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Last week, Taysha Gene Therapies announced that the company had entered into license and inventory purchase agreements with Abeona Therapeutics for ABO-202, an adeno-associated virus (AAV) gene therapy for CLN1 disease (disease of Batten).

In the long run, this news is a huge leap forward for kids like my sister, Taylor, who lost her life to CLN1 disease in 2018. For me, it’s also hugely personal, even though he came far too late to save Taylor. That’s because Taylor’s Tale launched ABO-202 more than seven years ago, when we led a coalition of local charities to fund a new research project led by Steven Gray, PhD, then at the University of North Carolina at Chapel Hill.

At the time, Taylor’s Tale had already invested hundreds of thousands of dollars in enzyme replacement therapy and, to a lesser extent, small molecule drug therapy, since our founding in 2007. But while these approaches looked promising, the Gene therapy was special because of its potential to fix Batten disease at its genetic root, eliminating the need for more surgeries or lifelong medications.

In my book, “Run to the Light,” I described the emotions that ran through my body the morning we announced our support for Dr. Gray:

“The pressure was high. I felt we were on the verge of something big, and I wanted my speech to capture that. Even as Taylor continued to slide, I had a strong feeling that 2013 would be the year we would sprint. This was where two roads diverged. We were racing in front and Batten’s disease was holding my sister back.

Today I remember that special day and the years that followed, and I think of how, even when my sister was still alive but so clearly spent on saving, I managed to dig deeply. Even as Taylor’s condition continued to deteriorate, I found a way to keep crawling forward.

And after running the Charlotte’s Thunder Road Half Marathon blindfolded in the fall of 2013, my energy for battling Batten’s disease skyrocketed. In the years that followed, I wrote a book, gave a TED Talk, and ran a race in nearly half of the 50 US states. During this time, I encouraged our friends Steve Gray and Ale Rozenberg, the postdoc working with Steve on gene therapy for CLN1 disease. When our CLN1 mice celebrated their first birthday (most die at eight months old), mom and I went to Chapel Hill and walked into the lab with pizza, beer, cake and stinky cheese, and we we threw a birthday party for these mice.

Our hearts soared even higher when in 2016 Abeona signed on to take the work to clinical stage. For several more years, the future looked bright. And when the company received clearance from the US Food and Drug Administration for its Investigational New Drug (IND) application for the CLN1 disease project, we could almost see the finish line. An IND, after all, gives trial sponsors the green light to begin treating patients.

But then, for more than a year, nothing happened. Rest assured, Taylor’s Tale hasn’t rested behind the scenes. Mom and I, in particular, did everything we could as founders of a small public charity with small pockets and big hearts to make sure Taylor’s legacy didn’t die on the shelf. Sometimes I thought about something Steve told me in 2013 when we announced the project at UNC. “We’ll have to be relentless, you know. Nothing worth having ever comes easy.

He was right, of course. But in the first eight months of 2020, I hit my lowest point in a long, long time. I remember now that even when Taylor died, so many things were going well. The promise of a cure (a cure she inspired). My mother’s ability to move mountains as a rare disease advocate. My beginnings as an author. The birth of my son.

But this year, I often struggled to find the will to keep running. In my book I recounted that some nights I wondered why I was up late at night battling computer issues with the Taylor’s Tale website when I should have been spending time with my sister then that she was still alive. Now I wondered why I was battling a rare disease and a pandemic and a stroke of bad luck when I should have been spending time with my son.

That’s why I cried tears of joy when I learned that Taysha’s gene therapies would drive Steve’s CLN1 disease work to the final stretch.

Like running, battling an ultra-rare disease is mostly a mental game. This is why miles 10 and 11 are often more difficult than the last mile of a 13.1 mile race. It’s easier to dig deep when you can see the finish line. It’s easier to believe. And even though I almost retired from racing this year, I never stopped believing in it.

To sprint forward.

To reach the finish lines.

To Taylor.

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To note: Batten Disease News is strictly a disease news and information site. It does not provide medical advice, diagnosis or treatment. This content is not intended to replace professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay seeking it because of anything you read on this website. The opinions expressed in this column are not those of Batten Disease News or its parent company, BioNews, and are intended to spark discussion about Batten disease issues.