Transposon Therapeutics, Inc. Science Pipeline Team News / Literature Connect

TPN-101


TPN-101, a potent inhibitor of LINE-1 reverse transcriptase, shows promise for the treatment of neurodegenerative diseases.
Transposon is developing TPN-101 (aka Censavudine) to treat neurodegenerative diseases of high unmet medical need. TPN-101 has excellent systemic and brain bioavailability following once-daily oral dosing and is well tolerated based on exposures of over 250 subjects for approximately 48 weeks.

Promising exploratory Phase 2 results support Phase 3 evaluation of TPN-101 in PSP and ALS, as well as initiation of studies in Alzheimer’s Disease. The Phase 2a evaluation of TPN-101 in AGS continues to enroll eligible patients who are 1 to 11 years of age.

PSP ALS/FTD AGS AD

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