Crispr Therapeutics Ag

Vertex Pharmaceuticals, Inc.

Chad Albert Cowan

Tirtha Chakraborty

Ante Sven Lundberg

Roman Lvovitch Bogorad

Thao Thi Nguyen

Ami Meda Kabadi

Jeffrey Li

Matthew Hebden Porteus

Michelle I-Ching Lin

Melanie Ruth Allen

Bibhu Prasad Mishra

Matthias Johannes John

Jeffrey William Stebbins

Kirsten Leah Beaudry

Ying Zhang

Elizabeth Jae-Eun Paik

Andrew Kernytsky

Todd Douglass Borland

Gregory Joseph Cost

Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.

Materials and methods for treatment of hereditary haemochromatosis

The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.

Materials and methods for treatment of Duchenne Muscular Dystrophy

The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

Materials and methods for treatment of hemoglobinopathies

The present application provides materials and methods for treating a patient with Glycogen Storage Disease type 1a (GSD1a) both ex vivo and in vivo. In addition, the present application provides materials and methods for modulating the expression, function, and/or activity of the glucose-6-phosphatase, catalytic (G6PC) and/or the glucose-6-phosphatase (G6Pase) protein in a cell by genome editing.

Materials and methods for treatment of glycogen storage disease type 1A

The present application provides materials and methods for treating a patient with Alpha-1 antitrypsin deficiency (AATD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing the SERPINA1 gene in a cell by genome editing.

Materials and methods for treatment of alpha-1 antitrypsin deficiency

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Cambridge, MA, United States of America

Chad Albert Cowan