|Report Code: PP10184||Report Type: Molecule-Type Pipeline Reports||Available format:|
Gene therapy is a technique that involves treatment or prevention of a disease by using genes. Several methodologies are being used in the gene therapy that involve replacing the mutated gene, inactivating or silencing the mutated gene, and introducing a new gene into the body. This results in the formation of beneficial protein. A gene cannot be directly delivered into the human body, because of the presence of certain nuclease enzymes that act upon the foreign nucleic acids. Some of the family of viruses that are being used as a vector for the administration of the desired gene are retrovirus and adenovirus. These vectors are genetically modified and do not harm the host. The National Institute of Health plays a prominent role in confirming the safety of gene therapy. The U.S. Food and Drug Administration (USFDA) approved first cell-based gene therapy manufactured by Novartis AG, named Kymriah, which is a CAR T cell therapy for the treatment of B-cell precursor acute lymphoblastic leukemia in patients up to 25 years of age, and B-cell lymphoma.
uniQure N.V. is also developing a wide range of innovative gene therapies for the treatment of liver-directed diseases and neurological disorders. Bioverativ Therapeutics Inc. is developing a gene therapy for the treatment of hemophilia A and hemophilia B. GlaxoSmithKline plc is also developing strimvelis as a gene therapy for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID), metachromatic leukodystrophy (MLD), Wiskott Aldrich syndrome (WAS), and beta thalassaemia. Some of the companies with gene therapy products in their pipeline are Sangamo Therapeutics Inc. and Spark Therapeutics Inc. among others.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.