HGB-206 (lovo-cel)
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease
Lentiviral β-globin gene therapy reduces or eliminates VOC events in severe sickle cell disease.
Source publication
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease
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Sponsor
Genetix Biotherapeutics Inc.
Principal investigator
Anjulika Chawla, MD, FAAP
Population
Sickle Cell Disease; n=54
Primary endpoint
Percentage of Group C Participants Who Achieved Complete Resolution of Vaso-occlusive Events (VOE-CR)
Methodology notes
Full methodology is under curation. This project aggregates 3 seeded claims across three temporal layers. The replication spec, pre-registered analysis, and version history will appear here as the project matures.
Funding progress
$645K / $2.9M
22%
Funding displayed here is indicative of collaborator interest. Sciync does not broker money, grants, or securities.
Temporal layers
Published findings under validation
1
Active replication in progress
1
Forecast horizons pending resolution
1