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Gene therapy group gears up for education campaign as CRISPR anxiety grows

A recent study linking gene-editing tool CRISPR to cancer comes just as industry groups ramp up efforts to reach consumers and physicians.

Two separate studies published in the medical journal Nature Medicine on June 11 suggested that cells edited by CRISPR-Cas9 — a technology meant to target and change precise sections of DNA strands — could begin growing tumors post-CRISPR.

Although the three companies leading the CRISPR charge — CRISPR Therapeutics AG, Intellia Therapeutics Inc. and Editas Medicine Inc. — say their approaches are different from the two studies' techniques, each saw its stock tumble June 11.

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CRISPR-Cas9 and other genome-editing technologies represent a transformative area of medical advances, the industry lobby group Alliance for Regenerative Medicine said in a statement to S&P Global Market Intelligence that was attributed to CEO Janet Lambert.

"ARM member companies developing these therapies understand the enormous responsibility that comes with making edits to a patient's DNA, and are pursuing the necessary, and very scientifically rigorous, research required to ensure these products are safe and effective for in-human clinical studies, including addressing the concerns these studies highlighted," Lambert said.

News of potential CRISPR complications come as the Alliance's newest project, the ARM Foundation, launches with planned education and outreach programs for patients and physicians.

The foundation is "affiliated with, but independent from ARM," its board chair, Stewart Parker, said in an interview on the sidelines of the BIO International Convention in Boston last week.

Parker is also board chair of gene therapy company Sangamo Therapeutics Inc. and the former CEO of Targeted Genetics, a Seattle biotech that struggled to recover after a patient died in a 2007 trial despite the U.S. Food and Drug Administration eventually determining that the company's gene therapy did not cause the death. Targeted Genetics is now known as AmpliPhi Biosciences Corp.

"It's really important for me personally that this field be successful, and yet I sense that the gap really needs to be filled in terms of education," Parker said.

While still in its early days, the foundation's plans also include an economic impact analysis and, eventually, an assessment of the type of infrastructure needed to distribute and administer complex medicines such as chimeric antigen receptor T-cell, or CAR-T cell, therapies that involve infusing patients with lab-enhanced versions of their own cells.

But first, ARM's focus is on educating the market. Between negative press such as a man self-administering CRISPR cells and later dying of unrelated causes, to a string of recent approvals — including Novartis AG's Kymriah and Gilead Sciences Inc.'s Yescarta, both CAR-T cell therapies, as well as Spark Therapeutics Inc.'s blindness therapy, Luxturna — "there's a sense of urgency that we have to get our job done," Parker said.

'The field is early'

"We think of CAR-Ts as a wonderful demonstration of what's possible if you engineer T-cells, but we think that T-cells can be better engineered if you use a T-cell receptor approach," John Leonard, Intellia Therapeutics CEO, said in an interview at BIO.

Intellia is one of three companies furthest along in CRISPR development, joined by Editas and CRISPR Therapeutics. While none are in human trials just yet, CRISPR Therapeutics was close — until the U.S. Food and Drug Administration put its new drug application for sickle cell disease, partnered with Vertex Pharmaceuticals Inc., on clinical hold to resolve certain questions.

The pair said they will continue with planned European trials in the second half of 2018. CRISPR Therapeutics' shares dipped after the May 30 news, and all three companies saw sharp drops after the June 11 Nature Medicine studies.

"There's this general reactivity where any time a paper comes out, there is a rush to do something within moments. My favorite example is a paper that was recently retracted," Leonard said at BIO, referring to a 2017 study, retracted this March, that concluded that CRISPR had significant off-target effects after assessing it in three mice.

"The field is early. Until we're in patients, which means you've gone through certain steps … people will react the way they do," Leonard said.

This interview with Leonard took place before the Nature Medicine studies were published. Reached for comment on the latest reports, Jennifer Smoter, Intellia's senior vice president for external affairs and communications, said in a statement that the company has observed no signs of this type of toxicity or cell transformation in any of its lab or animal studies.

"Despite extended observation in animals and in [lab cultures], we have not seen this effect. Intellia's current approaches are directed at different cell types," she said. "Developing safe and effective new medicines is a priority for Intellia, and we continuously study and monitor potential adverse effects."