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

Endocrine/metabolic
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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.
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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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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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Acquisition puzzle

Biomarin snags potential ‘first-in-disease’ ERT in $270M Inozyme buy

May 16, 2025
By Jennifer Boggs
No Comments
It’s been a big week for Inozyme Pharma Inc. On the heels of a promising interim readout for phase III-stage enzyme replacement therapy (ERT) candidate INZ-701 in ENPP1 deficiency, the firm agreed to be acquired by Biomarin Pharmaceuticals Inc. in a deal valued at about $270 million, putting the rare disease ERT in the hands of an experienced commercial team.
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Children’s Hospital of Philadelphia
ASGCT 2025

First bespoke gene editing therapy treats rare metabolic disease

May 15, 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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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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Illustration of liver with DNA double helixes
Endocrine/metabolic

ASGCT 2025: Gene and cell therapies transform metabolic diseases

May 15, 2025
By Mar de Miguel
No Comments
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
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