AT-003
From genetic discovery to broad therapeutic potential for autoimmune diseases.
A small-molecule oral mitophagy and autophagy modulator designed to treat a broad variety of autoimmune and neurodegenerative diseases based on interventions through the CLEC16A gene.
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18
CLEC16A-associated diseases
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16
of the 18 to date are autoimmune diseases, 2 are neurodegenerative (MS and PD)
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50%
increase in the total addressable market in the past decade across target indications
Treating multiple high-unmet-need diseases
AT-003 is designed to restore the biological pathways regulated by CLEC16A rather than treating a single disease in isolation. By targeting mechanisms that are genetically linked across numerous conditions, the program has the potential to extend beyond an initial indication into multiple autoimmune and neurodegenerative diseases where CLEC16A dysfunction contributes to disease risk and progression.
The origin of a therapeutic opportunity
In 2007, Arctic Therapeutics’ founder Dr. Hakon Hakonarson and his team at the Center for Applied Genomics (CAG) discovered and named the CLEC16A gene.
A genetically validated target
CLEC16A is an important regulator of immune homeostasis, autophagy, and mitochondrial quality control. Since its discovery, genetic variants in CLEC16A have been consistently associated with susceptibility across a growing number of autoimmune and neurodegenerative diseases through genome-wide association studies (GWAS).
What began with the first reported association in type 1 diabetes has since expanded to include multiple sclerosis, Crohn’s disease, rheumatoid arthritis, systemic lupus erythematosus, celiac disease, asthma, Parkinson’s disease, and many other disorders.
Although these conditions are clinically distinct, they share underlying biological pathways involving immune dysregulation, cellular stress responses, and impaired mitochondrial function, making CLEC16A a compelling therapeutic target with broad potential.