Biotechnology
Gene editing, cancer biotechnology, regenerative medicine, longevity science and the evidence moving from laboratories into clinical care.
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A Baby’s One-Off CRISPR Treatment Is Now a Blueprint for Rare Disease
A personalised CRISPR treatment built for one baby is reshaping rare-disease medicine. Here is what worked, what remains uncertain and what comes next.
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CRISPR Gene Therapy Approved for Children as Young as Two
Casgevy is approved from age two for sickle-cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta thalassaemia. Learn the risks.
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Pancreatic Cancer Prevention Vaccine Trains Immunity Before Disease
A small Phase 1 KRAS vaccine trial produced immune responses in people at inherited pancreatic cancer risk. It has not established cancer prevention.
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Tiny CRISPR Could Unlock Gene Editing Inside the Body
A compact CRISPR enzyme could fit a complete editor into one AAV vector. Explore the preclinical research and unresolved delivery and safety questions.
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Cholesterol Base Editing: Can One Infusion Replace Years of Treatment?
VERVE-102 lowered LDL in a small Phase 1b cholesterol base-editing trial. Explore the PCSK9 mechanism and why long-term safety and benefit remain unproven.
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In Vivo CRISPR Gene Editing Passes Its First Phase 3 Test
Intellia reports fewer hereditary angioedema attacks in a Phase 3 lonvo-z trial. In vivo CRISPR still needs regulatory review and longer safety follow-up.
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Partial Epigenetic Reprogramming Enters Human Trials
Partial epigenetic reprogramming has entered a Phase 1 eye-disease trial. Here is what ER-100 is testing, how OSK works and what remains unproven.
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Off-the-Shelf CAR-T Reaches a Pivotal Leukaemia Trial
Off-the-shelf CAR-T candidate lasme-cel has entered a pivotal B-ALL trial after encouraging early responses. Here is how it works and why the evidence remains preliminary.
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Personalised mRNA Cancer Vaccine Meets Its Phase 3 Goals in Melanoma
Moderna and Merck say a cancer vaccine built from each patient’s tumour has met the key goals of a large Phase 3 melanoma trial—a major validation of personalised mRNA biotechnology, although the full results are still pending.