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.
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.
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.
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.
Like the federal district court before it, the U.S. Court of Appeals for the Third Circuit said it lacks jurisdiction to rule on the merits of Novo Nordisk A/S’ claim that the CMS violated the Inflation Reduction Act (IRA) when it treated six of the company’s insulin aspart products as one negotiation-eligible single-source drug.
Among severe insulin-deficient diabetes patients, 12 weeks of 100-mg, once-daily dosing of Biomea Fusion Inc.’s icovamenib lowered hemoglobin A1c by 1.8% from placebo at the 52-week timepoint, an increased benefit over and above what was seen at 26 weeks.
Washington University in St. Louis has disclosed new autophagy inducers reported to be useful for the treatment of alpha-1 antitrypsin deficiency, amyotrophic lateral sclerosis, Alzheimer’s, Huntington’s and Parkinson’s disease.
A top-line readout of the 26-week phase IIa Cbeyond trial showed nimacimab, Skye Bioscience Inc.’s peripherally restricted CB1 inhibitor antibody for weight loss, fell short of statistical significance as a monotherapy vs. placebo on the primary endpoint of weight loss, sending the company’s shares down 60%. Skye executives, however, offered a more optimistic outlook for the findings, which they said provide clear direction for moving forward.
A major contributor to obesity-related pathology is inflammation involving a shift in macrophage polarization toward the inflammatory M1 phenotype and accumulation of such macrophages in the liver and adipose tissue. Activation of Toll-like receptor 4 (TLR4) on macrophages leads the downstream adaptor protein MyD88 to promote M1 polarization through altered gene expression mediated by the transcription factor NF-κB.
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