OF REVIEW: Hereditary hearing loss has historically been approached as a diagnostic category rather than a therapeutically modifiable disease. Recent advances in molecular genetics, cochlear gene delivery, and first-in-human clinical trials are changing that....
✦ The floor
Discussion
Signed responses from readers of the wire.
No actionable change for current clinical practice; this is a regulatory and ethical framework paper for researchers planning early-phase gene therapy trials, not a report of clinical outcomes.
A clear translational roadmap for inner-ear gene therapy trials could accelerate the path from laboratory discovery to safe first-in-human studies for hereditary hearing loss.
- 01Proposes a structured framework for Phase I/II inner-ear gene therapy trial design in hereditary hearing loss.
- 02Addresses regulatory strategy including FDA and EMA considerations for novel gene therapy products.
- 03Raises key ethical issues: patient selection, informed consent, and pediatric enrollment in gene therapy trials.
- 04Highlights the need for validated outcome measures and safety biomarkers before first-in-human trials.
- 05Intended to bridge the gap between preclinical gene therapy research and clinical translation.
A translational framework can standardize early-phase gene therapy trial design for hereditary hearing loss.
opinionunclearRegulatory and ethical considerations are a major barrier to inner-ear gene therapy clinical translation.
opinionpartially supported- PMID
- 42614064
- DOI
- 10.1097/MOO.0000000000001155.
- Journal
- Current Opinion in Otolaryngology & Head and Neck Surgery
- Publication type
- review
- Evidence level
- 5
- Population
- Not applicable — framework/review article for hereditary hearing loss gene therapy trials
- Intervention
- Translational framework for early-phase inner-ear gene therapy clinical trials
Primary outcomes
Proposed trial design elements; Regulatory strategy recommendations; Ethical considerations identified