LYFGENIA is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.2

Following treatment with LYFGENIA, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions. LYFGENIA has not been studied in patients with more than two α-globin gene deletions.2

HOW IT WORKS

LYFGENIA is a single dose autologous gene addition therapy2

Designed to produce anti-sickling adult hemoglobin (HbAT87Q) to potentially decrease or stop vaso-occlusive events (VOEs).2†‡§
Learn how LYFGENIA works

CLINICAL EFFECTIVENESS

A one-time transformational sickle cell disease (SCD) gene therapy with the potential to decrease or stop VOEs2

88% (n=28/32, CI: 71, 97) of evaluable patients in Study 1-C achieved complete resolution of VOEs (VOE-CR) between 6 and 18 months after infusion of LYFGENIA.2†‡§
See the efficacy data

VOE-CR = elimination of VOEs between 6 and 18 months post infusion.2

†VOEs were defined as any of the following events requiring evaluation at a medical facility: an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours, acute chest syndrome, acute hepatic sequestration, or acute splenic sequestration.2

‡After the primary evaluation period to last follow-up, 4 of 28 patients who achieved VOE-CR experienced VOEs.2

§The efficacy of LYFGENIA was studied in a single-arm, 24-month, open-label, multicenter Phase 1/2 study (Study 1-C) and a long-term follow-up study. In Study 1-C, 32 patients with a history of at least 4 VOEs in the 24 months prior to informed consent were evaluated for VOEs.2

Peer-to-Peer Insights on LYFGENIA

This video series is led by experts Beth Krieger, MD and Nirmish Shah, MD as they discuss LYFGENIA efficacy, safety, and patient identification. Each segment features a detailed review of its topic and a conversation about how it’s applied in practice.

Overview of SCD and Prevalence of VOEs

How LYFGENIA Works

Understanding Viral Vectors

LYFGENIA Safety and Efficacy

Identifying Patients for Treatment with LYFGENIA

Genetix Patient Support Program

Support that never stops

Even before LYFGENIA is prescribed, your patients can enroll in and receive access to Genetix Patient Support—a program that provides a range of support from helping to identify Qualified Treatment Center options for consultation to understanding insurance benefits and the process for prior authorization.

Patient Navigators are here to help with education, access, and logistics throughout the decision process and beyond.

patientsupport
@genetixbiotx.com

1-833-888-6378

Genetix Patient Support Program
Find a Qualified Treatment Center (QTC)

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