ODYLIA THERAPEUTICS INC
This organization has already been registered
Someone in your organization has already registered and setup an account. would you like to join their team?Profile owner : h*****e@o****a.o*g
Mission Statement
Odylia Therapeutics accelerates therapeutic development for people with rare diseases, changing the way treatments are brought from the lab to the clinic. Our ultimate goal is to bring life altering treatments to people with genetic disease regardless of prevalence or commercial interest. As a nonprofit biotech, Odylia is shifting the paradigm to focus on proven science and removing traditional roadblocks. Odylia Therapeutics works to lower costs, reduce development time, focus research, and continuously drive successful programs towards clinical trials and regulatory approval. Strategic collaborations with patient groups, research labs, and commercial partners facilitate our work to ensure treatments are safe, effective, and ultimately reach patients who need them.
About This Cause
In the United States, 1 in 10 people have a rare disease, yet 95% of rare diseases do not have a single FDA approved drug treatment. This is not acceptable. Potential profit should not be the determining factor for a life-altering therapy to be developed. Promising treatments for rare diseases that could prevent vision loss, should be moved from the lab to clinical trials no matter the number of patients. Odylia Therapeutics is a nonprofit organization working in a traditionally profit driven space. Our organization was formed because too many rare disease treatments are being left ‘sitting on the shelf’, in part because the ability to make a large financial profit is low or non-existent though science has proven them to be viable options. The consequence of not doing this work is not only the societal impact of lost productivity, lost wages, and high medical support costs. It is also a loss of significant opportunities for the patients and even potential loss of life for people diagnosed with a rare disease. Our goals include: • Complete the preclinical work for the therapeutic programs in our pipeline, leading to submission for regulatory approval to begin clinical trials in humans (called an IND submission.) The two current programs address vision loss, specifically for genetic forms of Leber Congenital Amaurosis and Usher Syndrome. • Raise the funds required for all aspects of these programs including manufacturing of the materials needed for toxicology, efficacy, & safety studies as well as for clinical trials. • Increase capacity with Patient Advocacy Groups working to find treatments for rare diseases • Add programs with strong proof-of-concept that prevent vision loss to our pipeline and efficiently move them to clinical trials All of Odylia Therapeutics’ activities are directed toward advancing the development of treatments for rare diseases in order to increase access for patients. Our strong partnerships with Patient Advocacy Groups help ensure patient needs are met. As a nonprofit organization, Odylia focuses on its mission and not the potential for financial profits. We stay nimble in order to address challenges quickly and strategically. Our two core programs work hand-in-hand to move treatments from academic labs and the ‘proof-of-concept’ stage into late-stage preclinical studies to enable submission for regulatory approval and then to clinical trials. • Gene Therapy Pipeline - Odylia creates and implements streamlined, strategic plans, to move gene therapies through preclinical development to clinical trials. We direct the critical activities required to ensure roadblocks are removed and efficiency is maximized. • Brydge Solutions - Patient Advocacy Groups are the primary drivers of rare disease research. These grass-root efforts often lack the bandwidth and scientific knowledge to develop a prudent approach to move research from the lab to clinical trials, resulting in loss of valuable time and resources. Odylia enables these groups to build capacity by working hand-in-hand to develop a research roadmap, gap analysis, clinical development planning, and other tools critical to achieving each group’s goals. In 2021, Odylia launched a key initiative to support our core programs. The creation of a designated Manufacturing Fund ensures treatments don’t stall. Through this approach, we are forging a new path for rare diseases. We are consolidating philanthropic donations of all sizes, to fund the costs of physically manufacturing gene therapies for patients with rare diseases. This will also advance a gene therapy platform that will be available to other nonprofits and strategic partners and ultimately impact a significant number of rare diseases.