Gene Therapy
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FDA Grants Accelerated Approval to First Gene Therapy for Severe Leukocyte Adhesion Deficiency, Capping a Two-Year Regulatory Journey
Rocket Pharmaceuticals' Kresladi becomes the first gene therapy approved for LAD-I, an ultra-rare immune disorder that leaves children vulnerable to fatal infections.
UC San Diego Engineers CRISPR Gene Drive That Spreads Through Bacteria to Strip Away Antibiotic Resistance
Researchers at the University of California San Diego have developed pPro-MobV, a second-generation CRISPR-based gene drive that spreads through bacterial populations via conjugal transfer to disable antibiotic resistance genes, demonstrating effectiveness even within biofilms.
Mass General Team's INSTALL Platform Solves Gene Therapy's Immune Toxicity Problem, Enabling Safe Non-Viral DNA Insertion in Living Mice
A Nature paper describes INSTALL, a gene editing platform using circular single-stranded DNA to insert therapeutic genes without triggering fatal immune responses, keeping mice healthy at doses where conventional DNA proved lethal.
FDA Lifts Clinical Hold on Intellia's CRISPR Gene-Editing Trial for Heart Disease After Patient Death and Safety Overhaul
The FDA has cleared Intellia Therapeutics to resume its MAGNITUDE Phase 3 trial of nexiguran ziclumeran for ATTR cardiomyopathy, five months after a patient death and subsequent clinical hold prompted a safety overhaul.
Sana Biotechnology Reports 14-Month Survival of Transplanted Islet Cells Without Immunosuppression in a Type 1 Diabetes Patient
Gene-edited pancreatic islet cells transplanted into a patient with type 1 diabetes continue producing insulin 14 months later with no immunosuppressive drugs, advancing the case for a functional cure.
In Vivo CAR-T Therapy Delivers First Human Proof-of-Concept for Autoimmune Reset, Bypassing Lab Manufacturing
MagicRNA's mRNA lipid nanoparticle therapy reprogrammed patients' own T cells inside the body, wiping out disease-driving B cells in refractory lupus patients without conventional cell manufacturing.
FDA Reverses Course on UniQure's Huntington's Gene Therapy, Demanding Sham Surgery Trial in Setback That Could Add Years to Approval Timeline
The FDA rejected uniQure's Phase I/II data for AMT-130 and now demands a sham surgery-controlled Phase 3 trial, reversing its own prior guidance and sending shares down 40 percent.
Base Editing Corrects a Genetic Disease for the First Time in Living Patients, Putting BEAM-302 on Track for FDA Accelerated Approval
Beam Therapeutics' BEAM-302 has achieved the first-ever in-vivo base editing correction of a disease-causing mutation in human patients, reaching therapeutic AAT levels in a Phase 1/2 trial for alpha-1 antitrypsin deficiency. A pivotal Q1 2026 data readout is now imminent.
UC San Diego Team Builds a CRISPR Gene Drive That Spreads Through Bacteria and Strips Away Antibiotic Resistance
A second-generation Pro-Active Genetics system spreads via bacterial conjugation to disable resistance genes on plasmids, working even inside biofilms.
The FDA's Plausible Mechanism Pathway: How Baby KJ's Personalized CRISPR Therapy Is Rewriting the Rules of Drug Approval
The FDA's new Plausible Mechanism Pathway enables approval of one-patient gene editing therapies without traditional clinical trials, inspired by the first personalized CRISPR cure for an infant with a fatal metabolic disorder — but ethicists warn of a regulatory Pandora's box.
Epigenetic CRISPR Technique Reactivates Silenced Genes Without Cutting DNA
UNSW and St. Jude researchers demonstrate that removing methyl tags from DNA can switch genes back on, opening safer paths for treating sickle cell disease.