Gene-editing techniques are on the cusp of transitioning from science fiction to reality.
Bayer, the European pharmaceutical giant, is establishing a partnership with a gene-editing startup, CRISPR Therapeutics, and investing at least $300 million over a five-year period, the companies announced earlier today. Bayer will also take a $35 million stake in the startup.
The goal is to develop new drugs for a range of ailments, including hemophilia, heart disease in infants, and a degenerative disease that causes blindness called Stargardt.
The CRISPR technique has been hailed in the press as the next great innovation in biotech. Scientists have struggled for decades to reliably edit genomes (viruses do it regularly, but randomly). The problem is that if you fix one aspect of the genome, you run the risk of making a lot of unintentional mistakes. CRISPR essentially offers a better tool for scientists to precisely control how they edit a genome.
Experts say that joint ventures like these bring much-needed validation to the still nascent field of gene editing. “It signals that big pharma sees this as a legitimate potential way to develop new drugs,” says Sabah Oney, a geneticist and biotech executive. In January of this year, drug maker Novartis secured the first of the CRISPR-pharma collaborations.
Oney predicts that many of the other major drug makers will follow suit through partnerships and further investment. “I expect all of the CRISPR startups to partner with pharma for many different disorders,” he says.
But Oney warns that CRISPR is still a ways off from becoming mainstream. “CRISPR is still far from clinics,” he said, and it still faces myriad scientific, regulatory, and legal hurdles.