#United States #San Diego #gene therapy #Hemophilia A #Expression Therapeutics

Expression Therapeutics Unveils Promising Results for Hemophilia A Gene Therapy

Expression Therapeutics, Inc., a leading innovator in gene therapy, recently disclosed significant results from a Phase 1 clinical trial of its pioneering lentiviral vector-based gene therapy known as ET3, targeting Hemophilia A. The findings, unveiled during the Annual Meeting of the American Society of Hematology (ASH) in San Diego, California, promise hope and a potential cure for individuals affected by this severe genetic disorder.

Understanding Hemophilia A

Hemophilia A is characterized by a deficiency in blood coagulation Factor VIII (FVIII), making it one of the most common severe congenital bleeding disorders, with incidence rates around 1 in 5,000 male births. Patients afflicted with severe Hemophilia A often face life-threatening bleeding episodes, especially during adolescence and young adulthood, if left untreated. The financial burden of managing this affliction is staggering, with annual treatment costs in the United States ranging from $200,000 to $850,000, making it one of the priciest genetic disorders to manage lifetime.

As such, the introduction of a curative gene therapy could significantly reduce these long-term costs while providing a better quality of life for patients.

Groundbreaking Trial Results

The clinical trial, executed at Christian Medical College in Vellore, India, involved five male participants aged between 22 and 41 with severe Hemophilia A. Utilizing autologous hematopoietic stem cells (HSCs), researchers employed a lentiviral vector containing a novel FVIII transgene under a myeloid-directed CD68 promoter. Before the therapy, each participant had reported at least 20 bleeding episodes annually.

Remarkably, after undergoing the therapy, none of the participants experienced spontaneous bleeding incidents, and their FVIII levels surged from undetectable to therapeutic levels. The safety profile of the trial was also encouraging; aside from expected side effects like neutropenia and thrombocytopenia, no severe adverse effects were noted. Furthermore, there were no indications of inhibitors to FVIII developing in any of the participants post-treatment.

The median follow-up duration of the study was 14 months, with the integration-site analysis, conducted 4 to 22 months after treatment, showing no safety concerns.

A New Hope for Treatment

This trial not only endorses a novel approach to gene therapy for Hemophilia A but suggests the potential for lifelong therapeutic benefits. Unlike other AAV gene therapy methods that struggled with expression durability and patient eligibility, the ET3 therapy appears to offer a robust solution that could cater to both adult and pediatric populations.

Dr. Pete Lollar, the Chairman of the Board of Expression Therapeutics, highlighted the exceptional safety and efficacy results from the trial, noting, "Lentiviral vector hematopoietic stem cell therapy offers the hope of permanent cure of Hemophilia A patients."

Mohan Rao, Ph.D., CEO of Expression Therapeutics, expressed gratitude towards collaborators and participants, emphasizing their unique therapeutic development initiative with curative potential. The company is committed to furthering the development of this promising lentiviral-mediated gene therapy to benefit Hemophilia A patients worldwide.

About Expression Therapeutics, Inc.

Based in Atlanta, Georgia, with a sophisticated GMP manufacturing facility in Cincinnati, Ohio, Expression Therapeutics stands at the forefront of clinical-stage gene and cell therapy. The company's mission is to advance curative therapies for Hemophilia A while also exploring innovative oncology solutions targeting neuroendocrine tumors and neuroblastoma.

The promising results from this Phase 1 trial not only illuminate the path forward for patients battling Hemophilia A but also set a foundation for future breakthroughs in the realm of genetic therapies.