DEVELOPING TREATMENTS FOR RARE GENETIC DISEASES

About Modis Therapeutics

Focused on disease-modifying therapies

Modis Therapeutics is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with high unmet medical need. We were formed in 2016 through a collaboration with academic experts in mitochondrial biology. Our lead program (MT1621) is in clinical development for thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disease.

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About TK2 Deficiency

A progressive, devastating mitochondrial disease

Thymidine kinase 2 deficiency is a genetic disorder that results in mitochondrial dysfunction, leading to inadequate energy production in cells. TK2d causes progressive and severe muscle weakness, respiratory insufficiency, loss of motor function, and is often fatal. There are currently no approved therapies for this disease.

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MT1621 for TK2d

Deoxynucleoside combination therapy to restore mitochondrial function

Our science is based on discoveries by our academic collaborators that deoxynucleoside combination therapy can restore cellular function in a subset of mitochondrial diseases – mitochondrial DNA (mtDNA) depletion disorders. Data from initial clinical studies suggest that this novel approach may reverse or slow disease progression in TK2 deficient patients.

Preclinical proof-of-concept established

Initial clinical data suggest meaningful treatment benefit

Accelerated development pathway

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