Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common mutation being the E342K mutation (Z allele) that introduces an amino acid change from Glutamic Acid (E) coding for M-AAT to a Lysine (K) coding for Z-AAT.
Celltrion Inc., of Incheon, South Korea, gained MFDS clearance of Eydenzelt (CT-P42) as a biosimilar referencing Regeneron Pharmaceuticals Inc./Bayer AG’s Eylea (aflibercept) on May 30. The news comes on the heels of the U.S. FDA approving the first Eylea interchangeable biosimilars on May 20.
Japanese industrial conglomerate Asahi Kasei Corp. has made an offer to acquire Swedish rare diseases specialist Calliditas Therapeutics AB for SEK11.8 billion (US$1.1 billion). The offer, at an 83% premium to the closing share price of SEK113.60 on Monday, May 27, is recommended by the three biggest shareholders and the board of Calliditas, which said the company would benefit from “being part of a larger platform.”
Researchers from Duke University presented findings from a study that aimed to assess the physiological role of Piezo1, a mechano-gated ion channel that is highly expressed in pancreatic acinar cells, in the exocrine pancreas.
Novo Nordisk A/S’s once-weekly human insulin analogue for adults with type 1 diabetes mellitus could be a useful tool for patients and physicians, the U.S. FDA’s Endocrinologic and Metabolic Drugs Advisory Committee said May 24, but it also agreed that the risks outweighed the benefits for improving glycemic control.
Shanghai- and San Diego-based Degron Therapeutics Inc. secured a potential $1.2 billion deal with Tokyo-headquartered Takeda Pharmaceutical Co. Ltd. May 23 for a multitarget collaboration and exclusive licensing agreement for molecular glue degraders. “It is a breakthrough technology in the small-molecule drug discovery field,” Degron CEO Lily Zou told BioWorld. “People talk about cell and gene therapy, but small molecules are still the mainstream of drug discovery, [with] more reach.”
Once-daily insulin treatment is getting a challenge from Novo Nordisk A/S’s once-weekly option. The U.S. FDA’s Endocrinologic and Metabolic Drugs Advisory Committee meets Friday, May 23, to review the BLA for once-weekly Awiqli (insulin icodec), a human insulin analogue from Novo for adults with type 1 diabetes mellitus.
Shanghai- and San Diego-based Degron Therapeutics Inc. secured a potential $1.2 billion deal with Tokyo-headquartered Takeda Pharmaceutical Co. Ltd. May 23 for a multitarget collaboration and exclusive licensing agreement for molecular glue degraders. “It is a breakthrough technology in the small-molecule drug discovery field,” Degron CEO Lily Zou told BioWorld. “People talk about cell and gene therapy, but small molecules are still the mainstream of drug discovery, [with] more reach.”
Prologue Medicines Inc. has launched to develop therapeutics created from the viral proteome, which are proteins produced across all viruses. It’s a newer twist on harnessing the power of the proteins that regulate biology. The company was founded on the premise of peering beyond the human proteome to see if there were viral proteins that could become therapeutics, Theonie Anastassiadis, Prologue’s founding president, told BioWorld.