Intellia cleared to test gene editing therapy in U.S. trials

The FDA is green-lighting a clinical trial for the experimental CRISPR medicine made by Intellia Therapeutics — suggesting that regulators are becoming more comfortable with in-vivo editing. Now, U.S. patients with hereditary angioedema, a rare swelling disorder, can join a Phase 2 trial of the treatment. Early stage studies have already been completed in patients in the Netherlands, the U.K., and New Zealand.

Companies like Sangamo Biosciences and Excision BioTherapeutics have also gotten FDA clearance to test gene editing therapies in the body. But others, like Verve Therapeutics and Beam Therapeutics, are facing regulatory delays as the agency takes its time to oversee their applications.

more editing

International genome editing summit to delve into CRISPR baby fallout

An international genome editing summit kicks off next week in London, and a top agenda item will be addressing the aftermath of the 2018 CRISPR baby scandal. The last time the summit had been held, Chinese researcher He Jiankui set the scientific community ablaze with news he'd created the world's first gene-edited children.

"Five years ago, at the last summit, he misled the world about the health of the babies who were born during his experiment," University of Oxford anthropologist Eben Kirksey said. "We still do not know if his venture with CRISPR in the fertility clinic actually produced any medical benefits for the children. The scientific community needs to keep pushing Dr. He to reveal the full truthful details about his 2018 experiment."

Although He is not invited to next week's summit, its first session will focus on how China has adopted stringent new laws that regulate gene editing research — and how they might be enforced. Other topics that will headline the summit include base editing, prime editing, and embryo editing.

podcast

Who invented CRISPR 2.0?

What happens when a drug company actually lowers prices? And is Novavax going to survive? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Our colleague Allison DeAngelis joins us to explain how the latest CRISPR breakthrough is shaping up to be a free-for-all among a multitude of companies and labs, including some of the biggest names in biotech. We also discuss the latest news in the life sciences, including Eli Lilly slashing the cost of insulin, succession at the FDA, and how pandemic boom times have turned to bust.

Listen here.

cancer

PARP inhibitor Lynparza will be scrutinized by FDA panel

PARP inhibitors have had a rough go of it — several of these cancer drugs have been withdrawn for the market, and the FDA is becoming increasingly wary of them. The agency will be gathering an expert panel on April 28 to review AstraZeneca and Merck's application for Lynparza to be used to treat metastatic castration-resistant prostate cancer (mCRPC).

The advsiory committee will review data, FiercePharma writes, over whether Lynparza worked better in combination with Johnson & Johnson's Zytiga, or whether Zytiga worked better in this form of prostate cancer alone. The Lynparza-Zytiga combination won approval in Europe in December, and in the U.S., Lynparza is already approved for patients with mCRPC in patients carrying a specific mutation.

That said, the FDA saw signs that Lynparza might be harmful to patients' overall survival, and had its approval pulled for late-line ovarian cancer.

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