Gemma Biotherapeutics
Clinical trials sponsored by Gemma Biotherapeutics, explained in plain language.
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Experimental gene therapy aims to halt devastating brain disease in infants
Disease control TerminatedThis study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best…
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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One-Time gene therapy aims to halt rare, fatal brain disease in children
Disease control OngoingThis study tests a single dose of PBGM01, a gene therapy delivered directly into the fluid around the brain and spinal cord, in children with GM1 gangliosidosis. The therapy uses a harmless virus to carry a working copy of the GLB1 gene, which is missing or faulty in these patien…
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC