November 1, 2022
To the CRD community,
As we have shared previously with our community, Terry Horgan, brother of Cure Rare Disease founder and CEO, Rich Horgan, recently passed away while participating in the CRD-TMH-001 clinical trial of a novel CRISPR therapeutic.
We know the CRD-TMH-001 trial and the outcome have been closely followed by the rare disease community and many are eager for more details. While these details are currently being studied by multiple teams across the country, this is a complex undertaking and could take up to four months.
The comprehensive work these teams are doing is critical to gaining a clear understanding of the outcome of the CRD-TMH-001 trial and to shedding additional light on the challenges of gene therapy broadly. Meanwhile, we will continue to work with our researchers, collaborators, and partners to develop therapies for the neuromuscular diseases in our pipeline.
Cure Rare Disease was founded with a commitment to transparency and collaboration as we believe both are critical to moving science forward for rare disease patients. To that end, we intend to share the findings from the CRD-TMH-001 trial with the scientific community, not only to support the continued advancement of the additional 18 therapeutics in the CRD pipeline, but also to further the understanding of AAV-based gene therapies and applications among the larger communities for the treatment of other rare diseases.
We also want to extend a heartfelt thank you to everyone who has sent their condolences following the passing of Terry. We appreciate all the support and continued patience as we work toward completing a final report on the trial.
Our research and development is powered by the generous financial support of families, rare disease communities and organizations unwilling to accept the status quo of drug development practices. As a 501c3 non-profit biotech, 93+% of all donations go directly to the research and development of life-saving therapies.
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