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DBA program
- Development stage
- Preclinical
- Lead developer
- Fulcrum Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
The DBA program is a therapeutic development initiative focused on Diamond-Blackfan Anemia (DBA), a rare genetic bone marrow failure syndrome. The program is a collaboration between Fulcrum Therapeutics and CAMP4 Therapeutics, leveraging CAMP4's proprietary GeneCircuit platform to identify regulatory RNAs (regRNAs) that control the expression of ribosomal protein genes. DBA is primarily caused by haploinsufficiency of genes encoding ribosomal proteins, most commonly RPS19, RPL5, and RPL11, which leads to impaired ribosome biogenesis and erythroid progenitor apoptosis. The program aims to discover and develop small molecule therapies that can upregulate the expression of the remaining functional allele of the affected ribosomal protein gene, thereby restoring normal red blood cell production (erythropoiesis).
- Other names
- Diamond-Blackfan Anemia program
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