Regulatory Documents
We believe open source principles and practices benefit everyone. We are walking the walk of data sharing.
We share everything we learn in a way that everyone can understand and use. By “building in public” and “showing our work,” we share our resources and guidance for any and all.
One critical step on the road to sustainable precision medicine is the regulatory process. We pledge to publish as much of the Investigational New Drug application for leosen as we can.
IND for leosen
We are publishing our submissions to the FDA. This investigator-initiated IND was approved by the FDA in June 2023.
Some sections of the document are protected by IP which do not belong to us so we are carefully checking each document, and working to secure the support of all necessary partners to publish.
These documents were drafted for a specific ASO with LNA chemistry modifications, and . They may serve as a starting point, or template for other investigator initiated ASO.
We thank Dr Nicole Coufal, MD PhD (UCSD/Rady Children's), and our partners at Creyon Bio, for their support in joining us to make this initial IND documents available to the public.
We hope this helps the families and patient advocacy groups who are pursuing a similar therapeutic strategy.
If this is helpful to your or your organization's efforts, we ask that you acknowledge this publication. This will enable us to continue our work.
