Gene-Editing hope: could BIVV003 free sickle cell patients from pain crises?

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria * Ages 18 to 40 * Confirmation of sickle cell disease (SCD) diagnosis (HbSS or HbS\[beta\]0 genotype) * Severe SCD, defined as having 1 or more of the following manifestations: Clinically significant neurologic event (example \[e.g.\], stroke) or any neurological deficit lasting more than 24 hours; History of 2 or more episodes or Acute Chest Syndrome (ACS) in 2 years prior to informed consent (despite adequate supportive therapies such as asthma therapy); Six or more pain crises per year in 2 years prior to informed consent (requiring intravenous \[IV\] pain management in the outpatient or inpatient hospital setting); History of 2 or more cases or priapism with participant seeking medical care in the 2-years prior to informed consent; Regular RBC transfusion therapy in the year prior to informed consent (having received 8 or more transfusions to prevent vaso-occlusive clinical complications); and Echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity of greater than or equal to 2.5 meter per second (m/s) * Clinically stable to undergo stem cell mobilization and myeloablative hematopoietic stem cell transplantation (HSCT) * Adequate physiological function, defined as the following: Karnofsky/Lansky Performance of greater than or equal to 60; Acceptable cardiac function as defined in protocol; Acceptable pulmonary function as defined in protocol; Acceptable renal function as defined in protocol; and Acceptable hepatic function as defined in protocol * Ability to understand purpose and risks of study, provide Informed Consent Form (ICF) and authorization to use protected health information * Completion of age-appropriate cancer screening * Willingness to use double-barrier method of contraception through entire study period (for participants of childbearing potential) * Willingness to receive blood transfusions * Willingness to discontinue hydroxyurea (HU) at least 30 days prior to stem cell mobilization through Day 100 post-transplantation Exclusion Criteria: * Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation * Previous treatment with gene therapy * Current enrollment in an interventional study or having received an investigational drug within 30 days of study enrollment * Pregnant or breastfeeding female * Female or male who plans to become pregnant or impregnate a partner, respectively, during the anticipated study period * Contraindication to plerixafor, apheresis, or busulfan * Treatment with prohibited medication in previous 30 days * Known allergy or hypersensitivity to plerixafor, busulfan, or investigational product excipients * History of active malignancy within past 5 years, any history of hematologic malignancy, or a family history of a cancer predisposition syndrome (without negative result of candidate) * Current diagnosis of uncontrolled seizures * History of significant bleeding disorder * Clinically significant infection * Any major organ dysfunction involving brain, kidney, liver, lung, or heart (e.g., congestive heart failure, pulmonary hypertension) * Corrected QT interval of more than 500 millisecond (ms) based on screening electrocardiogram (ECG) * Positive for human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV) * Known to have a gamma-globin variant associated with altered oxygen affinity * Hereditary persistence of fetal hemoglobin (HPFH) or HbF concentration of more than or equal to 20 percent (%) at screening * Absolute Neutrophil Count (ANC) of less than or equal to 1,000 per microliter * Platelet count of less than 100,000 per microliter * History of platelet alloimmunization (precluding ability to provide transfusion support) * Extensive Red Blood Cell (RBC) alloimmunization (precluding ability to provide transfusion support) * Judged unsuitable for participation by investigator and/or sponsor