Gene and cell therapy

About 300 products for gene and cell therapy are in development

Pharmacy online art.ia based on the materials of PhRMA: Medicines in development for cell therapy and gene therapy

Not so long ago, the idea of a technology that would use gene modification to achieve the desired medical effects was considered almost science fiction. However, today gene therapy, as well as cell therapy, is a reality that has marked a new era of medicine.

Potentially, technologies such as gene and cell therapy can be used in the treatment of more than 100 diseases. According to a report by the Association of Researchers and Manufacturers of Pharmaceutical Products of the USA (Pharmaceutical Research and Manufacturers of America) Currently, almost 300 candidates for drugs for gene or cell therapy of a wide range of diseases are at various stages of development.

Most of them (more than 100) are being developed for the treatment of oncological diseases. 28 candidates for drugs are intended for the treatment of eye diseases, 24 – cardiovascular system, 22 - neurological disorders, 21 – blood diseases, 15 – hereditary diseases. Research and development is also being carried out for the treatment of Alzheimer's disease, arthritis and musculoskeletal diseases, autoimmune disorders, bladder disorders, Crohn's disease, diabetes mellitus, infectious diseases, kidney diseases, liver, muscular dystrophy, respiratory diseases, skin, etc.

It is worth noting that technologies such as:

• a vaccine based on dendritic cells for the treatment of prostate cancer. In this case, the patient's own cells are used, which helps his immune system to develop a long-lasting immune response against cancer cells;
• gene therapy for the treatment of a rare inherited form of blindness associated with a mutation of the RPE65 gene. This is the first gene therapy approved in the United States aimed at treating a disease caused by mutations only in a specific gene;
• 2 drugs for CAR-T-cell therapy: one of them is intended for the treatment of B-cell acute lymphoblastic leukemia; the second is for the treatment of certain types of B-cell lymphoma. CAR-T cell immunotherapy uses the patient's own T cells, which are genetically modified in the laboratory and then injected back into the body. After modification, the patient's T cells become capable of recognizing and destroying cancer cells;
• a drug for targeted RNA therapy of amyloid polyneuropathy in adult patients, designed to interfere with the synthesis of RNA of the abnormal protein transthyretin. By preventing the production of TTP (transthyretin), the drug can reduce the accumulation of amyloid deposits in peripheral nerves, reduce the severity of symptoms and help patients better control the disease.

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