miRecule
miRecule is a clinical-stage biotechnology company developing RNA therapeutics for cancer and rare neuromuscular diseases, using its proprietary Antibody RNA Conjugate (ARC) technology and genomics-based DREAmiR discovery platform, with lead candidates MC-30 (head and neck cancer) and MC-DX4 (FSHD, licensed to Sanofi).
- Company typePrivate
- Founded2019
- HeadquartersGaithersburg, United States
- Headcount11–50
- GTM typeB2B
- OfferingHardware or Manufacturing
What miRecule does
miRecule is a Gaithersburg, Maryland-based clinical-stage biotechnology company developing RNA-based therapeutics for cancer and rare neuromuscular diseases. Founded in 2019 (per company history; firmographic data references 2018), the company operates with 11-50 employees and is led by founder and CEO Anthony D. Saleh, Ph.D., whose personal connection to Facioscapulohumeral Muscular Dystrophy (FSHD) shapes the company's mission. miRecule's technology rests on two proprietary platforms: the DREAmiR™ genomics-based discovery platform, which integrates genomic and clinical outcome data from thousands of patients to identify disease-driving genetic abnormalities and design RNA therapeutics targeting them, and the NAVIgGATOR™ conjugation chemistry, which joins RNA payloads to antibodies to form Antibody RNA Conjugates (ARCs) for targeted tissue delivery. The platform was developed from more than a decade of NIH-collaborated research. miRecule's lead clinical-stage candidates are MC-30, a microRNA-30 replacement therapy for head and neck cancer, and MC-DX4, an ARC therapeutic for FSHD targeting the DUX4 gene, which has been licensed to Sanofi.
The company operates a licensing and partnership business model rather than direct commercialization. Revenue mechanics consist of upfront payments, development/regulatory/commercial milestone payments, and tiered royalties on global net sales under pharmaceutical licensing agreements, with the Sanofi FSHD collaboration (October 2022, expanded March 2026) representing the anchor deal carrying potential milestone payments approaching $400 million plus royalties. Capital sources supplement licensing income through seed-stage equity (approximately $5.7M led by Alexandria Venture Investments in May 2021, with participation from Maryland Momentum Fund and SOLVE FSHD) and a stream of non-dilutive grants from NINDS SBIR, Maryland Industrial Partnerships, Friends of FSH Research, and the FSHD Society. miRecule's intellectual property is anchored by U.S. Patent No. 12,104,157 covering engineered miR-30 oligonucleotides with expiration expected in 2041. The company has received industry recognition including the MedCity INVEST 2024 Biopharma track competition win and has presented data at scientific venues including the Oligonucleotide Therapeutics Society annual meeting.
miRecule firmographics
Firmographics- Name
- miRecule
- Legal name
- miRecule, Inc.
- Website
- https://mirecule.com
- Company type
- Private
- Founded year
- 2019
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- miRecule is a clinical-stage biotechnology company developing RNA therapeutics for cancer and rare neuromuscular diseases, using its proprietary Antibody RNA Conjugate (ARC) technology and genomics-based DREAmiR discovery platform, with lead candidates MC-30 (head and neck cancer) and MC-DX4 (FSHD, licensed to Sanofi).
- Ownership category
- akta.pro rank
miRecule industry classification
Industry- Product category
- Biopharmaceuticals
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- akta.pro primary industry
- RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG)
- akta.pro secondary industry
- RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC) (HLAAADAF)
Keywords
Where miRecule is headquartered
LocationHeadquarters
- HQ city
- Gaithersburg
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
miRecule business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations
Revenue model
- Licensing and Milestone Payments: The company generates revenue through licensing agreements with pharmaceutical partners. Under the Sanofi collaboration, miRecule received an upfront payment and is eligible for near-term milestone payments exceeding $30M combined, with additional development, regulatory, and commercial milestones potentially reaching nearly $400M total, plus tiered royalties on global net sales.
- Equity Funding: The company has raised equity capital from venture investors including Alexandria Venture Investments to fund R&D activities. Raised $5.7M seed round in 2021.
- Government Grants: The company receives grant funding from government programs including Maryland Stem Cell Research Commission awards and NINDS SBIR program funding.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels5 records
miRecule product offering
Product offeringCore offering
miRecule is a clinical-stage biotechnology company that develops first-in-class RNA therapeutics using its proprietary DREAmiR™ discovery platform and NAVIgGATOR™ conjugation chemistry. The company creates Antibody RNA Conjugate (ARC) therapies that combine antibody targeting with RNA payloads to treat cancer and neuromuscular diseases, with lead candidates MC-30 for head and neck cancer and MC-DX4 for Facioscapulohumeral Muscular Dystrophy (FSHD).
Product overview
miRecule is a biotechnology company developing RNA therapies for cancer and muscular dystrophy. The company's proprietary DREAmiR™ discovery platform utilizes genomic and outcome data from thousands of patients to identify genetic abnormalities and create targeted RNA therapeutics. These therapeutics are delivered via the company's ARC (Antibody RNA Conjugate) technology using NAVIgGator™ conjugation chemistry. The core product portfolio includes MC-30 (lead candidate for head and neck cancer) and MC-DX4 (FSHD treatment licensed to Sanofi), representing first-in-class therapies for neuromuscular diseases and oncology applications.
Differentiator
Problem solved
Functional benefit
Brands
- DREAmiR™: Discovery platform that utilizes genomic and outcome data from thousands of patients to identify underlying genetic changes and create RNA therapeutics
- NAVIgGATOR™
- ARC Therapeutics
Products and services
- MC-30 Lead RNA therapeutic candidate designed as a microRNA replacement therapy for head and neck cancer. MC-30 replaces tumor suppressor activity of microRNA-30, which is lost in approximately half of head and neck cancer patients.
- MC-DX4 RNA maintenance therapy for Facioscapulohumeral Muscular Dystrophy (FSHD) that suppresses DUX4, the genetic driver of the disease. MC-DX4 is designed to prevent muscle loss safely and sustainably, and was licensed to Sanofi for clinical development and global commercialization.
- DREAmiR Discovery Platform Proprietary genomics-based discovery platform that utilizes genomic and outcome data from thousands of patients to identify underlying genetic changes causing disease, then creates novel RNA therapeutics that directly target those genetic abnormalities. Offered as a discovery engine to pharmaceutical partners.
Quantifiable outcome
- MC-DX4 targets DUX4 gene which causes disease in 95% of FSHD patients
- +2 more outcomes
Companies that use miRecule
Customer profileSegments2 records
Ideal customer profiles2 records
miRecule technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature5 records
miRecule partnerships and signals
Strategic signalPartnerships
Three partnerships are on record, tiered core and minor.
- SanoficoreStrategic collaboration combining miRecule's DREAmiR discovery platform and NAVIgGATOR conjugation technology with Sanofi's NANOBODY technology to develop ARC therapeutics for FSHD. miRecule granted exclusive worldwide license; Sanofi responsible for downstream clinical development and global commercialization. Expanded in March 2026 to advance best-in-class ARC for suppressing DUX4.
- University of Maryland, Baltimore (UMB)coreJoint Maryland Industrial Partnerships (MIPS) grant collaboration with Dr. Robert Bloch to develop translational biomarkers for FSHD and test miRecule's MC-DX4 therapy in Dr. Bloch's animal model of the disease.
- FSHD SocietyminorPatient advocacy organization providing funding and support for FSHD research. miRecule works with FSHD Society to bring treatment to patients.
Scale indicators5 records
Recent moves6 records
Expansion highlights6 records
miRecule competitors and assessment
Company assessmentDirect peers
- Avidity Biosciences: Avidity develops antibody-oligonucleotide conjugates (AOCs) that pair monoclonal antibodies with siRNA payloads for targeted RNA therapeutics, the same core Antibody-RNA Conjugate architecture that miRecule's NAVIgGATOR platform enables. Both companies pursue tissue-targeted RNA delivery in rare disease.
- Dyne Therapeutics: Dyne develops oligonucleotide therapeutics for serious muscle diseases including FSHD and Duchenne muscular dystrophy, overlapping directly with miRecule's MC-DX4 program in FSHD. Both apply antibody-based targeting to deliver RNA payloads to muscle tissue.
- Wave Life Sciences: Wave develops stereopure oligonucleotide therapeutics (PRISM platform) with a pipeline focused on neurological and neuromuscular diseases including Huntington's disease and Duchenne muscular dystrophy. Comparable to miRecule as an oligonucleotide therapeutic developer working on rare genetic disease.
- Stoke Therapeutics: Stoke develops antisense oligonucleotides that increase protein expression by modulating RNA splicing (TANGO platform), with lead programs in autosomal dominant optic atrophy and Dravet syndrome. Comparable to miRecule as a rare-disease-focused RNA therapeutics company.
Broad incumbents
- Alnylam Pharmaceuticals: Alnylam is the leading commercial-stage RNAi therapeutics company (Onpattro, Givlaari, Oxlumo, Amvuttra) and operates RNA interference-based drug discovery and GalNAc-conjugate delivery technology. It represents the established incumbent in the RNA therapeutics space in which miRecule competes.
- Ionis Pharmaceuticals: Ionis is the pioneer in antisense oligonucleotide (ASO) therapeutics with multiple approved products (Spinraza, Tegsedi, Waylivra) and a deep pipeline in rare neurological and neuromuscular diseases. It is a broad incumbent in oligonucleotide-based drug development adjacent to miRecule's ARC approach.
- Arrowhead Pharmaceuticals: Arrowhead develops RNA interference (RNAi) therapeutics using proprietary TRiM™ conjugation and delivery platforms to enable targeted siRNA delivery. It shares miRecule's focus on targeted RNA therapeutics but operates at a broader and more advanced commercial scale.
- Sarepta Therapeutics: Sarepta is a commercial-stage genetic medicines company with approved exon-skipping and gene therapies for Duchenne muscular dystrophy. It is a broad incumbent in RNA/DNA-based neuromuscular therapeutics relevant to miRecule's FSHD muscle-targeted program.
Emerging players
- PepGen: PepGen develops conjugated antisense oligonucleotides for neuromuscular diseases using its EDO peptide delivery platform. Comparable to miRecule in its combination of conjugation technology with oligonucleotide payloads and its neuromuscular disease focus.
- Entrada Therapeutics: Entrada develops endosomal escape vehicle (EEV)-enabled oligonucleotide and protein therapeutics for neuromuscular diseases including Duchenne muscular dystrophy. Comparable to miRecule in its focus on intracellular delivery of RNA-based payloads to muscle tissue.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
miRecule social profiles
Digital presencemiRecule financial estimates
Financial estimateRevenue estimate
Valuation estimate
miRecule leadership team
Management profileNumber of profiles
Profiles7 records
miRecule funding detail
Funding detailFunding overview
Funding rounds3 records
Investors7 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
miRecule M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about miRecule
What does miRecule do?
miRecule is a clinical-stage biotechnology company that develops first-in-class RNA therapeutics using its proprietary DREAmiR™ discovery platform and NAVIgGATOR™ conjugation chemistry. The company creates Antibody RNA Conjugate (ARC) therapies that combine antibody targeting with RNA payloads to treat cancer and neuromuscular diseases, with lead candidates MC-30 for head and neck cancer and MC-DX4 for Facioscapulohumeral Muscular Dystrophy (FSHD).
Is miRecule a public or private company?
miRecule is a private company. It is classified as venture growth investor backed and is currently operating.
When was miRecule founded?
miRecule was founded in 2019. It employs 11 to 50 people.
Where is miRecule based?
miRecule is headquartered in Gaithersburg, United States, in the North America region.
How does miRecule make money?
Three revenue lines are on record. Licensing and Milestone Payments are the primary driver. The others are equity Funding and government Grants.
Who are miRecule's main competitors?
Direct peers on record are Avidity Biosciences, Dyne Therapeutics, Wave Life Sciences and Stoke Therapeutics. Broad incumbents are Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Arrowhead Pharmaceuticals and Sarepta Therapeutics. Emerging players are PepGen and Entrada Therapeutics.
Does miRecule have an API?
No public API is recorded for miRecule.
What industry is miRecule in?
miRecule's product category is Biopharmaceuticals. Its primary akta.pro industry code is HLAAADAG, RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics), with a secondary code of HLAAADAF, RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC). Its NAICS code is 541714.