Affinia Therapeutics Inc. has nominated AFTX-201 as a development candidate for the treatment of BAG3 dilated cardiomyopathy. The gene therapy, using Affinia’s cardiotropic capsid, is given as a one-time intravenous injection.

About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.

After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.

Deficiencies in the SLC6A1 gene, encoding the γ-aminobutyric acid transporter GAT-1, are associated with infantile encephalopathy with intellectual disability. Like other neurodevelopmental disorders, SLC6A1-related disorders lack effective treatments; therefore, gene therapy using viral vectors has been proposed as a potential therapeutic strategy.

Arbor Biotechnologies Inc. has gained IND clearance from the FDA for ABO-101, a novel gene editing therapeutic designed to address primary hyperoxaluria type 1 (PH1). A phase I/II study in adult and pediatric patients with PH1 is expected to begin in the first half of 2025.

Vigeneron GmbH has received IND approval from the FDA for VG-801, a novel mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene. IND clearance enables initiation of a phase I/II trial, with a clinical trial application (CTA) submission to the EMA also planned in the coming months.

A gene therapy based on an enhanced lipid nanoparticle (LNP) loaded with vascular endothelial growth factor (VEGF) mRNA could be developed for the treatment of preeclampsia, according to a study in mice in which it alleviated maternal hypertension until the end of gestation and improved fetal health. Preeclampsia is a disease that affects 3% to 5% of pregnant women, who suffer from hypertension and proteinuria, elevated levels of protein in the urine, during pregnancy.

Kriya Therapeutics Inc. and the Universitat Autònoma de Barcelona have published preclinical data for KRIYA-497, a one-time intramuscular gene therapy being developed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).