Seeking in utero Treatments for Rare Diseases

The Impulse by Chiesi, the Open Innovation team within the global research-oriented biopharmaceutical group, is launching a call for innovative in utero therapeutic solutions for the treatment of rare diseases. Their focus is on approaches that enable early intervention to prevent or significantly reduce disease burden before birth.

Approaches of Interest:

• Indications of highest interest include inborn errors of metabolism; dermatologic, haematologic, and immunologic conditions; endocrine-metabolic diseases; and adjacent areas such as kidney disease 
• Ultra‑rare diseases (≤1 in 50,000) will be considered 
• Treatments capable of crossing the placenta are also of interest 
• Any therapeutic modalities that lead to rare disease treatment in utero would be accepted if they meet the criteria below: 
  • Demonstrate or plausibly support biological efficacy 
  • Be suitable for prenatal administration 
  • Present a clear translational pathway, including delivery, safety, and durability considerations 

Out of Scope:

• Opportunities related to oncology and infectious disease 

Developmental Stages of Interest:

• Opportunities from basic research phase to registration are within scope 
• Opportunities should provide, at a minimum, in vitro data, a detailed outline of the proposed next research steps, and the funding required to achieve the upcoming research milestones 

Submission Information and Opportunity for Collaboration

Submission of one-page, 200–300-word briefs is encouraged, along with any optional supplementary information e.g. relevant publications. Applicants are strongly encouraged to include a slide-deck (or similar presentation) outlining their solution. The Impulse by Chiesi welcomes submissions from all applicants, including early-stage researchers, using this submission form to describe their research and proposed next steps. In submitting to this campaign, you confirm that your submission contains only non-confidential information. 

The Impulse by Chiesi is open to a range of collaboration models, including support for small proof-of-concept or pilot studies to validate solutions that align with its needs. Details regarding intellectual property and licensing will be discussed on a case‑by‑case basis. By submitting to this campaign, you confirm that your submission contains only non-confidential information.