All eyes were on Valeant Pharmaceuticals (VRX) Monday morning, but another ‘V’ name deserves credit for enabling some real science. Vertex Pharmaceuticals (VRTX) on Monday climbed aboard the gene editing bandwagon with a $105 million commitment to CRISPR Therapeutics, one of a few recent startups employing the lauded gene editing technology, CRISPR-Cas9.
Vertex is doubling down on its commitment to cystic fibrosis, with the initial focus of the collaboration on the use of CRISPR-Cas9 to correct the mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that produce the defective protein that causes CF. Vertex already sells Kaleydeco and Orkambi for subsets of this population and has follow-ons in the pipeline that they believe will address 90% of the CF population; the CRISPR research is a logical extension. The two will also begin work on gene-based treatments for hemoglobinopathies like sickle cell disease, where bluebird bio (BLUE) has made noise with its lentivirus-driven gene therapy. Vertex also secures a seat on CRISPR’s board.
With a $75 million cash payment and $30 million equity investment, Vertex secures exclusive rights to license up to six CRISPR-Cas9-based treatments that emerge from the collaboration, with CRISPR eligible to development, regulatory and sales milestones from any success, up to $420 million per program. Vertex joins as an equity holder GlaxoSmithKline’s (GSK) venture arm, SR One, and Celgene (CELG), who put $64 million into CRISPR this April, plus New Enterprise Associates, Abingworth and Versant Ventures.
Who exactly owns the rights to this new, cheap gene-editing technology is still in limbo. and will remain so for the near future. CRISPR’s technology comes directly from Emmanuelle Charpentier, who previously worked with gene therapy rival, Editas, co-founded by Jennifer Doudna. Editas was founded in 2013 with $43 million from Polaris, Third Rock and Flagship Ventures; and Juno Therapeutics (JUNO) handed over $25 million of support in May. Doudna ultimately split from Editas and founded Caribou Bio, which out-licensed its technology to Intellia, launched by Atlas Ventures and Novartis (NVS).
The intellectual property dispute won’t be settled for a long, long time, and all three firms are years from having anything in human testing. But after a decade of moving its current CF franchise through the clinic and finally onto the market, Vertex’s latest research collaboration is, perhaps, the most exciting new endeavor in some time.
