What Modalis Therapeutics does

Modalis Therapeutics Corporation is a clinical-stage Japanese biotechnology company developing single-dose gene therapies for rare genetic diseases using its proprietary CRISPR-GNDM® epigenome editing platform. Founded in January 2016 in Tokyo (originally as EdiGene Corporation, renamed Modalis in August 2019), the company is publicly listed on the Tokyo Stock Exchange Growth Market under ticker 4883 since 2020, with corporate headquarters in Tokyo and R&D operations at a Greater Boston facility in Waltham, Massachusetts (relocated from Cambridge in October 2021). Modalis targets ultra-rare genetic disorders across muscular dystrophies (LAMA2-CMD, DMD, DM1, FSHD), cardiomyopathy (DCM), and central nervous system conditions (Tauopathy, Angelman Syndrome, Dravet Syndrome), with MDL-101 for LAMA2-CMD as the lead IND-enabling program.

The core technology, CRISPR-GNDM®, combines nuclease-deficient Cas9 (dCas9) with proprietary epigenome editors and guide RNA, packaged into AAV vectors for delivery. The platform modulates gene expression without altering DNA sequence or creating double-strand breaks, which Modalis positions as a safety advantage over conventional CRISPR nucleases. The company has also developed an engineered muscle-selective AAV capsid with greater than 10-fold improved tropism and a muscle-specific promoter, providing dual-layer tissue selectivity for muscle-targeted programs. Modalis licenses foundational IP from the University of Tokyo (engineered Cas9, 2017) and Editas Medicine (CRISPR/Cas9 patents, 2020), and has built a pipeline of eight named drug candidates leveraging the platform.

Modalis operates a hybrid business model: in-house pipeline assets are advanced to a defined preclinical or IND-enabling stage using internal funding, then out-licensed to pharmaceutical partners for late-stage development, manufacturing scale-up, and commercialization, generating revenue through upfront payments, development and sales milestones, and royalties. Key partnerships include two license agreements with Astellas Pharma (2019) and a collaboration with Eisai (2019). The company has FDA Rare Pediatric Disease and Orphan Drug designations for MDL-101 and conducts clinical manufacturing through CDMOs while bringing select CMC and clinical functions in-house. The acquired entity Air Flow (September 2024) extends capabilities beyond the core platform. End customers are patient populations with rare genetic conditions, served indirectly through licensed pharmaceutical partners rather than direct sales.

Name

Modalis Therapeutics

Legal name

Modalis Therapeutics Corporation

Website

https://modalistx.com

Company type

Public

Founded year

2016

Operating status

Operating

Headcount range

11–50 employees

Short description

Modalis Therapeutics is a clinical-stage Japanese biotech developing single-dose gene therapies using its proprietary CRISPR-GNDM® epigenome editing platform to treat rare genetic muscular, cardiac, and CNS disorders, licensing programs to pharmaceutical partners such as Astellas and Eisai for late-stage development and commercialization.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

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

SIC

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

akta.pro primary industry

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

akta.pro secondary industries

AAV Vector Gene Therapy Developers (HLAAACAA), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Neurology/CNS Gene Therapies (HLAAACAH), Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)

Keywords

• Epigenome editing therapeutics
• CRISPR-based gene therapy
• Rare disease treatments
• AAV gene therapy development
• Biopharmaceutical licensing

Headquarters

HQ city

Tokyo

HQ country

Japan

HQ region

Asia

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Licensing and milestone payments: Revenue from upfront payments at contract signing, development milestones as pipeline progresses, and royalties on sales after product launch. Partnering provides short-term licensing fees swapped for long-term profits from future product sales. Revenue recognized when contracts signed or when stipulated conditions are met.
2. Clinical/sales milestones: Revenue from achieving clinical trial milestones and sales milestones if specific sales targets are met under licensing agreements.
3. Royalty income: Ongoing royalties from product sales after commercialization under licensing agreements.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels6 records

Core offering

Modalis Therapeutics develops AAV-delivered gene therapy drug candidates using its proprietary CRISPR-GNDM® epigenome editing platform, which regulates gene expression without cutting DNA. The company focuses on rare genetic diseases including LAMA2-CMD, FSHD, and DM1, advancing a pipeline of drug candidates such as MDL-101, MDL-201, and MDL-202 through in-house development and partner licensing arrangements.

Product overview

Modalis Therapeutics is a biotech company developing a pipeline of gene therapy drug candidates using its proprietary CRISPR-GNDM® epigenome editing platform. The company's lead program MDL-101 targets LAMA2-CMD, with additional candidates including MDL-202 (DM1), MDL-201 (DMD), MDL-103 (FSHD), MDL-105 (DCM), MDL-104 (Tauopathy), MDL-206 (Angelman Syndrome), and MDL-207 (Dravet Syndrome). The CRISPR-GNDM® platform enables targeted gene expression modulation via AAV-delivered epigenome editors without altering DNA sequences.

Differentiator

Problem solved

Functional benefit

Products and services

• MDL-101 AAV-delivered CRISPR-GNDM® based gene therapy candidate for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), designed to upregulate compensatory LAMA1 expression to address the genetic deficiency in skeletal and cardiac muscle. Aimed at patients with this rare neuromuscular disease.
• MDL-201 AAV-delivered CRISPR-GNDM® based therapy candidate for facioscapulohumeral muscular dystrophy (FSHD), designed to suppress DUX4 expression and address the underlying pathology of this rare neuromuscular disease.
• MDL-202 AAV-delivered CRISPR-GNDM® based therapy candidate for myotonic dystrophy type 1 (DM1), designed to address the CTG repeat expansion pathology underlying this rare neuromuscular disease.
• CRISPR-GNDM® Platform Proprietary CRISPR-GNDM® epigenome editing platform technology that uses nuclease-dead Cas9 (dCas9) fused to transcriptional modulators to upregulate or downregulate target genes without cutting DNA. Available for licensing and partnership arrangements with pharmaceutical and biotechnology companies seeking differentiated gene therapy modalities.

Quantifiable outcome

• CRISPR-GNDM demonstrated 1) single-dose administration, 2) long-lasting effect, and 3) fundamental disease improvement in animal testing
• +2 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Seven partnerships are on record, tiered minor and core.

• Emily's Future is NowminorOthers · 14 May 2026Swiss non-profit patient organization dedicated to LAMA2-related congenital muscular dystrophy. Provided donation to support continued R&D of MDL-101. Organization raises awareness and funding for LAMA2-RD research, supporting patient initiatives and international collaboration.
• LAMA2 EuropeminorGTM or Marketing Partner · 20 January 2026European patient organization network (Netherlands, Spain, France) focused on accelerating LAMA2-CMD treatment development. Published article about Modalis MDL-101 research following Human Gene Therapy publication. Provides patient community engagement and advocacy support.
• Editas Medicine, Inc.coreTechnology or Integration · 1 April 2020License agreement for CRISPR/Cas9 foundational patents obtained April 2020. Non-exclusive license secures rights to foundational CRISPR IP in company's business areas, protecting business operations.
• Eisai Co., Ltd.coreStrategic or Co-development Partner · 1 November 2019Collaboration agreement established November 2019 for application of epigenome editing technology to agreed-upon targets. Joint development using CRISPR-GNDM technology.
• University of TokyocoreTechnology or Integration · 1 September 2017Exclusive license for CRISPR technology from University of Tokyo obtained September 2017. Provides access to engineered Cas9 technology for epigenome editing applications.
• Astellas Pharma Inc.coreStrategic or Co-development Partner · 1 April 2017Initial research collaboration formed April 2017; license agreement for genetic disorder established February 2019; second license agreement for additional genetic disorder established September 2019. Astellas provides funding and access to specific genetic targets for CRISPR-GNDM technology application.
• Cure CMDminorGTM or Marketing PartnerCongenital muscular dystrophy patient organization network. Collaboration for patient engagement and clinical trial readiness.

Scale indicators6 records

Recent moves19 records

Expansion highlights6 records

Emerging players

• Taysha Gene Therapies: Clinical-stage AAV gene therapy company focused on rare CNS and neuromuscular diseases. Comparable business model to Modalis as a small-cap AAV gene therapy developer with multiple pipeline programs targeting rare monogenic disorders.
• Verve Therapeutics: Clinical-stage company using in vivo base editing to treat cardiovascular diseases. Direct overlap with Modalis's MDL-105 (dilated cardiomyopathy) program in terms of indication, though using a different editing approach.
• Prime Medicine: Clinical-stage company developing prime editing technology for precise genome editing. Conceptually similar to Modalis in pursuing next-generation editing approaches, though prime editing uses a different mechanism than epigenome editing.

Broad incumbents

• Sarepta Therapeutics: Commercial-stage gene therapy leader with approved AAV-delivered therapy (Elevidys) for Duchenne muscular dystrophy. Directly competes with Modalis's MDL-201 DMD program and other muscular dystrophy programs.
• Sangamo Therapeutics: Pioneer in genome editing (zinc finger nucleases) and gene therapy with both in vivo AAV-delivered programs and ex vivo cell therapies. Competes in the same genetic disease space as Modalis but uses an older editing technology platform.
• PTC Therapeutics: Commercial-stage rare disease company with gene therapy programs for DMD and CNS disorders. Competitor in overlapping indications (DMD, Dravet Syndrome via partner programs) with broader therapeutic portfolio.

Direct peers

• Beam Therapeutics: Clinical-stage company developing base editing and prime editing therapies for genetic diseases. Closely comparable to Modalis as a next-generation gene editing platform company pursuing rare monogenic diseases with proprietary editing chemistries.
• Intellia Therapeutics: Clinical-stage CRISPR/Cas9-based gene editing company with in vivo and ex vivo programs. Both companies use CRISPR-based platforms delivered via AAV (or LNP) to address genetic diseases, making Intellia a close technological peer despite differing editing mechanisms.
• Editas Medicine: Clinical-stage CRISPR/Cas9 gene editing company developing in vivo and ex vivo gene therapies. Modalis licenses foundational IP from Editas and operates in overlapping genome editing space, though Modalis focuses specifically on epigenome editing rather than nuclease-based editing.
• CRISPR Therapeutics: Pioneer in CRISPR/Cas9 therapeutics with approved CASGEVY therapy (via Vertex partnership) for sickle cell disease and beta-thalassemia. Operates in the same gene editing category as Modalis with both in vivo and ex vivo approaches.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/11553498
• Xx.com/modalistx

Revenue estimate

Valuation estimate

Number of profiles

Subsidiaries1 record

Funding overview

Funding rounds6 records

Investors13 records

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

M&A1 record

Investments

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

Frequently asked questions about Modalis Therapeutics