Chord Clinical Trial
The CHORD Clinical Trial is looking at the use of the gene therapy DB-OTO for children with OTOF mutations. The goal of this trial is to assess different doses of DB-OTO to find out if it is:
• Safe and well-tolerated
• Effective in restoring hearing
DB-OTO is a gene therapy that has been created to deliver a working version of the OTOF gene to the inner ear. The DB-OTO injection is given into the inside of the ear during a surgical procedure that takes place under general anesthesia. This surgery is similar to cochlear implant surgery, which is well-established and has been used since the 1960s to treat deafness in babies.
DB-OTO contains a modified adeno-associated virus (AAV1) that carries the human OTOF gene. AAV vectors are protein shells that can deliver genetic material to specific cells and release it to restore function. Once AAVs deliver the genetic material, they are naturally eliminated from the body.