What Rznomics does

Rznomics Inc. is a South Korean clinical-stage biopharmaceutical company founded in 2017 and headquartered in Seongnam, South Korea, that develops RNA editing-based gene therapies built on its proprietary RNA trans-splicing ribozyme platform. Unlike DNA-level editing approaches, the platform performs precise genetic corrections at the RNA level via trans-splicing, theoretically offering improved safety by avoiding permanent genomic alteration. Its lead investigational candidate, RZ-001, is in Phase 1/2a development for hepatocellular carcinoma (HCC) and recurrent glioblastoma; interim HCC data presented at AACR 2026 showed a 61.5% objective response rate and 23% complete response rate (mRECIST) when RZ-001 was combined with atezolizumab and bevacizumab, with no Grade 3+ adverse events attributed to RZ-001, while the Phase 1/2a glioblastoma trial enrolled 10 patients with no dose-limiting toxicity and disease control exceeding six months in several patients. RZ-001 has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation in HCC, providing accelerated regulatory pathways including priority and rolling review and accelerated approval eligibility.

The company monetizes primarily through licensing and milestone-based partnerships with major pharmaceutical companies rather than direct product sales. In May 2026 Rznomics signed a global licensing agreement with Eli Lilly valued at up to $1.3 billion (upfront, research funding, and milestones) for hereditary hearing loss treatment development, marking entry into full implementation of joint R&D. It has also signed a strategic MOU with GluGene Therapeutics to combine its RNA editing platform with GluGene's AI-based AAV vector discovery technology for next-generation gene therapy delivery. Rznomics became a publicly listed company on KOSDAQ (ticker 476830) in December 2025, raising approximately $31 million, having previously raised approximately $64 million across Series A, B, and C rounds (2019–2024). Customer segments are organized vertically by indication: HCC patients, recurrent glioblastoma patients, and hereditary hearing loss patients, with the company operating in South Korea, the United States, and Japan.

Name

Rznomics

Legal name

Rznomics Inc.

Website

https://rznomics.com

Company type

Public

Founded year

2017

Operating status

Operating

Headcount range

11–50 employees

Short description

Rznomics Inc. is a South Korean clinical-stage biopharmaceutical company developing RNA editing-based gene therapies built on its proprietary trans-splicing ribozyme platform, with lead candidate RZ-001 in trials for hepatocellular carcinoma and glioblastoma.

Ownership category

akta.pro rank

Headquarters

HQ city

Seongnam

HQ country

South Korea

HQ region

Asia

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Licensing and Milestone Payments: Rznomics generates revenue through licensing agreements with pharmaceutical companies, including upfront payments, research funding, and milestone-based payments tied to development, regulatory, and commercial achievements. The company received research funding from Eli Lilly and entered full implementation phase of joint R&D.
2. Therapeutic Product Revenue: Future revenue expected from commercialization of RNA editing-based therapeutics following regulatory approval. The company is advancing RZ-001 through clinical development for hepatocellular carcinoma and glioblastoma, with plans to leverage FDA RMAT designation for accelerated approval pathways.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels3 records

Core offering

Rznomics is a South Korean clinical-stage biopharmaceutical company developing RNA editing-based gene therapies, with lead candidate RZ-001 in Phase 1/2a trials for hepatocellular carcinoma and recurrent glioblastoma. The company operates a proprietary RNA trans-splicing ribozyme platform that enables precision RNA editing without permanent DNA alterations, and monetizes its pipeline primarily through licensing deals and co-development partnerships with major pharmaceutical companies such as Eli Lilly.

Product overview

Rznomics is a South Korean biopharmaceutical company developing RNA-based gene therapies. The company's lead product is RZ-001, an RNA editing-based anticancer gene therapy currently in Phase 1/2a clinical trials for recurrent glioblastoma and hepatocellular carcinoma. The therapy is built on Rznomics' proprietary RNA trans-splicing ribozyme platform technology, which enables precision editing of RNA molecules for therapeutic purposes. The company has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for RZ-001 in hepatocellular carcinoma.

Differentiator

Problem solved

Functional benefit

Products and services

• RZ-001 Investigational RNA editing-based anticancer gene therapy utilizing the proprietary RNA trans-splicing ribozyme platform for precision RNA editing in patients with hepatocellular carcinoma and recurrent glioblastoma.
• RNA Trans-Splicing Ribozyme Platform Proprietary RNA editing technology platform enabling precise genetic corrections via trans-splicing mechanisms, licensed to pharmaceutical partners for therapeutic applications across cancer and genetic diseases.

Quantifiable outcome

• 61.5% objective response rate with 23% complete response rate (mRECIST criteria) in hepatocellular carcinoma patients
• +2 more outcomes

Segments3 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Two partnerships are on record, tiered core and flagship.

• GluGene TherapeuticscoreTechnology or Integration · 1 February 2026Strategic MOU signed to jointly develop next-generation RNA gene therapies using adeno-associated virus (AAV) delivery platforms. The collaboration combines Rznomics' RNA trans-splicing enzyme-based precision editing platform with GluGene's AI-based AAV vector discovery technology, aiming to overcome technical challenges including transgene size capacity, tissue specificity, immune responses, and low-dose delivery efficiency.
• Eli LillyflagshipStrategic or Co-development Partner · 1 May 2025Global license agreement with Eli Lilly for hereditary hearing loss treatment development, valued at over $1.3 billion. The partnership includes research funding separate from upfront payments and marks entry into full implementation phase of joint R&D. Rznomics plans to use funds to advance its RNA correction platform and hearing loss program, with potential expansion into central nervous system diseases.

Scale indicators5 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Beam Therapeutics: Clinical-stage biotechnology company developing base editing therapies that chemically convert one DNA letter to another without double-stranded breaks. Like Rznomics, Beam operates in the precision genetic medicine space, pursuing targeted editing approaches for serious diseases with platform-based pipeline expansion.
• Korro Bio: Biotechnology company developing RNA editing therapeutics using ADAR-mediated single-base editing. Directly comparable to Rznomics' RNA editing approach via a different enzymatic mechanism; both pursue indication-agnostic platform strategies for genetic diseases.
• Ascidian Therapeutics: Clinical-stage company using RNA editing to treat genetic diseases by reprogramming RNA. Ascidian shares Rznomics' focus on RNA-level rather than DNA-level editing, making it a direct technology comparable in modality and therapeutic ambition.
• ProQR Therapeutics: RNA therapeutics company developing Axiomer RNA editing technology and other RNA-targeting platforms. ProQR shares Rznomics' strategic positioning around RNA-based precision medicines and pursues similar rare disease and genetic disorder indications.
• Wave Life Sciences: Clinical-stage company leveraging proprietary PRISM platform for RNA-targeted therapeutics including RNA editing. Wave's siRNA and RNA editing programs overlap with Rznomics' trans-splicing approach in modality and target disease areas.
• Intellia Therapeutics: Clinical-stage gene editing company developing in vivo CRISPR/Cas9-based therapies. Comparable to Rznomics as a peer in the precision genomic medicine space, though Intellia uses DNA-level editing while Rznomics edits RNA.
• Editas Medicine: Clinical-stage company developing CRISPR-based genome editing therapies for serious diseases. Comparable in business model (clinical-stage gene medicine developer) and therapeutic ambition, though Rznomics differentiates via RNA editing.
• CRISPR Therapeutics: Leading clinical-stage gene editing company with approved therapy (Casgevy) and broad pipeline across hemoglobinopathies, oncology, and regenerative medicine. Comparable as a clinical-stage gene medicine company with platform technology approach.
• Verve Therapeutics: Clinical-stage company developing in vivo base editing therapies for cardiovascular disease. Comparable as a clinical-stage precision gene medicine developer pursuing platform-based indication expansion, with similar partnership-driven commercialization model.
• Sangamo Therapeutics: Clinical-stage genomic medicine company developing zinc finger nuclease-based gene editing and gene therapy. Comparable as a clinical-stage platform company in genomic medicine with multiple pipeline programs and partnership-based commercialization.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat2 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/rznomics

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds5 records

Investors19 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

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

Frequently asked questions about Rznomics