Alnylam Pharmaceuticals, Inc.

Editas Medicine, Inc.

Camp4 Therapeutics Corporation

Other

Curis, Inc.

Genentech, Inc.

Harvard College

Duke University

Arrowhead Pharmaceuticals, Inc.

Imperial Cancer Research Technology, Ltd.

Mayo Foundation for Medical Education and Research

David A Bumcrot

Ivanka Toudjarska

Morgan Lee Maeder

Muthiah Manoharan

Kallanthottathil G Rajeev

Pamela Tan

Jared Gollob

Brian R Bettencourt

Hans-Peter Vornlocher

Dinah Wen-Yee Sah

Alfica Sehgal

Shen Shen

Gregory Hinkle

Birgit Bramlage

Ari E Friedland

Philipp Hadwiger

Ingo Roehl

Andrew P McMahon

Clifford J Tabin

Philip W Ingham

Anke Geick

Michael Stefanidakis

Stuart Milstein

Justin Andrew Caravella

Satyanarayana Kuchimanchi

Charles A Gersbach

Jan Weiler

Bryan R Cullen

Markus Warmuth

Dinah Sah

Dieter Huesken

Akshay Vaishnaw

Jennifer Leah Gori

Frank Peter Stegmeier

Matthew J Farrer

Penrose O'Donnell

Elisa Marti-Gorostiza

G Grant Welstead

Donald L Hertzog

Rina J Mepani

Yun Joon Jung

Subhadeep Roy

Vaishnavi Rajagopal

Alla A Sigova

Greg Hinkle

Christina Gamba-Vitalo

Gordon Grant Welstead

Michael Ray Schlabach, Jr

Deepak Reyon

Igor Zlobine

Brian E Schwartz

Yuichi Nishi

Yuchun Guo

Jacqueline N Robinson-Hamm

Joshua C Tycko

Bryan J Matthews

Yuting Liu

Rachana S Kelkar

Mario Esteban Contreras Gamboa

Rutuja Sudhakar Pai

Demetrius Maraganore

Jack Michael Heath

Nicholas C Huston

CRISPR/CAS-related compositions and methods for treatment of Primary Open Angle Glaucoma (POAG) are disclosed.

Crispr/CAS-related methods and compositions for treating primary open angle glaucoma

Described herein are methods of modulating gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for modulating the levels of gene products, for example, increasing expression of Carbamoyl-Phosphatase Synthetase 1 (CPS1), thereby treating diseases associated with aberrant gene expression.

Modulation of gene transcription using antisense oligonucleotides targeting regulatory RNAs

CRISPR/CAS-related compositions and methods for treatment of Leber's Congenital Amaurosis 10 (LCA10) are disclosed.

CRISPR/Cas-related methods and compositions for treating Leber's congenital amaurosis 10 (LCA10)

The present invention provides phenothiazine compounds, processes for their preparation, pharmaceutical compositions comprising the compounds, and the use of the compounds or the compositions in the treatment of various diseases or conditions, for example ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA).

Phenothiazine derivatives and uses thereof

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.

GNAQ targeted dsRNA compositions and methods for inhibiting expression

CRISPR/CAS-related systems, compositions and methods for editing RS1, RL2, and/or LAT genes in human cells are described, as are cells and compositions including cells edited according to the same.

CRISPR/CAS-related methods and compositions for treating herpes simplex virus

This application relates to therapeutic siRNA agents and methods of making and using the agents.

Therapeutic compositions

Genome editing systems and genetic constructs that target a herpes simplex virus (HSV) viral gene, where the systems comprise one Cas9 molecule, and a gRNA molecule, compositions and cells comprising such genome editing systems and genetic constructs as well as methods for using the genome editing systems, genetic constructs, compositions and cells for genome engineering, and for preventing, treating or reducing HSV infection.

CRISPR/cas-related methods and compositions for treating herpes simplex virus

Compositions and methods for treatment of CEP290 related diseases are disclosed.

Compositions and methods for treating CEP290-associated disease

CRISPR/CAS-related methods and compositions for treating Leber's Congenital Amaurosis 10 (LCA10)

Provided are methods and compositions for the treating a patient with a urea cycle disorder. Methods and compositions are also provided for modulating genes encoding enzymes that participate in the urea cycle by altering gene signaling networks.

Methods and compositions for treating urea cycle disorders

CRISPR/CAS-related compositions and methods for treatment of Sickle Cell Disease (SCD) are disclosed.

CRISPR/CAS-related methods and compositions for treating sickle cell disease

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6.

Compositions and methods for inhibiting expression of TMPRSS6 gene

Growing community of inventors

Belmont, MA, United States of America

David A Bumcrot