Abstract: The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.

Abstract: Disclosed herein are methods of treating a subject having diabetes with a population of cells expressing ISL1 and one or more immunosuppressive reagents, such as an anti-thymocyte globulin binding moiety.

Abstract: Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic ? cell, ? cell, ? cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

Abstract: Provided herein, in some embodiments, are methods and compositions for treatment of subjects with ?-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.

Abstract: Provided herein are methods of producing ? cells and precursors thereof utilizing a Wnt signaling inhibitor or PKC activator, or both. Also provided herein are in vitro cultures comprising said cells, methods of treating a subject with a disease characterized by high blood sugar levels over a prolonged period of time by administering said cells, and devices for encapsulating said cells.

Abstract: The present invention relates to pharmaceutical compositions containing a solid dispersion of N-[2,4-Bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide including formulations of the solid dispersions into powders, granules and mini-tablets, methods for manufacturing and processing the powders, granules and mini-tablets, and methods for treating cystic fibrosis employing the pharmaceutical composition.

Abstract: Macroencapsulation implantation devices and containers for storing a macroencapsulation device disposed within a receptacle of a macroencapsulation implantation device as well as related methods are described. In some embodiments, a macroencapsulation implantation device may include a handle that is configured to be selectively engaged with a receptacle including a recess in which a macroencapsulation device may be disposed. In some embodiments, a container may be configured to maintain a receptacle of a macroencapsulation implantation device, and a macroencapsulation device disposed therein, in a desired pose within an internal volume of the container.

Abstract: The present invention provides compounds of Formula I?, or pharmaceutically acceptable salts thereof, pharmaceutical compositions thereof, and methods of use thereof for treating cellular proliferative disorders (e.g., cancer).

Abstract: The present invention provides devices and methods for delivering a population of cells or a therapeutic agent to a subject in need thereof.

Abstract: Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic ? cell, ? cell, ? cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

Abstract: The present invention provides crystalline forms of a compound of formula (I): Also provided are pharmaceutical compositions that include the provided crystalline forms and methods of using the provided crystalline forms and pharmaceutical compositions for the treatment of cancer.

Abstract: This application describes methods of treating cystic fibrosis or a CFTR mediated disease comprising administering Compound I or a pharmaceutically acceptable salt thereof. The application also describes pharmaceutical compositions comprising Compound I or a pharmaceutically acceptable salt thereof and optionally comprising one or more additional CFTR modulating agents.

Abstract: The present invention relates to compounds useful as inhibitors of ATR protein kinase. The invention also relates to pharmaceutically acceptable compositions comprising the compounds of this invention; methods of treating of various diseases, disorders, and conditions using the compounds of this invention; processes for preparing the compounds of this invention; intermediates for the preparation of the compounds of this invention; and methods of using the compounds in in vitro applications, such as the study of kinases in biological and pathological phenomena; the study of intracellular signal transduction pathways mediated by such kinases; and the comparative evaluation of new kinase inhibitors. The compounds of this invention have formula I: or a pharmaceutically acceptable salt, wherein the variables are as defined herein. Moreover, The compounds of this invention have formula I-A: or a pharmaceutically acceptable salt, wherein the variables are as defined herein.

Abstract: Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic ? cell, ? cell, ? cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

Abstract: Provided herein are, inter alia, compositions and methods for improved production of SC-? cells in vitro. For example, provided are novel formulations and differentiation methods that result in higher cell yields and recoveries, increased numbers and relative percentages of SC-? cells, enhanced stability and shelf-life of SC-? cells, SC-islet clusters with advantageous characteristics such as reduced size and increased uniformity, improved function of the SC-? cells in vitro, and improved viability, function, and reduced immunogenicity after transplantation. The disclosed compositions and methods can be employed in the manufacture of SC-islets for human therapeutic use.

Abstract: Provided are MTSP-1 polypeptides modified to have altered activity and/or specificity so that they cleave a complement protein, such as complement protein C3, to inhibit its activity and thereby inhibit complement activation. The modified MTSP-1 polypeptides that inhibit complement activation can be used for treatment of diseases and conditions in which complement activation plays a role. Such diseases and conditions include inflammatory diseases and diseases with an inflammatory component. Exemplary of these disorders are ischemic and reperfusion disorders, including myocardial infarction and stroke, sepsis, autoimmune diseases, ophthalmic disorders, such as diabetic retinopathies and macular degeneration, including age-related macular degeneration (AMD), and transplanted organ rejection, such as renal delayed graft function (DGF).

Abstract: Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic ? cell, ? cell, ? cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

Abstract: Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exons of the DMD gene are encompassed.

Abstract: The present invention features a compound of formula I: or a pharmaceutically acceptable salt thereof, where R1, R2, R3, W, X, Y, Z, n, o, p, and q are defined herein, for the treatment of CFTR mediated diseases, such as cystic fibrosis. The present invention also features pharmaceutical compositions, method of treating, and kits thereof.

Abstract: Provided herein, in some embodiments, are methods and compositions for treatment of subjects with ?-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.