Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Research programme: degenerative disorders therapeutics - Design Therapeutics

Next Previous
Drug Profile

Research programme: degenerative disorders therapeutics - Design Therapeutics

Alternative Names: FECD GeneTAC™ molecules - Design Therapeutics; FECD GeneTAC™ small molecules - Design Therapeutics; Syn-TEF1; Syn-TEF2; SynTEFs; Synthetic transcription elongation factors - Design Therapeutics

Latest Information Update: 15 May 2025

Price :
$50 *
Note:
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

  • Originator Design Therapeutics
  • Class Small molecules
  • Mechanism of Action Gene silencing; Genetic transcription modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Friedreich's ataxia; Fuchs' endothelial dystrophy; Huntington's disease; Myotonic dystrophy
  • Research Amyotrophic lateral sclerosis; Fragile X syndrome; Frontotemporal dementia; Spinocerebellar ataxias; X-linked bulbo-spinal atrophy

Most Recent Events

  • 07 May 2025 Design Therapeutics plan the selection of candidate in Myotonic dystrophy type-1 (DM1) in 2025
  • 05 Aug 2024 Design Therapeutics plans to file an IND application for Myotonic dystrophy and Huntington’s disease
  • 19 Mar 2024 Design Therapeutics has patent pending application for "compounds and methods for modulating the expression of the dystrophia myotonica protein kinase (DMPK) gene and methods for the treatment of DM1" in the US

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top