#Spain #Barcelona #SpliceBio #SB-007 #Stargardt Disease

SpliceBio Achieves Major Funding Milestone

SpliceBio, a pioneer in the field of genetic medicines, has recently announced a substantial financing round, raising $135 million in Series B funding. This funding is co-led by notable investors including EQT Life Sciences and Sanofi Ventures, alongside participation from existing stakeholders such as the Roche Venture Fund. This pivotal investment aims to support the advancement of SB-007, SpliceBio's innovative gene therapy program targeting Stargardt disease, while also expanding its pipeline of potential genetic therapies in various therapeutic areas.

Background on SpliceBio

Founded with a mission to combat diseases linked to large gene mutations, SpliceBio specializes in a groundbreaking technique called Protein Splicing. This method allows for the treatment of genetic disorders that have long been considered untreatable due to the limitations of conventional gene therapies, particularly with adeno-associated viral vectors (AAV) that struggle to accommodate large genes.

SB-007 is positioned to be a game-changer in treating Stargardt disease—a hereditary retinal disorder that results in progressive vision loss due to mutations in the ABCA4 gene. This gene therapy is tailored to generate a functional version of the ABCA4 protein, potentially addressing the root cause of the disease and offering hope to patients regardless of their specific genetic mutation.

Clinical Development and Regulatory Approvals

The funding will primarily facilitate the ongoing clinical studies for SB-007, including the Phase 1/2 ASTRA study and the observational POLARIS study. Notably, SB-007 has been recognized by the FDA as the first dual AAV gene therapy for clinical development in Stargardt disease, and it has also received clearance from the UK Medicines and Healthcare products Regulatory Agency (MHRA). With no existing approved treatments available, the progression of SB-007 through clinical trials heralds a significant advance for those affected by this condition.

Miquel Vila-Perelló, CEO and Co-Founder of SpliceBio, expressed optimism about this funding round, describing it as a crucial milestone for advancing clinical development and expanding their therapeutic offerings. "This financing will enable us to enhance our pipeline and explore additional treatment avenues in ophthalmology, neurology, and other yet undisclosed therapeutic areas," he stated.

Unique Protein Splicing Technology

What sets SpliceBio apart is its proprietary Protein Splicing platform, which ingeniously divides the gene into smaller segments that can fit within AAV vectors. This method paves the way for delivering larger genes, thus tackling a significant challenge in genetic medicine. The technology relies on engineered proteins known as inteins, developed through extensive research at Princeton University. This allows the therapeutic genes to be delivered separately and then combined within cells to form the complete, functional protein necessary for disease treatment.

Investor Confidence and Future Prospects

The backing from major investment firms underscores the confidence in SpliceBio’s innovative approach. Daniela Begolo from EQT Life Sciences remarked on SpliceBio's promise, emphasizing the company's potential to redefine how genetic diseases are addressed. Similarly, Laia Crespo from Sanofi Ventures highlighted the compelling data supporting SB-007 and the transformative impact it could have on the field of gene therapy.

In light of this funding success, SpliceBio aims to not only bring SB-007 to market but also to pioneer new gene therapies that can significantly improve patients' lives facing inherited genetic disorders. This endeavor aligns with the broader goal of developing treatments that effectively overcome the obstacles posed by larger gene mutations and their associated therapies.

As SpliceBio gears up for the next stages of clinical development, the biotech community and patients alike await further advancements that could revolutionize the landscape of genetic medicine and treatment for Stargardt disease and beyond.