Australia has a lot to celebrate when it comes to vaccines. The University of Queensland is where Ian Frazer invented the human papillomavirus vaccine Gardasil, and now Australia is projected to be the first in the word to eliminate cervical cancer, Queensland Deputy Premier Steven Miles said during the Ausbiotech 2023 conference held Nov. 1-3 in Brisbane, Australia.

Researchers from Coave Therapeutics SA presented a novel conjugated AAV (coAAV)-GBA1 candidate for the treatment of patients suffering from PD related to GBA1 mutations.

Researchers from Biontech SE and Genmab BV presented the discovery and preclinical evaluation of a novel Duobody-EpCAMx4-1BB (BNT314/GEN1059), a novel Fc-inert immunomodulatory bispecific antibody (bsAb) designed to boost antitumor immune responses through EpCAM-dependent 4-1BB agonistic activity.

Researchers from Centessa Pharmaceuticals plc presented the preclinical characterization of ORX-750, a novel selective orexin receptor 2 (OX2R) agonist aimed to be used for the treatment of narcolepsy.

At the IDWeek 2023 infectious disease conference, Rachelle Koch, a medical student from the University of Texas Southwestern Medical Center, presented the work done in David Greenberg’s Lab on a new strategy to tackle Pseudomonas aeruginosa infections using D-peptide-conjugated phosphorodiamidate morpholino oligomers (D-PPMOs). P. aeruginosa is an opportunistic pathogen showing a multidrug-resistance (MDR) pattern that is at the root of significant morbidity and mortality, especially in immunocompromised patients with severe chronic lung diseases such as cystic fibrosis.

Approximately 2% to 6% of familiar dilated cardiomyopathy (DCM) cases are caused by inherited mutations in the RBM20 gene, which encodes the RNA binding motif 20 (RBM20), a splicing factor that regulates the splicing of several targets involved in the regulation of sarcomeric structure and calcium handling in the myocardium.

Investigators from Biomarin Pharmaceutical Inc. have presented the first preclinical data for BMN-293, a novel adeno-associated virus (AAV) gene transfer vector carrying the MYBPC3 gene. MYBPC3 mutations can cause hypertrophic cardiomyopathy (HCM), a heart medical condition characterized by an abnormally thick myocardium, which makes it more difficult for the heart to pump blood.

Heterozygous familial hypercholesterolemia (HeFH) is caused by mutations in the LDL receptor gene, resulting in unusually high levels of low-density lipoprotein (LDL-C) in serum. Researchers from Epigenic Therapeutics Co. Ltd. presented the discovery of an epigenetic modulation therapeutic, EPI-001, for HeFH.