Blu Genesis raising funds to advance gene therapy for rare disease.  

There is tremendous research focused on rare genetic disorders being developed by talented researchers whose life's work is to treat or cure rare diseases.  Their work impacts a small population of people per disease and therefore presents a challenge to secure the large grants needed to progress their work to a clinical trial and ultimately a treatment option for patients.  There are over 7000 rare disease and Blu Genes is here to help spotlight and advance this research and offer hope to families facing a rare disease diagnosis.

Blu Genes has already helped to propel research to the first in-human clinical trial in history for a rare neurological disorder called Tay-Sachs disease.  We have now joined with SickKids in Toronto to continue research towards editing the disease causing gene, with hopes that it can be removed forever.  We have also joined with a team to advance research for Spastic Paraplegia Type 4 (SPG4) A genetic disease and neurological disorder that slowly causes paralysis in young children.

Rare disease research may treat small populations of people, but as scientists continue to break the code of gene therapies and gene editing, these thousands of diseases will begin to reveal how treatments find commonalities that allow for mutual gain and treatment impact.