Active programs
We are currently developing ASO therapies targeting the genetic variants listed below.
If you or someone for whom you are a legal guardian is affected by one of these conditions, we encourage you to register with RAREkey to stay informed about relevant treatment opportunities.
Select a program to learn more about the disease, its molecular mechanism, and our therapeutic approach. Select a stage heading to view the activities included in that phase. Pipeline positions are indicative and evolve as programs advance.
No active program for your disease?
If the patient’s condition or gene is not currently part of an active program, you can still register with RAREkey. Our scientific team will review the genetic information submitted to assess whether an experimental therapy could potentially be developed.
If a relevant opportunity arises, we may contact you to discuss potential next steps.
