C9orf72 ALS/FTD Prevention Trial Design
exploratoryresearch program · medium · Wed Jan 01 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Design disease prevention trials for clinically unaffected C9orf72 repeat expansion carriers at elevated risk for ALS and FTD.
Multi-stakeholder workshop recommendations covering outcome measures, eligibility criteria, biomarkers, digital health technologies, analytic frameworks, and regulatory considerations.
2025 publication describing recommendations from a June 2024 workshop on C9orf72 disease prevention trial design.
The publication recommends unified ALS/FTD prevention trial design considerations for C9orf72 repeat expansion carriers, including shared biomarkers and clinical progression measures.
TPN-101 for Aicardi-Goutieres Syndrome
phase 2drug program · high · Wed Mar 15 2023 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate TPN-101 in patients with Aicardi-Goutieres Syndrome.
Phase 2a multi-center, open-label, single dose level study of TPN-101 in AGS patients.
Phase 2a AGS clinical trial listed in public records.
C9orf72 Transposable Element Radiogenomics Biomarker
exploratorybiomarker · medium · Sun Jan 01 2023 00:00:00 GMT+0000 (Coordinated Universal Time)
Identify peripheral transcriptomic and neuroimaging biomarkers associated with C9orf72-mediated FTD/ALS severity and thalamic atrophy.
Whole-blood RNA sequencing integrated with FreeSurfer-based thalamic neuroimaging analysis to evaluate C9orf72 expression and transposable element activation.
2023 publication reporting peripheral transposable element derepression and associations with thalamic atrophy and clinical impairment in C9orf72 expansion carriers.
The study found reduced peripheral C9orf72 expression, global peripheral transposable element activation including L1HS, and associations between L1HS levels and atrophy of multiple pulvinar nuclei.
TPN-101 for Progressive Supranuclear Palsy
phase 2drug program · high · Sun Dec 12 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Assess TPN-101 as a therapy for progressive supranuclear palsy, a neurodegenerative disease listed in Transposon Therapeutics' pipeline.
Phase 2a clinical study assessing safety and tolerability of TPN-101 in patients with PSP.
Phase 2a PSP clinical trial listed in public records.
Novel NRTIs Platform
exploratoryplatform · medium
Develop novel nucleoside reverse transcriptase inhibitor approaches for neurodegenerative and aging-related diseases.
Novel NRTI therapeutic platform or approach referenced by the company site.
Novel NRTIs listed as a company science or pipeline focus area.
PKR Program
exploratoryresearch program · low
Investigate or target PKR biology as part of Transposon Therapeutics' therapeutic approach for neurodegenerative and aging-related diseases.
PKR-focused research or target program referenced by the company site; supplied material does not specify intervention details.
PKR listed as a company science or pipeline focus area.
TPN-101 for ALS/FTD
undiscloseddrug program · medium
Develop TPN-101 for ALS/FTD, a neurodegenerative disease area referenced in Transposon Therapeutics' pipeline navigation.
Drug program; supplied material does not provide trial design or results for ALS/FTD.
ALS/FTD listed as a pipeline focus area on the company site.
TPN-101 for Alzheimer's Disease
undiscloseddrug program · medium
Develop TPN-101 for Alzheimer's disease, an aging-related neurodegenerative disease area referenced in Transposon Therapeutics' pipeline navigation.
Drug program; supplied material does not provide trial design or results for AD.
AD listed as a pipeline focus area on the company site.