CLN7

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Not yet finished but already full! 1

One-Time gene injection holds promise for rare childhood brain disease

Disease control Ongoing

This early-stage trial tests a single injection of gene therapy (AAV9/CLN7) into the spinal fluid of children with CLN7 Batten disease, a rare and severe genetic disorder that causes progressive loss of motor and mental abilities. The main goal is to check safety, but researchers…

Matched conditions: CLN7

Phase: PHASE1 • Sponsor: Benjamin Greenberg • Aim: Disease control

Last updated Apr 26, 2026 20:01 UTC

CLN7

Get alerted when new CLN7 trials appear

One-Time gene injection holds promise for rare childhood brain disease