Company intelligence / Profile preview

Transposon Therapeutics, Inc.

San Diego, California, United Statestransposonrx.com ↗
Ownership
Private
Development stage
Phase 2
01

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
02

Drug pipeline

3 assets

censavudine

2

2 more assets in the full pipeline.

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