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BioWorld - Friday, December 26, 2025
Home » Alexion Pharmaceuticals Inc.

Articles Tagged with ''Alexion Pharmaceuticals Inc.''

Concept art for adeno-associated viral-based gene therapy.

JCR’s third deal with Alexion gives rights to AAV capsids

July 15, 2025
By Marian (YoonJee) Chu
No Comments
Under a new licensing deal announced July 8, JCR Pharmaceuticals Co. Ltd. granted Alexion Pharmaceuticals Inc. rights to its adeno-associated virus (AAV) capsids for use in up to five of Alexion’s genomic medicines programs.
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Concept art for adeno-associated viral-based gene therapy.

JCR’s third deal with Alexion gives rights to AAV capsids

July 9, 2025
By Marian (YoonJee) Chu
No Comments
Under a new licensing deal announced July 8, JCR Pharmaceuticals Co. Ltd. granted Alexion Pharmaceuticals Inc. rights to its adeno-associated virus (AAV) capsids for use in up to five of Alexion’s genomic medicines programs.
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Golden dollar coins
Analysis

Few biopharma mega-mergers hit the jackpot

Feb. 5, 2025
By Karen Carey
Due diligence plays a significant role in M&A transactions, but the eventual return on investments don’t always add up to the purchase price. While some companies such as Abbvie Inc. and Bristol Myers Squibb Co. – as shown in part one of this three-part series – have succeeded in acquiring products able to surpass M&A sticker prices, the vast majority of deals analyzed by BioWorld showed that most buyers remain significantly in the red.
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3D illustration of kidney cross section

Otsuka says IgAN, too, with phase III sibeprenlimab data

Oct. 29, 2024
By Marian (YoonJee) Chu
Visterra Inc., a subsidiary of Otsuka Pharmaceutical Co. Ltd., reported positive top-line data from the ongoing Visionary phase III study of sibeprenlimab, an anti-APRIL monoclonal antibody for immunoglobulin A nephropathy (IgAN).
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3D illustration of kidney cross section

Otsuka says IgAN, too, with phase III sibeprenlimab data

Oct. 25, 2024
By Marian (YoonJee) Chu
Visterra Inc., a subsidiary of Otsuka Pharmaceutical Co. Ltd., reported positive top-line data from the ongoing Visionary phase III study of sibeprenlimab, an anti-APRIL monoclonal antibody for immunoglobulin A nephropathy (IgAN).
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Immune

Alexion Pharmaceuticals patents complement C1s subcomponent inhibitors

March 18, 2024
An Alexion Pharmaceuticals Inc. patent describes new complement C1s subcomponent (C1S) inhibitors potentially useful for the treatment of immune thrombocytopenia purpura, lupus nephritis, amyotrophic lateral sclerosis, Guillain-Barre syndrome, Huntington’s disease, rheumatoid arthritis, traumatic brain injury, and autoimmune hemolytic anemia, among others.
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3D illustration of heart cross section
Genetic/Congenital

AAV-RBM20 gene therapy corrects splicing in cardiomyopathy model

Oct. 30, 2023
Approximately 2% to 6% of familiar dilated cardiomyopathy (DCM) cases are caused by inherited mutations in the RBM20 gene, which encodes the RNA binding motif 20 (RBM20), a splicing factor that regulates the splicing of several targets involved in the regulation of sarcomeric structure and calcium handling in the myocardium.
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Neurology/Psychiatric

Alexion Pharmaceuticals describes new C1s inhibitors

Oct. 19, 2023
Alexion Pharmaceuticals Inc. has identified complement C1s subcomponent inhibitors reported to be useful for the treatment of immune thrombocytopenia purpura, lupus nephritis, amyotrophic lateral sclerosis, Guillain Barré syndrome, Huntington’s disease, rheumatoid arthritis, traumatic brain injury, and autoimmune hemolytic anemia, among others.
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Twice as nice: With Alexion, Verge cuts another big AI deal

Sep. 8, 2023
By Lee Landenberger
Verge Genomics Inc. has entered a second big AI deal with a large drug company. Privately held Verge will receive up to $42 million, including up-front, equity and near-term payments from Alexion, Astrazeneca Rare Disease, to identify multiple targets for rare neurodegenerative and neuromuscular diseases. The deal could top out at $840 million. There also is potential for downstream royalties.
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Ultomiris

AZ gets CRL asking for REMS changes to rare disease sBLA

Sep. 6, 2023
By Lee Landenberger
While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated.
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