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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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Regeneron promised to comply with 23andMe’s consumer privacy policies and related data security laws.
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.
The restrictions on Novavax’s vaccine could portend changes at the FDA. Commissioner Marty Makary suggested last week that the agency could update its vaccine approval guidelines “in the coming days.”
Analysts at BMO Capital Markets said in a weekend note that a non-invasive blood test could help boost uptake of Alzheimer’s disease therapies.
The FDA and NIH recently announced plans to phase out animal testing requirements for some therapies. While organoid and AI providers celebrate, scientists warn that questions over safety, applicability and implementation remain.
While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.
While the Trump administration has painted the jettisoning of staff and regulations as good for business, there are multiple reasons it’s unlikely to work out that way.
The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.
The FDA also approved the use of Zynyz as a monotherapy for patients with squamous cell carcinoma of the anal canal who are intolerant to platinum chemotherapy or whose disease has progressed.
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.