Parkinson's Cure Journey

Gene therapy & delivery

The lane

Status
Active. Trials are running and the idea is still being tested.
Verdict
Dopamine-making gene therapies ended; brain-crossing capsids advance delivery for other branches.
Family
Restoration
Landmarks
14 in this lane, 5 more tagged with it. Advanced 7 · Open 4 · Dead end 3

The verdict and status come from the project's branches.yaml and use research wording.

Landmarks in this lane

14 landmarks drawn in this lane, oldest first. Select a column heading to sort.
AAV-AADC gene therapy2004–2019Trial in peoplePassed this stage––19 Oct 20262
MRI-guided convection-enhanced delivery2005–2011Research toolConfirmed and built on––19 Oct 20265
AAV2-GAD sham-controlled Phase 22008–2025Trial in peoplePassed this stage24519 Oct 20264
Intravenous AAV9 reaches the brain2009DiscoveryConfirmed and built on––19 Oct 20260
ProSavin and OXB-1022014–2026Trial in peopleDid not work––19 Oct 20263
AAV capsids that cross into the brain2016–2024Research toolConfirmed and built on––20 Oct 20264
Nusinersen: intrathecal antisense works2016–2018Trial in peoplePassed this stage3–19 Oct 20264
VY-AADC Phase 1b and RESTORE-12017–2021Trial in peopleDid not work21419 Oct 20267
Focused ultrasound opens the blood-brain barrier2018–2022Trial in peopleMixed or early results––19 Oct 20263
AAV-PHP.B fails in primates2018–2019DiscoveryConfirmed and built on––20 Oct 20264
Capsids that grip human TfR12024Research toolStill being studied––20 Oct 20265
CAP-003 intravenous GBA1 gene therapy (Capsida)2025–2026Trial in peopleDid not work2–19 Oct 20263
VCAP-102 capsid crosses primate blood-brain barrier2025Research toolStill being studied––20 Oct 20262
VGN-R08b GBA1 gene therapy (Vitalgen)2026–2027Trial in peopleMixed or early results21722 Oct 20265

Also tagged with this lane

5 landmarks drawn in another lane but tagged with this one. Select a column heading to sort.
GDNF: the growth-factor idea, from infusions to gene therapy2003–2026DiscoveryStill being studied––19 Oct 20264
CERE-120 (AAV2-neurturin)2008–2015Trial in peopleDid not work210919 Oct 20264
Gene editing of SNCA2018–2023Lab study (cells or animals)Still being studied––20 Oct 20263
GBA gene therapy (PR001)2020–2026Trial in peopleMixed or early results1/2–19 Oct 20263
REGENERATE-PD AB-10052024–2028Trial in peopleResult due Aug 2028212718 Oct 20263

Built on work from

Led to work in

See this lane on the map · All lanes