CAP-003 / Capsida Biotherap - LARVOL DELTA

CAP-003, a CNS-targeted IV-delivered AAV Gene Therapy Developed for Patients with Parkinson's Disease associated with GBA1 Mutations (MDS Congress 2025) - "CAP-003, targeting IND submission in 1H 2025, aims to achieve disease modifying clinical benefit for PD-GBA patients through a single intravenous dose."

Clinical • Gene therapy • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=31 | Suspended | Sponsor: Capsida Biotherapeutics, Inc. | Recruiting ➔ Suspended

Trial suspension • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=31 | Recruiting | Sponsor: Capsida Biotherapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=31 | Not yet recruiting | Sponsor: Capsida Biotherapeutics, Inc.

New P1/2 trial • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson’s Disease Associated With GBA Mutations (Businesswire) - "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....Capsida expects to dose the first patient in the Phase 1/2 clinical trial in the third quarter of this year."

IND • Trial status • Parkinson's Disease

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 (ASGCT 2025) - "Targeting a 1H 2025 IND filing, CAP-003 is being advanced with the goal of achieving disease modifying clinical benefit for patients with PD-GBA through a convenient single dose intravenous administration. Disease Focus of Abstract:Parkinson's Disease"

Clinical • Gene therapy • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease • GBA • GBA1

Capsida Presents New Data from GLP Toxicology Study in NHPs Demonstrating Potential Best-in-Class Profile of CAP-003 in Patients with Parkinson's Disease Associated with GBA Mutations (PD-GBA) (PRNewswire) - "Capsida Biotherapeutics...today announced new data from the three-month cohort of a GLP toxicology study in non-human primates (NHPs) with CAP-003, its intravenously (IV) delivered gene therapy for Parkinson's disease associated with GBA mutations (PD-GBA). CAP-003, enabled by one of Capsida's proprietary engineered capsids and cargo, demonstrates best-in-class potential to effectively and safely treat PD-GBA. These data will be presented in oral presentations at the International Conference on Alzheimer's and Parkinson's Diseases (AD/PD) being held April 1-5, 2025, in Vienna, Austria, and the American Academy of Neurology (AAN) annual meeting taking place April 5-9, 2025, in San Diego, CA. Capsida is on track to file an Investigational New Drug (IND) application in the second quarter of 2025."

IND • Preclinical • Parkinson's Disease

SYSTEMIC AAV GENE THERAPY WITH CNS-TARGETED ENGINEERED CAPSIDS SIGNIFICANTLY INCREASES GCASE ACTIVITY TO SUPPORT THE POTENTIAL TREATMENT OF PD-GBA (ADPD 2025) - "In addition, CAP-003 is well tolerated, de-targeted from the liver and DRGs, and lacks clinical pathology or immunogenicity associated with higher-dose systemic gene therapies. Conclusions Targeting a 1H 2025 IND filing, CAP-003 is being advanced with the ultimate goal of achieving disease modifying clinical benefit for patients with PD-GBA through a convenient single dose IV administration."

Gene therapy • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease • GBA • GBA1

Systemic AAV Gene Therapy Using a CNS-targeted Engineered Capsid Significantly Increases GCase Activity to Support the Potential Treatment of PD-GBA (AAN 2025) - "Objective:Capsida has developed a next-generation gene therapy candidate (CAP-003) for administration as a single intravenous (IV) infusion to Parkinson's disease patients with GBA1 mutations (PD-GBA).Background:Parkinson's disease (PD) is the second most common neurodegenerative disorder in the United States with no approved disease modifying treatments to date. Targeting a 1H 2025 IND filing, CAP-003 is being advanced with the goal of achieving disease modifying clinical benefit for patients with PD-GBA through a convenient single dose IV administration."

Gene therapy • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease • GBA • GBA1

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

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

Systemic AAV gene therapy with CNS-targeted engineered capsids achieves significant GCase activity increases in the primate brain to support the potential treatment of PD-GBA (Neuroscience 2024) - "Parkinson’s disease (PD) is the second most common neurodegenerative disorder with an estimated prevalence of over 1.2 million people in the United States by 2030 and no approved disease modifying treatments today. Importantly, these levels of GCase supplementation are achieved at doses that are well tolerated and without any clinical pathology or immunogenicity findings, including a lack of histopathology in the liver and dorsal root ganglia, that are associated with higher-dose systemic gene therapies. Taken together, CAP-003 is being advanced with the ultimate goal of achieving disease modifying clinical benefit for patients with PD-GBA through a convenient single dose IV administration."

Gene therapy • Late-breaking abstract • CNS Disorders • Parkinson's Disease • GBA

New Data Demonstrate Substantial Therapeutic Potential of Capsida's IV Gene Therapy for Parkinson's Disease Associated with GBA Mutations (PRNewswire) - "Results from Capsida's IND-enabling non-human primate (NHP) studies show brainwide RNA expression more than 200-fold greater than IV administered AAV9 and substantial increases in GCase protein and enzyme activity compared to untreated animals. A single IV infusion of CAP-003 resulted in up to six-fold increase in GCase protein compared to untreated animals in relevant brain regions. Across doses and brain regions, including the substantia nigra, the levels of GCase activity following infusion of CAP-003 was increased by up to approximately 250% compared to untreated animals. These data support the potential for meaningful clinical benefit in patients. Increases of 30% are expected to normalize GCase activity in patients with PD-GBA...'These data give us confidence that we are on track to enter the clinic with CAP-003 in the first half of 2025'...In addition, Capsida will present these data as part of a corporate presentation at the Cell & Gene Meeting on..."

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

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

Capsida Biotherapeutics to Present Preclinical Data for Parkinson’s Disease Associated with GBA Mutations Showing High Levels of GCase Enzyme Supplementation Following IV Administration (PRNewswire) - "Capsida Biotherapeutics...today announced new preclinical data that demonstrate the potential of...CAP-003, to safely and effectively treat Parkinson's disease (PD) associated with GBA mutations (PD-GBA)...Capsida's plans to advance the therapy into clinical trials in the first half of 2025...In non-human primates (NHPs), CAP-003 transduced the majority of neurons, including critical sub-cortical regions, resulting in GCase enzyme activity levels that exceed the threshold anticipated to restore GCase function in patients with PD-GBA. Importantly, CAP-003 also demonstrated significant liver detargeting compared with AAV9 with no clinical pathology, immunogenicity, or histopathology findings, including in dorsal root ganglia (DRGs)."

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