Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson’s Disease Associated With GBA Mutations
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.
Capsida Receives FDA Fast Track Designation for Its Potential First-in-Class IV-Administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
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.
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
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.
Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
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.
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)
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.
