Читать книгу Biologics, Biosimilars, and Biobetters - Группа авторов - Страница 106

Enzymes have been a long‐standing target for drug development, including small molecules as well as biologics. The biologic enzyme replacement therapies (ERTs) for the treatment of lysosomal storage diseases (LSDs) such as Fabry disease, Gaucher disease, and Pompe disease offer a vantage point into some of the current successes and future potential in this area of biotechnology. There are 50–60 different rare, genetically inherited disorders resulting in deficient lysosomal enzymes. When lysosomal enzymes do not work properly, fats and other enzyme substrates build up throughout the body resulting in widespread cellular complications including death. Enzyme replacement involves the production of enzyme proteins through rDNA technology, then these enzymes are infused into the patient on a recurrent basis as life‐long replacement therapy. ERTs have resulted in significant clinical benefit to patients including improved quality of life, walking ability, and respiratory function improvements. However, challenges exist such as neutralizing antibodies and other immune reactions. ERTs are not always able to reach the desired target cells such as those in the central nervous system. Potentially 75% of patients with neurologic dysfunctions may not be adequately treated with ERTs, often due to challenges with drug design and inability to penetrate the blood–brain barrier. Drugs marketed for ERT are some of the most expensive drugs in the world today. Gene therapy is one of many scientific advances being explored to treat these conditions in patients.¹⁴