Aerska develops next-generation RNA medicines specifically engineered for systemic administration and targeted delivery across the blood-brain barrier. The company leverages precision medicine approaches to silence disease-causing genes within the central nervous system. This focus aims to transform the treatment landscape for debilitating brain diseases by offering potent and durable therapeutic options.
Funding
$21M 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
Current therapies for central nervous system (CNS) disorders are limited by the blood‑brain barrier and an inability to modulate disease‑causing genes directly within neurons. This results in modest efficacy and a lack of disease‑modifying options for conditions such as Alzheimer’s, Parkinson’s, and ALS. Consequently, patients experience progressive cognitive and functional decline despite existing treatments.
Solution
Aerska builds next‑generation RNA interference (RNAi) medicines that silence pathogenic genes at the intracellular level. The platform couples chemically optimized siRNA constructs with proprietary brain‑shuttle vectors that enable systemic intravenous delivery across the blood‑brain barrier. By integrating biomarker‑driven patient stratification, the approach targets disease mechanisms early and supports precision dosing. The pipeline leverages established RNAi safety data and scalable GMP manufacturing to accelerate IND filing and clinical development. A cloud‑based analytics suite aggregates preclinical and clinical biomarker data, providing real‑time insight into target engagement and therapeutic response. This end‑to‑end solution aims to transform CNS drug development from discovery through to market.
Target Audience
Primary customers are pharmaceutical and biotech companies developing therapies for neurodegenerative and other CNS disorders, as well as academic and contract research organizations seeking a validated RNAi delivery system for brain targets.
Features
- Chemically stabilized siRNA molecules with enhanced potency and durability for CNS targets.
- Proprietary brain‑shuttle platform (receptor‑mediated transcytosis) that transports RNAi payloads across the BBB after systemic IV infusion.
- Integrated biomarker panel and companion diagnostics for patient selection and longitudinal monitoring.
- Scalable GMP‑compatible manufacturing process for oligonucleotide production and nanoparticle formulation.
- Data pipeline that links preclinical target validation, pharmacokinetic modeling, and clinical outcome metrics in a secure cloud environment.
- Regulatory strategy built on precedent from FDA‑approved RNAi therapeutics, facilitating accelerated pathways for CNS indications.
- Modular platform architecture allowing rapid adaptation to new gene targets and disease indications.