Volume 93 Issue 48 | p. 16
Issue Date: December 7, 2015

Cover Stories: 2015 Pharma Year In Review

2015 Saw Bets Placed On CRISPR/Cas9

Investments rose as technology adoption gained speed
Department: Business
Keywords: gene editing, genetic disease, genomics, gene therapy, patents

In 2015, more than $420 million went into funding four inventor-backed start-up companies exploiting CRISPR/Cas9 genome-editing methods. The investments came despite legal and ethical uncertainty around use of the breakthrough technology.

In April, the University of California system challenged a foundational patent awarded a year earlier to Feng Zhang of the Broad Institute of MIT & Harvard. UC asked for a U.S. Patent & Trademark Office interference review because of competing claims from UC Berkeley’s Jennifer Doudna and her European collaborator Emmanuelle Charpentier.

Meanwhile, because the gene-editing approach is simple, efficient, and accessible to most scientists, its use in R&D exploded during the year. Commercial reagents became more available, as did engineered cell lines and new animal models of disease. Work began on therapeutic applications.

Amid the R&D rush, Chinese scientists reported that they had tried to alter a disease-related gene in nonviable human embryos (Protein Cell 2015, DOI: 10.1007/s13238-015-0153-5). Scientific, legal, government, and other communities reacted quickly to consider the risks and ethical implications of editing human genes.

But the business community wasn’t deterred. In April, Caribou Biosciences, founded by Doudna and colleagues, completed an initial $11 million financing round in which Novartis participated. Later, DuPont cross-licensed technology with Caribou and took an equity position.

[+]Enlarge
GENOME EDITOR
The Cas9 enzyme (white) and guide RNA (green-yellow) form a complex with target DNA (red) and position themselves for gene editing. Blue spheres represent magnesium ions essential for the cutting of the DNA.
Credit: Ian Slaymaker and Lauren Solomon/Courtesy of the Broad Institute
Structure of the Cas9 complex depicts the Cas9 enzyme, along with guide RNA, target DNA, and the positions of magnesium ions essential for cutting the DNA.
 
GENOME EDITOR
The Cas9 enzyme (white) and guide RNA (green-yellow) form a complex with target DNA (red) and position themselves for gene editing. Blue spheres represent magnesium ions essential for the cutting of the DNA.
Credit: Ian Slaymaker and Lauren Solomon/Courtesy of the Broad Institute

Likewise, Intellia Therapeutics, a 2014 spin-off of Caribou, closed a $70 million second funding round in September. Prior to that, the company had signed a five-year deal with Novartis to develop ex vivo CRISPR/Cas9 therapies using chimeric antigen receptor T cells (CARTs) and hematopoietic stem cells.

In May, Editas Medicine, founded in 2013 by Zhang and others from Massachusetts Institute of Technology and Harvard University, formed an alliance with Juno Therapeutics to create anticancer CART therapies. Juno agreed to pay $25 million up front and up to $22 million on research. For each of three programs, Editas could receive milestone payments of at least $230 million. In August, Editas raised $120 million in its second financing round.

Crispr Therapeutics, which was set up last year around Charpentier’s intellectual property, managed to attract $89 million in 2015. Investors included Celgene and GlaxoSmithKline’s venture capital arm, SR One. In October, Crispr signed an R&D deal with Vertex Pharmaceuticals for $105 million up front and up to $420 million for each of six programs.

If events play out smoothly, initial CRISPR/Cas9 cell therapies are predicted to reach the clinic in 2016. Just last week, however, the National Academy of Sciences and the National Academy of Medicine’s Human Gene-Editing Initiative, along with the Chinese Academy of Sciences and the U.K.’s Royal Society, hosted a summit to discuss issues around human gene-editing research.

 
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