Technologies to Address Distribution of Macromolecules in Hard-to-reach Tissues

Chiesi Global Rare Diseases is seeking research proposals for new technologies from academic medical centres, universities, and research institutions globally. Their objective is to award funds to validate early‑stage projects with clear therapeutic potential. Successful applicants will be funded through sponsored research, with up to $275K/€250K available per project for 2 years duration. 

This should enable researchers to generate relevant preclinical data or demonstrate preclinical proof of concept for technologies enabling effective biodistribution of macromolecules (for example: nucleic acids, proteins) to the following tissues of interest, characterised by challenges in delivery (any or all): 

• Bone
• Skeletal/cardiac muscle
• Central nervous system

for the treatment of rare metabolic disorders, for example inborn errors of metabolism, including lysosomal storage disorders. The technology might be brought into the Chiesi Global Rare Diseases pipeline and ultimately benefit patients.

What Chiesi Is Looking For

Chiesi Global Rare Diseases is seeking new technologies with key attributes including some of the following:

1. Potential for platform development (for example to address multiple indications)
2. Preliminary preclinical evidence to support feasibility of technology
3. Innovative models for technology validation (e.g., organoids)
4. Strong potential for clinical impact

Therapeutic Area of Interest for 2022

Delivery technologies that can be applied to the treatment of rare metabolic disorders, for example inborn errors of metabolism, including lysosomal storage disorders.

Submission Information

The first step in the process is submission of a Pre‑Proposal which should contain a brief, non‑confidential 2‑3 page overview of the target, mechanism (including evidence for link to disease), and the proposed therapeutic opportunity. To submit your proposal, please visit our website at discover.in-part.com, register, and submit your research pre‑proposal under the appropriate Discover campaign. A more detailed Full Proposal will be solicited by invitation only.