Drugs / CAP-002
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06983158 | Jul 2025 → Mar 2026 | developmental and epileptic encephalopathy | Capsida Biotherapeutics, Inc. | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2025-05-29 | Capsida Biotherapeutics, Inc. | Regulatory Capsida Receives FDA Fast Track Designation for Its Potential First-in-Class IV-Administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy capsida.com ↗
Capsida Biotherapeutics (“Capsida”) today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its CAP-002 program. |
| 2025-05-14 | Capsida Biotherapeutics, Inc. | Results 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.com ↗
The three-month cohort data from the NHP GLP toxicology study demonstrate dose-dependent brain-wide expression of STXBP1 and simultaneous detargeting of the liver and dorsal root ganglia (DRGs). |
| 2025-05-12 | Capsida Biotherapeutics, Inc. | Regulatory Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy capsida.com ↗ |
| 2025-04-28 | Capsida Biotherapeutics, Inc. | Results 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.com ↗
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. |
| 2024-12-06 | Capsida Biotherapeutics, Inc. | Results New Preclinical Data Demonstrate Potential of Capsida’s IV Gene Therapy for STXBP1-DEE to Correct all Disease Manifestations capsida.com ↗
The new data in non-human primates (NHPs) and human cells establish the potential for CAP-002 to significantly correct seizures, motor abnormalities, and developmental/intellectual disabilities in STXBP1-DEE patients. |
| 2024-10-30 | Capsida Biotherapeutics, Inc. | Regulatory FDA Grants Orphan Drug Designation to Capsida Biotherapeutics for Potential Treatment of STXBP1 Developmental and Epileptic Encephalopathy capsida.com ↗
Capsida Biotherapeutics (“Capsida”) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Capsida for CAP-002. |
| 2024-05-07 | Capsida Biotherapeutics, Inc. | Results Capsida Biotherapeutics Presents New Preclinical Evidence Indicating Novel First-in-Class IV-Administered Gene Therapy Effectively Treats Genetic Epilepsy Due to STXBP1 Mutations capsida.com ↗
Capsida Biotherapeutics (“Capsida”) today announced new preclinical data supporting the potential of Capsida’s gene therapy candidate, CAP-002, to achieve levels of gene supplementation necessary to correct neurological phenotypes associated with genetic epilepsy due to syntaxin-binding protein 1 (STXBP1) mutations. |
| 2024-04-22 | Capsida Biotherapeutics, Inc. | Results Capsida Biotherapeutics to Present New Data on its Wholly Owned Gene Therapy Programs in Genetic Epilepsy and Parkinson’s Disease at the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) capsida.com ↗
Both development candidates are IV-administered and achieve breakthrough levels of neuronal transduction throughout the brain, up to 70% in certain brain regions, while simultaneously detargeting the liver. |
All press releases naming this drug 8 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | CAP-002 | “The goal of this clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy.” NCT06983158 ↗ |
| Route | Intravenous | “single intravenous infusion of CAP-002” NCT06983158 ↗ |