Frequently Asked Questions

Clinical studies are a type of research performed to learn about the effects of investigational new drugs or treatments. The studies follow predetermined procedures, called the clinical study protocol. Clinical studies are the most important way for researchers to better understand whether a new drug or treatment is safe and effective for a particular condition.

The most well-recognized use of mRNA technology has been in vaccines, such as COVID-19 vaccines; however, applications of mRNA extend to other disease areas. In vaccines (used for disease prevention), the mRNA carries the instructions for a viral compound that triggers the body to produce a protective immune response. By contrast, in medicines that treat disease, the mRNA carries the instructions for a missing or defective compound and enables its production.

Gene therapy has the potential to treat diseases at their root causes by correcting or replacing defective genetic material (DNA). A virus carrier or vector delivers the gene to the targeted cells of the body. Once inside the cell’s nucleus (the “control center”), gene therapy works to correct or replace the genetic material that is not properly working. This is done by gene editing or gene addition. Under ideal circumstances, the beneficial changes made to the genetic material are permanent, resulting in a long-lasting cure; however, if the therapy misses its intended target, side effects can also be long-lasting. By contrast, mRNA therapy does not enter the cell’s nucleus, nor does it change the genetic material. mRNA therapy only instructs cells how to assemble key ingredients to create compounds using the cell’s own internal “assembly line.” Its effects are temporary and after the compound is made, the mRNA therapy gets broken down. Repeated dosing is necessary to maintain the beneficial effects, but, if side effects occur, the therapy can be stopped to allow them to resolve.

Additional information about gene therapy can be found here.

There is no cost to participate in these clinical studies. Travel expenses, such as transportation and hotel, if required, will be covered. You will also be reimbursed for other travel expenses that are related to your study visits, such as meals, parking, and taxi fares.

Clinical study participation is an important decision. Below are some helpful questions to ask when speaking to your physician or family members to determine whether participation is right for you:

• Which type of treatment is being studied?
• Which tests or procedures will I need to have done if I participate?
• What type of travel support is provided in this study?
• How will this treatment impact my current diet or lifestyle?
• What risks and benefits can I expect if I participate in this study?