April 21, 2025 11:00 AM EDT

China startup injects CRISPR therapy into the brain for the first time

Ryan Cross

Senior Science Correspondent

Late last year, doctors injected an experimental CRISPR therapy into the brain of a 9-year-old boy with a rare neurodevelopmental condition that can cause intellectual …

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China startup injects CRISPR therapy into the brain for the first time

Ryan Cross

Senior Science Correspondent

Sign up to read this article for free.

Makary, in one of first comments on FDA policy, floats new pathway for rare disease drugs

Trodelvy and Keytruda combo succeeds in Phase 3 breast cancer study

Roivant brings rare disease program closer as Immunovant CEO retires

FDA approves Dupixent for hives, rejects new Eylea dosing schedule

FDA plans to ban pharma employees from serving on adcomms. The impact will be limited

ADSTILADRIN® (nadofaragene firadenovec-vncg) – A Growing Body of Clinical Data and Real-World Evidence

Chi­na start­up in­jects CRISPR ther­a­py in­to the brain for the first time

Ryan Cross

Senior Science Correspondent

Sign up to read this article for free.

Makary, in one of first com­ments on FDA pol­i­cy, floats new path­way for rare dis­ease drugs

Trodelvy and Keytru­da com­bo suc­ceeds in Phase 3 breast can­cer study

Roivant brings rare dis­ease pro­gram clos­er as Im­muno­vant CEO re­tires

FDA ap­proves Dupix­ent for hives, re­jects new Eylea dos­ing sched­ule

FDA plans to ban phar­ma em­ploy­ees from serv­ing on ad­comms. The im­pact will be lim­it­ed

AD­STI­LADRIN® (nad­o­fara­gene fi­rade­n­ovec-vncg) – A Grow­ing Body of Clin­i­cal Da­ta and Re­al-World Ev­i­dence

China startup injects CRISPR therapy into the brain for the first time

Makary, in one of first comments on FDA policy, floats new pathway for rare disease drugs

Trodelvy and Keytruda combo succeeds in Phase 3 breast cancer study

Roivant brings rare disease program closer as Immunovant CEO retires

FDA approves Dupixent for hives, rejects new Eylea dosing schedule

FDA plans to ban pharma employees from serving on adcomms. The impact will be limited

ADSTILADRIN® (nadofaragene firadenovec-vncg) – A Growing Body of Clinical Data and Real-World Evidence