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DNL952
- Development stage
- Phase 1
- Lead developer
- Denali Therapeutics
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
DNL952 is a brain-penetrant enzyme replacement therapy (ERT) developed by Denali Therapeutics for the treatment of Pompe disease. It consists of recombinant human acid alpha-glucosidase (GAA) fused to Denali's proprietary Transport Vehicle (TV) technology. The TV component is an Fc region engineered to bind the transferrin receptor (TfR), facilitating receptor-mediated transcytosis across the blood-brain barrier and enhancing uptake into muscle and central nervous system (CNS) tissues. By addressing both the systemic muscle weakness and the neurological deficits associated with glycogen accumulation in Pompe disease, DNL952 aims to overcome the limitations of conventional ERTs that have poor CNS penetration. It is currently being evaluated in Phase 1 clinical trials for late-onset Pompe disease (LOPD).
- Other names
- GAA-TVGAA-Transport Vehicle
02
Targets
GAA (Lysosomal acid alpha-glucosidase)TFRC (Transferrin Receptor)
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