beeldscherm schreef op 30 juli 2018 15:38:
[...]
dat ze dit maar eens goed lezen en laten inwerken,
Therapy of Pompe Disease
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• Enzyme replacement with recombinant human a-Glucosidase (rhaGLU)
• Two rhaGLU products on the market (from SANOFI-GENZYME): Myozyme® and Lumizyme®
• Current market size around $1.0~$1.2 billion
• Other products under development using similar CHO-cell derived rhaGLU base
• Current treatment: large (6 hour+) infusions of immunogenic version of a-Glu every 2 weeks
• Current estimation of market penetration 20-50%
• Infusion reactions in 50% of patients, and antibody formation in 80%+ of patients
• Immunogenic side effects in significant portion of patients
• Anaphylaxis/cardiac arrest in 1% of patients
• FDA Boxed Warnings on both products
Results of a Phase II clinical study with rabbit-derived a-glucosidase
Achievements in developing a-glucosidase (rhaGLU)
• Transgenic rhaGLU lines generated (cDNA and gDNA) and lead lines identified
• Milk expression confirmed (> 5 g/l)
• Glycosylation pattern significantly different from existing products and much closer to
human a-glucosidase
? Natural a-glucosidase has 20 mannose-6 phosphate (M6P) groups to enable cell
membrane entry – all approved products have fewer than 4 M6P groups
? New rabbit line has almost normal M6P number
• All key analytical assays developed (activity, purity, identity)
• Production of validation batches underway
• Initiation of clinical trial supplies
• IND filing/ start of clinical trials planned for 1H2019