ERAD Therapeutics, Inc. is currently developing our proprietary disease-specific gene therapy for targeted diseases and our broad-based mCT-gene therapy for delivery to a broad spectrum of diseases, including across the blood brain barrier (BBB) and into CNS tissues.
1/ Our novel gene therapy vector is initially being developed for targeted gene therapy in neuromuscular diseases (TBA).
2/ Many proteins that harbor a single amino acid mutation, though slightly mis-folded, still retain activity. Normally these mis-folded proteins are destroyed by the endoplasmic reticulum (Endoplasmic Reticulum-Associated Degradation aka ERAD). Our mCT-GT candidate is under initial development in certain lysosomal storage diseases, but has the capability of treating many rare genetic diseases based on successes in pre-clinical development in a variety of diseases. Our mCT-GT used in this approach is expected to have the ability to reversibly interfere with the ERAD process, allowing the mis-folded, but functional protein, to transit to its site of action where it can restore normal or near-normal cellular activity in multiple diseases creating a therapeutic platform.