CAMBRIDGE, Mass. — March 3, 2026 — Leads & Copy —
Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company focused on developing one-time curative genetic therapies, has released its financial results for the year ended December 31, 2025, along with a business update. The company is on track to file Investigational New Drug (IND) and/or Clinical Trial Application (CTA) for Wilson Disease and Alpha-1 Antitrypsin Deficiency (AATD) programs in the first half of 2026 and mid-2026, respectively. Initial clinical data for both programs is anticipated in 2027.
Prime Medicine is also engaged with the U.S. Food and Drug Administration (FDA) regarding PM359 in Chronic Granulomatous Disease (CGD) and plans to submit a Biologics License Application (BLA) following final alignment with the agency. The company reported cash, cash equivalents, investments, and restricted cash of $191.4 million, providing a cash runway into 2027.
According to Allan Reine, M.D., Chief Executive Officer of Prime Medicine, the company is shaping the future of genetic medicine by advancing a gene editing technology. Prime Editing offers the opportunity to correct disease-causing mutations across a range of indications.
Dr. Reine stated that the company’s vision is to deliver on the promise of Prime Editing and ensure patient access to transformative therapies. The company believes the data supports an accelerated approval in the United States for PM359, its ex vivo Prime Edited autologous HSC product for CGD, for which breakthrough data was announced in 2025.
Prime Medicine is also progressing toward regulatory milestones for its liver-focused programs in Wilson Disease (WD) and AATD, including planned IND and CTA submissions and the initiation of Phase 1/2 clinical trials. The company continues to generate preclinical data across its portfolio, including its program for Cystic Fibrosis (CF); optimize modular delivery and manufacturing approaches; and explore collaborations to expand the reach of its platform. The company is progressing in vivo gene editing programs aimed at treating Wilson Disease (WD) and Alpha‑1 Antitrypsin Deficiency (AATD).
Prime Medicine anticipates submitting an investigational new drug (IND) and/or clinical trial application (CTA) for its anchor WD program (targeting the H1069Q mutation) in the first half of 2026, and plans to leverage the modularity of the Prime Editing platform to subsequently advance follow-on programs targeting other mutations, which collectively address a majority of WD patients. Prime Medicine expects to file an IND and/or CTA for its AATD program in mid-2026, and to report initial clinical data from both WD and AATD programs in 2027.
Following positive proof-of-concept data from the first two patients treated in its Phase 1/2 study of PM359 for the treatment of Chronic Granulomatous Disease (CGD), Prime Medicine is working to ensure this therapy is available for patients.
Prime Medicine is also progressing an in vivo Cystic Fibrosis (CF) program with support from the Cystic Fibrosis Foundation, and anticipates generating preclinical proof of concept data in 2026. Additionally, its efforts to develop Prime Edited CAR-T products for hematology, immunology and oncology continue in partnership with Bristol Myers Squibb.
Based on recent interactions with the FDA, Prime Medicine believes clinical data generated to-date may be sufficient to support an accelerated approval of PM359 and intends to submit a Biologics License Application (BLA).
In December 2025, Prime Medicine announced the publication of Phase 1/2 clinical data with PM359 in the New England Journal of Medicine (NEJM). The data, which were also presented at the 67th American Society of Hematology (ASH) Annual Meeting, showed rapid neutrophil and platelet engraftment, as well as durable restoration of NADPH oxidase activity and early clinical benefit, without any safety concerns.
R&D expenses were $160.6 million for the year ended December 31, 2025, as compared to $155.3 million for the year ended December 31, 2024. G&A expenses were $52.3 million for the year ended December 31, 2025, as compared to $50.2 million for the year ended December 31, 2024.
Net loss was $201.1 million for the year ended December 31, 2025, as compared to $195.9 million for the year ended December 31, 2024. As of December 31, 2025, cash, cash equivalents, investments, and restricted cash were $191.4 million, as compared to $204.5 million as of December 31, 2024.
Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and investments as of December 31, 2025 will be sufficient to fund its operating expenses and capital expenditure requirements into 2027.
Prime Medicine is deploying its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology. Prime Editors have the potential to repair almost all types of genetic mutations and work in many different tissues, organs and cell types.
Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around its core areas of focus: hematology, immunology and oncology, liver and lung.
Source: Prime Medicine
