Your Impact on Advancing Gene Therapy

Our loyal supporters, both individual and corporate, have made research possible. Each donation gifted to Blu Genes granted the ability for Blu Genes Foundation to have made the $1.8 million commitment in 2018 to the University of Massachusetts.

In our endeavor to seek preeminent gene therapy programs in the world, Blu Genes came across the University of Massachusetts Medical School where a remarkable team of world class researchers focused on treating Tay Sachs disease. When we discovered that this research could ultimately help children in the immediate future, Blu Genes realized this was something incredibly meaningful as it presented a real chance of changing the face of this disease forever.
https://blugenes.org/blugenes-news-updates/tay-sachs-sees-hope-first-gene-therapy-clinical-trial/

Blu Genes Foundation’s commitment, with your support, ultimately advanced this gene therapy research to being the first-in-human clinical trial and a world first treatment option for Tay Sachs disease. This support was the catalyst in moving research from the preclinical phase to human trials. It allowed researchers to take more than a decade of scientific discovery into the clinic, where it will directly impact patient lives.

This gene therapy received FDA clearance of Investigational New Drug (IND) and is the first treatment for Tay-Sachs disease to enter the clinic. Through this clinical trial 9 patients were treated with the gene therapy.  The higher dose of the therapy has had the greatest impact.  We are proud that our first major philanthropic investment was a great success and for the first time in history patients with Tay-Sachs disease have participated in a clinical trial with a gene therapy.

This therapy made great strides in advancing research for a viable treatment for Tay-Sachs disease that will prolong patients lives in a meaningful way. The team at University of Massachusetts have now focused their talent on enhancing the vector or delivery of the gene therapy in hopes of improving the outcomes for patients in a future trial.

We now look to our next opportunity to advance gene therapy. There is tremendous research focused on rare genetic disorders being developed by talented researchers whose life work it is to treat or cure these underfunded, under-recognized rare genetic diseases and disorders. Blu Genes was created for this very purpose, we will continue to raise funds to advance gene therapy and strategically invest our donors support in world class research to make an impact and continue to offer hope to patients and families faced with rare genetic disorders.

Donate today to advance research and provide hope