Homology Medicines, Inc.

Takeda Pharmaceutical Company Limited

City of Hope

Shire Human Genetic Therapies, Inc.

Albert Barnes Seymour

Jason Boke Wright

Serena Nicole Dollive

Seemin Seher Ahmed

Hillard Rubin

James Anthony Mcswiggen

Jaime Michelle Prout

Danielle Lauren Sookiasian

Omar L Francone

Saswati Chatterjee

Laura Jane Smith

Teresa Leah Wright

Madhusudan Natarajan

Kamehameha K Wong, Jr

Paolo G V Martini

Patrick Anthony John Haslett

Diana Lamppu

Thia Baboval St Martin

Marwa BenHajSalah

Deborah A Kinch

Paolo GV Martini

Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions.

Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof

Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof

Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods for using the same to treat diseases associated with reduction of PAH gene function (e.g., PKU). Also provided are nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions.

Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

Adeno-associated virus compositions for PAH gene transfer and methods of use thereof

Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation. The methods generally comprise administering to the subject a therapeutically effective does of a recombinant adeno-associated virus (rAAV) that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell and thereby restore PAH gene function in the subject.

Methods of treating phenylketonuria

The present invention provides, among other things, bi-specific molecules including, but not limited to, antibodies, fynomers, aptamers, fusion proteins, and protein binding domains that bind both CCL2 and LOXL2 and uses thereof, in particular, for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions. In some embodiments, the present invention further provides methods and compositions for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions based on the combination of mono-specific anti-CCL2 and anti-LOXL2 molecules.

Anti-CCL2 and anti-LOXL2 combination therapy for treatment of scleroderma

Growing community of inventors

Westborough, MA, United States of America

Albert Barnes Seymour