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BioWorld - Sunday, April 12, 2026
Home » Topics » Science » New compound

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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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Lab glassware and scientist
Diagnostics

[68Ga]LW-02050, GRPR-targeted PET tracer with low hepatobiliary excretion and reduced pancreas uptake

June 20, 2024
Researchers from BC Cancer Research Institute (Provincial Health Services Authority) and the University of British Columbia have presented the discovery and preclinical characterization of [68Ga]LW-02050, a 68Ga-labeled hydroxamate derivative of SB3.
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Dermatologic

Allosteric TYK2 inhibitor demonstrates efficacy in preclinical models of psoriasis

June 19, 2024
Atomwise Inc. reported the development and preclinical evaluation data of ATMW-DC, a novel orally available allosteric inhibitor of TYK2 developed using Atomwise’s drug discovery platform Atomnet, for the potential treatment of psoriatic inflammation.
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Ribosome as part of an biological cell constructing mRNA molecules
Cancer

RGT-61159, an RNA-targeting small molecule with efficacy in cancer models driven by MYB dysregulation

June 14, 2024
Rgenta Therapeutics Inc. has presented their work on the discovery and development of RGT-61159, a potential first-in-class oral inhibitor of the oncogenic transcription factor c-MYB.
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Thyroid anatomy illustration
Endocrine/metabolic

Orally bioavailable TSHR antagonist shows disease-modifying effects in models of Graves’ disease

June 12, 2024
Septerna Inc. scientists have reported proof-of-concept data on a novel oral small-molecule thyroid-stimulating hormone receptor (TSHR) antagonist, SP-1351, with potential for the treatment of Graves’ disease (GD), including ophthalmic manifestations.
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Infection

JNJ-9117, a potent pan-genotype HEV replication inhibitor with promising antiviral profile

June 12, 2024
At last week’s EASL meeting, Janssen Pharmaceutica NV disclosed the discovery and preclinical evaluation of a novel pan-genotypic hepatitis E virus (HEV) replication inhibitor, JNJ-64779117 (JNJ-9117).
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