CAP-002 / Capsida Biotherap - LARVOL DELTA

A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy (clinicaltrials.gov) - P1/2 | N=12 | Suspended | Sponsor: Capsida Biotherapeutics, Inc. | Recruiting ➔ Suspended

Trial suspension • CNS Disorders • Epilepsy • Gene Therapies • Pediatrics

A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Capsida Biotherapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Epilepsy • Gene Therapies • Pediatrics

Capsida Receives FDA Fast Track Designation for Its Potential First-in-Class IV-Administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy (Businesswire) - "Capsida Biotherapeutics ('Capsida') today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its CAP-002 program."

Fast track • CNS Disorders • Epilepsy

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 (ASGCT 2025) - "These results demonstrate that AAV gene therapy, if able to achieve wide-spread biodistribution and supplementation of STXBP1 protein in neurons across the brain, has the potential to be disease-modifying and provide a long-lasting treatment for STXBP1 encephalopathy. Based on these data and the well-tolerated efficacy profile of CAP-002, Capsida's STXBP1 program is targeting a 1H 2025 IND filing with the goal of achieving a disease-modifying treatment for patients suffering from STXBP1 encephalopathy. Disease Focus of Abstract:Central Nervous System Disorders"

Gene therapy • CNS Disorders • Cognitive Disorders • Developmental Disorders • Epilepsy • Gene Therapies • Mental Retardation • Psychiatry • XBP1

A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: Capsida Biotherapeutics, Inc.

New P1/2 trial • CNS Disorders • Epilepsy • Gene Therapies • Pediatrics

Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy (Businesswire) - "Capsida Biotherapeutics...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....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."

IND • New P1/2 trial • CNS Disorders

Capsida Announces AbbVie Opt-in for First Genetic Medicine Program from Neurodegenerative Disease Collaboration (PRNewswire) - "Capsida Biotherapeutics...announced that AbbVie has exercised an option for the first neurodegenerative disease program under their ongoing collaboration. Capsida will receive a $40 million license payment and is eligible for additional milestones and royalties...The opt-in decision was based on results from primate studies using Capsida's engineered adeno-associated virus (AAV) capsids to deliver AbbVie's novel therapeutic cargo intravenously (IV). Broad neuronal expression was achieved while simultaneously de-targeting the liver and dorsal root ganglia. AbbVie will be responsible for advancing the program into IND-enabling studies and performing clinical trials, while Capsida will be responsible for manufacturing....'In addition, we are looking forward to advancing two of our wholly owned CNS programs into clinical trials in the first half of 2025.'"

Licensing / partnership • New trial • CNS Disorders

Systemic Gene Therapy with Engineered AAV Demonstrates Preclinical Efficacy and Safety Supporting a Disease-Modifying Treatment for STXBP1 Developmental and Epileptic Encephalopathy (AES 2024) - "We also utilized non-human primates (NHPs) to assess the DNA and RNA biodistribution and safety of an engineered AAV vector designed to cross the blood-brain barrier after IV administration and deliver the therapeutic gene extensively across the brain. Our findings indicate that STXBP1 supplementation through AAV gene therapy reversed disease phenotypes in a dose-dependent manner across key behavioral domains, including robust and long-lasting correction of cognition and motor deficits and correction of myoclonic and spike-wave discharge seizures, with effects observed up to one-year post-treatment. These studies demonstrate potential for a first- and best-in-class, disease-modifying gene therapy for genetic epilepsy due to STXBP1 mutations. The CAP-002 gene therapy achieved levels of STXBP1 protein expression brain-wide in NHPs that were sufficient to produce meaningful and sustained phenotypic correction in the mouse model, highlighting the potential for significant..."

Gene therapy • Preclinical • CNS Disorders • Developmental Disorders • Epilepsy • Gene Therapies • Mental Retardation • Psychiatry • XBP1

Capsida Biotherapeutics to Present at 7th Annual Evercore HealthCONx Conference (PRNewswire) - "Capsida Biotherapeutics...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...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."

IND • Preclinical • CNS Disorders • Epilepsy • Parkinson's Disease

FDA Grants Orphan Drug Designation to Capsida Biotherapeutics for Potential Treatment of STXBP1 Developmental and Epileptic Encephalopathy (PRNewswire) - "Wholly owned first-in-class program anticipated to enter clinical trials in first half of 2025...Capsida Biotherapeutics...announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Capsida for CAP-002....CAP-002 is currently in IND-enabling studies....'We are committed to advancing CAP-002, with our IND filing in the first half of 2025...'"

IND • New trial • Orphan drug • CNS Disorders • Epilepsy

Capsida Biotherapeutics to Participate in Upcoming Investor and Industry Conferences Highlighting Progress With its Wholly Owned Programs in IND-enabling Studies (PRNewswire) - "Capsida Biotherapeutics...announced that the company will participate in the following investor and industry conferences in September and October. Management will highlight the progress with Capsida's wholly owned IV-administered programs – CAP-002 in genetic epilepsy due to STXBP1 mutations and CAP-003 in Parkinson's disease associated with GBA mutations. Both programs are on track to enter the clinic in the first half of 2025."

New trial • Preclinical • CNS Disorders • Epilepsy • Parkinson's Disease