Novel approaches to developing strategies for IND applications and clinical trial design are necessary, especially for rare and ultra-rare diseases. By coming together to use Odylia’s status as a non-profit as a central sponsor, the ability to cross-reference studies, reuse of physical materials such plasmids and GMP grade vectors, and redefinition of lean clinical trial design, there are cost and time savings to be realized. Using Odylia as the sponsor, a platform delivery system could have one comprehensive study conducted to test toxicity and safety and hold a letter of cross-reference to make available that platform for use with other indications. In addition, by standardizing the manufacturing method used for AAV production within the Odylia network, a temple CMC section, including reduced burden for adventitious agent testing, may be developed for the use of all Odylia members which may be used whenever an Odylia AAV manufacturer is selected for GMP virus manufacture.
Using Odylia’s non-profit status there is opportunity to make both the pre-clinical and clinical regulatory process more efficient as the FDA may be more approachable with a smaller, non-profit entity. Holding letters of cross-reference, making and storing larger batches of platform vectors and plasmids, using Odylia’s network of discounted services and utilizing Odylia’s independent, non-profit status to make these available and collaborate with development companies are only a few of the opportunities to change the ability to bring rare and ultra-rare indications from the lab into the clinic.
By taking a cross-section of pre-competitive Odylia Members with the common goal of strategizing and developing a new protocol for rare and ultra-rare IND submissions and clinical trial design, there is opportunity to dramatically change the frequency and costs associated with bringing novel therapies to trial. Through working together in a pre-competitive landscape, Odylia members are defining where non-competitive synergies exist, providing value for all and helping advance the field as a whole.