At TransformaTx Biotherapeutics LLC, our approach to treating rare genetic disorders is built on a foundation of scientific excellence, collaboration, and a deep commitment to patient well-being.

 

We understand that developing therapies for rare diseases requires more than just innovation; it requires a focus on safety, precision, and a relentless drive to overcome challenges and deliver results that can potentially transform lives.

Our approach is patient-centric, driven by a clear vision: to provide hope and potentially transformative therapies for individuals and families affected by Angelman syndrome and other rare genetic disorders.

 

Through innovation, rigorous research, and unwavering dedication, we are committed to transforming the future of rare disease treatment.

Our lead development program is an investigational lentiviral gene therapy (ube-cel) developed in collaboration with the University of California at Davis.

 

Designed to restore the production of the UBE3A protein, which is missing or deficient in individuals living with Angelman syndrome, ube-cel represents a novel approach the treatment of rare neurological disorders. The robust phenotypic effects of ube-cel in animal models of Angelman syndrome has laid the foundation for its continued development.

Our approach goes beyond scientific innovation; we are committed to collaboration at every step.

 

By working closely with the Foundation for Angelman Syndrome Therapeutics (FAST), renowned scientists, and clinicians, we ensure that every decision is made with patient safety and the integrity of the science in mind.

We embrace a spirit of partnership and inclusivity, bringing together diverse expertise to tackle the unique challenges of developing gene therapies for rare diseases.