Pre-clinical development of CDK8/19 Inhibitors to Restore Erythropoiesis in Diamond-Blackfan Anemia.
| Reference number | |
| Coordinator | Lunds universitet - Institutionen för laboratoriemedicin |
| Funding from Vinnova | SEK 2 999 976 |
| Project duration | January 2026 - January 2029 |
| Status | Ongoing |
| Venture | European Joint Programme on Rare Diseases |
| Call | International collaborations with preclinical therapy studies of rare diseases |
Purpose and goal
The goal is to develop a safe, oral CDK8/19 inhibitor that restores erythropoiesis in Diamond Blackfan Anemia Syndrome (DBAS). The consortium combines rational drug design, validated human and animal models, and biomarker strategy to develop a preclinical candidate with a clear regulatory pathway to clinical trials.
Expected effects and result
Optimized CDK8/19 inhibitors with improved selectivity and PK/PD, validated efficacy in patient cells and models, mechanistic insight, and response biomarkers. Long‑term: a non‑invasive, cost‑effective therapy reducing transfusion dependence and improving quality of life for DBAS patients.
Planned approach and implementation
The project is coordinated by Lund University and divided into seven work packages. WP1 secures patient engagement, communication, and ethics. WP2 delivers optimized CDK8/19 leads. WP3–WP4 validate efficacy in iPSC‑based human models and mouse models. WP5 maps mechanisms and biomarkers. WP6 validates in primary patient cells. WP7 implements FAIR data, IP management, and the regulatory route toward clinical trials.