25 patents
Page 2 of 2
Utility
Closed-ended Dna Vectors Obtainable from Cell-free Synthesis and Process for Obtaining Cedna Vectors
11 Mar 21
The application describes methods for synthetic synthesis and cell-free synthesis of DNA vectors, particularly closed-ended DNA vectors (e.g., ceDNA vectors) having linear and continuous structure for delivery and expression of a transgene.
Ozan Alkan, Robert Michael Kotin, Matthew Stanton, Douglas Anthony Kerr, Carolyn Pelletier
Filed: 18 Jan 19
Utility
Lipid Nanoparticle Formulations of Non-viral, Capsid-free Dna Vectors
4 Mar 21
Provided herein are lipid nanoparticle formulations that comprise an ionizable lipid and non-viral, capsid-free DNA vectors with covalently-closed ends.
Robert Michael Kotin, Ozan Alkan, Douglas Anthony Kerr, Ara Karl Malakian, Matthew John Simmons, Matthew G. Stanton, Jie Su, Teresa L. Wright
Filed: 7 Sep 18
Utility
Closed-ended Dna (Cedna) Vectors for Insertion of Transgenes at Genomic Safe Harbors (GSH) In Humans and Murine Genomes
25 Feb 21
The application describes ceDNA vectors having linear and continuous structure for insertion of a transgene into a gene safe harbor (GSH) in a genome, e.g., mammalian genome. ceDNA vectors can comprise at least one ITR sequence, or two ITR sequences, a transgene, and at least one nucleic acid sequence that specifically binds to, or hybridizes to a GSH locus.
Robert M. Kotin
Filed: 1 Mar 19
Utility
Identifying and Characterizing Genomic Safe Harbors (GSH) In Humans and Murine Genomes, and Viral and Non-viral Vector Compositions for Targeted Integration at an Identified GSH Loci
16 Dec 20
The technology described herein relates to methods, compositions and in silico screening approaches for identifying and validating genomic safe harbors (GSHs) in mammalian genomes, including human genomes.
Robert M. Kotin, Evin Hildebrandt
Filed: 28 Feb 19
Utility
Modified Closed-ended Dna (Cedna)
9 Sep 20
CeDNA vectors having linear and continuous structure can be produced in high yields and used for effective transfer and expression of a transgene. ceDNA vectors comprise an expression cassette and two different ITR sequences derived from AAV genomes in a specified order.
Robert Michael Kotin, Ozan Alkan, Annaliese Jones, Douglas Anthony Kerr, Ara Karl Malakian, Matthew John Simmons, Teresa L. Wright
Filed: 6 Sep 18