Odylia
Odylia Therapeutics is a nonprofit 501(c)(3) biotech developing AAV gene therapies for ultra-rare inherited retinal diseases using the proprietary Anc80 vector platform. It serves patient advocacy groups through a three-program pipeline and a Brydge Solutions consulting service.
- Company typePrivate
- Founded2017
- HeadquartersAtlanta, United States
- Headcount1–10
- GTM typeB2B
- OfferingServices
What Odylia does
Odylia Therapeutics is a nonprofit 501(c)(3) biotechnology organization founded in 2017 and headquartered in Atlanta, Georgia, that develops AAV-based gene therapies for rare diseases—predominantly inherited retinal dystrophies—that lack commercial interest from traditional pharmaceutical companies. The organization holds exclusive rights to the Anc80 AAV vector platform (an ancestral adeno-associated virus discovered in 2015 at Massachusetts Eye and Ear) for rare and ultra-rare retinal diseases, and operates a three-program pipeline: OT-004 (RPGRIP1 gene therapy for LCA6, CORD13, and juvenile retinitis pigmentosa, which holds FDA Orphan Drug and Rare Pediatric Disease Designations), a USH1C gene therapy partnered with the Usher 2020 Foundation, and an NPHP1 gene therapy co-developed with the NPHP1 Family Foundation and Boston Children's Hospital. Ancillary to the pipeline, Odylia runs Brydge Solutions, a consulting and portfolio management service for patient advocacy groups, and The Odylia Collective, a marketplace platform (merged with Comend in 2025) that connects patient advocacy groups with drug development service providers such as CROs and CDMOs.
Odylia's business model is philanthropic rather than commercial: the organization is funded by competitive research grants (e.g., $1.5 million from Foundation Fighting Blindness in 2024 and $249,719 from the Critical Path Institute in 2026), individual and corporate donations, and fee-based Brydge Solutions consulting engagements offered at reduced rates to patient groups. Manufacturing partnerships with Andelyn Biosciences (Anc80-capable CDMO) and Aldevron (plasmid DNA) support pipeline progression, while a strategic collaboration with PTC Therapeutics covers retinal disease development using the Anc80 system. Co-founder Luk Vandenberghe (Anc80 discoverer) and CEO/CSO Ashley Winslow lead scientific direction, supported by a Board of Directors with deep rare-disease industry expertise (Ultragenyx founder Emil Kakkis, former Sarepta/Alexion executives).
The organization serves three primary constituents: rare disease patient advocacy groups seeking to develop therapies for conditions with no commercial sponsor, early-stage biotechs and academic researchers needing translational guidance, and pharmaceutical companies pursuing nonprofit partnership models for ultra-rare indications. Go-to-market is community-led and event-driven, leveraging industry conferences (ASGCT, World Orphan Drug Congress), educational webinars, and direct patient-group engagement. As a pre-clinical stage nonprofit, Odylia has no commercial product revenue, and its measurable outputs are grant dollars secured, partnerships established, and IND-enabling milestones achieved across the pipeline.
Odylia firmographics
Firmographics- Name
- Odylia
- Legal name
- Odylia Therapeutics
- Website
- https://odylia.org
- Company type
- Private
- Founded year
- 2017
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Odylia Therapeutics is a nonprofit 501(c)(3) biotech developing AAV gene therapies for ultra-rare inherited retinal diseases using the proprietary Anc80 vector platform. It serves patient advocacy groups through a three-program pipeline and a Brydge Solutions consulting service.
- Ownership category
- akta.pro rank
Odylia industry classification
Industry- Product category
- Nonprofit Rare Disease Gene Therapy Development
- NAICS
- Scientific Research and Development Services (5417)
- SIC
- Services-Commercial Physical & Biological Research (8731)
- akta.pro primary industry
- Ophthalmology Gene Therapies (HLAAACAG)
- akta.pro secondary industries
- AAV Vector Gene Therapy Developers (HLAAACAA), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Rare Ophthalmology Disorder Therapies (HLAIAIAK)
Keywords
Where Odylia is headquartered
LocationHeadquarters
- HQ city
- Atlanta
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Odylia business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure
Revenue model
- Donations and Charitable Contributions: Odylia is a 501(c)(3) nonprofit organization that relies on contributions from individuals, nonprofit organizations, patient groups, and corporate donors to fund drug development activities.
- Research Grants: Odylia receives competitive research grants from foundations and organizations such as the Critical Path Institute ($249,719 grant for USH1C gene therapy) and Foundation Fighting Blindness ($1.5 million Translational Research Award for RPGRIP1).
- Brydge Solutions Services: Odylia provides consulting and program management services to patient groups, early-stage biotech companies, and academic researchers through its Brydge Solutions initiative, offering services at reduced rates to patient groups.
Go-to-market motion2 records
Distribution channels1 record
Marketing channels9 records
Odylia product offering
Product offeringCore offering
Odylia Therapeutics is a nonprofit biotech developing investigational AAV gene therapies for rare genetic diseases, with a primary focus on inherited retinal dystrophies. Its pipeline includes OT-004 (RPGRIP1), USH1C, and NPHP1 gene therapy programs built on the Anc80 AAV vector platform. The organization also operates Brydge Solutions, providing drug development consulting and portfolio management to patient advocacy groups, and runs The Odylia Collective (merged with Comend), a marketplace connecting patient groups with drug development service providers.
Product overview
Odylia Therapeutics is a nonprofit biotech that operates primarily through its gene therapy pipeline, offering three investigational AAV gene therapies (OT-004/RPGRIP1, USH1C, and NPHP1) built on exclusive Anc80 vector technology. The company complements its internal pipeline with Brydge Solutions—a consulting and portfolio management service for patient groups—and The Odylia Collective marketplace (merged with Comend) that connects patient advocacy groups with drug development service providers. Anc80 Resources provides technical information on the AAV vector platform. The Odylia Library offers educational content and landscape analyses to support the rare disease community.
Differentiator
Problem solved
Functional benefit
Brands
- Brydge Solutions: A consulting and portfolio management service for rare disease patient groups, providing scientific team support, strategic briefs, landscape analyses, and portfolio management services.
- The Odylia Collective
Products and services
- OT-004 (RPGRIP1 Gene Therapy) Investigational AAV gene therapy utilizing the Anc80 vector for efficient RPGRIP1 gene transfer to treat vision loss in patients with Leber Congenital Amaurosis 6 (LCA6), Cone Rod Dystrophy 13 (CORD13), and juvenile retinitis pigmentosa caused by RPGRIP1 mutations.
- USH1C Gene Therapy Investigational gene therapy designed to address vision loss in Usher Syndrome Type 1C by delivering a functional copy of the USH1C gene to retinal cells using an AAV delivery system. Developed in partnership with Usher 2020 Foundation.
- NPHP1 Gene Therapy AAV-based gene replacement therapy for retinal dystrophy caused by mutations in the NPHP1 (nephrocystin-1) gene, developed in co-development partnership with the NPHP1 Family Foundation and Boston Children's Hospital (Dr. Friedhelm Hildebrandt's team).
- Brydge Solutions Consulting and program management services for rare disease patient groups, providing scientific expertise, strategic briefs, landscape analyses, and portfolio management to accelerate therapeutic development. Services offered at reduced rates to patient groups and include Science Team support, Strategic Briefs, Landscape Analyses, Portfolio Management, and Strategy Evaluations.
- The Odylia Collective (merged with Comend) Centralized marketplace designed to make essential drug development resources findable and accessible for patient advocacy groups and the rare disease research community. Connects patient groups with service providers including manufacturers, clinical CROs, animal testing services, cell line producers, and biobanking services.
Quantifiable outcome
- Accelerated timelines and reduced costs compared to traditional biotech model
- +1 more outcomes
Companies that use Odylia
Customer profileNamed customers7 records
Segments3 records
Ideal customer profiles3 records
Odylia technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Odylia partnerships and signals
Strategic signalPartnerships
Twelve partnerships are on record, tiered core, major and minor.
- Usher 2020 FoundationcorePartnership to pursue treatment for Usher Syndrome Type 1C-associated vision loss. Preclinical studies supported by the foundation demonstrated successful gene delivery with expression of human USH1C transcript and protein, providing proof-of-concept.
- ComendcoreStrategic partnership to launch a patient-driven platform for rare disease drug development. The Odylia Collective concept merged with Comend's platform to help patient advocacy groups identify, evaluate, and partner with service providers like CROs, academic groups, and drug discovery organizations. The partnership was formalized in late 2024 with platform launch planned for April 2025.
- NPHP1 Family FoundationcoreInnovative co-development partnership to create an AAV-based gene replacement therapy for retinal dystrophy caused by NPHP1 gene mutations. Preclinical work scheduled throughout 2025, targeting IND submission and clinical trial in late 2027.
- Boston Children's Hospital / Dr. Friedhelm HildebrandtcoreCollaboration for NPHP1 gene therapy program. Dr. Hildebrandt's team at Boston Children's Hospital is conducting natural history, dosing, and efficacy studies on two different NPHP1 mouse models. Dr. Hildebrandt is a renowned expert on nephrocystin genes and associated ciliopathies.
- PURA Syndrome FoundationmajorPartnership to evaluate current state of research and clinical progress toward treatments for PURA Syndrome. Odylia provides scientific and strategic guidance to help the foundation seek new treatments and develop research strategies.
- Smith Kingsmore Syndrome FoundationmajorPartnership to provide scientific and strategic guidance regarding current state of research and clinical progress toward treatments for Smith-Kingsmore syndrome. Odylia's work enables the foundation to deploy resources effectively and develop short- and long-term research strategies.
- JEM TherapeuticsmajorPartnership to accelerate development of JEM's portfolio, starting with TECPR2-related disease (SPG49). Odylia develops and implements scientific strategy to accelerate development timelines and increase likelihood of success.
- Andelyn BiosciencescorePartnership with gene therapy CDMO to manufacture OT-004 (RPGRIP1 gene therapy) using the novel Anc80 AAV capsid. Andelyn has unique capabilities to manufacture this novel AAV capsid and bring the treatment to clinical trials.
- CLOVES Syndrome CommunityminorPartnership to provide scientific and strategic guidance regarding research and clinical progress for CLOVES syndrome treatments. Odylia enables the community to deploy resources effectively and develop ongoing research strategies.
- SATB2 Gene FoundationminorPartnership to provide scientific and strategic guidance regarding current state of research and understanding of SATB2-associated syndrome. Odylia enables the foundation to deploy resources appropriately to accelerate research.
- PTC TherapeuticsmajorStrategic collaboration to develop novel gene therapies for rare inherited retinal diseases utilizing the Anc80 vector system. Lead program is RP-GRIP1 (RPGRIP1) for Leber Congenital Amaurosis 6 (LCA6).
- AldevroncorePartnership with contract manufacturing organization specializing in plasmid DNA production. Aldevron is a member of Odylia and serves as the exclusive provider of plasmid DNA for Odylia-sponsored projects.
Scale indicators5 records
Recent moves7 records
Expansion highlights5 records
Odylia competitors and assessment
Company assessmentDirect peers
- Cystic Fibrosis Foundation: The pioneering nonprofit drug development organization that funded and incubated therapies now marketed by Vertex Pharmaceuticals. Operates an analogous mission-driven, patient-anchored model for ultra-rare genetic disease drug development, the exact organizational archetype Odylia aspires to replicate in retinal disease.
- MeiraGTx: Clinical-stage gene therapy company developing AAV-based treatments for inherited retinal diseases (including achromatopsia, X-linked retinitis pigmentosa) as well as CNS and salivary gland conditions. Directly competes with Odylia's RPGRIP1, USH1C, and NPHP1 programs in the inherited retinal dystrophy space.
- REGENXBIO: Clinical and commercial-stage AAV gene therapy platform company with approved therapy ABECMA-related assets and a broad pipeline including ABBV-RGX-314 for wet AMD and diabetic retinopathy. Operates the same AAV delivery platform category as Odylia's Anc80-based programs.
- 4D Molecular Therapeutics: Clinical-stage gene therapy company with a proprietary AAV discovery platform (Therapeutic Vector Evolution) developing treatments for rare diseases including choroideremia and other inherited retinal dystrophies. Competes directly with Odylia on next-generation AAV platforms for ultra-rare ophthalmic diseases.
- Nanoscope Therapeutics: Clinical-stage biotechnology company developing gene therapies for inherited retinal diseases including retinitis pigmentosa and Stargardt disease. Directly comparable target indication space (inherited retinal dystrophies) and development stage to Odylia's OT-004 program.
- Parent Project Muscular Dystrophy: Nonprofit organization that funds and accelerates Duchenne muscular dystrophy drug development, including direct investment in gene therapy programs and partnerships with Sarepta and other developers. Direct operational analog to Odylia's nonprofit biotech model applied to a different rare disease.
- PTC Therapeutics: Strategic collaboration partner with Odylia on inherited retinal disease gene therapies since 2019, with shared interest in the RPGRIP1 program. Also operates its own gene therapy pipeline and could be a future acquirer, co-development partner, or competitor depending on pipeline evolution.
Broad incumbents
- Spark Therapeutics (Roche): Acquired by Roche in 2019, Spark developed and commercializes LUXTURNA, the first FDA-approved AAV gene therapy for an inherited retinal dystrophy (RPE65-related). Sets the regulatory and commercial precedent for the exact disease category Odylia targets.
- Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech with multiple approved therapies and an active gene therapy pipeline including programs in ophthalmology. Shares board overlap with Odylia (Emil Kakkis as CEO, Khandan Baradaran as VP Regulatory CMC) and represents the scaled commercial destination of Odylia's nonprofit drug development model.
Others
- Foundation Fighting Blindness: Largest nonprofit funder of retinal degenerative disease research, and a direct financial backer of Odylia via a $1.5M Translational Research Award. Functions as an adjacent ecosystem enabler/funder rather than a competitor, but shapes the competitive landscape by funding competing academic and commercial retinal disease programs.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks6 records
Key highlights7 records
Customer concentration
Odylia social profiles
Digital presenceOdylia financial estimates
Financial estimateRevenue estimate
Valuation estimate
Odylia leadership team
Management profileNumber of profiles
Profiles10 records
Odylia funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Odylia 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 Odylia
What does Odylia do?
Odylia Therapeutics is a nonprofit biotech developing investigational AAV gene therapies for rare genetic diseases, with a primary focus on inherited retinal dystrophies. Its pipeline includes OT-004 (RPGRIP1), USH1C, and NPHP1 gene therapy programs built on the Anc80 AAV vector platform. The organization also operates Brydge Solutions, providing drug development consulting and portfolio management to patient advocacy groups, and runs The Odylia Collective (merged with Comend), a marketplace connecting patient groups with drug development service providers.
Is Odylia a public or private company?
Odylia is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was Odylia founded?
Odylia was founded in 2017. It employs 1 to 10 people.
Where is Odylia based?
Odylia is headquartered in Atlanta, United States, in the North America region.
How does Odylia make money?
Three revenue lines are on record. Donations and Charitable Contributions are the primary driver. The others are research Grants and brydge Solutions Services.
Who are Odylia's main competitors?
Direct peers on record are Cystic Fibrosis Foundation, MeiraGTx, REGENXBIO, 4D Molecular Therapeutics, Nanoscope Therapeutics, Parent Project Muscular Dystrophy and PTC Therapeutics. Broad incumbents are Spark Therapeutics (Roche) and Ultragenyx Pharmaceutical. Foundation Fighting Blindness is listed as an others.
Does Odylia have an API?
No public API is recorded for Odylia.
What industry is Odylia in?
Odylia's product category is Nonprofit Rare Disease Gene Therapy Development. Its primary akta.pro industry code is HLAAACAG, Ophthalmology Gene Therapies, with a secondary code of HLAAACAA, AAV Vector Gene Therapy Developers. Its NAICS code is 5417 and its SIC code is 8731.