First Rare Diseases in the program

To initiate the program, CCP has established research and development projects on the following rare diseases:

• Netherton syndrome
• Angelman syndrome
• Prader-Willi syndrome
• SPATA5 syndrome
• Diamond-Blackfan anemia

Preclinical Therapeutics Development in CCP – Objectives:

• GLP studies.
• To provide fully supported access to preclinical development in accordance with the requirements of regulatory authorities.

CCP obtained the good laboratory practice (GLP) certificate that improves our possibilities of preclinical testing of therapies:

Since 2025, the RD-Factory Program has been part of the CCP Annual Conference. As part of the conference, one afternoon is dedicated to a workshop on selected rare diseases, where patient representatives meet with the scientific community to discuss recent developments in RD- research.

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