SpliceBio | AI Transformation Index
SpliceBio is among the most AI-durable assets in a healthcare portfolio: its value creation is locked inside a proprietary biological platform, a regulated...
Overview
SpliceBio is a clinical-stage gene therapy company that discovers and develops genetic medicines using its proprietary Protein Splicing platform, generating value through the de-risking and advancement of therapeutic pipeline assets toward regulatory approval and eventual commercialization or partnership/acquisition.
Executive Summary
SpliceBio is among the most AI-durable assets in a healthcare portfolio: its value creation is locked inside a proprietary biological platform, a regulated clinical development pathway, and orphan IP that no AI-native entrant can replicate or bypass, making it structurally resilient to LLM-era disruption. The AI opportunity is genuine but modest in PE-transformation terms — AI can accelerate the scientific design and regulatory preparation functions, but the company's critical path is dominated by physical clinical execution and manufacturing that AI cannot compress. For EQT, the investment thesis here is squarely on scientific platform value and clinical de-risking, not AI transformation — AI is a helpful tailwind for discovery productivity rather than a step-change lever on the business model.
Durability
How resilient is this business against AI disruption?
Market Substitutability
Profit Pool Migration
Barrier to Entry
Disintermediation Exposure
Switching Costs
Opportunity
How big a deal is AI for this business?
AI Applicability
Improvement Magnitude
Transformation Upside
Domain Intelligence Amplification
69.5
Ciridae AI Score
Durability Rationale
SpliceBio's durability is anchored in three interlocking structural facts: the paid outcome is a physical biologic requiring years of regulated clinical development that AI cannot shortcut; the company sits at the origin of its own proprietary IP rather than in any intermediary or coordination layer; and the structural barriers — FDA/EMA approval pathways, GMP manufacturing, orphan drug designations, and a patent-protected platform — are among the highest in any industry. The primary durability risk is not AI disruption but rather pipeline failure, competitive modalities (RNA editing, small molecules), or a future where AI-accelerated drug discovery at large pharma narrows the platform differentiation premium — none of which are LLM-driven threats in the 2025-2026 timeframe.
Opportunity Rationale
The AI opportunity for SpliceBio is real but structurally bounded: the scientific discovery, protein engineering design, and regulatory documentation workflows are genuinely AI-applicable and would benefit from step-change improvements in speed and coverage, and the company's 20+ years of proprietary protein splicing knowledge represents domain intelligence that AI compute could amplify in ways a general-purpose model cannot. However, because the rate-limiting constraints on SpliceBio's value creation are clinical trial timelines, biological manufacturing, and regulatory review — none of which bend to LLM capabilities — the overall transformation potential is meaningful but not order-of-magnitude; AI accelerates the scientific funnel feeding a physically constrained pipeline, rather than transforming the whole production function.
Key Risks
The durability assessment could be wrong if competing therapeutic modalities (RNA editing, small molecule approaches) advance faster than expected and erode the clinical lead SpliceBio currently holds in Stargardt disease — this is a scientific competition risk, not an AI disruption risk, but it could compress the value of the protein splicing platform.
The opportunity assessment could be wrong in the optimistic direction if AI-driven protein design tools (e.g., AlphaFold successors, generative protein models) dramatically accelerate intein optimization and large-gene construct engineering — potentially compressing years of wet-lab iteration into months and making AI more transformative to the core R&D production function than the current LLM/agent framing suggests.
Company Overview
| Headquarters | Parc Científic de Barcelona, Baldiri Reixac, 10, 08028 Barcelona, Spain |
| Products / Services | SpliceBio focuses on developing gene therapies using its proprietary Protein Splicing platform. Their lead program is SB-007, a gene therapy for Stargardt disease. The company is engaged in multiple gene therapy programs aimed at treating genetic diseases caused by mutations in large genes. |
| Deal Partners |
PE / Ownership History
| Event | Details |
|---|---|
Series B Round June 11, 2025 | The $135 million Series B financing supports the clinical development of SpliceBio's lead gene therapy candidate SB-007 and pipeline expansion. Daniela Begolo, Laia Crespo, and Carole Nuechterlein joined SpliceBio's Board of Directors following the financing. |
Series A Round February 2022 | The Series A financing was oversubscribed and aimed to advance SpliceBio's protein splicing platform and gene therapy pipeline. The board included representatives from these firms. |
Seed Round September 2020 | Seed round led by Ysios Capital and joined by Asabys Partners. Joël Jean-Mairet expressed excitement about the investment and the potential of SpliceBio's platform. Sylvain Sachot highlighted the quality of the initial data and the promise of the technology. |
Executives
| Name | Background |
|---|---|
Miquel Vila-Perelló, Ph.D Co-Founder & CEO | Co-founder and CEO of SpliceBio since its inception in 2014 (formerly ProteoDesign). Led SpliceBio from an early-stage concept to a global clinical-stage biotechnology company. Advanced multiple programs into development, including the lead program for Stargardt disease. Secured over $200 million in funding from top-tier investors. Scientific foundation rooted in his pioneering research on protein splicing and engineering at Princeton University. Former Research Scholar and Protein Center manager in the Department of Chemistry at Princeton University. Postdoctoral researcher at The Rockefeller University for six years, contributing to innovative protein ligation technologies. Over 20 years of experience in protein engineering for therapeutic development. Author of 23+ peer-reviewed publications and inventor of multiple patents. Holds a PhD in Chemistry from the University of Barcelona. |
Silvia Frutos, Ph.D Co-Founder & CTO | Co-founder and Chief Technology Officer of SpliceBio. Leads development of the company’s core technology platform and scientific strategy. Over two decades of experience in chemical biology, protein chemistry, and peptide synthesis. Key contributor to SpliceBio’s Protein Splicing platform for gene therapy. Former CIBER researcher at the University of Barcelona. Postdoctoral training at The Rockefeller University, contributing to intein-mediated protein splicing mechanisms. Holds a PhD in Chemistry from the University of Barcelona. |
Gerard Caelles Chief Business Officer | Joined SpliceBio in 2016. Played a pivotal role in company growth and financing rounds including seed, $57M Series A, and $135M Series B. Forged strategic partnerships in the biopharmaceutical industry. Influenced R&D strategy and pipeline definition. Previously led business development at Bionure, contributing to drug development for Acute Optic Neuritis. Holds a degree in Biotechnology from Universitat Autònoma de Barcelona. |
Aniz Girach, MD Chief Medical Officer | Joined SpliceBio in 2024. Over 25 years of experience in medical retina and gene therapy for inherited retinal diseases. Involved in development and approval of four ophthalmology drugs. Former Global Head of Ophthalmology at Merck & Co, VP Clinical Development at Alcon, CMO at Oxurion NV, Nightstar Therapeutics, and ProQR Therapeutics. Led gene therapy programs culminating in Nightstar’s acquisition by Biogen for $800 million. 11 years in clinical practice and academia before pharmaceutical industry roles. Visiting and Honorary Professor at Wills Eye Hospital, Philadelphia. Author of four textbooks and 90+ peer-reviewed publications. Holds independent board directorships in two biotech companies. |
Donald Munoz Chief Financial Officer | CFO of SpliceBio (detailed background not fully available from current sources). |

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