Oxyrane – enzyme replacement therapies

Oxyrane develops Enzyme Replacement Therapies (ERTs) for the treatment of lysosomal storage diseases (LSDs) using a proprietary engineered yeast expression platform based on Yarrowia lipolytica.

Oxyrane – enzyme replacement therapies

Oxyrane

Lysosomal storage diseases (LSDs) are pathologies where an inherited deficient enzyme leads to undesired cellular waste accumulation and disease. Oxyrane focuses on LSDs where the deficient enzyme can be effectively targeted to the lysosome by interaction with the cation-independent mannose-6 phosphate receptors (CI-M6PR). Indeed, Oxyrane develops recombinant enzymes using a proprietary engineered yeast expression platform based on Yarrowia lipolytica. This Yarrowia lipolytica has been engineered to produce proteins with a specific “targeting” sugar structure required for an effective intracellular localization (mannose 6-phosphate (M6P)). Thus, Oxyrane develops enzyme replacement therapies (ERTs)-based solutions to address the unmet clinical needs across a range of lysosomal storage diseases and can rapidly explore various product concepts to find the best possible engineered solutions. Oxyrane’s lead product is a pre-clinical ERT for Pompe Disease produced by Yarrowia lipolytica engineered yeast cells.

Pompe disease is a rare disease where the progressive accumulation of glycogen results in muscle weakness (mainly heart and skeletal muscle). Pompe disease is caused by a deficiency of the lysosomal acid alpha glucosidase (GAA), responsible for breaking down glycogen. Deficiency in GAA induces an increase in glycogen, disrupting cell functions and impacting muscle function. If left untreated, the early-onset infantile form is fatal before 18 months. OXY2810 is a recombinant human GAA used as an enzyme replacement therapy for Pompe disease. OXY2810 has a natural targeting structure required for efficient localisation to lysosomes.

Founded in 2006, Oxyrane is a private venture-capital funded company headquartered in Manchester (UK) with research operations in Gent (Belgium) and Medical/Regulatory activities in Boston (USA). Management team is composed by Evert Küppers (CEO), Wouter Vervecken (CTO), Charlie Richard and John Tagliamonte. Board Of Directors also includes Philip Astley-Sparke, Christopher Bull, Gerald Chan, Sander Slootweg and Somu Subramanian. Oxyrane’s world-class scientific team has a strong expertise in the engineering of its yeast expression host (Yarrowia lipolytica) to improve protein expression with desirable sugar structures (glycans). Oxyrane’s platform is highly efficient, enabling to go from gene to product in 3 months, allowing the company to evaluate dozens of product variants in parallel. Furthermore, Oxyrane’s engineering ensures batch-to-batch product consistency for both enzyme and sugar structure using a flexible, scalable production.

More about Oxyrane : www.oxyrane.com

Oxyrane – Enzyme Replacement Therapies – ERT – Enzyme Replacement Therapy – lysosomal storage disease – LSD – yeast expression platform – Yarrowia lipolytica – acid alpha glucosidase – GAA – OXY2810 – lysosome – lysosomal – glycan

Oxyrane – mannose-6 phosphate receptor – CI-M6PR – mannose 6-phosphate – M6P – Pompe Disease – Evert Küppers – Wouter Vervecken – Charlie Richard – John Tagliamonte – Philip Astley-Sparke – Christopher Bull – Gerald Chan – Sander Slootweg – Somu Subramanian – Enzyme Replacement Therapies – ERT – Enzyme Replacement Therapy – lysosomal storage disease