Betibeglogene autotemcel

Lonza and Genetix Biotherapeutics Inc. extended their long-term commercial manufacturing agreement to expand production capacity for the gene therapy betibeglogene autotemcel, or Zynteglo. The therapy is approved by the U.S. Food and Drug Administration for pediatric and adult patients with transfusion-dependent beta-thalassemia. Under the expanded agreement, Lonza will increase manufacturing capacity at its cell and gene therapy facility in Houston, Texas to support rising patient demand. The collaboration between the companies began in 2013 and supported the therapy’s commercial approval in 2022. Lonza said the Houston site has nearly a decade of combined clinical and commercial manufacturing experience supporting production of the therapy. The agreement also includes provisions to scale manufacturing capacity in the future for additional Genetix gene therapy programs. Brian Riley, president and chief technology officer at Genetix, said demand for the company’s therapies has increased over the past year and that expanding manufacturing capacity will support long-term supply. The agreement comes as Lonza continues to reshape its portfolio around contract development and manufacturing services. Last week, the company announced plans to divest its Capsules and Health Ingredients business to investment firm Lone Star Funds for $2.2 billion in upfront cash as part of its strategy to become a pure-play contract development and manufacturing organization. Lonza has also recently expanded technical capabilities across its network. In February, the company integrated an antibody-drug conjugate development platform into its advanced synthesis offering at its site in Oss, the Netherlands, to support early-phase bioconjugate development and manufacturing.

Genetix delivers strong 2025 revenue growth resulting in the Company’s first ever profitable quarter In 2025, >100 U.S. patients were treated and >150 U.S. patients completed their first cell collection cycle The vast majority of patients treated in 2025 required a single cell collection cycle (since launch, patients have required a median of 1 collection cycle) In 2026, Genetix is doubling manufacturing capacity, enabling a substantial increase in annual patients treated 2026 lifecycle management priorities include enabling cryopreserved apheresis and advancing efforts to expand LYFGENIA access to pediatric patients 100 patients received infusions in 2025, including >40 patients infused in Q4 The vast majority of patients treated in 2025 required a single cell collection cycle (since launch, patients have required a median of 1 collection cycle) >150 new patients completed their first cell collection An important leading indicator of commercial performance, 150 sickle cell patients completed their enrollment for LYFGENIA treatment (~100% increase from prior year) LYFGENIA and ZYNTEGLO experience meaningful access for >90% of eligible patients with commercial or Medicaid coverage Ended the year with 70 active Qualified Treatment Centers (QTCs) in the U.S., the largest network for hemoglobinopathy gene therapies nationwide 2026 Strategic Priorities Building on the success of 2025, the Company is focused on advancing several strategic priorities this year to further expand access to the tens of thousands of patients in the U.S. that could benefit from Genetix’s transformative therapies. Continued Commercial Growth and Profitability: targeting significant commercial growth and the Company’s first full year of profitability in 2026 driven by substantial increase in patient demand and treatments infused Manufacturing Expansion: strengthening partnerships with industry leading CDMOs—Minaris, Lonza, and MilliporeSigma (the U.S. and Canada business of Merck KGaA, Darmstadt, Germany) — to double commercial manufacturing capacity, expected by mid-2026 Manufacturing Optimization: finalizing several process development initiatives, including cryopreserved apheresis, to enhance the treatment experience for patients and providers LYFGENIA Label Expansion: efforts are underway via the HGB-210 clinical study to expand access to pediatric sickle cell patients 50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia. Drug Interactions Drug-drug interactions between iron chelators and the myeloablative conditioning agent must be considered. Iron chelators should be discontinued at least 7 days prior to initiation of conditioning. The prescribing information for the iron chelator(s) and the myeloablative conditioning agent should be consulted for the recommendations regarding co-administration with CYP3A substrates. Some iron chelators are myelosuppressive. After ZYNTEGLO infusion, avoid use of these iron chelators for 6 months. If iron chelation is needed, consider administration of non-myelosuppressive iron chelators. Phlebotomy can be used in lieu of iron chelation, when appropriate. Pregnancy/Lactation Advise patients of the risks associated with conditioning agents, including on pregnancy and fertility. ZYNTEGLO should not be administered to women who are pregnant, and pregnancy after ZYNTEGLO infusion should be discussed with the treating physician. ZYNTEGLO is not recommended for women who are breastfeeding, and breastfeeding after ZYNTEGLO 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 ZYNTEGLO 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 ZYNTEGLO. Advise patients of the option to cryopreserve semen or ova before treatment if appropriate. Please see full Prescribing Information and Patient Information for ZYNTEGLO. 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 treatments 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. Contacts For media inquiries: info@GenetixBioTx.com Website: