Patent classifications
A61P7/06
FORMULATION COMPRISING DAPRODUSTAT
The present disclosure relates to an immediate release tablet of daprodustat having good tensile strength. In other aspects, medical uses of the immediate release tablet and dosage regimens for using the immediate release tablet are disclosed.
MONOMERIC AND OLIGOMERIC COMPOUND EMBODIMENTS AS CONTRACEPTIVES AND THERAPIES AND METHODS OF MAKING AND USING THE SAME
Disclosed herein are monomeric and oligomeric compound embodiments for use as contraceptive agents. Monomeric compound embodiments disclosed herein comprise substituents that facilitate the ability of the compounds to exhibit progestogenic, androgenic, and estrogenic activity, which can prevent or inhibit bone density loss in subjects. Oligomeric compound embodiments disclosed herein provide the ability to control receptor activation and/or treatment by incorporating a tunable linker group which couples steroidal-based compounds to one another or with therapeutic agents and facilitates selective cleavage of the monomeric components of the oligomeric compound.
Prolyl Hydroxylase Inhibitors
The invention described herein relates to certain pyrimidinetrione N-substituted glycine derivatives of formula (I)
##STR00001##
which are antagonists of HIF prolyl hydroxylases and are useful for treating diseases benefiting from the inhibition of this enzyme, anemia being one example.
PYRUVATE KINASE ACTIVATORS FOR USE IN TREATING BLOOD DISORDERS
Described herein are compounds that activate pyruvate kinase, pharmaceutical compositions and methods of use thereof. These compounds are represented by Formula (I) wherein R.sup.1, R.sup.2, R.sup.a, R.sup.b, R.sup.j, R.sup.k, and Q are as defined herein.
##STR00001##
Therapeutic retroviral vectors for gene therapy
Provided are improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. Also provided are improved gene therapy methods for treating hematopoietic-related disorders. Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.
Chemical compounds
- Ariamala Gopalsamy ,
- Shawn Cabral ,
- Agustin Casimiro-Garcia ,
- Ming Zhu Chen ,
- Chulho Choi ,
- Robert Lee Dow ,
- Olugbeminiyi Omezia Fadeyi ,
- David Hepworth ,
- Jayasankar Jasti ,
- Lyn Howard Jones ,
- Arjun Venkat Narayanan ,
- Mihir Dineshkumar Parikh ,
- David Walter Piotrowski ,
- Lee Richard Roberts ,
- Ralph Pelton Robinson, Jr. ,
- Hatice Gizem Yayla
The invention relates to pyrazole derivatives, to their use in medicine, to compositions containing them, to processes for their preparation and to intermediates used in such processes. More particularly the invention relates to HbS modulators of formula (I) ##STR00001##
or tautomers thereof, or pharmaceutically acceptable salts of said modulators or tautomers thereof, wherein X, Y, R.sup.2 and R.sup.3 are as defined in the description. HbS modulators are potentially useful in the treatment of a wide range of disorders, including sickle cell disease (SCD).
Chemical compounds
- Ariamala Gopalsamy ,
- Shawn Cabral ,
- Agustin Casimiro-Garcia ,
- Ming Zhu Chen ,
- Chulho Choi ,
- Robert Lee Dow ,
- Olugbeminiyi Omezia Fadeyi ,
- David Hepworth ,
- Jayasankar Jasti ,
- Lyn Howard Jones ,
- Arjun Venkat Narayanan ,
- Mihir Dineshkumar Parikh ,
- David Walter Piotrowski ,
- Lee Richard Roberts ,
- Ralph Pelton Robinson, Jr. ,
- Hatice Gizem Yayla
The invention relates to pyrazole derivatives, to their use in medicine, to compositions containing them, to processes for their preparation and to intermediates used in such processes. More particularly the invention relates to HbS modulators of formula (I) ##STR00001##
or tautomers thereof, or pharmaceutically acceptable salts of said modulators or tautomers thereof, wherein X, Y, R.sup.2 and R.sup.3 are as defined in the description. HbS modulators are potentially useful in the treatment of a wide range of disorders, including sickle cell disease (SCD).
Recombinant Heme Oxygenase-1 (HO-1) for the Treatment of Sickle Cell Disease
The present invention provides, among other things, methods and compositions for making and using recombinant heme oxygenase for treating sickle cell disease. In some embodiments, recombinant heme oxygenase proteins are truncation variants, or Fc fusion proteins with increased half-life and/or reduced aggregation.
Recombinant Heme Oxygenase-1 (HO-1) for the Treatment of Sickle Cell Disease
The present invention provides, among other things, methods and compositions for making and using recombinant heme oxygenase for treating sickle cell disease. In some embodiments, recombinant heme oxygenase proteins are truncation variants, or Fc fusion proteins with increased half-life and/or reduced aggregation.
METHODS FOR TREATING NEUTROPENIA
The present invention relates to methods of treating patients with neutropenia, such as severe, chronic neutropenia, or a related disorder, in which mavorixafor, or a pharmaceutically acceptable salt thereof, is administered to such patients. In some cases, the methods have the advantage of reducing or eliminating the need for administration of G-CSF, which is frequently associated with severe bone pain.