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Iris sbRNA
- Development stage
- Preclinical
- Lead developer
- Iris Medicine
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Intravenous, Intraperitoneal, Intramuscular, Subcutaneous, Intradermal, Oral, Topical, Rectal, Vaginal, Intranasal, Inhalation, Ophthalmic, Transdermal, Buccal, Sublingual, Intravitreal
01
Overview
Iris sbRNA is a proprietary platform of small binding RNA (sbRNA™) therapeutics developed by Iris Medicine. These molecules utilize RNA interference (RNAi) to selectively target and silence transcripts containing expanded nucleotide repeats, which are the genetic drivers of several neurodegenerative and neuromuscular diseases. The platform encompasses multiple programs, including those for Huntington's Disease (targeting CAG repeats in the HTT gene), C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) (targeting GGGGCC repeats), and Myotonic Dystrophy Type 1 (targeting CTG repeats in the DMPK gene). Additionally, the technology is being applied to various Spinocerebellar Ataxias (SCA1, 2, 3, 6, 7, 12, 17), Dentatorubral-Pallidoluysian Atrophy (DRPLA), and Fuchs Endothelial Corneal Dystrophy (FECD). By specifically binding to the expanded repeat regions, Iris sbRNA aims to reduce the levels of toxic mutant RNA and proteins while preserving the function of normal alleles.
- Brand names
- Iris sbRNA
- Other names
- small binding RNAsbRNA
02
Targets
Chromosome 9 open reading frame 72 hexanucleotide repeat expansion RNAMutant huntingtin messenger RNA with expanded CAG repeatsExpanded CAG/CTG repeat-containing RNA
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