Myrtelle

What Myrtelle does

Myrtelle, Inc. is a clinical-stage gene therapy company developing first-in-class treatments for rare monogenetic disorders of the central nervous system, with a focus on myelin-based diseases. The company's proprietary rAAV-Olig001 vector platform selectively targets oligodendrocytes (myelin-producing cells), enabling precise delivery of therapeutic genes or gene-silencing miRNAs to cells affected in leukodystrophies and other white matter disorders. Its lead asset, MYR-101 (rAAV-Olig001-ASPA), is an investigational gene therapy for Canavan disease currently in Phase 1/2 clinical trials, with a Biologics License Application (BLA) submission planned and potential FDA approval targeted for 2027.

Myrtelle's broader pipeline addresses Pelizaeus-Merzbacher Disease (PMD), H-ABC, Multiple System Atrophy (MSA), and DFNB8 genetic hearing loss (MYR-201, via a licensed TMPRSS3 gene therapy). The company operates with 11-50 employees, is incorporated in Delaware with operational headquarters in New York, and has secured extensive regulatory designations including FDA RMAT, Fast Track, Rare Pediatric Disease, and Orphan Drug status, as well as EMA Orphan Drug and ATMP classifications and UK MHRA ILAP designation. Strategic IP licensing agreements with Pfizer (Canavan disease) and Rescue Hearing (DFNB8) anchor the pipeline.

The business model is pre-revenue and clinical-stage. Future revenue is expected to be generated through one-time gene therapy product sales at premium per-patient pricing typical of ultra-rare disease treatments, supplemented by potential licensing and milestone payments from existing partnerships. Commercial-stage GMP manufacturing was launched in July 2025 through partnerships with Charles River (UK) and Viralgen Vector Core/AskBio (Spain). Distribution is structured for hospital-based intracerebroventricular administration at specialized treatment centers. Myrtelle has raised over $57 million across multiple SEC-registered securities offerings between 2021 and 2025, with the most recent activity including a Series B-2 Preferred Stock convertible note exchange in December 2025.

Name

Myrtelle

Legal name

Myrtelle, Inc.

Website

https://myrtellegtx.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

11–50 employees

Short description

Myrtelle is a clinical-stage gene therapy company developing its proprietary rAAV-Olig001 oligodendrocyte-targeting vector platform to treat rare monogenetic CNS disorders, with lead program MYR-101 for Canavan disease in Phase 1/2 trials and a pipeline spanning PMD, H-ABC, MSA, and DFNB8 hearing loss.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

Scientific Research and Development Services (5417)

akta.pro primary industry

Neurology/CNS Gene Therapies (HLAAACAH)

akta.pro secondary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• Gene therapy
• Rare disease therapeutics
• Oligodendrocyte targeting
• Viral vector platform
• Leukodystrophy treatments

Headquarters

HQ city

Wakefield

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, Supply Chain, Marketing or Sales

Revenue model

1. Gene therapy product sales: Once approved, the company plans to commercialize MYR-101 as a treatment for Canavan disease, potentially generating revenue through product sales. As a ultra-rare disease treatment, pricing may be substantial per-patient.
2. Licensing and milestone payments: The company has entered into exclusive worldwide licensing agreements including with Pfizer for Canavan disease and with Rescue Hearing for DFNB8 hearing loss, generating potential milestone and royalty payments.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels6 records

Core offering

Myrtelle is a clinical-stage gene therapy company developing first-in-class recombinant adeno-associated virus (rAAV) gene therapies targeting oligodendrocytes for the treatment of rare monogenetic neurological disorders, with lead candidate MYR-101 for Canavan disease and additional pipeline programs for Pelizaeus-Merzbacher Disease, H-ABC, Multiple System Atrophy, and DFNB8 hearing loss.

Product overview

Myrtelle is a clinical-stage gene therapy company with a proprietary oligodendrocyte-targeting rAAV-Olig001 vector platform. The company's lead product is MYR-101 (rAAV-Olig001-ASPA), an investigational gene therapy for Canavan disease currently in Phase 1/2 clinical trials. The platform enables targeted delivery of therapeutic genes to oligodendrocytes, the myelin-producing cells affected in various leukodystrophies. Myrtelle's pipeline includes programs for Canavan disease (MYR-101), Pelizaeus-Merzbacher Disease (PMD), Multiple System Atrophy (MSA), H-ABC, and DFNB-8 hearing loss (MYR-201), with the company having launched commercial-stage manufacturing partnerships in 2025.

Differentiator

Problem solved

Functional benefit

Products and services

• MYR-101 (rAAV-Olig001-ASPA) A first-in-class oligotrophic recombinant adeno-associated virus (rAAV) gene therapy that delivers a functional ASPA gene directly to oligodendrocytes for the treatment of Canavan disease, restoring ASPA enzyme function to reduce toxic NAA accumulation and support myelin production.
• MYR-201 A novel gene therapy for monogenic hearing loss targeting DFNB8 genetic deafness, utilizing low-dose rAAV delivery of a therapeutic TMPRSS3 gene by local administration to the inner ear.
• PMD Gene Therapy Program A gene therapy program for Pelizaeus-Merzbacher Disease (PMD) using a nonpathogenic rAAV vector to deliver a gene silencing microRNA (miRNA) to suppress toxic Plp1 accumulation in oligodendrocytes, addressing an X-linked recessive brain disorder affecting approximately 3,500 patients in the US.
• H-ABC Program Gene therapy program for H-ABC (hypomorphic ASPA mutation causing a milder form of Canavan disease), leveraging the same oligodendrocyte-targeting rAAV approach to treat this white matter disorder.
• MSA Program Gene therapy program targeting Multiple System Atrophy (MSA), a rare neurodegenerative disorder affecting myelin-producing cells, using the proprietary oligodendrocyte-targeting rAAV-Olig001 vector platform.

Quantifiable outcome

• Signals of therapeutic benefit and meaningful clinical improvement in children treated to date
• +4 more outcomes

Named customers2 records

Segments3 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

Six partnerships are on record, tiered core and major.

• Charles RivercoreTechnology or Integration · 8 July 2025Global leader in gene therapy infrastructure providing GMP plasmid manufacturing and analytical excellence at their CDMO center of excellence in Keele, UK. Part of commercial-stage manufacturing partnership for MYR-101.
• Viralgen Vector Core (AskBio Inc.)coreTechnology or Integration · 8 July 2025Cutting-edge GMP vector production at their world-class facility in San Sebastián, Spain. Commercial manufacturing partner for MYR-101 gene therapy. Hosted symposium at ASGCT 2026 featuring Myrtelle CEO.
• rAAVen TherapeuticsmajorStrategic or Co-development Partner · 11 January 2023Partnership to develop novel recombinant AAV vectors for gene therapy treatments targeting diseases of the CNS where myelin is affected. rAAVen provides AAV engineering expertise; Myrtelle tests vectors and owns vector compositions with exclusive worldwide commercialization rights. rAAVen receives milestone and sales-based royalty payments.
• Forge BiologicsmajorTechnology or Integration · 3 October 2022Manufacturing partnership to advance Myrtelle's novel gene therapy for monogenic hearing loss (DFNB8) into clinical trials. Forge provides research-grade and GMP-Pathway plasmid manufacturing and cGMP AAV process development and scale-up manufacturing at their 200,000 sq ft Hearth facility in Columbus, Ohio.
• Rescue Hearing Inc. (RHI)coreOEM/ Whitelabel/ Licensing Partner · 19 May 2022Worldwide exclusive licensing agreement for novel gene therapy for DFNB8 genetic hearing loss using TMPRSS3 gene. Myrtelle to develop and commercialize the therapy leveraging its gene therapy capabilities.
• Pfizer Inc.coreOEM/ Whitelabel/ Licensing Partner · 14 December 2021Exclusive worldwide licensing agreement for investigational rAAV gene therapy to bring potential new medicine to patients with Canavan disease. Following the licensing agreement, Myrtelle is conducting Phase I/II clinical study under open U.S. IND.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Taysha Gene Therapies: Clinical-stage AAV gene therapy company developing treatments for rare CNS/monogenic neurological diseases using intrathecal delivery. Directly comparable in modality (AAV), target organ (CNS), and ultra-rare pediatric indications.
• Passage Bio: Clinical-stage AAV gene therapy company focused on rare CNS disorders of the central nervous system with an intrathecal AAV delivery approach. Highly comparable in modality, delivery route, and patient population (rare monogenic pediatric CNS diseases).
• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based therapeutics for severe CNS diseases including rare neurological indications. Comparable in modality (AAV), platform approach, and CNS focus.
• uniQure: Clinical/commercial-stage gene therapy company with AAV platform targeting rare diseases including CNS indications (e.g., Huntington's program). Comparable as an AAV gene therapy developer with rare CNS focus, more advanced commercially.

Broad incumbents

• REGENXBIO: Established AAV gene therapy platform company with both internal pipeline and licensed NAV technology partners across multiple rare disease indications. Broader and more commercially advanced than Myrtelle but directly comparable as an AAV gene therapy developer.
• Pfizer Inc. Global biopharmaceutical company that licensed the MYR-101 Canavan program to Myrtelle and has its own gene therapy franchise. Relevant as both a strategic partner and a benchmark for large-pharma AAV gene therapy commercialization.
• Novartis (AveXis): Owner of Zolgensma, the landmark AAV9 gene therapy for SMA, via its AveXis acquisition. Multiple Myrtelle executives came from AveXis. Highly relevant commercial benchmark for pediatric CNS AAV gene therapy pricing, delivery, and launch dynamics.
• Sarepta Therapeutics: Commercial-stage gene therapy company with multiple approved rare neuromuscular disease products (Elevidys, etc.). Comparable as a rare disease gene therapy commercializer though focused on muscular dystrophies rather than leukodystrophies.
• Asklepios BioPharmaceutical (AskBio): AAV gene therapy platform company (now part of Bayer) operating Viralgen Vector Core, Myrtelle's commercial-stage manufacturing partner. Relevant as an AAV manufacturing and platform peer with comparable production technology.

Emerging players

• AVROBIO: Clinical-stage gene therapy company developing lentiviral-based therapies for rare diseases including lysosomal storage disorders. Comparable as a small rare-disease gene therapy developer; previous employer of Myrtelle's CMO.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/myrtelleinc
• Facebookfacebook.com/myrtelleinc
• LinkedInlinkedin.com/company/myrtelle-inc
• Instagraminstagram.com/myrtelleinc

Compliance8 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds4 records

Investors1 record

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

M&A

Investments

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

Frequently asked questions about Myrtelle