the third quarter of this year." About CAP-003 and the Phase 1/2 Clinical Trial In non-human primate (NHP) studies to date, a single IV infusion of CAP-003 resulted in dose-dependent increases in GCase activity in critical brain regions including the substantia nigra, frontal cortex, caudate nucleus, and putamen substantially above the established 30% efficacy threshold expected to restore enzyme activity levels back to normal in patients with PD-GBA. The NHP Good Laboratory Practices (GLP) toxicology study demonstrated a well-tolerated safety profile with no adverse histopathology. Capsida expects to dose the first patient in the Phase 1/2 clinical trial in the third quarter of this year. For more information about the Phase 1/2 clinical trial, please visit (NCT07011771). About Parkinson's disease associated with GBA mutations (PD-GBA) Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of Parkinson's patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients compared to healthy individuals1. There are no approved treatments that target GCase and there are no approved disease modifying treatments for PD. Other investigational treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, those treatments have required invasive direct brain or cerebrospinal fluid (CSF) administration, with limited results, and a significant burden for patients. 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), CAP-002; and potential best-in-class investigational treatment for Parkinson's disease associated with GBA mutations (PD-GBA) CAP-003. Both have received FDA Investigational New Drug (IND) clearance to initiate clinical trials. Capsida's pipeline also includes a potential best-in-class treatment for Friedreich's ataxia (FA). 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 1Leyns, C. E, G. et al (2023). npj Parkinson's Disease, 74(9). View source version on Contacts Media Contact Inizio Evoke CommsKatherine Sign in to access your portfolio
Business Wire
28-04-2025
- Business
- Business Wire
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)
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced it will present new data on its positive progress across its wholly owned pipeline, proprietary capsid engineering, and manufacturing in seven scientific presentations accepted 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. Capsida will deliver three oral presentations highlighting advances in Capsida's pipeline of intravenously (IV)-delivered genetic medicines enabled by its proprietary engineered capsids and cargo. The Company will deliver an oral presentation on non-human primate (NHP) GLP toxicology results of CAP-002 for STXBP1-DEE demonstrating widespread and safe STXBP1 expression throughout the brain that exceeds thresholds needed to correct seizures, motor abnormalities, and developmental disabilities. A second oral presentation will feature preclinical data on CAP-004 for Friedreich's ataxia (FA), Capsida's single IV-delivered gene therapy indicating high rates of expression across central nervous system (CNS), cardiac, and sensory tissues, establishing potential to treat all manifestations of the disease. A third oral presentation will showcase Capsida's identification of novel blood-brain barrier receptors and highly CNS tropic and peripherally detargeted capsids. 'These data reflect the significant progress we are making in translating Capsida's innovative capabilities into differentiated clinical therapies,' said Peter Anastasiou, Capsida's Chief Executive Officer. '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.' The presentations are listed below. Abstracts can be found at Data from presentations are embargoed until 6:00 AM CT on the presentation day for oral abstracts and until 6:00 AM CT on May 13, 2025 for poster abstracts. Oral Presentations: Pipeline Systemic AAV Gene Therapy with Next Generation Engineered Capsid Demonstrates Expression Levels Supporting Potential Therapeutic Benefit for CNS, Cardiac, and Sensory Symptoms in Friedreich's Ataxia Date and Time: Wednesday, May 14, 2025, 1:45-2:00 PM CT Session: Neurologic Diseases – Vectorology and Gene Therapy Abstract Number: 75 Location: New Orleans Theater A Presenter: Celeste Stephany, Ph.D., Director of CNS and Ophthalmology Preclinical Research, Capsida 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: Wednesday, May 14, 2025, 3:45-4:00 PM CT Session: Viral Vectors in Large Animal Models Abstract Number: 123 Location: New Orleans Theater B Presenter: Nick Flytzanis, Ph.D., Founder, Chief Research and Innovation Officer, Capsida Engineering Identification of Multiple Novel Blood-Brain-Barrier Receptors for CNS Gene Therapy and Other Drug Modalities via an Integrated AAV Capsid Engineering Platform Date and Time: Wednesday, May 14, 2025, 2:45-3:00 PM CT Session: AAV Gene Transfer (A): Crossing the Blood-Brain Barrier Abstract Number: 93 Location: New Orleans Theater C Presenter: Nick Goeden, Ph.D., Founder, Chief Technology Officer, Capsida Poster Presentations: Preclinical CAP-003, a CNS-Targeted IV-delivered AAV Gene Therapy, Safely Increases Brain GCase in NHPs to Level Supporting Potential Normalization of Activity in PD-GBA Patients Date and Time: Wednesday, May 14, 2025, 5:30-7:00 PM CT Session: Wednesday Poster Reception Abstract Number: 1435 Location: Hall I2 Presenter: Kim McDowell, Ph.D., Director, Preclinical Research, Capsida Process Development, Analytical Development, Manufacturing rAAV Manufacturing Solutions: Strategic Designs of Engineered rAAV Two Plasmid Systems for Cost Effective Scaling and Product Safety Date and Time: Tuesday, May 13, 2025, 5:30-7:00 PM CT Session: Tuesday Poster Reception Abstract Number: 962 Location: Hall I2 Presenter: Jenna Rodden, Senior Research Associate, Capsida Dual-Platform NGS for Comprehensive Characterization of Engineered rAAV Vector Integrity Date and Time: Wednesday, May 14, 2025, 5:30-7:00 PM CT Session: Wednesday Poster Reception Abstract Number: 1326 Location: Hall I2 Presenter: Zach Mason, Associate Scientist, Capsida Development of a Novel Automated Loading Approach Which Significantly Reduces Processing Time for Enriching Full AAV Capsids Using Ultracentrifugation Date and Time: Thursday, May 15, 2025, 5:30-7:00 PM CT Session: Thursday Poster Reception Abstract Number: 1833 Location: Hall I2 Presenter: Varun Gejji, Ph.D., Senior Scientist, Capsida 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 a potential first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), best-in-class treatment for Parkinson's disease associated with GBA mutations (PD-GBA), and best-in-class therapy for Friedreich's ataxia (FA). The STXBP1-DEE and PD-GBA programs are on track to enter the clinic in 1H 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
