This therapeutics platform develops nucleic acid medicines that precisely target and silence mutated genes. By selectively suppressing disease-causing genes without affecting normal mRNA, the company aims to provide transformative treatments for patients.
Funding
Funding not disclosed
Founders
Product
Problem
Traditional gene silencing methods in nucleic acid therapeutics can inadvertently suppress normal mRNA alongside mutated mRNA, leading to potential off-target effects and adverse side effects for patients. This lack of specificity limits the therapeutic window and poses challenges in treating cancers and genetic diseases where preserving normal gene function is crucial.
Solution
ANRis is developing siRNA therapeutics using its proprietary SNPD-siRNA (Single Nucleotide Polymorphism-Distinguishable siRNA) technology, which enables precise targeting and silencing of mRNA containing single nucleotide mutations while sparing normal mRNA. This innovative approach allows for selective suppression of disease-causing genes without affecting healthy gene expression, potentially reducing off-target effects and improving treatment outcomes. By focusing on driver gene mutations in cancers and genetic disorders, ANRis aims to create personalized medicines that offer a less burdensome treatment option for patients. The SNPD-siRNA technology leverages the RNA interference (RNAi) pathway, a Nobel Prize-winning mechanism, to selectively degrade target mRNA.
Target Audience
The primary target audience includes patients with cancers and genetic diseases characterized by driver gene mutations, as well as pharmaceutical companies seeking more precise and effective nucleic acid therapeutics.
Features
- SNPD-siRNA design for allele-specific silencing of mutated mRNA
- Selective targeting of single nucleotide polymorphisms in mRNA sequences
- Reduced off-target effects compared to traditional siRNA approaches
- Application to driver gene mutations in cancer and genetic diseases
- siRNA therapeutics delivered via injection with potential for infrequent dosing (e.g., once every few months)
- Validated RNA interference (RNAi) mechanism for mRNA degradation
- Demonstrated in vitro and in vivo efficacy in preclinical models, including pancreatic cancer cell lines and xenograft models