biotech
A historic FDA approval for CRISPR-based medicine
The FDA on Friday approved the world's first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases.
In a clinical trial, Casgevy was shown to eliminate recurrent episodes of debilitating pain caused by sickle cell, which afflicts approximately 100,000 people in the U.S., a vast majority of whom are Black. The same day, the FDA also approved another sickle cell treatment, a gene therapy from Bluebird Bio called Lyfgenia. Patients will now have the option of two cutting-edge therapies that provide potentially curative benefits.
Read more from STAT's Adam Feuerstein about the approvals. And our amazing biotech team also had two Q&As on the news. Read on...
biotech
Inside the historic approval with CRISPR CEO and Vertex founder
First, Adam spoke with the CEO of CRISPR Therapeutics, Samarth "Sam" Kulkarni:
When writing about the approval of Casgevy, we focus mostly on the clinical data showing how the treatment benefits patients, but can you describe some of the other, less-recognized elements that got you to this point?
One of the things that really helped us was our interaction with the regulators on manufacturing issues. They were willing to provide feedback on what would be required, but you have to take what they say seriously, and then just do it.
And we aren't done yet! Check out the conversation that STAT's Allison DeAngelis had with Vertex's founder, Joshua Boger, during a visit to his Boston-Seaport-area penthouse:
If you had gone back 29 years and told yourself where we've ended up, what would you have thought?
I would have lost a house in a bar bet if I had bet that Vertex would be the first company to bring a CRISPR drug to the world.
biotech
The quest to develop a new class of non-opioid painkillers
SANDY HUFFAKER FOR STAT
For decades, the biotech industry has tried — and failed — to develop new and effective painkillers without the addictive potential of opioids. There's reason to think that could change soon.
Vertex Pharmaceuticals is on the cusp of announcing results from late-stage trials of a drug that precisely blocks pain-sensing neurons from signaling the brain. The company will announce data early next year from Phase 3 studies testing the molecule's effectiveness at treating post-surgical pain. And before the end of the month, Vertex will share results from a mid-stage trial in people with diabetes who have chronic nerve pain.
If the Phase 3 trials are successful, Vertex plans to seek approval to treat moderate to severe acute pain — such as the kind experienced after surgery, a bad fall, or other specific events. Market analysts believe the therapy could become a future blockbuster if approved. But opinions surrounding its current experimental drug, a small molecule dubbed VX-548, are mixed. Vertex has argued that the treatment's benefits are clinically meaningful, while some outside researchers and observers say those benefits seem modest based on data the company has shared so far. Read more from STAT's Jonathan Wosen.
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