Frequently Asked Questions
LUNAR-OTC Studies FAQs
The most well-recognized use of mRNA technology has been in vaccines, such as COVID-19 vaccines. But 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.
As with all clinical studies, there can be risks and possible side effects associated with taking the study drug and undergoing the standard medical tests carried out as part of the studies. Information on the possible side effects that may be experienced are explained in the consent form and will be discussed with you by the study doctor.
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?
If you or someone you know may be interested in learning more about clinical trials at Arcturus, contact us by filling out this form here.
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.
Participation in clinical studies can allow you to play a more active role in your own healthcare, gain access to investigational new treatments, assist researchers as they gather valuable information, and help others by contributing to medical research. Clinical trial participation is not always easy, but it is rewarding. It is important to discuss with your family or doctor if this decision is right for you.
There are always risks involved with participating in clinical studies; however, clinical study protocols are carefully designed and controlled to monitor the safety of all participants.
Placebos are inactive drugs or treatments used in clinical studies. Placebos are used in clinical studies as “controls.” They allow the effects of the active drug or treatment to be compared with the effects of the placebo, giving a much clearer picture of safety and effectiveness. Clinical studies that use placebos can sometimes be performed faster than those that do not use placebos because the safety and effectiveness of the investigational new drug or treatment can be better understood in a shorter period of time.
You can start by asking your physician about clinical studies that might be right for you. If you are part of a patient support group or organization, they may have resources or know of other clinical studies you may be interested in. ClinicalTrials.gov is an excellent resource and registry of all ongoing clinical studies with additional details and contact information.
If you or someone you know may be interested in learning more about clinical trials at Arcturus, contact us by filling out the form here.
Connect with the advocacy organizations below for additional assistance, education, and resources
- Baine’s Legacy
- Belgian Organization for Kids and Adults with Metabolic Disease (BoKS)
- Connecting Families UCD Foundation
- Metabolic Support UK
- National Organization for Rare Disorders (NORD)
- National Urea Cycle Disorders Foundation
Follow us on social media for the latest updates on our mRNA science, clinical pipeline, and community events. Please contact us at Community@ArcturusRx.com.