Splice Bio
SpliceBio is a clinical-stage genetic medicines company developing gene therapies for rare genetic diseases using its proprietary Protein Splicing platform based on engineered split inteins from Princeton University. Its lead program, SB-007, is in Phase 1/2 trials for Stargardt disease.
- Company typePrivate
- Founded2014
- HeadquartersBarcelona, Spain
- Headcount11–50
- GTM typeB2B
- OfferingHardware or Manufacturing
What Splice Bio does
SpliceBio is a clinical-stage genetic medicines company headquartered at the Parc Científic de Barcelona, Spain, founded in 2014 (originally as ProteoDesign) by Miquel Vila-Perelló and Silvia Frutos. The company develops gene therapies for rare genetic diseases using its proprietary Protein Splicing platform, a technology based on engineered split inteins originating from over 20 years of research at Princeton University's Muir Lab. SpliceBio's lead clinical program, SB-007, is a dual AAV gene therapy candidate in Phase 1/2 development (ASTRA study) for Stargardt disease, with the potential to treat all patients regardless of ABCA4 mutation type. The platform addresses a fundamental limitation in gene therapy: standard AAV vectors cannot package genes exceeding 4.7 kb, and SpliceBio's split intein technology reconstitutes full-length therapeutic proteins from dual AAV vectors in vivo.
SpliceBio's technology foundation combines engineered split inteins with additional proprietary elements enabling efficient delivery of large genes via dual AAV vectors, a platform that has been validated across multiple organs beyond the retina. The pipeline targets ophthalmology and neurology indications, and SB-007 has received FDA Orphan Drug Designation, FDA Fast Track Designation, European Commission Orphan Drug Designation, FDA IND clearance (the first-ever for a Protein Splicing gene therapy), and UK MHRA clearance. The company is supported by the POLARIS natural history study to streamline clinical trial enrollment.
SpliceBio is a privately held, pre-revenue biotech with no commercialized products. Its near-term revenue model is one-time licensing — exemplified by the October 2023 exclusive worldwide collaboration and licensing agreement with Spark Therapeutics for an undisclosed inherited retinal disease, eligible for up to $216 million in upfront, opt-in, and milestone payments plus royalties. Future revenue will derive from commercialization of SB-007 and pipeline gene therapy products, with pricing expected to be consistent with high-cost one-time gene therapies (industry reference points such as CSL's $3.5M hemophilia B therapy). The company pursues clinical trial site engagement, regulatory interactions, and biopharma partnerships, and is backed by over $200 million in total funding from top-tier investors including EQT Life Sciences, Sanofi Ventures, Roche Venture Fund, NEA, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners.
Splice Bio firmographics
Firmographics- Name
- Splice Bio
- Legal name
- SPLICEBIO, S.L.
- Website
- https://splice.bio
- Company type
- Private
- Founded year
- 2014
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- SpliceBio is a clinical-stage genetic medicines company developing gene therapies for rare genetic diseases using its proprietary Protein Splicing platform based on engineered split inteins from Princeton University. Its lead program, SB-007, is in Phase 1/2 trials for Stargardt disease.
- Ownership category
- akta.pro rank
Splice Bio industry classification
Industry- Product category
- Genetic Medicines / Gene Therapy
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)
- akta.pro primary industry
- Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization) (HLAAAIAC)
- akta.pro secondary industry
- Recombinant Protein Therapeutics (enzymes, hormones, growth factors) (HLAAAAAB)
Keywords
Where Splice Bio is headquartered
LocationHeadquarters
- HQ city
- Barcelona
- HQ country
- Spain
- HQ region
- Europe
Offices2 records
Markets served
Splice Bio business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Personnel, Technology or R&D, Operations, Infrastructure, Marketing or Sales
Revenue model
- Gene Therapy Development and Commercialization: Pre-revenue clinical-stage biotech company. Revenue will be generated through commercialization of gene therapy products, primarily SB-007 for Stargardt disease and future pipeline programs in ophthalmology, neurology, and other therapeutic areas. Additional potential revenue from licensing agreements and milestone payments from partnerships.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels4 records
Splice Bio product offering
Product offeringCore offering
SpliceBio is a clinical-stage genetic medicines company developing gene therapies using its proprietary Protein Splicing platform based on engineered split inteins developed at Princeton University's Muir Lab. The platform enables delivery of large genes exceeding the 4.7 kb packaging capacity of standard AAV vectors by splitting genes across dual AAV vectors and reconstituting full-length therapeutic proteins in vivo via protein trans-splicing. The lead clinical candidate SB-007 is a dual AAV gene therapy for Stargardt disease currently in Phase 1/2 ASTRA clinical development.
Product overview
SpliceBio is a clinical-stage genetic medicines company offering a proprietary Protein Splicing platform technology and a pipeline of gene therapy programs. The core platform enables delivery of large genes using dual AAV vectors with engineered split inteins that reconstitute full-length therapeutic proteins in vivo. The lead product is SB-007, a dual AAV gene therapy for Stargardt disease currently in Phase 1/2 clinical development (ASTRA study), supported by the POLARIS natural history study. Additional pipeline programs span ophthalmology and neurology therapeutic areas.
Differentiator
Problem solved
Functional benefit
Products and services
- SB-007 Lead dual AAV gene therapy candidate for Stargardt disease that uses protein splicing technology to restore expression of full-length ABCA4 protein in retinal photoreceptor cells, with potential to treat all patients regardless of ABCA4 mutation type. Currently in Phase 1/2 ASTRA clinical trial; administered via subretinal injection.
- Protein Splicing Platform Proprietary gene therapy technology platform based on engineered split inteins that enables delivery of large genes exceeding the 4.7 kb packaging capacity of standard AAV vectors. Splits large genes into multiple transgenes delivered via dual AAV vectors, which are reconstituted into full-length therapeutic proteins through protein trans-splicing in vivo. Licensed to biopharmaceutical partners for development of gene therapies in ophthalmology, neurology, and other therapeutic areas.
Quantifiable outcome
- First patient dosed in Phase 1/2 ASTRA clinical trial in March 2025 - first dual AAV gene therapy for Stargardt disease to enter clinical development
- +2 more outcomes
Companies that use Splice Bio
Customer profileSegments2 records
Ideal customer profiles2 records
Splice Bio technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Splice Bio partnerships and signals
Strategic signalPartnerships
One partnership is on record.
- Spark TherapeuticscoreExclusive collaboration and licensing agreement to utilize SpliceBio's Protein Splicing platform to develop a gene therapy for an undisclosed inherited retinal disease. Spark has exclusive worldwide rights to develop, manufacture, and commercialize. SpliceBio eligible to receive upfront, opt-in and milestone payments up to $216 million plus royalties.
Scale indicators6 records
Recent moves6 records
Expansion highlights6 records
Splice Bio competitors and assessment
Company assessmentDirect peers
- REGENXBIO: Clinical and commercial-stage AAV gene therapy company with a broad pipeline including ophthalmology assets (e.g., RGX-314 for wet AMD/DR). Direct platform peer in AAV-based gene therapy for rare diseases including inherited retinal conditions.
- Adverum Biotechnologies: Clinical-stage AAV gene therapy company focused on ocular diseases (wet AMD, diabetic retinopathy). Directly comparable as an ophthalmology-focused AAV gene therapy peer at a similar clinical stage with intravitreal delivery approaches.
- ProQR Therapeutics: Clinical-stage RNA therapeutics company developing ultevursen (formerly QR-421a) for Stargardt disease and other ophthalmology programs. Most direct competitor for SpliceBio's SB-007 lead indication, using antisense oligonucleotides rather than AAV gene therapy.
- uniQure: Clinical and commercial-stage AAV gene therapy company (Hemgenix approved for hemophilia B). Comparable as a rare-disease AAV gene therapy platform with similar regulatory, manufacturing, and capital-intensity dynamics.
- Voyager Therapeutics: Clinical-stage AAV gene therapy company focused on CNS and rare neurological diseases with proprietary capsid engineering. Similar stage and business model to SpliceBio, using engineered AAV vectors for hard-to-treat monogenic indications.
- Passage Bio: Clinical-stage AAV gene therapy company developing treatments for rare CNS disorders, partnered with Penn's Gene Therapy Program. Comparable clinical-stage AAV peer in monogenic rare diseases.
- Taysha Gene Therapies: Clinical-stage gene therapy company with a broad AAV-based pipeline across CNS and rare diseases. Comparable clinical-stage AAV gene therapy peer with similar platform strategy and indication breadth.
Emerging players
- Nanoscope Therapeutics: Clinical-stage gene therapy company developing optogenetic therapies for retinal diseases including Stargardt disease (MCO-010). Directly competing in the same lead indication with an alternative gene-therapy-based mechanism.
- Generation Bio: Clinical-stage non-viral gene therapy company using cell-targeted lipid nanoparticles (ctLNP) for large-gene delivery, including in ophthalmology and hemophilia. Emerging competitor pursuing the same large-gene delivery opportunity as SpliceBio but via a non-AAV modality.
Broad incumbents
- BioMarin Pharmaceutical: Large rare-disease-focused biopharmaceutical company with a commercial gene therapy product (Roctavian for hemophilia A) and pipeline spanning ophthalmology, CNS, and metabolic rare diseases. A broader incumbent in the rare-disease gene therapy commercial landscape that SpliceBio's lead program could eventually compete in.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks1 record
Key highlights7 records
Customer concentration
Splice Bio social profiles
Digital presenceSplice Bio compliance and trust
Trust signalCompliance4 records
Splice Bio financial estimates
Financial estimateRevenue estimate
Valuation estimate
Splice Bio leadership team
Management profileNumber of profiles
Profiles6 records
Splice Bio funding detail
Funding detailFunding overview
Funding rounds4 records
Investors10 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Splice Bio 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 Splice Bio
What does Splice Bio do?
SpliceBio is a clinical-stage genetic medicines company developing gene therapies using its proprietary Protein Splicing platform based on engineered split inteins developed at Princeton University's Muir Lab. The platform enables delivery of large genes exceeding the 4.7 kb packaging capacity of standard AAV vectors by splitting genes across dual AAV vectors and reconstituting full-length therapeutic proteins in vivo via protein trans-splicing. The lead clinical candidate SB-007 is a dual AAV gene therapy for Stargardt disease currently in Phase 1/2 ASTRA clinical development.
Is Splice Bio a public or private company?
Splice Bio is a private company. It is classified as venture growth investor backed and is currently operating.
When was Splice Bio founded?
Splice Bio was founded in 2014. It employs 11 to 50 people.
Where is Splice Bio based?
Splice Bio is headquartered in Barcelona, Spain, in the Europe region.
How does Splice Bio make money?
One revenue line is on record: gene Therapy Development and Commercialization.
Who are Splice Bio's main competitors?
Direct peers on record are REGENXBIO, Adverum Biotechnologies, ProQR Therapeutics, uniQure, Voyager Therapeutics, Passage Bio and Taysha Gene Therapies. Emerging players are Nanoscope Therapeutics and Generation Bio. BioMarin Pharmaceutical is listed as a broad incumbent.
Does Splice Bio have an API?
No public API is recorded for Splice Bio.
What industry is Splice Bio in?
Splice Bio's product category is Genetic Medicines / Gene Therapy. Its primary akta.pro industry code is HLAAAIAC, Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization), with a secondary code of HLAAAAAB, Recombinant Protein Therapeutics (enzymes, hormones, growth factors). Its NAICS code is 541714 and its SIC code is 2836.