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Home » Topics » Disease categories and therapies » Endocrine/metabolic

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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HKEX exterior

China’s Pegbio launches HK$300M Hong Kong IPO

May 20, 2025
By Tamra Sami
No Comments
China’s Pegbio Co. Ltd. launched its IPO on the Hong Kong stock exchange May 19, to raise up to HK$300.82 million (US$38.4 million) to advance visepegenatide (PB-119), its glucagon-like peptide 1 (GLP-1) receptor agonist.
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Children’s Hospital of Philadelphia
ASGCT 2025

First bespoke gene editing therapy treats rare metabolic disease

May 20, 2025
By Anette Breindl
No Comments
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
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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 20, 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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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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HKEX exterior

China’s Pegbio launches HK$300M Hong Kong IPO

May 19, 2025
By Tamra Sami
No Comments
China’s Pegbio Co. Ltd. launched its IPO on the Hong Kong stock exchange May 19, to raise up to HK$300.82 million (US$38.4 million) to advance visepegenatide (PB-119), its glucagon-like peptide 1 (GLP-1) receptor agonist.
Read More
Financial chart, upward arrow

Protagenic and Phytanix merger prompts a stock surge

May 19, 2025
By Lee Landenberger
No Comments
The merger of Protagenic Therapeutics Inc. and Phytanix Bio Inc. combines two different approaches to treating obesity and metabolic issues. The all-stock deal will merge Protagenic’s peptide candidate in IND-enabling development for treating depression, anxiety, posttraumatic stress disorder and additional indications, along with Phytanix’s cannabinoid and cannabinoid-like molecules for bladder pain syndrome and treatment-resistant focal seizures.
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