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.
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 has now received FDA clearance of Investigational New Drug (IND) and is officially the first potentially curative treatment for Tay-Sachs disease to enter the clinic. We are extremely excited to know that our first major philanthropic investment was a home run, and the therapy will now be treating infants and children.
Great thanks to our donors, for the first time in history, there is hope for Tay Sachs patients as this treatment can give them a chance at a new future.
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