Nerai builds custom CRISPR‑based genome editors using its MORPHEME platform, which combines automated high‑throughput directed evolution, large‑scale cell assays, and machine‑learning‑guided protein design to create PAM‑specific Cas9 variants with superior specificity and activity. These bespoke editors enable precise correction of mutations that standard tools cannot target, supporting one‑time curative gene‑editing therapies for rare inherited diseases, initially focused on liver and retinal indications.
Funding
Funding not disclosed
Founders
Product
Problem
Many rare inherited diseases lack effective treatments because existing genome editing tools have limited DNA targeting range and pose off‑target safety risks, making it difficult to correct disease‑causing mutations in a precise and reliable manner.
Solution
Nerai develops custom CRISPR‑based genome editors using its MORPHEME platform, which integrates automated high‑throughput directed evolution, large‑scale cell‑based functional screening, and machine learning‑guided protein design. This approach produces bespoke Cas9 variants with enhanced PAM specificity, higher on‑target activity, and reduced off‑target effects, enabling the correction of mutations that are inaccessible to standard editors. The company applies these super‑precise editors to a pipeline of rare genetic disorders, initially focusing on liver and retinal targets, with the goal of delivering one‑time curative therapies.
Target Audience
Primary customers are biopharmaceutical companies and research organizations developing gene‑editing therapeutics for rare inherited diseases, particularly those targeting liver and retinal indications.
Features
- Fully automated 100‑fold multiplexed directed evolution of CRISPR enzymes to explore diverse protein variants
- High‑throughput human cell assays that evaluate on‑target activity, precision, and efficiency across thousands of therapeutic loci
- Machine‑learning models that predict and optimize protein designs for maximal specificity and safety
- Generation of PAM‑specific Cas9 variants tailored to individual disease‑causing DNA sequences
- Scalable platform capable of producing personalized genome editors for multiple rare disease programs