UMass Chan launches Translational Institute for Molecular Therapeutics

Study supported by Blu Genes Published in Nature Medicine

Blu Genes Foundation made a commitment of $1.4million (USD) to the University of Massachusetts Chan Medical School to advance the Phase I/II clinical trial for the rare genetic disorder, Tay Sachs. Blu Genes gift was the catalyst in moving research from the preclinical phase to early stage human trials. Dr. Gray-Edwards shared “This philanthropic investment in our Tay-Sachs research from Blu Genes Foundation will allow us to take more than a decade of scientific discovery into the clinic, where our novel gene therapy approach will directly impact patient lives.”

“This week, an important milestone in human gene therapy was reached. Dr. Sena-Esteves, associate professor of neurology and a member of the Horae Gene Therapy Center at UMass Chan, and colleagues reported on the first ever gene therapy for infantile Tay Sachs to reach clinical trials in humans. Published in Nature Medicine, the study shows that an adeno-associated virus (AAV) gene therapy can be safely administered to infants and further, it can increase enzymatic activity and improve imaging biomarkers.”