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Regenxbio’s Duchenne gene therapy shows improved muscle function in trial

By Thomson Reuters Mar 11, 2026 | 11:00 AM

March 11 (Reuters) – Regenxbio said on Wednesday that interim ​data from ‌a early-to-mid stage study of its experimental gene therapy in ‌patients ​with Duchenne ⁠muscular dystrophy showed ⁠continued improvement in muscle function and a clean safety ​profile.

The company was testing the ⁠treatment, called ⁠RGX-202, in ​boys aged 1 to ​12.

The company said children ‌who received the therapy showed better performance at ⁠one year across standard tests used to track ⁠how ‌quickly Duchenne ⁠progresses, such ​as ‌how fast they ​can stand ⁠up or climb stairs.

(Reporting by Kamal Choudhury in Bengaluru; Editing by Leroy ​Leo)