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

PI Anjulika Chawla, MD, FAAP

Primary endpoint

Percentage of Group C Participants Who Achieved Complete Resolution of Vaso-occlusive Events (VOE-CR)

Population

Sickle Cell Disease; n=54

Historical

Current

Future

3 noemes

Historical

1 noeme

Published findings from this trial. Vote whether the expert body still believes each noeme stands given current evidence.

The HGB-206 (lovo-cel) trial's stated primary conclusion — Lentiviral β-globin gene therapy reduces or eliminates VOC events in severe sickle cell disease. — replicates in independent cohorts.

gene-therapy

Current

1 noeme

Noemes tracking active replication, subgroup analysis, or long-term follow-up. Vote on the likelihood the original effect holds.

Recent follow-up analyses of HGB-206 (lovo-cel) are confirming the original effect size in real-world data.

gene-therapy

Future

1 noeme

Open forecast horizons tied to this trial. Vote your probability that each resolves TRUE by the declared horizon.

By 2028, at least two sickle cell gene-therapy products will compete head-to-head on real-world VOC-free survival and cost-effectiveness.

gene-therapy

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