Iris Medicine is developing small binding RNAs (sbRNAs) that selectively target expanded repeat sequences in mRNA to inhibit the expression of mutant genes associated with repeat expansion disorders. This technology aims to provide effective treatments for conditions like Huntington’s Disease and C9orf72 ALS, where current therapeutic options are limited.
Funding
$400K raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Repeat expansion disorders, such as Huntington’s Disease and C9orf72 ALS, are characterized by expanded repeat sequences in mRNA, leading to the expression of mutant genes. Current therapeutic options for these conditions are limited, creating a need for more effective and targeted treatments.
Solution
Iris Medicine is developing small binding RNAs (sbRNAs) that selectively target expanded repeat sequences in mRNA to inhibit the expression of mutant genes associated with repeat expansion disorders. The sbRNA platform mimics natural RNAs and modulates gene expression through tightly regulated, highly selective binding events. This approach enables allele selectivity, allowing for the targeting of mutant transcripts while preserving the expression of healthy genes. The non-viral delivery modality allows for flexible dosing, which can be increased, decreased, or stopped as needed.
Target Audience
The primary target audience includes patients with repeat expansion disorders such as Huntington’s Disease, C9orf72 ALS, and Spinocerebellar Ataxias, as well as the healthcare providers who treat them.
Features
- Utilizes small binding RNA™ (sbRNA™) constructs, short (~20nt) RNA sequences that mimic natural RNAs.
- Employs a non-viral delivery modality for redosable treatment.
- Achieves allele selectivity by directly targeting the expanded repeat sequence.
- Designed to have non-cooperative binding to wild-type transcripts, preserving healthy gene expression.
- Exhibits cooperative binding to expanded, mutant transcripts, potently inhibiting mutant gene expression.
- Targets the root cause of pathology in repeat expansion disorders.