our commitment
Prime Medicine is committed to transforming the lives of patients with debilitating diseases through the application of our Prime Editing platform and technology. We believe that strong partnerships with patient communities and advocacy organizations are essential to achieving our mission. We strive to:
- Learn directly from patients and caregivers to better understand the patient journey
- Communicate in a transparent and timely way about our programs to patient communities
- Work collaboratively with advocacy organizations to identify and pursue common goals
Through these guiding principles, we aim to deliver on the promise of one-time, potentially curative therapies to address genetic diseases.
about our programs
About Our Programs
about chronic granulomatous disease
Chronic Granulomatous Disease (CGD) is a rare inherited hematologic disorder characterized by susceptibility to severe, difficult-to-treat infections, and inflammatory/autoimmune complications. CGD is caused by mutations in any one of the subunits comprising the NADPH oxidase complex, which is a group of proteins found in immune cells that work together to kill pathogens such as bacteria and fungi. CGD causative mutations are estimated to occur in between one in 100,000 and one in 200,000 births in the United States, and most children are diagnosed within the first three years of life. One type of CGD is p47phox CGD, which occurs when a person has mutations in both copies of the NCF1 gene.
Beginning in childhood, patients with CGD develop infections from a range of both typical and unusual bacteria, fungi and mycobacteria. These infections may present in various organ systems, and protracted infections can lead to long-term organ damage and failure. In addition, patients have non-infectious inflammatory disease, most commonly presenting as inflammatory bowel disease, soft tissue granulomas, and strictures of the urinary or digestive tract.
Undiagnosed or untreated, the infectious manifestations of CGD are rapidly fatal, with refractory or antimicrobial resistant infection the leading cause of mortality.
current clinical trials
Clinical trials are designed to help determine whether an investigational therapy or treatment is safe and effective.
We are currently enrolling patients in Prime-0101, a Phase 1/2 clinical trial evaluating the safety and efficacy of PM359 in patients with p47phox autosomal recessive CGD. Learn more about Prime-0101 here or visit clinicaltrials.gov. If you have questions or may be interested in participating in our Prime-101 clinical trial, please email us at cgdtrial@primemedicine.com for more information.
expanded access
Expanded access, or compassionate use, enables patients with serious or immediately life-threatening conditions to gain access to investigational therapies outside of clinical trials. We believe that participation in our clinical trials is currently the most appropriate way to access our investigational therapies. Prime Medicine does not provide access to investigational therapies outside of clinical trials at this time.
Prime Medicine’s investigative therapies are not approved by the FDA or by any other country’s regulatory authority, and their safety and efficacy are still being studied in clinical trials.
external resources
These resources are for informational purposes only and do not represent an endorsement by Prime Medicine of any individual organization or viewpoint. This list is not exhaustive, and the resources within should not be construed as medical advice.
American Society of Gene + Cell Therapy Clinical Trials Finder: A curated list of gene and cell therapy clinical trials, updated daily and sourced from clinicaltrials.gov.
Chronic Granulomatous Disease Association of America: An independent, nonprofit organization with the world’s largest community of CGD patients.
Global Genes: A non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally.
Genetic and Rare Diseases Information Center (GARD): A program of the National Center for Advancing Translational Sciences. It provides the public with access to current, reliable information about rare or genetic diseases.
National Organization for Rare Disorders (NORD): NORD advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives.
patient stories
Coming Soon…
If you are living with CGD and are interested in sharing your story, please email us at info@primemedicine.com.