An internationally recognised RNA therapeutics scientist, I was an integral member of the group that pioneered therapeutic antisense oligomer development. We were the first to recognise the clinical potential of morpholino oligomers and designed Eteplirsen, the first FDA approved drug for the treatment of Duchenne muscular dystrophy (DMD). Golodirsen and Casimersen, ASOs designed to address other subsets of DMD mutations, were approved by the FDA in 2019 and 2021 respectively. In 2021, I was appointed an Officer of the Order of Australia for distinguished service to medical research and neurological science.

I serve as the Chief Scientific Officer at To Cure A Rose (TCAR), leading scientific strategy and therapeutic development for children with rare monogenic diseases. I earned my Ph.D. in Medical Sciences from Texas A&M University Health Science Center, Institute of Biosciences and Technology in 2008. I have led large scientific teams through the design, optimisation and advancement of RNA and gene-based therapies, with many programs now progressing through FDA and international clinical trials.

I am a senior scientist and biotech innovator with a passion for advancing RNA therapeutics and precision medicine to transform patient outcomes. My expertise spans RNA metabolism, DNA damage and repair, and RNA-based platform technologies, in particular oligonucleotide therapeutics, with a strong focus on translating cutting-edge research into impactful treatments. I led the preclinical development of a first-in-class RNA oligonucleotide therapy for cancer, taking it from discovery to a clinic-ready drug product.

Before joining To Cure A Rose (TCAR) as a senior scientist, I earned my Ph.D. from Oklahoma State University in 2015, completed my postdoctoral training at the University of Texas at Austin, and managed a biomedical engineering laboratory as a research associate. With over a decade of laboratory experience, I am deeply committed to advancing personalised therapies for children with rare genetic disorders. My passion lies in helping children and families access the promise of targeted, life-changing RNA therapies.

A distinguished molecular parasitologist with over 35 years of experience spanning both academic and industrial science, my research career has made significant contributions to global health and biotechnology, including immunological strategies for controlling seasonal and pandemic influenza. In September 2002, I was appointed the inaugural Director of the Institute for the Biotechnology of Infectious Diseases at University of Technology Sydney (UTS), with appointments at Rockefeller University in New York, Hadassah Medical School in Jerusalem, and the University of Bern in Switzerland. In 2008, I established a Bio-Innovation course at UTS. In 2013, I was appointed the inaugural Director of the SPARK Sydney program, modelled on the successful initiative at Stanford University. My career is dedicated to improving human health through scientific innovation and global collaboration.

Ken VanLuvanee is a serial entrepreneur with over 25 years of pharmaceutical and technology expertise. Throughout his career, Mr. VanLuvanee has enjoyed great success in the regulatory process and technology sector of the life sciences industry.

Mr. VanLuvanee is currently the President/CEO of Facet Life Sciences. Prior to creating this organization, Mr. VanLuvanee was the founder of Apyx, a life sciences regulatory consultancy which was acquired by Image Solutions, which was subsequently acquired by Computer Sciences Corporation. He held leadership roles in newly formed organizations and was responsible for both strategic and operational performance. Prior to creating Apyx, Mr. VanLuvanee held consulting roles for Barnett International and Accenture, where he gained a practical understanding of regulatory processes and emerging enabling technologies being used across the life sciences industry.

Mr. VanLuvanee was a key participant in the development of the DIA EDM Reference Model, a cross-industry effort to apply standards to the organization and structure of R&D documentation.

Lisa Jenkins VanLuvanee, Ph.D., is the Chief Operating Officer & Vice President of Research and Development at Facet Life Sciences.  Dr. VanLuvanee has over 20 years of experience in the life sciences industry.  She specializes in product, program, and business R&D strategy; CNS, ophthalmic, and PET radiopharmaceutical product development; trial design, endpoint selection, and analysis; and FDA interactions.  She has provided strategic program and product guidance, tactical support, and content review for over 100 products.  Her experience spans blockbuster products, products being developed for rare diseases and special populations (geriatrics and pediatrics), and products with orphan, fast track, and breakthrough designations.  On behalf of Sponsors, she provides executive-level guidance on corporate strategies and supports board-level discussions and investor relations activities.

I’m an MBA-trained health economist with a Master of Health Economics and experience across academia, consulting and the biotech industry in both Australia and the United States. I now run my own advisory practice so I can focus my work where it creates the greatest impact, particularly in rare diseases. I support Saving Sasha by helping shape the ASO development framework and guiding our value, access and sustainability strategy, as well as contributing to fundraising and partnership development. My goal is to help build a clear and workable pathway that supports Sasha today and paves the way for future families who need personalised therapies.