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BioWorld - Monday, June 16, 2025
Home » Topics » Disease categories and therapies » Endocrine/metabolic

Endocrine/metabolic
Endocrine/metabolic RSS Feed RSS

Woman receiving chemotherapy
Endocrine/metabolic

Kexing’s GB-18 receives IND clearances in China and US for cancer cachexia

May 29, 2025
No Comments
Kexing Biopharm Co. Ltd. has announced IND clearances this month by China’s National Medical Products Administration (NMPA) and the U.S. FDA for GB-18, a biologic product for the treatment of cancer cachexia.
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Endocrine/metabolic

Université de Montréal divulges new compounds for cancer and Menkes disease

May 28, 2025
Université de Montréal has synthesized compounds reported to be useful for the treatment of cancer and Menkes disease.
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Heart and DNA

Houston, a problem: Rocket death in Danon stalls phase II program

May 27, 2025
By Randy Osborne
No Comments
Rocket Pharmaceuticals Inc. CEO Gaurav Shah said his firm is investigating how its gene therapy for Danon disease may have created an “unexpected and paradoxical” effect that led to problems for a phase II patient who ultimately died.
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DNA data illustration
Genetic/congenital

Latus Bio’s LTS-101 is a potential therapeutic approach for Batten disease

May 26, 2025
No Comments
Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.
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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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