Company intelligence / Profile preview
Transposon Therapeutics, Inc.
- Ownership
- Private
- Development stage
- Phase 2
Overview
TPN-101 received FDA Fast Track designation for PSP in May 2024. Phase 2a study in ALS/FTD patients with C9ORF72 mutation showed TPN-101 slowed vital capacity decline in ALS patients, with biomarker responses suggesting therapeutic benefit on neuroinflammation and neurodegeneration. All doses were well-tolerated, and clinical benefit in ALS emerged with prolonged treatment. A Phase 3 clinical trial is in planning.[5][10]
- Therapeutic areas
- Neurology
- Modalities
- Small molecule drugs
- Industry
- Biotech
Drug pipeline
3 assets2 more assets in the full pipeline.
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