Plants are a promising alternative for the production of biotherapeutics. Manufacturing in-planta adds plant specific glycans. To understand immunogenic potential of these glycans, we developed a validated method to detect plant specific glycan antibodies in human serum. Using this assay, low prevalence of pre-existing anti-plant glycan antibodies was found in healthy humans (13.5%) and in glucocerebrosidase-deficient Gaucher disease (GD) patients (5%). A low incidence (9% in naïve patient and none in treatment experienced patients) of induced anti-plant glycan antibodies was observed in GD patients after up to 30 months replacement therapy treatment with taliglucerase alfa, a version of human glucocerebrosidase produced in plant cells. Detailed evaluation of clinical safety and efficacy endpoints indicated that anti-plant glycan antibodies did not affect the safety or efficacy of taliglucerase alfa in patients. This study shows the benefit of using large scale human trials to evaluate the immunogenicity risk of plant derived glycans, and indicates no apparent risk related to anti-plant glycan antibodies.
Study Results - ClinicalTrials NCT00376168
Results from Study PB-06-001, ‘A Phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease’. ERT-naïve adult patients (N=32) with a total treatment duration of 9 months (study started on August 2007 and ended September 2009). Results available at ClinicalTrials.gov: NCT00376168. Please acknowledge the National Library of Medicine (NLM) in subsequent use of this document.
While using these results please cite the study in PLOS One and acknowledge the National Library of Medicine.
Study Results - ClinicalTrials NCT00712348
Results from Study PB-06-002, ‘A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (Taliglucerase alfa) in Patients with GD Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy.’ Adult and pediatric patients (N=26 and 5, respectively) with a total treatment duration of 9 months (study started on December 2008 and ended January 2013). Results available at ClinicalTrials.gov: NCT00712348. Please acknowledge the National Library of Medicine (NLM) in subsequent use of this document.
While using these results please cite the study in PLOS One and acknowledge the National Library of Medicine.
Study Results - ClinicalTrials NCT01132690
Results for Study PB-06-005, ‘A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of TGA in Pediatric patients with Gaucher Disease’. Pediatric patients (N=11) with a total treatment duration of 12 months. (study started on August 2010 and ended July 2012). Results available at ClinicalTrials.gov: NCT01132690. Please acknowledge the National Library of Medicine (NLM) in subsequent use of this document.
While using these results please cite the study in PLOS One and acknowledge the National Library of Medicine.