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RFK Jr. invested in CRISPR while criticizing gene editing

January 23, 2025
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National Biotech Reporter
Good morning. We've got lots of news coming out of D.C. today — let's get straight into it.

politics

RFK Jr. invested in CRISPR while criticizing gene editing

New financial disclosures from Robert F. Kennedy Jr., President Trump's pick as health secretary, showed that he had invested under $15,000 into CRISPR Therapeutics, which makes the sickle cell gene-editing therapy Casgevy. He plans to divest his stake in CRISPR, as well as in another biotech called Dragonfly Therapeutics, my colleagues in D.C. report.

RFK Jr., it turns out, has a years-long track record of voicing concerns about CRISPR, a technology he would be in position to regulate if confirmed to lead the Department of Health and Human Services. Children's Health Defense, the nonprofit that RFK Jr. founded and led for years, has repeatedly raised similar concerns. Read more from STAT's Jonathan Wosen.



politicS

Trump administration cancels key science meetings

In other political news, a flurry of scientific gatherings and panels across federal science agencies were canceled yesterday, prompting confusion and concern among researchers and others.

As STAT's Anil Oza reports, at least some meetings of National Institutes of Health study sections, which review applications for fellowships and grants, were canceled without being rescheduled. A Feb. 20-21 meeting of the National Vaccine Advisory Committee, a panel that advises the leadership of the Department of Health and Human Services on vaccine policy, was also canceled. 

The scope of the cancellations was unclear. It was also unclear whether they were related to the Trump administration's freeze on external communications until Feb. 1. 

Read more.


biotech

Tris Pharma, looking to compete with Vertex, reports acute pain trial success

From my colleague Jonathan Wosen: For decades, pain has been a graveyard for drug developers. But in yet another sign that the field's fortunes might be improving, Tris Pharma yesterday reported that its experimental drug helped tamp down the pain of patients who underwent abdominoplasty, or "tummy tuck" surgery.

The Phase 3 trial, ALLEVIATE-1, recruited around 300 patients, half of whom were randomly assigned to take a placebo and the other half to take cebranopadol, Tris' drug. Patients then reported their pain levels on a standard rating scale from 0 (no pain) to 10 (the worst pain imaginable). Those taking cebranopadol once a day for two days reported less pain than the placebo group, with a 1.34-point lower pain level on average across multiple time points, the company announced in a press release. The firm also noted that the drug was generally safe and well-tolerated, with nausea as the most common adverse event.

Tris, a privately held specialty pharma company, is soon planning to announce results from a separate trial of the drug in patients getting bunion surgery. The New Jersey firm, which is looking to show its drug can treat moderate-to-severe acute pain, plans to apply for regulatory approval later this year. If that strategy sounds familiar, that's because it is. Vertex ran trials of its own therapy, suzetrigine, in these same patient populations before applying for approval to treat acute pain; an FDA decision on that drug is now just a week away. Tris is also exploring whether cebranopadol can help treat people with substance use disorders and chronic pain.


biotech

Looking ahead to Akero's MASH study next month

Akero Therapeutics next month will be reporting results from a nearly two-year trial of its drug efruxifermin in patients with cirrhosis caused by the fatty liver disease known as MASH.

It's Akero's second chance at demonstrating a benefit for a group of advanced patients with few treatment options. (Akero's trial is testing F4 patients, patients with the most severe stage of MASH, while Madrigal's recently approved MASH drug is for F2 and F3 patients.)

Akero earlier reported initial results from this MASH cirrhosis study. At 36 weeks, more patients on the drug showed a meaningful reduction in liver fibrosis, or scarring, without other symptoms of MASH getting worse than patients on placebo. The difference though, of 10 percentage points, was not statistically significant.

Akero has kept patients in the trial for another 60 weeks, and those results are what are expected next month. 

Read more from STAT's Adam Feuerstein.


Correction: Yesterday's newsletter incorrectly stated the date of YolTech's announcement of its in vivo beta-thalassemia trial. The announcement was made on Monday.


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