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M0X4
- Development stage
- Preclinical
- Lead developer
- X4 Pharmaceuticals
- Modality
- Acoustic/Optical: Sonoporation, laser photoporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Mechanical: Microinjection, microfluidics, gene gun → Physical Methods → Gene Addition/Replacement → Gene Therapies, Electrical: Electroporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Pressure-driven: Hydrodynamic injection → Physical Methods → Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Magnetic: Magnetofection → Physical Methods → Gene Addition/Replacement → Gene Therapies, Lipid-mediated Delivery → Gene Addition/Replacement → Gene Therapies
01
Overview
M0X4 is a preclinical-stage non-viral gene therapy program being developed by M6P Therapeutics. It utilizes a proprietary non-viral gene delivery platform designed to enable the expression of therapeutic genes for the treatment of multiple lysosomal storage disorders (LSDs). The platform aims to overcome limitations associated with viral vectors, such as immunogenicity and limited cargo capacity, while leveraging M6P Therapeutics' expertise in lysosomal targeting to ensure efficient delivery and processing of therapeutic enzymes within the lysosome. The program is currently in the preclinical phase of development.
- Other names
- M0X4 (non-viral)
02
Targets
mPR (Membrane progesterone receptors)IGF2R (Cation-independent mannose-6-phosphate receptor)Neuraminidase
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