drugset
Drugs / AAV9 carrying the IGHMBP2 gene
last change Nov 2021 re-read 3 minutes ago

AAV9 carrying the IGHMBP2 gene

Gene therapy (AAV / viral vector)

Developed for
autosomal recessive distal spinal muscular atrophy 1 · Charcot-Marie-Tooth disease type 2
Investigated by
Megan Waldrop

Trials 1

today NCT05152823 · Ph 1/2
2022202320242025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT05152823 Nov 2021 → Jul 2028 expected Charcot-Marie-Tooth disease type 2, autosomal recessive distal spinal muscular atrophy 1 Megan Waldrop Enrolling by invitation No outcome recorded

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as AAV9 carrying the IGHMBP2 gene “AAV9 carrying the IGHMBP2 gene.” NCT05152823 ↗
Modality Gene therapy (AAV / viral vector) “AAV9 vector carrying the IGHMBP2 gene” NCT05152823 ↗
Route Intrathecal “single intrathecal injection” NCT05152823 ↗