Pulmonary arterial hypertension (PAH) is a life-threatening disease that predominantly affects women, progressively causing damage to the pulmonary vessels, leading to heart failure. Current treatments are limited, offering only symptomatic relief without addressing the underlying disease. However, a breakthrough is on the horizon, thanks to a multidisciplinary team from Monash University, including the Australian Translational Medicinal Chemistry Facility (ATMCF), a research infrastructure supported by Therapeutic Innovation Australia.
Led by Dr. Chengxue Helena Qin, the team is working on a novel, disease-modifying therapy that targets a G-protein coupled receptor involved in lung vessel inflammation and remodelling, with the goal of halting disease progression and reducing mortality. The expertise within the team is extensive, including Prof Rebecca Ritchie, Prof Paul Stupple, and Dr. Lauren May. Their collaboration with clinicians from Alfred Hospital and Monash Health ensures the research remains closely aligned with real-world patient needs.
Crucial to this project’s advancement is the support from BioCurate’s Proof of Concept (POC) Fund. This funding, combined with BioCurate’s strategic insights, has helped the team bridge the gap between early-stage discovery and the development of drug candidates. As Dr. Qin remarked, “The POC fund targets early-stage therapeutic projects, which aligned perfectly with the stage of our project.”
Beyond financial support, BioCurate has played a pivotal role in pushing the project forward by providing valuable expertise. Discussions with the BioCurate team prompted the researchers to critically evaluate their approach, ensuring that both the scientific and commercial aspects of the project were well aligned. These insights have led to streamlined workflows and an enhanced focus on the project’s translatability.
TIA is proud to have been one of the foundational supporters of this groundbreaking work through our Pipeline Accelerator scheme. This scheme has enabled Dr Qin the access to ATMCF, one of TIA’s Small Molecules Therapeutics facilities, which has been instrumental in the medicinal chemistry efforts that underpin this project. Early lead candidates were identified through high-throughput screening performed at the National Drug Discovery Centre (NDDC), which is recently supported by TIA and is another vital partner in this collaborative effort.
What truly stands out in this initiative is the shared vision among all collaborators—researchers, clinicians, and industry experts alike—to develop a therapeutic that can make a meaningful difference in the lives of patients suffering from PAH. “This process has brought us closer to our consumers and end-users,” Dr. Qin said, “which feels really special. We all have the same alignment of goals and mission – delivering a disease-modifying therapeutic for this devastating disease.”
As the team continues to work toward their goal, TIA is excited to see how these efforts will translate into tangible impact for the pulmonary hypertension community. Together with BioCurate and Monash University, we are proud to be part of this critical journey toward delivering a new hope for PAH patients.
