Context
Help Lily Bloom was established in honour of Lily, who is living with an ultra-rare genetic condition caused by a mutation in the CASK gene. Fewer than 600 individuals have been diagnosed with CASK-related disorders worldwide.
CASK-related disorders are severe neurological conditions that affect brain development and can impair movement, coordination, communication and other essential functions. There is currently no cure or approved treatment targeting the underlying genetic cause of these disorders.
Two promising gene therapy research programmes are exploring complementary approaches to address the genetic cause of CASK-related disorders. Both are currently at the preclinical stage, where further research is required to establish their safety, optimal dosing and effectiveness before clinical trials can be considered.
Goals
The initiative aims to accelerate the development of gene therapies targeting the underlying genetic cause of CASK-related disorders and bring potential treatments closer to clinical application.
A fundraising target of USD 1 million to USD 1.5 million has been set to support the next stages of research and development, generate the scientific evidence required to advance towards clinical trials, and help attract future partnerships with biotechnology and pharmaceutical companies.
Activities
Donations will support two complementary research approaches:
- Gene replacement therapy: this programme focuses on delivering a healthy copy of the CASK gene to brain cells to compensate for the defective gene. The research received the 2025 Oxford-Harrington Rare Disease Scholar Award, providing access to drug development expertise and potential industry partnerships.
- CASK gene reactivation: this programme aims to reactivate the healthy copy of the CASK gene already present in girls with the condition but silenced in some of their brain cells. In March 2026, the successful activation of the healthy gene in laboratory-grown brain cells was demonstrated. Further research in mouse models is underway.
Myriad Canada is working with Texas Children’s Hospital and Baylor College of Medicine, where Dr. Mingshan Xue’s team is leading the gene replacement therapy programme, and the University of California, Davis (UC Davis), which is conducting research into CASK gene reactivation. These renowned institutions are advancing two complementary approaches, both of which require further preclinical research to establish the safety, optimal dosing and effectiveness of the proposed therapies before progressing towards potential clinical trials in children.
Myriad Canada’s role is to carry out charitable activities across the globe in accordance with its charitable purposes. We control and monitor the use of funds and the progress of charitable activities to ensure that donations made to Myriad Canada have the greatest impact. As a member of the Myriad Alliance with offices in Canada, the US, Europe, Asia and Australia, Myriad Canada has a vast international network and extensive practical expertise.
How to support this project?
By credit card: At the top right of this page, indicate the amount you wish to donate, and make the donation online by credit card. You will receive a receipt for tax purposes by e-mail just a few minutes after making the donation. It is simple and quick!
By cheque:
- Make your cheque out to Myriad Canada.
- IMPORTANT NOTE: We receive a lot of cheques! You must please indicate “Project G419 – Help Lily Bloom Fund” in the memo line of your cheque to ensure that it is allocated correctly.
- Mail your cheque to: Myriad Canada, 1 Place Ville Marie, Suite 1670, Montreal, QC, H3B 2B6, Canada.
By Direct Deposit:
- For transfer instructions, please email or call Caroline Chaulet: caroline@myriadcanada.org or +1 438 300 8122.
For Donation of Securities:
- Please email or call Caroline Chaulet: caroline@myriadcanada.org or +1 438 300 8122.
For Monthly Donations:
- We use CanadaHelps. Please find the link here.
If you need any assistance, please do not hesitate to contact us.
All donations are eligible for a tax receipt in Canada.
The projects are managed with the greatest care and professionalism.