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Genetix Biotherapeutics

Develops and commercializes autologous gene therapies

Overview

Genetix Biotherapeutics develops autologous gene therapies using a lentiviral vector to insert a correct gene into a patient’s own blood stem cells, then re-infuses the cells to treat the underlying disease. It has three FDA-approved products: LYFGENIA for sickle cell disease, ZYNTEGLO for transfusion-dependent β-thalassemia, and SKYSONA for cerebral adrenoleukodystrophy. The company differentiates itself by using an established LVV-based platform and by expanding manufacturing capacity and access through partnerships with Qualified Treatment Centers. Its goal is to provide durable, curative treatments for genetic disorders and to broaden global patient access to these therapies.

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About Genetix Biotherapeutics

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Industries

BiotechnologyHealthcare

Company Size

51-200

Company Stage

N/A

Total Funding

N/A

Headquarters

Somerville, MA

Founded

N/A

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Benefits

  • Health Insurance
  • Life Insurance
  • Disability Insurance
  • 401(k) Company Match
  • Hybrid Work Options
  • Tuition Reimbursement
  • Student Loan Assistance
  • Parental Leave
  • Paid Holidays
  • Commuter Benefits

Company News

Business Wire
Oct 19th, 2026
Genetix Biotherapeutics names Meredith Manning chief commercial officer

Genetix Biotherapeutics has appointed Meredith Manning as chief commercial officer. She will lead commercial strategy for the Somerville, Massachusetts company's three gene addition cell therapies approved by the US Food and Drug Administration (FDA). Manning has more than 25 years of biopharmaceutical experience. She was most recently chief commercial officer at Soleno Therapeutics, which Neurocrine Biosciences acquired. She also held senior roles at PharmaEssentia and resTORbio, and led a $3 billion global hemophilia portfolio at Shire. "Her leadership will help accelerate our significant growth trajectory," said chief executive officer David Meek. Genetix aims to reach tens of thousands of eligible patients. The therapies treat sickle cell disease, ß-thalassemia, and cerebral adrenoleukodystrophy.

boerse.de Group
Mar 8th, 2026
Lonza and Genetix Biotherapeutics Extend Commercial Manufacturing Agreement for ZYNTEGLO(TM)

Lonza and Genetix Biotherapeutics Extend Commercial Manufacturing Agreement for ZYNTEGLO(TM). Montag, 09.03.26 14:00 Link kopieren Bildquelle: fotolia.com | Lonza Group AG / Key word(s): Partnership/Agreement Lonza and Genetix Biotherapeutics Extend Commercial Manufacturing Agreement for ZYNTEGLO(TM) 09.03.2026 / 14:00 CET/CEST * Extended agreement will expand manufacturing capacity at Lonza Houston (US) to support growing demand for Genetix's ZYNTEGLO(TM)(betibeglogene autotemcel), the only FDA-approved gene therapy for pediatric and adult patients with transfusion dependent beta-thalassemia * Long-term collaboration began in 2013 and enabled commercial approval and rapid uptake for ZYNTEGLO(TM) in 2022 Basel, Switzerland, 9 March 2026 - Lonza, one of the world's largest contract development and manufacturing organizations (CDMOs), and Genetix Biotherapeutics Inc. (Genetix), a commercial-stage biotechnology company dedicated to delivering genetic therapies for patients with severe rare diseases, today announced the extension of their long-term commercial manufacturing agreement. Under the expanded agreement, Lonza will expand manufacturing capacity to support growing demand for Genetix's ZYNTEGLO(TM), the only FDA-approved gene therapy for pediatric and adult patients with transfusion-dependent beta-thalassemia. The extended agreement further strengthens the strategic collaboration established in 2013 between Lonza and Genetix, which later enabled ZYNTEGLO(TM) commercial approval in 2022. Under the renewed agreement, commercial manufacturing for Genetix will continue at Lonza's Houston (US) facility, a dedicated cell and gene therapy site with nearly 10 years of combined clinical and commercial experience supporting the manufacture of this innovative treatment. The collaboration also makes provisions to scale up manufacturing in the future for additional Genetix therapies. Daniel Palmacci, Head of Specialized Modalities at Lonza, said: "Our extended agreement with Genetix underscores the value of our services and expertise in commercial cell and gene therapy manufacturing. We are proud to continue our collaboration by expanding the commercial production of ZYNTEGLO(TM) at our state-of-the-art manufacturing facility in Houston." Brian Riley, President and Chief Technology Officer at Genetix, added: "Our long-standing partnership with Lonza reflects our shared commitment to commercial excellence and operational discipline to bring curative therapies to patients impacted by rare disease. Their scientific rigor, quality focus and consistent execution have been important in enabling a reliable supply of ZYNTEGLO(TM). In the last year, patient demand for Genetix's therapies has grown consistently and rapidly. Extending the agreement with Lonza and expanding capacity provides a strong foundation as we invest to meet the growing patient demand and broaden our long-term manufacturing strategy." About Lonza Lonza is one of the world's largest contract development and manufacturing organizations (CDMOs) dedicated to serving the healthcare industry. Working across five continents, our global team of approximately 20,000 colleagues works alongside pharma and biotech companies to turn their breakthrough innovations into viable therapies. We support our customers in bringing life-saving and life-enhancing treatments to patients worldwide with a combination of cutting-edge science, smart technology and lean manufacturing. Our company generated sales of CHF 6.5 billion with a CORE EBITDA of CHF 2.1 billion in Full-Year 2025. Find out more at www.lonza.com 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 ZYNTEGLO(TM)(betibeglogene autotemcel) ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene addition therapy approved for eligible patients with transfusion-dependent beta-thalassemia who require regular red blood cell (RBC) transfusions, in which functional copies of a modified form of the beta-globin gene (β[A-T87Q-] globin gene) is added into a patient's own HSPCs. This addition results in the production of adult hemoglobin (HbA[T87Q]) enabling total hemoglobin to reach normal or near normal levels. ZYNTEGLO has been shown to eliminate the need for regular RBC transfusions. ZYNTEGLO is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular RBC transfusions. The FDA previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation for betibeglogene autotemcel. Lonza Contact Details Victoria Morgan Head of External Communications Lonza Group Ltd [email protected] Daniel Buchta Head of Investor Relations Lonza Group Ltd Tel +41 61 316 2985 [email protected] Genetix Media Contact [email protected] Additional Information and Disclaimer Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited ("SGX-ST"). Lonza Group Ltd is not subject to the SGX-ST's continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual. Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release. All trademarks belong to Lonza and are registered in CH, US and/or EU, or belong to their respective third-party owners and are used only for informational purposes. Privacy Policy link To immediately delete all the data End of Media Release View original content: EQS News | | Language: | English | | Company: | Lonza Group AG | | / | Münchensteinerstrasse 38 | | / | 4052 Basel | | / | Switzerland | | Phone: | +4161 316 81 11 | | Internet: | www.lonza.com | | ISIN: | CH0013841017 | | Valor: | 1384101 | | Listed: | SIX Swiss Exchange | | EQS News ID: | 2274042 | | End of News | EQS News Service | 2274042 09.03.2026 CET/CEST Quelle: DGAP

Business Wire
Mar 2nd, 2026
Genetix achieves first profitable quarter with >100 gene therapy patients treated in 2025

Genetix Biotherapeutics, a leader in genetic therapies for hemoglobinopathies, announced strong 2025 commercial results and outlined 2026 strategic priorities. The company achieved its first profitable quarter after a take-private acquisition and strategic turnaround. Over 100 patients received infusions in 2025, with more than 150 completing their first cell collection. Sickle cell patient enrolments for LYFGENIA treatment doubled year-over-year to approximately 150. The company ended 2025 with 70 active Qualified Treatment Centres across the US, the largest network for hemoglobinopathy gene therapies nationwide. For 2026, Genetix aims to achieve its first full year of profitability whilst doubling commercial manufacturing capacity through partnerships with Minaris, Lonza and MilliporeSigma. The company is also pursuing label expansion for LYFGENIA to include paediatric sickle cell patients under 12 years of age.

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