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Synthetic solutions for immuno-oncology

How Synthorx is harnessing synthetic DNA to enhance biologics

Synthorx Inc. is poised to enter the clinic this year with an IL-2 therapy that could validate its synthetic DNA platform. Marrying its click chemistry and unnatural amino acid technology with pegylation, the company is building a pipeline of cytokines with tailored pharmacology, and plans to expand to other modifications and other modalities.

Fresh off a December IPO that raised $150.7 million on NASDAQ, Synthorx is lining up a suite of preclinical candidates, starting with its IL-2 immuno-oncology program THOR-707. The compound is slated for IND submission next quarter, and the company expects Phase I/II data within 12 months.

It also will select IND candidates by year-end for IL-10 and IL-15 programs in immuno-oncology, and a different IL-2 candidate for autoimmune disease.

Synthorx’s platform features synthetic DNA base pairs that encode anchor points on proteins where additional structures, such as polyethylene glycols (PEGs), can be attached to enhance their function (see “Base Instincts”).

Using synthetic DNA the company encodes unnatural amino acids; using click chemistry it can then add a PEG or other groups at any point it wants in a protein, meaning it can tailor biologics to

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