Historic operation in the Spanish biotechnology ecosystem. The Catalan startup SpliceBio has closed a financing round of 135 million dollars (about 118 million euros), which represents the largest round for a company in the sector registered in Spain. The company, based in the Barcelona Science Park (PCB), will invest the funds in the development of its gene therapy to treat Stargardt disease, a hereditary retinal disorder that causes vision loss and, ultimately, blindness and for which there is still no drug on the market.
The operation has been co-led by two of the major firms in the sector, such as the Dutch EQT Life Sciences and Sanofi Ventures, the investment arm of the French pharmaceutical giant; in addition to the participation of Roche Venture Fund, the capital management firm of the Swiss pharmaceutical company. Likewise, existing investors, who already participated in the company's previous financing in 2022, when the startup raised 50 million euros, which at the time also represented a record investment, have also joined this round. Thus, in this new operation, which has more than doubled the previous one, New Enterprise Associates, UCB Ventures, the fund based in San Sebastián and Barcelona Ysios Capital, Gilde Healthcare, Novartis Venture Fund and Asabys Partners, through its second fund with Banco Sabadell, are participating again.
Founded in 2020 by researchers Silvia Frutos and Miquel Vila-Perelló, SpliceBio will allocate the funds to advance the clinical development of its gene therapy SB-007, which is based on the use of its own technology known as Protein Splicing, which allows addressing genetic mutations such as Stargardt disease. The technology, created from research at Princeton University, uses special proteins called inteins. Thus, the gene, capable of treating the mutations addressed in each case, is divided into two parts that are introduced separately into the body by vectors, and the inteins are responsible for joining these two halves to reconstruct the complete protein to treat the disease in question.
An innovative approach that allows addressing genetic diseases that require the use of large genes for their treatment and that, until now, could not be cured because the vectors responsible for introducing them into the body did not allow transporting such long genes. However, by dividing and then splicing these proteins directly in the body, still untreatable disorders, such as Stargardt disease, can be addressed. This is a rare pathology that affects the central area of the retina, the macula, and which already represents the most common cause of degeneration of this area in young people, causing progressive vision loss and, finally, leading to blindness.
"The support of such high-quality investors underscores the strength of our programs and our unique protein splicing platform, as well as its potential to develop gene therapies for diseases that remain untreatable today," highlighted SpliceBio CEO, Miquel Vila-Perelló, who described this round as a "fundamental milestone" for the biotechnology company's trajectory.
In fact, the therapy being developed by the startup is the first of its kind to have received authorization from the US Food and Drug Administration (FDA) to initiate its clinical trials, as well as from the MHRA (Medicines and Healthcare products Regulatory Agency) in the United Kingdom. After receiving authorization from the US department late last year, this March the treatment has already been administered to the first patient in the ASTRA study.
The funds will be used to advance this study, as well as the POLARIS observational clinical trial, with the aim of bringing this drug to market in the coming years, becoming the first available treatment for this pathology. In addition, the 118 million euros raised in this financing round will also allow accelerating the expansion of SpliceBio's gene therapy portfolio, as the technology they are developing to transport long genes can be applied to all types of genetic diseases beyond ophthalmology, such as neurology.
The general director of EQT Life Sciences, Daniela Begolo, has underlined that the technology developed by the biotechnological startup will offer an "innovative solution for the introduction of large genes into the human body," a challenge she considers one of the "greatest challenges in the genetic field." Laia Crespo, from Sanofi Ventures, who co-led the round, expressed herself in similar terms, assuring that the company "has the potential to transform the field of gene therapy and to offer reference therapies for patients." Through the financing round, Daniela Begolo (EQT Life Sciences), Laia Crespo (Sanofi Ventures), and Carole Nuechterlein (Roche Venture Fund) will join SpliceBio's board of directors.