Our unique gene therapy platform enables high-efficiency, cost-effective approaches to gene therapy. This allows us a two-fold approach to treatment of rare genetic diseases:
- Targeted, disease-specific gene therapy therapeutics with an initial focus on neuromuscular diseases
- A broad-based, “gene-agnostic” platform approach pursuing multiple rare genetic diseases with a single gene therapy product
ERAD Therapeutics Inc. has also developed a novel biomolecule called mCT to rescue mutated, misfolded proteins from destruction by Endoplasmic Reticulum Associated Degradation (ERAD). This unique biomolecular platform employs a modified, inactivated cholera toxin (mCT) that has had its cytotoxic activity eliminated by genetic manipulation of the DNA. mCT can enter all cells in the body, and is capable of crossing the blood brain barrier.
mCT has been demonstrated to rescue mutated, misfolded, partially functional proteins in multiple rare genetic diseases, including cystic fibrosis and several lysosomal storage diseases, by interfering with their destruction. These accomplishments give us a “gene-agnostic” approach to treatment of numerous rare and very rare genetic diseases with unmet clinical needs. mCT received Orphan Drug Designation for Gaucher Disease in 2017
Recent developments enable us to take a gene therapy approach for mCT (mCT-GT) offering new hope to many patients, families and stakeholders.