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The first gene editor designed entirely by AI and validated in human cells. OpenCRISPR-1 offers superior

Dual AAV systems split large transgenes across two vectors to bypass the 4.7 kb cargo limit. Early clinical

Development costs, clinical trial advantages, pricing realities, and what makes rare disease gene therapy

AAV vectors can package approximately 4.7 kb of genetic cargo. SpCas9 and its regulatory elements require

Three design principles separate successful AAV programs from those that fail: receptor-aware capsid