Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I). Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene

Any significant infection, defined as those requiring hospitalization or intravenous antimicrobials

Any significant infection, defined as those requiring hospitalization or intravenous antimicrobials

Any new skin or oral lesion potentially caused by underlying LAD-I disease.

Any new skin or oral lesion potentially caused by underlying LAD-I disease.

Any late-occurring (more than 2 years post-infusion) SAE considered at least possibly related to the investigational RP-L201 study treatment.

Any late-occurring (more than 2 years post-infusion) SAE considered at least possibly related to the investigational RP-L201 study treatment.

Incidence of any malignancy

Incidence of any malignancy

Any new incidence of secondary graft failure defined as a sustained decrease in PBMC VCN of <0.1 or PB neutrophil CD18 expression of <10% on 2 consecutive evaluations separated by an interval of at least 1 month, and not considered related to a concurrent infection or due to non-RP-L201 drug-related toxicity.

Any new incidence of secondary graft failure defined as a sustained decrease in PBMC VCN of <0.1 or PB neutrophil CD18 expression of <10% on 2 consecutive evaluations separated by an interval of at least 1 month, and not considered related to a concurrent infection or due to non-RP-L201 drug-related toxicity.

Any new concerns for GvHD based on GVHD clinical classifications defined

Any new concerns for GvHD based on GVHD clinical classifications defined

Allogeneic HSCT-free survival;

Allogeneic HSCT-free survival;

Event free survival defined as survival in the absence of graft failure or GvHD;

Event free survival defined as survival in the absence of graft failure or GvHD;

Reduction of significant infections, defined as those requiring hospitalization or intravenous antimicrobials

Reduction of significant infections, defined as those requiring hospitalization or intravenous antimicrobials

Reduction of infection-related hospitalizations, and infection-related prolonged hospitalization (lasting ≥7 days) beyond the initial 24 months of RP-L201-0318;

Reduction of infection-related hospitalizations, and infection-related prolonged hospitalization (lasting ≥7 days) beyond the initial 24 months of RP-L201-0318;

Improvement or resolution of LAD-I-related neutrophilia and leukocytosis

Improvement or resolution of LAD-I-related neutrophilia and leukocytosis

Resolution of LAD-I-related skin rash or periodontal abnormalities;

Resolution of LAD-I-related skin rash or periodontal abnormalities;

Persistence of transgene in PB cells as demonstrated by VCN of at least 0.1 in PBMCs and PB CD15+ granulocytes;

Persistence of transgene in PB cells as demonstrated by VCN of at least 0.1 in PBMCs and PB CD15+ granulocytes;

Persistence of CD18 neutrophil expression defined by PB neutrophil CD18 expression to at least 10% of normal;

Persistence of CD18 neutrophil expression defined by PB neutrophil CD18 expression to at least 10% of normal;

Persistence of CD11 a/b neutrophil co-expression.

Persistence of CD11 a/b neutrophil co-expression.