Aperture Therapeutics develops oligonucleotide therapeutics by identifying naturally occurring gene variants that confer protection against neurodegenerative diseases. The platform uses machine learning to analyze genomic and clinical datasets to prioritize protective gene variants for therapeutic mimicry. This approach targets genetically validated pathways to address shared drivers in ALS and FTD, expanding patient applicability beyond rare mutations.
Funding
Funding not disclosed
Founders
Product
Problem
Neurodegenerative diseases lack effective treatments, and current drug discovery methods often fail to translate to clinical success. Identifying protective genetic factors and translating them into therapies is challenging due to the complexity of the human genome and the difficulty of modeling brain aging.
Solution
Aperture Therapeutics is developing a drug discovery engine that leverages extensive, genetically diverse cell libraries to identify gene variants that confer protection against neurodegenerative diseases. The company focuses on understanding the protective mechanisms encoded by these variants and translating them into targeted RNA therapeutics. By targeting specific RNA transcripts, Aperture aims to create therapies that can delay brain aging and improve outcomes for individuals at risk of or affected by neurodegenerative conditions. Their approach seeks to transform the genetic strengths observed in a small subset of the population into broad-spectrum protection for many.
Target Audience
The primary target audience includes individuals at risk of or affected by neurodegenerative diseases, as well as pharmaceutical companies seeking novel therapeutic targets and drug candidates in the neurodegeneration space.
Features
- Massive cell libraries representing the genetically diverse human population
- Focus on identifying gene variants that provide protection from neurodegenerative diseases
- Discovery of therapeutics that target specific RNA transcripts
- High-throughput screening to accelerate the identification of promising drug candidates