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
Liver
Wilson’s Disease
LNP
Alpha-1 Antitrypsin Deficiency (AATD)
LNP
Lung
Cystic Fibrosis1 (Including PASSIGE™)
LNP/AAV
Immunology & Oncology
Ex vivo CAR-T2 (with PASSIGE™)
ex vivo
Prime Medicine is identifying opportunities to advance its other programs, including CGD, neurological diseases, cell therapy, ocular diseases and hearing loss, in partnership or through internal efforts in the future.
1 In January 2024, entered into agreement with CF Foundation for up to $15 million to support development of Prime Editors for Cystic Fibrosis.
2 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.
Clinical Programs
Prime-0101 Study: Chronic Granulomatous Disease
Although the CGD study is not enrolling new participants right now, the positive results seen so far are encouraging. These results support the potential of prime editing, and we plan to continue working with regulatory agencies (health authorities) to move PM359 forward. For more information, please see “Prime Medicine Announces The New England Journal of Medicine Publication of PM359 Clinical Data for the Treatment of Chronic Granulomatous Disease.”
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.