Axovia Therapeutics
Axovia Therapeutics is a UK-based, clinical-stage biotech developing AXV-101, a first-in-human gene therapy for BBS1-related retinitis pigmentosa causing childhood blindness, with a first-in-human trial planned for early 2026.
- Company typePrivate
- Founded2024
- HeadquartersLondon, United Kingdom
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
What Axovia Therapeutics does
Axovia Therapeutics is a clinical-stage biotechnology company headquartered in London, United Kingdom, focused on developing gene therapies for rare genetic disorders. Its sole and lead asset, AXV-101, is a gene therapy candidate targeting childhood blindness caused by Bardet-Biedl Syndrome 1 (BBS1)-related retinitis pigmentosa and retinal degeneration. The company received Clinical Trial Application (CTA) clearance in Q3 2025 and is positioned to begin a first-in-human open-label dose-escalation study in early 2026, conducted through UK clinical trial sites. BBS1 and BBS10 genes together account for over 20% of all BBS cases, representing a sizeable underserved population in which IMCIVREE remains the only FDA-approved therapy (since 2022). The company frames its mission around the broader ciliopathy disease space, though its current pipeline is a single gene therapy program.
The business is pre-revenue with no approved products, no disclosed pricing, and no commercial customers. Axovia has been funded exclusively through grant awards from a single nonprofit, A Race Against Blindness, with cumulative commitments of $5.1 million across three tranches from October 2024 through January 2026. The 11–50 employee organization is led by co-founder and CEO Philip Beales and co-founder and CSO Victor Hernandez. Current go-to-market activity is limited to scientific publications, nonprofit partnerships, and regulatory/PR communications rather than commercial channels. Upon any future regulatory approval, distribution would likely follow a specialty rare disease model via specialty pharmacies, hospital-based dispensing, and rare disease treatment centers. The company's near-term value driver is clinical trial execution and proof-of-concept data for AXV-101, not commercial revenue.
Axovia Therapeutics firmographics
Firmographics- Name
- Axovia Therapeutics
- Legal name
- Axovia Therapeutics
- Website
- https://axoviatherapeutics.com
- Company type
- Private
- Founded year
- 2024
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Axovia Therapeutics is a UK-based, clinical-stage biotech developing AXV-101, a first-in-human gene therapy for BBS1-related retinitis pigmentosa causing childhood blindness, with a first-in-human trial planned for early 2026.
- Ownership category
- akta.pro rank
Axovia Therapeutics industry classification
Industry- Product category
- Ophthalmology Gene Therapy
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), 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
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), AAV Vector Gene Therapy Developers (HLAAACAA)
Keywords
Where Axovia Therapeutics is headquartered
LocationHeadquarters
- HQ city
- London
- HQ country
- United Kingdom
- HQ region
- Europe
Markets served
Axovia Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales, Supply Chain
Distribution channels1 record
Marketing channels3 records
Axovia Therapeutics product offering
Product offeringCore offering
Axovia Therapeutics is developing AXV-101, a single gene therapy candidate targeting Bardet-Biedl Syndrome 1 (BBS1)-related retinitis pigmentosa and retinal degeneration. The product is administered as a first-in-human open-label dose-escalation study aimed at halting or slowing vision loss in patients with BBS1 genetic mutation. The company is clinical-stage with no approved products and no additional pipeline candidates beyond AXV-101.
Product overview
Axovia Therapeutics is developing AXV-101, a single gene therapy product targeting Bardet-Biedl Syndrome 1 (BBS1)-related vision loss and retinal degeneration. The company is a clinical-stage biotech with no platform architecture or multiple products—their pipeline consists of this one lead therapy candidate advancing through early-phase human trials.
Differentiator
Problem solved
Functional benefit
Products and services
- AXV-101 A gene therapy candidate designed to treat Bardet-Biedl Syndrome 1 (BBS1)-related retinitis pigmentosa by addressing the underlying genetic cause of retinal degeneration. It is intended for patients with BBS1 genetic mutation causing progressive childhood blindness, delivered as a first-in-human open-label dose-escalation clinical trial.
Quantifiable outcome
- Clinical trial for AXV-101 targeting retinal degeneration in BBS1 patients expected to initiate in early 2026
Companies that use Axovia Therapeutics
Customer profileSegments1 record
Ideal customer profiles2 records
Axovia Therapeutics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature1 record
Axovia Therapeutics partnerships and signals
Strategic signalScale indicators4 records
Recent moves5 records
Expansion highlights5 records
Axovia Therapeutics competitors and assessment
Company assessmentDirect peers
- MeiraGTx: Clinical-stage gene therapy company developing treatments for inherited retinal diseases, including AAV-based candidates targeting forms of retinitis pigmentosa. Directly comparable to Axovia's AXV-101 in modality, indication class, and clinical stage.
- REGENXBIO: AAV gene therapy developer with a pipeline including ocular programs (e.g., RGX-314 for wet AMD in partnership with AbbVie). Comparable as an AAV platform developer with ophthalmology gene therapy assets advancing through clinical trials.
- Applied Genetic Technologies Corp (AGTC): Clinical-stage gene therapy company historically focused on inherited retinal diseases using AAV vectors. Comparable in modality (AAV gene therapy) and target indication (inherited retinal dystrophies), though AGTC has undergone restructuring.
- 4D Molecular Therapeutics: Clinical-stage AAV gene therapy developer with ophthalmology pipeline including 4D-150 for retinal indications. Comparable as a next-generation AAV platform company advancing ocular gene therapy candidates through clinical trials.
- ProQR Therapeutics: RNA therapeutics company developing treatments for inherited retinal diseases such as Usher syndrome and retinitis pigmentosa. Comparable in rare inherited retinal disease focus and clinical-stage development, though using a different modality (RNA editing vs AAV gene therapy).
- Editas Medicine: Clinical-stage genome editing company with an ophthalmology program (EDIT-101 for CEP290-related retinal degeneration, though discontinued). Comparable in targeting inherited retinal disease with a novel therapeutic modality.
- Beacon Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments for inherited retinal diseases including X-linked retinitis pigmentosa. Comparable in modality (AAV gene therapy), indication (inherited retinal dystrophy), and clinical-stage development profile.
Broad incumbents
- Spark Therapeutics (Roche): Subsidiary of Roche that developed and commercialized Luxturna, the first FDA-approved AAV gene therapy for an inherited retinal disease (RPE65-related). Sets the benchmark for regulatory pathway, pricing, and commercial model in ocular gene therapy that Axovia's AXV-101 would follow.
Emerging players
- Rhythm Pharmaceuticals: Commercial-stage rare disease company with IMCIVREE (setmelanotide), the only FDA-approved therapy for BBS since 2022. The most direct competitive reference point for Axovia in the BBS treatment landscape, though IMCIVREE targets metabolic/systemic rather than retinal manifestations.
- Generation Bio: Clinical-stage genetic medicines company developing non-viral gene therapy delivery with an early ophthalmology focus. Comparable as an early-stage rare disease gene therapy developer exploring ocular indications, though using a different delivery platform than AAV.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat3 records
Key risks6 records
Key highlights6 records
Customer concentration
Axovia Therapeutics social profiles
Digital presenceAxovia Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Axovia Therapeutics leadership team
Management profileNumber of profiles
Profiles2 records
Axovia Therapeutics funding detail
Funding detailFunding overview
Funding rounds3 records
Investors1 record
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Axovia Therapeutics 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 Axovia Therapeutics
What does Axovia Therapeutics do?
Axovia Therapeutics is developing AXV-101, a single gene therapy candidate targeting Bardet-Biedl Syndrome 1 (BBS1)-related retinitis pigmentosa and retinal degeneration. The product is administered as a first-in-human open-label dose-escalation study aimed at halting or slowing vision loss in patients with BBS1 genetic mutation. The company is clinical-stage with no approved products and no additional pipeline candidates beyond AXV-101.
Is Axovia Therapeutics a public or private company?
Axovia Therapeutics is a private company. It is classified as founder individual operated bootstrapped and is currently operating.
When was Axovia Therapeutics founded?
Axovia Therapeutics was founded in 2024. It employs 1 to 10 people.
Where is Axovia Therapeutics based?
Axovia Therapeutics is headquartered in London, United Kingdom, in the Europe region.
Who are Axovia Therapeutics's main competitors?
Direct peers on record are MeiraGTx, REGENXBIO, Applied Genetic Technologies Corp (AGTC), 4D Molecular Therapeutics, ProQR Therapeutics, Editas Medicine and Beacon Therapeutics. Spark Therapeutics (Roche) is listed as a broad incumbent. Emerging players are Rhythm Pharmaceuticals and Generation Bio.
Does Axovia Therapeutics have an API?
No public API is recorded for Axovia Therapeutics.
What industry is Axovia Therapeutics in?
Axovia Therapeutics's product category is Ophthalmology Gene Therapy. Its primary akta.pro industry code is HLAAACAG, Ophthalmology Gene Therapies, with a secondary code of HLAAACAE, Gene Therapy for Rare/Monogenic Diseases. Its NAICS code is 541714 and its SIC code is 8731.