Spark Therapeutics, Inc.

Stargardt disease is a genetic retinal condition, resulting in progressive loss of vision and blindness. Credit: Hryshchyshen Serhii/Shutterstock. SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007, its gene therapy candidate for Stargardt disease. Jointly led by Sanofi Ventures and EQT Life Sciences, the funding round also saw participation from Roche Venture Fund and current investors including Novartis Venture Fund, UCB Ventures, New Enterprise Associates and Ysios Capital. The Series B financing will support the ongoing interventional Phase I/II ASTRA trial and the observational POLARIS study of SB-007. The funds will expedite the development of SpliceBio’s AAV gene therapy programme pipeline across fields including neurology, ophthalmology and other undisclosed indications, leveraging the company’s protein splicing platform. Stargardt disease is a genetic retinal condition that occurs due to mutations in the ATP-binding cassette subfamily A member 4 (ABCA4) gene, resulting in progressive loss of vision and eventual blindness. SB-007 has received clearance from the Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) to enter clinical development. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence It aims to deliver a functional copy of the full-length ABCA4 protein, potentially treating individuals with Stargardt disease, irrespective of their specific ABCA4 mutation. Three new members will join the company’s board of directors:  EQT Life Sciences managing director Daniela Begolo, Sanofi Ventures partner Laia Crespo and Roche Venture Fund head Carole Nuechterlein. SpliceBio co-founder and CEO Miquel Vila-Perelló stated: “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.” In 2023, SpliceBio signed a licensing agreement with Spark Therapeutics, allowing the latter to use its protein splicing platform and develop a gene therapy for an undisclosed inherited renal disease. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic You have a right to withdraw your consent at any time, by clicking here . We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy . Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now

SV-007 is designed to produce a functional copy of the full-length ABCA4 protein.\n 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.Stargardt disease causes vision loss in children and adults, and SpliceBio’s adeno-associated viral (AAV) gene therapy SB-007 is already undergoing a phase 1/2 trial for the indication. The series B round was co-led by new investors EQT Life Sciences and Sanofi Ventures with support from Roche Venture Fund, New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund and Asabys Partners.As well as fund the phase 1/2 study of SV-007, which is designed to produce a functional copy of the full-length ABCA4 protein, the biotech said it would also use the series B proceeds to “accelerate” its pipeline of AAV gene therapy programs in ophthalmology, neurology and “other undisclosed indications.”“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,” SpliceBio CEO Miquel Vila-Perelló, Ph.D., said in a June 11 release.“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,” Vila-Perelló added. “We are building a company positioned to lead the next wave of genetic medicines.”Roche’s Spark Therapeutics unit previously penned a $126 million alliance with SpliceBio back in 2023 to use the Barcelona-based biotech’s platform, which aims to sidestep a common challenge in gene therapy where the necessary gene is too large to fit within traditional AAV vectors.In this morning’s release, Carole Nuechterlein, head of Roche Venture Fund, said the fund had been “impressed by the … momentum behind SB-007 in Stargardt disease, and the platform’s potential to unlock a new class of genetic medicines.”Splice emerged in 2022 with 50 million euros ($57 million) to tackle delivery barriers in gene therapy, backed by UCB Ventures and the Novartis Venture Fund. The biotech’s work is based on technology developed in the Muir Lab at Princeton University.

SpliceBio pulled in another $135 million in funding as it continues to usher its lead gene therapy candidate, SB-007 for Stargardt disease, through Phase I/II development and expand its wider pipeline. The series B financing, announced Wednesday, was co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund, as well as all existing backers, including UCB Ventures and Novartis Venture Fund.SB-007 is designed to address the underlying genetic cause of Stargardt disease by producing a functional copy of the full-length ABCA4 protein in the retina, with the potential to treat all patients, regardless of their specific mutation. The protein splicing dual AAV gene therapy is currently being investigated in the Phase I/II ASTRA study, which dosed its first participant in March, as well as the POLARIS natural history trial. The new funds will also be used to accelerate SpliceBio's pipeline of AAV gene therapy programmes in ophthalmology, namely SB008 and SB009, and SB011 in neurology, as well as other undisclosed indications that utilise the company's protein splicing platform."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," remarked co-founder and CEO Miquel Vila-Perelló.SpliceBio's platform is designed to overcome the size limitations of AAV vectors, which are unable to deliver genes that are bigger than 4.7 kilobases. The firm's protein splicing technology enables the splitting of the gene into two or more transgenes that are then delivered using dual AAV vectors. Once inside the cell, the DNA of each transgene is transcribed into mRNA and translated into protein, with SpliceBio's engineered inteins then assembling the full-length protein that is needed to treat the disease.SpliceBio emerged in 2022 with €50 million ($57 million) in series A financing having been seeded in 2020 by Ysios Capital and Asabys Partners. At the tailend of 2023, the biotech entered a licensing agreement with Roche's Spark Therapeutics unit to utilise the former's protein splicing platform to develop a gene therapy for an undisclosed inherited retinal disease.