Prime Medicine
Prime Medicine is a biotechnology company developing gene-editing therapies based on prime editing.
Last updated August 31, 2026
AI crawler reads
No AI crawler has read this profile since Oct 28, 2026.
Overview
Prime Medicine is an American biotechnology company focused on developing gene therapies that use prime editing, a form of genome editing designed to make precise changes to DNA without requiring double-strand breaks. The company is headquartered in Cambridge, Massachusetts, and its work centers on translating prime-editing research into treatments for inherited diseases. The company’s development model includes both ex vivo and in vivo approaches. In its ex vivo programs, a patient’s cells are collected, genetically modified outside the body, tested or expanded, and then returned to the patient. This strategy is being applied to hematopoietic stem cells, including the autologous CD34-positive stem-cell product PM359. Prime Medicine’s in vivo research seeks to deliver prime-editing systems directly to tissues affected by genetic disease, including the liver. A major milestone came in 2024 when the United States Food and Drug Administration accepted Prime Medicine’s investigational new drug application for PM359. The therapy is designed for chronic granulomatous disease caused by mutations in the NCF1 gene, which encodes the p47phox component of the neutrophil respiratory burst pathway. PM359 also received Rare Pediatric Disease and Orphan Drug designations from the FDA. A Phase 1/2 open-label study began in October 2024, making PM359 the first prime-editing therapy to enter clinical testing according to the company’s cited reports. Prime Medicine has also pursued collaborations to expand the use of its platform. In January 2024, it announced an agreement with the Cystic Fibrosis Foundation to support prime-editing therapies for cystic fibrosis. In September 2024, the company announced a collaboration with Bristol Myers Squibb focused on optimized prime-editing reagents for ex vivo T-cell therapies. These collaborations place Prime Medicine within both the rare-disease gene-therapy field and the broader cell-therapy technology ecosystem. In May 2025, Prime Medicine said it would prioritize clinical development programs for Wilson’s disease and alpha-1 antitrypsin deficiency, two genetic liver disorders. This represented a concentration of development resources on liver-directed applications in addition to the company’s hematopoietic stem-cell program. Preliminary PM359 results were announced in May 2025, and later reports in December 2025 described two treated chronic granulomatous disease patients as effectively cured. Those findings remain part of the early clinical-development record and require interpretation alongside longer-term follow-up, additional patients, and regulatory review. Prime Medicine’s positioning is based on the potential precision, versatility, and broader editing range of prime editing. As with other clinical-stage gene-editing companies, its progress depends on demonstrating durable benefit, reliable delivery, manufacturing consistency, and an acceptable safety profile in human studies. The company remains an active developer rather than a mature commercial pharmaceutical brand, and its principal offerings are investigational therapies and genome-editing technologies rather than approved products.
History
Prime Medicine emerged as a biotechnology company dedicated to developing therapies based on prime editing. Prime editing is a genome-editing approach intended to install targeted DNA changes through a programmable guide and reverse-transcriptase system. In contrast with editing methods that commonly depend on creating double-strand DNA breaks, prime editing is designed to make more precise substitutions, insertions, and deletions. Prime Medicine’s corporate focus has been to convert this platform into therapeutic programs for inherited disorders. The company’s early development strategy combined disease-specific programs with platform partnerships. Its work has included ex vivo modification of a patient’s own blood-forming stem cells, as well as research into delivery of editing systems to organs such as the liver. The ex vivo approach is represented by PM359, which uses autologous CD34-positive stem cells edited outside the body before reinfusion. The program targets chronic granulomatous disease associated with mutations in NCF1, a gene involved in the function of immune-cell respiratory burst machinery. In January 2024, Prime Medicine announced a partnership with the Cystic Fibrosis Foundation to support development of prime-editing treatments for cystic fibrosis. The collaboration broadened the company’s disease focus beyond blood disorders and connected its platform with a major disease-specific research and advocacy organization. In September of the same year, Prime Medicine announced a collaboration with Bristol Myers Squibb to develop optimized prime-editing reagents for ex vivo T-cell therapies. This agreement linked Prime Medicine’s editing technology with Bristol Myers Squibb’s capabilities in cellular immunotherapy and expanded the potential application of the platform beyond direct treatment of inherited disease. A central regulatory turning point occurred in May 2024, when the FDA accepted Prime Medicine’s IND application for PM359. The agency also granted the program Rare Pediatric Disease and Orphan Drug designations. A Phase 1/2 open-label trial began in October 2024. PM359 was described as the first prime editor to reach clinical testing, giving the program significance beyond its individual disease indication: it served as an initial human test of the company’s platform, manufacturing process, delivery method, and editing strategy. In May 2025, Prime Medicine announced a sharper clinical-development focus on Wilson’s disease and alpha-1 antitrypsin deficiency. Both conditions involve genetic defects affecting the liver, making them relevant to the company’s efforts to develop liver-directed editing therapies. The prioritization indicated a strategy of concentrating resources on selected clinical opportunities while continuing to evaluate the wider prime-editing pipeline. The company announced preliminary PM359 results in May 2025. In December 2025, Prime Medicine and clinical collaborators reported that two patients treated with PM359 had been effectively cured of chronic granulomatous disease. Such reports represent an important early signal for the platform, but the clinical significance of the findings depends on the durability of correction, the number and characteristics of treated patients, safety monitoring, and subsequent regulatory and scientific review. Prime Medicine remains a clinical-stage biotechnology company. Its principal assets are investigational programs and enabling editing technologies rather than marketed medicines. Its long-term prospects depend on whether prime editing can deliver durable, clinically meaningful genetic correction while meeting the safety, manufacturing, and delivery requirements of gene therapy. The company’s collaborations and disease prioritization show an effort to build a portfolio spanning rare disease, liver-directed therapy, cystic fibrosis, and engineered immune-cell applications.
- 2025
Clinical priorities shift toward genetic liver diseases
[1]Prime Medicine prioritized development programs for Wilson’s disease and alpha-1 antitrypsin deficiency.
- 2024
FDA accepts PM359 IND application
[1]The FDA accepted Prime Medicine’s investigational new drug application for PM359, an ex vivo prime-edited autologous stem-cell therapy for NCF1-related chronic granulomatous disease.
- 2024
PM359 receives FDA special designations
[1]PM359 received Rare Pediatric Disease and Orphan Drug designations from the FDA.
- 2024
First prime-editing therapy enters clinical testing
[1]The Phase 1/2 PM359 study began in October, marking the first reported entry of a prime-editing therapy into the clinic.
Products and positioning
A clinical-stage biotechnology company specializing in precise gene correction and gene therapy using prime-editing technology, with programs spanning ex vivo stem-cell treatment, inherited diseases, liver disorders, and cell-therapy reagents.
Ex vivo gene therapy · 2024
PM359
PM359 is an investigational autologous cell therapy for chronic granulomatous disease caused by mutations in NCF1. Patient-derived CD34-positive hematopoietic stem cells are modified outside the body using prime editing and then intended for transplantation back into the patient. The program entered a Phase 1/2 open-label clinical trial in October 2024 and was the first reported prime-editing therapy to reach clinical testing. It is not described here as an approved treatment.
In vivo gene therapy · 2025
Wilson’s disease program
Prime Medicine identified Wilson’s disease as one of the genetic liver-disease programs it would prioritize for clinical development. The approach is intended to use prime editing to address the underlying genetic cause, although the supplied reference material does not specify a product code, clinical stage, delivery system, or approved product status.
In vivo gene therapy · 2025
Alpha-1 antitrypsin deficiency program
Prime Medicine selected alpha-1 antitrypsin deficiency as a priority clinical-development area in 2025. The program belongs to the company’s broader effort to apply prime editing to inherited liver diseases. The supplied reference does not provide a product name, clinical-stage designation, delivery details, or evidence of regulatory approval.
Brand decisions
2025
Prioritization of genetic liver-disease programsStrategyBackground
Prime Medicine was developing multiple applications of its prime-editing platform, including programs for inherited liver disorders.
Action taken
The company announced that it would prioritize clinical development of programs for Wilson’s disease and alpha-1 antitrypsin deficiency.
Aftermath
The decision concentrated the company’s clinical strategy on two genetic liver diseases while PM359 continued as its clinical-stage chronic granulomatous disease program.
Recent events
- 2025
Prime Medicine prioritizes Wilson’s disease and alpha-1 antitrypsin deficiency programs[1]
Other
Prime Medicine announced that it would prioritize clinical development programs for Wilson’s disease and alpha-1 antitrypsin deficiency, both genetic liver diseases.
- 2025
Prime Medicine reports preliminary PM359 results[1]
Other
The company announced preliminary results from the PM359 clinical program in chronic granulomatous disease.
- 2025
Reports describe two PM359 patients as effectively cured[1]
Other
Prime Medicine and its clinical collaborators reported that two patients treated with PM359 for chronic granulomatous disease had been effectively cured. The finding is an early clinical result requiring continued follow-up and broader validation.
- 2024
FDA accepts investigational new drug application for PM359[1]
Product launch · Regulation
Prime Medicine announced that the FDA had accepted its IND application for PM359, an autologous CD34-positive stem-cell therapy using ex vivo prime editing for chronic granulomatous disease caused by NCF1 mutations. The program also received Rare Pediatric Disease and Orphan Drug designations.
- 2024
PM359 clinical trial begins[1]
Product launch · Product generation
A Phase 1/2 open-label clinical study of PM359 began in patients with NCF1-related chronic granulomatous disease. The program was described as the first prime-editing therapy to enter clinical testing.
- 2024
Prime Medicine announces cystic fibrosis collaboration[1]
M&A
Prime Medicine entered a partnership with the Cystic Fibrosis Foundation to support development of prime-editing therapies for cystic fibrosis.
- 2024
Prime Medicine and Bristol Myers Squibb announce cell-therapy collaboration[1]
M&A
The companies announced a collaboration to develop optimized prime-editing reagents for ex vivo T-cell therapies.
Sources2 sources
Cite this profile: Cite the canonical profile. /brand-wiki/prime-medicine · Editorial policy · How profiles are compiled