Research
Where we are on our journey
Last year, we made a $50K donation to the Gray Lab at UTSW to obtain animal models and kick off research. In parallel, we negotiated a $400K two-year sponsored research agreement to complete Phase 1: Develop and test an LNPK gene replacement therapy. This a critical first step and if it goes well, we will move forward with manufacturing the drug for safety testing and human clinical trial. UTSW hosts one of the top gene therapy labs in the country and has successfully brought multiple drugs to clinical trial in the rare disease space.
Gene therapy experts at UTSW are developing an AAV to deliver LNPK to brain cells and demonstrate efficacy in restoring the LNPK protein.
The AAV gene therapy created during proof of concept will be tested in animals to ensure its safety.
We’ll partner with a biotech and other manufacturers to produce doses for use in human clinical trial.
We’ll submit our safety and efficacy studies to the FDA for their approval to use our drug for clinical trial.
What is Gene Therapy?
Gene therapy is a technique that uses a gene to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patient’s cells with a healthy version of that gene. Both inherited genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia) have been treated with gene therapy.
Source: NIH National Human Genome Research Institute
The most common way to deliver a new copy of a gene to the body is through an Adeno-Associated Virus (AAV) vector, which is a repurposed virus used as a delivery vehicle to transport the gene to cells in the body.
“The ability to generate recombinant AAV particles lacking any viral genes and containing DNA sequences of interest for various therapeutic applications has thus far proven to be one of the safest strategies for gene therapies.”
Source: nih.gov
Our scientific collaborators
We’re working with an incredible team of gene therapy and rare disease experts to push forward our science and cure. We are currently partnering with the Gray Lab at UTSW as part of a funded scientific research agreement to develop a proof of concept gene therapy for LNPK. We are eternally grateful for their time and expertise.
Lauren Flickinger founded RareOlive after her daughter was diagnosed with an ultra-rare neurodevelopmental syndrome in 2022. She is a wife, mother of two and accomplished Product Director in the FinTech industry. Lauren oversees strategy, fundraising and scientific engagements for RareOlive.
Lauren brings a unique skillset of solving complex problems, curiosity for learning and an unbridled passion for helping her daughter to push forward the understanding of LNPK and associated treatment options.
She is passionate about helping families in a similar position and enjoys connecting with parents in the rare-disease community to pay forward the incredible support she has received from Terry Pirovolakis of SPG50 and the community of rare disease crusaders.
Dr. Stephen Gray has pioneered AAV gene therapy treatments for several rare neurological diseases. He is Associate Professor at UTSW in the departments of Pediatrics, Neurology & Neurotherapeutics, and Molecular Biology, with cross appointments in the Center for Regenerative Medicine and the McDermott Center for Growth and Human Development.
Dr. Gray serves as the Director of UTSW’s Viral Vector Facility, where his major focus is in AAV vector development tailored to serve specific clinical and research applications involving the nervous system. These include preclinical studies for Rett Syndrome, Giant Axonal Neuropathy (GAN), Tay-Sachs, Krabbe, AGU, and Batten Disease, and has expanded to human clinical studies for GAN and SPG50.
The Gray lab is overseeing the pre-clinical development of a gene therapy for LNPK, funded by a research award from RareOlive.
Dr. Xin Chen is an Assistant Professor in the Department of Pediatrics at UT Southwestern Medical Center. His scientific efforts focus on developing adeno-associated virus (AAV) based gene therapy treatments for neurological diseases with the goal of translating preclinical results into clinical trials.
He has been developing gene therapy treatments for many neuromuscular disorders including AGU, CLN7, and SPG50, which are currently moving into clinical trials. An CLN7 clinical trial is ongoing at Children’s Health in Dallas, TX in collaboration with UTSW Medical Center, with four patients treated up to date. An SPG50 clinical trial was approved more recently by Health Canada and the FDA, and the first SPG50 patient received treatment in March 2022. Dr. Chen is also providing support for other gene therapy projects in other laboratories.
Dr. Chen and his team began pre-clinical development of a gene therapy for LNPK, funded by a research award from RareOlive in January 2023.