Unravel Biosciences

What Unravel Biosciences does

Unravel Biosciences is a clinical-stage therapeutics company that operates an AI-driven drug discovery platform to identify and validate drug repurposing opportunities for rare and complex diseases. Founded in 2021 as a spinout from the Wyss Institute at Harvard University, the company uses patient-derived RNA transcriptomics combined with proprietary computational network biology models to predict therapeutic response before clinical trials. Its core platform, BioNAV, is a probabilistic network model of human health that screens 40,000+ compounds in silico against 121 million drug-gene interactions and 364 million unique network patterns and claims a 200x higher hit rate than typical phenotypic screening. The technology stack centers on Living Molecular Twins (LMTs), digital representations of real patients created by combining patient RNA data with BioNAV analysis that function as in silico personalized clinical trials, supported by SquishyWare, an in vivo screening system using Xenopus tadpole avatars, and rareSHIFT, a patient-driven drug discovery program that has collected approximately 3,500 patient and control RNA samples across 100 disorders from over 30 countries. The pipeline includes RVL-001 (vorinostat) for Rett syndrome and Pitt Hopkins syndrome, which received FDA Orphan Drug Designation in May 2024 and entered Phase I clinical trials in Colombia in January 2026, plus RVL-002 (mitochondrial metabolism) and RVL-069 (dystonias/demyelination).

Unravel operates a hybrid revenue model combining platform services, therapeutic development, and clinical co-development. The rareSHIFT service program provides BioNAV and LMT access to patient foundations, families, and biotech partners under a not-for-profit model for foundations, while Unravel retains IP and commercial rights that can transition to its for-profit biopharma arm. Therapeutic candidates are advanced through co-funding arrangements with patient foundations, academic medical centers, and biopharma partners such as Evogene (multiple sclerosis), Vanderbilt University Medical Center (Rett syndrome, backed by a $13M grant), and Jackson Laboratory (preclinical validation). The company has raised approximately $3.3M in disclosed equity funding since 2021 (BoxOne Ventures, Good Ventures, KdT Ventures, RTK Group), with additional undisclosed RTK Group investment in 2022 and a $550K MGH grant in 2023. With 11–50 employees headquartered in Medford, Massachusetts, the company is led by co-founders Richard Novak (CEO) and Frederic Vigneault (CSO), both former Wyss Institute researchers, and supported by an advisory board that includes Don Ingber (Founding Director of the Wyss Institute) and Michael Levin (Tufts University).

Name

Unravel Biosciences

Legal name

Unravel Biosciences, Inc.

Website

https://unravel.bio

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

11–50 employees

Short description

Clinical-stage biotech that operates the AI-driven BioNAV platform and patient-derived Living Molecular Twins to repurpose existing drugs for rare diseases. Serves 30+ patient foundations and biopharma partners; lead asset RVL-001 is in Phase I trials for Rett syndrome with FDA Orphan Drug Designation.

Ownership category

akta.pro rank

Product category

AI Drug Discovery

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Research and Development in the Physical, Engineering, and Life Sciences (54171), Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731), Pharmaceutical Preparations (2834), Services-Prepackaged Software (7372)

akta.pro primary industry

AI/ML Platforms for Drug Discovery & Experiment Optimization (HLAGAJAO)

akta.pro secondary industries

Bioinformatics & Multi-omics Analysis Software (HLAGAJAD), Bioinformatics, Multi-Omics Data Analysis & Interpretation Services (HLAGANAI)

Keywords

• AI drug discovery
• Rare disease therapeutics
• Computational biology platform
• RNA-based drug discovery
• Patient stratification technology

Headquarters

HQ city

Boston

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Software

Cost components

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

Revenue model

1. Platform Services (rareSHIFT): Unravel offers access to its BioNAV platform and Living Molecular Twins technology through the rareSHIFT service program. Patient advocacy groups, families, and biotech companies commission drug discovery studies. The program has a not-for-profit business model for foundations, lowering costs while Unravel retains IP and commercial possibilities. Programs can transition to Unravel's for-profit biopharma arm as they mature.
2. Therapeutic Development & Licensing: Unravel develops proprietary therapeutic candidates (RVL-001, RVL-002, RVL-069) through clinical stages. The company retains commercial rights to discovered molecules and targets, with potential for out-licensing or spinout companies for mature programs. FDA Orphan Drug Designation for RVL-001 provides regulatory exclusivity incentives.
3. Clinical Trial Co-Development: Unravel co-develops clinical trials with patient foundations and academic medical centers. Partners (e.g., Pitt Hopkins Research Foundation, Evogene) co-fund clinical development in exchange for program access, data, and co-commercialization rights. Manufacturing and development partnerships (Quality Chemical Laboratories) support clinical supply.

Go-to-market motion2 records

Distribution channels4 records

Marketing channels5 records

Core offering

Unravel Biosciences operates an AI-driven Predictable Medicine platform (BioNAV) that integrates patient-derived RNA transcriptomics with proprietary computational network biology models to discover, derisk, and clinically validate drug targets and therapeutic mechanisms. The company develops proprietary therapeutic candidates (RVL-001, RVL-002, RVL-069) and provides platform access to patient advocacy groups, biotech companies, and academic researchers through its rareSHIFT service program.

Product overview

Unravel Biosciences operates as a clinical-stage therapeutics company built on its Predictable Medicine™ innovation stack, a unified platform architecture centered on BioNAV™ (computational AI platform) and Living Molecular Twins™ (digital patient models). The platform is supported by SquishyWare™ (in vivo tadpole screening) and accessed via rareSHIFT™ service program. The company is developing a pipeline of drug candidates including RVL-001 (Phase I for Rett/Pitt Hopkins), RVL-002 (mitochondrial metabolism), and RVL-069 (dystonias/demyelination), all discovered through its AI-driven platform.

Differentiator

Problem solved

Functional benefit

Brands

• BioNAV: Proprietary probabilistic network model of human health that predicts molecules to restore disease back to health, screening 40,000+ compounds in silico.
• rareSHIFT
• Living Molecular Twins
• SquishyWare
• RVL-001

Products and services

• BioNAV Proprietary AI-powered computational platform that uses probabilistic network models of human health to predict therapeutic responses by screening 40,000+ molecules in silico against 121 million drug-gene interactions and 364 million unique network patterns. Enables target-agnostic identification of novel drug targets and disease mechanisms.
• Living Molecular Twins (LMTs) Digital representations of real patients created by combining patient RNA data with BioNAV computational analysis, functioning as in silico personalized clinical trials. Enables prediction of therapeutic response before patient exposure and represents patient heterogeneity at scale.
• SquishyWare In vivo screening platform using Xenopus tadpole avatars to validate in silico predictions in a whole organism. Allows clinically-relevant questions about efficacy and safety to be asked from the start of drug development.
• rareSHIFT Patient-driven drug discovery service program providing access to Unravel's BioNAV platform and Living Molecular Twins to patient advocacy groups, families, and biotech partners. Collects patient and matched healthy control RNA globally using self-collected nasal swab kits and has generated approximately 3,500 RNA samples across 100 disorders from over 30 countries.
• RVL-001 (Vorinostat) Lead clinical-stage drug candidate identified by BioNAV for treatment of Rett syndrome and Pitt Hopkins syndrome. Received FDA Orphan Drug Designation in May 2024. Currently in Phase I clinical trials in Colombia with first patient dosed in May 2026.
• RVL-002 First-in-class small molecule targeting mitochondrial metabolism with multiple clinical applications including neurodegenerative and metabolic disorders. Discovered through BioNAV platform.
• RVL-069 Small molecule targeting a novel mechanism for treatment of dystonias and demyelination disorders. Discovered through BioNAV platform.
• Predictable Medicine Unravel's integrated innovation stack combining patient RNA data, Living Molecular Twins, BioNAV computational platform, and clinical validation to forecast and optimize the entire therapeutic development path from preclinical models to clinical translation.

Quantifiable outcome

• 200x higher in silico hit rate compared to typical phenotypic screening
• +6 more outcomes

Named customers10 records

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability7 records

Feature5 records

Partnerships

17 partnerships are on record, tiered core and minor.

• The SCN2A FoundationcoreStrategic or Co-development Partner · 28 January 2026Research collaboration to advance preclinical research on SCN2A-related disorders caused by loss-of-function mutations. The project aims to identify strategies to increase functional SCN2A protein in the brain, potentially informing future therapeutic development.
• Evogene Ltd.coreStrategic or Co-development Partner · 7 January 2026Scientific collaboration to jointly develop a first-in-class small-molecule therapeutic capable of restoring myelin and reversing neurological damage in demyelinating diseases such as Multiple Sclerosis. Partnership combines Evogene's ChemPass AI generative molecular design platform with Unravel's BioNAV predictive biology platform and patient-derived Living Molecular Twins to identify and optimize brain-penetrant inhibitors targeting a novel demyelination mechanism. Affects approximately 3 million MS patients worldwide with no approved therapy to effectively reverse myelin loss.
• Zymo Research CorporationminorStrategic or Co-development Partner · 20 November 2025Partnership with Zymo Research and Channel Islands Marine & Wildlife Institute to identify an injectable therapy for sea lions dying of domoic acid (DA) toxicity. Zymo sequences RNA from sea lion patients; Unravel uses BioNAV to predict treatments using the RNAseq data. A therapeutic candidate was discovered in 2025 and CIMWI is ready for dosing of future stranded animals.
• Channel Islands Marine & Wildlife Institute (CIMWI)minorStrategic or Co-development Partner · 20 November 2025Partnership with Zymo Research and Unravel Biosciences to identify injectable therapy for sea lions suffering from domoic acid toxicity. CIMWI collects RNA samples from stranded sea lions and conducts critical tests of the therapeutic molecule at its facilities.
• COMBINEDBraincoreStrategic or Co-development Partner · 27 February 2025Large-scale drug discovery collaboration enabling the consortium of rare neurological disorder advocacy groups to identify drug candidates and therapeutic mechanisms through Unravel's rareSHIFT program. This is described as one of the largest biopharma-patient community partnerships of its kind, accelerating identification of potential drug candidates across multiple ultra-rare diseases and catalyzing the goal of defragmenting rare disease.
• CURE AP-4coreStrategic or Co-development Partner · 27 February 2025Pioneering AI-driven drug discovery for ultra-rare AP-4 hereditary spastic paraplegias and related disorders. Partnership generates clinical data from patients globally to accelerate identifying and developing therapeutics, expanding to other related disorders through innovative patient stratification.
• PECET (Universidad de Antioquia)coreStrategic or Co-development Partner · 24 October 2024Collaboration to conduct clinical trials of RVL-001 in Rett syndrome and Pitt Hopkins syndrome in Colombia. Clinical trials are conducted at Universidad de Antioquia's clinical trial site in Medellin, Colombia. IRB approval received and clinical trial agreement finalized as the final step toward initiation of RVL-001 proof-of-concept clinical studies.
• The Jackson Laboratory (JAX)coreTechnology or Integration · 17 September 2024Collaboration to accelerate discovery of promising drugs for diseases with high unmet need. Unravel uses BioNAV to predict potential therapies, which are validated in JAX's preclinical models in a seamless preclinical pipeline, potentially shaving years off the development process.
• CSNK2A1 FoundationcoreStrategic or Co-development Partner · 28 August 2024Expanded collaboration to identify drug candidates for Okur-Chung Neurodevelopmental Syndrome (OCNDS). New study uses Unravel's AI platform to stratify patients into subgroups based on their likelihood to respond to prioritized drugs identified in the earlier phase of research. Also advancing OCNDS and sleep disorder portfolio.
• Pitt Hopkins Research FoundationcoreStrategic or Co-development Partner · 11 June 2024Partnership to conduct clinical study of RVL-001 in Pitt Hopkins patients. The foundation funds proof-of-concept clinical trial in Colombia to assess the efficacy of vorinostat (RVL-001) in patients with Pitt Hopkins Syndrome.
• Vanderbilt University Medical CentercoreStrategic or Co-development Partner · 14 May 2024Collaboration to support upcoming multicenter U.S. clinical study in Rett syndrome that includes RVL-001. Vanderbilt's multisite trial evaluates repurposed FDA-approved drugs as treatment options for Rett syndrome patients, with Unravel providing liquid formulation of vorinostat (RVL-001) predicted by Unravel's BioNAV platform. The trial is backed by a $13M grant.
• Quality Chemical Laboratories, Inc. (QCL)minorStrategic or Co-development Partner · 19 March 2024Manufacturing and development partnership to manufacture RVL001 clinical trial material for use in upcoming Rett syndrome clinical trials in the US and Colombia. QCL developed a new manufacturing process with lower toxicity and lower manufacturing costs.
• CHOPS FoundationcoreStrategic or Co-development Partner · 5 January 2024CHOPS Foundation initiated an AI-driven therapeutics discovery program with Unravel Biosciences to tackle an ultra-rare disorder (Cornelia de Lange syndrome) by joining forces with Cornelia de Lange syndrome families and leveraging Unravel's BioNAV platform to rapidly identify therapeutic mechanisms for clinical testing.
• Vascular Perfusion SolutionsminorStrategic or Co-development Partner · 27 November 2023Partnership to modulate endothelial activation during heart preservation to improve heart transplant outcomes. Co-development of a cardiac perfusion solution aims at extending viability of hearts in transport and reducing acute organ rejection.
• MeCo DiagnosticsminorStrategic or Co-development Partner · 26 October 2023Prostate cancer biomarker and drug discovery partnership to rapidly identify and evaluate novel biomarker-targeted therapies for solid tumors, starting with prostate cancer. Unlocks synergies between MeCo's biomarkers and Unravel's drug discovery capabilities.
• TMA Precision HealthminorStrategic or Co-development Partner · 5 July 2022Partnership to accelerate clinical translation of Unravel's therapeutic for Rett syndrome into the clinic. TMA Precision Health brings global patient networks and clinical trial infrastructure to support the program.
• Cure Mito FoundationcoreStrategic or Co-development PartnerPartnership to rapidly identify the minimum number of therapeutic interventions to treat all children with Leigh Syndrome (~110 genes). Used BioNAV and rareSHIFT to identify three patient subgroups corresponding to potential drug candidates and response markers. Analyzed 22 patients and 22 controls within three months.

Scale indicators12 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Recursion Pharmaceuticals: Clinical-stage AI drug discovery platform combining high-throughput biology with proprietary ML to discover novel therapeutics. Closely comparable to Unravel in mission (AI-driven drug discovery) and target customer base (biopharma, rare disease), but with broader pipeline and significantly larger scale (publicly listed, multi-billion dollar valuation).
• Insitro: ML-driven drug discovery platform combining induced pluripotent stem cell biology with proprietary ML models to predict drug response. Comparable to Unravel's BioNAV in applying AI/ML to patient-derived biology for therapeutic discovery, with similar target customers in biopharma and rare disease.
• Exscientia: AI-driven precision medicine company using patient-derived data and ML to design novel small molecules. Comparable to Unravel in AI-first drug discovery approach and emphasis on translating from in silico to clinical assets, though focused on new chemical entity design rather than repurposing.
• BenevolentAI: AI-driven drug discovery company using knowledge graphs and ML to identify novel targets and drug candidates, with a focus on rare diseases and inflammation. Comparable to Unravel in AI target identification and clinical translation, with overlapping therapeutic area focus.

Broad incumbents

• Schrödinger: Computational drug discovery platform combining physics-based simulation with ML for molecular design. Comparable to Unravel in computational drug discovery technology, but operates as a broader incumbent serving large biopharma via software platform and internal pipeline.
• BioMarin Pharmaceutical: Established rare disease biopharmaceutical company with multiple approved therapies. Comparable to Unravel's commercial ambitions in the rare disease space and Orphan Drug Designation strategy, though operates as a much larger incumbent.
• Ultragenyx Pharmaceutical: Clinical-stage biopharmaceutical company focused on rare and ultra-rare genetic diseases. Comparable to Unravel in rare disease focus and clinical-stage development for genetic disorders, with overlapping Rett syndrome and CNS disorder therapeutic areas.

Emerging players

• Atomwise: AI-driven small molecule discovery company using structure-based deep learning. Comparable in AI-first drug discovery approach, with focus on early-stage partnerships with biopharma and academic institutions, though Unravel is differentiated by its patient RNA data focus.
• Verge Genomics: AI-driven drug discovery company focused on neurological diseases, using patient genomics data. Comparable to Unravel in AI + patient data approach and in CNS/rare disease focus, but uses genomic rather than transcriptomic data.
• Lantern Pharma: AI-driven oncology drug development company using ML to repurpose and rescue oncology drug candidates. Comparable to Unravel in AI-driven drug repurposing strategy and clinical-stage development, with overlapping focus on identifying mechanisms and patients most likely to respond.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/unravel-biosciences

Revenue estimate

Valuation estimate

Number of profiles

Profiles13 records

Funding overview

Funding rounds5 records

Investors6 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 Unravel Biosciences