Genetix Biotherapeutics to Highlight U.S. Gene Therapy Implementation and Patient Experience Data at ASH 2025
Genetix Biotherapeutics Inc. announced that an analysis of recent patient experience data from the U.S. commercial implementation of its landmark gene therapies LYFGENIA™ and ZYNTEGLO™ has been selected for an oral presentation at the 67th American Society of Hematology (ASH) Annual Meeting, scheduled to take place December 6–9, 2025, in Orlando, Florida. This recognition underscores the growing significance of real-world gene therapy data and reflects Genetix’s commitment to transforming the lives of patients with severe inherited blood disorders through cutting-edge genetic medicine.
The presentation will deliver new insights into how the company’s commercial rollout of LYFGENIA and ZYNTEGLO has translated into tangible patient benefits, highlighting lessons learned from early implementation, access acceleration, and treatment delivery across specialized centers in the United States.
Transforming Care with One-Time Gene Therapy
“We are pleased to share this analysis for the first time at ASH, which demonstrates the rising acceptance and administration of our one-time gene therapies,” said Joanne Lager, MD, Chief Medical Officer of Genetix Biotherapeutics. “The presentation highlights the growing demand for ZYNTEGLO, the first FDA-approved gene therapy for eligible patients with transfusion-dependent beta-thalassemia, and how experience from the ZYNTEGLO launch paved the way for more rapid access to LYFGENIA treatment for sickle cell patients nationwide.”
Dr. Lager emphasized that this real-world experience marks a turning point in the treatment of hemoglobinopathies, complex genetic blood disorders that historically required lifelong management and chronic transfusions. Both therapies represent durable, one-time gene therapy solutions designed to address the underlying genetic causes of disease rather than merely alleviating symptoms.
The analysis presented at ASH will trace the patient journey from initial enrollment through infusion, providing a comprehensive overview of the milestones achieved and challenges overcome during the transition from clinical development to commercial implementation. According to Genetix, the data demonstrates meaningful progress in streamlining logistics, accelerating patient access, and ensuring equitable delivery of care across diverse populations.
“These advancements set the standard for delivery of ex vivo gene therapy in hemoglobinopathies,” Dr. Lager added. “They reinforce the value of durable, one-time gene therapy to significantly improve patient outcomes and provide meaningful benefits to patients, providers, and payers.”
Gene Therapy at a Pivotal Moment in Hematology
The upcoming ASH 2025 presentation, titled “Accelerating Access to Gene Therapy: Lessons from Commercial Implementation in Sickle Cell Disease and Transfusion-Dependent Thalassemia” (Abstract #948), will explore how coordinated partnerships among treatment centers, payers, and patient support programs have enabled scalable delivery of advanced therapies that once seemed out of reach.
ZYNTEGLO™, approved by the U.S. Food and Drug Administration (FDA) as the first gene therapy for transfusion-dependent beta-thalassemia (TDT), offers patients the potential to achieve independence from chronic blood transfusions. The treatment works by inserting a functional copy of the β-globin gene into a patient’s hematopoietic stem cells, allowing them to produce sufficient hemoglobin and effectively reduce or eliminate the need for transfusions. Since its introduction, ZYNTEGLO has transformed the standard of care for eligible patients who previously relied on lifelong supportive therapies.
Building on this foundation, LYFGENIA™ represents a breakthrough for sickle cell disease (SCD), a condition that has historically lacked curative options for most patients. Approved as a one-time, autologous gene therapy, LYFGENIA modifies a patient’s stem cells to produce functional hemoglobin capable of preventing the polymerization that causes sickling of red blood cells. This innovation aims to reduce vaso-occlusive events, organ damage, and other serious complications associated with SCD.
The company’s latest analysis evaluates how the successful commercial implementation of ZYNTEGLO informed and accelerated the national rollout of LYFGENIA. Early real-world data demonstrate improvements in patient throughput, enhanced coordination between authorized treatment centers, and the development of best practices that streamline complex manufacturing and infusion logistics. These insights are expected to guide future gene therapy deployments for other genetic diseases.
Elevating the Standard of Care for Genetic Blood Disorders
Genetix Biotherapeutics has positioned itself as a global leader in ex vivo lentiviral gene therapy, pioneering a commercial model that combines scientific innovation with robust patient support systems. The company’s integrated approach focuses not only on the delivery of gene therapy products but also on comprehensive care coordination, including patient education, financial navigation, and long-term follow-up.
As the number of patients treated with gene therapy continues to rise, Genetix’s data offer valuable perspective on what successful, sustainable implementation looks like in real-world healthcare settings. The findings to be presented at ASH will highlight both the measurable patient outcomes and the operational advancements that have enabled broader access to these transformative therapies.
The presentation will also showcase the company’s collaborations with leading treatment centers and hematology experts, illustrating how multidisciplinary teamwork has accelerated time-to-treatment for eligible patients. Genetix’s commercial strategy emphasizes equitable access, particularly for populations historically underrepresented in advanced therapeutic research.
Setting the Stage for the Future of Genetic Medicine
The 67th ASH Annual Meeting represents one of the world’s premier scientific gatherings in hematology, bringing together clinicians, researchers, and industry leaders to share breakthroughs shaping the future of blood disorder treatment. Genetix’s participation at this year’s meeting underscores the company’s ongoing role in driving innovation and expanding the reach of gene therapy across clinical and commercial landscapes.
The company’s oral presentation is scheduled for Monday, December 8, 2025, at 4:00 PM EST, and will provide a detailed account of the progress made since the launch of ZYNTEGLO and LYFGENIA. Attendees can find additional session details through the ASH 2025 online program and visit the Genetix Biotherapeutics booth (#1261) onsite for further engagement and discussion with the company’s medical and scientific teams.
Through continued commitment to innovation, patient-centered design, and real-world data generation, Genetix Biotherapeutics is helping define the next era of treatment for genetic blood diseases—where a single infusion can change a lifetime of care.
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