What Evolyra Therapeutics does

Evolyra Therapeutics is a preclinical-stage biotechnology company developing next-generation adeno-associated virus (AAV) gene replacement therapies for limb-girdle muscular dystrophy types 2C (LGMD2C) and 2D (LGMD2D) — rare, progressive neuromuscular diseases with no approved treatments affecting approximately 20,000 people globally. Founded in August 2025 and headquartered in Richmond, Virginia, the company is built on a second-generation AAV vector platform engineered to deliver full-length functional genes to skeletal muscle tissue while eliminating the liver toxicity risks that have limited earlier gene therapy programs. Unlike Duchenne muscular dystrophy therapies that use partial gene replacement, Evolyra's approach delivers complete functional genes as a single-dose, long-term corrective infusion.

The company's core technology is a modular AAV platform explicitly designed for scalability across 20+ LGMD subtypes and beyond. Its translational infrastructure is anchored by a partnership with the GRASP-LGMD Consortium, which provides access to 500+ patients, natural history data, biomarkers, and clinical trial-ready sites, and by a clinical manufacturing agreement with Andelyn Biosciences (announced April 2026) leveraging Andelyn's AAV Curator Platform for IND-enabling studies and GMP production. The founding team comprises physician-scientists from Virginia Commonwealth University's Center for Inherited Myology Research, led by Dr. Nicholas E. Johnson (CEO), Kevin L. Passarello (COO), Dr. Melissa Hale (CTO), and Dr. Ellie Carrell (CSO).

Evolyra's revenue model is contingent on future commercialization of one-time gene therapy products for rare disease patients, consistent with premium orphan-drug pricing upon regulatory approval. The company is currently pre-revenue, having completed a $5 million seed round and actively raising a $20 million Series A to fund IND submission planned for H2 2026 and subsequent Phase I/II clinical trials. Go-to-market will occur through specialized rare disease treatment centers and hospital pharmacies, with clinical operations conducted through enterprise partnerships with clinical trial sites and patient consortiums.

Name

Evolyra Therapeutics

Legal name

Evolyra Therapeutics

Website

https://evolyratherapeutics.com

Company type

Private

Founded year

2025

Operating status

Operating

Short description

Evolyra Therapeutics is a preclinical biotechnology company developing second-generation AAV gene replacement therapies for limb-girdle muscular dystrophy types 2C and 2D, targeting a rare disease population of approximately 20,000 patients globally.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• AAV gene therapy
• Gene replacement therapy
• Neuromuscular disease therapeutics
• Limb-girdle muscular dystrophy
• Rare disease biotechnology

Headquarters

HQ city

University Of Richmond

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, Infrastructure, Supply Chain, Marketing or Sales

Revenue model

1. Gene Therapy Product Sales: As a preclinical biotech company developing gene replacement therapies, Evolyra plans to generate revenue through commercialization of gene therapy treatments for LGMD. The company is currently in preclinical stages with IND submission planned for H2 2026.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Evolyra Therapeutics is a preclinical biotechnology company developing a second-generation Adeno-Associated Virus (AAV) gene therapy platform engineered to deliver full-length functional genes directly to skeletal muscle while avoiding liver toxicity. Its lead therapeutic programs target Limb-Girdle Muscular Dystrophy types 2C (LGMD2C/LGMDR3) and 2D (LGMD2D/LGMDR5), designed as single-dose, long-term corrective treatments for these rare, currently untreatable neuromuscular diseases.

Product overview

Evolyra Therapeutics is a biotechnology company developing a platform-based portfolio of gene replacement therapies for Limb-Girdle Muscular Dystrophy (LGMD). The core offering consists of a second-generation AAV vector platform that enables safe, full-length gene delivery to muscle tissue, supporting pipeline programs targeting LGMD2C and LGMD2D subtypes. The modular platform is designed to scale across 20+ LGMD subtypes and beyond.

Differentiator

Problem solved

Functional benefit

Products and services

• Second-Generation AAV Vector Platform A next-generation Adeno-Associated Virus (AAV) gene therapy delivery platform engineered to deliver full-length functional genes directly to muscle tissue while avoiding liver toxicity, addressing safety limitations of earlier gene therapy approaches. The modular platform is designed to scale across 20+ LGMD subtypes.
• Gene Replacement Therapy for LGMD2C and LGMD2D A single-dose, long-term corrective gene therapy targeting Limb-Girdle Muscular Dystrophy types 2C and 2D (also referred to as LGMDR3 and LGMDR5), designed to deliver complete functional genes for effective treatment of these rare neuromuscular diseases affecting approximately 20,000 patients globally.

Quantifiable outcome

• Successful preclinical results demonstrating complete protein expression and muscle restoration in animal models
• +1 more outcomes

Segments1 record

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

Four partnerships are on record, tiered core and supporting.

• Andelyn BiosciencescoreTechnology or Integration · 14 April 2026Andelyn Biosciences partnered with Evolyra for clinical manufacturing of AAV gene therapies for LGMD types 2C and 2D. Andelyn will utilize its proprietary AAV Curator Platform to support IND-enabling studies and GMP manufacturing for Evolyra's programs.
• GRASP-LGMD ConsortiumcoreStrategic or Co-development PartnerGlobal network of clinicians, scientists, and patient advocates focused on accelerating LGMD research. Provides Evolyra access to clinical trial-ready infrastructure, natural history data, and patient voices that guide therapy development. Consortium includes 500+ patients providing biomarkers and disease progression tools.
• The Speak FoundationsupportingOthersNon-profit organization dedicated to improving quality of life for those living with muscular dystrophy. Founder Kathryn Bryant Knudson authored introduction to Evolyra highlighting the company's mission and approach.
• Virginia Commonwealth UniversitycoreStrategic or Co-development PartnerFounding team members (Dr. Nicholas Johnson, Dr. Melissa Hale, Dr. Ellie Carrell) are affiliated with VCU's Center for Inherited Myology Research. Kevin Passarello serves as Entrepreneur-in-Residence. Provides research infrastructure and academic expertise.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Pfizer: Pfizer commercializes Beqvez, an AAV gene therapy for hemophilia B, and has broader rare disease gene therapy capabilities. Operates as a large incumbent in the broader AAV gene therapy space and is a potential acquirer or competitor for the same patient populations and partnerships.
• Regenxbio: Regenxbio is an established AAV platform company with multiple partnered gene therapy programs across rare diseases. Comparable AAV vector technology and rare disease focus, but operates a broader portfolio rather than a single neuromuscular niche.
• Asklepios BioPharmaceutical (AskBio): AskBio is a clinical-stage AAV gene therapy platform with programs across neuromuscular and other rare diseases, owned by Bayer. Comparable AAV technology and rare disease focus; operates a broader pipeline rather than LGMD-specific focus.

Direct peers

• Solid Biosciences: Solid Biosciences develops AAV gene therapies for Duchenne and other neuromuscular diseases, with programs spanning multiple subtypes. Comparable in modality (AAV), indication overlap, and clinical-stage development profile.
• Sarepta Therapeutics: Sarepta is the leading commercial-stage gene therapy company for Duchenne muscular dystrophy (Elevidys) and other neuromuscular diseases. Direct overlap with Evolyra in muscle-targeted AAV gene therapy, patient population (muscular dystrophy), and rare disease commercialization strategy.
• Atamyo Therapeutics: Atamyo is a clinical-stage gene therapy company developing AAV-based treatments specifically for multiple LGMD subtypes (including 2C/R5 and 2D/R3). The closest direct peer in indication, modality, and target population.

Emerging players

• Lexeo Therapeutics: Lexeo is a clinical-stage gene therapy company focused on rare diseases including cardiac and neurological indications. Comparable in AAV modality and early-stage development strategy with overlapping patient advocacy-driven trial design.
• Taysha Gene Therapies: Taysha develops AAV-based gene therapies for rare CNS and neuromuscular diseases. Comparable in AAV platform approach and rare disease focus, though with different lead indications.

Market position

Strengths5 records

Weaknesses4 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds1 record

Investors

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 Evolyra Therapeutics