Patents
Application number: 18375206
Abstract: Provided herein are conjugated oligonucleotides that are characterized by efficient and specific eye distribution.
Type: Application
Filed: September 30, 2022
Publication date: May 24, 2024
Inventors: Claudio Punzo, Anastasia Khvorova, Dimas Echeverria Moreno, Annabelle Biscans, Julia F Alterman, Matthew Hassler, Shun-Yun Cheng
Application number: 18375206
Abstract: Provided herein are oligonucleotides (e.g., siRNA) targeting S6K1. Also provided are methods of treating a disease associated with S6K1 expression.
Type: Application
Filed: September 30, 2022
Publication date: July 6, 2024
Inventors: Claudio Punzo, Anastasia Khvorova, Dimas Echeverria Moreno, Annabelle Biscans, Julia F Alterman, Matthew Hassler, Shun-Yun Cheng, Jillian Caiazzi
METHODS AND COMPOSITION FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION
Application number: 17996711
Abstract: Aspects of the disclosure relate to methods and compositions for treatment of certain ocular diseases and disorders, for example age-related macular degeneration (AMD). In some embodiments, the methods comprise administering a subject having AMD one or more therapeutic agents that modulate the mTORCl pathway (or a component thereof). The disclosure is based, in part, on methods for treating AMD in a subject by administering one or more kinase inhibitors, for example one or more serine/threonine kinase inhibitors. In some embodiments, at least one of the serine/threonine kinase inhibitors is a Ribosomal protein S6 kinase beta-1 (S6K1) inhibitor.
Type: Application
Filed: April, 21, 2020
Publication date: July 13, 2023
Inventors: Claudio Punzo, Shun-Yun Cheng
Publication number: 17685457
Abstract: Aspects of the disclosure relate to compositions and methods for expressing anti-Vascular endothelial cell growth factor (VEGF) agent in a cell or subject. In some embodiments, the disclosure provides rAAVs comprising a capsid protein (e.g., AAV2 variants, AAV2/3 hybrid variants, AAV8 variants, etc.), and a transgene encoding an anti-VEGF agent (e.g., KH902) and one or more regulatory sequences. In some embodiments, compositions described herein are useful for treating subjects having diseases associated with angiogenesis or aberrant VEGF activity/signaling.
Type: Application
Filed: November 26, 2019
Publication date: February 16, 2023
Inventors: Guangping Gao, Phillip Tai, Claudio Punzo, Haijiang Lin
METHODS FOR INHIBITING STARVATION OF A CELL
Publication number: 20120232130
Abstract: The present invention is directed to methods for the treatment or prevention of starvation in a cell, e.g., a neuronal cell, and methods for the treatment and prevention of disorders associated therewith by the administration of an agent, e.g., a nucleic acid molecule, which enhances the intracellular generation and/or uptake of glucose, pyruvate, lactate, and/or NADPH.
Type: Application
Filed: April 15, 2010
Publication date: September 13, 2012
Inventors: Constance L. Cepko, Claudio Punzo
Publication number: 20110318424
Abstract: The present invention is directed to the use of modulators of the mammalian target of rapamycine (mTOR) pathway, glucose and/or glucose enhancers for treating retinal disorders and, in particular, for prolonging the viability of cone cells.
Type: Application
Filed: December 3, 2009
Publication date: December 29, 2011
Applicant: PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Inventors: Constance Louise Cepko, Claudio Punzo