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PATHOLOGY-RESPONSIVE RECOMBINANT CELLS AND USES THEREOF

Resumen de: US2025290037A1

Modified cells that express and present or secrete at least one therapeutic molecule that can treat or ameliorate a disease of interest such as but not limited to Alzheimer's disease. In the modified cells, expression of the therapeutic molecule is induced when the modified cells are proximate to or in contact with pathology related to the disease of interest. The present disclosure also relates to compositions and kits comprising the disclosed cells. The present disclosure also relates to methods of using the disclosed cells for treating disease.

PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING ALZHEIMER'S DISEASE, COMPRISING NEURAL CREST-DERIVED NASAL TURBINATE STEM CELLS EXPRESSING SSEA3 AND CD105 AS ACTIVE INGREDIENT

Resumen de: WO2025192800A1

The present invention relates to a pharmaceutical composition for preventing or treating Alzheimer's disease, the composition comprising, as an active ingredient, neural crest-derived nasal turbinate stem cells (NTSCs) expressing SSEA3 and CD105. Treatment with the NTSCs expressing SSEA3 and CD105 or with an NTSC cell line including at least a predetermined proportion of the NTSCs was found to result in remarkably good therapeutic activity against Alzheimer's disease. Therefore, the present invention is expected to be effectively used not only as a composition for preventing or treating Alzheimer's disease in which the composition includes, as an active ingredient, NTSCs expressing SSEA3 and CD105 or an NTSC cell line including at least a predetermined proportion of the NTSCs, but also for uses such as screening of NTSC formulations that can be used to treat Alzheimer's disease, or prediction of the therapeutic efficacy thereof.

VARIANT RNAi

Resumen de: US2025290075A1

Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

CRYSTAL FORMS OF TETRAHYDRO-N,N-DIMETHYL-2,2-DIPHENYL-3-FURANMETHANAMINE HYDROCHLORIDE, PROCESSES OF MAKING SUCH FORMS, AND THEIR PHARMACEUTICAL COMPOSITIONS

Resumen de: EP4616852A2

Polymorphic forms of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (ANAVEX2-73) and a metabolite of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanaminehydrochloride (ANAVEX2-73) are disclosed and characterized. Compositions and method for treatment of Alzheimer's disease that includes the polymorphic forms and metabolite of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (ANAVEX2-73).

TREM2 ANTIGEN BINDING PROTEINS AND USES THEREOF

Nº publicación: EP4617286A2 17/09/2025

Solicitante:

AMGEN INC [US]
Amgen Inc

Resumen de: EP4617286A2

The present invention relates to antigen binding proteins, such as monoclonal antibodies, that specifically bind to and activate human triggering receptor expressed on myeloid cells-2 (TREM2) and pharmaceutical compositions comprising such antigen binding proteins. The agonist antigen binding proteins (e.g. antibodies) of the invention are capable of activating TREM2/DAP12 signaling in myeloid cells in the absence of Fe-mediated crosslinking of the antigen binding proteins. Methods of treating or preventing conditions associated with TREM2 loss of function, such as Alzheimer's disease and multiple sclerosis, using the antigen binding proteins are also described.