Genprex Inc. and University of Pittsburgh have entered into a new sponsored research agreement (SRA) to study GPX-002, Genprex’s gene therapy for diabetes in animal models of type 1 and type 2 diabetes.

“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”

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.

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.

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.

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.

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.

Syntis Bio Inc. has presented preclinical data regarding their antiobesity compound SYNT-101 in the preclinical setting.

In a newly published study, researchers from the CSIR Institute of Genomics and Integrative Biology and collaborators further explored the potential of Cdk5 targeting as a therapeutic approach for type 2 diabetes and cognitive deterioration.