Abstract: The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and/or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.

Abstract: Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.

Abstract: Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.

Abstract: The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and/or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.

Abstract: Disclosed is a crystalline form of sepiapterin, a method of preparing the crystalline form, pharmaceutical compositions containing the crystalline form, and a method for treating patients with a disease associated with low intracellular BH4 levels or with dysfunction of various BH4 dependent metabolic pathways, which involves administering to the patient an effective amount of the crystalline form.

Abstract: The present invention is directed to an improved method for preparing bisantrene, specifically bisantrene dihydrochloride, for intravenous administration, as well as to preparations of bisantrene dihydrochloride for intravenous administration. The present invention is also directed to methods for treatment of malignancies treatable by administration of bisantrene, which can include administration of additional anti-neoplastic agents, wherein the bisantrene is prepared by a method according to the present invention.

Abstract: Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.

Abstract: Disclosed is a crystalline form of sepiapterin, a method of preparing the crystalline form, pharmaceutical compositions containing the crystalline form, and a method for treating patients with a disease associated with low intracellular BH4 levels or with dysfunction of various BH4 dependent metabolic pathways, which involves administering to the patient an effective amount of the crystalline form.

Abstract: The present invention relates to new pharmaceutical salts and/or co-crystals of sepiapterin which exhibit improved properties. In particular, the invention relates to salts of sepiapterin with improved stability. The invention also relates to pharmaceutical compositions including a pharmaceutically effective amount of one or more salts and/or co-crystals of sepiapterin, as well as methods of treating tetrahydrobiopterin-related disorders including administration of a sepiapterin salt and/or co-crystal of the invention to a subject in need thereof.

Abstract: Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.

Abstract: This invention is directed to nanoparticles for delivery of nucleic acids to target cells of interest for transfection and expression. The nanoparticles typically include a complex of a cationic peptide bound to a protective hydrophilic polymer through a chelator. The nucleic acid is held to the complex by ionic interactions with the cationic peptide. The chelator is adapted to allow release of the hydrophilic polymer in a time frame suitable to facilitate transfection with the nanoparticle at the target cell surface.

Abstract: A gastric tract acid (GTA) compound having the structure of formula I, as well as salts, esters, prodrugs, or labelled derivatives thereof, are provided. Such GTA compounds may be used for determining GTA levels of a sample, for diagnosing a subject as having or being at risk of developing colorectal cancer, or for raising antibodies. Antibodies, or fragments thereof, which specifically bind to the GTA of formula I are described, as well as uses of such antibodies or fragments for determining GTA levels in a sample, or for diagnosing a subject as having or being at risk of developing colorectal cancer. Kits comprising such GTA compounds and/or antibodies are also provided.

Abstract: The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and/or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.

Abstract: Disclosed is a crystalline form of sepiapterin, a method of preparing the crystalline form, pharmaceutical compositions containing the crystalline form, and a method for treating patients with a disease associated with low intracellular BII4 levels or with dysfunction of various BII4 dependent metabolic pathways, which involves administering to the patient an effective amount of the crystalline form.

Abstract: Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.

Abstract: The present invention relates to novel adenine based inhibitors of adenylyl cyclase of the formula: wherein X, L, R1, R2, R5 are those defined herein. Compounds of the present invention are useful to treat cardiovascular diseases. The present invention also relates to a method of preventing heart failure by administering an effective amount of compound according to the invention following vascular injury and reperfusion therapy.

Abstract: The present invention provides compounds useful as inhibitors of Ca2+/calmodulin-dependent protein kinase (CaMKII), compositions thereof, and methods of using the same.

Abstract: The present invention relates to novel adenine based inhibitors of adenylyl cyclase of the formula: wherein X, L, R1, R2, R5 are those defined herein. Compounds of the present invention are useful to treat cardiovascular diseases. The present invention also relates to a method of preventing heart failure by administering an effective amount of compound according to the invention following vascular injury and reperfusion therapy.

Abstract: The present invention relates to C-20 steroid compounds, compositions and methods of use thereof to treat, minimize and/or prevent traumatic brain injury (TBI), including severe TBI, moderate TBI and mild TBI, including concussions.

Abstract: The present invention relates to the synthesis of ent-progesterone and intermediates thereof.