A61K31/4365

COMPOSITIONS AND METHODS FOR TREATING NEURODEGENERATIVE, MYODEGENERATIVE, AND LYSOSOMAL STORAGE DISORDERS

Provided herein are compositions and methods for treating or preventing a neurodegenerative disease, a neurodevelop-mental disease, a myodegenerative disease, a prion disease, a lysosomal storage disease or cancer in a subject.

COMPOSITIONS AND METHODS FOR TREATING NEURODEGENERATIVE, MYODEGENERATIVE, AND LYSOSOMAL STORAGE DISORDERS

Provided herein are compositions and methods for treating or preventing a neurodegenerative disease, a neurodevelop-mental disease, a myodegenerative disease, a prion disease, a lysosomal storage disease or cancer in a subject.

MULTI-TARGET TYROSINE KINASE INHIBITOR

The present invention relates to a multi-target tyrosine kinase inhibitor, which is a compound having protein kinase inhibitory activity as shown in formula (I). The compound is metabolized into an active small molecule td32-4 that inhibits tumor growth in vivo, so as to achieve the anti-tumor effect. Compared with td32-4, the compound of the present invention has significantly improved solubility and oral bioavailability, and shows strong anti-tumor activity in animal in vivo experiments. The present invention relates to a synthetic method for the compound of formula (I) and an application thereof in the field of pharmaceuticals.

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MULTI-TARGET TYROSINE KINASE INHIBITOR

The present invention relates to a multi-target tyrosine kinase inhibitor, which is a compound having protein kinase inhibitory activity as shown in formula (I). The compound is metabolized into an active small molecule td32-4 that inhibits tumor growth in vivo, so as to achieve the anti-tumor effect. Compared with td32-4, the compound of the present invention has significantly improved solubility and oral bioavailability, and shows strong anti-tumor activity in animal in vivo experiments. The present invention relates to a synthetic method for the compound of formula (I) and an application thereof in the field of pharmaceuticals.

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PHARMACEUTICAL COMPOSITION FOR PREVENTION OR TREATMENT OF CANCER IN WHICH KRAS MUTATION AND ACTIVATED RON ARE PRESENT

A pharmaceutical composition that is useful preventing or treating cancer with a KRAS mutation and activated RON is disclosed. The pharmaceutical composition may be utilized as an anticancer drug that exhibits an excellent cell death-inducing ability on cancer cells with KRAS G13 mutation or KRAS G12 mutation and a RON mutation or tyrosine-phosphorylated RON (pTyr-RON).

PHARMACEUTICAL COMPOSITION FOR PREVENTION OR TREATMENT OF CANCER IN WHICH KRAS MUTATION AND ACTIVATED RON ARE PRESENT

A pharmaceutical composition that is useful preventing or treating cancer with a KRAS mutation and activated RON is disclosed. The pharmaceutical composition may be utilized as an anticancer drug that exhibits an excellent cell death-inducing ability on cancer cells with KRAS G13 mutation or KRAS G12 mutation and a RON mutation or tyrosine-phosphorylated RON (pTyr-RON).

EPAC1 INHIBITORS FOR THE TREATMENT OF IDIOPATHIC PULMONARY FIBROSIS
20230030517 · 2023-02-02 ·

The present invention relates to the treatment of idiopathic pulmonary fibrosis. Today, there is no cure for IFF. The inventors showed that EPAC1 inhibitors, in particular CE3F4 and AM-001, represent a promising therapeutic strategy for treating patients with pulmonary fibrosis. The present invention thus relates to an EPAC1 inhibitor for use in the treatment and/or prevention of idiopathic pulmonary fibrosis.

EPAC1 INHIBITORS FOR THE TREATMENT OF IDIOPATHIC PULMONARY FIBROSIS
20230030517 · 2023-02-02 ·

The present invention relates to the treatment of idiopathic pulmonary fibrosis. Today, there is no cure for IFF. The inventors showed that EPAC1 inhibitors, in particular CE3F4 and AM-001, represent a promising therapeutic strategy for treating patients with pulmonary fibrosis. The present invention thus relates to an EPAC1 inhibitor for use in the treatment and/or prevention of idiopathic pulmonary fibrosis.

CHEMOTHERAPEUTIC DRUG IMPLANT
20230030886 · 2023-02-02 ·

This invention relates to chemotherapeutic drug implants, and more particularly, to biodegradable chemotherapeutic drug implants comprising irinotecan, or derivatives or pharmaceutically acceptable salts thereof. The present invention also relates to processes for the preparation of these chemotherapeutic drug implants and to their use in therapy, in particular in treating brain tumours.

CHEMOTHERAPEUTIC DRUG IMPLANT
20230030886 · 2023-02-02 ·

This invention relates to chemotherapeutic drug implants, and more particularly, to biodegradable chemotherapeutic drug implants comprising irinotecan, or derivatives or pharmaceutically acceptable salts thereof. The present invention also relates to processes for the preparation of these chemotherapeutic drug implants and to their use in therapy, in particular in treating brain tumours.