8 patents
Utility
Recombinant Aav Production In Mammalian Cells
4 Nov 21
The present invention includes methods and compositions for the production of high titer recombinant Adeno-Associated Virus (rAAV) in a variety of mammalian cells.
Kyu-Kye Hwang, David R. Knop, James Conway, Barry J. Byrne
Filed: 15 Mar 21
Utility
Codon Optimized Nucleic Acid Encoding a Retinitis Pigmentosa Gtpase Regulator (RPGR)
10 Jun 21
This invention relates generally to a codon optimized nucleic acid encoding a retinitis pigmentosa GTPase regulator (RPGR) protein.
Guo-jie Ye, Jilin Liu
Filed: 9 Sep 20
Utility
Promoters, Expression Cassettes, Vectors, Kits, and Methods for the Treatment of Achromatopsia and Other Diseases
27 May 21
The present invention provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of genetic diseases that affect the cone cells of the retina.
Guo-jie Ye
Filed: 1 Dec 20
Utility
Adeno-associated Virus (Aav) Systems for Treatment of Progranulin Associated Neurodegenerative Diseases or Disorders
20 May 21
The disclosure provides, in part, optimally-modified progranulin (PGRN) cDNA and associated genetic elements for use in recombinant adeno-associated virus (rAAV)-based gene therapy for neurodegenerative disorders characterized by cognitive disruption, behavioral impairment, and deficient lysosomal storage, including familial frontotemporal dementia (FTD), frontotemporal lobar degeneration (FTLD), neuronal ceroid lipofuscinosis (NCL), or Alzheimer's disease (AD).
Juan Li, Tanaz Farivar, Savitri Mandapadi, Steven Pennock, Mark Shearman, Judith Newmark, Adrian Timmers
Filed: 22 Oct 20
Utility
Triple Function Adeno-associated Virus (Aav)vectors for the Treatment of C9ORF72 Associated Diseases
20 May 21
The present disclosure provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of C9ORF72 associated diseases, including ALS and FTD.
Peixin Zhu, Xijia Wang, Steven Pennock, Mark Shearman
Filed: 22 Oct 20
Utility
Formulation Optimization for Viral Particles
29 Apr 21
The present disclosure provides formulations for maintaining viral particles in a stable form.
David R. Knop, Chantelle Gaskin, Yuyu Long
Filed: 5 Jun 20
Utility
Adeno-associated Virus (Aav)vectors for the Treatment of Age-related Macular Degeneration and Other Ocular Diseases and Disorders
29 Apr 21
The present invention provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of age-related macular generation and other genetic diseases that affect the cone cells of the retina.
Mark Shearman, Adrian M Timmers, Judith Newmark, Steven Pennock, Rakshaa Mureli, Chunjuan Song, Lisa Keyes
Filed: 22 Oct 20
Utility
Adeno-associated Virus (Aav) Systems for Treatment of Genetic Hearing Loss
1 Apr 21
Described herein are optimally-modified GJB2 cDNA and associated genetic elements for use in recombinant adeno-associated virus (rAAV)-based gene therapy for the treatment of genetic hearing loss.
Chris Bartolome, Luis David Jaramillo, Adrian Timmers, Steven Pennock, Mark Shearman
Filed: 30 Sep 20
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