What Prime Medicine does

Prime Medicine, Inc. is a clinical-stage biotechnology company founded in 2019/2020 and headquartered at 21 Erie Street in Cambridge, Massachusetts, that develops Prime Editing-based gene editing therapies for rare genetic diseases. The company's core platform, Prime Editing, was developed in Dr. David R. Liu's lab at the Broad Institute of MIT and Harvard and published in Nature in December 2019; it acts as a "DNA word processor" to search and replace genetic sequences at their native genomic location without inducing double-strand DNA breaks, theoretically addressing approximately 90% of known disease-causing mutations. The product portfolio spans three therapeutic areas: hematology/immunology/oncology (PM359, an ex vivo autologous HSC therapy for p47phox Chronic Granulomatous Disease, and ex vivo CAR-T programs partnered with Bristol Myers Squibb), liver diseases (PM577a for H1069Q-mutated Wilson Disease via LNP delivery, and PM647 for Alpha-1 Antitrypsin Deficiency), and lung disease (a cystic fibrosis program supported by Cystic Fibrosis Foundation funding targeting hotspot editing and the PASSIGE™ platform extension). Two proprietary technology modules extend the platform: PASSIGE™ combines Prime Editing with integrase enzymes for gene-sized insertions, and Dual Flap (twin Prime Editors) enables deletions, large hotspot corrections, exon replacements, and sequence insertions without double-strand breaks.

The company is pre-revenue with no approved products; FY2025 net loss was approximately $201.1 million against a cash position of $191.4 million at year-end (and $149.2 million at end of Q1 2026), funded by a $315 million Series A-B round in 2021 (led by ARCH Venture Partners and GV), a $110 million upfront from the BMS ex vivo T-cell collaboration plus potential milestones exceeding $3.5 billion, approximately $39 million in cumulative Cystic Fibrosis Foundation grants, and a $144 million public offering in July 2025. Q1 2026 collaboration revenue was $4.6 million (up 55.3% year-over-year). The go-to-market is enterprise-pharma partnerships (BMS handles manufacturing and commercialization for ex vivo programs) and clinical trial execution for wholly-owned in vivo programs, with regulatory designations accumulated on PM359 including FDA RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease status. The company underwent a May 2025 strategic restructuring with a 25% workforce reduction, pivoting focus to liver diseases, cystic fibrosis, and partnered programs; clinical milestones include the first-in-human NEJM publication of Prime Editing data in December 2025 and New Zealand CTA clearance for PM577a in June 2026 marking the first in vivo Prime Editing clinical authorization.

Name

Prime Medicine

Legal name

Prime Medicine, Inc.

Website

https://primemedicine.com

Company type

Public

Founded year

2020

Operating status

Operating

Headcount range

101–250 employees

Short description

Prime Medicine is a clinical-stage biotechnology company developing Prime Editing-based gene therapies for rare genetic diseases across hematology, liver, and lung indications, operating through pharma partnerships and clinical trials.

Ownership category

akta.pro rank

Product category

Gene Editing Biotechnology and Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Scientific Research and Development Services (5417)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Oncology Gene Therapies (incl. oncolytic vectors & gene-modified approaches) (HLAAACAF), Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI)

Keywords

• Gene editing therapies
• Prime editing platform
• Rare genetic diseases
• Cell therapy development
• Biotechnology research

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Strategic Collaboration Revenue: Prime Medicine receives upfront payments, research funding, and potential milestone payments from pharmaceutical partners. The Bristol Myers Squibb collaboration includes $110 million upfront and potential milestones exceeding $3.5 billion for ex vivo T-cell therapies.
2. Foundation Funding: Non-dilutive funding from disease foundations supporting research programs. The Cystic Fibrosis Foundation provided up to $15 million initially and committed an additional $24 million for cystic fibrosis program development.
3. Future Product Revenue: Pre-revenue company. Future revenue anticipated from commercialized gene editing therapies, though no products are currently approved. Q1 2026 revenue was $4.6 million (up 55.3%), likely from collaborations.
4. Equity Financing: Raised $315 million through Series A and Series B funding rounds in 2021. Additional public equity offerings in 2025 raised approximately $144 million.

Go-to-market motion2 records

Distribution channels3 records

Marketing channels5 records

Core offering

Prime Medicine develops investigational one-time curative gene editing therapies using its proprietary Prime Editing platform, which can search and replace disease-causing DNA sequences without double-strand breaks. The company is advancing clinical-stage programs for Chronic Granulomatous Disease (PM359), Wilson Disease (PM577), Alpha-1 Antitrypsin Deficiency (PM647), Cystic Fibrosis, and an ex vivo CAR-T cell therapy program partnered with Bristol Myers Squibb.

Product overview

Prime Medicine is a biotechnology company developing a diversified portfolio of investigational gene editing therapies organized around core areas: hematology/immunology/oncology, liver, and lung diseases. The company's core technology is the Prime Editing platform, a versatile gene editing approach that acts like a DNA word processor to search and replace genetic sequences. The portfolio includes clinical-stage programs: PM359 (ex vivo therapy for Chronic Granulomatous Disease), PM577 (in vivo liver therapy for Wilson Disease), PM647 (in vivo liver therapy for Alpha-1 Antitrypsin Deficiency), plus cystic fibrosis and CAR-T programs. Key technology modules include PASSIGE™ for gene-sized insertions and Dual Flap Prime Editing for broader genomic edits. Prime Editing can theoretically address about 90% of known disease-causing mutations across many organisms, organs and cell types, offering potentially curative one-time therapies.

Differentiator

Problem solved

Functional benefit

Brands

• PASSIGE: Prime Assisted Site Specific Integrase Gene Editing - a proprietary therapeutic approach combining Prime Editing with integrase enzymes to insert gene-sized sequences precisely in the genome.

Products and services

• PM359 Investigational ex vivo autologous hematopoietic stem cell therapy using Prime Editing to treat p47phox-deficient Chronic Granulomatous Disease (CGD), delivered as an all-RNA Prime Editor via electroporation.
• PM577 (Wilson Disease Program) Investigational in vivo liver-directed Prime Editing therapy targeting the ATP7B gene (H1069Q mutation) for Wilson Disease, delivered via lipid nanoparticle (LNP) technology.
• PM647 (Alpha-1 Antitrypsin Deficiency Program) Investigational in vivo liver-directed Prime Editing therapy for Alpha-1 Antitrypsin Deficiency (AATD), delivered via lipid nanoparticle (LNP) technology with preclinical data showing 95% protein restoration.
• Cystic Fibrosis Program Investigational lung-directed Prime Editing program for Cystic Fibrosis targeting CFTR mutations using hotspot editing and PASSIGE approaches, with initial focus on the G542X mutation.
• Ex Vivo CAR-T Cell Therapy Program Ex vivo T-cell therapy program using Prime Editing for CAR-T products, co-developed with Bristol Myers Squibb under a strategic collaboration where BMS handles development, manufacturing, and commercialization while Prime Medicine designs the gene editing reagents.

Quantifiable outcome

• PM359 achieved 66% DHR positivity by Day 30 in first patient (May 2025), exceeding the 20% threshold considered potentially curative. Second patient achieved 83% DHR neutrophils.
• +3 more outcomes

Named customers3 records

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

One partnership is on record.

• Bristol Myers SquibbflagshipStrategic or Co-development Partner · 30 September 2024Strategic collaboration and license agreement to develop and commercialize ex vivo T-cell therapies using Prime Editing technology. Prime Medicine designs gene editing reagents while Bristol Myers Squibb handles development, manufacturing, and commercialization. Prime receives $110 million upfront with potential milestones exceeding $3.5 billion. This is a major validation of Prime Editing technology for cell therapy applications.

Scale indicators8 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Beam Therapeutics: Beam Therapeutics is a direct peer developing base editing, a closely related next-generation gene editing modality that also avoids double-strand DNA breaks. Both companies emerged from the David Liu / Broad Institute ecosystem and target similar monogenic and rare disease indications, including sickle cell disease and other hematologic conditions.
• CRISPR Therapeutics: CRISPR Therapeutics is a direct peer and pioneer of CRISPR/Cas9-based gene editing therapies, with CASGEVY (exa-cel) becoming the first FDA-approved CRISPR therapy. Like Prime Medicine, it focuses on ex vivo and in vivo gene editing for rare genetic diseases and has established major pharma partnerships (Vertex).
• Editas Medicine: Editas Medicine is a direct peer developing CRISPR/Cas9 and CRISPR/Cas12a gene editing therapies for serious diseases, including sickle cell disease and inherited retinal diseases. It competes with Prime Medicine for talent, partner attention, and clinical milestones in the gene editing field.
• Intellia Therapeutics: Intellia Therapeutics is a direct peer developing both ex vivo and in vivo CRISPR/Cas9 gene editing therapies, including programs for transthyretin amyloidosis and hereditary angioedema. Its in vivo LNP delivery approach for liver targets directly parallels Prime Medicine's liver-directed Prime Editing programs.
• Verve Therapeutics: Verve Therapeutics is a direct peer developing in vivo base editing therapies for cardiovascular disease using LNP delivery. Like Prime Medicine's in vivo programs, it leverages lipid nanoparticle delivery to the liver and pursues one-time curative treatments, making its modality and delivery mechanism highly comparable.
• Sangamo Therapeutics: Sangamo Therapeutics is a direct peer pioneering zinc finger nuclease (ZFN) gene editing and gene therapy for rare genetic diseases including hemophilia and lysosomal storage disorders. It is among the longest-operating clinical-stage genome editing companies, providing a comparable reference point for Prime Medicine's modality-focused pipeline.

Broad incumbents

• bluebird bio: bluebird bio is a broad incumbent in ex vivo gene therapy with approved products for beta-thalassemia, sickle cell disease, and cerebral adrenoleukodystrophy. Its ex vivo lentiviral gene therapy approach overlaps with Prime Medicine's ex vivo HSC program (PM359 for CGD), and Prime Medicine's CMO previously led clinical development at bluebird bio.

Others

• Moderna: Moderna is an adjacent player whose LNP delivery platform technology overlaps with Prime Medicine's PM577 and PM647 in vivo liver-directed programs. While Moderna's primary focus is mRNA vaccines and therapeutics rather than gene editing, it represents a relevant reference for LNP manufacturing scale-up and a potential future partner or competitor for in vivo delivery.
• Alnylam Pharmaceuticals: Alnylam Pharmaceuticals is an adjacent peer in nucleic acid-based therapeutics, pioneering RNAi-based therapies for rare genetic diseases including hereditary ATTR amyloidosis and primary hyperoxaluria. It competes with Prime Medicine in liver-directed genetic disease treatment paradigms and LNP/GalNAc delivery approaches.
• Vertex Pharmaceuticals: Vertex Pharmaceuticals is a strategic partner of Prime Medicine (BMS competing in CF space; also historically a partner for CASGEVY with CRISPR Therapeutics). Vertex's CF franchise (TRIKAFTA) represents both a near-term competitive threat and a potential commercialization partner for Prime Medicine's cystic fibrosis program.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xtwitter.com/PrimeMedicine
• LinkedInlinkedin.com/company/prime-medicine

Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles16 records

Funding overview

Funding rounds7 records

Investors14 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Prime Medicine