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Grace Science, Llc

Clinical trials sponsored by Grace Science, Llc, explained in plain language.

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Trials from this sponsor expected to report results within the next 12 months.

Experimental gene therapy aims to halt rare childhood disease Results expected Mar 2027 (estimated)

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Not yet finished but already full! 1

Experimental gene therapy aims to halt rare childhood disease

Disease control Ongoing

This study tests a one-time gene therapy called GS-100 in 10 children aged 2 to 18 with NGLY1 deficiency, a rare genetic disorder. The therapy is given directly into the brain fluid to deliver a working copy of the missing gene. The goal is to improve motor skills and development…

Phase 3 • Sponsor: Grace Science, LLC • Aim: Disease control

Last updated Jun 27, 2026 12:29 UTC