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BioWorld - Saturday, February 14, 2026
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Endocrine/metabolic

Cyclodextrin-based therapy improves outcomes in Krabbe disease models

Feb. 4, 2026
No Comments
Krabbe disease, also called globoid-cell leukodystrophy, is caused by a deficiency of the lysosomal enzyme β-galactosylceramidase (GALC), resulting from pathogenic variants in both copies of the GALC gene. The GALC deficiency leads to increased levels of the primary galactosphingolipid substrates, galactosylceramide (GalCer) and psychosine (galactosylsphingosine, PSY), in the nervous system.
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Art concept for mouse model
Immune

A humanized mouse mimics Fcγ receptor biology in antibody assays

Feb. 4, 2026
By Mar de Miguel
No Comments
Scientists at Ghent University have created a mouse model that incorporates human versions of the receptors that recognize the fragment crystallizable (Fc) region of immunoglobulin G (IgG), one of the most abundant antibodies in the blood and a key mediator of essential immune functions such as cellular activation, pathogen elimination and the regulation of inflammatory responses. These human Fcγ receptors allow the humanized mouse to more accurately reproduce IgG-driven biology, enabling more reliable and safer preclinical assays before evaluating monoclonal antibodies in clinical trials with people.
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Neurology/psychiatric

Shenzhen Hanlin Biomedical Technology reports urolithin A derivatives

Feb. 3, 2026
Shenzhen Hanlin Biomedical Technology Co. Ltd. has identified new urolithin A derivatives. They are reported to be useful for the treatment of neurological disorder, aging, muscle disorder and metabolic diseases.
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Neurology/psychiatric

Nav1.8 blockers revealed in Alicorn Pharmaceutical patent

Feb. 3, 2026
Work at Alicorn Pharmaceutical Co. Ltd. has led to the synthesis of new sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers. They are reported to be useful for the treatment of pain, respiratory and neurological disorders.
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Neurology/psychiatric

Connecta Therapeutics discloses 3-phenoxy-3-phenylpropanamine derivatives

Feb. 3, 2026
Connecta Therapeutics SL has discovered 3-phenoxy-3-phenylpropanamine derivatives described as potentially useful for the treatment of fragile X and Rett syndrome.
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Ocular

Alchemedicine patents CFD inhibitors

Feb. 3, 2026
Alchemedicine Inc. has disclosed complement factor D (CFD) inhibitors described as potentially useful for the treatment of age-related macular degeneration, arthritis, Alzheimer’s disease, multiple sclerosis, ulcerative colitis, diabetic retinopathy, glaucoma and autoimmune diseases.
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Nephrology

University of Antwerp patent divulges ferroptosis inhibitors

Feb. 3, 2026
A group at the University of Antwerp has patented ferroptosis inhibitors. They are reported to be useful for the treatment of acute renal failure, sepsis, ischemia-reperfusion injury and more.
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Interfering RNA (RNAi)
Drug design, drug delivery & technologies

Genentech licenses Sanegene’s RNAi asset in $1.7B deal

Feb. 3, 2026
By Marian (YoonJee) Chu
No Comments
Genentech Inc. is paying $200 million up front and up to $1.5 billion in milestone payments to license one of Suzhou Sanegene Bio Inc.’s RNAi programs. Metabolic and autoimmune-focused Sanegene did not disclose specifics around the licensed candidate, except that it was derived from its LEAD (Ligand and Enhancer Assisted Delivery) platform.
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Illustration of muscle anatomy
Musculoskeletal

S100a8 knockdown mitigates symptoms in Marfan syndrome

Feb. 3, 2026
No Comments
Researchers from the University of São Paulo (Brazil) first proposed using eccentric training as a promising intervention to address musculoskeletal impairments associated with Marfan syndrome. Eccentric training is a form of resistance exercise that focuses on muscle lengthening under load and can induce robust skeletal muscle adaptations, including the attenuation of muscle wasting, promotion of myofiber hypertrophy and stimulation of satellite cell activation and proliferation, as previously demonstrated.
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Radiotherapy of cancer
Cancer

Advancell and 48Hour Discovery partner on 212Pb radiotherapeutic

Feb. 3, 2026
No Comments
Advancell Pty Ltd. has entered into a collaboration and exclusive licensing agreement with 48Hour Discovery Inc. to develop a novel peptide-based lead-212 (212Pb) radiotherapeutic with an initial focus on a gastrointestinal cancer with significant medical need.
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