CLN3

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Not yet finished but already full! 1

Experimental gene therapy aims to halt rare fatal brain disease in children

Disease control Ongoing

This early-stage trial tests a one-time gene therapy called CLN-301 in 7 children aged 3 to 10 with CLN3 Batten disease, a rare genetic disorder that causes progressive loss of vision, movement, and thinking skills. The therapy delivers a working copy of the CLN3 gene directly in…

Matched conditions: CLN3

Phase: PHASE1, PHASE2 • Sponsor: Neela Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 13:06 UTC

CLN3

Get alerted when new CLN3 trials appear

Experimental gene therapy aims to halt rare fatal brain disease in children