Funding will support clinical development of SB-007 and expand pipeline of genetic medicines using Protein Splicing platform

SpliceBio, a clinical-stage genetic medicines company based in Barcelona, has closed a $135 million Series B financing round to progress its lead therapy for Stargardt disease and accelerate a broader pipeline of genetic medicines.

The round was co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from the Roche Venture Fund. Existing investors, including New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund and Asabys Partners, also joined the round.

Proceeds from the financing will fund the continued development of SpliceBio’s lead gene therapy candidate, SB-007, currently in a Phase 1/2 trial for Stargardt disease. SB-007 is the first dual adeno-associated viral (AAV) gene therapy cleared by the US FDA to enter clinical development for this inherited retinal disorder. The candidate has also received clinical trial clearance from the UK’s MHRA.

Tackling the challenge of large genes

Stargardt disease is caused by mutations in the ABCA4 gene and currently has no approved treatment. SB-007 is designed to deliver a functional copy of the full-length ABCA4 protein, with the aim of treating patients regardless of their specific mutation.

The therapy uses SpliceBio’s proprietary Protein Splicing platform, which addresses a key challenge in gene therapy: the inability of AAV vectors to carry large genes. AAV has a packaging limit of around 4.7 kilobases—too small for many gene targets. SpliceBio’s technology enables large genes to be split across multiple vectors and reassembled in the cell using engineered proteins called inteins, originally developed at Princeton University. Once inside the cell, the transgenes are expressed and the inteins guide the reformation of the functional full-length protein.

“This financing marks a pivotal milestone for SpliceBio,” said Miquel Vila-Perelló, CEO and Co-Founder. “It supports our efforts to bring SB-007 through the clinic and allows us to expand our pipeline across ophthalmology, neurology and other areas.”

Investor confidence in a differentiated platform

New board members joining SpliceBio in connection with the financing include Daniela Begolo (EQT Life Sciences), Laia Crespo (Sanofi Ventures), and Carole Nuechterlein (Roche Venture Fund).

“SpliceBio’s Protein Splicing platform offers a novel solution to one of the field’s most pressing challenges—delivering large genes via AAV,” said Begolo. “It exemplifies our commitment to supporting transformational science.”

Crespo added: “With compelling data for SB-007 and a highly differentiated technology, SpliceBio is well positioned to make a significant impact in gene therapy.”

“We’re impressed by the team’s execution and the momentum behind SB-007,” said Nuechterlein. “SpliceBio has the potential to unlock a new class of genetic medicines.”

The new funds will also support continued research into additional ophthalmic and neurological targets, as well as undisclosed indications, expanding the potential reach of the Protein Splicing approach.