Research

Taylor’s Tale is working with academic institutions, the pharmaceutical industry, government agencies and other patient organizations to develop lifesaving treatments and change how doctors approach devastating rare diseases.

For nearly a decade, Taylor’s Tale has been a leading supporter of infantile Batten disease research. Our donors have helped advance important work in gene therapy, enzyme replacement therapy and small molecule drug therapy.

In 2013, Taylor’s Tale helped start an innovative gene therapy research program for Batten disease at the University of North Carolina at Chapel Hill. This treatment developed by Steven Gray, PhD, is the first-ever gene delivery approach administered directly into the spinal fluid to treat a neurological disorder.

In September 2016, Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, added Dr. Gray’s promising work to its pipeline. The gene therapy program is anticipated to enter clinical trials in 2017, meaning children like Taylor could, for the first time, have a meaningful treatment. Our dream is coming true.

This groundbreaking and innovative research is also paving the way to life-changing treatments for other diseases such as Alzheimer’s disease, Lewy body dementia, ALS (Lou Gehrig’s disease), metabolic diseases and many others.