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BioWorld - Saturday, December 13, 2025
Home » Topics » Disease categories and therapies » Endocrine/metabolic

Endocrine/metabolic
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Black dollar sign on ombre blue background

Series B rounds abound with Kardigan’s $254M, Kailera’s $600M

Oct. 14, 2025
By Karen Carey
No Comments
Two biopharma companies launched within the past year raised top amounts in series B financings for 2025, seeking to advance their therapeutic candidates for cardiovascular diseases and obesity.
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Hand pointing at digital M&A screen

Low frequency sounds good to Biocryst in $700M Astria HAE buy

Oct. 14, 2025
By Randy Osborne
No Comments
Biocryst Pharmaceuticals Inc.’s near-term dilution should “pave the way for entrenchment” in the hereditary angioedema (HAE) space, thanks to the buyout of Astria Therapeutics Inc., RBC analyst Brian Abrahams said.
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Cardiovascular

EDIT-401 lowers LDL cholesterol by modulating LDLR

Oct. 14, 2025
No Comments
When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed by Editas Medicine Inc.
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Illustration of DNA double helixes inside drop of blood
Hematologic

Gene therapy for hemophilia stalls but doesn’t stop at ESGCT

Oct. 13, 2025
By Mar de Miguel
No Comments
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.
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Woman measuring her body fat with caliper
Endocrine/metabolic

Ascletis selects ASC-35 as clinical development candidate

Oct. 13, 2025
No Comments
Ascletis Pharma Inc. has selected ASC-35, a once-monthly, subcutaneously administered GLP-1 receptor (GLP-1R)/GIP receptor (GIPR) dual peptide agonist, as a clinical development candidate. Ascletis expects to submit an IND application to the FDA for ASC-35 for the treatment of obesity in the second quarter of next year.
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Illustration of DNA double helixes inside drop of blood
Hematologic

Gene therapy for hemophilia stalls but doesn’t stop at ESGCT

Oct. 10, 2025
By Mar de Miguel
No Comments
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.
Read More
Endocrine/metabolic

Pfizer patents SLC6A19 inhibitors

Oct. 9, 2025
Pfizer Inc. has identified new indole analogues acting as sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatotic liver disease (NAFLD; MASLD), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism-spectrum disorders, among others.
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Dollar sign droplet above test tube
Endocrine/metabolic

Grant supports Confo’s work in endocrine and metabolic disorders

Oct. 9, 2025
No Comments
Confo Therapeutics NV has secured a 2-year €1 million (US$1.2 million) grant from Flanders Innovation & Entrepreneurship (VLAIO) to advance research and development of ultra-long-acting medicines targeting G protein-coupled receptors (GPCRs), including bi- and multispecific antibody formats for obesity and other metabolic and endocrine disorders.
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Obesity, fat cell research concept image
Endocrine/metabolic

Fractyl Health reports preclinical data on RJVA-002 for obesity

Oct. 8, 2025
No Comments
Fractyl Health Inc. has announced data on RJVA-002, a dual GIP/GLP-1 gene therapy candidate.
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Red CAR T cell on blue blackground
Drug design, drug delivery & technologies

ESGCT 2025: Redefining CAR T cells across cancer and autoimmunity

Oct. 8, 2025
By Mar de Miguel
No Comments
As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.
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