Advancing Craniosynostosis Treatment (ACT) Rare Diseases Consortium Pilot Study Cycle 2 Letter of Intent

The Advancing Craniosynostosis Treatment (ACT) Rare Diseases Consortium (https://act.rarediseasesnetwork.org/) is pleased to announce a Call for Letters of Intent/Inquiry (LOIs) for the second cycle of its Pilot Study Program.

The LOI process is intended to help estimate the number of reviewers needed and to determine whether proposed projects are responsive to the goals and priorities of the ACT Rare Diseases Consortium before preparation of a full application. Investigators are strongly encouraged to submit an LOI. Feedback may be provided on the proposed project's responsiveness to the forthcoming Request for Applications; applicants who choose not to submit an LOI do so without the benefit of this feedback.

The ACT Rare Diseases Consortium Pilot Study Program is designed to support early-stage, high-impact pilot projects that generate preliminary data, validate novel concepts, or de-risk innovative approaches with strong potential for future translational, clinical, or commercialization impact. Proposed projects should align with the mission of the ACT Rare Diseases Consortium and complement or expand the Consortium's ongoing Clinical Projects. Clinical Project 1 aims to study the longitudinal natural history of syndromic CS. Clinical Project 2 aims to study surgical treatment outcomes of syndromic CS after cranial vault remodeling. Clinical Project 3 aims to study surgical treatment outcomes of syndromic CS after midface advancement.

Responsive topics include, but are not limited to:

  • Disease mechanisms, pathophysiology, or target identification
  • Biomarker discovery or validation
  • Therapeutic development (e.g., small molecules, biologics, cell or gene therapies, and medical devices)
  • Diagnostic or prognostic tools
  • Data science, artificial intelligence/machine learning, and digital health approaches
Projects should be feasible within a one-year project period (June 1, 2027–May 31, 2028) and an estimated budget of $40,000 in total costs, and should clearly reflect the exploratory, pilot nature of the proposed work.

Letters of Intent/Inquiry are due September 15, 2026. LOIs should be limited to one page. Additional information regarding eligibility, application requirements, review criteria, and funding details will be provided in the full Request for Applications.