In an era where advancements in biotechnology have the potential to alter treatment paradigms, SpliceBio, a promising Spanish startup, has taken a significant leap forward by securing $135 million in its Series B funding round, announced on June 11,
2025.
This influx of capital is earmarked for the company’s innovative pursuit of gene therapy targeting Stargardt disease, a progressive retinal disorder caused by mutations in the large ABCA4 gene.
This article delves into SpliceBio’s unique dual AAV approach to gene therapy, its funding journey, and how it aligns with broader trends in the biotechnology sector.
Key Takeaways
- SpliceBio has raised $135 million to enhance gene therapy targeting Stargardt disease using an innovative dual AAV approach.
- Their technique involves using separate AAVs to deliver gene segments, enabling the reassembly of the full-length protein necessary for treatment.
- The company’s lead therapy, SB-007, is currently in clinical trials and is supported by prominent investors in the biotech industry.
Innovative Dual AAV Approach to Gene Therapy
In a groundbreaking development within the realm of gene therapy, SpliceBio, a Spanish biotechnology startup, has secured $135 million in a Series B funding round, disclosed on June 11,
2025.
This significant infusion of capital highlights investor confidence in SpliceBio’s innovative approach to treating Stargardt disease, a hereditary condition that can lead to severe vision impairment due to mutations within the large ABCA4 gene.
Conventional gene therapies frequently rely on adeno-associated viruses (AAVs) to deliver the necessary genetic information; however, these AAVs have a limitation in their capacity to accommodate larger genes, such as ABCA4.
SpliceBio’s novel strategy involves the use of two separate AAVs designed to transport distinct segments of the large gene.
Once inside the target cells, engineered splicing molecules facilitate the reassembly of these segments, allowing for the successful production of the full-length protein essential for effective disease treatment.
The lead candidate, SB-007, is currently undergoing Phase 1/2 clinical trials, which could pave the way for further advancements in gene therapy applications.
The financial backing from notable investors such as Sanofi Ventures, Roche Venture Fund, and Novartis Venture Fund not only strengthens SpliceBio’s position in the competitive biotech landscape but also emphasizes the industry’s recognition of the potential in innovative gene therapy methods.
Additionally, the company builds upon rigorous research originating at Princeton University, providing a robust scientific foundation for its pioneering technology.
As SpliceBio progresses towards clinical testing, its reimagined approach to gene delivery may unlock new therapeutic possibilities for currently untreatable conditions, ultimately positioning it as a key player in the evolving narrative of gene therapy.
Funding Milestones and Future Directions for SpliceBio
The recent funding milestone achieved by SpliceBio is pivotal not just for the company, but also for the broader gene therapy landscape.
With a focus on the complex challenges associated with delivering large genes, SpliceBio’s innovative use of dual AAVs coupled with engineered splicing molecules represents a shift towards more flexible gene therapy solutions.
By navigating around the size limitations typically posed by AAVs, SpliceBio is not only attempting to treat Stargardt disease but also laying the groundwork for potential applications in a variety of other genetic disorders linked to large genes.
The engagement from high-profile investors reinforces the market’s increasing interest in overcoming existing barriers in gene delivery mechanisms.
This investment trend may signal a growing recognition that addressing the unmet needs of patients with rare genetic disorders is not only a humanitarian endeavor but also a substantial business opportunity.
Additionally, as SpliceBio continues to refine its trials for SB-007, updates on safety and efficacy will be critical for maintaining investor confidence and fostering partnerships within the biotech ecosystem.
By anchoring its technology in robust academic research, SpliceBio exemplifies how collaborations between academia and industry can catalyze innovations that stand to benefit both patients and investors alike.
