The gene-editing technique known as CRISPR was declared safe and feasible after a trial in cancer patients, potentially opening the door for the controversial tool’s wider use in fighting disease and improving health.

The clinical trial, with just three patients, was designed simply to determine the safety of the technique, not to cure the disease. While the scientists conducting the study cautioned it’s too early to declare success, they did say in a paper published in Science the technology “is possible at clinical scale” and that the initial results showed it was safe.

“Now we can move on to a whole new frontier of further engineering these cells and expanding the number of patients treated,” Edward A. Stadtmauer, an oncologist at the University of Pennsylvania and the study’s principal investigator, told Wired.

The results likely will add to the debate over whether the gene-altering tool should be banned or regulated. Some critics say they fear CRISPR will be used to create designer babies and others concerned about its use in modifying crops and livestock, which may have unintended impacts on the environment. Supporters of CRISPR research say it has the potential to prevent inherited diseases such as hemophilia and cystic fibrosis by altering the defective genes. The tool also may be effective against acquired conditions including AIDS and cancer.

“Two things worry me,” said bioethicist Hank Greely, a professor of law at Stanford University. “One is the intentional misuse of CRISPR. The other is that people with good intentions will inadvertently cause harm.”

But for treating diseases like sickle cell, “I think CRISPR will be transformative,” he told the Stanford Medicine magazine, “and that’s a great thing.”

 In December, a scientist and two associates in China were sentenced to three years in prison after using CRISPR to create the first genetically edited babies. When news broke of their action, there was an outcry among some researchers who called it “unconscionable” and “immoral and ethical.”

Private investors’ interests in startups focusing on gene-editing have surged in the last three years. Venture capitalists spent a record $809 million in the filed last year, a 19% jump from 2018 and more than five-fold of 2016, according to PitchBook data. 

Poseida Therapeutics, after forgoing an IPO a year ago, landed a $142 million series C investment led by Novartis Pharma last April, the largest amount raised in the field in 2019. The most active investors in the gene-editing field are Alexandria Venture Investments from Pasadena, California and Chicago-based ARCH Venture Partners, which invested seven deals each in the past five years.

  • In the cancer trial, three people received infusions of their own T cells, which had been genetically modified with enhanced cancer-recognizing receptors. The cells didn’t trigger any immune response and generated only mild adverse effects, and the patients’ health held steady or improved. The trial was conducted by researchers from the University of Pennsylvania and Stanford.
  • CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.