TransformaTx Biotherapeutics LLC is dedicated to advancing groundbreaking therapies for the treatment of rare genetic disorders, starting with Angelman syndrome. Born out of a partnership with the Foundation for Angelman Syndrome Therapeutics (FAST), TransformaTx was formed to bring the investigational ube-cel lentiviral gene therapy program to early-stage clinical trials.

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​Ube-cel was developed through a 2016 research collaboration with the University of California at Davis and the University of California at Los Angeles and demonstrated significant promise in rescuing the broad phenotype of both newborn and adult mice with Angelman syndrome in nonclinical studies. This cutting-edge technology is designed to restore UBE3A protein production which is missing or deficient in individuals living with Angelman syndrome.​