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Therapeutic Pipeline


In vitro & In vivo POC

Patent Protection

Addressable Market

First in Class Drug Candidate 

High Unmet Clinical Need

CRISPRi Gene Therapy for FSHD is not an approved therapeutic product. The above information is indicative only of progress of the Therapeutic Program through the drug development pathway.  

 Any suggested pre-treatment protocol, diagnostic process or mechanism of drug action is purely for example and illustration purposes only and is entirely proposed only. No suggestion is made as to the efficacy or safety of any gene therapy, and any final approved therapeutic product and/or treatment regimen for CRISPRi Gene Therapy may be materially different in scope, nature and practice than any suggested here. No reliance should be made upon the following as being fit for any purpose. Please see details of forward-looking statements and risk which can be found in the Terms of Use for this website and apply to any drug development program described, suggested or referenced, in whole or part on this website. 

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