SpliceBio raises $135m for gene therapy development

SpliceBio has successfully secured $135 million through a Series B financing round primarily led by Sanofi Ventures and EQT Life Sciences. This funding is designated for the progression of SB-007, a gene therapy candidate intended for treating Stargardt disease, a genetic retinal disorder that can lead to vision impairment and eventually blindness due to mutations in the ABCA4 gene. The financing round also saw participation from the Roche Venture Fund and existing investors, including Novartis Venture Fund, UCB Ventures, New Enterprise Associates, and Ysios Capital.

The newly acquired funds are allocated to support the ongoing clinical trials, specifically the Phase I/II ASTRA trial and the observational POLARIS study, both evaluating the efficacy of SB-007. SpliceBio aims to advance its AAV gene therapy pipeline across various fields such as neurology and ophthalmology, utilizing its innovative protein splicing technology. This platform is crucial for developing gene therapies that could potentially treat diseases currently lacking effective treatments.

The candidate, SB-007, has received approval from both the U.S. Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) to proceed with clinical development stages. SB-007’s main objective is to deliver a functional version of the full-length ABCA4 protein, potentially offering treatment solutions to a broader range of patients with Stargardt disease, regardless of the specific mutation present.

In line with the growth and progression, SpliceBio is also expanding its board of directors with three new members joining: Daniela Begolo from EQT Life Sciences, Laia Crespo from Sanofi Ventures, and Carole Nuechterlein from Roche Venture Fund. These additions bring a wealth of expertise and strategic insights to the company as it navigates the complexities of developing advanced gene therapies.

Miquel Vila-Perelló, co-founder and CEO of SpliceBio, expressed optimism about the future, stating, “This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our pipeline across ophthalmology, neurology, and beyond. The support from such high-quality investors underscores the strength of our programmes and our unique protein splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today.”

Apart from the developments in Stargardt disease treatment, SpliceBio has entered into a licensing agreement with Spark Therapeutics. This collaboration allows Spark Therapeutics to utilize SpliceBio’s protein splicing platform to develop a gene therapy for an undisclosed inherited renal disease, marking another significant step in expanding the applications of its technology.

The progress of SpliceBio reflects a growing momentum in the field of gene therapy, as industries and research entities invest in innovative solutions to transform treatment paradigms and address unmet medical needs. The backing from prominent investors not only represents a vote of confidence in SpliceBio’s capabilities but also highlights the potential impact of its gene therapy platform in revolutionizing the way genetic disorders are treated. As SpliceBio continues to push forward with its clinical trials and partnership strategies, it stands at the forefront of next-generation therapeutic developments, offering hope for patients with conditions once deemed untreatable.