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BioWorld - Thursday, July 9, 2026
Home » Topics » Disease categories and therapies » Endocrine/metabolic

Endocrine/metabolic
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Cholesterol plaque in artery
Cardiovascular

Epigenetic editing demonstrates long-lasting control of LDL-C levels in vivo by targeting PCSK9

March 3, 2025
Epigenetic editing is a promising method for gene regulation in vitro and in vivo, allowing precise control of gene expression without altering the DNA sequence, thereby minimizing genotoxic risks.
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Biomarkers

Modulation of miRNAs has therapeutic efficacy in models of fibrosis and HCC

Feb. 27, 2025
Researchers from Medizinische Hochschule Hannover and affiliated organizations reported data from studies aimed to identify non-coding microRNAs (miRNAs) with therapeutic potential against liver fibrosis in hepatocellular carcinoma (HCC). Functional screening of patient-derived primary human hepatic myofibroblasts, followed by in vivo validation in mouse models of fibrosis, were performed in search of antifibrotic miRNAs.
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Gold wireframe handshake

Harbour Biomed newco HBM Alpha pens $395M hyperplasia drug deal

Feb. 26, 2025
By Marian (YoonJee) Chu
HBM Alpha Therapeutics Inc. signed a potential $395 million licensing deal Feb. 26 with an unnamed “business partner” for its endocrine asset, HAT-001, adding another contender to the congenital adrenal hyperplasia space.
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Person testing glucose level
Endocrine/metabolic

Pre-IND meeting guides path forward for ITOL-102 for type 1 diabetes

Feb. 26, 2025
Kadimastem Ltd. and Itolerance Inc. have held a type B pre-IND meeting with the FDA regarding the development of ITOL-102, an investigational biologic for the treatment and potential cure of type 1 diabetes that would not require life-long chronic immune system suppression. It comprises Kadimastem’s allogenic human stem cell-derived pancreatic islets (Isletrx cells) combined with Itolerance’s immunomodulator, ITOL-100.
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New admin toes the Biden line on Rx price negotiations

Feb. 25, 2025
By Mari Serebrov
The Trump administration dashed hopes that it would temper the Medicare price negotiations mandated by the Inflation Reduction Act when it filed the government’s brief in response to Novartis AG’s appeal to the U.S. Court of Appeals for the Third Circuit.
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Nasal spray
Drug design, drug delivery & technologies

Intranasal bacterium for targeted brain delivery

Feb. 25, 2025
In a recent publication in Cell, researchers from the National University of Singapore and collaborators have proposed using commensal bacteria in the nasal cavity as a delivery vector for precision therapy targeting the OE and brain.
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Endocrine/metabolic

PD-1-FABP5 axis regulates metabolism in obesity

Feb. 25, 2025
Resident immune cells, including type 2 innate lymphoid cells (ILC2s), play a key role in adipose tissue homeostasis. Researchers from Sungkyunkwan University and affiliated organizations evaluated the impact of ILC2s on systemic metabolic regulation in obesity.
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Endocrine/metabolic

Nerio Therapeutics divulges new PTPN1 and PTPN2 degradation inducers

Feb. 25, 2025
Nerio Therapeutics Inc. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to a tyrosine-protein phosphatase non-receptor type 1 (PTPN1; PTP-1B) and/or tyrosine-protein phosphatase non-receptor type 2 (PTPN2; TCPTP) targeting moiety through a linker reported to be useful for the treatment of cancer, type 2 diabetes, obesity and metabolic diseases.
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Nasal spray
Drug design, drug delivery & technologies

Intranasal bacterium for targeted brain delivery

Feb. 24, 2025
In a recent publication in Cell, researchers from the National University of Singapore and collaborators have proposed using commensal bacteria in the nasal cavity as a delivery vector for precision therapy targeting the OE and brain.
Read More
3D rendered medical illustration of the pituitary gland
Endocrine/metabolic

Study links higher USP11 levels to increased Cushing’s disease risk in women

Feb. 24, 2025
A recent study published in Acta Neuropathologica Communications sheds light on the gender disparity in the incidence of ACTH-secreting pituitary neuroendocrine tumors (PitNETs), which occur more frequently in females than in males.
Read More
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