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

‹ Pipeline › TPN-101

AGS

Aicardi Goutières Syndrome


About AGS

Why TPN-101?
✕

Aicardi Goutières Syndrome (AGS)


About AGS
AGS is an ultrarare severe pediatric disease generally presenting in infancy; many patients do not survive childhood. Neuronal degeneration causes massive brain damage and loss of developmental milestones beginning shortly after birth and can result in heart and muscle damage.

✕

Aicardi Goutières Syndrome (AGS)


Why TPN-101?
Genetic mutations of LINE-1 repressing enzymes (e.g., TREX-1, RNASEH2) cause AGS and result in uncontrolled LINE-1 activation with severe interferon-1 autoimmune reaction. Clinical validation of LINE-1 reverse transcriptase as a therapeutic target was demonstrated in a study of AGS patients receiving reverse transcriptase inhibitors (abacavir, lamivudine and zidovudine), which lowered interferon scores and increased blood flow to the brain

Clinical Trial Information

An open-label, Phase 2a study of TPN-101 in AGS patients is open for enrollment. Cohort 1, comprised of adult patients, has been completed. The study is recruiting patients aged 1 to 17 years to fill the remaining cohorts. The study is being conducted in France and Italy.

For More Information

ClinicalTrials.gov