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It’s early days for xenotransplantation, but eGenesis, Eledon, United Therapeutics and more are working to develop solutions to make this approach a viable option and help ease the organ shortage crisis.
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials.
The FDA approval of Alnylam’s Amvuttra sets up a three-way race with Pfizer and BridgeBio, which both market transthyretin stabilizers for transthyretin amyloid cardiomyopathy.
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Pfizer Inc. announced that the U.S. Food and Drug Administration has approved BEQVEZ™ for the treatment of adults with moderate to severe hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid as detected by an FDA-approved test.
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The HHS secretary recently canceled $500 million worth of BARDA contracts around mRNA vaccine research. But the U.S. government has already spent billions on this work, which has saved millions of lives.
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The medium-sized biopharma is showing off new results from dordaviprone and Zepzelca, both of which were acquired through Jazz Pharmaceuticals’ dealmaking over the last five years.
BioSpace is on site to keep you updated on all of the biggest data and news from the conference.
Although the FDA has rejected Stealth’s new drug application for Barth syndrome candidate elamipretide, the agency identified a potential accelerated approval pathway. The company has pared down its staff to conserve resources to fund a potential resubmission.
Analysts at Truist Securities admitted that the result for itepekimab, which the companies were hoping to follow their blockbuster Dupixent in the I&I space, was “contrary to our expectations—we were wrong.”
Despite mixed results, analysts maintained faith in ivonescimab’s ability to cross over between Eastern and Western patient populations.
The National Legal and Policy Center, a right-wing advocacy and watchdog group, had asked the company to revisit its DEI goals in executive pay incentives.
At the heart of the deal is an anti-Claudin18.2 antibody-drug conjugate being developed for solid tumors, including gastric cancer and pancreatic cancer.
Keros will be down to 85 full-time employees after the layoffs and expects to generate annualized savings of $17 million.
According to the nonprofit news outlet NOTUS, at least seven studies cited in the Make America Healthy Again report released last week are nonexistent. The White House shrugged off questions about the errors.
While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of patient deaths in other trials for the modality and sent the company’s stock tumbling in pre-market trading.