A61K9/5107

Compositions and methods of treating muscle atrophy and myotonic dystrophy

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.

Compositions and methods of treating muscle atrophy and myotonic dystrophy

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.

Anti-infective formulations

The present disclosure provides pharmaceutical compositions for treating fungal and bacterial infections. The pharmaceutical compositions of the disclosure comprise a cationic surfactant, a chelating agent, and at least one solvent. The pharmaceutical compositions of the disclosure can be used to treat drug-sensitive or multi drug-resistant bacterial or fungal infections.

Use of dianhydrogalactitol and analogs or derivatives thereof in combination with platinum-containing antineoplastic agents to treat non-small-cell carcinoma of the lung and brain metastases

The use of dianhydrogalactitol provides a novel therapeutic modality for the treatment of non-small-cell lung carcinoma (NSCLC) and ovarian cancer, as well as other types of malignancy, including brain metastases of NSCLC. Dianhydrogalactitol acts as an alkylating agent on DNA that creates N.sup.7 methylation. Dianhydrogalactitol is effective in suppressing the growth of cancer stem cells and is active against tumors that are refractory to temozolomide, cisplatin, and tyrosine kinase inhibitors; the drug acts independently of the MGMT repair mechanism. Dianhydrogalactitol can be used together with other anti-neoplastic agents and can possess additive or super-additive effects.

MUCUS PENETRATING PARTICLE COMPOSITIONS AND METHODS OF USE THEREOF ENHANCING IMMUNE RESPONSE
20220370488 · 2022-11-24 ·

Mucus penetrating nanoparticles for inducing, increasing, or enhancing an immune response typically include core of a blend of a biodegradable hydrophobic polymer and a hydrophilic polymer, wherein ≥50% of the biodegradable polymer is conjugated to the hydrophilic polymer, and the hydrophilic polymers forms a coating on the particle. The particles encapsulate a cargo, typically an antigen, adjuvant or other immunomodulator, or a nucleic acid encoding the antigen, or combination thereof. Pharmaceutical compositions including an effective amount of particles to induce an immune response in a subject in need thereof are also provided. Methods of inducing an immune response are also provided, and typically include administering to a subject, preferably via the respiratory tract, the pharmaceutical composition. In some embodiments, the subject has cancer or an infection of the lung.

METABOLITE-BASED POLYMERS, MICROPARTICLES, AND NANOPARTICLES FOR IMMUNOTHERAPY AND METHODS OF TREATING A DISEASE OR DISORDER
20230058160 · 2023-02-23 ·

The present invention provides polymers, microparticles, nanoparticles, and compositions thereof for inducing an immune response and preventing or treating a metabolic inhibition in a subject. The present invention additionally provides kits that find use in the practice of the methods of the invention.

PEPTIDE COMPOUNDS AND PEPTIDE CONJUGATES FOR THE TREATMENT OF CANCER THROUGH RECEPTOR-MEDIATED CHEMOTHERAPY

The present disclosure relates to peptide compounds and conjugate compounds, processes, methods and uses thereof for treating cancer. For example, the compounds can comprise compounds of formula

TABLE-US-00001 X.sub.1X.sub.2X.sub.3X.sub.4X.sub.5GVX.sub.6AKAGVX.sub.7NX.sub.8FKSESY (I) (SEQ ID NO: 1) (X.sub.9).sub.nGVX.sub.10AKAGVX.sub.11NX.sub.12FKSESY (II) (SEQ ID NO: 2) YKX.sub.13LRRX.sub.14APRWDX.sub.15PLRDPALRX.sub.16X.sub.17L (III) (SEQ ID NO: 3) YKX.sub.18LRR(X.sub.19).sub.nPLRDPALRX.sub.20X.sub.21L (IV) (SEQ ID NO: 4) IKLSGGVQAKAGVINMDKSESM (V) (SEQ ID NO: 5) IKLSGGVQAKAGVINMFKSESY (VI) (SEQ ID NO: 6) IKLSGGVQAKAGVINMFKSESYK (VII) (SEQ ID NO: 7) GVQAKAGVINMFKSESY (VIII) (SEQ ID NO: 8) GVRAKAGVRNMFKSESY (IX) (SEQ ID NO: 9) GVRAKAGVRN(Nle)FKSESY (X) (SEQ ID NO: 10) YKSLRRKAPRWDAPLRDPALRQLL (XI) (SEQ ID NO: 11) YKSLRRKAPRWDAYLRDPALRQLL (XII) (SEQ ID NO: 12) YKSLRRKAPRWDAYLRDPALRPLL (XIII) (SEQ ID NO: 13) wherein X.sub.1 to X.sub.21 and n can have various different values and wherein at least one protecting group and/or at least one labelling agent is optionally connected to said peptide compound at an N- and/or C-terminal end.

NANOPARTICLE COMPOSITIONS FOR GENE THERAPY

A nanoparticulate composition comprises a gene editing ribonucleoprotein system complexed within a cationic polymer. The cationic polymer may be a Poly-beta amino ester hyperbranched polymer, especially a 4-branching hyperbranched polymer. The gene editing ribonucleoprotein system may be a CRISPR-Cas9 gene editing system configured to excise a mutation or exon in a gene, replace a mutation in a gene, or produce a knock-down or knock-out of a gene, and in particular configured to excise exon 80 of the COL7A1 gene which codes for the collagen VII protein. Data shows that using

HETEROCLITIC CANCER VACCINES

In some aspects, the present invention provides heteroclitic CALRMUT peptides designed and selected to elicit an immune response to CALRMUT in subjects with JAK2 mutant-negative myeloproliferative neoplasms, nucleic acid molecules encoding such peptides, compositions comprising such peptides or nucleic acid molecules, and various associated compositions and methods.

COMPOSITIONS AND METHODS OF TREATING MUSCLE ATROPHY AND MYOTONIC DYSTROPHY

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.