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.

CLN1 disease (Batten disease)

Since 2007, Taylor’s Tale has been a leading supporter of CLN1 disease (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 spearheaded an innovative gene therapy research program for Batten disease at UNC 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 2021, Taysha Gene Therapies announced the addition of Dr. Gray’s CLN1 disease approach to its pipeline. The clinical trial is expected to begin in late 2021. If successful, children like Taylor could, for the first time, have a meaningful treatment.

This groundbreaking 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.