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

Endocrine/metabolic
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Test tube, dropper, DNA illustration
Cardiovascular

Mammoth nominates development candidate for FCS and severe hypertriglyceridemia

May 5, 2025
No Comments
Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.
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Illustration showing cross section of skeletal muscle
Musculoskeletal

Lack of NAD does not alter muscle function or accelerate aging

May 5, 2025
By Mar de Miguel
No Comments
Adult skeletal muscle tolerates a lack of the coenzyme nicotinamide adenine dinucleotide (NAD), according to a study led by scientists at the University of Copenhagen. Their results suggest that adverse effects previously associated with congenital NAD depletion in this tissue may be due to impaired muscle development rather than to a deficiency of this molecule.
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Regulus’ rare kidney disease drug draws Novartis in potential $1.7B buyout

April 30, 2025
By Jennifer Boggs
Regulus Therapeutics Inc. CEO Jay Hagan told investors in a January call the company had no interest in “simply out-licensing” rights to lead candidate farabursen, an oligonucleotide targeting autosomal dominant polycystic kidney disease set to start phase III testing on a path to a potential accelerated approval. And now, there’s no need to, as Regulus found a buyer for the whole company in a deal with Novartis AG valued at about $1.7 billion.
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China in red on globe
Chinabio 2025

US capital crunch pushes China biotechs to roundabout financings

April 29, 2025
By Marian (YoonJee) Chu
Tightening of U.S. regulation and capital is leading Chinese biotechs to alternative and new models of financing, ranging from cross-border licensing deals, M&As, the so-called newco model and overseas listings.
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Illustration of double helix
Endocrine/metabolic

Innorna’s mRNA therapy granted US orphan drug designation for Wilson disease

April 28, 2025
Innorna Co. Ltd.’s investigational mRNA therapy, IN-013, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Wilson disease.
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3D illustration of organoid models in a petri dish
Gastrointestinal

Stem cell-derived liver organoids as a model of fatty liver disease

April 28, 2025
Metabolic dysfunction-associated steatotic liver disease (MASLD) is a growing global health concern, with an estimated prevalence of around 25% worldwide. This chronic liver condition is characterized by lipid deposition in the liver, which can lead to inflammation, scarring and even liver cancer if left untreated.
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Illustration of cancer tumor on pancreas
Cancer

PIKfyve enzyme is target to ‘starve’ pancreatic cancer cells

April 25, 2025
By Mar de Miguel
A metabolic vulnerability of pancreatic ductal adenocarcinoma (PDAC) could be used to address this type of cancer that often resists treatments. Scientists at the University of Michigan have discovered that inhibiting the PIKfyve enzyme prevented tumor development and reduced tumor growth by altering the lipid synthesis these cells rely on. The KRAS-MAPK pathway is involved in this process, leading the researchers to suggest that dual inhibitors of PIKfyve and KRAS-MAPK could be an effective therapeutic strategy.
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China in red on globe
Chinabio 2025

US capital crunch pushes China biotechs to roundabout financings

April 24, 2025
By Marian (YoonJee) Chu
Tightening of U.S. regulation and capital is leading Chinese biotechs to alternative and new models of financing, ranging from cross-border licensing deals, M&As, the so-called newco model and overseas listings.
Read More
Obesity, fat cell research concept image
Endocrine/metabolic

SYNT-101 causes weight reduction in obese rodents

April 22, 2025
Syntis Bio Inc. has presented preclinical data regarding their antiobesity compound SYNT-101 in the preclinical setting.
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Person holding pills and glass of water

Post-Pfizer dropout, Lilly Achieves GLP-1 win in phase III

April 17, 2025
By Randy Osborne
Days after Pfizer Inc. pulled the plug on its oral GLP-1 candidate danuglipron, Eli Lilly and Co. aired positive top-line data from the phase III trial called Achieve-1 testing orforglipron vs. placebo in adults with type 2 diabetes and inadequate glycemic control with diet and exercise alone.
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