Our Pipeline
Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: hematology, immunology & oncology, liver and lung. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing’s broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. For more information, please visit www.primemedicine.com.
prime medicine pipeline
Modular
Platform
Indication
Delivery
Discovery
Lead
Optimization
IND-
Enabling
Phase 1/2
Hematology, Immunology & Oncology
p47phox Chronic Granulomatous Disease (CGD)
ex vivo
X-linked CGD (with PASSIGE™)
ex vivo
Ex vivo CAR-T1 (with PASSIGE™)
ex vivo
Liver
Wilson’s Disease
LNP
Alpha-1 Antitrypsin Deficiency (AATD)
LNP
Lung
Cystic Fibrosis2
LNP/AAV
Prime Medicine is identifying opportunities to advance its other programs, including neurological diseases, cell therapy, ocular diseases and hearing loss, in partnership or through internal efforts in the future.
1 In September 2024, Prime entered into a strategic license and broad collaboration agreement with Bristol Myers Squibb to develop and commercialize ex vivo T-cell Prime Editor products.
2 In January 2024, entered into agreement with CF Foundation for up to $15 million to support development of Prime Editors for Cystic Fibrosis.
Clinical Programs
Prime-0101 Study: Chronic Granulomatous Disease
Prime Medicine is currently enrolling patients in Prime-0101, a Phase 1/2 clinical trial evaluating the Prime Editing program PM359 for the treatment of p47phox Chronic Granulomatous Disease (CGD). People with p47phox CGD inherit a NCF1 gene mutation from each parent, which causes the body to produce abnormal white blood cells that lead to inflammation and increased susceptibility to infections. Prime-0101 will evaluate the safety and efficacy of investigational therapy PM359 in reducing or preventing infections and inflammatory conditions in people living with p47phox CGD. Learn more about CGD here.
Learn more about Prime-0101 here, visit clinicaltrials.gov or email us at cgdtrial@primemedicine.com for more information.
About PM359
PM359, Prime Medicine’s first product candidate within its hematology, immunology & oncology area of focus, is an investigational therapy designed to treat the p47phox variant of CGD. PM359 is comprised of autologous hematopoietic stem cells (HSCs) modified ex vivo (that is, outside the body) using an all RNA-based Prime Editor that has been designed to correct a high percentage of cells containing the disease-causing mutation. The Prime Editor is delivered into the HSCs by electroporation, which is a clinically and commercially validated delivery technology. PM359 has received rare pediatric drug designation and orphan drug designation from the U.S. Food and Drug Administration.