Our Vision and Team

On February 8th, 2024, we received the news of Elly’s diagnosis. In the hospital, our doctor shared what little he knew about the disease. Although there is no cure or readily available treatment, he gave us hope that gene therapy is a possibility.  After further research and countless calls with experts we immediately set upon a path to pursue this novel therapy.

We are thankful that in a few short months, we brought together a team of experts in gene therapy and drug discovery. Additionally, we assembled a remarkable group of therapists and doctors to join Elly’s Team.

Elly’s Team is a foundation established with a singular goal of translating medical research to treatment in record time. Our path to treatment involves:

1. Funding additional research to understand the disease mechanism and effects on the body
2. Design and discover treatment modalities
3. Conduct safety and toxicity studies for treatment options
4. Develop and manufacturing drugs
5. Navigate the FDA approval process and clinical trials
6. Dose and treat children!

While traditionally these steps are sequential, we are advancing multiple steps simultaneously to shorten the timeline for translating research into treatment. We are also funding various treatment options concurrently, including drug repurposing, gene replacement, antisense oligonucleotides (ASOs), and gene editing.

In the future, another family will sit in the hospital and receive the same diagnosis, but their doctor will tell them there is a path to treatment.  Help us make this future a reality now. Please join Elly’s Team.