29 patents
Utility
Chemically Modified Oligonucleotides with Improved Systemic Delivery
23 Mar 23
The disclosure relates, in some aspects, to methods and compositions for targeting chemically-modified double stranded nucleic acid molecules to a site of interest, such as the liver.
James Cardia, Dingxue Yan
Filed: 31 Dec 20
Utility
RNA interference in ocular indications
21 Feb 23
The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
Lyn Libertine, Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia, Pamela A. Pavco
Filed: 12 May 20
Utility
Chemically Modified Oligonucleotides Targeting Bromodomain Containing Protein 4 (BRD4) for Immunotherapy
5 Jan 23
The disclosure relates, in some aspects, to methods and compositions for production of immunomodulatory compositions.
James Cardia, Rolf Kiessling, Jeroen Melief
Filed: 6 Nov 20
Utility
Neutral nanotransporters
26 Jul 22
Neutral lipid formulations for nucleic acid delivery are provided according to the invention.
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Michelle Miller, Karen G. Bulock
Filed: 12 Oct 18
Utility
Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
22 Mar 22
Aspects of the invention relate to methods for treating cancer by administering to a subject in need thereof a therapeutically effective amount of a nucleic acid molecule that is directed against a gene encoding mouse double minute 1 homolog (MDM1), mouse double minute 2 homolog (MDM2), mouse double minute 3 homolog (MDM3), mouse double minute 4 homolog (MDM4) or V-myc myelocytomatosis viral related oncogene (MYCN) for treating cancer.
Michael Byrne, Karen G. Bulock, James Cardia
Filed: 28 Apr 15
Utility
Inhibition of MAP4K4 through RNAi
22 Feb 22
RNAi constructs directed to MAP4K4 that demonstrate unexpectedly high gene silencing activities, and uses thereof are disclosed.
Tod M. Woolf, Joanne Kamens, Anastasia Khvorova, William Salomon
Filed: 2 Jun 15
Utility
Methods for Treating Aging and Skin Disorders Using Nucleic Acids Targeting Tyr or MMP1
11 Nov 21
The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal applications.
Gerard Cauwenbergh
Filed: 15 Jan 21
Utility
Immunotherapy of Cancer
11 Nov 21
Immunogenic modulators and compositions comprising oligonucleotide agents capable of inhibiting suppression of immune response by reducing expression of one or more gene involved with an immune response suppression mechanism.
Alexey Wolfson, Alexey Eliseev, Taisia Shmushkovich
Filed: 15 Jan 21
Utility
Reduced size self-delivering RNAI compounds
14 Sep 21
The present invention relates to methods for in vivo administration of sd-rxRNA molecules.
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
Filed: 7 Feb 19
Utility
Rna Interference In Dermal and Fibrotic Indications
26 Aug 21
The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal and fibrotic applications.
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, Lyn Libertine, James Cardia, Karen G. Bulock
Filed: 15 Jan 21
Utility
Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
1 Jun 21
The present disclosure relates to RNAi constructs with improved cellular uptake characteristics and methods of use of these compounds for silencing expression of long coding RNAs (IncRNAs).
James Cardia, Karen G. Bulock, Joke Hedwig Vandesompele, Gert Van Peer
Filed: 19 Oct 16
Utility
Reduced Size Self-delivering Rnai Compounds
20 May 21
The present invention relates to RNAi constructs with minimal double-stranded regions, and their use in gene silencing.
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
Filed: 21 Jul 20
Utility
Nucleic acid molecules targeting superoxide dismutase 1 (SOD1)
11 May 21
Aspects of the invention relate to methods for treating ALS comprising administering to a subject in need thereof a therapeutically effective amount of a nucleic acid molecule that is directed against a gene encoding superoxide dismutase 1 (SOD1).
James Cardia, Michael Byrne, Karen G. Bulock, Pamela A. Pavco, Lyn Libertine
Filed: 6 Jul 16
Utility
Rna Interference In Ocular Indications
4 Mar 21
The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
Lyn Libertine, Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia, Pamela A. Pavco
Filed: 12 May 20
Utility
Immunotherapy of cancer
2 Mar 21
Immunogenic modulators and compositions comprising oligonucleotide agents capable of inhibiting suppression of immune response by reducing expression of one or more gene involved with an immune suppression mechanism.
Alexey Wolfson, Alexey Eliseev, Taisia Shmushkovich
Filed: 2 Dec 14
Utility
RNA interference in dermal and fibrotic indications
9 Feb 21
The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal and fibrotic applications.
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, Lyn Libertine, James Cardia, Karen G. Bulock
Filed: 12 Mar 18
Utility
Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1
26 Jan 21
The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal applications.
Gerard Cauwenbergh
Filed: 4 Sep 15
Utility
Reduced size self-delivering RNAI compounds
28 Dec 20
The present invention relates to RNAi constructs with minimal double-stranded regions, and their use in gene silencing.
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
Filed: 27 Jun 18
Utility
RNA interference in skin indications
26 Oct 20
The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal applications.
Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, James Cardia
Filed: 25 Feb 18
Utility
Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
19 Oct 20
In one aspect, the disclosure provides methods for treating a neurodegenerative disorder, comprising administering to a subject in need thereof a therapeutically effective amount of a nucleic acid molecule and a therapeutically effective amount of a small molecule that activates NF-κB p65 and increases expression of manganese superoxide dismutase (MnSOD/SOD2) and/or expression of a related protein or enzyme.
Michael Byrne, Antonella Favit-VanPelt
Filed: 5 Jul 16