Regeneron gene therapy improves hearing in 10 children with rare form of hearing loss

Regeneron's Gene Therapy Breakthrough for Childhood Deafness

Regeneron's gene therapy DB-OTO has shown significant improvements in hearing for children with otoferlin-related genetic deafness134.

In the Phase I/II CHORD trial, 10 out of 12 children with profound genetic hearing loss demonstrated clinically meaningful improvements in hearing after receiving DB-OTO4.

The therapy involves delivering a working copy of the OTOF gene to the inner ear using a modified, non-pathogenic virus4.

Opal Sandy, an 11-month-old British baby, was among the youngest to receive the therapy and showed dramatic improvements in hearing within 24 weeks16.

The first child treated in the trial, at 10 months old, showed speech and development progress following hearing improvements, with results lasting up to 72 weeks4.

DB-OTO is administered via a single intracochlear injection, similar to the procedure used for cochlear implantation4.

The therapy aims to restore hearing in patients with congenital otoferlin hearing loss, a rare condition caused by variants in the OTOF gene13.

Improvements in hearing were assessed using behavioral sound confirmation, pure tone audiometry (PTA), and auditory brainstem response (ABR)3.

The surgical procedure and DB-OTO were well-tolerated, with no related adverse or serious adverse events reported3.

This breakthrough could potentially extend to individuals with hearing loss from other common genetic conditions3.

Sources:

1. https://www.clinicaltrialsarena.com/news/regenerons-gene-therapy-triumphs-twice-restoring-hearing-in-children/

3. https://www.regmednet.com/breakthrough-in-gene-therapy-for-genetic-deafness/

4. https://hearingpractitionernews.com.au/regeneron-gene-therapy-improves-hearing-in-10-deaf-children-in-chord-trial-aro-conference-hears/

6. https://www.cuh.nhs.uk/news/baby-born-deaf-can-hear-after-breakthrough-gene-therapy/