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.

Lu-AG-22515, also known as velaprumig, is a recombinant fusion protein that targets CD40 ligand (CD40L) and human serum albumin (HSA), thereby blocking the binding between CD40 and CD40L, a known signaling pathway involved in several autoimmune diseases. Researchers at H. Lundbeck A/S evaluated the pharmacokinetic (PK) and pharmacodynamic (PD) profile of Lu-AG-22515 in cynomolgus monkeys, as well as the impact of its inhibitory effect in the preclinical setting of thyroid eye disease (TED).

Vascular-type bleeding disorder (BDVAS) is a rare, autosomal dominant disorder mainly caused by impaired vascular integrity.

Acromegaly is a rare endocrine condition caused by hypersecretion of human growth hormone (GH) that results in elevated circulating levels of GH and insulin-like growth factor-1 (IGF-1). Opko Biologics Ltd. has presented data on the development and characterization of OPKO-8801001, a long-acting human GH receptor (GHR) antagonist with prolonged systemic exposure and pharmacological activity designed for subcutaneous administration.

Researchers at Theramir Ltd. presented preclinical proof-of-concept data for a new extracellular vesicle (EV) platform engineered to deliver miR-885-5p (EVmiR885-5p), targeting leukocyte-specific protein-1 (LCP-1)-mediated cytoskeletal remodeling in triple-negative breast cancer (TNBC) models.

The alternative pathway (AP) of the complement system is crucial for innate immunity and its dysregulation may lead to several diseases, including paroxysmal nocturnal hemoglobinuria (PNH). Alnylam Pharmaceuticals Inc. and Regeneron Pharmaceuticals Inc. recently presented data on ALN-CFB, a siRNA targeting hepatic complement factor B (CFB) that inhibits the production of CFB in the liver.