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
Nr. of Employees
small (1-50)
ERAD Therapeutics
900 Foulk Road, Suite 201, Wilmington, DE 19803, United States
Patents
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
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.
Out-licensing or collaborative development of modular gene therapy technologies and engineered biomolecules for rare disease indications.
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.
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
News & Updates
Presentation of preclinical research on glycosphingolipids and treatment approaches for protein misfolding diseases at the Gordon Research Conference (Galveston, TX).
Presentation entitled on ERAD blockade correcting CFTR-dependent saliva secretion in CF mice at the ICCF 2018 in London, UK.
Regulatory orphan drug designation granted for a candidate intended to treat Gaucher disease.
Issued and pending patents covering platform technologies, with stated coverage extending to 2044 and beyond.
Presentation of preclinical research on glycosphingolipids and treatment approaches for protein misfolding diseases at the Gordon Research Conference (Galveston, TX).
Presentation entitled on ERAD blockade correcting CFTR-dependent saliva secretion in CF mice at the ICCF 2018 in London, UK.
Regulatory orphan drug designation granted for a candidate intended to treat Gaucher disease.
Issued and pending patents covering platform technologies, with stated coverage extending to 2044 and beyond.