| | | |   | It's Meghana today, with some help from both Matt and Adam. We talk Vertex, we talk biosimilars, we talk Bluebird restructuring. Happy Tuesday. | | | Vertex gets top stem cell scientist for diabetes work Vertex Pharmaceuticals is hiring Douglas Melton, one of the top stem cell researchers in the world. He’s leaving his post at Harvard to pursue new cellular therapies for type 1 diabetes. Both his children have the disease, so this quest is particularly meaningful to him. “Given my personal interest in type 1 diabetes, I’m convinced that I could be most effective at the company that’s leading the area,” Melton told STAT exclusively. He’ll be using stem cells to create beta cells, which are absent in the pancreases of people with type 1 diabetes. Vertex is already developing these sorts of beta cells, so Melton will be where the action is.
Read more. | Bluebird begins restructuring, layoffs to save money Bluebird Bio is reducing expenses and laying off nearly a third of its employees to preserve cash ahead of two pivotal, gene therapy approval decisions expected later this year, the company said this morning.
The restructuring, which aims to save $160 million over the next two years, comes one month after Bluebird warned investors there was "substantial doubt" about its ability to remain solvent beyond this year. Bluebird is laying off approximately 150 employees, or 30% of its total workforce, and halting spending on certain research programs, including an effort aimed at making bone-marrow conditioning — a prerequisite for gene therapy treatment – less toxic.
Bluebird’s financial difficulties are the result of a prolonged pileup of clinical, business, and regulatory setbacks that have put at risk its ability to deliver potentially curative treatments to patients in the U.S. born with two rare diseases — the blood disorder beta-thalassemia and the pediatric brain disease cerebral adrenoleukodystrophy, or CALD.
Read more. | “Silencing” the expression of disease-causing genes using RNA interference (RNAi) During gene expression, errors in DNA can result in faulty mRNA that cause or contribute to disease. Alnylam’s RNAi therapeutics utilize specially designed small interfering RNA (siRNA) to target and degrade these mRNA, treating disease at source. Learn how. | Medicare could've saved millions with biosimilars (but didn't) Medicare Part D spending on biologics in 2019 could have been $84 million less than it was, a new government analysis shows, had these medicines been used more frequently. Beneficiaries themselves could have saved $1.8 million. But this didn’t come to pass because not all Medicare plans cover biosimilars — and those that did “rarely encouraged” prescribing. Instead, the brand-name biologics tend to be on formularies. This data suggests that biosimilars, which are 20% to 30% less costly than brand-name biologics before discounts, could indeed make a dent in health care spending. Read more. | More reads - The creator of the CRISPR babies has been released from a Chinese prison. (MIT Technology Review)
- Sanofi, Regeneron score fast FDA review for Dupixent in eosinophilic esophagitis. (FiercePharma)
| Thanks for reading! Until tomorrow,  | | | |
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