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COL4A5 gene therapy vector
- Development stage
- Preclinical
- Lead developer
- Sonotherapeutics
- Modality
- Lipid-mediated Delivery → Gene Addition/Replacement → Gene Therapies, Electrical: Electroporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Magnetic: Magnetofection → Physical Methods → Gene Addition/Replacement → Gene Therapies, Mechanical: Microinjection, microfluidics, gene gun → Physical Methods → Gene Addition/Replacement → Gene Therapies, Acoustic/Optical: Sonoporation, laser photoporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Pressure-driven: Hydrodynamic injection → Physical Methods → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
The COL4A5 gene therapy vector is an investigational nonviral gene therapy being developed by SonoThera for the treatment of X-Linked Alport Syndrome (XLAS). The therapy utilizes SonoThera's proprietary RIPPLE (Remote Induced Pressure-Pulse Localized Emission) platform, which employs ultrasound-mediated delivery (UMD) to deliver a full-length COL4A5 gene construct to target cells, specifically in the kidney. By delivering the full-length gene, the therapy aims to restore the production of the type IV collagen alpha 5 chain, which is deficient in patients with XLAS, a hereditary condition leading to progressive renal dysfunction, hearing loss, and ocular abnormalities. Preclinical data presented at the American Society of Nephrology's Kidney Week 2025 demonstrated successful expression of the full-length COL4A5 gene in both murine and non-human primate models.
- Other names
- SonoThera COL4A5 gene therapyCOL4A5 nonviral gene therapyCOL-4A5 nonviral gene therapyCOL 4A5 nonviral gene therapyRIPPLE COL4A5 therapy
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