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Bluebird has another rare disease in its near-term sights: adrenoleukodystrophy, better known as the disease featured in the movie Lorenzo’s Oil . In the disorder a malfunction of the gates that transport fats into and out of cells causes buildups of fatty acids throughout the body, including in the brain. Instead of injecting the virus into the body, in Bluebird’s approach cells (in this case, bone marrow stem cells) are removed and treated with another virus, lentivirus. Unlike AAV, lentivirus inserts the genes permanently into the cell’s DNA-containing chromosomes. These stem cells are reinjected; Bluebird is running a phase III trial in 15 patients and aims to get its treatment on the market in the next several years.
These treatments won’t be cheap. The first gene therapy ever to clear a regulator was approved in Europe in 2012 by the Nasdaq-listed firm uniQure. Reports pegged the price at $1 million; CEO Joern Aldag disputes that any price has yet been set. The treatment is not yet on the market. But that kind of pricing power–beyond even the norm of $300,000 per patient per year and up for rare-disease drugs–is enticing more and more companies to enter the field.
One of the hottest areas is already one of the biggest markets for expensive biotech drugs: hemophilia A and B, the genetic diseases that cause the blood not to clot properly, potentially leading patients to bleed to death.
“Gene therapy for hemophilia A possibly could be our biggest product,” says Jean-Jacques Bienaime, chief executive of rare-disease specialist Biomarin, which forecasts revenue of at least $650 million this year.
Bienaime says his company estimates there are 50,000 type A hemophiliacs in territories where it markets drugs and who are receiving hemophilia medicine; that would be his addressable market. A company called Dimension Therapeutics is competing with Biomarin; Spark, Dimension and hemophilia expert Baxter International BAX +1.55% are chasing hemophilia B.
The promise of the technology has some venture capitalists pushing far beyond rare genetic diseases. San Diego’s Celladon, which raised $51 million in an IPO earlier this year, is aiming to treat heart diseases like advanced heart failure, in which the heart muscle becomes too weak to function.
Mark Levin, the former chief executive of Millennium Pharmaceuticals and founder of Third Rock Ventures, is now serving as interim chief executive of a company named Voyager Therapeutics, which aims to treat Parkinson’s disease. Patients now get a drug to raise dopamine levels, but it eventually stops working; Voyager’s gene therapy would cause the brain itself to produce more dopamine.
“It is really exciting to see how rapidly science and medicine are changing to make a big difference for patients,” Levin says.
The viruses used in gene therapy have also joined the war on cancer. Novartis NVS -0.02%, Bluebird and Juno Therapeutics, which raised $120 million in venture capital last year, are all working on cells genetically engineered to hunt down tumors. Early results in blood cancer show dramatic reductions in the number of cancer cells in patients who had few other options.
And biotech researchers are dreaming of doing far more. Even viruses like lentivirus, which write DNA directly into a cell’s chromosome, can make only limited additions to the patient’s genes. They’re like adding an ingredient to life’s recipe book. But what if you need to change the recipe?
Recently, researchers have been working with a series of proteins, originally found in the immune systems of bacteria, that allow them to cut DNA in specific places and splice it back together. This technology, called CRISPR/Cas9, might be deliverable to cells using gene therapy. Editas Medicine was launched in November with $43 million in capital by Flagship Ventures, Polaris Partners and Third Rock to turn that dream into a reality.
Still, laboratory success doesn’t ensure financial success. “We may be taking a really simplistic view that if you’re able to come close to treating grievous illness, the pricing and getting paid and developing a valid business model will take care of itself,” says Kevin Bitterman of Polaris, who is serving as Editas’ interim president. “The goal very simply is to leverage this technology to develop a new class of therapeutics against genetic disorders.” Eager investors, take note.