SpliceBio, a genetic medicines company centered on Protein Splicing to combat diseases stemming from mutations in large genes, has successfully closed a $135 million Series B financing round. The funding, co-led by EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund and existing investors, aims to drive the clinical development of SB-007, SpliceBio’s lead gene therapy candidate for Stargardt disease. This funding milestone comes as a significant boost to advancing SB-007 through ongoing clinical trials and expanding SpliceBio’s pipeline to encompass various genetic disorders.
SB-007, the first dual adeno-associated viral (AAV) gene therapy endorsed by the FDA for clinical trials in Stargardt disease, has also received regulatory clearance from the UK’s MHRA. Stargardt disease, an inherited retinal disorder resulting from mutations in the ABCA4 gene, causes progressive vision loss and blindness without any approved treatments currently available. SB-007 is engineered to counter the genetic root of the disease by generating a functional copy of the complete ABCA4 protein, offering potential treatment to all patients regardless of their specific ABCA4 mutation. The financing will not only support the advancement of SB-007 but also expedite the development of SpliceBio’s pipeline across ophthalmology, neurology, and undisclosed indications utilizing the Protein Splicing platform.
SpliceBio’s innovative approach aims to address a critical limitation of AAV vectors, which struggle to deliver genes exceeding their packaging capacity. By leveraging their Protein Splicing platform, which employs engineered inteins to split genes into transgenes for delivery via dual AAV vectors, SpliceBio can overcome this barrier. Once inside the cell, the transgenes are transcribed into messenger RNA and translated into proteins, with the engineered inteins facilitating the assembly of the full-length protein necessary for treating the disease. This technology opens new possibilities for treating genetic disorders previously deemed untreatable due to gene size constraints.
The involvement of prominent investors like EQT Life Sciences, Sanofi Ventures, and Roche Venture Fund underscores the potential of SpliceBio’s unique platform to revolutionize genetic medicines. The addition of these investors to the SpliceBio Board of Directors further solidifies the company’s strategic growth and development plans. With the support of these investors and the promising data from SB-007, SpliceBio is well-positioned to lead the next wave of genetic therapies, addressing unmet medical needs across various disease areas.
SpliceBio’s commitment to advancing genetic medicines is exemplified through SB-007’s progress in Stargardt disease. With Orphan Drug Designation from both the FDA and European Commission, SB-007’s journey from IND clearance to clinical trials marks a significant milestone in the field of gene therapy. The initiation of Phase 1/2 ASTRA study and the ongoing POLARIS study highlight SpliceBio’s dedication to bringing novel therapies to patients in need. Through their continuous recruitment efforts, SpliceBio aims to further strengthen the evidence base supporting the efficacy of SB-007 in treating Stargardt disease.
In conclusion, SpliceBio’s recent funding success not only propels the advancement of SB-007 for Stargardt disease but also signifies a significant step towards expanding the scope of genetic medicines across various therapeutic areas. The company’s innovative Protein Splicing platform has the potential to revolutionize gene therapy by overcoming traditional limitations, offering hope to patients with genetic disorders previously considered untreatable. With a strong investor base and a robust pipeline of genetic medicine programs, SpliceBio is at the forefront of pioneering novel treatments that could transform the landscape of genetic medicine.
Key Takeaways:
– SpliceBio’s $135 million Series B financing will drive the clinical development of SB-007 for Stargardt disease and expand the company’s genetic medicine pipeline.
– The Protein Splicing platform enables SpliceBio to deliver large genes via dual AAV vectors, overcoming traditional limitations in gene therapy.
– Involvement of leading investors like EQT Life Sciences, Sanofi Ventures, and Roche Venture Fund underscores the transformative potential of SpliceBio’s approach in genetic medicines.
– SB-007’s progress from IND clearance to clinical trials marks a significant milestone in gene therapy, offering hope to patients with Stargardt disease and other genetic disorders.
Tags: gene therapy, regulatory, protein engineering
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