A message of hope & thanks from BGF’s Board of Directors – 2022

To all of our supporters, sponsors, advocates and friends,
This year has been filled with some great gains and advancements for research as well as some setbacks and personal loss.

Our mission remains the same; to offer hope to patients, families, and the rare disease community. We are committed to raising funds to advance gene therapy research for those life-saving research projects that don’t always get the spotlight or allocated budgets needed to progress.

We thank each and every one of our donors, sponsors, event participants and advocates for their support; we couldn’t do this without you.

Looking back over the past five years, Blu Genes has made great strides. We have partnered with two prominent North American research hospitals to propel research initiatives that offer real and tangible treatments for infants with Tay-Sachs disease.

We share this success with each of you who have made it possible.

In 2017, we embarked on a mission to launch a charitable foundation to specifically seek out and support critical research needed to save the lives of infants and children faced with rare genetic diseases. Blu Genes Foundation was created from a personal mission, as the granddaughter of our founders was diagnosed with Tay-Sachs disease, a rare and fatal genetic disorder for which there is no cure. Siena was the spark that started this mission and Blu Genes will forever be her legacy as we continue to fund research that will provide meaningful treatments and hopefully one-day, a cure.

Our founders discovered that great research is taking place across the globe for rare diseases like Tay-Sachs. However, they are seriously under-funded and often stall due to financial hurdles. Five years later, we continue to raise funds to advance gene therapy and strategically invest our resources in world-class research.

Our initial philanthropic investment was with the University of Massachusetts. It became the catalyst in advancing a decade of research for a gene therapy treatment for Tay-Sachs to the first in-human clinical trial of its kind in the world. This extraordinary achievement continues with the progressing trial and goal of treatment being available in the clinic within the near future.  Fundraising continues for this trial as industry partners ebb and flow with invested support, but our foundation and many other advocacy groups, continue to push this research forward.

Blu Genes has now joined with SickKids Hospital in launching gene editing research here in Toronto. This is a tremendous achievement as SickKids Research Institute is Canada’s largest hospital-based child health research institute and has the expertise and ability to translate ground-breaking research into in-clinic treatments. Dr. Zhenya Ivakine is focusing his gene editing expertise on Tay-Sachs disease – working towards a treatment with curative potential. A collaboration with SickKids is a great honour, and we are excited for you to share in this success.

We are filled with hope as we move into a new season of opportunity in finding treatments for Tay-Sachs disease.

Thank you for being an important part of the Blu Genes Foundation’s success story.

Sasha Cucuz
Sara Margani
Maria Crignano
Nick Torchetti

Sam Crignano
Peter Politis
Gianpiero Di Rocco



Nicola Dent,

BGF Executive Director