Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated in the Past With HSCT (REKLAIM)

Inclusion Criteria: Group 1: Subjects transplanted for infantile onset Krabbe disease (IKD onset <12 months) with initial diagnosis based on: Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING: Elevated psychosine levels predictive of infantile onset by dried blood spot (DBS); OR Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR Two GALC mutations predictive to result in infantile onset phenotype. Group 2: Subjects transplanted for late infantile onset Krabbe (LIKD onset 12-47 months) with signs or symptoms of Krabbe disease, and with initial diagnosis based on: Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of late infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING: Elevated psychosine levels predictive of late infantile onset by DBS; OR Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR Two GALC mutations predictive to result in late infantile onset phenotype; OR Neurological/developmental exam findings consistent with late infantile Krabbe disease Participants must have received HSCT at least 90 days prior to dosing date Chimerism should reflect at least 30% of myeloid cells from the donor by month 3 post-transplant or 10% by one-year post-transplant that result in GALC leukocyte levels > or equal to 0.2 nmol/h/mg. Participant's parents or legal guardian consent to participate in the study and provide informed consent according to IRB/IEC guidelines prior to any study procedures being performed Parent(s) and/or legal guardian able to comply with the clinical protocol Exclusion Criteria: Immunoassay with total anti-AAVrh10 antibody titers of >1:100 History of prior treatment with a gene therapy product Inability to actively move upper extremities against gravity Grade 2 or higher abnormalities in LFTs, bilirubin, creatinine, white count, hemoglobin, platelets, PT/INR and PTT according to latest version of CTCAE Presence of any neurocognitive deficit, motor deficit, or brain damage not attributable to Krabbe disease Signs of active infections or disease from cytomegalovirus, adenovirus, EBV, hepatitis B or C, and HIV or other viruses excluding rhinovirus from RVP and asymptomatic norovirus presence in stool Active bacterial or fungal infection documented in the preceding 7 days Presence of any contraindication for MRI or lumbar puncture (LP) Use of any investigational product prior to study enrollment or current enrollment in another study that involves clinical interventions Immunizations with live viruses in the 30 days prior to immune suppression Ejection fraction of <50% by echocardiogram or other appropriate study without evidence of pulmonary hypertension Active acute Graft Versus Host Disease (GvHD) Grade II or higher according to modified Glucksberg criteria (Przepiorka et al., 1995) or active, moderate, or severe, chronic GvHD according to revised NIH criteria (Jagasia et al., 2015) Any other medical condition, serious intercurrent illness, other genetic condition or extenuating circumstance that, in the opinion of the PI, would preclude participation in the study