Brainstorm Health: Gene Edited Babies, Medical Device Overhaul, CVS…

Happy Monday, readers. I hope you had a wonderful Thanksgiving break. Let’s get back into it.

A shock claim from a Chinese researcher saying that the world’s first known gene edited babies have been born is stirring major controversy among the life sciences community, drawing criticism from one of the pioneering minds behind CRISPR gene-editing tech itself and China’s Southern University of Science and Technology, according to the MIT Technology Review.

To be clear, the claim by Jiankui He has not been verified. But if it is, in fact, true, it would be a simultaneous milestone for genomic technology and a divisive ethical flashpoint in medicine.

He says that twin baby girls were born following embryonic gene modification meant to make them immune to HIV transmission. The scientist also claimed that only the intended genes were modified with no other unexpected editing.

A wide range of life sciences leaders and academic institutions issued stern responses to the reports, emphasizing that the claims had to be investigated and the research team ostensibly behind the development must explain themselves.

“On November 26, 2018, the Southern University of Science and Technology (hereafter SUSTech or the University) was informed through media news reports that Dr. Jiankui HE (who has been on no-paid leave from February 2018 through January 2021) released a public announcement that he has carried out genetic editing on human embryos,” wrote the organization in a statement. “The University was deeply shocked by this event and has taken immediate action to reach Dr. Jiankui HE for clarification. Dr. Jiankui HE’s previous affiliation, the Department of Biology (hereafter the Department) called an emergency meeting of the Department Academic Committee.”

Texas’ Rice University issued its own missive as several of the reports implicated that one of its own professors, Michael Deem, was involved in some shape or form.

“Recent press reports describe a case of genomic editing of human embryos in China. These reports include a description of involvement by Dr. Michael Deem, a professor of bioengineering at Rice University. This research raises troubling scientific, legal and ethical questions…We have begun a full investigation of Dr. Deem’s involvement in this research,” wrote the university.

Feng Zhang of the Broad Institute of MIT and Harvard, one of the trio of scientists credited for pioneering CRISPR gene editing in the first place, added that, “Not only do I see this as risky, but I am also deeply concerned about the lack of transparency surrounding this trial.”

This story isn’t going away anytime soon. Stay tuned as the dust settles.

Read on for the day’s news.

Sy Mukherjee

@the_sy_guy

[email protected]

DIGITAL HEALTH

FDA to overhaul medical device approval process. The Food and Drug Administration (FDA) on Monday expounded on its plans to revamp the the regulatory pathways used to approve medical devices. FDA Commissioner Scott Gottlieb issued an extensive statement on several potential proposals that could enhance the safety of such products as digital health and other technologies take flight. "We believe that newer devices should be compared to the benefits and risks of more modern technology; that is why we’re looking at ways to promote the use of more recent predicates," he wrote. "To advance these goals, in the next few months CDRH is considering making public on its website those cleared devices that demonstrated substantial equivalence to older predicate devices. We’re considering focusing on predicates that are more than 10 years old as a starting point, so that the public is aware of those technologies." (Coins2Day)

INDICATIONS

Zafgen stock plunges on clinical trial hold. Shares of biotech Zafgen tumbled more than 40% in Monday trading after the company announced that the FDA had put a clinical trial hold on its experimental diabetes drug ZGN-1061. This is far from the first time Zafgen has faced a devastating setback; in 2016, it was forced to drop development of its lead drug candidate, a treatment for obesity related to a rare disorder.

U.K.'s Vectura ditches asthma drug. Another clinical debacle on the horizon: U.K.-based Vectura's shares fell 10% on Monday as the company stopped development of its experimental asthma drug. The firm said that its treatment for severe asthma was unlikely to show a clinical benefit in patients following a clinical study. (Reuters)

THE BIG PICTURE

CVS, Aetna get final clearance for their historic merger. The final domino has fallen in the $69 billion CVS-Aetna merger quest, setting up a close within the next few days, according to the companies. Federal regulators at the Justice Department had already given the deal a preliminary blessing back in October; on Monday, the final state holdout, New York, also cleared the corporate marriage. The deal could now close by mid-week, according to regulatory filings. (CNBC)