Sanofi Genzyme goes to Phase III with Rare Neuromuscular Disease

08/03/2016 - 2 minutes

Sanofi Genzyme (Boston and France) have obtained good phase I/II results with a candidate for the rare Pompe Disease and expects to move to phase III in the second quarter of 2016.

sanofi_genzyme_pompe_disease_neoGAA_neo1_phase_III_gaa_enzyme_replacement_therapy_ertSanofi Genzyme continues to bet on rare diseases and neurodegenerative disorders, now with a therapy for late-onset Pompe Disease.

Pompe Disease is a neuromuscular condition caused by a dysfunction in acid alpha-glucosidase (GAA), a lysosomal enzyme that is key to break down glycogen (a common sugar ‘storage’ molecule in muscles). Without the enzyme, glycogen accumulates and damages the muscles.

This condition can affect babies (early onset) or develop later in life (late onset). Though the prognosis improves with later onset, Pompe is a debilitating disease and patients experience difficulties with walking and breathing.

Treatment involves enzyme replacement therapy (ERT), and Sanofi Genzyme is developing a second-generation therapy (neoGAA) based on new engineered enzymes.

These enzymes have a greater affinity for M6P receptors (a type of cellular ‘gate’

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