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    CRISPR Therapeutics CEO discounts concerns raised in an unpublished paper

    SAN FRANCISCO — The new head of a genome editing company whose main research lab is in Cambridge on Tuesday brushed aside an unpublished paper that raised concerns about preexisting immune responses to the biotech’s gene-editing technology, known as CRISPR.

    Samarth Kulkarni, who last month rose from chief business development officer of the Swiss company CRISPR Therapeutics to chief executive, acknowledged that the paper, posted Friday on the bioRxiv website, presented data that caused the stocks of his company and two Cambridge rivals to tumble. But, Kulkarni said, the findings had already been presented at conferences six to eight months ago and were irrelevant to the science.

    “The data are not new,” he told the Globe in a hotel conference room during the J.P. Morgan Healthcare Conference, the biggest annual investors event in biotechnology. “What’s new is that someone in the press picked it up and sensationalized it, and all of a sudden everyone else picked it up.”


    Kulkarni said the research paper will not delay CRISPR Therapeutics, which has almost all of its 120 employees in Cambridge, from beginning tests of a gene-editing therapy in clinical trials this year.

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    The company applied late last year to begin trials in Europe on patients with beta thalassemia, a rare inherited blood disorder. It is asking the Food and Drug Administration to let it test the same treatment in the United States this year on patients with sickle cell disease, another rare blood disorder.

    Boston-based Vertex Pharmaceuticals is working with CRISPR to develop and market the treatment.

    Kulkarni said CRISPR “has the pole position” and would be the first to begin clinical trials of gene editing in patients. It was the last of three gene-editing startups — the others are Editas Medicine and Intellia Therapeutics — to go public.

    The three companies are among several parties in a patent fight that is currently in the US Court of Appeals for the Federal Circuit. Gene editing has attracted major investment because of its potential to cure deadly intractable diseases.


    The bioRxiv paper’s senior author, Dr. Matthew Porteus of Stanford University, recently told the online health and life sciences publication STAT — launched in 2015 by Boston Globe owner John Henry — that the findings on immune responses shouldn’t stop development of CRISPR therapies. But Porteus said the field needs to address possible problems as the first clinical trials get closer. If the immune system did attack proteins used in a CRISPR therapy, he said, it could make the treatment useless, or even provoke a dangerous inflammatory attack in the patient. Porteus is a scientific founder of CRISPR Therapeutics.

    Kulkarni said any problems with the immune response would be fleeting and have no impact on patients’ health.

    Jonathan Saltzman can be reached at