Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing the RNA activation (RNAa) mechanism to up-regulate endogenous gene expression. RAG-01 is a pioneering small activating RNA (saRNA) drug engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. It is currently being investigated in a phase Ⅰ, open label, multi-center study in patients with non-muscle-invasive bladder cancer (NMIBC) who have failed Bacillus Calmette-Guérin (BCG) therapy. We are conducting the clinical trial aiming at gaining regulatory approval to make RAG-01 available to all eligible patients as quickly as possible.
Expanded Access, which is sometimes known as “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational therapy for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. The U.S. FDA advises that Expanded Access may be appropriate when all the following apply:
- Patient has a serious or immediately life-threatening disease or condition.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- Patient enrollment in a clinical trial is not possible.
- Potential patient benefit justifies the potential risks of treatment.
- Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
For more information about expanded access in the U.S., please visit the FDA website at https://www.fda.gov/news-events/public-health-focus/expanded-access.
Ractigen is not currently making its investigational products available on an expanded access basis anywhere in the world, as we believe that our clinical trials are the most appropriate way to access our investigational products. We encourage patients who are interested in accessing RAG-01 treatment to talk to their physician about participating in a clinical trial. Information about the trial of RAG-01, including enrollment status, eligibility criteria, and locations, is available at https://clinicaltrials.gov/study/NCT06351904.
If we determine that expanded access to RAG-01 becomes appropriate in the future, this website and policy will be updated. If you have further questions, please feel free to contact us at expanded.access@ractigen.com with any questions.
Consistent with the 21st Century Cures Act, Ractigen Therapeutics may revise this policy at any time.
