Gene Therapy Project Fundraising
September 2026 Update:
3 Million of 5 Million Raised
Loving Loic Foundation began with an urgent goal: to move a gene therapy for PKAN toward children who need it. That work remains important. As we grow, we are also building a foundation that can support families and pursue promising paths to treatment beyond a single program.
We are working to bring more parents and other family stakeholders into the organization so that the people living with PKAN help set its priorities. We want families to have regular opportunities to connect, share what they are experiencing, and tell us where support and research are most needed. We also received a recent grant to start Selah's Sibling Support Program in Montana.
We are also developing clearer policies for how the Foundation evaluates and funds research and family outreach. That work will help us make careful decisions, use donor funds responsibly, and respond to opportunities across different lines of research. Our goal is to help move every credible path toward a treatment or cure as quickly as we can.
Gene therapy update
The PANK2 gene therapy vector has been manufactured. The next major step is the toxicology and safety work needed to support a future human trial. We have submitted a pre-IND request to the FDA and are working through questions about the studies required. FDA dialogue has taken longer than we hoped, so we cannot yet give families a reliable trial date.
Families often ask how much more funding is needed. It is a moving target because the remaining studies, regulatory work, and trial preparations depend in part on FDA feedback. Loving Loic Foundation has raised more than $2 million for this effort. Together with another funding source, support for the program has hit $3 million. The Foundation has provided $1.054 million this year alone to move the work forward. We still need significant funding to complete the remaining steps, and we will share a more reliable estimate as those requirements become clear.
We will share meaningful milestones as they happen and be candid about delays. At the same time, we will keep building the parent-led foundation that PKAN families deserve—one equipped to support families now and push promising research forward wherever it may lead.
September 2025 Donation Update:
2 Million of 5 Million Raised
We are pleased to announce a major advancement in the gene therapy research project for PKAN. Due to a generous donation of $1.2 million from a private PKAN family, the gene therapy project is moving forward as needed. This substantial contribution will ensure the gene therapy project will be fully funded through the Phase III toxicology studies, marking a critical milestone in our mission to develop a life-saving gene therapy for PKAN. We extend our deepest gratitude to this family for their remarkable support and investment in the OHSU and UMass Chan team on this exciting journey towards a breakthrough in PKAN research.
Implications for the PKAN Community
Given current timelines, clinical trials with gene therapy for our children may be ready to start in 2026. As a reminder, given the single location and precise known mutation of PKAN, it is ideally suited for gene therapy, making this research exceptionally promising.
Call to Action
To ensure the successful development and delivery of this vital treatment, we must continue to secure additional funding. We appeal to friends, family, and all willing supporters to help us achieve our financial goals. Each donation brings us one step closer to halting the progression of PKAN and preventing this disease in future generations.
Together, we can make a significant impact and offer hope to those affected by PKAN. Thank you for your unwavering support.
September 2025 Gene Therapy Update
We are excited to share important progress on the path toward a cure for PKAN. Thanks to the incredible generosity of our donors, we have now raised $2 million toward the gene therapy project. Our goal remains to raise a total of $5 million to bring this treatment from design through to human clinical trials.
We are now entering Phase III/IV of development, with multiple components advancing in parallel to accelerate progress:
Pre-IND Submission to the FDA:
OHSU has submitted a Pre-Investigational New Drug (Pre-IND) application. This is an important step in which researchers share their data and development plans with the FDA to receive early feedback. It helps ensure that when the formal IND is submitted, the trial design, safety studies, and manufacturing processes meet regulatory standards—saving valuable time later.
Large Mammal Study:
The critical large mammal study is on track to begin Spring 2026 and will be conducted at the OHSU Primate Center. These studies are a necessary step to demonstrate safety and effectiveness before moving into human trials.
Human Trial Preparation:
Parameters for the first-in-human trial are actively being developed, positioning us to launch as soon as funding and regulatory clearance are in place.
The only thing that will slow this momentum is funding. To reach the clinic, we must raise the next $3 million. Every dollar brings us closer to giving children with PKAN a chance at life-saving treatment.
October 2024 Gene Therapy Update
What we are doing to help:
The Loving Loic Foundation has established the Loving Loic PKAN Gene Therapy Research Fund to develop a gene therapy treatment for PKAN.
The Loving Loic Foundation supports a partnership between PKAN physician-scientists at Oregon Health & Science University and scientists from the Horae Gene Therapy Center at UMass Chan Medical School. These experts from OHSU and UMass bring complementary skill sets, decades of experience, and a collaborative approach to the partnership, positioning them to successfully develop gene therapy for PKAN.
Dr. Miguel Sena-Esteves received his PhD in biomedical sciences from the University of Porto, Portugal, where he started his work in gene therapy. As a member of the Horae Gene Therapy Center, his lab has developed adenoassociated virus (AAV)-based gene therapies for several rare neurologic diseases in children and adults. Dr. Sena-Esteves and his team work to optimize gene delivery and incorporation into the central nervous system, the area to be targeted in PKAN. He has also established the Translational Institute for Molecular Therapeutics (TiMT) at UMass Chan and serves as its director.
Dr. Heather Gray-Edwards is a Doctor of Veterinary Medicine with a PhD in biomedical sciences. She is the Director of the Umass Chan Transgenic Core, the Large Animal Program at UMass Chan and the Associate Director of the TiMT. Her lab in the Horae Gene Therapy Center uses animal models to enhance understanding of disease progression and develop and test novel therapeutics, including new AAV vectors and delivery routes.
Dr. Susan Hayflick, a medical geneticist, has dedicated much of her career to PKAN. Her accomplishments include identifying the causative gene in 2001, developing a PKAN mouse model, creating a potential therapeutic for PKAN, and overseeing early gene therapy experiments in her lab using PKAN mice
Dr. Penny Hogarth, a movement disorders neurologist and clinical trial expert, has developed deep experience in the management of PKAN patients. In partnership with Dr. Hayflick & other collaborators, she designed a potential therapeutic for PKAN and was awarded a large NIH grant to support the related clinical trial, for which she is the principal investigator.
Dr. Sena-Esteves, Dr. Gray-Edwards, and the Horae Gene Therapy Center have built critical infrastructure to support a gene therapy pipeline. Their expertise in bringing gene therapies from bench to bedside, including experience working closely with the FDA, makes them an ideal partner.
Dr. Hayflick, Dr. Hogarth, and their team at OHSU have laid the foundation for the development of gene therapy using PKAN mice, and their team will provide key experience as PKAN clinicians and clinical trialists when gene therapy is moved into humans for the first time.
We need help to raise $5,000,000
The Loving Loic Foundation has raised $2 million for the gene therapy project over the past two years through the generosity of our community. Our goal is to secure a total of $5 million by the end of 2026. This funding will support the development and advancement of a gene therapy treatment—moving from design to human clinical trials on an accelerated timeline. Below is the current project and development roadmap. We have now entered Phase III/IV of development, with several components progressing in parallel to accelerate the path toward a human clinical trial.