Genterapier stoppas efter tumör hos femåring

Utvecklingen av en hjärntumör hos en studiedeltagare har fått FDA att tills vidare stoppa två pågående genterapiprogram. Det drabbade bolaget, Regenxbio, förlorade med ens en nästan femtedel av sitt börsvärde.

By Anders Göransson

January 29, 2026 11:31 AM

Tumören upptäcktes vid en rutinmässig magnetkameraundersökning av en symtomfri femårig deltagare som fyra år tidigare behandlats med genterapikandidaten RGX-111 mot den sällsynta, ärftliga ämnesomsättningssjukdomen Hurlers syndrom.

En utredning pågår för att fastställa om tumören är läkemedelsrelaterad, uppger Regenxbio i ett pressmeddelande.

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