#Sweden #Stockholm #gene therapy #EQT Life Sciences #SpliceBio

EQT Life Sciences Co-Leads Significant Funding for SpliceBio

In a major development for the biotech sector, EQT Life Sciences has announced a substantial investment in SpliceBio totaling USD 135 million in a Series B funding round. This investment was co-led by Sanofi Ventures and saw participation from existing investors including New Enterprise Associates and Roche Venture Fund, highlighting the growing interest in innovative approaches to genetic medicine.

The Innovative Approach of SpliceBio

SpliceBio, a biotechnology firm based in Spain, is known for its cutting-edge gene therapy solutions aimed at treating genetic disorders. The company utilizes a unique intein platform to create novel therapies, primarily focusing on conditions pertaining to ophthalmology and neurology. This platform allows for efficiently addressing large genes that would typically be too cumbersome for traditional gene delivery methods. By splitting the genes into smaller segments, they can be delivered separately before being reassembled intracellularly to produce functioning full-length proteins necessary for therapeutic action.

The funding secured will push forward the Phase 1/2 clinical development of SpliceBio's lead program, SB-007, specifically designed to combat Stargardt disease, a genetic eye disorder inflicting progressive vision loss and potential blindness. Currently, there are no approved treatment options for Stargardt disease, which affects approximately 1 in every 8,000 to 10,000 individuals, stemming from mutations of the ABCA4 gene.

Addressing a Pressing Need

Stargardt disease represents the most prevalent inherited condition responsible for macular degeneration, demanding innovative therapeutic interventions. The existing therapies have proven inadequate, and the advent of SB-007 may finally provide affected patients with hope. By leveraging pioneering Protein Splicing technology first developed at Princeton University, SpliceBio aims to deliver a functional ABCA4 protein directly to retinal cells, potentially reversing the effects of this debilitating condition.

What sets this initiative apart is its ability to cater to the entirety of the patient population suffering from Stargardt disease by overcoming the inherent challenge of delivering large genes, as this hindrance extends across numerous genetic disorders. This expansive applicability reinforces the potential impact of SpliceBio's technology well beyond the confines of Stargardt disease.

Vision for the Future

Miquel Vila-Perelló, the Chief Executive Officer and Co-Founder of SpliceBio, expressed optimism regarding this funding, stating, "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." His sentiments reflect a strong belief in the potency of their unique Protein Splicing platform and its promise to unlock gene therapies for currently untreatable diseases.

EQT Life Sciences Managing Director Daniela Begolo emphasized their commitment to progressive science that benefits patients' lives by supporting SpliceBio, underscoring the novel solutions the company provides to some of the most serious challenges in the field of genetic medicine.

With substantial funding backing their innovative efforts, SpliceBio is poised to innovate further in the field of genetics, bringing hope to individuals plagued by genetic disorders and aiming at a future where previously untreatable conditions can finally receive effective therapies. This significant investment is not just a leap for the company but a beacon of hope for patients and healthcare providers looking for solutions in genetic medicine.