- The chairman of the largest biotech trade group voiced concern over U.S. leaders' understanding of science, as well as the potential for politicians and the public to take a detrimental, knee-jerk reaction to the controversy over gene-edited babies that erupted in late November.
- John Maraganore, chair of the Biotechnology Innovation Organization, expressed worry in an interview with BioPharma Dive over how "uninformed leaders of our country" will react to news of a Chinese scientist claiming he genetically edited the embryos of two babies born last month.
- Furthermore, Maraganore said he's broadly observed a "concerning sentiment from our country's leadership" on science. "When you see the country pulling out of the Paris Accord, when you see the country [and] policymakers wondering whether vaccines are good or bad, that's a very, very dangerous situation," he said. "And one that I personally — I'm talking for myself — reject as an acceptable position by leaders of our country."
The BIO chair and CEO of Alnylam Pharmaceuticals took on skeptics of vaccines and climate change, concepts that have well-established and broad support within the scientific community but habitually find doubters in the broader public and the Trump administration.
These questions were brought into the national spotlight again Tuesday, when Mark Green, a newly elected member of Congress, expressed skepticism on the value of vaccines at a town hall, referencing unsubstantiated and debunked claims of a link with autism.
"I have committed to people in my community, up in Montgomery County, to stand on the CDC's desk and get the real data on vaccines," said the Tennessee Republican who is also an emergency physician. "Because there is some concern that the rise in autism is the result of the preservatives that are in our vaccines."
Green is the latest example of skepticism from U.S. leaders, which, coupled with doubts over climate science, has put the scientific community on the defensive. President Trump himself has met with vaccine doubters and pulled the U.S. out of the Paris climate agreement.
The BIO chair did not mention any official by name but issued a rebuke of this nation's uninformed leaders.
"There's no doubt that the science around climate change, which is embraced by almost everybody, the science around vaccines, which is incontrovertible, and the attack on that science is just not good," Maraganore said. "It's concerning. Hopefully, it's short-lived in many ways and we can go back to being a country that focuses on the right scientific-based decisions for our society."
The other concern stemmed from reports of a scientist claiming to have genetically modified the embryos of two children born last month in China. While doubts remain over the validity of He Jiankui's work, his claims have captured the world's attention and reverberated throughout a research field in the midst of major advances.
"I would call that a rogue activity by a scientist who is off the reservation," Maraganore said, "who should not be doing that at this point in time and has been publicly questioned in terms of what he's done, and I think appropriately questioned for what he's done."
In reaction, BIO, as well as leaders of the Food and Drug Administration and National Institutes of Health, have all expressed disapproval over that use of CRISPR-based gene editing.
Food and Drug Administration head Scott Gottlieb warned of "potential regulations and laws that could be far more restrictive than they might otherwise be if there were more confidence that the community was able to self-impose appropriate standards" in an interview with BioCentury.
The CRISPR case in China may be isolated, but it isn't the first example of concerns over gene-based therapeutic approaches.
In 1999, 17-year-old Jesse Gelsinger died in a clinical trial for gene therapy at the University of Pennsylvania, resulting in new regulations and delays on similar studies. And in 2003, news of a few children developing leukemia from a gene therapy trials led to the FDA halting 27 studies as a precaution.
Now, the consequences of He Jiankui's claims are still being assessed. Whether or not He's claims are validated, the public debate playing out in the media and among the scientific community carries the power to shape public attitudes and understanding of such technology.
For biotech, questions now turn to whether worries will translate into policy actions that could affect the momentum of gene editing research in the U.S., where Congress banned human germline editing in 2016.
As a field, gene therapy is one of biotech's hottest. Dozens of companies are exploring research into gene replacement as well as gene editing like CRISPR. And last year, the FDA approved the first gene therapy for an inherited disease, opening a new chapter for the sector.
Research, however, has focused on using genetic tools to treat or even cure disease in children and adults, rather than changing germline DNA before a baby is born.
"It's an act like that that can cause a knee-jerk reaction by the uninformed public and by the uninformed leaders of our country to do something which can be very harmful for future innovation," Maraganore said. "I do worry about that as well."