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For $300 million upfront, Bayer is purchasing Perfuse Therapeutics to advance an eye implant for glaucoma and diabetic retinopathy, marking the company’s first pharma acquisition since 2021.
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Biogen’s Qalsody won FDA approval in 2023 to treat a rare, genetic form of amyotrophic lateral sclerosis. On Tuesday, QurAlis presented interim Phase 2 data showing the potential of a similar drug to more broadly treat the neurodegenerative disease.
As Q1 earnings arrive, three biotechs have big quarters ahead, with two—Amylyx and Neumora Therapeutics—betting at least partly on novel assets for obesity.
Comprehending the spate of recent rejections in the cell and gene therapy space may require looking no further than early-stage clinical trials of candidates from REGENXBIO, Excision BioTherapeutics and Intellia Therapeutics.
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Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
Drug development powered by artificial intelligence is countering incentives from the Inflation Reduction Act and making small molecules more attractive in the complex inflammatory & immunology disease space.
Around 300 FDA staffers laid off last week are being asked to return. So far, the Trump administration has terminated some 1,000 employees from the agency.
Non-opioid pain therapies are entering an unprecedented era, marked by the landmark FDA approval of Vertex’s Journavx and a growing number of alternative approaches. Their ultimate uptake, however, remains to be seen.
With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery breakthroughs are needed to propel RNA editing to the next stage.
The proposed acquisition by global investment firms Carlyle and SK Capital Partners could net shareholders $3 per share plus potential CVR dollars and provide bluebird bio with primary capital to expand the commercial reach of its gene therapies.
Acelyrin earlier this month agreed to an all-stock merger with fellow immune player Alumis, with their combined cash tiding the combined company over until 2027.
The move comes weeks after Pfizer terminated its partnership with Sangamo Therapeutics for another hemophilia gene therapy.
Many of these unlawful and unauthorized shipments were explicitly tagged for compounding, according to a new analysis. Separately, a group of state attorneys general has raised concerns about the unsafe GLP-1 drugs finding their way to American consumers.
The postponed ACIP meeting comes barely a week after Robert F. Kennedy, Jr. was confirmed as Secretary of Health and Human Services, despite controversy regarding his anti-vaccine history.