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BioWorld - Wednesday, December 24, 2025
Home » Topics » Science » New compound

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Profile view of the human head highlighting the eye
Ocular

Partial epigenetic reprogramming to treat ischemic optic neuropathy

Sep. 12, 2024
By Mar de Miguel
A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates (NHP) models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.
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Illustration of leg anatomy with target around knee
Musculoskeletal

Novel TAK1 inhibitor from Novartis for treating osteoarthritis

Sep. 6, 2024
Novartis AG described the identification and activity of TAK-756, a novel, selective and potent TAK1 inhibitor, as a potential intra-articular therapy for the treatment of osteoarthritis. Previous studies demonstrated that TAK1 appears to be a potential target for controlling inflammation and catabolism through NF-κB and MAPK pathways.
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Art concept for hematologic cancer
Cancer

Identification of highly potent, orally bioavailable BCL6 ligand-directed degraders

Aug. 23, 2024
At the recently concluded ACS Fall meeting, Bristol Myers Squibb Co. reported the discovery of potent orally bioavailable B-cell lymphoma 6 protein (BCL6) ligand-directed degraders (LDDs).
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Woman with blanket on couch blowing her nose with a tissue
Infection

New antirhinoviral compound with broad-spectrum activity described

Aug. 20, 2024
Human rhinoviruses (hRVs) are associated with upper respiratory tract infections such as the common cold, otitis media or sinusitis. In immunocompromised individuals or older people, hRVs can exacerbate existing pulmonary conditions such as asthma or chronic obstructive pulmonary disease.
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Cross section illustration of ion channel in cell membrane
Neurology/psychiatric

Nav1.8 inhibitor MSD-199 has activity in inflammatory, neuropathic pain models

Aug. 7, 2024
Researchers from Merck & Co Inc. have presented preclinical data for the novel Nav1.8 inhibitor MSD-199, being developed for the treatment of inflammatory and neuropathic pain.
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Kidney illustration
Nephrology

META-001-PH gets rare pediatric disease designation

Aug. 6, 2024
Meta Pharmaceuticals Inc. announced that the FDA has granted rare pediatric disease designation to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH), an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body.
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Mark Mitchnick, CEO, Red Queen
Infection

Red Queen targets infections with predatory lipopeptides

Aug. 6, 2024
By Karen Carey
Armed with $55 million in series A funds and a U.S. government contract, Cambridge, Mass.-based Red Queen Therapeutics Inc. launched operations this week, with plans to advance its novel stapled lipopeptide platform, which creates new antiviral therapies that do not rely on the immune system to work.
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Art concept for pain
Neurology/psychiatric

Novel CCR2-targeting pepducin alleviates neuropathic and bone cancer pain

July 29, 2024
Researchers from Universite de Sherbrooke and University of California San Francisco presented the discovery and preclinical characterization of CCR2-targeting pepducin, PP-101.
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3D illustration showing presence of tumor inside prostate gland
Diagnostics

[68Ga]SDTWS-01, a PSMA-targeting radiotracer for the diagnosis of prostate cancer

July 23, 2024
Researchers from Shandong University and affiliated organizations reported the discovery and preclinical characterization of novel 68Ga-labeled PSMA-targeting tracers for the diagnosis of prostate cancer.
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Human colon cancer cells.
Cancer

JD-02 induces cytoprotective autophagy in colorectal cancer models

June 28, 2024
Since heat shock protein 90 (Hsp90) is highly overexpressed in cancer, it is considered both a tumor marker and a therapeutic target for intervention.
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