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Triplet Therapeutics is a biotechnology company developing transformational treatments for patients with repeat expansion disorders (REDs) -- a group of more than 50 known genetic diseases including Huntington`s disease, myotonic dystrophy, spinocerebellar ataxias, fragile X syndrome, and familial amyotrophic lateral sclerosis (ALS) -- leveraging insights from patient genetics. Triplet designs and develops potential therapeutics for REDs using its proprietary thRED Engine, which enables the Company to develop a single oligonucleotide targeting the DNA Damage Response (DDR) pathway to potentially treat multiple REDs. Triplet is backed by investments from Atlas Venture, MPM Capital and Pfizer Ventures, along with Invus, Partners Innovation Fund and Alexandria Venture Investments.
Exicure is developing a new class of immunomodulatory and gene silencing drugs against validated targets. Our 3-dimensional, spherical nucleic acid (SNA™) architecture unlocks the potential of nucleic acid therapeutics in multiple organs. Our lead programs address diseases from inflammatory disorders to oncology. SNA constructs overcome one of the most difficult obstacles to nucleic acid therapeutics: safe and effective delivery into cells and tissues. SNA constructs exhibit unparalleled transfection efficiency into numerous cell and tissue types including the skin without carriers or transfection agents. Moreover, SNAs can be used as potent immunotherapeutic agents for the treatment of cancer or infectious disease.
Engineering next‑generation, brain‑penetrant cancer therapies.
Comprehensively mapping the immune system with single-cell biology and AI to power new therapeutic discoveries, accelerate drug development and improve patient outcomes