SpliceBio Secures $135M Series B Funding for Innovative Gene Therapy Approach
Spanish biotechnology company SpliceBio has successfully raised $135 million in a Series B funding round, positioning itself at the forefront of next-generation gene therapies. The funding, co-led by EQT Life Sciences and Sanofi Ventures, will primarily support the advancement of SpliceBio's lead program for Stargardt disease, a genetic eye condition causing progressive vision loss.
Overcoming Gene Therapy Limitations with Dual AAV Approach
SpliceBio's technology aims to address a critical limitation in current gene therapy approaches. Traditional adeno-associated virus (AAV) vectors used for gene delivery are constrained by their small size, unable to carry larger corrective genes. SpliceBio's innovative solution employs two separate AAVs to deliver pieces of a large gene into cells, where specially engineered splicing molecules reassemble the cargo into full-length proteins.
This approach is particularly relevant for treating Stargardt disease, which is caused by mutations in the ABCA4 gene – too large to fit within a single AAV vector. SpliceBio's lead program, SB-007, currently in a Phase 1/2 trial, aims to produce a functional version of the ABCA4 protein using this dual AAV method.
Strategic Backing from Pharmaceutical Giants
The substantial Series B funding round demonstrates strong industry confidence in SpliceBio's platform. Notable participants include the venture arms of major pharmaceutical companies:
- Sanofi Ventures (co-lead investor)
- Roche Venture Fund
- Novartis Venture Fund
Additional investors comprise New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, and Asabys Partners. This diverse group of backers underscores the potential of SpliceBio's Protein Splicing platform to unlock gene therapies for previously untreatable diseases.
Expanding Beyond Ophthalmology
While SpliceBio's initial focus is on ophthalmology with its Stargardt disease program, the company has broader ambitions. The Series B proceeds will also support the acceleration of AAV gene therapy programs in neurology and other undisclosed therapeutic areas. This expansion aligns with SpliceBio's vision to lead the next wave of genetic medicines across multiple disease domains.
SpliceBio CEO Miquel Vila-Perelló emphasized the significance of the funding, stating, "The support from such high-quality investors underscores the strength of our programs and our unique Protein Splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today."
References
- SpliceBio lands $135M for a new kind of eye gene therapy
The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to help overcome the packaging constraints of current genetic medicines.
- Sanofi-backed SpliceBio secures $135M series B for eye disease gene therapy trials
Spain’s SpliceBio will use $135 million from a series B round backed by Sanofi and Roche to advance its lead gene editing therapy for Stargardt disease through clinical studies.
Explore Further
What are the basic details of SpliceBio's executive team, pipeline overview, and funding history?
What are the highlights and potential advantages of SpliceBio's innovative dual AAV approach in gene therapy?
What are the clinical data and progress details for SpliceBio's SB-007 program targeting Stargardt disease?
What is the estimated target market size for SpliceBio's core pipeline, particularly its gene therapy for Stargardt disease?
Who are the major competitors of SpliceBio in the gene therapy space addressing the limitations of AAV vectors?