Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD

Inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cells, Pyruvate Kinase Deficiency

1.Insertional mutagenesis:Evaluation of gene-modified clonal repertoire and lentiviral ISA in blood and, if feasible, BM cells via modified gene sequencing (MGS)-PCR. 2.RCL (as required and in settings where there is clinical suspicion of unexplained viral illness) in blood. 3. Immunogenicity: Evidence of antibodies against coRPK (or other LV components) in blood (serum)

*Achievement of stem cell engraftment ≤42 days after infusion. Neutrophil engraftment is defined as the first of three consecutive days with an absolute neutrophil count ≥500/μL. Platelet engrafment is defined as the first of three consecutive days with a platelet count ≥ 20,000/μL in the absence of thrombopoietin mimetics and independent of platelet transfusions for at least 7 days. *Time to stem cell engraftment. *Incidence of respiratory complications

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