Patent classifications
C12Y304/21021
Short-acting Factor VII polypeptides
Short-acting Factor VII peptides are disclosed. A shortened half-life is desirable for treatment of acute bleeding and similar disorders. Modification of the sialylation and/or glycosylation of Factor VII and variants thereof produced peptides useful in treating conditions of acute bleeding.
CIRCULAR RNA COMPOSITIONS AND METHODS
Disclosed herein are circular RNA s and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.
Tissue factor pathway inhibitor antibodies and uses thereof
The invention relates to antibodies, and antigen-binding fragments thereof, that specifically bind TFPI and inhibit an activity thereof. Such antibodies and fragments are useful for treating bleeding disorders and shortening clotting time.
Nucleic acids encoding clotting factor variants and their use
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.
COMPOSITIONS, DEVICES, AND METHODS FOR FACTOR VII THERAPY
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.
CIRCULAR RNA COMPOSITIONS AND METHODS
Disclosed herein are circular RNA s and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.
CIRCULAR RNA COMPOSITIONS AND METHODS
Disclosed herein are circular RNAs and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.
Method for purifying active GLA-domain coagulation proteins
The invention relates to a method for purifying biologically active GLA-domain coagulation proteins, comprising the following steps: a) bringing a sample that contains one or more GLA-domain coagulation proteins and may contain biologically inactive molecules of GLA-domain protein(s), into contact with an affinity support on which nucleic aptamers that bind specifically to at least one biologically active GLA-domain coagulation protein are immobilized, in order to form complexes between (i) said nucleic aptamers and (ii) said GLA-domain coagulation protein(s), b) releasing the GLA-domain coagulation protein(s) from the complexes formed in step a), and c) recovering said biologically active GLA-domain coagulation protein(s) in a purified form.
FACTOR VII CONJUGATES
The present invention relates to the conjugation of Factor VII polypeptides with heparosan polymers. The resultant conjugates may be used to deliver Factor VII, for example in the treatment or prevention of bleeding disorders.
ANTI-GPIIB/IIIA ANTIBODIES AND USES THEREOF
Antibodies and antibody fragments that specifically bind to glycoprotein IIb/IIIa (GPIIb/IIIa) are disclosed. Chimeric molecules comprising such antibodies or antigen-binding fragments are also disclosed. In addition, methods of using the disclosed antibodies, antibody fragments, and chimeric molecules, e.g., to target agents to platelets and for the treatment or prevention of diseases or disorders are provided.