“What good does it do in the world to create life-altering treatments and never bring them to patients?”
Treatments for rare diseases are being discovered in academic labs all over the world. These viable therapies could save a patient’s sight, their independence, or even their life. Unfortunately, they are often ‘left on the shelf’ because the disease prevalence is too low to warrant commercial interest and there is little consideration for the potential impact it could make on the lives of patients and their families.
One of the longest and most expensive stages in developing new gene therapy treatments is the manufacturing process. The typical path for a promising new treatment developed in an academic lab includes establishing a partnership with a pharmaceutical company to fund and manage the program through late-stage preclinical and clinical studies. The drug is commercialized once it has been approved by the FDA, ensuring patients have access to the new treatment. Rare disease treatments often stall because there is no commercial interest due to the small patient population.
Odylia is transforming how therapeutics are developed for rare disease. As a nonprofit, we are leveraging philanthropic donations to ensure viable drug treatments continue to be developed and manufactured. Patients deserve every treatment option. Odylia, with support from donors, is making that possible through our Manufacturing Fund.
Contributions from individuals, foundations, and corporations to the Odylia Manufacturing Fund will help fund the manufacturing costs of viable drug treatments for rare diseases. We are raising $8 million for the three gene therapy programs currently in our pipeline. Each of these treatments has the potential to preserve or fully prevent vision loss, if treated early enough. But we won’t stop there. We will continue to discover and enable the development of new treatment options for patients with rare diseases.
Support this vital work. The need is urgent. We are seeking $950,000 to begin manufacturing a drug treatment for RPGRIP1 as soon as possible. Sight-saving treatment will be put on hold until funding is secured. Odylia is looking to secure donations from individuals and companies, if you or your company would like to have an impact on hundreds of patients through the Manufacturing Fund, please complete this form. Or you can make a donation online today.
Patients with rare diseases need treatments. Odylia is committed to shifting the paradigm for therapeutic development, bringing together people, companies, patients, and donors to reach our goal. We are working to manufacture and deliver therapies that are proven, ensuring patients and their families don’t have to wait for life-changing options.