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BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
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Nusano will bring a massive new radioisotope facility in Salt Lake City online by the end of the year, establishing a supply of starting materials for the next generation of radiopharmaceuticals.
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
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Read our takes on the biggest stories happening in the industry.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year.
At a Thursday ASGCT 2024 session, CBER Director Peter Marks made the case for a better, “more convergent” global framework on cell and gene therapies, especially for rare diseases.
The Bay Area–based biotech plans to use the funds to advance two lead programs, one to treat atopic dermatitis and another for immune-mediated diseases.
As competition with Eli Lilly heats up, Novo Nordisk has partnered with Flagship’s Metaphore Biotechnologies to take a biomimicry approach to GLP-1s.
After a combination with Eisai’s Lenvima failed to improve survival in advanced disease in December, adjuvant Keytruda plus chemotherapy missed the mark in newly diagnosed, high-risk patients.
An autologous and personalized regulatory T cell therapy is safe in patients with type 1 diabetes, but does not help preserve β-cell function.
Takeda saw a 57% drop in profit in its 2023 fiscal year and has lowered its outlook for the coming fiscal year, mostly driven by increasing generic competition for its ADHD treatment Vyvanse.
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated virus gene therapies to more rare disease patients.
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On this episode of Denatured, Lori Ellis and guests discuss President Biden’s Executive Order on Advancing Women’s Health Research and Innovation and the outlook of investment in women’s health.