Regulatory
What's an 'off-target' genome editing?
If there's one outstanding concern about what would be the first FDA-approved CRISPR medicine, it's the imprecision of the revolutionary genome-editing technology.
At yesterday's public hearing on a therapy from Vertex Pharmaceuticals and CRISPR Therapeutics, the potential for off-target editing — in which the wrong bit of DNA gets snipped — emerged as a real, if theoretical, concern for the FDA's independent advisers.
The therapy, called exa-cel, is designed to home in on the genetic defects that cause sickle cell disease and leave the rest of patients' genomes untouched. According to Vertex, the company used cutting-edge methods to search for potential off-target edits and found none, affirming exa-cel's safety. But FDA staff took issue with the scope of Vertex's analysis, and the agency's expert advisers said more work is needed to understand the risk of errant editing.
None of this is likely to derail the approval of exa-cel, expected by Dec. 8, but it sets the tone for long-term discussions of CRISPR-based medicines and the regulatory vigilance they're going to elicit.
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Markets
Biotech volatility cuts both ways
Some time before the end of the year, Cytokinetics will have pivotal data on its investigational cardiovascular treatment, results that could double — or halve — its stock price. Because it's such a binary proposition, the likelihood anyone would buy the company before knowing the results of that study would seem negligible.
And yet. Yesterday, Bloomberg reported that Cytokinetics has attracted buyout interest from at least one major drugmaker in recent months, noting that no deal is imminent and that any suitor would probably want to see some data before spending billions of dollars. The news sent Cytokinetics' stock price up as much as 15% before triggering a volatility halt on trading. The company ended the day up about 7%.
The reaction reads like yet another symptom of biotech's protracted slump. Just as declining sentiment has made bad news go over that much worse on the market, investors' desire for any and all good news might explain Cytokinetics' volatile day on Wall Street. After all, regardless of who approached whom about what, the company's investment thesis — it needs its drug to work for shareholders to make money — hasn't changed at all.
Policy
Gene therapy doesn't have to be so expensive
The dawn of gene therapy has given rise to a vexing problem. Companies invent life-changing medicines and argue that they're worth upward of $1 million, pointing to their long-term value and the expense of developing them. Payers and governments, operating with finite budgets, point out that $1 million is quite a lot of dollars, and restrict patient access. And patients, the people whose lives stand to be changed, either struggle to get treated or lose out entirely.
It doesn't have to be this way, writes Vivian Cheung, a physician-scientist at the University of Michigan who is also a patient with a rare disease. To date, the government has taken action to make medicines more affordable, but that can only address a symptom of the problem rather than its root cause. In addition, the U.S. should mount an aggressive federal effort to invest in a shared biomedical infrastructure that would make the process of developing gene therapies faster, more efficient, and cheaper, Cheung writes.
"Engineers may have invented the internal combustion engine, and Henry Ford may have debuted the production line," she writes. "But cars would have never become so commonplace had private auto companies been expected to build the national highway system."
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