A61K38/50

Composition and use of Cas protein inhibitors

Provided are amino acid sequences capable of binding to and inhibiting a Cas protein's ability to bind to a nucleic acid molecule, thereby inhibiting the Cas protein's function in genome editing. Such Cas protein inhibitors, which can be comprised of a major coat protein (G8P), an extracellular region of the G8P (G8P.sub.EX), or a biological equivalent, are useful in improving the specificity of Cas protein-based genome editing procedures.

ANC80 ENCODING SPHINGOLIPID-METABOLIZING PROTEINS FOR MITIGATING DISEASE-INDUCED TISSUE DAMAGE
20220347276 · 2022-11-03 ·

The present disclosure relates generally to the use of sphingolipid-metabolizing proteins to mitigate or minimize tissue damage resulting from injury or from disease, for example, pulmonary arterial hypertension (PAH) when the sphingolipid-metabolizing protein is delivered via expression from an Anc80 vector.

ANC80 ENCODING SPHINGOLIPID-METABOLIZING PROTEINS FOR MITIGATING DISEASE-INDUCED TISSUE DAMAGE
20220347276 · 2022-11-03 ·

The present disclosure relates generally to the use of sphingolipid-metabolizing proteins to mitigate or minimize tissue damage resulting from injury or from disease, for example, pulmonary arterial hypertension (PAH) when the sphingolipid-metabolizing protein is delivered via expression from an Anc80 vector.

Beta-lactamases with improved properties for therapy

This invention relates to, in part, compositions of beta-lactamases and methods of using these enzymes in, for example, gastrointestinal tract (GI tract) disorders such as C. difficile infection (CDI).

Beta-lactamases with improved properties for therapy

This invention relates to, in part, compositions of beta-lactamases and methods of using these enzymes in, for example, gastrointestinal tract (GI tract) disorders such as C. difficile infection (CDI).

INJECTABLE HIGH CONCENTRATION PHARMACEUTICAL FORMULATIONS AND METHODS OF MANUFACTURING AND USE THEREOF

The present invention provides compositions comprising one or more active pharmaceutical ingredients, wherein the compositions are in the form of high solids concentration pastes capable of being injected in relatively low volumes into an animal using standard commercially available syringes. The invention also provides methods of making such compositions, particularly those compositions comprising high molecular weight active ingredients (e.g., antibodies, enzymes and other proteins and peptides) at relatively high therapeutic concentrations in the high solids concentration pastes. The invention further provides methods of using such formulations in treating, preventing and/or ameliorating certain diseases and physical disorders in animals, including humans, in need thereof. The invention also provides kits comprising the formulations of the invention and a suitable syringe, which in some aspects may be pre-loaded or pre-filled with a composition of the invention.

INJECTABLE HIGH CONCENTRATION PHARMACEUTICAL FORMULATIONS AND METHODS OF MANUFACTURING AND USE THEREOF

The present invention provides compositions comprising one or more active pharmaceutical ingredients, wherein the compositions are in the form of high solids concentration pastes capable of being injected in relatively low volumes into an animal using standard commercially available syringes. The invention also provides methods of making such compositions, particularly those compositions comprising high molecular weight active ingredients (e.g., antibodies, enzymes and other proteins and peptides) at relatively high therapeutic concentrations in the high solids concentration pastes. The invention further provides methods of using such formulations in treating, preventing and/or ameliorating certain diseases and physical disorders in animals, including humans, in need thereof. The invention also provides kits comprising the formulations of the invention and a suitable syringe, which in some aspects may be pre-loaded or pre-filled with a composition of the invention.

THERAPEUTIC CONJUGATE
20220331411 · 2022-10-20 ·

The invention provides conjugates comprising L-asparaginase and a water-soluble polymer for use in treating a disease treatable by L-asparagine depletion in a patient that has been previously administered E. coli derived L-asparaginase. The invention also provides methods of treatment, compositions comprising said conjugate, and methods of producing the conjugate.

THERAPEUTIC CONJUGATE
20220331411 · 2022-10-20 ·

The invention provides conjugates comprising L-asparaginase and a water-soluble polymer for use in treating a disease treatable by L-asparagine depletion in a patient that has been previously administered E. coli derived L-asparaginase. The invention also provides methods of treatment, compositions comprising said conjugate, and methods of producing the conjugate.

ANTIMICROBIAL NANOWORMS
20220331262 · 2022-10-20 ·

At least one nanoworm comprises a plurality of alkene units and a plurality of macroCTA polymer units. The macroCTA polymer units include R.sup.1 groups from reversible addition-fragmentation chain-transfer agents. In certain aspects, the R.sup.1 groups of the macroCTA polymer units are functional groups, such as a carboxylic acid, an alkyne, a pyridine, a dopamine, a thiolactone, a biotin, an azide, a peptide sequence, a sugar sequence, a protease, a glycanase, a polymer, other functional groups, and combinations thereof. In certain aspects, the macroCTA polymer units comprise quaternized amines, In certain aspects, the macroCTA polymer units comprise functionalized quaternized amines, such as an alkyl group, a carboxylic acid, an alkyne, a pyridine, a dopamine, a thiolactone, a biotin, an azide, a peptide sequence, a sugar sequence, a protease, a glycanase, a polymer, other functional groups, and combinations thereof. In certain aspects, the coating comprises the at least one nanoworm.