October 27, 2025

Intellia CRISPR treatment studies paused following case of liver toxicity

Intellia Therapeutics has paused two clinical trials of its CRISPR gene-editing treatment after a patient in one of the studies was hospitalized with serious liver toxicity, the company said this morning.

Shares of Intellia were nearly cut in half in pre-market trading.

The two studies being suspended are testing a gene-editing treatment for two forms of a disease called ATTR amyloidosis, in which misshapen proteins can build up in the liver and damage the heart or the nerves. Intellia's drug aims to permanently knock out the gene that codes for those proteins.

biotech

Chinese biotechs are unfazed by threat of U.S. restrictions

Chinese drugmakers are continuing to strike major deals with their counterparts in the U.S. and across the globe, even as American officials attempt to guard against their growing competitiveness.

Last week, Innovent Biologics announced a deal with Takeda worth up to $11.4 billion for rights to a pair of cancer medications. Earlier this month, Zenas Biopharma, a U.S. biotech company developing therapies for autoimmune diseases, said it would license three drugs from China's InnoCare for up to $100 million.

The deals come as U.S. officials reportedly consider heavier scrutiny of Chinese licensing agreements and as legislators revive the BIOSECURE Act. But Chinese biotechs are showing no sign of slowing down.

Read more from STAT contributor Brian Yang.

RARE DISEASE

BridgeBio reports positive data from muscular dystrophy drug

BridgeBio said this morning that an experimental therapy for a rare form of muscular dystrophy succeeded in a Phase 3 trial, potentially setting up the first approval of a drug to treat the disease.

The oral drug, known as ribitol, is for one subtype of limb-girdle muscular dystrophy, a broad collection of over 30 genetic diseases that cause similar patterns of muscle loss.

The company plans to file with the FDA next year.

immunology

CAR-T therapies from Cabaletta, BMS show promise in autoimmune disorders

Experimental CAR-T therapies from Cabaletta Bio and Bristol Myers Squibb have induced complete remissions in patients living with a severe inflammatory muscle disease, results from dual clinical trials being presented this week show.

The new data, while still preliminary, add to evidence reported over the past several years that personalized cell therapies — already used to treat blood cancers — may prove to be curative for patients with serious autoimmune disorders.

DEALS

Novartis to buy RNA-focused Avidity in $12 billion deal

Novartis said yesterday it would buy Avidity Biosciences for roughly $12 billion in cash. The deal, at $72 a share, represents a 46% premium on Avidity's closing share price on Friday.

With the purchase, Novartis is picking up three late-stage programs: one treatment for Duchenne muscular dystrophy, another for myotonic dystrophy type 1, and a third for facioscapulohumeral muscular dystrophy.

But the Swiss pharma also framed the deal as one that would give it a new platform, with Avidity designing a class of drugs focused on delivering RNA to muscle.

Syndax leukemia drug wins FDA expanded approval

Syndax Pharmaceuticals' drug, called Revuforj, was cleared Friday to treat patients with relapsed or refractory acute myeloid leukemia caused by a mutation in a gene called NPM1 that makes the blood cancer more difficult to treat. Approximately 30% of AML cases involve an NPM1 mutation.

Revuforj won an initial FDA approval last year for a different and less common type of genetically altered AML.

The Revuforj news was not all good. The newly expanded FDA prescribing label includes a black box safety warning for QTc prolongation and Torsades de Pointes — serious heart rhythm disorders that, in Torsades cases, can be life-threatening.

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