Story perspectives

FDA Approves First Thymidine Treatment Amid Breakthroughs in Gene Therapy

11/6/2025

Summary

Story summary

UCB received FDA approval for Kygevvi, the first treatment for thymidine kinase 2 deficiency, based on a trial showing an 86% reduction in death risk.
GeneEditBio treated its first patient with GEB-101, a Crispr-Cas gene-editing therapy for TGFBI corneal dystrophy, as part of a trial with Fudan University.
Praveen Khandelwal, Indian Member of Parliament, urged Prime Minister Modi to accelerate policy changes to expedite treatment for LAMA2-related congenital muscular dystrophy.

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Story summary

UCB received FDA approval for Kygevvi, the first treatment for thymidine kinase 2 deficiency, based on a trial showing an 86% reduction in death risk.
GeneEditBio treated its first patient with GEB-101, a Crispr-Cas gene-editing therapy for TGFBI corneal dystrophy, as part of a trial with Fudan University.
Praveen Khandelwal, Indian Member of Parliament, urged Prime Minister Modi to accelerate policy changes to expedite treatment for LAMA2-related congenital muscular dystrophy.