Gene therapy research is coming into its own lately. Gene replacement and gene editing technology treatments for rare diseases, caused by errors in a single gene, have been making landmark strides in laboratories. Following suit, pharmaceutical companies are increasingly investing time and money (some $10 billion in 2015, according to The Alliance for Regenerative Medicine) in gene therapy research.
Gene therapies typically involve one-time treatments that allow “fixed” genes to reproduce in the body. Certainly an improvement over repeated trips to doctors’ offices, long-term dependence on medication, or even highly invasive treatments such as bone marrow transplants. The road to commercialization has been wrought with obstacles, though, so many are just cautiously optimistic of the renewed excitement around gene therapy.
There are currently hundreds of gene therapies in clinical development, with about 70 in late-stage trials. One treatment about to launch is GlaxoSmithKline’s drug Strimvelis (licensed in 2010), which treats the rare condition severe combined immune deficiency, also known as “bubble boy” disease. It looks as though Strimvelis will soon be approved in Europe, where it will be made available in 27 countries. GlaxoSmithKline will push for US approval in 2017.
These treatments can be quite expensive and, if they’re targeting rare diseases, have limited earning potential. For example, the only gene therapy currently approved in the West is UniQuire’s Glybera, which treats the ultrarare lipoprotein lipase deficiency and was approved in Europe back in 2012. However, many consider Glybera a flop. It has been used exactly one time (the treatment worked), at the whopping cost of $1 million. The approval process to get the insurance company to cover that cost proved quite complex. And due to the great expense of conducting additional trials, UniQuire has scrapped plans to introduce the drug to the US. Its CEO, Dan Soland, stated that Glybera “still drains a lot from the company.”
The gene editing technology CRISPR (which stands for clustered regularly interspaced short palindromic repeats) has been in the news as scientists battle for bragging, patent, and commercial rights. Several companies are engaged in CRISPR, or other genetic technology research and development. Novartis-backed Intellia, which raised $108 million in an IPO yesterday, is pursuing gene editing treatments for transthyretin amyloidosis, a rare liver disease. Another biotech, Editas, has a program in the works to treat Leber congenital amaurosis, a rare form of blindness. French firm Cellectis is advancing its proprietary TALEN treatment for acute lymphoblastic leukemia, a previously incurable form of the disease. And Sangamo Biosciences has the world’s most advanced (non-CRISPR) gene editing program, a phase 2 treatment targeting HIV infection. It also has a hemophilia B therapy in the works.
To improve the chances for profitability, and even for finding the next blockbuster drug, pharmaceuticals are also developing gene therapies that treat more common diseases, including cancer and sickle cell disease. For example, Novartis holds the rights to Intellia’s CRISPR technology to develop cancer and blood disorder treatments. By targeting more common ailments, and making money in the process, these firms will establish a foundation in gene therapy that can then be further developed to treat some of the world’s more esoteric diseases.
Diane Ramirez has been a member of the D&B editorial department for more than a decade. She currently covers the health care and insurance industries for Hoover’s.