Publication of CRD-TMH-001 clinical trial results

To our community,

Today, we released a case report with comprehensive findings from the CRD-TMH-001 clinical trial on the pre-print server, We have made our manuscript available via an open access server as part of our commitment to sharing information that may benefit the rare disease community, while we concurrently submit our manuscript to a peer-review journal.

As we’ve communicated, the evidence in the 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 we developed for Terry was efficacious. We did, however, learn much that we can apply to our ongoing programs and share with the broader neuromuscular and gene therapy fields.

We want to thank you for your patience as our focus has been on sharing these learnings in a thorough, responsible manner. In February, we introduced some preliminary results for conversation among the medical community, so that we could take their insight into our manuscript development. Today, we are releasing the comprehensive findings.

Cure Rare Disease remains firmly committed to the rare disease community as we move forward with other therapies, all in honor of Terry’s legacy. 

For additional perspective on these newly published findings, please read Rich Horgan’s letter to the Cure Rare Disease community on the preliminary findings and our FAQs on the report.

Previous communication on the CRD-TMH-001 trial:

Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic | August 9, 2022