ERAD Therapeutics
Private, pre-clinical biotherapeutics company focused on developing gene- and protein-based therapies for rare genetic (orphan) diseases. The company develops a modular gene therapy platform and a modified, inactivated toxin–derived biomolecule that transiently interferes with Endoplasmic Reticulum Associated Degradation (ERAD) to rescue misfolded but partially functional proteins. Development emphasis includes neuromuscular diseases, lysosomal storage diseases (including Gaucher disease), and cystic fibrosis, with preclinical data in cell lines and animal models, an orphan drug designation for Gaucher disease, and a patent portfolio protecting platform technologies.
Industries
Products
Modified, inactivated toxin–derived biomolecule for ERAD inhibition
A genetically inactivated toxin-derived biomolecule engineered to enter cells, transiently interfere with ERAD, and rescue misfolded but functional proteins to restore cellular activity.
Gene therapy encoding ERAD-modulating biomolecule (gene-based platform)
A gene therapy approach that encodes the ERAD-modulating biomolecule to enable sustained or controlled expression and delivery across tissues, including CNS.
Proprietary plasmid vector for large-payload delivery
A plasmid/vector system developed to carry larger genetic payloads for gene therapy applications, intended to improve payload capacity and production efficiency.
Modified, inactivated toxin–derived biomolecule for ERAD inhibition
A genetically inactivated toxin-derived biomolecule engineered to enter cells, transiently interfere with ERAD, and rescue misfolded but functional proteins to restore cellular activity.
Gene therapy encoding ERAD-modulating biomolecule (gene-based platform)
A gene therapy approach that encodes the ERAD-modulating biomolecule to enable sustained or controlled expression and delivery across tissues, including CNS.
Proprietary plasmid vector for large-payload delivery
A plasmid/vector system developed to carry larger genetic payloads for gene therapy applications, intended to improve payload capacity and production efficiency.
Services
Preclinical research and development of gene- and protein-based therapeutics
End-to-end preclinical R&D services including plasmid/vector design, biomolecule engineering, cell-based assays, and animal model testing to advance candidates toward clinical development.
Platform licensing and partnership
Out-licensing or collaborative development of modular gene therapy technologies and engineered biomolecules for rare disease indications.
Preclinical research and development of gene- and protein-based therapeutics
End-to-end preclinical R&D services including plasmid/vector design, biomolecule engineering, cell-based assays, and animal model testing to advance candidates toward clinical development.
Platform licensing and partnership
Out-licensing or collaborative development of modular gene therapy technologies and engineered biomolecules for rare disease indications.
Expertise Areas
- Gene therapy platform development
- Protein engineering for therapeutic modulation of cellular pathways
- Preclinical development (cell-line assays and animal models)
- Plasmid vector design and manufacturing
Key Technologies
- Plasmid-based gene delivery
- Gene therapy vector engineering
- Protein engineering of modified toxin-derived biomolecules
- ERAD pathway blockade/modulation