Odylia Therapeutics Inc

Odylia works to bring life-changing therapies to people with rare diseases - accelerating and de-risking the process to get treatments from the lab through clinical trials to patients. We remove roadblocks that might delay or stop a potential treatment, creating innovative collaborations to ensure drug development for rare disease continuously moves forward.
Work with 6-10 Patient Advocacy Groups each year to create strategic research plans. Patient Advocacy Groups (PAGs) are the primary drivers of rare disease research. They are usually grass-root efforts of patient families who are looking for answers about a rare disease but they often lack scientific knowledge and bandwidth to determine a strategic, streamlined plan to get research from the lab to clinical trials and are left to navigate the process alone. Valuable time and resources are wasted and the groups end up no closer to finding a therapeutic for their patients after years of effort.
Manage the gene therapies we are developing directly and foster creative collaborations to ensure continuous progress toward clinical trials. Currently, Odylia oversees two therapeutic programs that are in preclinical testing. We will continue to further work with contract research organizations, key opinion leaders,vendors, and clinicians to lower costs, maintain timelines, and ensure treatments continue to move forward.
Secure $5-8 million in venture philanthropy to support our Manufacturing Fund. The costs to manufacture materials needed to test therapies are high. In the absence of a commercial partner who would fund these costs, Odylia will seek donors who believe in our mission and desire to see treatments created for people with rare diseases.
Odylia is committed to shifting the paradigm to focus on proven science and the unmet need for rare disease patients to create synergy to drive therapeutic development. The consequence of not doing this work is not only lost opportunity, it is potential loss of life for people diagnosed with a rare disease.

Working with Patient Advisory Groups: We will help PAGs identify short and long term goals for their research programs or research investments. Patients with a rare disease may want different treatments for symptoms, improved quality of life, or even a cure, understanding these differences and defining the needs for each organization is important for guiding activities. Working with the PAGs, we will provide a therapeutic or research roadmap, experimental plan (development and oversight), disease area resource gap analysis, key opinion leader engagement, clinical development planning, patient data strategy, program management & operations - dependent on the group’s needs. Odylia will educate and empower PAGs-working together to create and drive strategic research for their disease to better serve the needs of the patients they represent. Moreover, we will help PAGs determine which research may not best serve their goals, and therefore is not the best investment of their resources. PAGs will have increased capacity to create and drive a strategic research program to achieve their goals on behalf of their patients. Their increased capacity will also enable them to better initiate and implement research using the detailed roadmap we provide.
Pipeline Program Management: Odylia Therapeutics has created and is implementing a streamlined and strategic plan to move two gene therapies from preclinical development to clinical trials. Our staff directs the day-to-day work and scientific guidance required to move each therapy forward. We focus on completing the research that is specifically aimed at proving the treatment is safe and effective and does not digress to answer interesting but not useful scientific questions. The work includes proof-of-concept studies, IND-enabling studies, coordinating the manufacturing process for materials needed for studies, and clinical development activities. We work with a network of for-profit companies to innovatively align interests and expertise creating strategic partnerships with third party vendors who provide their services to Odylia at a discount, enabling us to produce therapies as a significantly reduced price. Odylia leverages our nonprofit designation to keep costs low.
Manufacturing Funding: Rare disease treatments often stall because there is no commercial interest. One of the longest and most expensive stages of getting a treatment to patients is the manufacturing process. Odylia is actively seeking donors to support our Manufacturing Fund. A Development Manager was hired in October 2020 to focus on building philanthropic support. Donations to this fund will ensure that therapeutic materials are produced promptly, and the treatments can continue moving, reaching clinical trials and the patients as soon as possible.

Potential profit should not be the delineation for a life-altering therapy to be developed. Promising treatments for rare diseases should be moved from the lab to clinical trials no matter the

Odylia is led by President and Chief Scientific Officer Ashley Winslow, PhD. She received her PhD in Medical Genetics from the University of Cambridge and completed her postdoctoral work at Massachusetts General Hospital and Harvard Medical School. Ashley has extensive experience engaging stakeholders especially in the rare disease community. At Pfizer, she worked with patient communities to design more effective clinical trials. While at Univ of Pennsylvania, she worked at the Orphan Disease Center (ODC) with a wide variety of patient groups on all aspects of research and drug-development implementation including scientific advisement, diversifying research portfolios, budgeting, contracting, assembling advisory boards, engaging key opinion leaders, vendor selection, patient engagement, and clinical trial design. She founded and led ODC’s International Patient Registry and Natural History Study Initiative to harness human-derived data to support community-wide research and clinical development across rare diseases and the JumpStart program to guide Patient Groups just starting research initiatives. At Penn she was a ‘CSO on loan’ to the Loulou Foundation.

Scientist Kathryn Post received her PhD in Neuroscience from the University of British Columbia followed by a post-doctoral fellowship at The Translational Genomics Research Institute in Phoenix, Arizona. She has experience with cellular and molecular biology and most recently studied the effects of de novo gene mutations associated with neurodevelopmental disorders. With a focus on genetics, her studies have included both neurodevelopmental and neurodegenerative disorders. She provides scientific guidance as well as operational strategy to facilitate execution of Odylia programs.

Our CEO, Scott Dorfman serves as an Operations Partner for Fulcrum Equity Partners and sits on the Board of Dropoff Inc., Complemar Corporation, Fulcrum Equity, and Usher 2020 Foundation. Prior to Odylia, Mr. Dorfman founded and was Chairman & CEO of Innotrac Corporation (NASDAQ: INOC), which was a full-service provider of eCommerce technology, fulfillment, and call center services until combining with eBay Enterprises to form Radial Corporation. Scott is father of two children with a rare disease called Usher Syndrome and runs a separate foundation focused on raising funds for research for this disorder.

Odylia’s cofounder, Luk Vandenberghe, PhD, is Assistant Professor, Harvard Medical School and Director, Grousbeck Gene Therapy Center, Massachusetts Eye and Ear. He has discovered and developed new technologies in the gene therapy field and started numerous companies to bring treatments to patients. His work has focused on a number of rare genetic disorders and the delivery of functional genes to treat these disorders.

The Board of Directors and Advisory Board members have vast knowledge and experience in the rare disease field and are also instrumental in guiding our work to achieve our mission.

Odylia Therapeutics began working on a drug development program for Leber Congenital Amaurosis (LCA) type 6 caused by mutations in the RPGRIP1 gene, in 2019. In the last three years, we put together a strategic research plan, sourced vendors, created the experimental plans, reviewed and interpreted outcomes, and established a Clinical Development plan. In 2022, we formed the Clinical Advisory Board to review and help design the clinical trials for the gene therapy and we formed the Patient Advisory Board to provide patient and care giver insight about the trial design. We also initiated the manufacturing of the materials needed for the final toxicology study.

In fall 2021, the FDA granted Orphan Drug and Rare Pediatric Drug Designations to the RPGRIP1 program. Because of Odylia, this therapy is ready for late-stage preclinical testing. We are focused on identifying funding for manufacturing of the gene therapy for clinical trials and beginning engagement with the FDA to prepare for clinical trials.

In June 2020, Odylia partnered with patient advocacy groups to develop a gene therapy to treat vision loss in patients with Usher Syndrome caused by mutations of the USH1C gene. We have identified the academic collaborators, recruited a clinical advisory board and research vendors, created strategic research and program risk mitigation plans, and managed the experimental execution. We are working with our partners to manufacture a gene therapy and generate proof-of-concept data to assess the preliminary efficacy of the gene therapy.