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Illustration of thymus in relation to lungs and thyroid
Immune

Tolerance Bio and Zipcode partner on thymus-targeting therapeutics

Sep. 9, 2025
No Comments
Tolerance Bio Inc. and Zipcode Bio have established a strategic research and development collaboration aimed at pioneering novel delivery methods for targeted thymus therapeutics.
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Art concept for gene therapy research
Endocrine/metabolic

Remedium and Lilly partner on gene therapies for obesity and diabetes

Sep. 9, 2025
No Comments
Remedium Bio Inc. has entered into a multitarget research and development collaboration with Eli Lilly & Co. to advance gene therapies for type 2 diabetes and obesity using Remedium’s Prometheus dose-adjustable gene therapy platform.
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Human body skeleton with DNA science background art
Genetic/congenital

Vantictumab reverses bone defects in mice with osteopetrosis

Sep. 9, 2025
No Comments
At the American Society for Bone and Mineral Research (ASBMR) in Seattle, researchers from Ashibio Inc. reported preclinical efficacy data on vantictumab, a human monoclonal IgG2 lambda antibody that binds to multiple frizzled (FZD) receptors.
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Microscopic image showing histology of a glioblastoma multiforme
Cancer

Neuronos’ BA-101 designated orphan drug for glioblastoma

Sep. 9, 2025
No Comments
Neuronos Ltd., a subsidiary of Beyond Air Inc., has announced the granting of orphan drug designation by the FDA to BA-101 for the treatment of glioblastoma (GBM). The company is advancing development of BA-101 toward first-in-human studies.
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Neurology/psychiatric

Evaluation of AAV-delivered miRNA therapy for myotonic dystrophy type 1

Sep. 9, 2025
No Comments
Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic disease that causes severe muscle weakness and other debilitating symptoms, such as compromised respiration and cardiac conduction abnormalities. No disease-modifying therapy exists for DM1, so care focuses on managing symptoms like arrhythmia, myotonia, hypertension, cataracts, respiratory issues and sleep disorders.
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Illustration showing steps to business success
Immune

IMU Biosciences' platform reshaping immune system understanding

Sep. 8, 2025
By Shani Alexander
IMU Biosciences Ltd. is working to transform society’s understanding of the immune system. The company’s platform maps the immune system at molecular, cellular and system levels, to unlock new insights into immune-related health and diseases, paving the way for clinical applications that could improve patient outcomes.
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Other news to note for Sept. 8, 2025

Sep. 8, 2025
Additional early-stage research and drug discovery news in brief, from: Clene, Hoth Therapeutics, Skye Biosciences, Umecrine Cognition, Valitor.
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Cancer

University of Texas researchers prepare dual MALT1/BTK inhibitors

Sep. 8, 2025
Investigators at the University of Texas have synthesized new mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) and Bruton tyrosine kinase (BTK) dual-target inhibitors reported to be useful for the treatment of cancer, autoimmune and inflammatory disorders.
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Cancer

Olema Pharmaceuticals patent discloses new ER-α modulators

Sep. 8, 2025
Olema Pharmaceuticals Inc. has patented new estrogen receptor α (ER-α; ESR1) modulators reported to be useful for the treatment of cancer.
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Neurology/psychiatric

SORT inhibitors described in Otsuka Pharmaceutical patent

Sep. 8, 2025
Otsuka Pharmaceutical Co. Ltd. has identified new piperidine- and morpholine-carboxylate compounds acting as sortilin (SORT; NT3; Gp95) antagonists. As such, they are described as potentially useful for the treatment of amyotrophic lateral sclerosis, frontotemporal dementia, Alzheimer’s and Parkinson’s disease.
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