Preliminary findings from the CRD-TMH-001 clinical trial

To our community,

I want to thank you for all the heartfelt messages of support since my brother, Terry Horgan, passed away in October 2022, and for respecting our privacy as we continue to grieve the loss of a brother, son, friend, and inspiration to the rare disease community.

I also want to thank you for your patience as a team of researchers and clinicians led by Yale University and UMass Chan Medical School have been studying the outcome of the CRD-TMH-001 clinical trial. Today, we have shared some preliminary findings, reported in a STAT News article, and the research team will release a full report on Terry’s case that further explains those findings within the next few weeks on

The evidence from the forthcoming report suggests that Terry had an adverse reaction to the viral vector used to deliver the potential CRISPR therapeutic, worsened by his age and the advanced stage of his condition. Unfortunately, we didn’t have an opportunity to learn whether the potential CRISPR therapeutic that our team has spent many years developing for Terry was efficacious, but we learned much that we can apply to our ongoing programs.

We have felt a great responsibility in studying what happened to Terry, as the findings are not just important to Cure Rare Disease, but also to the broader neuromuscular and gene therapy fields. Cure Rare Disease is advancing next-generation delivery methods for all potential therapeutics currently in preclinical development, and we will do our part to tackle the challenges of gene therapy delivery, alongside the many other challenges, including barriers to access, that patients and families with rare and ultra-rare neuromuscular diseases face every day. Our commitment to rewriting the drug development playbook for rare and ultra-rare diseases, evident in our pipeline of 20 potential therapies, continues in Terry’s honor.

While this outcome for Terry is not what any of us wanted, he is a medical hero who has advanced the rare disease field. For Terry, the trial was about more than just him. He was at the end stage of his disease, he was running out of time, and he understood that death was a very real possibility. The trial was his sole opportunity to stop a disease he’d been fighting his whole life, and while his outcome is devastating for all of us, his motivation to move science forward for other rare and ultra-rare disease patients continues in our work today. Our hope is that these learnings will help other families receive safe and efficacious treatment – and that they will no longer be left behind. 

That is our steadfast commitment, in Terry’s honor.

With gratitude,

Rich Horgan

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