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While BMO Capital Markets said that zimislecel is “highly encouraging” for type 1 diabetes, questions regarding its target population and Vertex’s execution hang over the cell therapy’s commercial potential.
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Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
As multiple companies vie to expand on Alnylam’s success in commercializing RNAi therapeutics, the pioneering company has set a goal of targeting small interfering RNA to any tissue by 2030.
Vaccine skepticism is at an all-time high in the U.S., and HHS Secretary Robert F. Kennedy Jr. is making some drastic moves in the name of reversing that trend. But misinformation and inconsistencies within the country’s healthcare agencies highlight problems with his approach.
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Known as the home of big-name companies including Johnson & Johnson, New Jersey has become a destination for more and more small and midsize life sciences businesses. Choose New Jersey, Grace Therapeutics and Onyx Equities executives discuss the state’s life sciences scene.
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BioSpace Senior Editor Annalee Armstrong headed to the J.P. Morgan Healthcare Conference with a months-long story idea brewing. Unfortunately, it was one she’s written before.
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Andembry is the first monthly Factor XIIa inhibitor for the prevention of HAE attacks and will be available to patients “before the end of June,” CSL announced.
Another patient has died from acute liver failure after receiving Sarepta’s gene therapy for DMD ; After a quiet start to the year, M&A is back with one deal for a gene editing biotech reinvigorating that sector; and RFK Jr. installs a suite of new vaccine board members who share his skeptical views on vaccines.
Findings that U.S. companies can sue foreign rivals despite limited business operations in the country could dissuade drug developers from targeting the U.S. market, potentially benefiting domestic producers of biosimilars.
BioNTech said in 2022 that it faced “threats of a groundless patent infringement suit” from a company that was “unable to bring to market any product to help in the fight against COVID-19.” Now, the mRNA biotech is buying that very company.
Sanofi paid a more than 300% premium on its acquisition of Vigil Neuroscience, suggesting a fierce battle to seal the deal. Across biopharma, companies are sometimes willing to put it all on the line for the right buyout. Novartis’ recent acquisition of Regulus for $800 million upfront provides a case study.
The program will bring together experts from across the FDA for a team-based review, rather than having an application move across numerous offices within the agency before getting a yay or nay.
Nearly $30 million in tax incentive awards are expected to add 1,519 new life sciences jobs in Massachusetts, with about 52% of the positions spread over Boston and Cambridge. Vertex, which received the largest award, will create hundreds of roles in Boston.
Jefferies analysts said on Tuesday that Ventyx could leverage its mid-stage data for VTX3232 to position the oral drug candidate as a treatment for other neurodegenerative diseases, including Alzheimer’s.
The pending deal was rumored overnight after a report from the Financial Times, spurring analysts to speculate that if true, the entire gene editing space would see a boost at the markets.
Venclexta, when combined with azacitidine, elicited an overall survival benefit below 10% in patients with myelodysplastic syndromes.