Neurogene (NGNE) Goldman Sachs 45th Annual Global Healthcare Conference summary

Corporate overview and strategic positioning

• Focuses on neurological gene therapy with a proprietary EXACT technology platform targeting commercially attractive indications, notably Rett syndrome as the lead program.

• Maintains internal manufacturing capabilities in Houston, supporting quality control and cost efficiency.

• Cash runway extends into the second half of 2026, supporting key development milestones.

• Two clinical-stage programs: Rett syndrome and CLN5 Batten disease.

Rett syndrome program highlights

• Rett syndrome is caused by MECP2 gene deficiency, affecting 6,000–9,000 potential U.S. patients, with significant global market potential.

• EXACT technology enables tight regulation of gene expression, minimizing toxicity and maximizing therapeutic window.

• Preclinical data in mice show dose-dependent survival benefits and strong safety profile, with the microRNA component critical for safety.

• Intracerebroventricular (ICV) administration chosen for optimal brain delivery, supported by non-human primate studies.

• Direct pediatric trial entry approved by FDA, with global regulatory concurrence in the UK and Australia.

Clinical trial design and progress

• Phase 1/2 trial in children aged 4–10, with low and high dose cohorts enrolling concurrently.

• Objective, clinically meaningful endpoints focus on communication, hand function, and ambulation.

• Early safety data show favorable tolerability, with only transient liver enzyme elevations and no serious adverse events.

• Key interim data expected in Q4 2024, with more mature data in the second half of 2025.