Challenges in biomanufacturing individualized CRISPR therapeutics

“...collaborative, public–private approach ensures that the next generation of genomic therapies can successfully transition from bespoke treatments to accessible, scalable medicines.”

Genome editing drugs are rapidly moving into the clinic, and the prospect of individualized CRISPR drugs has demonstrated clinical promise, transitioning rapidly from design to infusion into patients. New initiatives build on the precedent set by individualized antisense oligonucleotide therapies. However, scaling these therapies from bespoke, artisanal interventions to generalizable platforms requires resolving critical bottlenecks in biomanufacturing workflows, supply chains, clinical trial structures, and strict regulatory frameworks. Transitioning to scale‑out, automated, and AI‑enabled just‑in‑time manufacturing is operationally necessary to overcome current economic barriers to patient access. This commentary discusses the path forward, emphasizing the need for scale‑out manufacturing capacity, phase‑appropriate quality specifications for novel excipients, risk‑based stability and comparability standards for multi‑variant umbrella trials, bioinformatic off‑target assessment of individualized designs, and regulatory requirements for complex clinical challenges such as long‑term follow‑up and germline risk assessment. Academic medical centers can be on the leading edge, as recently demonstrated by China’s rapid investigator‑initiated trial pathways and proactive US funding and regulatory initiatives. These efforts provide a blueprint to accelerate clinical translation of CRISPR drugs for individualized medicine and rare disease.

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