SpliceBio Secures FDA Approval for Innovative Stargardt Disease Treatment SB-007

SpliceBio Achieves FDA Clearance for SB-007

A Significant Breakthrough in Gene Therapy

SpliceBio, an innovative genetic medicines company, has announced that it received FDA clearance for its investigational new drug (IND) application for SB-007, a groundbreaking gene therapy designed to address Stargardt disease. This is a pivotal moment not only for SpliceBio but also for the thousands affected by this rare genetic condition, which previously lacked any approved treatments.

What is SB-007?

SB-007 is the only clinical-stage therapeutic that addresses the genetic root cause of Stargardt disease. This condition, which is characterized by progressive vision loss due to the degeneration of the retina, primarily results from mutations in the ABCA4 gene. The unique aspect of SB-007 lies in its ability to target all ABCA4 mutations, making it a comprehensive solution for all patients suffering from Stargardt disease.

Crucial to this therapy is its Protein Splicing technology, which delivers a full-length version of the ABCA4 protein through an adeno-associated viral (AAV) vector. According to SpliceBio's CEO, Dr. Miquel Vila-Perelló, the FDA’s clearance represents a major milestone in showing the potential of this novel approach to effectively treat diseases caused by large gene mutations.

The Clinical Study: Phase 1/2 ASTRA

Plans are in motion to commence a Phase 1/2 clinical study, named the ASTRA study, in the first half of 2025. This study aims to evaluate the safety and effectiveness of SB-007 when administered subretinally in Stargardt disease patients. Furthermore, the ASTRA study will be supported by the POLARIS trial, a natural history study focused on the progression of Stargardt disease that was launched by SpliceBio in March 2024. POLARIS will help to refine eligibility criteria for the clinical study, ensuring a streamlined approach to patient enrolment.

As noted by Professor Paul Yang from the Casey Eye Institute, the ability to replace the full-size ABCA4 protein through this innovative therapy could dramatically impact the lives of those affected by this degenerative disease. With no existing approved treatments available, the progress of SB-007 instills hope for many.

Outlook for Stargardt Disease Patients

Stargardt disease impacts approximately 1 in 8,000 to 10,000 individuals, making it the most common inherited form of juvenile macular degeneration. Patients face significant daily challenges as central vision deteriorates, affecting their quality of life and leading to greater dependence on others.

The FDA’s recognition of SB-007 as an Orphan Drug signifies its potential to significantly benefit this underserved community. As SpliceBio prepares for the upcoming clinical study, there is optimism that SB-007 could provide a viable treatment option, thereby addressing long-standing treatment gaps in the field of genetic medicine.

Future Developments

In addition to the Phase 1/2 clinical trials, SpliceBio's management team will be present at the J.P. Morgan's 43rd Annual Healthcare Conference scheduled for January 2025, where they will provide updates about SB-007 and its future trajectory.

As the landscape for genetic medicines evolves, the clearance of SB-007 underscores a newly forged path for treating genetic conditions, offering renewed hope to patients and their families—and shining a light on the transformative potential of innovative gene therapies.

For further updates on SpliceBio and its innovative therapeutic approaches, you can visit their website at www.splice.bio.