Bayer Healthcare LLC

Crispr Therapeutics Ag

Ryo Takeuchi

Abraham Scaria

Akiko Noma

Albena Kantardzhieva

Jan Tebbe

Wayne Coco

Moritz Schmidt

Ashish Gupta

Christian Pitzler

Michael Biag Gamalinda

Florian Richter

Christopher J Cheng

Andre Cohnen

Shuying He

Caroline W Reiss

The present application provides materials and methods for treating a patient with autosomal dominant RP, both ex vivo and in vivo; materials and methods for editing a RHO gene in a human cell; and materials and methods for editing a P23H mutation in a RHO gene in a human cell. In addition, the present application provides one or more gRNAs or sgRNAs for editing a RHO gene; one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene. The present application provides a therapeutic for treating a patient with autosomal dominant RP. The present application provides a kit for treating a patient with autosomal dominant RP. In addition, the present application provides a self-inactivating CRISPR-Cas system.

Materials and methods for treatment of autosomal dominant retinitis pigmentosa

Aspects of this invention, inter alia, relate to novel systems for targeting, editing or manipulating DNA in a cell, using novel synthetic RNA-guided nucleases (sRGNs). The sRGNs are derived from wildtype or parental small type II CRISPR Cas9 endonucleases.

RNA-programmable endonuclease systems and uses thereof

The present application provides a CRISPR/Cas system comprising a nuclease segment that encodes a Cas9 nuclease or variant thereof, a guide RNA segment comprising a nucleotide sequence that encodes a gRNA or sgRNA, and a promoter segment comprising a nucleotide sequence that encodes a first promoter comprising one or more tetracycline operator sequence, wherein the gRNA segment is operably linked to the promoter segment. The present application also provides materials and methods for controlling transcriptional expression of guide RNAs and/or post-transcriptional expression of Cas nuclease.

Materials and methods for controlling gene editing

The present application provides materials and methods for treating a patient with autosomal dominant CORD, both ex vivo and in vivo; materials and methods for editing a GUCY2D gene in a human cell; and materials and methods for editing a R838H, R838C, or R838S mutation in a GUCY2D gene in a human cell. The present application also provides one or more gRNAs or sgRNAs for editing a GUCY2D gene; one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene. The present application provides a therapeutic for treating a patient with autosomal dominant CORD. The present application also provides a kit for treating a patient with autosomal dominant CORD. In addition, the present application provides a self-inactivating CRISPR-Cas system.

Materials and methods for treatment of autosomal dominant cone-rod dystrophy

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Cambridge, MA, United States of America

Ryo Takeuchi