RD-Factory Program Announces Open Call for Gene and Disease Nominations to Revolutionize Rare Disease Research

Since 2015, the Czech Centre for Phenogenomics (CCP) of the Institute of Molecular Genetics of the Czech Academy of Sciences (IMG) has been at the forefront of gene editing and the creation of mouse models to explore human diseases. With over 1,000 mouse models CCP has become a pivotal player in the discovery of gene functions and the preclinical testing of potential therapies. In parallel, the Institute of Molecular and Translational Medicine (IMTM) at Palacký University has been advancing drug and biomarker development, forwarding them to clinical trials and medical implementations.

The RD-Factory Program has been established to intensify these efforts, focusing on new models for rare genetic diseases and exploring innovative therapeutic strategies, especially in cell and gene therapies, along with novel and repurposed therapeutic compounds. By doing so, the program seeks to translate these advances into clinical settings, significantly impacting the rare disease landscape.

Open Call for Proposals: Nominate Your Gene or Disease

The RD-Factory Program invites proposals for preclinical studies aimed at rare human genetic diseases. This open call seeks contributions from the scientific community, rare disease experts, societies, and patient families to:

• develop mouse models for disease understanding and therapeutic testing,
• design and/or preclinical testing of therapies (gene therapy or other such as small molecule, biological, and cellular therapies),
• evaluate emerging drugs and therapeutic strategies,
• foster novel diagnostic approaches encompassing diagnostic, prognostic, and predictive biomarkers.

Application Process

Beginning October 1, 2024, the RD-Factory Program welcomes nominations year-round, without deadlines. Up to six proposals will be selected annually for complimentary support. Initial submissions, detailing the rare disease and relevant gene, can be forwarded via e-mail to ccp@phenogenomics.cz.

Scientific Evaluation and Selection Criteria

Each proposal will undergo rigorous evaluation by CCP and IMTM experts to assess scientific merit, clinical need, technical feasibility, and translational potential. The selection will consider factors such as technical feasibility, cost, resource capacity, and patient impact.

For more information and to submit nominations, visit www.phenogenomics.cz.

Join us in making strides against rare diseases and bring hope to affected populations worldwide.