Our 
Mission

There are currently over 12,000 Rare Diseases affecting 400 million people worldwide. Most of these affect young children who will never see the age of 5, what Leni is now.  After realizing that 95% of these have no cure, let alone any form of a treatment, we realized just how big this problem was, so much more than just our Leni. We knew had to help the countless others with similar diseases and no options or hope for a future.  

 

We have assembled the best team in the world and have done the impossible, creating a successful Gene Therapy CURE that has been developed and working in multiple animal models. This AAV9 therapy uses a virus to remove the defective or missing gene in ones DNA and replaces it with a healthy version, correcting the issue at the core and curing the disease. We have been able to accomplish this by establishing unprecedented partnerships with Dr Steven Gray at UTSW, Harvard Medical School, Boston Children's Hospital, Cambridge University, top biotech's, and the Weizmann Institute of Science in Israel.  

 

Our next step and most costly, is to have the Gene Therapy manufactured in a large enough batch for a clinical trial. This is where you come in!  The pieces are in place and we have done the impossible, we just need your help for the final push to FDA approval and providing a safe treatment for these affected children.

 

The Eleanor Kaplan Foundation has accomplished more than 99.9% ever have and with your help to bring this CURE and many others in our pipeline to completion. Through curing TECPR2, we have identified the key breakthroughs in Lysosomal Storage Disorders and Neurodegenerative Diseases. We have made revolutionary discoveries for treatments of many major diseases that affect millions, including Alzheimer's, ALS and Parkinson's.  It has been a very humbling, yet amazing journey of not accepting no for an answer and actually doing what no one thought could ever be done. With this tremendous progress, we have created the network and blueprint for all of the 12,000 other rare diseases. We are paving the way for the future of bespoke medicine and proving that these children's lives matter. We were given a true gift through a devastating diagnosis, and cant wait to help and share it with the world.