The present invention provides stable amorphous calcium carbonate (ACC) compositions, and food articles comprising said compositions.

Methods of treating or reducing risk of developing cardiomyopathy or heart failure in a subject, comprising administering to a subject in need thereof a therapeutically effective amount of an inhibitor of NADPH oxidase 4 (Nox4).

This invention provides polypeptides represented by the following formula:

H.sub.2N-A-X—B-A-X—B—HOOC

which are based on HMGB1 as well as compositions comprising, and treatment methods using, such polypeptide.

This invention provides polypeptides represented by the following formula:

H.sub.2N-A-X—B-A-X—B—HOOC

which are based on HMGB1 as well as compositions comprising, and treatment methods using, such polypeptide.

The present invention relates to compounds for the treatment of diseases related to DUX4 expression, such as muscular dystrophies. It also relates to use of such compounds, or to methods of use of such compounds.

An isolated, non-naturally occurring cell-penetrating peptide (CPP) comprising the amino acid sequence:

TABLE-US-00001 [SEQ ID NO: 1] RRSRTARAGRPGRNSSRPSAPR
and sequences which have at least 60% similarity to SEQ ID NO: 1.

The present invention relates to methods for developing engineered T-cells for immunotherapy that are non-alloreactive. The present invention relates to methods for modifying T-cells by inactivating both genes encoding T-cell receptor and an immune checkpoint gene to unleash the potential of the immune response. This method involves the use of specific rare cutting endonucleases, in particular TALE-nucleases (TAL effector endonuclease) and polynucleotides encoding such polypeptides, to precisely target a selection of key genes in T-cells, which are available from donors or from culture of primary cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

The present disclosure relates to: a) solid state forms of hydrobromide salts of Compound 1; b) pharmaceutical compositions comprising one or more solid state forms of hydrobromide salts of Compound 1, and, optionally, a pharmaceutically acceptable carrier; c) methods of treating tumors or cancers by administering one or more solid state forms of hydrobromide salts of Compound 1 to a subject in need thereof; and d) methods for the preparation of solid state forms of Compound 1.