Wikipedia -WSS

Wikipedia

author：entertainment 2025-10-02 11:38:04 - browse：1572

Earlier this month, Intellia Therapeutics announced it was scrapping plans to include U.S.-based sites in one of its mid-stage CRISPR trials after the Food and Drug Administration requested additional data to support including female patients of childbearing potential. The move left many researchers wondering whether the specter of accidental germline transmission — which haunted early gene therapy trials — had now risen over the field of gene editing.

Over the weekend, Intellia sought to assure the gene editing community that no such thing is happening. At a meeting at Cold Spring Harbor Laboratory that drew more than 400 of the field’s top scientists, the company shared more detailed information about studies it has conducted to assess the risks that renegade CRISPR components could be getting into an egg or sperm. These studies demonstrate “that there’s no evidence of vertical germline transmission of those edits,” Jonathan Phillips, head of pharmacology and toxicology at Intellia, said during a presentation Saturday.

Intellia is one of the leading companies working to advance in vivo gene editing therapies. In a landmark study published in the New England Journal of Medicine in 2021, Intellia revealed data from six patients in its trial for a transthyretin amyloidosis therapy — showing for the first time that it was possible to precisely alter the DNA of particular cells within the human body to treat disease with a one-time infusion of CRISPR. Included in the supplementary material was a redacted version of the company’s package requesting authorization to start its clinical trial. It noted that during toxicology studies in non-human primates, Intellia scientists discovered that editing had taken place at low levels in organs other than the liver, including the spleen and adrenal glands. In three out of 12 female monkeys, they also found editing in the animals’ ovaries.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED Log In

Previous article： Bright Health sells Medicare Advantage business to Molina
Next article： Merck, Daiichi strike cancer drug pact worth up to $22 billion

leisure time

explore

WSS

STAT Summit: Ending the crisis of Black deaths in the U.S.

J&J lung cancer data amount to new salvo against AstraZeneca

STAT Readout Newsletter: Biotech news today

Doctors increasingly discourage vaping amid mounting health concerns

Russian and Belarusian players banned from a women's tennis tournament in Prague

Europe is fastest

Wikipedia

Wikipedia

explore

Alkermes shareholders re

Campaign finance charge dropped from case against Sam Bankman

At least 13 dead in Texas as scorching temps continue

Japan raises alarm over China's military, Russia ties and Taiwan tensions in new defense paper

Cancer drug shortages should be causing more outrage

Merck, Daiichi strike cancer drug pact worth up to $22 billion

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Typhoon Doksuri makes landfall in China after bringing deadly landslides to Philippines

Affirmative action in medical school literally saved lives

Harvard's Jason Buenrostro wins MacArthur grant for genetics work

Wikipedia

HCA says smaller share of spending going to labor

Teacher posts viral 'school supply rant' with advice for parents

Europe is fastest

The future of Biogen: Pragmatism, ‘moonshots,’ and no Twinkies

Young Chinese opt out of the rat race and pressures at home to pursue global nomad lifestyle

ADCs take center stage as promising cancer treatments

knowledge

leisure time

explore

leisure time