Everyone at the Napa meeting had access to a gene-editing technique called Crispr-Cas9. The first term is an acronym for “clustered regularly interspaced short palindromic repeats,” a description of the genetic basis of the method; Cas9 is the name of a protein that makes it work. Technical details aside, Crispr-Cas9 makes it easy, cheap, and fast to move genes around—any genes, in any living thing, from bacteria to people. ... Using the three-year-old technique, researchers have already reversed mutations that cause blindness, stopped cancer cells from multiplying, and made cells impervious to the virus that causes AIDS. Agronomists have rendered wheat invulnerable to killer fungi like powdery mildew, hinting at engineered staple crops that can feed a population of 9 billion on an ever-warmer planet. Bioengineers have used Crispr to alter the DNA of yeast so that it consumes plant matter and excretes ethanol, promising an end to reliance on petrochemicals. Startups devoted to Crispr have launched. International pharmaceutical and agricultural companies have spun up Crispr R&D. Two of the most powerful universities in the US are engaged in a vicious war over the basic patent. Depending on what kind of person you are, Crispr makes you see a gleaming world of the future, a Nobel medallion, or dollar signs. ... It brings with it all-new rules for the practice of research in the life sciences. But no one knows what the rules are—or who will be the first to break them. ... As it happened, the people who found it weren't genome engineers at all. They were basic researchers, trying to unravel the origin of life by sequencing the genomes of ancient bacteria and microbes called Archaea (as in archaic), descendants of the first life on Earth. Deep amid the bases, the As, Ts, Gs, and Cs that made up those DNA sequences, microbiologists noticed recurring segments that were the same back to front and front to back—palindromes. The researchers didn't know what these segments did, but they knew they were weird. In a branding exercise only scientists could love, they named these clusters of repeating palindromes Crispr. ... Pick your creature, pick your gene, and you can bet someone somewhere is giving it a go.
The DNDi is an unlikely success story in the expensive, challenging field of drug development. In just over a decade, the group has earned approval for six treatments, tackling sleeping sickness, malaria, Chagas' disease and a form of leishmaniasis called kala-azar. And it has put another 26 drugs into development. It has done this with US$290 million — about one-quarter of what a typical pharmaceutical company would spend to develop just one drug. The model for its success is the product development partnership (PDP), a style of non-profit organization that became popular in the early 2000s. PDPs keep costs down through collaboration — with universities, governments and the pharmaceutical industry. And because the diseases they target typically affect the world's poorest people, and so are neglected by for-profit companies, the DNDi and groups like it face little competitive pressure. They also have lower hurdles to prove that their drugs vastly improve lives. ... Now, policymakers are beginning to wonder whether their methods might work more broadly. ... If successful, the work could challenge standard assumptions about drug development, and potentially rein in the runaway price of medications.