Minaris expands commercial manufacturing for LYFGENIA™, a first-in-class FDA approved gene therapy for patients with sickle cell disease
Expanded capacity meets growing patient demand while delivering improved manufacturing efficiency and accelerated treatment timelines for patients
PHILADELPHIA, Jan. 12, 2026 /PRNewswire/ -- Minaris, a global cell and gene therapy (CGT) contract development and manufacturing organization (CDMO) and multimodality biosafety testing provider, and Genetix Biotherapeutics Inc., a commercial-stage biotechnology company dedicated to delivering genetic therapies for patients with severe rare diseases, today announced an expanded manufacturing partnership to increase commercial-scale production of LYFGENIA, Genetix's FDA-approved, one-time gene therapy for individuals 12 years and older with sickle cell disease. The collaboration significantly increases manufacturing capacity, enabling Genetix to meet growing commercial demand while maintaining the quality, consistency, and regulatory rigor required for commercial CGT manufacturing. The partnership builds on a strong existing relationship and reflects Minaris' ability to grow alongside its partners as products advance and supply needs expand.
To facilitate the expansion, Minaris Advanced Therapies has implemented process and operational improvements that increase manufacturing efficiencies and reduce turnaround times, while significantly increasing output. Minaris has continued to invest in infrastructure, talent, and capacity to ensure it can support long-term commercial supply and respond to increasing demand without disruption.
"Minaris is proud to partner with Genetix to continue to bring this transformative therapy to patients. This expansion reflects the reality of commercial cell and gene therapies, where demand can increase quickly," said Dr. Eytan Abraham, Chief Commercial & Technology Officer at Minaris. "Our focus is on investing early, building efficiency into the process, and ensuring our partners have the capacity and cost structure to scale as patient needs grow."
The collaboration reinforces Minaris' broader mission to support advanced therapy developers through the transition from development to sustained commercial supply. With strengthened capacity and a focus on continuous improvement, Minaris is positioned to support Genetix's growth while maintaining the operational excellence required for regulated commercial manufacturing.
"The increasing growth in commercial demand for LYFGENIA requires us to meaningfully expand our manufacturing capacity. Minaris has been a reliable, highly effective and innovative partner bringing our curative therapies to patients in need.," said Brian Riley, President & Chief Technical Officer at Genetix. "They understand the technical demands of manufacturing a genetic therapy and have consistently demonstrated the essential capabilities needed to support our growing commercial supply needs."
About Minaris
Minaris is a global cell and gene therapy (CGT) CDMO and a multimodality biosafety testing provider. With more than 25 years of CGT development and manufacturing expertise and more than 40 years in biosafety testing, Minaris combines deep legacy experience with state-of-the-art GMP facilities, and is a fully US-owned organization under new ownership and management, built for today's evolving therapeutic landscape. Our CDMO team supports CGT innovators from IND through commercial supply, and our testing business delivers GMP analytics, biosafety, viral clearance, and product characterization across CGT and biologics. With 5 global sites on 3 continents, we operate with one mission—to support clinical and commercial therapeutic programs hand in hand with our partners to get more therapies to more patients. We are united by our commitment to excellence, reliability, and a patient-focused purpose as we enable innovators to deliver new therapies and expand patient access worldwide. For more information, visit https://minaris.com or follow us on LinkedIn.
About Genetix Biotherapeutics
Genetix Biotherapeutics Inc. is a privately held, commercial-stage biotechnology company dedicated to delivering genetic therapies for patients with severe rare diseases. Backed by more than 30 years of pioneering genetic therapy innovation, the company has FDA-approved therapies for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy that directly treat the underlying cause of disease. Genetix is committed to commercial execution, scaling patient access, and improving the treatment experience for patients and providers. Genetix is headquartered in Somerville, Massachusetts.
About LYFGENIA™ (lovotibeglogene autotemcel)
LYFGENIA is a one-time ex-vivo lentiviral vector (LVV) gene addition therapy approved for eligible patients with sickle cell disease, in which a functional β-globin gene is added to patients' own hematopoietic (blood) stem and progenitor cells (HSPCs). This addition results in the production of adult hemoglobin with anti-sickling properties (HbAT87Q) which has a similar oxygen-binding affinity to wild-type HbA. LYFGENIA has been shown to limit sickling of red blood cells and reduce or eliminate vaso-occlusive events (VOEs).
LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of VOEs.
The FDA previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation for lovotibeglogene autotemcel.
Limitations of Use
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.
IMPORTANT SAFETY INFORMATION FOR LYFGENIA (lovotibeglogene autotemcel)
Boxed WARNING: HEMATOLOGIC MALIGNANCY
Hematologic malignancy has occurred in patients treated with LYFGENIA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 6 months and through integration site analysis at Months 6, 12, and as warranted.
Hematologic Malignancy
Hematologic malignancy has occurred in patients treated with LYFGENIA (Study 1, Group A). At the time of initial product approval, two patients treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A) developed acute myeloid leukemia (AML). One patient with α-thalassemia trait (Study 1, Group C) has been diagnosed with myelodysplastic syndrome (MDS).
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. Patients with sickle cell disease have an increased risk of hematologic malignancy as compared to the general population.
Patients treated with LYFGENIA may develop hematologic malignancies and should have lifelong monitoring. Monitor for hematologic malignancies with a complete blood count (with differential) at least every 6 months for at least 15 years after treatment with LYFGENIA, and integration site analysis at Months 6, 12, and as warranted.
In the event that a malignancy occurs, contact Genetix Biotherapeutics at 1-833-999-6378 for reporting and to obtain instructions on collection of samples for testing.
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.
Delayed Platelet Engraftment
Delayed platelet engraftment has been observed with LYFGENIA. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia. Two patients (4%) required more than 100 days post treatment with LYFGENIA to achieve platelet engraftment.
Patients should be made aware of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding according to standard guidelines. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise.
Neutrophil Engraftment Failure
There is a potential risk of neutrophil engraftment failure after treatment with LYFGENIA. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 0.5 × 109 cells/L obtained on different days by Day 43 after infusion of LYFGENIA. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with LYFGENIA, provide rescue treatment with the back-up collection of CD34+ cells.
Insertional Oncogenesis
There is a potential risk of lentiviral vector-mediated insertional oncogenesis after treatment with LYFGENIA.
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of LYFGENIA. The dimethyl sulfoxide (DMSO) or dextran 40 in LYFGENIA may cause hypersensitivity reactions, including anaphylaxis.
Anti-retroviral Use
Patients should not take prophylactic HIV anti-retroviral medications for at least one month prior to mobilization and until all cycles of apheresis are completed. There are some long-acting anti-retroviral medications that may require a longer duration of discontinuation for elimination of the medication. If a patient is taking anti-retrovirals for HIV prophylaxis, confirm a negative test for HIV before beginning mobilization and apheresis of CD34+ cells.
Hydroxyurea Use
Patients should not take hydroxyurea for at least 2 months prior to mobilization and until all cycles of apheresis are completed. If hydroxyurea is administered between mobilization and conditioning, discontinue 2 days prior to initiation of conditioning.
Iron Chelation
Drug-drug interactions between iron chelators and the mobilization process and myeloablative conditioning agent must be considered. Iron chelators should be discontinued at least 7 days prior to initiation of mobilization or conditioning. Do not administer myelosuppressive iron chelators (e.g., deferiprone) for 6 months post-treatment with LYFGENIA. Non-myelosuppressive iron chelation should be restarted no sooner than 3 months after LYFGENIA infusion. Phlebotomy can be used in lieu of iron chelation, when appropriate.
Interference with PCR-based Testing
Patients who have received LYFGENIA are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to integrated BB305 LVV proviral DNA, resulting in a possible false-positive PCR assay test result for HIV. Therefore, patients who have received LYFGENIA should not be screened for HIV infection using a PCR-based assay.
Adverse Reactions
The most common adverse reactions ≥ Grade 3 (incidence ≥ 20%) were stomatitis, thrombocytopenia, neutropenia, febrile neutropenia, anemia, and leukopenia.
Three patients died during LYFGENIA clinical trials; one from sudden cardiac death due to underlying disease and two from acute myeloid leukemia who were treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A).
Pregnancy/Lactation
Advise patients of the risks associated with myeloablative conditioning agents, including on pregnancy and fertility.
LYFGENIA should not be administered to women who are pregnant, and pregnancy after LYFGENIA infusion should be discussed with the treating physician.
LYFGENIA is not recommended for women who are breastfeeding, and breastfeeding after LYFGENIA infusion should be discussed with the treating physician.
Females and Males of Reproductive Potential
A negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before LYFGENIA administration.
Women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra-uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of LYFGENIA.
Advise patients of the options for fertility preservation.
Please see full Prescribing Information, including Boxed WARNING and Medication Guide for LYFGENIA.
Minaris Media Contact
Kristen White, Co-Founder & Partner
Oak Street Communications
[email protected]
Genetix Media Contact
SOURCE Minaris Advanced Therapies
Share this article