α1-Antitrypsin deficiency is a hereditary disorder that affects the liver in children and adults, as well as the lungs in adults. The disease is mostly caused by the Z allele mutation in the SERPINA1 gene, where a glutamic acid amino acid is substituted by lysine (E342K) leading to protein misfolding and aggregation.

Many patients with peripheral nerve diseases do not have a sufficient regenerative response because of genetic inheritance, infections or chronic disease, leading them to lifelong pain and disability.

Genmab A/S, in collaboration with Biontech SE, has presented data on a novel OX40 agonist antibody –Hexabody-OX40 (GEN-1055/BNT-315), developed using Genmab’s proprietary Hexabody platform, which promotes the formation of antibody hexamers upon target binding to cell surfaces.

Lowering intraocular pressure (IOP) is still the only approach for treating glaucoma, in which there is mitochondrial damage in the retinal ganglion cells (RGCs). Activating survival pathways in RGCs was thus hypothesized by Indiana University scientists as a strategy for treating glaucoma independently of IOP modulation.

Researchers from Fate Therapeutics Inc. presented preclinical data for the multiplexed-engineered, off-the-shelf chimeric antigen receptor (CAR) natural killer (NK) cell therapy, FT-522, as a potential therapeutic against autoimmune diseases.

Kate Therapeutics Inc. recently presented data on a novel muscle- and heart-targeted, liver de-targeted development candidate for the treatment of Duchenne muscular dystrophy (DMD) – KT-809.

Vrise Therapeutics Inc. has developed and presented a USP1 inhibitor, VRTX-531, for the potential treatment of BRCA1/2-mutated HDR+ breast cancer.