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).

Background: Sickle cell disease (SCD) is an inherited blood disorder.

One in 300 Jamaicans have HbSS sickle cell disease, and of these, up to 70% will suffer from sickle cell leg ulcers (SCLUs).

This is a phase 1, first-in-human, multi-center, open-label, single ascending dose (SAD) cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with stable sickle cell disease (SCD).

The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease.

This study will evaluate two genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) products - OTQ923 and HIX763 - each reducing the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life.

Sickle cell disease is the most common single-gene disease in the world.

Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis and acute thoracic syndrome are the most frequent.

The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease.