Story perspectives

Breakthrough Gene Therapies Show Promise for Hearing, Brain Disorders

10/14/2025

Summary

Story summary

Regeneron's DB-OTO gene therapy for profound hearing loss achieved meaningful hearing improvements in 11 of 12 participants.
Myrtelle's MYR-101 reduced N-Acetylaspartate levels and increased brain myelin volume in Canavan disease patients in a phase 1/2 trial.
In mice with SYNGAP1-related disorders, gene therapy restored brain function and reduced seizures.
The FDA supports innovative therapies through the Rare Disease Endpoint Advancement Pilot Program to boost collaboration for drug development.

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Story summary

Regeneron's DB-OTO gene therapy for profound hearing loss achieved meaningful hearing improvements in 11 of 12 participants.
Myrtelle's MYR-101 reduced N-Acetylaspartate levels and increased brain myelin volume in Canavan disease patients in a phase 1/2 trial.
In mice with SYNGAP1-related disorders, gene therapy restored brain function and reduced seizures.
The FDA supports innovative therapies through the Rare Disease Endpoint Advancement Pilot Program to boost collaboration for drug development.