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Gene Therapy

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UCSF-Led Study Engineers CAR-T Cells In Vivo, Clearing Tumors in Mice

Researchers say the preclinical method now requires scaling followed by human trials.

FDA Demands Placebo-Controlled Trial for UniQure’s Huntington’s Gene Therapy

FDA Proposes ‘Plausible Mechanism’ Pathway for Individualized Rare-Disease Therapies

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million

Lilly to Buy Adverum in Gene‑Therapy Deal Worth Up to $12.47 a Share

FDA Names First 9 Drugs for ‘National Priority’ Fast-Track Reviews, Targeting 1–2-Month Decisions

Former FDA Biologics Chief Peter Marks Joins Eli Lilly to Lead Infectious Disease and Molecule Discovery

Gene Therapy Slows Huntington’s Progression by 75% in Early Trial, uniQure Says

Capsida Halts STXBP1 Gene Therapy Trial After First Pediatric Participant Dies

Kriya Nets $320 Million Series D as Odyssey Raises $213 Million and Amgen Retools Research Leadership

Single Birth Injection Protects Primates From HIV, Paves Way for Newborn Safety Trials

Precision RNA Therapy Reverses Progeria in Preclinical Models

Eli Lilly to Acquire Verve Therapeutics for $1.3 Billion

mRNA Nanoparticles Reverse HIV Latency in Resting T Cells