4DMT to Share Interim Data from 4D-710 AEROW Trial at European Cystic Fibrosis Conference

4D Molecular Therapeutics (4DMT), a prominent clinical-stage genetic medicines enterprise, is set to unveil interim data from its Phase 1/2 AEROW clinical trial at the imminent 47th European Cystic Fibrosis Conference in Glasgow, UK. This significant event is scheduled for June 6, 2024, where Dr. Jennifer L. Taylor-Cousar, the Principal Investigator of the AEROW clinical trial, will present the findings. The data focuses on the potential of 4D-710, an aerosolized gene therapy aimed at treating cystic fibrosis (CF) lung disease.

The detailed presentation, titled "CFTR transgene expression in airway epithelial cells following aerosolized administration of the AAV-based gene therapy 4D-710 to adults with cystic fibrosis lung disease," is part of the session WS06, which explores new therapeutic approaches. Dr. Taylor-Cousar's presentation will occur between 5:00 and 5:15 p.m. BST.

4DMT has also planned a comprehensive webcast to delve into the clinical data and provide updates on the program. This webcast is scheduled for June 6, 2024, at 8:00 a.m. ET. Interested parties can register for this event, and an archived version will be accessible for up to one year on the company's website.

Cystic fibrosis is a severe inherited disorder arising from mutations in the CFTR gene, impacting the lungs, pancreas, and other organs. A significant health concern, it affects around 40,000 individuals in the United States and over 105,000 globally. The disease is characterized by progressive lung function decline, chronic infections, and inflammation, necessitating lifelong treatment and frequent hospitalization.

4D-710, the focal point of the AEROW trial, incorporates a next-generation AAV vector, A101, and a codon-optimized CFTR∆R transgene. This therapy aims to address the needs of approximately 15% of CF patients who do not respond to current CFTR modulator treatments due to specific gene variants or drug intolerance. Additionally, 4DMT envisions expanding the application of 4D-710 to a broader CF population, either as a standalone therapy or in combination with existing treatments, addressing the variable and often incomplete response seen with current modulators.

4D-710's distinction is further solidified by its Rare Pediatric Disease Designation and Orphan Drug Designation granted by the U.S. Food and Drug Administration (FDA), underscoring its potential to fill an unmet medical need.

4DMT, headquartered in Emeryville, California, leverages its proprietary Therapeutic Vector Evolution platform to advance its diverse pipeline of genetic medicine candidates. This platform combines directed evolution technology with a vast library of synthetic AAV capsid sequences to engineer customized vectors. Currently, 4DMT is progressing five clinical-stage and two preclinical candidates aimed at treating diseases in ophthalmology, pulmonology, cardiology, and the central nervous system.

As a company dedicated to pioneering genetic medicines, 4DMT aims to revolutionize the treatment landscape for both rare and widespread diseases, offering potentially curative therapies for millions of patients. The company’s innovative approach and robust pipeline position it at the forefront of genetic medicine development.

While 4DMT's products are still under clinical investigation and not yet approved for marketing by regulatory authorities, the ongoing research and promising interim results contribute to a hopeful outlook for advancements in cystic fibrosis treatment and beyond.