The gut microbiota may be altered in people with depression as a result of treatment. These microorganisms reorganize differently in individuals who respond to therapy. In a multiomics study of antidepressant-naive patients presented at the 2026 World Congress of Neuropsychopharmacology (CINP), scientists from National Taiwan University found that patients who improved after antidepressant treatment maintained a more balanced and functional microbial ecosystem, recovered beneficial metabolites, and displayed blood-based biological signals that aligned with these changes.

Gensci-134 (Genescience Pharmaceuticals Co. Ltd.) is a growth hormone receptor (GHR) agonist fusion protein that mimics the biological activity of growth hormone (GH), does not contain GH sequence and selectively activates human GHR for the treatment of GH deficiency (GHD). It is designed to enable once-every-4-week dosing.

Genescience Pharmaceuticals Co. Ltd. has been developing an SLC6A19 inhibitor – Gensci-144 – for the potential treatment of chronic kidney disease (CKD).

The gut microbiota may be altered in people with depression as a result of treatment. These microorganisms reorganize differently in individuals who respond to therapy. In a multiomics study of antidepressant-naive patients presented at the 2026 World Congress of Neuropsychopharmacology (CINP), scientists from National Taiwan University found that patients who improved after antidepressant treatment maintained a more balanced and functional microbial ecosystem, recovered beneficial metabolites, and displayed blood-based biological signals that aligned with these changes.

At the BIO International Convention, nonprofit and for-profit companies discussed ways to fund orphan drug development, especially for ultra-rare diseases where development costs can be challenging. One of the simplest ways to keep costs down is to skip the discovery phase and use repurposed drugs or rescue shelved therapeutic assets as the Orphan Therapeutics Accelerator has done. The Cambridge, Mass.-based accelerator was founded in 2024 to rescue shelled therapeutic assets for ultrarare diseases.

Graves’ disease is a form of autoimmune hyperthyroidism where the main extrathyroidal feature is thyroid eye disease. Autoantibodies that stimulate thyroid-stimulating hormone receptor (TSHR) are released and orchestrate hormone production in the thyroid tissue and the orbit. Regeneron Pharmaceuticals Inc. has developed REGN-24493, a monoclonal antibody (MAb) that binds TSHR and blocks its activation by those autoantibodies.

Hypoparathyroidism is a rare disorder characterized by low levels of parathyroid hormone (PTH) that lead to low blood calcium levels and hyperphosphatemia. Septerna Inc. is developing a small-molecule PTH1 receptor (PTH1R) agonist, SEP-479, designed to replace PTH and normalize serum calcium.

Amyotrophic lateral sclerosis (ALS)-associated genes provide direct therapeutic targets and reveal pathways that can be used to develop treatments that counteract their harmful molecular effects. Because the underlying causes of most ALS cases remain unknown, identifying disease-associated variants is essential to uncover the mechanisms that drive the disease, as shown at the European Network to Cure ALS (ENCALS) meeting, held in Madrid from June 24 to 26, 2026.

The eighth version of the Prescription Drug User Fee Act (PDUFA) is up for renewal next year, but the bulk of the negotiations between the drug development industry and the U.S. FDA is largely complete. At the BIO International Convention, representatives from the industry and trade organization provided an overview of what was agreed upon during the 127 negotiating sessions that were needed to arrive at the commitment letter, which will be sent to Congress later in 2026.