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In September 2020, Novartis changed the company's name to Novartis Gene Therapies. [13] [14] In 2019, AveXis's first gene therapy drug onasemnogene abeparvovec (Zolgensma) received regulatory approval in the United States [15] [16] and, with a list price of USD 2.125 million per injection, became the most expensive drug in the world. [16]
(Reuters) -Voyager Therapeutics said on Tuesday Swiss-based drugmaker Novartis would pay $100 million upfront as part of a licensing deal to develop gene therapy candidates for genetic disorders.
ZURICH (Reuters) -Swiss drugmaker Novartis said on Tuesday it has bought gene therapy specialist Arctos Medical for an undisclosed amount, aiming to boost its efforts to find treatments for severe ...
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Precision BioSciences, Inc. is a publicly traded American clinical stage gene editing company headquartered in Durham, North Carolina. [3] Founded in 2006, Precision is focused on developing both in vivo and ex vivo gene editing therapies using its proprietary "ARCUS" genome editing platform. [4]
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), [6] [7] a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. [6] It works by providing a new copy of the SMN gene that produces the SMN protein. [6]
She knows new gene therapies may provide long-term relief to some of the 100,000 Americans like her who suffer from sickle cell disease. ... Novartis' Zolgensma for spinal muscular atrophy in ...
This page was last edited on 10 November 2024, at 06:28 (UTC).; Text is available under the Creative Commons Attribution-ShareAlike 4.0 License; additional terms may apply.
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