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BioWorld - Monday, May 18, 2026
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Home » Topics » BioWorld Science, Endocrine/metabolic

BioWorld Science, Endocrine/metabolic
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Endocrine/metabolic

Maze Therapeutics discovers SLC6A19 inhibitors

March 27, 2025
Maze Therapeutics Inc. has disclosed new sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria, metabolic syndrome, hyperphenylalaninemia, tyrosinemia, isovaleric acidemia, propionic acidemia, maple syrup urine disease and chronic kidney disease, among others.
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Endocrine/metabolic

Agios Pharmaceuticals develops PAH mutant stabilizers for phenylketonuria

March 26, 2025
Agios Pharmaceuticals Inc. has patented new 4-pyrazolo[1,5-a]pyridin-2-yl-4,5,6,7-tetrahydro-1h-imidazo[4,5-c]pyridine derivatives acting as phenylalanine hydroxylase (PAH) R408W mutant stabilizers reported to be useful for the treatment of phenylketonuria.
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Tape measure wrapped around scale
Endocrine/metabolic

PL-7737 gets orphan drug status for leptin receptor deficiency

March 26, 2025
Palatin Technologies Inc. has obtained U.S. orphan drug designation for PL-7737 for leptin receptor (LEPR) deficiency, including obesity caused by this condition.
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3D illustration of liver and photomicrograph showing triglyceride fat accumulated in liver cells.
Endocrine/metabolic

ALKBH5 blockade reverses type 2 diabetes and MASLD in mice

March 24, 2025
The liver is a key at maintaining glucose and lipid homeostasis, crucial processes for metabolic health. When these processes are disrupted, a series of metabolic disorders may occur, including type 2 diabetes and metabolic dysfunction-associated steatotic liver disease (MASLD).
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Endocrine/metabolic

Gasherbrum Bio discovers new GLP-1R agonists

March 21, 2025
Gasherbrum Bio Inc. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, schizophrenia, hypertension, obesity, stroke, myocardial infarction and gout, among others.
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Endocrine/metabolic

Agios Pharmaceuticals divulges new PAH stabilizers for phenylketonuria

March 21, 2025
Agios Pharmaceuticals Inc. has synthesized phenylalanine hydroxylase (PAH) R408W mutant stabilizers reported to be useful for the treatment of phenylketonuria.
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Dollar sign droplet above test tube
Immune

Series B financing at Ampersand Biomedicines to advance immuno-inflammation and immuno-oncology programs

March 20, 2025
Ampersand Biomedicines Inc. has secured $65 million in series B funding from investors including Ampersand’s founder, Flagship Pioneering, to support advancement of two lead programs and a new discovery partnership in obesity.
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Scientist, microscope and dropper
Endocrine/metabolic

Prime Medicine announces new α1-antitrypsin deficiency program

March 19, 2025
Prime Medicine Inc. has announced its new preclinical program for the treatment of α1-antitrypsin deficiency (AATD). The program is advancing through its final stages of lead optimization, with an IND and/or clinical trial application (CTA) filing anticipated around the middle of next year.
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Scientists shaking hands in the lab
Endocrine/metabolic

Opko Health, Entera Bio sign agreement for oral GLP-1/glucagon dual agonist peptide

March 18, 2025
Opko Health Inc. and Entera Bio Ltd. have entered into a collaboration and license agreement to advance an oral dual agonist GLP-1/glucagon peptide as a once-daily tablet into the clinic for patients with obesity, metabolic and fibrotic disorders.
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Endocrine/metabolic

Pluvia divulges new pharmacological chaperones for PKU

March 17, 2025
Pluvia AS has synthesized pharmacological chaperones able to stabilize phenylalanine hydroxylase (PAH) reported to be useful for the treatment of hyperphenylalaninemia, particularly phenylketonuria (PKU).
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