MyPEAK-1:

Clinical trials of AAV gene therapies are still ongoing and not all of the risks are known. Known risks today include:

• The immune system may recognize the gene therapy as a harmful intruder. AAVs do not cause diseases in people. However, the immune system is designed to remove anything it does not recognize as part of a person’s body. The immune system may react by attacking the treatment before it has a chance to work.^1,2
  • Some studies of AAV and other forms of gene therapy show that immune system reactions may affect a person’s liver²
  • Medicines that suppress or subdue the immune system are typically given before a person receives AAV gene therapy to help prevent or reduce this reaction³
• Currently, a person can receive AAV gene therapy only once; it cannot be re-dosed or administered in a larger dose at a later time.^1,2
  • After the first treatment with AAV gene therapy, a person’s immune system makes antibodies to the AAV capsid. If the body sees the same AAV again, it will attack it before it has a chance to work⁴
• AAV gene therapy may deliver a working gene to cell or tissue types that are not their intended target. This may cause illness or harm to other cells.^1,4
  • This is why the working gene is packaged with specific instructions which tell the gene what to do in target cells and reduces potential effects on other cells⁵
• Participation in an AAV gene therapy clinical trial may prevent an individual from participating in clinical trials for other AAV gene therapies in the future. Although many clinical trials of AAV gene therapies have been completed to date, ask your healthcare provider about the known risks of AAV gene therapy.