FDA Approves Pfizer’s BEQVEZ™ Gene Therapy for Hemophilia B
Pfizer announced on Friday that the U.S. FDA has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate
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Rare Diseases
AstraZeneca Expands Rare Disease Pipeline with Amolyt Pharma Acquisition
AstraZeneca has announced it is set to acquire Amolyt Pharma, a French clinical-stage biotechnology company focused on developing novel treatments
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FDA Approves Krystal Biotech’s VYJUVEK™: First Redosable Skin-disorder Gene Therapy
Krystal Biotech, Inc., a Pennsylvania-headquartered biotech company specialising in genetic medicines for rare diseases, announced on Friday that the US
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ReCode Starts Phase 1 Trial of Inhaled Genetic Medicine for PCD
ReCode Therapeutics, announced the dosing of the first participants in a Phase 1 trial designed to evaluate the safety and
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Pfizer Reports Positive Results from Phase 3 Hemophilia B Gene Therapy Study
Pfizer Inc. (NYSE: PFE) announced in late December, positive top-line results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating fidanacogene
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Tezepelumab gets ODD in the US for Eosinophilic Esophagitis
Tezepelumab, developed by AstraZeneca in collaboration with Amgen, has been granted Orphan Drug Designation (ODD) in the US FDA for
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Takeda and JCR to Commercialize Next-Gen Hunter Syndrome Therapy
Takeda and JCR Pharmaceuticals announced today a geo-based exclusive collaboration and license agreement to commercialize JR-141 (INN: pabinafusp alfa), an
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Sanofi to buy Kadmon and its First-in-class Transplant Drug in $1.9B Deal
Sanofi announced that it will purchase U.S. biotech Kadmon to enhance its transplant business for approximately $1.9 billion. Sanofi and
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Eli Lilly enters $1 billion agreement to acquire Prevail Therapeutics
Eli Lilly & Co and Prevail Therapeutics announced a definitive agreement for Lilly to acquire Prevail for a total consideration
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