C12Y301/03016

Drug Delivery Enhancement Agents
20200325173 · 2020-10-15 ·

A peptide comprising a sequence for opening a tight junction of an epithelial surface and optionally a cell penetrating sequence; and related compositions, optionally comprising further pharmaceutical agents; and related methods.

Peptides and Method for Treatment of Cardiac Arrest
20200297813 · 2020-09-24 ·

Modified peptides based on C-terminal PDZ binding domains of PTEN and PHLPP, or PDK1 interacting fragment of PKN2 are described as are methods of using the modified peptides for blocking the activity of PTEN, PHLPP and PKN2 and treating sudden cardiac arrest. A method for guiding treatment of cardiac arrest based on sorbitol or taurine levels is also provided.

Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
10781434 · 2020-09-22 · ·

The present disclosure provides for compositions comprising a chimeric polypeptide comprising a polypeptide effective for treating glycogen storage disease and an internalizing moiety that promotes delivery into cells. In certain embodiments, the polypeptide effective for treating glycogen storage disease is an acid alpha-glucosidase (GAA), a laforin, an amyloglucosidase (AGL), a malin, or an alpha amylase. The present disclosure also provides for methods for decreasing glycogen accumulation in cells or for treating glycogen storage diseases, including Forbes-Cori Disease, Andersen Disease, von Gierke Disease, Pompe Disease, and Lafora Disease, comprising administering the chimeric polypeptide disclosed herein.

METHODS AND COMPOSITIONS FOR TREATMENT OF GLYCOGEN STORAGE DISEASES AND GLYCOGEN METABOLISM DISORDERS
20200224184 · 2020-07-16 ·

The present disclosure provides for compositions comprising a chimeric polypeptide comprising a polypeptide effective for treating glycogen storage disease and an internalizing moiety that promotes delivery into cells. In certain embodiments, the polypeptide effective for treating glycogen storage disease is an acid alpha-glucosidase (GAA), a laforin, an amyloglucosidase (AGL), a malin, or an alpha amylase. The present disclosure also provides for methods for decreasing glycogen accumulation in cells or for treating glycogen storage diseases, including Forbes-Cori Disease, Andersen Disease, von Gierke Disease, Pompe Disease, and Lafora Disease, comprising administering the chimeric polypeptide disclosed herein.

Peptides and method for treatment of cardiac arrest

Modified peptides based on C-terminal PDZ binding domains of PTEN and PHLPP, or PDK1 interacting fragment of PKN2 are described as are methods of using the modified peptides for blocking the activity of PTEN, PHLPP and PKN2 and treating sudden cardiac arrest. A method for guiding treatment of cardiac arrest based on sorbitol or taurine levels is also provided.

Targeted sensitization of non-del(5q) malignant cells

Disclosed are molecules for treating non-del(5q) MDS that mimic allelic deficiency in del5q MDS to sensitize the malignant clones of patient without del(5q). The disclosed molecule contains an inhibitor of Cdc25C, an inhibitor of PP2Ac, or a combination thereof, and a toll like receptor-9 (TLR9) targeting ligand. The molecule can also contain lenalidomide, or an analogue or derivative thereof. Also disclosed is a composition comprising the disclosed molecule in a pharmaceutically acceptable carrier. Also disclosed is a method for treating non-del(5q) meylodysplastic syndrome (MDS) in a subject by administering to the subject a therapeutically effective amount of the disclosed pharmaceutical composition.

THERAPY AND DIAGNOSIS OF DISEASE CHARACTERIZED BY ALTERATIONS IN THE DNA DAMAGE RESPONSE

The present invention relates to at least one modulator of PP2A or at least one modulator of PP2A-like phosphatase or at least one modulator of PP2A and PP2A-like phosphatase or a combination of said modulators for use in the treatment of a disease characterized by an alteration in the DNA damage response. The present invention also relates to a method to identify a subject to be treated with a PP2A modulator comprising detecting in the genome of said patient a mutation in PP2A.

Drug delivery enhancement agents
10662222 · 2020-05-26 · ·

A peptide comprising a sequence for opening a tight junction of an epithelial surface and optionally a cell penetrating sequence; and related compositions, optionally comprising further pharmaceutical agents; and related methods.

Use of recombinant human calcineurin B subunit

Provided is a use of a recombinant human calcineurin B subunit (rhCNB) in the preparation of a medicine for killing and/or inhibiting the gastric cancer.

COMPOSITIONS AND METHODS FOR MODULATING PPP2R1A

Disclosed herein, inter alia, are compositions and methods useful for modulating PPP2R1 A and for the treatment of cancer.