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TD101

Development stage: Phase 1
Lead developer: TransDerm
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intralesional, Topical

Overview

TD101 is a small interfering RNA (siRNA) designed specifically for the treatment of pachyonychia congenita, a rare genetic skin disorder. It works through RNA interference by targeting the keratin 6a (K6a) N171K mutant mRNA without affecting wild-type K6a mRNA. This highly specific approach allows it to inhibit the expression of the mutant keratin that causes the disease.

Brand names: Reveker
Other names: Pachyonychia congenita siRNA

Targets

KRT6A (Keratin 6A)

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