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The results are nevertheless “unprecedented,” UniQure executives said on an investor call Tuesday, emphasizing that the updated natural history database used as the control group for the Phase 1/2 study of AMT-130 underestimates disease progression.
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The approval of Mirum Pharmaceuticals’ ALK2 inhibitor for fibrodysplasia ossificans progressiva on Friday adds a third option for patients who had long been without any treatments, joining Regeneron’s recently approved Pasatru and Ipsen’s Sohonos.
The recent FDA approval of Fayuvi for a rare, childhood neurodegenerative disease was “particularly special” for Ultragenyx CEO Emil Kakkis. It could also, along with an August nod for another gene therapy, help offset recent challenges brought on by two asset failures.
Johnson & Johnson, Pfizer, Argenx and more are unlocking new immunology targets and looking to improve on existing ones in an attempt to catch some of the magic brought about by AbbVie’s Humira and Sanofi and Regeneron’s Dupixent.
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Big Pharma needs more—not fewer—bold bets on rare and difficult diseases after Novartis’ efforts to invest in muscular dystrophy and ALS hit major roadblocks this month.
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Merck continues to diversify its cancer efforts as the clock ticks down on Keytruda’s patent, picking up rights to a preclinical oncology asset in a deal that could exceed $2 billion.
What drives meaningful employee engagement in today’s life sciences workplace? This BioSpace report explores the factors that shape how employees feel about their organizations and what employers can do to create workplaces where talent wants to stay and thrive.
In the Phase 3 TEMPO program, AbbVie’s Juvmo elicited significant improvements in motor symptoms and prolonged periods with good symptom control.
Merck and Daiichi Sankyo’s withdrawal of an accelerated approval request for the antibody-drug conjugate ifinatamab deruxtecan is a “net positive” for rivals like GSK and Roche, according to RBC Capital Markets.
Stem cell therapies vary widely yet many physicians still treat them as interchangeable. A more credible model requires diagnostics, product characterization and conventional medical context to determine who and when should receive regenerative treatment.
Manufacturers are redesigning their processes to balance both better future emergency vaccine mobilization and the more demanding work of maintaining flexible, commercially viable manufacturing networks.
Nektar Therapeutics has been awarded $90 million in damages after a jury sided with the biotech, which had accused Big Pharma Eli Lilly of “botching” an analysis of their formerly partnered autoimmune drug.
Two RNA biotechs, ADARx Pharmaceuticals and City Therapeutics, are taking advantage of the IPO frenzy, which has led to a total of 27 newly public biotechs so far this year.
Ten patients out of 20 who received Johnson & Johnson and Legend Biotech’s Carvykti remained alive at five years of follow up.
Genentech’s agreement with Earendil Labs came the same day its parent company, Roche, inked a cardiometabolic and renal deal with Atavistik Bio for up to $2 billion.