A new strategy
An Overview of Enzerna’s Technology for RNA Interference
Our therapeutic platform, called Artificial Site Specific RNA Endonucleases (ASREs) is modular system consisting of an RNA binding module (PUF) that can be engineered to bind any RNA sequence of choice and an RNA degrading enzyme (PIN) that will destroy the RNA. ASREs can be used to specifically cleave (and thus inactivate) any disease-causing RNA. Combined with gene delivery vectors, ASREs provide a new strategy for selective degradation of pathogenic transcripts associated with nucleotide expansion disorders.
ASRE Technology: Highly Specific and Highly Differentiated
Targeting the pathogenic RNA or protein with ASRE-Technology offers an avenue for curative therapies. Antisense oligonucleotide (ASO) and RNA interference (RNAi)- based therapies have been shown to be effective in isolated cells. However, these therapies are limited by the need for lifelong administration, poor delivery across the blood brain barrier, and passive delivery to target cells in vivo. While antisense RNAs could be delivered via gene therapy, to date, targeting efficiency remains unacceptably low.
For many diseases, gene editing, most notable using CRISPR/Cas DNA editing technology, offers an opportunity to correct mutant alleles. Unfortunately, given the mechanism of the gene editing process, CRISPR/Cas currently does not offer a viable therapeutic approach for nucleotide expansion disorders.
ASRE's Have the Following Competitive Advantages:
- Non CRISPR-based
- No competing Intellectual Property
- ASREs preferentially bind and destroy RNAs carrying expanded repeats leaving normal RNAs intact
- One ASRE therapeutic can be used to treat multiple indications