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Gene Editing (CRISPR, Base Editing, Prime Editing)

Precision tools for modifying DNA sequences in living cells. CRISPR-Cas9 achieved the first approved therapy (Casgevy); next-generation approaches like base editing and prime editing offer even greater precision without double-strand breaks.

Key Companies

CRISPR TherapeuticsIntellia TherapeuticsVerve TherapeuticsBeam TherapeuticsPrime Medicine

Recent Signals

DEEP DIVE

CAR-T Beyond Cancer: The Autoimmune Revolution Nobody Predicted

From a single lupus patient in Erlangen to pediatric trials in Rome, allogeneic programs at Allogene, and dual-targeted CD19/CD70 designs — CAR-T cell therapy is rewriting autoimmune medicine. We map the landscape: which diseases, which constructs, and who's winning the race to scale.

Apr 16, 2026 · 14 min read

REGULATORY

ACC Issues First Scientific Statement on Gene Editing for Heart Disease

The American College of Cardiology publishes its 2026 scientific statement on gene editing therapy for cardiovascular disease, covering CRISPR-based approaches to PCSK9, TTR amyloidosis, and hypertrophic cardiomyopathy. A formal signal that cardiology's regulatory framework is catching up to the science.

Apr 14, 2026 · 7 min read

CLINICAL SIGNAL

Intellia Pauses Nex-Z Phase 3 Trials After Grade 4 Liver Event

Intellia places its CRISPR gene editing therapy nex-z on partial clinical hold following a serious liver adverse event, raising questions about in vivo LNP-delivered editing safety at scale. Over 450 patients had been dosed across MAGNITUDE trials.

Apr 14, 2026 · 7 min read

REGULATORY

Casgevy Approvals Expand to 10 Countries as CRISPR Enters Mainstream

The first CRISPR-based therapy, Casgevy for sickle cell disease and beta-thalassemia, is now approved in the US, EU, UK, Canada, and six additional countries. Real-world data confirms durable transfusion independence.

Apr 2, 2026 · 6 min read

CLINICAL SIGNAL

eGenesis Gene-Edited Pig Kidney Recipient Reaches One Year

The longest-surviving pig-to-human kidney transplant patient crosses the 12-month milestone with stable graft function, powered by 69 genetic edits that removed pig antigens and added human immune genes.

Mar 28, 2026 · 7 min read

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