Gene therapy is the process of inserting genes into human cells and tissues for the treatment of diseases, including hereditary diseases. The first gene therapy procedure was performed in 1990 on a four-year old girl who was born with severe combined immunodeficiency, a rare genetic disease, also known as 'bubble boy' disease or SCID.
Gene therapy can also involve the use of stem cells. In 1993, a baby was tested with genetic screening before birth which showed that he had SCID. Cord Blood was removed from the baby boy's placenta and umbilical cord immediately after he was born and the stem cells were extracted, mixed with retroviruses, and injected with working ADA genes into the boy's blood stream. The boy's T-cells made their own ADA enzymes for a period of four years after the treatment.
The saving and storing of cord blood has become very popular recently, and is a growing industry. Even Richard Branson, founder and head of Virgin Records and Virgin Atlantic Airways, is jumping on the bandwagon, by setting up a cord blood bank.
The following are a selection of some of the larger, publicly traded companies involved in a significant way in gene therapy. All of the following stocks have market capitalizations above $300 million.
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) develops therapeutic products based on RNA interference. It is working on therapeutics for the treatment of respiratory syncytial virus infection, hypercholesterolemia, liver cancer, Huntington's disease, Ebola virus viral hemorrhagic fever, progressive multifocal leukoencephalopathy, pandemic flu, Parkinson's disease, and cystic fibrosis. The stock has a market cap of $1.13 billion. Its latest reported earnings were negative.
Cell Genesys' (CEGE) is a biotechnology company that develops cell-based cancer immunotherapies and oncolytic virus therapies. Its therapies are used to treat prostate cancer, pancreatic cancer, acute and chronic myelogenous leukemia, myelodysplastic syndrome, and recurrent bladder cancer. The stock has a market cap of $307 million. Its latest reported earnings were negative.
Enzo Biochem Inc. (NYSE:ENZ) makes and sells biomedical research products and tools used in the fields of gene expression, non-radioactive labeling and detection, apoptosis, cell cycle, cytoskeletal research, DNA damage and repair, immunology and cancer research. The stock has a market cap of $336 million. Its latest reported earnings were negative.
Genzyme (GENZ) is a biotechnology company involved in the products and therapies relating to renal, therapeutics, transplants, biosurgery, and genetics. It is working on a first phase 2 gene therapy trial, which investigates the proprietary HIF-1-alpha gene for the treatment of peripheral arterial disease, and has an ongoing phase 1-2 gene therapy trial for Parkinson's disease. The company is also working in gene therapy for age-related macular degeneration and lysosomal storage disorders. The stock has a market cap of $18.5 billion. The stock has a P/E of 41, and a PEG of 0.94.
Isis Pharmaceuticals (ISIS) is a biopharmaceutical company involved in RNA-based drugs. The company has 18 drugs in development, to treat familial hypercholesterolemia patients, type 2 diabetes, kidney glucose issues, advanced prostate cancer, advanced lung cancers, tumor progression, multiple sclerosis, eye diseases, and respiratory diseases. The stock has a market cap of $1.09 billion. Its latest reported earnings were negative.
Onyx Pharmaceuticals (NASDAQ:ONXX) develops and markets oral anticancer therapies used to prevent cancer cell proliferation and angiogenesis through the process of inhibiting proteins that signal or support tumor growth. The stock has a market cap of $1.93 billion. The stock has a forward P/E of 26, and a PEG of 4.47.
Disclosure: The author does not own any of the above.