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Home » Topics » BioWorld Science, Endocrine/metabolic

BioWorld Science, Endocrine/metabolic
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Art concept for mouse model
Drug design, drug delivery & technologies

From mice to big animal models in gene therapy for rare diseases

May 23, 2025
By Mar de Miguel
No Comments
The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.
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Endocrine/metabolic

Chengdu Easton Biopharmaceuticals discovers new MEN1/MLL interaction inhibitors

May 22, 2025
Chengdu Easton Biopharmaceuticals Co. Ltd. has described menin (MEN1)/KMT2A (MLL) interaction inhibitors reported to be useful for the treatment of cancer and diabetes.
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Endocrine/metabolic

Esperion Therapeutics discovers new ATP citrate lyase inhibitors

May 21, 2025
Esperion Therapeutics Inc. has described ATP citrate lyase (ACLY) inhibitors reported to be useful for the treatment of cancer, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), type 2 diabetes, chronic kidney disease, autoimmune disease and inflammatory disorders.
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DNA double helix under a magnifying glass
Drug design, drug delivery & technologies

Base and prime editions to achieve genetic cure

May 21, 2025
By Mar de Miguel
No Comments
Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
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High-density lipoprotein particles and red blood cells
Endocrine/metabolic

Repair Biotechnologies’ REP-0003 designated orphan drug for homozygous familial hypercholesterolemia

May 20, 2025
No Comments
Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
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Illustration of man holding magnifying glass to human body model showing muscle anatomy
Endocrine/metabolic

TAOK1 identified as a potential therapeutic target in cancer cachexia-associated muscle atrophy

May 19, 2025
No Comments
Cancer cachexia is a devastating condition that affects up to 80% of advanced cancer patients and causes approximately 2 million deaths worldwide annually. Cancer cachexia is characterized by uncontrolled weight loss and severe muscle wasting. Despite its significant impact, effective treatments remain elusive.
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Art concept for metabolism
Endocrine/metabolic

Validation of a fatty acid receptor agonist in a fly model of metabolic syndrome

May 19, 2025
No Comments
The search continues for effective treatments against metabolic syndrome and its related complication, diabetes. Current treatments fail in many patients to provide long-term glycemic control or lead to weight loss.
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Man measuring waist
Endocrine/metabolic

Preclinical data for Wave’s INHIBE GalNAc-siRNA in obesity

May 19, 2025
No Comments
At the recent American Society of Gene and Cell Therapy (ASGCT) meeting, Wave Life Sciences Ltd. presented siRNAs designed to suppress expression of the liver gene inhibin subunit β E (INHBE). Human genetic data show that heterozygous INHBE loss-of-function carriers exhibit a healthy metabolic profile.
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Woman at doctors office visit
Women's health

Abcellera gains Canadian clearance for phase I study of ABCL-635 for vasomotor symptoms in menopause

May 15, 2025
No Comments
Abcellera Biologics Inc. has received a no objection letter from Health Canada authorizing its clinical trial application (CTA) for ABCL-635.
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Cardiovascular

VERVE-102: A single-course gene editing therapy for permanent inactivation of PCSK9

May 15, 2025
No Comments
One of the main goals in the prevention of cardiovascular disorders is to maintain low-density lipoprotein cholesterol (LDL-C) at consistently low levels to ensure long-term cardiovascular protection. Investigators at Verve Therapeutics Inc. reported preclinical data on VERVE-102, a GalNAc base editing strategy designed to sustainably inactivate the PCSK9 gene and lower LDL-C in familial hypercholesterolemia.
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