Novartis Ag

Editas Medicine, Inc.

Michael Stefanidakis

Joy Gispati Ghosh

Michael Roguska

Barbara Brannetti

Morgan Lee Maeder

Igor Splawski

Ute Jaeger

Yong-In Kim

Christian Carsten Silvester Kunz

Sha-Mei Liao

Leslie Ngozi Anuna Johnson

Rina J Mepani

David A Bumcrot

Shen Shen

Bijan Etemad-Gilbertson

Nicole Haubst

Chad Eric Bigelow

Engin Toksoz

Stephen Hedrick Poor

Nina Moker

Michael Zbigniew Twarog

Nalini Velamur Rangaswamy

Yubin Qiu

Andrew Anh Nguyen

Ganesh Rajan Iyer

Shawn Michael Hanks

Kirk Lee Clark

James Edgar Chastain

Ana Maria Carrion

Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.

Compositions and methods for treating CEP290 associated disease

The present disclosure provides anti-BTC antibodies, methods of producing said antibodies, pharmaceutical compositions comprising said antibodies, and methods of using said antibodies. The present disclosure also provides multi-specific binding molecules, e.g., bispecific antibodies, comprising a BTC binding moiety and an anti-VEGF binding moiety.

Anti-betacellulin antibodies, fragments thereof, and multi-specific binding molecules

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

Compositions and methods for treating CEP290-associated disease

The present invention relates to antibodies or antigen binding fragments thereof that bind to complement Factor P and used thereof as well as combinations of anti-Factor P antibodies with antibodies or antigen binding fragments thereof that bind to complement component 5 (C5).

Nucleic acids encoding anti-Factor P antibodies

Compositions comprising antibodies targeting factor P

Antibodies that bind factor P

Growing community of inventors

Brookline, MA, United States of America

Michael Stefanidakis