Capsida Biotherapeutics develops targeted, intravenously administered gene therapies using proprietary engineered capsids that achieve over 70% neuronal expression while minimizing liver targeting. The company focuses on treating rare and common central nervous system disorders, including STXBP1 developmental and epileptic encephalopathy and Parkinson’s disease associated with GBA mutations.
Funding
$300M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
AFounders
Product
Problem
Current gene therapies often struggle with targeted delivery to the central nervous system (CNS) while minimizing off-target effects in other organs like the liver and dorsal root ganglion (DRG). Achieving sufficient neuronal expression throughout the brain remains a challenge, limiting the therapeutic potential for many neurological disorders.
Solution
Capsida Biotherapeutics develops intravenously administered gene therapies that utilize engineered capsids to enhance targeted delivery to the CNS. Their proprietary capsids achieve high levels of neuronal expression, exceeding 70% in certain brain regions, while simultaneously reducing targeting to the liver and DRG. This approach aims to improve the therapeutic index of gene therapies for both rare and common neurological diseases. Capsida's integrated platform encompasses capsid engineering, cargo optimization, translational biology, process development, manufacturing, and clinical development.
Target Audience
The primary target audience includes patients with rare and common central nervous system disorders, as well as their caregivers and advocacy groups.
Features
- Engineered capsids with enhanced tropism for CNS tissues and reduced off-target effects in the liver and DRG
- Intravenous administration for non-invasive delivery
- High neuronal expression throughout the brain, exceeding 70% in certain regions
- Broad cargo delivery capabilities, including gene replacement, gene editing, gene silencing, and vectorized antibodies
- Integrated platform encompassing capsid engineering, cargo optimization, translational biology, process development, and manufacturing
- Pipeline programs targeting STXBP1 developmental and epileptic encephalopathy, Parkinson’s disease associated with GBA mutations, and Friedreich’s ataxia