Karikó, Weissman awarded Nobel Prize in medicine

Katalin Karikó and Drew Weissman, two pioneers of mRNA research, won the 2023 Nobel Prize in medicine or physiology this morning.

The pair made key discoveries about messenger RNA that enabled scientific teams to start developing the tool into therapies, immunizations, and — as the pandemic spread in 2020 — vaccines targeting the SARS-CoV-2 coronavirus. Moderna and the Pfizer-BioNTech partnership unveiled their mRNA-based Covid-19 shots in record time thanks to the foundational work of Karikó and Weissman, helping save millions of lives.

The pair performed their world-changing research at the University of Pennsylvania, where Weissman remains a professor in vaccine research. Karikó, who later went to work at BioNTech, is now a professor at Szeged University in her native Hungary, and remains an adjunct professor at Penn's Perelman School of Medicine.

FDA

What we owe ALS patients — and why one company fell short

"What do you say to a patient when the FDA rejects 'their' experimental drug," STAT's Matthew Herper writes in a poignant new column this morning, "one that they believe can alleviate at least some of their suffering?"

That's the question that arises following last week's FDA advisory committee meeting on an ALS treatment from BrainStorm Therapeutics, when the agency methodically dismantled the company's application.

There are no easy answers, Matt writes, but he argues the FDA did the right thing. "When it comes to drug development," he says, "we want the system that generates the greatest good for people with illnesses. This means that we don't want to waste resources on medicines that are harmful or don't work."

biotech scorecard

Another installment of the quarterly biotech scorecard

We're in for an eventful fourth quarter, so STAT's Adam Feuerstein has compiled another biotech scorecard.

What's on tap? We may see the FDA broaden the label of Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy to include older boys, based on a Phase 3 clinical trial that'll read out in a couple months. Similarly, we'll see an interim analysis from Pfizer for its own Duchenne gene therapy.

On Halloween, an FDA ad comm will review the first CRISPR-based treatment for sickle cell disease, which is being developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics. A decision is expected on Dec 8. Other approval decisions are coming down the pike this quarter for companies including Bluebird Bio, Aldeyra Therapeutics, and Alnylam Pharma.

vaccines

A surprising hurdle in developing an HIV vaccine

For all the decades of progress we've seen in the development of HIV therapies and prophylactics, we still have no vaccine. And the scientists who have dedicated decades to pursuing such an inoculation continue to find hurdle after hurdle: "To be honest," said Louis Picker, a founder of Vir Biotechnology, a company that's attempting to develop one, "I'm not completely sure that an HIV vaccine will ever be developed."

The field is at a crossroads, STAT's Jason Mast writes. Yet another large study failed earlier this year, and today there's only one late-stage HIV vaccine trial that's ongoing — experts think the technology is too dated to be effective. And even if the technology is excellent, it's devilishly hard to test it these days.

One part of the problem is that PrEP, or pre-exposure prophylaxis, is quite effective and therefore muddying the clinical trials. Those at highest risk are already receiving PrEP, and so researchers can't ethically randomize volunteers to receive a placebo if a 99% effective pill is available. But this means that millions more who aren't receiving PrEP miss out on a vaccine.

global health

In about-face, J&J opens up patent access to TB drug

Johnson & Johnson has announced it no longer will enforce any patents for its critical tuberculosis drug in 134 low- and middle-income countries.

The pharma giant has faced mounting pressure from advocates to change its pricing and patent policies for bedaquiline, a medicine that's a key component in treating multidrug-resistant tuberculosis. Even Unitaid, a global health initiative that invested in the J&J pill, publicly criticized the company Friday — saying it "ignored the public health community's calls" to improve access.

J&J's new move is meant to address "any misperception that access to our medicine is limited or restricted and builds on our decade of investments in collaborative efforts to help countries sustainably scale up access" so that more people with drug-resistant forms of the disease can be treated, the company said.

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