SpliceBio secures US$135M to advance gene therapy for Stargardt disease

SPAIN—SpliceBio, a clinical-stage genetic medicines company based in Spain, has successfully completed a Series B financing round, raising US$135 million to advance its lead gene therapy program, SB-007, for Stargardt disease.

The round was co-led by EQT Life Sciences and Sanofi Ventures, with additional participation from Roche Venture Fund and existing investors such as Novartis Venture Fund, UCB Ventures, New Enterprise Associates, Ysios Capital, Gilde Healthcare, and Asabys Partners.

The newly secured funds will primarily support the clinical development of SB-007, including the ongoing Phase I/II ASTRA interventional trial and the POLARIS observational study.

SB-007 is notable as the first dual adeno-associated viral (AAV) gene therapy to receive clearance from both the US Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) for clinical trials targeting Stargardt disease.

Stargardt disease is a genetic retinal disorder caused by mutations in the ABCA4 gene, leading to progressive vision loss and, ultimately, blindness.

Currently, there are no approved treatments for this condition.

SB-007 aims to address the underlying cause by delivering a functional copy of the full-length ABCA4 protein, potentially offering a therapeutic solution for all patients with Stargardt disease, regardless of their specific ABCA4 mutation.

Beyond the immediate focus on Stargardt disease, the Series B financing will also accelerate SpliceBio’s broader pipeline of AAV gene therapies.

The company’s proprietary protein splicing platform enables the development of treatments for diseases caused by mutations in large genes, which have traditionally been challenging to address with standard gene therapy approaches.

This platform is being leveraged to expand SpliceBio’s research and development efforts in fields such as ophthalmology, neurology, and other undisclosed therapeutic areas.

As part of this new phase of growth, three new members will join SpliceBio’s board of directors: Daniela Begolo, Managing Director at EQT Life Sciences; Laia Crespo, Partner at Sanofi Ventures; and Carole Nuechterlein, Head of Roche Venture Fund.

Their expertise is expected to further strengthen the company’s strategic direction.

Commenting on the financing milestone, SpliceBio’s co-founder and CEO, Miquel Vila-Perelló, emphasized the significance of the investment, stating that it marks a pivotal moment for the company as it advances SB-007 in clinical development and continues to expand its pipeline across multiple therapeutic areas.

He highlighted that the support from leading investors underscores the strength of SpliceBio’s programs and the potential of its unique protein splicing technology to unlock gene therapies for diseases that currently lack effective treatments.

In addition to its internal pipeline, SpliceBio entered into a licensing agreement in 2023 with Spark Therapeutics, granting Spark the rights to use SpliceBio’s protein splicing platform to develop a gene therapy for an undisclosed inherited renal disease.

With this substantial financial backing, SpliceBio is well-positioned to accelerate the development and potential commercialization of innovative genetic medicines, offering new hope to patients affected by Stargardt disease and other severe genetic conditions.

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