Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.

The invention relates to C. acnes strains carrying DNA vectors for the production of recombinant C. acnes phages. The invention encompasses a C. acnes producer cell carrying DNA vectors, with a template for recombination with C. acnes phage genome leading to the insertion of a gene of interest, for the production of recombinant phages that can lead to the transgene expression into C. acnes infected by the recombinant phage. The invention encompasses, C. acnes strains containing these vectors, C. acnes recombinant phages and methods of using these recombinant phages.

An object of the present invention is to provide a production method for a synovium-derived mesenchymal stem cell, which makes it possible to obtain a sufficient amount of synovium-derived mesenchymal stem cells from the synovial tissue, a production method for a cell preparation for joint medical treatment using the production method for a synovium-derived mesenchymal stem cell, synovium-derived mesenchymal stem cell, and a cell preparation for a joint medical treatment. According to the present invention, there is provided a production method for a synovium-derived mesenchymal stem cell, which includes treating a synovial tissue with an enzyme for 2 hours or more; and washing a mixture after the enzyme treatment until a residual enzyme concentration in a supernatant is 0.5 ng/mL or less, to obtain a synovium-derived mesenchymal stem cell.

The present disclosure provides compositions and methods of use involving binding proteins, e.g., antibodies and antigen-binding fragments thereof, that bind to the matrix metalloproteinase-9 (MMP9) protein (MMP9 is also known as gelatinase-B), such as where the binding proteins comprise an immunoglobulin (Ig) heavy chain (or functional fragment thereof) and an Ig light chain (or functional fragment thereof).

Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins involved in clotting into the genome of a cell for treating conditions including hemophilias.

Aspects of the disclosure relate to chimeric antigen receptors (CARs) comprising an antigen binding domain (e.g., anti-TSHR), transmembrane domain (e.g., CD28), and a cytoplasmic domain (e.g., CD27, CD-137, etc.) and a safety mechanism comprising an inducible apoptosis trigger. In some aspects, the disclosure relates to use of the CARs in T cells, compositions, kits and methods for the treatment of thyroid cancers.

Disclosed herein are novel variants of clotting factors VII, VIII, and IX and their use, for example, in methods of treating a subject with a clotting disorder, such as hemophilia A or hemophilia B.

Described herein are bispecific antibodies that bind to plasma kallikrein (pKal) and Factor XII and methods of producing and using such bi-specific antibodies for treating diseases or disorders associated with the contact system, e.g., hereditary angioedema or thrombosis.

Described herein are bispecific antibodies that bind to plasma kallikrein (pKal) and Factor XII and methods of producing and using such bi-specific antibodies for treating diseases or disorders associated with the contact system, e.g., hereditary angioedema or thrombosis.

Described herein are RPE cells engineered to secrete a FVII protein, as well as compositions, pharmaceutical preparations, and implantable devices comprising the engineered RPE cells, and methods of making and using the same for treating a patient with hemophilia or FVII deficiency.