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3D cross-section illustration of muscle anatomy
Musculoskeletal

Dyne Therapeutics’ DYNE-302 normalizes muscular pathology in preclinical FSHD

Oct. 11, 2024
Facioscapulohumeral muscular dystrophy (FSHD) is a severe muscle disorder caused by aberrant DUX4 mRNA expression in skeletal muscle. DUX4 activates downstream target transcriptome, known as D4T, leading to myofiber loss and muscle weakness.
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Obesity, fat cell research concept image
Endocrine/metabolic

Lead molecule identified under Ibio and Astralbio’s myostatin program for obesity

Oct. 11, 2024
Ibio Inc. has announced progress under its collaboration with Astralbio Inc. on a joint myostatin program for cardiometabolic disease and obesity. Ibio leveraged its technology to rapidly advance the program from inception to in vitro proof of concept in human muscle cells.
Read More
Illustration of neurons with Lewy bodies
Neurology/psychiatric

Lewy bodies do not feed only on synuclein in Parkinson’s disease

Oct. 11, 2024
By Mar de Miguel
To recreate in the laboratory the formation of Lewy bodies as they would occur in a Parkinson’s patient, two ingredients are required: the protein α-synuclein and the participation of the immune system. The results could prevent the development and progression of this neurodegenerative disorder and help in the search for new therapies.
Read More
Illustration of kidney with DNA structures
Nephrology

Advancing gene therapy for kidney disease, Purespring adds £80M

Oct. 10, 2024
By Nuala Moran
Purespring Therapeutics Ltd. has raised £80 million (US$104.6 million) in a series B, putting it on course to be the first to take a gene therapy for a kidney disease into the clinic. The money enables the company to move the lead program, PS-002, for the treatment of IgA nephropathy to clinical proof of concept and advance programs in other complement-mediated kidney diseases, and in an undisclosed glomerular kidney disease.
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Neurology/psychiatric

Haisco Pharmaceutical patent describes Nav1.8 channel blockers for pain

Oct. 10, 2024
Haisco Pharmaceutical Group Co. Ltd. has disclosed new tetrahydrofuran derivatives acting as sodium channel protein type 10 subunit alpha (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain.
Read More
Nephrology

Ono Pharmaceutical identifies new EP3 receptor agonists

Oct. 10, 2024
Ono Pharmaceutical Co. Ltd. has patented tricyclic prostaglandin E2 receptor EP3 subtype (PTGER3; EP3) agonists potentially useful for the treatment of renal diseases.
Read More
Cancer

PI3Kα (H1047R mutant) inhibitors reported in Reactive Biosciences patent

Oct. 10, 2024
Work at Reactive Biosciences Inc. has led to the discovery of new phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors reported to be useful for the treatment of cancer.
Read More
Cancer

D3 Bio discloses GTPase KRAS (G12D mutant) inhibitors

Oct. 10, 2024
A D3 Bio (Wuxi) Co. Ltd. patent describes heterocyclic-substituted pyrimidopyran compounds acting as GTPase KRAS (G12D mutant) inhibitors reported to be useful for the treatment of cancer.
Read More
Multiple myeloma cells in the bone marrow.
Cancer

Mcl-1 inhibitor KS-18 fights resistance in multiple myeloma models

Oct. 10, 2024
Researchers from Rowan University, Pennsylvania State University, and affiliated organizations presented preclinical data for the selective Mcl-1 inhibitor, KS-18, in models of multiple myeloma (MM).
Read More
Cancer

Chengdu Zeling Biomedical Technology patents NLRP3 inflammasome inhibitors

Oct. 10, 2024
Chengdu Zeling Biomedical Technology Co. Ltd. has identified triazine compounds acting as NLRP3 inflammasome inhibitors.
Read More
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