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Odylia accelerates therapeutic development for rare diseases. We are scientists discovering and developing the next generation of rare disease treatments and increasing access to transformative treatments for rare disease patients. I’ve written to you before about why we chose to make Odylia a nonprofit in a traditionally for-profit industry. Today, I want to share more about how being a nonprofit supports the way we carry out the science needed for our two pipeline programs, our Brydge Solutions initiative, and what it means for future rare disease drug development.
Six years ago, Odylia was founded through the partnership of scientists and the rare disease community to address an unmet need. The need to move stalled treatments to clinical trials for the patients who need them. We pride ourselves on our team’s deep knowledge of genetics, molecular and cellular biology, and novel drug development, as well as the deep empathy and direct experience we have with rare diseases. These traits are core to the entire organization- the staff, Board of Directors, and our Scientific Advisors, as well as the amazing volunteers that join our efforts.
Odylia operates as a biotech, developing novel and life-changing therapies for rare diseases - bringing gene therapies from the lab to clinical trials. We leverage our internal scientific expertise and strategic team differently for each program, building critical partnerships as a part of a well-rounded team that addresses the needs of each therapeutic, we have in development. Our scientific and operational strategy allows us to be nimble in the ever-changing environment of drug development. It also allows us to work on programs that are in different phases of development (i.e., discovery, late-stage preclinical studies, clinical planning), or affect different organ systems (i.e., the eye, the brain, systemic tissue overgrowth issues, etc), or using different therapeutic modalities (i.e. gene therapy, small molecules, antisense oligonucleotides).
The technical, hands-on aspects of drug development take time. As scientists, we design the most straightforward set of experiments to bring a therapy from ideation to the clinic, recognizing that discovery rarely proceeds in a linear path. Our team adapts experimental plans to maximize progress and to minimize unnecessary delays or scientific questions not relevant to developing a treatment. Collectively, our scientific strategy defines Odylia and we believe this approach gives rare disease treatments a better chance of success.
In each newsletter we will spotlight one of our Odylia Team members, so you can meet the people working on the exciting therapies we have in development. We’ve also included a quick poll to get feedback from you about new resources you’d like to see in our library.
As I write this, we are putting the finishing touches on our pre-IND submission for the RPGRIP1 gene therapy and finalizing the next study for the USH1C program, while also evaluating new potential programs and Brydge Solutions partnerships. We are busy and are excited about the future. The overall timeline to get treatments to clinical trials is lengthy and the path is circuitous. Building a nimble and passionate team that can navigate this complex process has been key to Odylia’s success.
From the beginning, the goal of Odylia has been to bring safe, effective treatments to patients regardless of potential for economic gain.
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