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Current Pipeline
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HSC-GT

TMX

Transformatx Biotherapeutics LLC (TMX) is exploring an investigational Hematopoietic Stem Cell Gene Therapy (HSC-GT) approach to treating Angelman Syndrome.

Pre-clinical

Discovery & Dev

Pre-clinical

Phase 1

Phase 2

Phase 3

To patients

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Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.

Therapeutic Approach

A therapeutic approach where an individual’s own bone marrow stem cells are removed from their body, modified ex vivo, or outside the body, and returned to the body with a replaced copy of the missing or nonfunctional gene (UBE3A in this case). Once the bone marrow cells are transplanted back into the individual, they go to the bone marrow to grow and repopulate. The goal is for those cells to continuously supply the body with a healthy version of the gene. These cells can cross from the blood to the brain, through the blood brain barrier (BBB). Once they cross the BBB they become a cell type called microglia and secrete the UBE3A protein throughout the brain for neurons to take it up and use it.

Media

  • Transformatx Update: Hematopoietic Stem Cell Gene Therapy Program

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.