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Home » Topics » BioWorld Science, Endocrine/metabolic

BioWorld Science, Endocrine/metabolic
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Doctor with illustration of kidneys
Endocrine/metabolic

NEDD4L and CaMKKβ are targets for diabetic kidney disease

June 3, 2024
Previous studies have demonstrated that CaMKKβ/AMPK signaling plays an important role in regulating mitochondrial homeostasis and that down-regulation of CaMKKβ could contribute to the pathogenesis of diabetic kidney disease (DKD).
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Nephrology

WWP2 drives fibrosis in diabetic nephropathy

June 3, 2024
Chronic kidney disease (CKD) during diabetes may manifest several phenotypes, including diabetic nephropathy (DN), nondiabetic renal disease (NDRD) or a mixed form.
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3D rendering of a liposome containing RNA strand
Endocrine/metabolic

KRRO-110 normalizes AAT levels in models of alpha-1 antitrypsin deficiency

May 31, 2024
Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common mutation being the E342K mutation (Z allele) that introduces an amino acid change from glutamic acid (E) coding for M-AAT to a lysine (K) coding for Z-AAT.
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Test tube, dropper, DNA illustration
Endocrine/metabolic

US orphan drug status granted to Hanmi and GC Biopharma candidate for Fabry disease

May 28, 2024
The U.S. FDA has granted orphan drug status to HM-15421 (GC-1134A, LA-GLA) for the treatment of Fabry disease.
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Endocrine/Metabolic

Piezo1 mechano-gated ion channel as therapeutic target for pancreatic insufficiency

May 27, 2024
Researchers from Duke University presented findings from a study that aimed to assess the physiological role of Piezo1, a mechano-gated ion channel that is highly expressed in pancreatic acinar cells, in the exocrine pancreas.
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3D dollar sign
Cancer

Prologue wants to harness individually tiny, collectively giant viral proteome

May 22, 2024
By Lee Landenberger
Prologue Medicines Inc. has launched to develop therapeutics created from the viral proteome, which are proteins produced across all viruses. It’s a newer twist on harnessing the power of the proteins that regulate biology. The company was founded on the premise of peering beyond the human proteome to see if there were viral proteins that could become therapeutics, Theonie Anastassiadis, Prologue’s founding president, told BioWorld.
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Endocrine/metabolic

Sixpeaks Bio emerges with pipeline of weight loss therapies

May 22, 2024
Sixpeaks Bio AG has emerged from stealth with $30 million in series A financing and a pipeline of therapies for healthy weight loss, including a dual-specific antibody targeting activin type IIA and B receptors for preservation of skeletal muscle mass.
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Drug R&D concept image.
Endocrine/metabolic

Intracerebroventricular ERT for MPS IIIA receives IND clearance in US

May 21, 2024
The FDA has cleared an IND application for GC-1130A, a treatment for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) being jointly developed by GC Biopharma Corp. and Novel Pharma Inc.
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Cholesterol plaque in artery
Endocrine/metabolic

CSPC Pharmaceutical’s JMT-202 cleared to enter clinic in China for hypertriglyceridemia

May 17, 2024
CSPC Pharmaceutical Group Ltd.’s anti-βKlotho monoclonal antibody drug JMT-202 has received clearance from China’s National Medical Products Administration (NMPA) to enter clinical trials to lower triglyceride levels in patients with hypertriglyceridemia.
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Art concept for gene therapy research
Genetic/congenital

Decades of studies on gene and cell therapies lead to ASGCT hits

May 16, 2024
By Mar de Miguel
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
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