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Good morning, everyone. Damian here with the latest megaround in biotech venture capital, a rough month for biotech’s neuroscience renaissance, and the Novo Nordisk Foundation’s big computer.
The need-to-know this morning
- On Monday, the Food and Drug Administration approved a gene therapy for children with a fatal neurodegenerative disease called metachromatic leukodystrophy, or MLD. Today, Orchard Therapeutics said it would charge $4.25 million for the treatment, called Lenmeldy, making it the most expensive drug in history. But for the 40-100 children born with MLD each year, Lenmeldy will be the only lifeline away from a progressive, fatal disease.
- Acelyrin reported preliminary results from an early study investigating an antibody treatment for thyroid eye disease that can be administered with a simple skin injection.
A bright idea in cancer raises $150 million
Biotech startup Clasp Therapeutics has a plan to turn the body’s natural defenses against cancer while minimizing the risk to healthy bystander cells, an idea that helped the nascent company raise $150 million in venture dollars.
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