What RheumaGen does

RheumaGen is a clinical-stage biotechnology company developing a class of one-time, curative cell and gene therapies for treatment-resistant autoimmune diseases. The company's core technology is precision HLA gene editing: by editing specific markers on the human leukocyte antigen (HLA) gene, the therapy aims to convert a patient's harmful autoimmune-predisposing HLA alleles into healthy, disease-resistant ones while preserving the rest of the immune system, in contrast to broadly immunosuppressive standard-of-care biologics. The lead asset, RG0401, targets the 10-20% of rheumatoid arthritis patients who are refractory to existing therapies, with a Phase I clinical trial planned for 2027. Additional pipeline programs extend the platform to multiple sclerosis, type 1 diabetes, celiac disease, and ankylosing spondylitis, all autoimmune indications with established HLA epidemiological linkages.

Underlying technology rests on two proprietary advances: anchor-position editing (a single amino acid edit at position 82 of an HLA molecule that blocks presentation of three critical self-peptides in RA) and broader pocket engineering of HLA alleles. Manufacturing and clinical development are enabled through a long-standing partnership with ClinImmune Center for Clinical Immunology at the University of Colorado Anschutz Medical Campus, which operates a 25,000-square-foot cGMP facility. In October 2025, RheumaGen expanded its delivery options via a licensing and joint development agreement with SiVEC Biotechnologies, integrating the BactPac in vivo bacterial delivery platform and co-developing an in vivo HLA gene-editing program.

RheumaGen generates no current product revenue. Its business model is built around future commercialization of approved one-time cell and gene therapies at premium pricing typical of advanced therapies, supplemented by technology licensing and strategic partnerships. The company is privately held, headquartered in Aurora, Colorado, with a wholly owned Australian subsidiary (RheumaGen Australia) responsible for Phase I execution and commercialization in Australia. The go-to-market motion is enterprise-focused, targeting specialized treatment centers and academic medical centers for clinical trial enrollment and eventual administration of approved therapies. To date, the company has raised approximately $15 million in Series A financing (2025) plus earlier grants and a Series A-1 investment from Beyond Celiac Investments for the celiac program.

Name

RheumaGen

Legal name

RheumaGen, Inc.

Website

https://rheumagen.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

1–10 employees

Short description

RheumaGen is a clinical-stage biotechnology company engineering one-time, curative HLA gene-editing cell and gene therapies for treatment-resistant autoimmune diseases. Its lead program RG0401 targets refractory rheumatoid arthritis, with additional pipeline programs in multiple sclerosis, type 1 diabetes, celiac disease, and ankylosing spondylitis.

Ownership category

akta.pro rank

Product category

Cell and Gene Therapy for Autoimmune Diseases

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Research and Development in the Physical, Engineering, and Life Sciences (54171)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Cell Therapy Developers (Autologous) (HLAAABAA)

akta.pro secondary industries

Rare Immunology, Autoimmune & Autoinflammatory Therapies (HLAIAIAE), Immunology & Autoimmune Specialty Pharmaceuticals (HLAIACAB)

Keywords

• Cell gene therapy
• HLA gene editing
• Autoimmune therapeutics
• Rheumatoid arthritis treatment
• Precision genome editing

Headquarters

HQ city

Aurora

HQ country

United States

HQ region

North America

Offices2 records

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. Cell and Gene Therapy Commercialization: Pre-commercial stage company developing one-time curative cell and gene therapies. Revenue will be generated through commercialization of approved therapies, likely high-value treatments for rare/refractory autoimmune diseases with substantial pricing typical of advanced therapies.
2. Licensing and Partnerships: Technology licensing and strategic partnerships with larger pharmaceutical companies for development and commercialization rights in specific indications or territories.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels6 records

Core offering

RheumaGen is a clinical-stage biotechnology company developing one-time, curative cell and gene therapies that edit the human leukocyte antigen (HLA), or "immune gene," to cure autoimmune diseases at their source. The company's lead program, RG0401, targets treatment-resistant rheumatoid arthritis, with additional pipeline programs for multiple sclerosis, type 1 diabetes, celiac disease, and ankylosing spondylitis.

Product overview

RheumaGen is engineering a new class of cell and gene therapies to cure major autoimmune diseases. The company's core offering is its HLA Gene-Editing Therapy Platform, which enables precision editing of the human leukocyte antigen (HLA) to create one-time, curative treatments. The lead product is RG0401, an HLA gene-editing therapy for treatment-resistant rheumatoid arthritis currently in IND-enabling studies with Phase I trial planned for 2027. The platform supports a pipeline of programs including RG0401 for RA, plus development programs for multiple sclerosis, type 1 diabetes, celiac disease, and ankylosing spondylitis — all autoimmune diseases where HLA alleles play a critical epidemiological role.

Differentiator

Problem solved

Functional benefit

Products and services

• RG0401 One-time, curative HLA gene-editing therapy targeting treatment-resistant or refractory rheumatoid arthritis (RA). RG0401 makes a patient's HLA molecules mirror those of a person resistant to RA by editing a DNA marker(s) of the HLA gene, preventing T cells from activating chronic autoimmune responses. Targets the 10-20% of RA patients who are refractory or treatment-resistant.
• HLA Gene-Editing Therapy Platform A breakthrough cell and gene therapy platform that edits the human leukocyte antigen (HLA), or 'immune gene,' to develop one-time, curative treatments for autoimmune diseases. The platform enables precision editing to make a patient's HLA molecules mirror those of a disease-resistant individual while maintaining the rest of the immune system as normal.
• Multiple Sclerosis Program HLA gene-editing therapy in development for progressive forms of multiple sclerosis, targeting autoimmune diseases where HLA alleles play a critical epidemiological role.
• Type 1 Diabetes Program

Quantifiable outcome

• Anchor-position editing at amino acid 82 substantially blocks three of the most critical self-peptides in rheumatoid arthritis (both citrullinated peptides and collagen) from being presented to T cells
• +1 more outcomes

Segments5 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Three partnerships are on record, tiered core and minor.

• SiVEC BiotechnologiescoreStrategic or Co-development Partner · 7 October 2025Licensing and joint development agreement utilizing SiVEC's BactPac drug delivery platform to streamline manufacturing and increase scalability of RheumaGen's HLA gene-editing therapies. The partnership also establishes an in vivo program co-developed by both companies. Richard Freed, CEO of RheumaGen, was appointed to SiVEC's Board of Directors. Both companies are Colorado-based, collaborating to develop cost-effective, scalable, and accessible therapies.
• University of Colorado Anschutz Medical Campus / ClinImmunecoreStrategic or Co-development PartnerRheumaGen works closely with ClinImmune Center for Clinical Immunology at CU Anschutz Medical Campus for clinical development and manufacturing. ClinImmune, founded by Dr. Brian Freed, operates a state-of-the-art 25,000-square-foot cGMP facility and has facilitated more than 10,000 organ and stem-cell transplants with 30 active cell therapy trials and 2 FDA licenses. The partnership enables RheumaGen to expeditiously advance programs through CGT regulatory approval processes.
• Prof. Georg SchettminorOthersScientific Advisory Board member and Vice President of Research at Friedrich-Alexander-Universität Erlangen-Nürnberg. Dr. Schett is an internationally recognized rheumatologist who pioneered CAR T-cell therapy in refractory systemic lupus, paving the way for autoimmune cell therapy. He has published more than 750 articles in peer-reviewed journals.

Scale indicators7 records

Recent moves7 records

Expansion highlights6 records

Broad incumbents

• Editas Medicine: Clinical-stage gene-editing company developing CRISPR-based therapies for genetic diseases and oncology. Comparable as a gene-editing incumbent with overlapping editing modality, though RheumaGen's HLA-anchor mechanism is differentiated.
• Beam Therapeutics: Clinical-stage base-editing company developing precision genetic medicines for hematologic and other diseases. Comparable as a next-generation gene-editing incumbent, with adjacent precision-editing approach relevant to RheumaGen's anchor-position editing.
• Atara Biotherapeutics: Broader cell therapy company with allogeneic T-cell and CAR-T programs across oncology and autoimmune indications. Comparable as a cell-therapy incumbent pushing into autoimmune disease, with greater scale and platform breadth than RheumaGen.
• CRISPR Therapeutics: Leading gene-editing company with clinical-stage programs across hemoglobinopathies, oncology, and regenerative medicine. Comparable as a CRISPR-based gene-editing incumbent, though focused on ex vivo editing of hematopoietic stem cells rather than HLA-specific anchor editing.
• Fate Therapeutics: Clinical-stage cell therapy platform company developing off-the-shelf iPSC-derived NK and T-cell therapies for cancer and autoimmune disease. Comparable as a cell-therapy platform player, though with broader oncology focus and a different modality.

Direct peers

• Kyverna Therapeutics: Clinical-stage cell therapy company developing autologous CAR-T therapies for autoimmune diseases including lupus, myositis, and MS. Directly comparable in cell-therapy modality, autoimmune focus, and target patient populations.
• Sonoma Biotherapeutics: Developing regulatory T-cell (Treg) therapies for autoimmune and inflammatory diseases. Comparable in autologous cell-therapy approach and autoimmune disease focus, though targeting immune regulation via Tregs rather than HLA editing.
• Cabaletta Bio: Clinical-stage biotech developing CAR-T cell therapies for autoimmune diseases, including refractory rheumatoid arthritis and lupus. Most directly comparable to RheumaGen in target indications and autologous cell-therapy modality, though using a different engineered-receptor mechanism.
• Quell Therapeutics: Cell therapy company engineering Treg-based therapies for autoimmune and inflammatory diseases including liver transplant tolerance. Comparable in autologous cell-therapy modality and autoimmune focus, with overlap on indications like inflammatory bowel disease.

Emerging players

• Caribou Biosciences: Clinical-stage allogeneic CAR-T cell therapy company using CRISPR-based genome editing. Comparable as an emerging gene-edited cell-therapy player with overlapping editing modality, though focused on oncology rather than autoimmune indications.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks7 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/rheumagen

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Subsidiaries1 record

Funding overview

Funding rounds3 records

Investors3 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 RheumaGen