We are looking for drugs for Wolfram syndrome to prevent or halt the progression of the disease. In parallel, we are developing “cell-based” therapies.
What is “cell-based” therapy? What I mean by this is the following.
1. Take the cells from patients.
2. Correct the genetic mutations and add additional features to the cells to enhance viability.
3. Bring these cells back to patients.
This strategy has been successful in the cancer field, and has provided a cure for patients with certain forms of cancer. I will elaborate this and keep you updated about our progress in my future blogs.