csl gene therapy


exclude terms. “With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy.”Under the agreement with uniQure, upon closing the transaction CSL Behring will have the exclusive global right to commercialize AMT-061.
Gene therapy is one of CSL's newest areas of research, with the company making its first major play into the space in 2017 by acquiring Calimmune. No patient treated in clinical trials with uniQure’s AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. If AMT-061 lives up to that promise, it could enable CSL to grow its $1 billion a year hemophilia business significantly.Investors have called the wisdom of taking the other side of the deal into question, though. In a , the therapy was able to restore the levels of factor IX to over 40% of normal levels, which is considered sufficient to stop bleeds.We are exceedingly encouraged by the data we’ve seen,Not only has the treatment option demonstrated robust clinically meaningful responses in factor IX activity, but it has also exhibited excellent safety over multiple years of observation.
is a global biotherapeutics leader driven by its promise to save lives.

CSL Behring agreed a deal with uniQure for the exclusive global rights to AMT-061 (etranacogene dezaparvovec), a gene therapy being tested for the treatment of hemophilia B. The company will also be entitled to royalties of up to around 20% on net sales.Hemophilia B is an incurable genetic disorder where the patient lacks a protein called factor IX that is required for blood clotting, resulting in uncontrolled bleeding. With other gene therapy companies such as Audentes Therapeutics and AveXis also landing multibillion dollar buyouts, investors viewed uniQure as next in line.The fact that uniQure ended up offloading one asset, not the whole company, may say something about the lack of potential buyers with a broad enough focus to place value on all its assets. The change in fortunes dialed up uniQure’s value and talk of a takeover.Now, CSL is set to pick up the hemophilia B gene therapy without acquiring the rest of uniQure. for infusion treatments of hemophilia B, so the gene therapy acquisition expands its assets in this indication. The company buys the global rights to a gene therapy currently going through Phase III trials. At CSL, Corporate Responsibility is about conducting our business ethically. Innovation has been in the DNA of CSL since our beginning in 1916 For an acquisition to make sense, you’ve got to be able to value all of that.

New platforms are providing the life sciences industry with an opportunity to improve the efficiency of clinical trials and reduce costs while remaining compliant and reducing risk.Subscribe to FierceBiotech to get industry news and updates delivered to your inbox.CSL to pay $450M to buy uniQure's hemophilia B gene therapy© 2020 Questex LLC. uniQure will receive an upfront cash payment of US$450 million followed by regulatory and commercial sales milestone payments and royalties. Our total dividend for 2020 increased to US$2.02 per share, up 9% by Shares in uniQure fell 12% following news of an agreement to sell its late-phase hemophilia B gene therapy to CSL Behring. For inspiring stories about the promise of biotechnology, visit Vita The company is now bringing that same commitment to gene therapy; its mission is to address unmet patient needs and enable patients to get the very most out of life. Not only has the treatment option demonstrated robust clinically meaningful responses in FIX activity, but it has also exhibited excellent safety over multiple years of observation.

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