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BioWorld - Monday, December 22, 2025
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Alpha-galactosidase enzyme
Endocrine/metabolic

GC Biopharma and Hanmi’s once-monthly subcutaneous treatment for Fabry disease gains US IND clearance

Sep. 3, 2024
The FDA has cleared an IND for a phase I/II trial of LA-GLA (GC-1134A, HM-15421), an innovative enzyme replacement therapy for Fabry disease being co-developed by GC Biopharma Corp. and Hanmi Pharmaceutical Co. Ltd.
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3D illustration of acute lymphoblastic leukemia in blood.
Immuno-oncology

Hemogenyx develops improved version of CDX for leukemia

Sep. 3, 2024
Hemogenyx Pharmaceuticals plc has developed a new and improved version of its bispecific antibody CDX for the treatment of relapsed/refractory acute myeloid leukemia, a subset of acute lymphoblastic leukemia, and potentially for conditioning in bone marrow transplantations.
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Heart illustration
Cardiovascular

Ghrelin receptor agonist improves cardiac function in preclinical heart failure

Sep. 3, 2024
At the recent European Society of Cardiology meeting, Anacardio AB and Helsinn Healthcare SA presented data on their oral small-molecule ghrelin receptor agonist and first-in-class calcium sensitizing inotrope, AC-01, in preclinical models of heart failure.
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Cancer

UBR1 as therapeutic target for anaplastic thyroid carcinoma

Sep. 3, 2024
Scientists from Wuhan University and affiliated organizations have published findings from their investigation into the role of Yes-associated protein (YAP), which is a critical effector of the Hippo pathway, in anaplastic thyroid carcinoma.
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Illustration of tau proteins in Alzheimer's disease
Neurology/psychiatric

RING-nanobody degraders clear and prevent aggregation in tauopathy models

Sep. 3, 2024
By Coia Dulsat
Researchers from the UK Dementia Research Institute at the University of Cambridge have found how to prevent and reverse tau aggregation using target-specific nanobodies. The team holds great expertise in the role of TRIM21 in the tau environment since William McEwan, senior author of the study, first discovered TRIM21 and, a bit later, defined its contribution to tau immunotherapy efficacy.
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3D dollar sign
Cancer

Cumulus raising $50M, spinning GPR68 small molecules into GIO

Sep. 2, 2024
By Nuala Moran
Cumulus Oncology Ltd. is in the thick of raising a $50 million series A round as its model of sourcing novel drug targets emerging from academia, shaping them up for clinical development, and spinning them into startups, gathers pace. At the same time, Nodus Oncology Ltd., the first spinout created around an acquired asset, has just reached in vivo proof of concept with its lead DNA damage response inhibitor, and it, too, is looking to raise a series A to take the program through to the end of phase I.
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Gastrointestinal

Roche discovers new CCR6 antagonists

Sep. 2, 2024
Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have described C-C chemokine receptor type 6 (CCR6) antagonists reported to be useful for the treatment of asthma, psoriasis, Crohn’s disease, ulcerative colitis, multiple sclerosis, rheumatoid arthritis and systemic lupus erythematosus.
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Dermatologic

Wellpep presents new peptidomimetic compounds for atopic dermatitis

Sep. 2, 2024
Wellpep Co. Ltd. has divulged peptidomimetic compounds reported to be useful for the treatment of atopic dermatitis.
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Cancer

Latvian Institute of Organic Synthesis describes new ER-α modulators

Sep. 2, 2024
Latvian Institute of Organic Synthesis has identified selenium compounds acting as estrogen receptor α (ER-α; ESR1) modulators reported to be useful for the treatment of cancer.
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Endocrine/metabolic

Purdue Research Foundation divulges new PTPN1/2 degradation inducers

Sep. 2, 2024
Purdue Research Foundation has synthesized proteolysis targeting chimeras (PROTACs) comprising a E3 ubiquitin ligase binding moiety covalently linked to a tyrosine-protein phosphatase non-receptor type 1 (PTPN1; PTP-1B) and 2 (PTPN2; TCPTP) dual targeting moiety reported to be useful for the treatment of cancer, type 2 diabetes and obesity.
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