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Today, we talk about the much-awaited CRISPR-based drug Casgevy, discuss the new Gavi initiative that plans to dramatically ramp up vaccine manufacturing in Africa, and more. Have a nice weekend!
An historic moment for CRISPR is here
At some point today, the FDA is expected to approve the first CRISPR-based medicine. Called Casgevy, it’s a groundbreaking treatment for sickle cell disease that offers a potential cure for people living with the chronic, life-shortening blood disorder. The drug is made by Vertex Pharmaceuticals and CRISPR Therapeutics.
Casgevy’s 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.
Soon after today’s inevitable approval announcement, we’ll learn from Vertex how much the medicine will cost. (It won’t be cheap.)
But for all its virtues, whether or not Casgevy becomes a widely used treatment for people living with sickle cell — undoing decades of scientific neglect and medical racism — remains an open question that will only be answered in the months ahead.
How does the first CRISPR treatment work?
Can biotech rebound? And when is $300 million not $300 million? We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast.
With the first CRISPR-based medicine expected to win FDA approval any minute now, STAT’s Megan Molteni joins us to discuss how we got here and what it means for the future. Then, Bruce Booth from Atlas Venture joins us to discuss what was yet another downbeat year for biotech and whether there’s reason for optimism heading into 2024.
Using ultrasound-guided 3D printing in tissues
Researchers at Harvard and Duke have created a 3D printer that uses ultrasound to print biomaterials inside an organ. They were able to print a bone-shaped structure through 10 millimeters of pig skin and muscle to simulate bone reconstruction. They also demonstrated a way to potentially treat atrial fibrillation, by printing a patch on the left atrial appendage of an ex vivo pig heart.
The machine has a focused ultrasound transducer, which uses sound waves to travel through tissue to create intricate structures. It uses an “ink cocktail” that can contain a “concoction of polymers, particles, and chemical initiators,” one of the researchers told STAT. The components vary depending on whether they’re meant to turn into bone-like or tissue-like structures.
Gavi commits $1 billion to manufacture vaccines in Africa
Gavi, the Vaccine Alliance, has set up a new accelerator to help fund vaccine manufacturing in Africa — allocating up to $1 billion to improve access to important vaccines. The initiative is a response to the glaring gap in access to vaccines that became apparent during the Covid-19 pandemic. It’ll help boost domestic production of shots to treat diseases like cholera and malaria, which kill hundreds of thousands of African children each year, Reuters writes.
The African Union aims to have the African vaccine manufacturing industry supply more than 60% of vaccines needed on the continent by 2040. Right now, only about 1% of the vaccines are made there now.
The funding comes from leftover money from the COVAX initiative, an effort created during the pandemic to help disseminate vaccines in low-income countries.
More reads
- Biden proposal targeting pharma patents spurs industry fears and consumer doubts, STAT
- Klobuchar questions drug company behind 10-fold spike in price of lead poisoning antidote, CNN
- AbbVie CEO Gonzalez brushes off FTC concerns for Cerevel buy: ‘This acquisition is not anti-competitive,’ FierceBiotech