Delivery of therapeutic proteins using
Lectin-Mediated Transport
BioStrategies’ proprietary technology exploits natural plant lectins (sugar-binding proteins) to direct the uptake and intracellular trafficking of proteins in human cells, tissues and organs. This provides unique biodistribution patterns compared to currently approved biotherapeutics. These therapeutic proteins typically use receptor-mediated mechanisms (e.g. lysosomal enzyme replacement therapies) to enter cells. While effective for many diseases, they are unable to treat tissues and organs protected by biological barriers, such as central nervous system, bone, eye and cartilage. In contrast, our lectins bind to diverse glycoproteins and glycolipids abundant on human cell surfaces enabling delivery into a wide range of cell types. Advantages of our platform includes:
CNS delivery
Lectin-enzyme fusions are able to cross the blood brain barrier after intravenous administration. In vivo pre-clinical studies in three lysosomal storage disease models have provided evidence for transport of corrective doses of enzymes across the blood–brain barrier to treat CNS pathologies.
Review Article
https://www.mdpi.com/1422-0067/21/3/971
Bone and Muscle Delivery
Delivery of therapeutics to bone and muscle is challenging. The avascular nature of cartilage in the growth plate limits the delivery of therapeutics for prevention of abnormal bone growth. Delivery to the muscle is also difficult and requires very high doses of product to achieve improvement due to the type of receptors present in muscle cells. Our lectin-fusion therapeutics are capable of delivering corrective doses of product to these tissues, proving to prevent the development of pathologies in animal models.
Mitigates the effect of anti-drug antibodies
Therapies design to replace the missing protein in lysosomal storage disorders are often compromised by the development of neutralizing antibodies to the enzyme (anti-drug antibodies; ADA). ADA responses hinder treatment efficacy of the majority of therapies available to patients, blocking the entry of the replacement therapeutic to key organs and tissues. Over 90% of the patients with Hurler (MPS I), MPS VIA, Pompe and Fabry develop antibodies to their approved drug.
Our lectin delivery platform is able to deliver corrective doses of enzyme to the brain, heart, kidney, bone and muscle even in the presence of blocking antibodies in animal models.