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BioWorld - Thursday, January 8, 2026
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Immuno-oncology

Antengene's Claudin 18.2 ADC ATG-022 awarded US orphan drug designations for gastric and pancreatic cancer

May 24, 2023
Antengene Corp. Ltd.'s Claudin 18.2 antibody-drug conjugate (ADC) ATG-022 has been granted two orphan drug designations consecutively by the FDA for the treatment of gastric cancer and pancreatic cancer.
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Concept art for adeno-associated viral-based gene therapy.
Ocular

CNGA1-linked retinitis pigmentosa gene therapy safe in preclinical studies

May 24, 2023
LMU Klinikum recently presented data from studies of an intravitreal gene therapy for CNGA1-linked retinitis pigmentosa (CNGA1-RP).
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Liver illustration
Gastrointestinal

Small-molecule AXIN stabilizer from University of Maryland inhibits liver fibrosis

May 24, 2023
Researchers from the University of Maryland presented preclinical data for YA-6060, a novel small molecule showing dual activities of Wnt inhibition and AMPK activation via the mechanism of AXIN stabilization. Since both Wnt/β-catenin and AMPK signaling pathways have been previously shown to play a key role in liver fibrosis, this study aimed to assess the potential of YA-6060 as an antifibrosis agent.
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Concept art for systemic lupus erythematosus R&D
Musculoskeletal

Exo Therapeutics announces TBK1 exosite inhibitor program for autoimmune diseases

May 24, 2023
Exo Therapeutics Inc. has announced its lead program directed against TANK-binding kinase 1 (TBK1) for the treatment of autoimmune diseases. The company is now approaching identification of a development candidate for its lead program, an exosite-targeted compound that selectively reprograms the activity of TBK1 in the STING pathway that drives pathogenic signaling in diseases such as systemic lupus erythematosus, Aicardi-Goutières syndrome and others.
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Cancer

Allosteric TKI HS-10382 shows efficacy in several CML models

May 24, 2023
Chronic myeloid leukemia (CML) is a myeloproliferative neoplasm characterized by a chromosomal translocation that results in the generation of the BCR-ABL1 oncogene, which encodes the chimeric BCR-ABL1 protein with tyrosine kinase activity. Allosteric tyrosine kinase inhibitors (TKIs) represent an improvement of 2-fold in efficacy over standard inhibitors and are better tolerated.
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Zebrafish heart muscle cells.
Cardiovascular

Mechanism controlling heart regeneration in zebrafish revealed

May 24, 2023
By Helen Albert
Research led by the Hubrecht Institute and the University Medical Center Utrecht has uncovered a key molecular mechanism that helps control heart regeneration in zebrafish. A protein known as leucine-rich repeat-containing 10 (LRRC10) acts as a switch to stop uncontrolled proliferation of cardiomyocyte cells when regeneration is complete. As reported in the May 18, 2023, issue of Science, lead author Jeroen Bakkers and colleagues found that the LRRC10 pathway was conserved in mouse and human cells.
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Cardiovascular

JV-001 targets telomeric ends and restores cardiac function in murine models of heart failure

May 23, 2023
There is a need regarding cardiovascular disease and heart failure for a therapy that reverses the progression of ventricular dysfunction. Previous findings have shown that cardiomyocytes during cardiac dysfunction show an accelerated telomere shortening, thus leading to DNA damage.
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UCSFDBS2.jpg
Neurology/Psychiatric

Where does it hurt? For chronic pain, in the orbitofrontal cortex

May 23, 2023
By Anette Breindl
Long-term brain recordings from four patients with chronic pain have led investigators at the University of California at San Francisco to identify brain signals that could serve as biomarkers for each individual patients’ pain. The study, which was published online in Nature Neuroscience on May 22, 2023, demonstrated that “chronic pain can successfully be tracked, can successfully be predicted, in the real world while patients are ... going about their lives,” lead author Prasad Shirvalkar told reporters at a press conference announcing the findings. Shirvalkar is a neurologist at the University of California at San Francisco.
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Other news to note for May 23, 2023

May 23, 2023
Additional early-stage research and drug discovery news in brief, from: Keros Therapeutics, Pieris Pharmaceuticals, Reviva Pharmaceuticals, Revolo Biotherapeutics, Serca Pharmaceuticals, Tune Therapeutics.
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Gastrointestinal

Insilico Medicine identifies new PHD2 inhibitors

May 23, 2023
An Insilico Medicine IP Ltd. patent describes Egl nine homolog 1 (EGLN1; HPH-2; PHD2) inhibitors reported to be useful for the treatment of ulcerative colitis and Crohn’s disease.
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