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Novo Nordisk, Moderna, Novartis, raw milk

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Novo Nordisk, Moderna, Novartis, raw milk

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What you need to know this morning

Moderna is experiencing some turbulence in its gene editing efforts

We learned yesterday about some hiccups in Moderna’s gene-editing efforts: It’s ending a partnership with a gene-editing startup and, as a source told STAT, the head of its genomics division has left.

The developments mark a reversal from the company’s plans to invest in gene editing three years ago, when money came from the sale of Covid vaccines. Moderna President Stephen Hogue told investors at the time: “This will be a completely new approach for Moderna and a dramatic expansion of our modality strategy.”

The company’s technology has long seemed like a good fit for gene editing. To deliver CRISPR and other gene-editing systems into the body, researchers often encode the proteins as mRNA and encase them in a lipid bubble called a lipid nanoparticle — the same system Moderna used to deliver spike proteins for its coronavirus vaccines.

Read more from STAT’s Jason Mast on what this means for Moderna.

BridgeBio says goodbye to oncology

The biotech sector is building its oncology subsidiary into a fully independent company as it focuses its resources on the expected launch of its pivotal heart drug.

The subsidiary, which develops treatments for cancers with mutations in the KRAS gene, will become BridgeBio Oncology Therapeutics. It is backed by $200 million in private financing led by Cormorant Asset Management and Omega Funds.

It’s an interesting development in BridgeBio’s unique business model. The biotech was born from the idea of ​​being a hub-and-spoke company, meaning it consists of several subsidiaries, each independently working on different drugs. The aim is for the subsidiaries to share central resources while having the freedom and expertise to conduct their own research. But as BridgeBio develops more drugs, each of which costs more money, its business model has become more complicated.

Read more from me about BridgeBio’s plans and what the CEO told me about the business model.

Novartis is expanding its radiopharmaceutical plans

Novartis, which already produces the most important radiopharmaceutical drug, is doubling its position in this field. It will pay $1 billion upfront and up to $750 million if certain milestones are met to acquire startup Mariana Oncology.

Mariana is a three-year-old company developing drugs that drop radioactive isotopes on cancer cells, decimating them more powerfully than traditional radiation treatment. It was founded by investors Atlas Venture, Access Biotechnology and RA Capital Management.

The success of Novartis’ own radiopharmaceutical drug, Pluvicto, has created a domino effect in the industry. Eli Lilly, AstraZeneca and Bristol Myers Squibb have spent billions of dollars to acquire radiopharmaceutical startups.

Read more from STAT’s Allison DeAngelis about this new deal.

Promising results in a rare skeletal disease

A team of academic researchers reported that a gene therapy alleviated skeletal abnormalities linked to a rare inherited disease called Hurler syndrome in a small trial.

This treatment, called OTL-203, has been licensed to Orchard Therapeutics, which was recently acquired by Japanese drugmaker Kyowa Kirin.

People with the disease suffer from short stature, spinal defects and extremely stiff joints, complications that significantly limit their quality of life. But in the nearly four-year study, the children who received the therapy grew to a height within average standards, and had much more flexible shoulder, hip and knee joints than untreated children.

If longer studies show the treatment to be effective, it is possible it could provide these patients with more life-changing results than bone marrow transplants, the current standard approach for Hurler syndrome.

Read more from STAT’s Andrew Joseph about these new results.

Read more

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  • Lawmakers Grill UnitedHealth CEO Over Cyberattack: ‘Your Company Let the Country Down,’ STAT
  • Lawmakers, as part of ‘lab leak’ Covid research, push to ban EcoHealth from federal research funds, STAT
  • Pfizer’s strong Vyndaqel sales draw attention to the patent life of rare disease drugs, BioPharma Dive
  • Boehringer sets a target for Humira, says it’s necessary to shift it away from formularies. Endpoints