ReviR Therapeutics

What ReviR Therapeutics does

ReviR Therapeutics is a privately held, clinical-stage biotechnology company founded in 2021 and headquartered at 150 North Hill Drive, Brisbane, California. The company develops orally bioavailable, small-molecule RNA modulators that target disease-relevant RNA — including mRNA and RNA structural elements — to modulate gene expression, reduce toxic protein accumulation, or correct splicing defects in rare neurogenetic and neurological diseases. Its lead clinical candidate, RTX-117, is an oral small molecule that activates eIF2B to modulate the Integrated Stress Response pathway and is being evaluated in Phase 1 first-in-human trials for Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM); it holds U.S. FDA Orphan Drug Designation and IND clearance, with parallel China CDE IND clearance. Additional earlier-stage programs target an mRNA pathway in Huntington's disease (funded by a $4.6M CIRM grant), an RNA modulator for SBMA / Kennedy's Disease (partnered with the Kennedy's Disease Association), and an oral splice modulator for CMT1A (co-developed with the CMT Research Foundation).

The company's core technology is the proprietary VoyageR Discovery Engine, which combines a proprietary RNA-targeting compound library, transcriptome-wide compound screening, and a VoyageR AI layer for drug and target discovery. VoyageR is integrated with XtalPi's AI+robotics drug discovery engine — XtalPi is also an incubator investor — and is being extended with an isogenic iPSC + AI drug-perturbation modeling platform for CMT subtype stratification, funded by CureCMT and intended to be opened to the broader CMT research community. ReviR's business model is pre-commercial: it has no products on the market and no recurring revenue, generating no licensing royalties or product sales today. Capital is sourced from a mix of equity (a 2024 $30M Series A led by Lapam Capital bringing total funding to $54M including a 2021/2022 seed) and non-dilutive foundation/government grants (CIRM, CMTRF, KDA, CureCMT). The go-to-market is partnership- and foundation-driven: disease-foundation co-development alliances, an AI+robotics platform partnership with XtalPi, and a dual U.S. (FDA) and China (CDE/NMPA) clinical strategy intended to leverage Orphan Drug Designation and future specialty-pharma partnerships for commercialization in the two largest pharmaceutical markets. Leadership includes CEO Peng Yue and Chief Scientific Officer Paul August; the company has 11–50 employees and no listed public ticker.

Name

ReviR Therapeutics

Legal name

ReviR Therapeutics

Website

https://revirtx.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

11–50 employees

Short description

ReviR Therapeutics is a clinical-stage, AI-enabled biotechnology company developing orally bioavailable, RNA-targeting small molecule therapies for rare neurogenetic diseases, with lead candidate RTX-117 in Phase 1 trials for Charcot-Marie-Tooth and Vanishing White Matter disease.

Ownership category

akta.pro rank

Product category

Rare Disease RNA-Targeting Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

siRNA Therapeutics (gene silencing) (HLAAADAB)

Keywords

• RNA-targeting drug discovery
• Small molecule therapeutics
• AI drug discovery
• Rare neurogenetic diseases
• Orphan drug development

Headquarters

HQ city

Brisbane

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

Revenue model

1. Research grants from disease foundations: Non-dilutive funding sourced from disease-focused foundations (CMT Research Foundation, Kennedy's Disease Association, CIRM) tied to specific program milestones. Not a recurring commercial revenue stream.
2. Venture capital financing (equity): Capital raised through priced equity rounds; $30M Series A in July 2024 led by Lapam Capital, bringing total funding to $54M including 2021 seed. Not commercial revenue.
3. Future: Out-licensing / commercialization of small molecule therapeutics: Pre-commercial clinical-stage biotech. RTX-117 has FDA Orphan Drug Designation and is in Phase 1 trials (CMT, Vanishing White Matter). Future revenue expected from drug commercialization or out-licensing of pipeline assets; no current product revenue.

Go-to-market motion2 records

Distribution channels2 records

Marketing channels3 records

Core offering

ReviR Therapeutics is an AI-enabled biotechnology company that develops orally bioavailable, RNA-targeting small molecule therapeutics for rare neurogenetic diseases. Its proprietary VoyageR Discovery Engine combines a proprietary RNA-targeting compound library, transcriptome-wide compound screening, and VoyageR AI to identify drug targets and candidate molecules. The lead clinical candidate, RTX-117, targets the Integrated Stress Response pathway via eIF2B activation and is in Phase 1 trials for Charcot-Marie-Tooth disease and Vanishing White Matter disease.

Product overview

ReviR Therapeutics is a single-platform AI-enabled biotechnology company whose offering centers on the proprietary VoyageR Discovery Engine — an RNA-targeting drug discovery platform that combines a proprietary RNA-targeting compound library, transcriptome-wide compound screening, and VoyageR AI for drug and target discovery. This platform underpins ReviR's pipeline of small-molecule RNA modulators for neurogenetic and CNS diseases, including the lead clinical candidate RTX-117 (an oral small molecule targeting the Integrated Stress Response via eIF2B activation for CMT and Vanishing White Matter disease, currently in Phase 1 trials with FDA Orphan Drug Designation), an mRNA-targeting program for Huntington's disease funded by a $4.6M CIRM grant, an SBMA / Kennedy's Disease program partnered with the Kennedy's Disease Association, and an oral splice-modulator program for CMT1A developed with the CMT Research Foundation.

Differentiator

Problem solved

Functional benefit

Brands

• VoyageR: Proprietary AI-enabled drug/target discovery engine combining a proprietary RNA-targeting compound library with transcriptome-wide compound screening; used to originate lead candidates such as RTX-117.
• RTX-117

Products and services

• VoyageR Discovery Engine Proprietary AI-enabled RNA-targeting drug discovery platform combining a proprietary RNA-targeting compound library, transcriptome-wide compound screening, and VoyageR AI for drug and target discovery across CNS and inflammatory disease indications. Used internally to originate ReviR's clinical and preclinical pipeline.
• RTX-117 Lead investigational oral small-molecule therapy that targets the Integrated Stress Response (ISR) pathway via eIF2B activation for Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM); in Phase 1 clinical trials with U.S. FDA Orphan Drug Designation and parallel FDA and China CDE IND clearance.
• Huntington's Disease mRNA-Targeting Therapy Program Preclinical RNA-targeted small-molecule program designed to reduce toxic protein buildup by targeting the mRNA of genes involved in Huntington's disease progression; supported by a $4.6M CIRM grant with plans to advance into clinical trials.
• Spinal and Bulbar Muscular Atrophy (SBMA / Kennedy's Disease) Program Small-molecule RNA modulator program for SBMA / Kennedy's Disease, a rare neuromuscular disorder lacking effective treatments; developed in partnership with the Kennedy's Disease Association.
• CMT1A Oral Splice Modulator Program Orally administered splice-modulating small molecule designed to reduce overexpression of the PMP22 disease gene in Charcot-Marie-Tooth disease type 1A (CMT1A), co-developed with the CMT Research Foundation.
• CMT Precision Medicine Platform (isogenic iPSC + AI Drug Perturbation Modeling) First-of-its-kind precision medicine platform for Charcot-Marie-Tooth disease combining isogenic induced pluripotent stem cell (iPSC) disease models with AI-driven drug perturbation modeling across multiple genetically defined CMT subtypes; supports patient stratification, accelerates RTX-117 development, and is intended to be shared with the global CMT research community.

Quantifiable outcome

• $30M Series A closed (July 2024), bringing total raised to $54M
• +4 more outcomes

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability6 records

Feature4 records

Partnerships

Two partnerships are on record, tiered flagship.

• XtalPiflagshipStrategic or Co-development Partner · 2 March 2026VoyageR AI platform integrated with XtalPi's AI+robotics drug discovery engine; RTX-117 was discovered through this collaboration. XtalPi is also ReviR's incubator company backer and participated as a new investor in the $30M Series A.
• CMT Research Foundation (CMTRF)flagshipStrategic or Co-development Partner · 19 June 2024Strategic co-development partnership to develop small-molecule therapeutics for Charcot-Marie-Tooth disease type 1A (CMT1A). ReviR is creating an oral splice modulator that reduces overexpression of the disease gene (PMP22). Plans to progress from cell models to animal and clinical testing. CMTRF also participated as an investor in ReviR's $30M Series A.

Scale indicators6 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Remix Therapeutics: Develops small molecules that modulate RNA processing to address disease-driving transcripts; comparable to ReviR's strategy of using small molecules to rebalance disease gene expression via RNA-level intervention.
• Accent Therapeutics: Focused on small molecule inhibitors of RNA-modifying proteins (e.g., METTL3, PRMT5), with programs in cancer and rare disease. Comparable to ReviR as a small-molecule RNA-biology platform company.
• Twentyeight-Seven Therapeutics: Pioneered small molecule modulation of RNA-modulating proteins (e.g., LIN28, miR-21) for cancer and rare disease; acquired by Otsuka in 2023. Comparable to ReviR as a small-molecule RNA-targeting platform company.
• Skyhawk Therapeutics: Develops small molecules that modulate RNA splicing for neurological and oncological diseases; acquired by Biogen in 2024. Closely comparable to ReviR's CMT1A oral splice modulator program and broader RNA-modulator pipeline.
• Arrakis Therapeutics: Pioneer in RNA-targeting small molecule drug discovery with a platform focused on structured RNA elements; directly comparable to ReviR's VoyageR approach and pipeline of small molecules that bind and modulate RNA targets.
• Expansion Therapeutics: Focused on small molecules targeting repeat expansion diseases (e.g., DM1, FXN, C9orf72), directly comparable to ReviR's neurogenetic disease focus and RNA-targeting small molecule approach.

Broad incumbents

• Recursion Pharmaceuticals: AI-driven drug discovery platform using high-throughput biology and machine learning to develop small molecule therapeutics; broader scope and significantly more capital than ReviR, but directly comparable as an AI-native biotech pursuing CNS and rare disease targets.
• InSilico Medicine: End-to-end AI-driven drug discovery company with proprietary generative chemistry, target identification, and clinical-stage pipeline; broader therapeutic scope than ReviR but directly comparable as an AI-discovered small molecule platform.
• Insitro: Machine learning-driven drug discovery combining induced pluripotent stem cell biology and genomics to identify novel targets and therapeutics; comparable to ReviR's combination of AI with iPSC-derived disease modeling in CNS indications.

Others

• XtalPi: AI + robotics drug discovery company that is both ReviR's strategic co-development partner and an investor (incubator backer that participated in the $30M Series A). Comparable as an AI-driven drug discovery platform, with a direct operational linkage to ReviR through the VoyageR integration.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Subsidiaries1 record

Funding overview

Funding rounds5 records

Investors11 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 ReviR Therapeutics