Ocular AAV capsid engineering program
preclinicalresearch program · high · Thu Jan 01 2026 00:00:00 GMT+0000 (Coordinated Universal Time)
Identify high-performing ocular AAV capsids for eye indications across intravitreal and suprachoroidal delivery routes.
Directed engineering of AAV capsids for ocular delivery.
2026 ASGCT publication/poster on identification of high-performing ocular AAV capsids.
Identified high-performing ocular AAV capsids through directed engineering across intravitreal and suprachoroidal routes.
CAP-003 for GBA1-associated Parkinson's disease
phase 1drug program · high · Mon Aug 18 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Treat adults with GBA1-associated Parkinson's disease and evaluate safety and evidence of efficacy.
Single intravenous infusion of CAP-003, an investigational CNS-targeted intravenous gene therapy, with 2 years of follow-up visits and tests.
Clinical trial in adult patients with GBA1-associated Parkinson's disease published on 2025-08-18.
CAP-002 for STXBP1 encephalopathy
phase 1drug program · high · Thu Jul 03 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Treat pediatric patients with Syntaxin-Binding Protein 1 (STXBP1) encephalopathy, including patients with and without seizures.
Single-infusion gene therapy using Capsida's CNS-targeted AAV genetic medicine approach; trial participants are followed for 2 years with clinic visits, testing, and caregiver seizure diaries.
Clinical trial in pediatric patients with STXBP1 encephalopathy published on 2025-07-03.
Preclinical/publication material states CAP-002 showed potential for disease-modifying treatment of seizures and motor and cognitive deficits in STXBP1-DEE.
CAP-004 for Friedreich's ataxia
preclinicaldrug program · medium · Wed Jan 01 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Treat CNS and cardiac symptoms in Friedreich's ataxia.
Systemic AAV gene therapy using engineered capsids.
2025 publication/poster listed for CAP-004 systemic AAV gene therapy in Friedreich's ataxia.
CNS-targeted AAV capsid engineering platform
exploratoryplatform · high · Wed Jan 01 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Engineer AAV capsids that cross biological barriers and deliver genetic medicines to CNS indications while reducing off-target delivery such as liver targeting.
Integrated AAV capsid engineering platform combining capsid engineering, preclinical work, and in-house cGMP manufacturing capabilities.
2025 publication listed on identification of multiple novel blood-brain-barrier receptors for CNS gene therapy and other drug modalities.
Published work describes engineered AAV variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset, and AAV.CAP-Mac with improved delivery in multiple non-human primate species.
STXBP1 encephalopathy natural history study
exploratoryresearch program · high · Tue Oct 01 2024 00:00:00 GMT+0000 (Coordinated Universal Time)
Characterize the natural history of STXBP1-related encephalopathy with epilepsy in pediatric participants under 5 years of age.
Prospective, non-interventional, longitudinal natural history study.
ClinicalTrials.gov record published on 2024-10-01.
AAV.CAP-Mac non-human primate brain delivery research program
preclinicalresearch program · high · Sat Jul 01 2023 00:00:00 GMT+0000 (Coordinated Universal Time)
Enable non-invasive systemic gene transfer throughout the brain of non-human primates.
Engineered AAV variant identified by screening in adult marmosets and newborn macaques; single intravenous dose used to deliver functional GCaMP or fluorescent reporter cocktails across brain regions.
2023 Nature Nanotechnology publication on functional intravenous gene transfer throughout the non-human primate brain.
AAV.CAP-Mac showed improved brain delivery across marmoset, rhesus macaque, and green monkey; it was neuron-biased in infant Old World primates, broad-tropic in adult rhesus macaques, and vasculature-biased in adult marmosets.
Amyotrophic lateral sclerosis genetic medicine program
undiscloseddrug program · medium
Develop a targeted genetic medicine program for amyotrophic lateral sclerosis.
Capsida pipeline program using its targeted genetic medicines and engineered AAV capsid platform.
Listed in Capsida's pipeline.
