25 patents
Utility
Non-viral Dna Vectors and Uses Thereof for Expressing Fviii Therapeutics
30 Nov 23
The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding FVIII protein.
Debra Klatte, Russell Monds, Luke S. Hamm, Nathaniel Silver, Phillip Samayoa, Douglas Anthony Kerr, Jessica Lynn Keenan
Filed: 16 Sep 21
Utility
Methods for Encapsulating Polynucleotides into Reduced Sizes of Lipid Nanoparticles and Novel Formulation Thereof
12 Oct 23
Provided herein are lipid formulations of reduced size, comprising a lipid and a capsid free, non-viral vector (e.g., ceDNA), and methods of producing said lipid formulations.
Nolan Gallagher, Matthew G. Stanton, Gregory Feinstein
Filed: 16 Jul 21
Utility
Novel Lipids and Nanoparticle Compositions Thereof
25 May 23
Matthew G. Stanton, Birte Nolting
Filed: 26 Mar 21
Utility
Non-viral Dna Vectors and Uses Thereof for Expressing Gaucher Therapeutics
4 May 23
The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding GBA protein.
Douglas Anthony Kerr, Phillip Samayoa, Nathaniel Silver, Luke S. Hamm
Filed: 24 Mar 21
Utility
Non-viral Dna Vectors and Uses Thereof for Expressing Factor IX Therapeutics
4 May 23
The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding FIX protein.
Douglas Anthony Kerr, Phillip Samayoa, Nathaniel Silver, Sebastian Aguirre, Debra Klatte, Eli Hertzel Pasackow
Filed: 24 Mar 21
Utility
Non-viral Dna Vectors and Uses Thereof for Expressing Phenylalanine Hydroxylase (Pah) Therapeutics
26 Jan 23
The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding PAH protein.
Douglas Anthony Kerr, Phillip Samayoa, Nathaniel Silver, Matthew Chiocco
Filed: 13 Mar 20
Utility
Ionizable Lipids and Nanoparticle Compositions Thereof
24 Nov 22
Matthew G. Stanton, Birte Nolting, Gregory Feinstein, Michelle LeBlanc, Jon Edward Chatterton
Filed: 23 Nov 20
Utility
Methods and Compositions for Reducing Gene or Nucleic Acid Therapy-related Immune Responses
15 Sep 22
This disclosure provides methods and pharmaceutical compositions for attenuating immune response in a subject suffering from a genetic disorder and receiving gene or nucleic acid therapy.
Sebastian Aguirre, Ching-Hung Chang, Debra Klatte
Filed: 11 Aug 20
Utility
Gene Editing Using a Modified Closed-ended Dna (Cedna)
15 Sep 22
The application describes ceDNA vectors having linear and continuous structure for gene editing. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a gene editing molecule.
Robert M. Kotin, Douglas Kerr, Phillip Samayoa, Ozan Alkan, Matthew J. Simmons
Filed: 6 Dec 18
Utility
Lipid Nanoparticle Compositions Comprising Closed-ended Dna and Cleavable Lipids and Methods of Use Thereof
8 Sep 22
Provided herein are lipid formulations comprising a lipid and a capsid free, non-viral vector (e.g. ceDNA).
Jie Su, Prudence Yui Tung Li, Debra Klatte, Leah Yu Liu, Matthew James Chiocco, Matthew G. Stanton, Jeff Moffit, Jon Edward Chatterton
Filed: 3 Sep 20
Utility
Compositions and Production of Nicked Closed-ended Dna Vectors
21 Jul 22
The present application discloses methods for synthetic production and cell-free synthesis of DNA vectors, particularly closed-ended linear DNA vectors having one or more gaps (e.g., nicked ceDNA vectors, “neDNA”) and adenoassociated-virus (AAV) vector which is single strand DNA having linear and continuous structure, for delivery and expression of a transgene in the host cell.
Ozan Alkan, Robert Michael Kotin, Douglas Anthony Kerr, Russell Monds, Carolyn Pelletier, Matthew Stanton
Filed: 17 Jul 20
Utility
Synthetic Production of Single-stranded Adeno Associated Viral Dna Vectors
14 Jul 22
The present application discloses methods for synthetic production and cell-free synthesis of single stranded adeno-associated virus (AAV) vectors, for delivery and expression of a transgene in host cells.
Ozan Alkan, Robert Michael Kotin, Douglas Anthony Kerr, Russell Monds, Carolyn Pelletier, Matthew Stanton
Filed: 17 Jul 20
Utility
Closed-ended Dna (Cedna) and Immune Modulating Compounds
16 Jun 22
Provided herein are methods and constructs related to minimizing immune responses using modified dexamethasone compounds when administering a desired transgene in a cell achieved by delivery of the transgene with one or more doses of a ceDNA construct.
Debra Klatte, Matthew Stanton
Filed: 6 Mar 20
Utility
Non-viral Dna Vectors and Uses Thereof for Expressing Fviii Therapeutics
9 Jun 22
The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene encoding FVIII protein.
Douglas Anthony Kerr, Debra Klatte, Phillip Samayoa, Nathaniel Silver
Filed: 13 Mar 20
Utility
Controlled Expression of Transgenes Using Closed-ended Dna (Cedna) Vectors
9 Jun 22
Provided herein are methods and constructs comprising close-ended DNA (ceDNA vectors) for maintaining or sustaining a level of transgene expression at a predetermined level or range for a predefined time, or increasing the level of transgene expression in a cell or a subject, where the transgene expression level can be modulated (e.g., increased) with one or more subsequent administrations (e.g., a re-dose or a booster administration) after an initial priming administration.
Douglas A. Kerr, Matthew G. Stanton, Matt Chiocco, Mark D. Angelino, Robert M. Kotin, Phillip Samayoa
Filed: 21 Feb 19
Utility
Non-active Lipid Nanoparticles with Non-viral, Capsid Free Dna
9 Jun 22
Provided herein are compositions and methods for delivering non-viral, capsid-free DNA vectors (ceDNA) to cytosol of a target cell in subject while reducing or inhibiting an immune response.
Matthew G. Stanton, Matthew Manganiello
Filed: 6 Mar 20
Utility
Modulation of Rep Protein Activity In Closed-ended Dna (Cedna) Production
28 Apr 22
Provided herein are methods for producing DNA vectors comprising incubating a population of cells harboring the vector polynucleotide encoding a heterologous nucleic acid operatively positioned between a first and a second AAV inverted terminal repeat DNA polynucleotide sequence (ITRs), with at least one of the ITRs having nucleotide sequences corresponding to AAV wild type ITR in the presence of only a single species of Rep protein having at least DNA binding and DNA nicking functionality, under conditions effective and for a time sufficient to induce production of the DNA within the cells and harvesting and isolating the resultant DNA with the ITRs from the cells.
Robert Michael Kotin, Anna Ucher, Ara Karl Malakian
Filed: 14 Feb 20
Utility
Closed-ended Dna (Cedna) and Use In Methods of Reducing Gene or Nucleic Acid Therapy Related Immune Response
21 Apr 22
Provided herein are methods and constructs related to minimizing immune responses using inhibitors of the immune response, in particular the innate immune response, when administering a desired transgene in a cell achieved by delivery of the transgene with repeated doses of a ceDNA vector.
Douglas Anthony Kerr, Phillip Samayoa, Robert M. Kotin, Matthew G. Stanton, Ozan Alkan, Matthew Chiocco, Raj Rajendran
Filed: 24 Jan 20
Utility
Non-viral Dna Vectors and Uses Thereof for Antibody and Fusion Protein Production
10 Feb 22
The application describes ceDNA vectors having linear and continuous structure for delivery and expression of a transgene. ceDNA vectors comprise an expression cassette flanked by two ITR sequences, where the expression cassette encodes a transgene.
Ozan ALKAN, Douglas Anthony KERR, Robert Michael KOTIN, Debra KLATTE, Leah LIU, Nathaniel SILVER
Filed: 14 Feb 19
Utility
Modified Closed-ended Dna (Cedna) Comprising Symmetrical Modified Inverted Terminal Repeats
16 Dec 21
Described herein are ceDNA vectors having linear and continuous structure can be produced in high yields and used for effective transfer and expression of a transgene.
Robert Michael Kotin, Ozan Alkan, Annaliese Jones
Filed: 8 Nov 19