I’m thrilled to share news of an incredible milestone achieved last week: FDA clearance of an investigational new drug (IND) application for a novel, one-time gene therapy to treat CLN1 disease (a form of Batten disease). This gives Abeona Therapeutics the green light to begin a Phase 1/2 clinical trial evaluating the treatment in patients. You can read the entire press release on Abeona’s website here.
Though we’re proud of everything we’ve done for children and others like Taylor, this is why we founded Taylor’s Tale more than a decade ago: to spearhead and enable the development of a viable treatment for CLN1 disease. So many called it an impossible mission, but we believed enough to try, even after it became clear Taylor wouldn’t survive long enough to benefit from such an achievement. The photo above was taken in the lab of Dr. Steven Gray, our partner and friend who developed the technology, on the day we celebrated the first birthday of mice treated with his innovative gene therapy approach. It was a happy day and a sign of more good things to come.
We’re grateful for Steve as well as Ale Rozenberg, who played an instrumental role in moving Steve’s work forward, and the entire team at Abeona Therapeutics for all they’ve done to guide it to the clinical stage. Thanks also to you, our donors, friends and volunteers, for your gifts of love, money and time. Our dream is coming true, but we couldn’t have done it alone.
Though this is a significant win, our work is far from complete, and I hope you’ll stay on the journey with us. To learn how you can continue supporting Taylor’s Tale, send us a note.