City of Hope

Homology Medicines, Inc.

Other

Cedars-sinai Medical Center

Saswati Chatterjee

Laura Jane Smith

Kamehameha Wong

Kamehameha K Wong, Jr

Jason Boke Wright

Jeff Lynn Ellsworth

Hillard Rubin

James Anthony Mcswiggen

Cam Mroske

Kamehameha Kay-Min Wong, Jr

Yu-Chong Tai

James A McSwiggen

Prediman Krishan Shah

Yuman Fong

Colin A Cook

Albert Barnes Seymour

Serena Nicole Dollive

Jaime Michelle Prout

Grace Fisher-Adams

Thia Baboval St Martin

Marwa BenHajSalah

Joel Conrad

Stella Kim

Christie Wong

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

A cell cultivation apparatus for cultivating microorganisms and growing cells at high density is provided. The apparatus includes a membrane comprising multiple surface features on a first side of the membrane for cell placement. The surface features comprising one or more compartments within which a cell can be located. The membrane includes a material that is at least partially permeable to gas. A second side of the membrane defines a gas region. The second side of the membrane is separated from the first side of the membrane by the membrane. The apparatus further includes a media region for receiving media. The compartments are configured to at least partially reduce media flow shear forces on one or more cells in the compartments. The surface features may be ridges, protrusions, fins, wells, and/or posts.

System, device and method for production of bioproduct including high density cell respirator (HDCR) for intensified production of adeno-associated viruses (AAV) and cell-based production

Adeno-associated virus (AAV) Clade F vectors or AAV vector variants (relative to AAV9) for precise editing of the genome of a cell and methods and kits thereof are provided. Targeted genome editing using the AAV Clade F vectors or AAV vector variants provided herein occurred at frequencies that were shown to be 1,000 to 100,000 fold more efficient than has previously been reported. Also provided are methods of treating a disease or disorder in a subject by editing the genome of a cell of the subject via transducing the cell with an AAV Clade F vector or AAV vector variant as described herein and further transplanting the transduced cell into the subject to treat the disease or disorder of the subject. Also provided herein are methods of treating a disease or disorder in a subject by in vivo genome editing by directly administering the AAV Clade F vector or AAV vector variant as described herein to the subject.

Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in a cell without co-transducing or co-administering an exogenous nuclease or a nucleotide sequence that encodes an exogenous nuclease. Also provided are methods of using the AAV compositions to correct an F8 gene mutation and/or treat a disease or disorder associated with an F8 gene mutation. Packaging systems for making the adeno-associated virus compositions are also provided.

Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof

Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.

Recombinant adeno-associated vectors for targeted treatment

In some embodiments, an antiviral vector is provided. The antiviral vector includes a replication competent adeno-associated virus (AAV) and an inhibitory expression cassette that includes a nucleotide sequence that encodes an RNAi molecule that inhibits expression of a targeted helper virus (THV) gene. The THV gene may be part of an Adenovirus (Ad) genome, a Human Papillomavirus (HPV) genome, a Human Herpes Virus (HHV) genome, or a Vaccinia virus (VV) genome.

Replication capable rAAV vectors encoding inhibitory siRNA and methods of their use

Growing community of inventors

Altadena, CA, United States of America

Saswati Chatterjee