Drug intelligence / Profile preview

DSL101

Development stage
Phase 1
Lead developer
DSciLab
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

DSL101 is an mRNA-based therapeutic encapsulated in lipid nanoparticles (LNPs) designed for the treatment of Wilson's disease. The therapy consists of mRNA encoding the functional copper-transporting P-type ATPase (ATP7B) protein. Wilson's disease is a rare genetic disorder characterized by mutations in the *ATP7B* gene, which lead to impaired copper excretion from the liver and subsequent toxic accumulation in various tissues, particularly the liver and brain. By delivering the functional mRNA directly to hepatocytes via LNPs, DSL101 aims to restore the liver's ability to transport and excrete copper, thereby mitigating the systemic toxicity associated with the disease. Developed by DSciLab Co., Ltd., DSL101 is currently undergoing early-phase clinical evaluation to assess its safety, tolerability, and preliminary efficacy as a potential gene therapy-like intervention that bypasses the need for permanent genomic modification.

Other names
ATP7B mRNA/LNPDSL-101DSL101DSL 101
02

Targets

ATP7B (Copper ion transporter ATPase 7B)

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