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Intestine
Gastrointestinal

WTX-921: An IL-10 Indukine designed to minimize systemic toxicity

May 8, 2025
No Comments
Interleukin-10 (IL-10) is an immunoregulatory cytokine produced by a variety of immune cells, including type 1 regulatory T cells and macrophages, as well as nonimmune cells such as epithelial cells and fibroblasts.
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Ocular

ABI-201 confers protection against age-related macular degeneration

May 8, 2025
No Comments
ABI-201 is an engineered AAV.N54 vector that expresses a soluble CD59 variant and a complement 3 (C3) inhibitor fusion protein. ABI-201 was developed by Avirmax Biopharma Inc. to confer photoreceptor protection against retinal damage. The effects of ABI-201 were tested in a sodium iodate-induced retinal damage model in nonhuman primates, a widely used model for testing therapies for age-related macular degeneration (AMD).
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DNA and silhouette
Genetic/congenital

Organoids plus gene editing bring insights into pediatric metabolic disease

May 8, 2025
By Anette Breindl
No Comments
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
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3D illustration of human digestive system
Immuno-oncology

LM-350 shows strong antitumor activity in CDH17-positive models

May 7, 2025
No Comments
Cadherin 17 (CDH17) is a membrane-bound cell adhesion molecule involved in tumor cell proliferation and is selectively overexpressed in several gastrointestinal malignancies, including colorectal cancer, gastric cancer and pancreatic cancer.
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Drug R&D concept image.
Ocular

Surrozen’s SZN-8141 ameliorates retinopathy in murine models

May 7, 2025
No Comments
Recent evidence has suggested that the use of norrin mimetics targeting both frizzled-4 (FZD4) and low-density lipoprotein receptor-related protein 5 (LRP5) may be highly effective at modulating retinal vascular leakage. When combined with an anti-VEGF therapy, it was hypothesized to have an additive benefit potential for treating retinal vascular disorders.
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Immuno-oncology

Beigene’s BGB-A3055 inhibits tumor growth in cancer rodent models

May 7, 2025
No Comments
Beigene Co. Ltd. has presented data on their anti-CCR8 antibody BGB-A3055 as a cancer immunotherapeutic approach.
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Ocular

PBI-671 induces retinal ganglion cell survival in glaucoma models

May 7, 2025
No Comments
Glaucoma is a leading cause of blindness and is characterized by the death of retinal ganglion cells (RGCs). Genetic screening data have identified dual leucine zipper kinase (DLK) and leucine zipper kinase (LZK) as important mediators of RGC death.
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Immuno-oncology

Xenothera details preclinical characterization of XON-9

May 6, 2025
No Comments
Hepatocellular carcinoma (HCC) is the second leading cause of cancer-associated deaths worldwide.
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Cancer

Novel PROTAC shows robust antitumor activity in preclinical models of prostate cancer

May 6, 2025
No Comments
Researchers from Jiangsu Hengrui Pharmaceuticals Co. Ltd. reported the discovery of SHR-3591, an orally bioavailable AR proteolysis targeting chimera (PROTAC) designed to treat prostate tumors.
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Cancer

HSK-46256, a brain-penetrant PARP-1 inhibitor for the treatment of HRD-associated tumors

May 6, 2025
No Comments
PARP inhibitors have been approved for the treatment of several cancers, including ovarian, breast, pancreatic and prostate cancers with BRCA mutations or other homologous recombination repair deficiencies (HRD). However, their therapeutic potential is limited by challenges such as hematologic toxicity and lack of target selectivity.
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