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Hello my dear subscribers. This is my last Readout newsletter – just kidding! This is just my last reading for the next few weeks. I’m going on vacation for three weeks, so you won’t see my name here for a while. In the meantime, my colleague Meghana Keshavan will direct the newsletter.

See you all in December!

The need to know this morning

• In order to preserve the company’s dominant position in the field of cancer, Merck said that would be the case authorize a new cancer drug Since LaNova Medicinesa Shanghai-based company, for $588 million upfront and up to $2.7 billion in potential milestone payments.

GOP-controlled Congress gives Trump broad powers

Now that it has been determined that Republicans will have full control of the House and Senate, President-elect Trump will have broad authority to assert his will on health policy.

Major health care issues are potentially at stake, including subsidies for Affordable Care Act plans, funding for Medicaid, restructuring of important federal agencies like the FDA and CDC, access to telehealth, pharmaceutical intermediary reforms, drug price negotiations and the rise of biotechnology in China.

Democrats are in a weak position to assert their preferences on many of these issues, although arcane congressional rules and divisions within the Republican Party could potentially give the party at least some openings. In the Senate, 60 votes are needed to overcome a filibuster, and Republicans are far from reaching that number.

Learn more by STAT’s John Wilkerson.

Looking forward to reading Vertex’s High Stakes Pain

Vertex Pharmaceuticals’ next quest is to transform pain treatment, and a crucial milestone is fast approaching.

The biotech plans to announce results by the end of the year from a mid-stage trial of an experimental treatment for patients with lumbosacral radicular syndrome, more commonly known as sciatica.

Analysts view the trial as a major moment for the company, and the stakes are high. My colleague Jonathan Wosen describes the background and design of the trial, as well as expectations for the results.

Learn more.

CDC and FDA officials warn of anti-vaccine consequences

Two Biden administration officials – CDC Director Mandy Cohen and the FDA’s top vaccine regulator, Peter Marks – warned yesterday that there could be serious consequences for the nation’s children if anti-vaccine views prevailed.

The comments, made at separate conferences yesterday, come as the country waits to see who will fill key health care posts in the new Trump administration and how much influence anti-vaccine figures like Robert F. Kennedy Jr. might have. exercise.

“The natural consequences of not believing in the science or the potential benefits of these vaccines could be unnecessary deaths,” Marks said. “I’m sorry to say that. I hope it doesn’t have to come to that, but it looks like we’re there.

Learn more by Helen Branswell and Anil Oza of STAT.

Tune Therapeutics to Launch HBV Gene Editing Trial

From my colleague Jason Mast: Tune Therapeutics will soon begin human testing of a gene-editing therapy for hepatitis B, the startup announced today. It is the second company to obtain regulatory approval for such a study, after Precision Biosciences announced in October that it would launch the first-ever trial of a gene-editing treatment for HBV.

The efforts come more than a decade after researchers first proposed that gene editing could eventually provide a cure for a chronic virus that still infects more than 300 million people worldwide. The virus has evaded other treatment attempts by forming stable DNA loops in liver cells or integrating into human DNA, beyond the reach of conventional approaches.

Intellia Therapeutics and Beam Therapeutics toyed with VHB programs before ultimately shelving them. Precision Biosciences is trying to extract a fragment of DNA from patients’ livers, using a DNA-cutting enzyme found in algae. Tune doesn’t cut DNA at all. Instead, the company uses an approach called epigenetic editing, in which researchers can use certain enzymes to delete or activate genes without breaking them.

Neither Precision nor Tune are starting their trials in the United States, where CRISPR companies often complain that regulators are too strict in their preclinical requirements. Tune begins in New Zealand. Precision decided to study its therapy in Moldova. If successful, both will likely apply to begin studies in the United States.

Amgen defends its obesity drug over bone problems

And to come full circle, if you remember, on Tuesday, a research note raising potential safety concerns with Amgen’s lead obesity candidate triggered the company’s stock to plummet, wiping out $12 billion of market value.

The memo was based on data found in hidden tabs of a file attached to a release of early trial results for the drug, called MariTide. The tabs contained what appeared to be data showing that study participants were experiencing loss of bone mineral density, the memo said.

Amgen’s development chief defended the drug at an investors conference yesterday, saying the data tables referenced in the memo were not finalized and were not subject to standard review. The company therefore asked the newspaper to publish a correction and add the finalized data.

There were overlapping margins of error between the treatment and placebo groups, leading researchers to conclude that there was no association between MariTide and changes in bone density, a- he added.

Observers have speculated that MariTide may cause bone density loss due to its mechanism of blocking GIP hormone receptors. But the Amgen executive said the company studied the genetic data and found no signs that genetic variants associated with reduced GIP receptor activity were linked to reduced bone density or bone diseases. .

Read all about this saga here.

Neurogene and the race to the bottom of gene therapy

From my colleague Adam Feuerstein: Forgive this grumpy rant, but I think companies developing genetic drugs, even for the rarest and most serious diseases, should conduct randomized, placebo-controlled clinical trials. Anything less is scientifically irresponsible and shows a lack of respect for patients and their families. These therapies are irreversible and carry significant risks, so it is preferable that their benefits be clearly demonstrated.

Yet at Neurogene, the developer of a gene therapy for Rett syndrome, timeliness appears to be the priority. The company collects as little data as possible from single-arm, open-label studies that, by definition, will not demonstrate compelling evidence of effectiveness or safety.

Randomized, placebo-controlled studies are the gold standard for conclusively determining the benefits and risks of an experimental treatment. Yet gene therapy developers make an exception, saying it is too difficult logistically or ethically to ask patients to participate in such scientifically rigorous trials.

Read more about me here.

More reading

• Struggling Ginkgo Bioworks announces it will move away from soon-to-open seaport building, Boston Globe
• The new Senate Majority Leader, Thune, comes from a state that relies on rural hospitals, telehealth, STATUS
• Moderate GOP senators: Don’t ask me to confirm RFK Jr., STATUS