In the high-stakes world of gene therapy, Barcelona-based SpliceBio has carved a unique path by focusing on delivering replacement proteins for mutated genes. This innovative approach is a refreshing departure from traditional gene splicing or editing techniques, and it’s making waves in the biotech community.
At the heart of their groundbreaking work is SB-007, a lead gene therapy designed to treat Stargardt disease, a debilitating genetic disorder that causes progressive vision loss and eventual blindness. Stargardt disease is caused by a mutation in the ABCA4 gene, which, in its full-length form, is too large to fit inside a traditional adeno-associated viral (AAV) vector.
SpliceBio deftly circumvents this obstacle by breaking the gene into two parts, each delivered by a separate AAV. Each part of the gene is then translated separately, and sequences called inteins engineered into each section splice together the full healthy protein. It’s a biotechnological jigsaw puzzle, with the end result being a functioning protein standing in for its mutated counterpart.
“We’re like master puzzle solvers,” said SpliceBio CEO Miquel Vila-Perelló. “We’re taking these large, complex genes and breaking them down into manageable pieces, then putting them back together again inside the body.”
This approach is particularly significant for the treatment of Stargardt disease, for which there are currently no approved treatments on the market. Moreover, targeting the eye, an immune-privileged area compared to other parts of the body, allows for a significantly smaller dose of AAVs, according to Vila-Perelló. This positions SpliceBio’s SB-007 as a potential game-changer in the field of gene therapy.
The promise of SpliceBio’s innovative approach has not gone unnoticed. The company recently secured a hefty $135 million Series B funding round, backed by an international syndicate of investors, including the investment arms of pharmaceutical giants Sanofi, Roche, and Novartis Venture Funds. This marks the company’s third funding round, following a seed round in 2020 and a $57 million Series A in 2022.
As the field of gene therapy continues to evolve, SpliceBio’s unique approach could set a new precedent. The ability to deliver complex genes in segments opens up an exciting realm of possibilities, potentially revolutionizing the treatment of a wide range of genetic diseases. The success of SpliceBio’s innovative protein splicing technique could have far-reaching implications, paving the way for more effective and targeted treatments, and ultimately improving outcomes for patients with genetic disorders.
The future of gene therapy is still being written, but with its novel approach and strong financial backing, SpliceBio is well-positioned to be a major player in the narrative. The biotech industry will be watching with keen interest as they bring their protein-splicing techniques to bear on the challenges of genetic diseases. The promise of a future where these diseases can be effectively treated, or even cured, is a compelling vision indeed.
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