Treatment pathway

You guide the care. We guide the process.

Overview of treatment with LYFGENIA™

LYFGENIA is a one-time treatment process that is administered at a Qualified Treatment Center (QTC). The main steps are1:

  • Pre-treatment
  • Stem cell collection
  • LYFGENIA production
  • Conditioning and washout
  • LYFGENIA infusion
  • Monitoring and follow-up

Referring to Qualified Treatment Centers

QTCs are independently owned and operated specialized hospitals. Each QTC has been carefully selected and trained to administer LYFGENIA, and the broad network of QTCs provides patients with convenient coast-to-coast access within the US.

The primary hematologist plays a pivotal role in identifying and referring appropriate patients for LYFGENIA. Through a shared-care approach, QTCs provide the specialized expertise, infrastructure, and treatment coordination required for gene therapy while keeping referring providers connected to key patient care decisions throughout the treatment journey.

Find a Qualified Treatment Center

6 important steps to treatment with LYFGENIA

REMINDER

Preparation matters before, during, and after treatment

Preparation matters before, during, and after treatment

Knowing what to expect helps patients move forward with confidence, so it's important to set realistic expectations around treatment and recovery.

The LYFGENIA journey does not end at infusion

Patients treated with LYFGENIA require ongoing monitoring and long-term follow-up after infusion. Set expectations early that long-term monitoring remains an important part of care.

Advise patients on the following:

  • The risk of manufacturing failure—in case of manufacturing failure or the need for additional cells, additional cell collection and manufacturing of LYFGENIA would be needed
  • The risks associated with mobilization and myeloablative conditioning agents

Additionally advise patients of the following:

  • Hematologic malignancy has occurred in patients treated with LYFGENIA. Patients with sickle cell disease have an increased risk of hematologic malignancy, compared to the general population. The additional hematopoietic stress associated with mobilization, conditioning, and infusion of LYFGENIA, including the need to regenerate the hematopoietic system, may increase the risk of a hematologic malignancy
  • Delayed platelet engraftment has been observed with LYFGENIA. Patients should be made aware of the risk of bleeding until platelet recovery has been achieved
  • Risk of neutrophil engraftment failure—patients who experience neutrophil engraftment failure will receive rescue treatment with their back-up collection of CD34+ cells
  • Insertional oncogenesis—there is a potential risk of insertional oncogenesis after treatment with LYFGENIA. Patients should be monitored lifelong
  • Monitoring will include assessment for hematologic malignancies with a complete blood count at least every 6 months for at least 15 years after treatment with LYFGENIA. This will include integration site analysis at Months 6, 12, and as warranted

Post-marketing long-term follow-up study:

Patients who intend to receive treatment with LYFGENIA are encouraged to enroll in the study, as available, to assess the long-term safety of LYFGENIA and the risk of malignancies occurring after treatment with LYFGENIA by calling Genetix Biotherapeutics at 1-833-999-6378. The study includes monitoring (at pre-specified intervals) for clonal expansion

Advise patients to:

  • Have their treating physician contact Genetix Biotherapeutics at 1-833-999-6378 if patients are diagnosed with a malignancy
  • Monitor for signs and symptoms of bleeding and have frequent blood draws for platelet counts until platelet recovery has been achieved
  • Not donate blood, organs, tissues, or cells at any time in the future
  • Understand that they may have a false-positive result for HIV if tested using a PCR-based assay after being treated with LYFGENIA
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