From gene target
to CRO brief.
In hours.
Gene therapy programmes spend weeks assembling design briefs before manufacturing runs begin. Mishale computes the complete pre-manufacturing design package — target rationale, guide selection, off-target safety, vector fit, dosing, regulatory pathway — from a single input.
Sample output · BCL11A programme
Gene therapy design is
the bottleneck.
Not biology.
Casgevy — the first approved CRISPR therapy — took eleven years to reach patients. The biology was understood in 2012. The bottleneck was design, safety characterisation, and regulatory preparation. That work is still done manually. We compute it.
Manual assembly
Research teams pull alignment scores from separate tools, search literature for dosing precedents, write regulatory narratives by hand. Six to twelve weeks of work. No audit trail. High error rate at the CRO handoff.
Expensive iteration
A single manufacturing run costs $3–5M. Errors in the design brief discovered at the CRO stage require full restarts. One missed high-consequence off-target site can fail Phase I and terminate a programme.
No standard output
Every team delivers a different design document. CROs, regulators, and BD partners receive incomparable packages. There is no computational basis for cross-programme comparison or portfolio decision-making.
Mishale is the computational infrastructure layer that gene therapy programmes are missing.
One input — gene target + indication. Six sections. CRO-ready in hours. Every decision traceable to a data source. Every assumption documented.
Pre-Manufacturing
Design Package.
Six structured sections. Every programme. Computed from a proprietary biological knowledge base spanning genes, variants, approved therapies, and regulatory precedents.
Target Rationale
Is this gene the right target?
Mechanism class, inheritance pattern, therapeutic modality fit, and target confidence score — assessed against our proprietary biological knowledge base and approved therapy precedents.
Three layers.
One output.
A biological knowledge graph. A computational design engine. A structured output layer. Together they reduce weeks of manual work to a single query.
The Biological Foundation
A comprehensive, continuously updated graph of human genes, variants, cell types, approved therapies, and regulatory precedents — assembled from leading biological and clinical databases.
Computational Reasoning
Queries the knowledge graph in real time, runs safety analysis across the reference genome, scores guide candidates against the target profile, and applies dosing and regulatory logic against curated precedent libraries.
The Output Layer
Six structured sections assembled into a CRO-ready PDF in under 24 hours. Every field traceable to a source. Every decision documented with a confidence score and biological rationale.
Request a Design Package.
Tell us the gene target and indication. We will run the full pipeline and deliver a CRO-ready package within 24 hours.
We work with pharma BD teams, biotech founders, and academic translational groups.