You are the gateway to gene therapy
Help connect patients living with sickle cell disease (SCD) to gene therapy sooner.
Patients living with SCD may continue to experience irreversible disease burden over time as a result of recurring vaso-occlusive events (VOEs).1
As a referring physician, you play a critical role in identifying appropriate patients and helping to initiate the path toward gene therapy evaluation.
You are the gateway to gene therapy
Help connect patients living with sickle cell disease (SCD) to gene therapy sooner.
Patients living with SCD may continue to experience irreversible disease burden over time as a result of recurring vaso-occlusive events (VOEs).1
As a referring physician, you play a critical role in identifying appropriate patients and helping to initiate the path toward gene therapy evaluation.
Refer early to help halt the progression of SCD.1-5
Every VOE contributes to irreversible organ damage, narrowing the therapeutic window for meaningful intervention.1,2,4 Referring earlier is how you interrupt the cycle of VOEs before organ damage accumulates.2,3,5
You don’t need to manage gene therapy, just recognize when it’s time to act. Timely referral can open the door to transformative treatment.1,4,5
A referral isn't a handoff—it's a clinical escalation.
Referring for gene therapy with LYFGENIATM helps patients access specialized evaluation while preserving continuity of care.
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Patient Identification QTC Partnership Eligibility Criteria When & How to Refer Request a RepJump to topics on page
Patient IdentificationQTC Partnership
Eligibility Criteria
When & How to Refer
Request a Rep
Rethink patient eligibility. Reimagine what’s possible with LYFGENIA.
Not just for the most severe—LYFGENIA may be right for more patients than you think.
The impact of SCD is not one-size-fits-all. VOEs can vary in frequency and severity, but even patients with fewer VOEs can experience long-term organ damage and disruptions to their lives.6,7
LYFGENIA is a gene addition therapy with the potential to decrease or stop VOEs.8
When considering treatment, expand how you see eligibility:
Younger patients (≥ 12 years of age) who may benefit from earlier intervention to help prevent cumulative organ damage2,3,5,8; older patients are eligible as well and have already been successfully enrolled9
Those experiencing fewer VOEs can still mean hospitalizations, school/work interruptions, and long-term health risks6,7
History of inadequate response to disease-modifying therapies (DMTs) such as hydroxyurea or dependence on chronic transfusion therapy8,10,11
Individuals looking to lessen the impact of their VOEs, including crises, and pursue what matters most in their lives8
Those seeking a different future, defined less by unpredictable VOEs and more by control8
Identifying Patients for Treatment with LYFGENIA
A peer-to-peer discussion of key considerations for identifying patients living with SCD who may be appropriate to refer for LYFGENIA
Broadening how you identify patients is critical.
Proactive identification can help prevent continued disease burden, rather than reacting after irreversible damage.2,3,5 We can help you get started.
Confidently identify, evaluate, and discuss LYFGENIA with appropriate patients.
Help patients and caregivers determine their readiness for LYFGENIA and referral for care.
Identify patients first. Access can be navigated.
We're here to help. Genetix Biotherapeutics is committed to supporting patient access and coverage throughout the treatment journey.
Coverage approvals have been achieved across multiple payer types, with access pathways being established across commercial and government insurance plans.
Patients continue to gain access to LYFGENIA, with no ultimate denials from payers reported to date.12
Shared care can help reduce practice burden.
Qualified Treatment Centers (QTCs) are specialized treatment sites that deliver gene therapy while partnering with referring providers to manage evaluation, coordination, and treatment logistics.
QTCs work in coordination with you to deliver LYFGENIA to your patients.
LYFGENIA QTCs provide the specialized expertise, infrastructure, and multidisciplinary support required for gene therapy. After referral, you may stay informed and involved as part of the continuation of care for your patients.
QTCs follow rigorous quality and oversight standards, which include accreditation by organizations such as the Foundation for the Accreditation of Cellular Therapy (FACT).
Not sure how to get started?
Your representative can help connect you with a QTC and support next steps in the referral process.
Help patients prepare for their first appointment with the QTC team: Patient Treatment Readiness Tool
Confirm the right patients and prepare them to move forward.
Identifying potential patients is the first step. From there, evaluating their ability to participate in the treatment journey includes both their clinical and personal readiness.
Clinical considerations
- Confirm patients meet core clinical eligibility criteria8
- Consider lifestyle factors that may affect treatment planning
- Provide education early to support informed consideration of gene therapy from the start
Patient readiness
- Consider whether patients have support in place for treatment and recovery
- Assess their ability to manage time away
- Discuss willingness to undergo conditioning, monitoring, and long-term follow-up8
Move from consideration to action with Genetix CARES.
Enroll your patients at no cost to help:
- Confirm eligibility and identify coverage from the start
- Access support and resources across the treatment journey
Support that never stops
Know the moment to act:
When disease control is no longer enough
If your patient continues to experience worsening symptoms or recurrent VOEs despite current therapy, it may be time to consider gene therapy.8,10,11
When today's symptoms signal tomorrow's risk
Ongoing VOEs and disease burden don’t impact just the present; they increase the risk of irreversible organ damage and long-term complications.1,2,13
When SCD begins to limit life
If your patient’s disease is putting their current or future goals at risk, it may be time to explore a different path.14
Gene therapy conversations don't have to wait.
When disease burden continues to accumulate, earlier action matters.2,3,5
Referral is the first step toward halting the progression of SCD, and it's simple2,3,5:
Identify a patient who may benefit
Reach out to a Genetix representative
Genetix identifies a QTC for referral
From there, Genetix and the QTC guide the process so you and your patient aren't navigating it alone.
Request a RepresentativeRequest a Representative
Have questions about eligibility, referrals, or next steps? Please fill out the form below to have a Genetix representative contact you about LYFGENIA.