Transformatx Biotherapeutics

The investigational lentiviral therapy called ube-cel, first came out of a 2016 sponsored research agreement with the University of California at Davis. The technology resulted in broad phenotype rescue in both newborn and adult mice with Angelman syndrome, similar to their wild-type counterparts.

Transformatx is working closely with Donald Kohn, MD of the UCLA Broad Stem Cell Research Center and his research team to advance this important program Dr. Kohn has worked for over 35 years on the development of therapies to treat genetic diseases, which includes extensive work on hematopoietic stem cell gene therapy.

Here is a recent update on this program from the 2023 FAST Science Summit. The efficacy of this approach in the mouse is remarkable.