Abstract: The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme. The disclosure also provides a method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid, and a promoter operably linked to a nucleic acid sequence that encodes iduronate-2-sulfatase (I2S), and wherein administering results in an increase in I2S enzymatic activity in the subject.
Abstract: Disclosed are Respiratory Syncytial Virus (RSV) antigens including a recombinant RSV F protein stabilized in a prefusion conformation. Also disclosed are nucleic acids encoding the antigens and methods of producing the antigens. Methods for generating an immune response in a subject are also disclosed. In some embodiments, the method is a method for treating or preventing a RSV infection in a subject by administering a therapeutically effective amount of the antigen to the subject.
Type:
Grant
Filed:
September 11, 2023
Date of Patent:
May 14, 2024
Assignee:
The United States of America, as represented by the Secretary, Department of Health and Human Services
Inventors:
Peter D. Kwong, Barney S. Graham, Jason S. McLellan, Jeffrey Boyington, Lei Chen, Man Chen, Gwo-Yu Chuang, Ivelin Stefanov Georgiev, Jason Gorman, Michael Gordon Joyce, Masaru Kanekiyo, Gilad Ofek, Marie Pancera, Mallika Sastry, Cinque Soto, Sanjay Srivatsan, Guillaume Stewart-Jones, Yongping Yang, Baoshan Zhang, Tongqing Zhou
Abstract: The present invention provides compositions and methods for regulating the specificity and activity of immune effector cells for use in immunotherapy. In one embodiment, the invention provides a type of chimeric antigen receptor (CAR) wherein the CAR is termed a “NKR-CAR” which is a CAR design comprising a component of a receptor naturally found on natural killer (NK) cells. In one embodiment, the NK receptor includes but is not limited to a naturally occurring activating and inhibitory receptor of NK cells known as a killer cell immunoglobulin-like receptor (KIR).
Type:
Grant
Filed:
January 15, 2020
Date of Patent:
May 14, 2024
Assignees:
The Trustees of the University of Pennsylvania, Novartis AG
Inventors:
Gregory Beatty, Boris Engels, Neeraja Idamakanti, Carl H. June, Andreas Loew, Michael C. Milone, Huijuan Song, Enxiu Wang, Qilong Wu
Inventors:
Jody Akana, Molly Anderson, Bartley K. Andre, Shota Aoyagi, Anthony Michael Ashcroft, Marine C. Bataille, Jeremy Bataillou, Abidur Rahman Chowdhury, Clara Geneviève Marine Courtaigne, Markus Diebel, Alexandre B. Girard, Jonathan Gomez Garcia, M. Evans Hankey, Anne-Marie Heck, Moises Hernandez Hernandez, Richard P. Howarth, Jonathan P. Ive, Julian Jaede, Duncan Robert Kerr, Kainoa Kwon-Perez, Nicolas Pedro Lylyk, Peter Russell-Clarke, Benjamin Andrew Shaffer, Clement Tissandier, Eugene Antony Whang