EditForce strives to discover and develop RNA-targeted drugs based on our proprietary platform technology - PPR (PentatricoPeptide Repeat) protein based RNA modulation - in the areas of CNS, neuromuscular diseases and rare disease area. PPR is small enough to be delivered via AAV by a single module whereas CRISPR-Cas systems require two modules.
Our lead program EF-210 has cleared the critical milestone of in vivo POC (efficacy, immunogenicity, toxicity, etc. ) on Myotonic dystrophy type 1, and thus we are looking for a partner to bring this asset to clinical/commercial stage.
EF-210 is a recombinant AAV integrated with a PPR gene. It is specifically binds to the pathogenic RNA and release splicing factors but does not degrade the pathogenic RNA unlike ASOs. It has long term effect by sustained in vivo expression when delivered via AAV.
We are also open to research collaboration with you through our platform technology.
Our lead program EF-210 has cleared the critical milestone of in vivo POC (efficacy, immunogenicity, toxicity, etc. ) on Myotonic dystrophy type 1, and thus we are looking for a partner to bring this asset to clinical/commercial stage.
EF-210 is a recombinant AAV integrated with a PPR gene. It is specifically binds to the pathogenic RNA and release splicing factors but does not degrade the pathogenic RNA unlike ASOs. It has long term effect by sustained in vivo expression when delivered via AAV.
We are also open to research collaboration with you through our platform technology.
Company Type:
Privately Funded Company
Company Website:
Company HQ State:
Not Provided
Company HQ Country:
Japan
Year Founded:
2015
Main Therapeutic Focus:
CNS/Neurological
Lead Product in Development:
EF-210
Development Phase of Primary Product:
Pre-Clinical
Number Of Unlicensed Products (For Which You Are Seeking Partners):
Not Provided
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