A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

Summary

This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).

Timeline

Start

2020-02-14

Primary completion

2027-05

Completion

2027-11

Drugs

Indications