We strive to discover and develop gene therapy drugs based on our PPR Platform Technology, mainly in CNS, muscular, and rare diseases.
We believe our RNA targeting approaches will achieve safer and more innovative therapies to reverse genomic DNA damage and restore normal gene function.
We are committed to leveraging our innovative PPR Platform to discover and deliver breakthrough therapies to patients suffering from diseases worldwide.
Targetable RNA-related diseases
PPR platform technology enables various RNA controls, including RNA editing, translation enhancement, splicing control, and RNA blocking, and allows targeting a wide range of RNA-related diseases.
Mechanism of action
・Diseases caused by point mutations (C>T, T>C mutations) in RNA
・Disease not amenable to gene replacement therapy with AAV due to the large gene size
・Diseases in which the splicing pattern is abnormal due to mutation
・Diseases caused by abnormal RNA that traps a specific RNA-binding protein.
・Repeat RNA disease
・Myotonic dystrophy type 1 (DM1)
PPR is Amenable to Multiple Delivery Systems
We are developing our PPR platform to be delivered by viral vectors such as an adeno-associated virus (AAV). However, other delivery approaches are in development better suited to different disease settings.
Target: Myotonic Dystrophy Type 1
DM1 is a multisystem disorder affecting skeletal muscle, eye, heart, CNS, and endocrine systems. More than 1 in 2,300 people are afflicted with DM1 worldwide. Patients with DM1 suffer from severe symptoms. Unfortunately, there are currently no FDA-approved treatment options for DM1.
DM1 is the most prevalent triplet repeat disease caused by the expansion of a CTG repeat in the noncoding region of DMPK.
EF-210 is a novel gene therapy program for the potential treatment of Myotonic Dystrophy Type 1 (DM1) and was designed using our proprietary PPR platform to target specific RNA sequences. EF-210 binds to the CUG repeat RNAs and releases splicing factors to restore splicing networks and muscular function.