Segment & Geographic Data
Quant-Grade Normalized (live API)
API & CSV Delivery
Uniqure N V's Business Segments
Uniqure N V's reported revenue by business segment and by geographic region, quarterly and annual, normalized against the consolidated income statement. Free below: the top 3 rows per table, this quarter and this fiscal year. Subscriber access adds the full segment history and operating income by segment.
Segment Data As of Q2 FY2025
Reportable Segments
1
Largest Segment
Single Reportable Segment
Total Revenue
$ 5
Regions Reported
-
Revenue Share by Reportable Segment - Q2 FY2025
- Single Reportable Segment100%
Revenue by Reportable Segment - Q2 FY2025
| Segment | Revenue (Millions) | % of Total |
|---|---|---|
| Single Reportable Segment | $ 5 | 100% |
Description of Uniqure N V
Uniqure N.V. is a gene therapy company headquartered in the Netherlands, with a focus on developing treatments for genetic and rare diseases. The company was founded in 1999 and has since become a leading player in the gene therapy industry.
The company's pipeline includes several candidates in clinical and pre-clinical development. One of its most promising programs is AMT-061, a gene therapy for patients with hemophilia B. Uniqure N.V. also has a collaboration with CSL Behring to develop gene therapies for hemophilia A and B.
Uniqure N.V. has established partnerships with major pharmaceutical companies, including Pfizer, Bristol-Myers Squibb, and AbbVie. The company has also received funding from the European Union to support its research and development efforts.
Overall, Uniqure N.V. is working to advance gene therapy as a new approach to treating genetic and rare diseases, and its innovative therapies are expected to have a significant impact on patients' lives.
The company's pipeline includes several candidates in clinical and pre-clinical development. One of its most promising programs is AMT-061, a gene therapy for patients with hemophilia B. Uniqure N.V. also has a collaboration with CSL Behring to develop gene therapies for hemophilia A and B.
Uniqure N.V. has established partnerships with major pharmaceutical companies, including Pfizer, Bristol-Myers Squibb, and AbbVie. The company has also received funding from the European Union to support its research and development efforts.
Overall, Uniqure N.V. is working to advance gene therapy as a new approach to treating genetic and rare diseases, and its innovative therapies are expected to have a significant impact on patients' lives.
uniQure N.V. is a pioneering Dutch gene therapy company committed to revolutionizing the treatment landscape for genetic diseases. It achieves this by developing cutting-edge, innovative therapies specifically tailored to meet the unique needs of patients suffering from rare genetic disorders. Below is a detailed overview of uniQures segments, products, and services.
Segments
uniQures business model encompasses two primary segments, each targeting distinct categories of genetic diseases:
1. Hematology:
- This segment concentrates on advancing gene therapy solutions for various blood disorders, particularly focusing on:
- Hemophilia B: uniQure has developed AMT-061, a transformative gene therapy designed to provide a functional copy of the factor IX gene. This therapy aims to significantly reduce incident bleeding episodes and enhance overall patient quality of life. The treatment introduces a stable version of the missing protein directly into the patients system, addressing the root cause of the disorder.
- Hemophilia A: For patients with hemophilia A, uniQure is working on AMT-180. This innovative therapy intends to deliver a working copy of the factor VIII gene, thereby restoring the production of this critical protein and mitigating the severe effects of clotting deficiency.
2. Central Nervous System (CNS):
- This segment is focused on the development of gene therapies for neurodegenerative disorders, including:
- Huntington’s Disease: AMT-130 is currently under development as a gene therapy aimed at treating Huntingtons disease. Its mechanism involves targeting and reducing the levels of the mutant huntingtin protein in the brain— a protein which contributes to the pathophysiology of Huntington’s— with the goal of alleviating symptoms and slowing disease progression.
- Parkinson’s Disease: uniQure is also advancing AMT-150, a therapy that is designed to amplify the production of Glial cell-derived neurotrophic factor (GDNF) in the brain. GDNF is known for its neuroprotective effects, particularly on dopamine-producing neurons, thereby addressing the neuronal loss seen in Parkinson’s disease.
Products
uniQure is dedicated to producing transformative gene therapy products that focus not just on symptom management but on the underlying genetic causes of diseases. Key products include:
- Glybera (alipogene tiparvovec): Glybera holds the distinction of being the first gene therapy approved in Europe (2012) and is specifically indicated for the treatment of adults suffering from lipoprotein lipase deficiency (LPLD). This rare genetic disorder leads to dangerously elevated triglyceride levels due to the absence of the lipoprotein lipase enzyme.
- AMT-060: This product represents an earlier version of the gene therapy for hemophilia B, currently in clinical development and designed to provide a functional copy of the factor IX gene.
- AMT-061: An enhanced version of AMT-060, AMT-061 aims to improve efficacy in treating hemophilia B and is currently undergoing clinical evaluation.
- AMT-130: This investigational gene therapy product targets Huntingtons disease, in clinical development with the goal of modifying the disease course by targeting mutant huntingtin levels.
- AMT-150: This therapy, currently in preclinical development, is aimed at addressing Parkinson’s disease through GDNF augmentation.
Services
In addition to developing gene therapies, uniQure offers a suite of services that facilitate the development, delivery, and implementation of their innovative treatments:
- Research and Development: A significant focus on R&D allows uniQure to explore novel therapeutic strategies and refine existing treatments. The company employs a robust team of experts specializing in gene therapy, bioprocessing, analytical development, and clinical research, enabling them to stay at the forefront of gene therapy advancements.
- Manufacturing: uniQure operates a state-of-the-art manufacturing facility located in Lexington, Massachusetts, where it produces its gene therapy products. The facility adheres to stringent quality standards to ensure the safety and efficacy of treatments. Additionally, the company collaborates with contract manufacturing organizations (CMOs) to enhance and scale up production as necessary.
- Patient Advocacy: uniQure places a strong emphasis on working with patient advocacy groups to promote awareness around genetic diseases. Their commitment ensures that patients have access to the necessary therapies and support services, enhancing the overall patient experience.
- Professional Education: To guarantee the safe and effective administration of gene therapies, uniQure provides education and training resources for healthcare professionals. This service aims to equip practitioners with the knowledge and skills needed to deliver these advanced therapies, facilitating better patient management.
Conclusion
In summary, uniQure N.V. stands out as a leader in the field of gene therapy, providing a diverse range of segments, innovative products, and comprehensive services focused on treating rare genetic diseases. The companys efforts in developing therapies for conditions like hemophilia B, hemophilia A, Huntingtons disease, and Parkinsons disease illustrate its commitment to transforming patient care and enhancing quality of life through groundbreaking gene therapies. By integrating robust research and development, advanced manufacturing capabilities, patient advocacy, and healthcare professional training, uniQure is positioned to make a significant impact in the field of genetic disease treatment.
Segments
uniQures business model encompasses two primary segments, each targeting distinct categories of genetic diseases:
1. Hematology:
- This segment concentrates on advancing gene therapy solutions for various blood disorders, particularly focusing on:
- Hemophilia B: uniQure has developed AMT-061, a transformative gene therapy designed to provide a functional copy of the factor IX gene. This therapy aims to significantly reduce incident bleeding episodes and enhance overall patient quality of life. The treatment introduces a stable version of the missing protein directly into the patients system, addressing the root cause of the disorder.
- Hemophilia A: For patients with hemophilia A, uniQure is working on AMT-180. This innovative therapy intends to deliver a working copy of the factor VIII gene, thereby restoring the production of this critical protein and mitigating the severe effects of clotting deficiency.
2. Central Nervous System (CNS):
- This segment is focused on the development of gene therapies for neurodegenerative disorders, including:
- Huntington’s Disease: AMT-130 is currently under development as a gene therapy aimed at treating Huntingtons disease. Its mechanism involves targeting and reducing the levels of the mutant huntingtin protein in the brain— a protein which contributes to the pathophysiology of Huntington’s— with the goal of alleviating symptoms and slowing disease progression.
- Parkinson’s Disease: uniQure is also advancing AMT-150, a therapy that is designed to amplify the production of Glial cell-derived neurotrophic factor (GDNF) in the brain. GDNF is known for its neuroprotective effects, particularly on dopamine-producing neurons, thereby addressing the neuronal loss seen in Parkinson’s disease.
Products
uniQure is dedicated to producing transformative gene therapy products that focus not just on symptom management but on the underlying genetic causes of diseases. Key products include:
- Glybera (alipogene tiparvovec): Glybera holds the distinction of being the first gene therapy approved in Europe (2012) and is specifically indicated for the treatment of adults suffering from lipoprotein lipase deficiency (LPLD). This rare genetic disorder leads to dangerously elevated triglyceride levels due to the absence of the lipoprotein lipase enzyme.
- AMT-060: This product represents an earlier version of the gene therapy for hemophilia B, currently in clinical development and designed to provide a functional copy of the factor IX gene.
- AMT-061: An enhanced version of AMT-060, AMT-061 aims to improve efficacy in treating hemophilia B and is currently undergoing clinical evaluation.
- AMT-130: This investigational gene therapy product targets Huntingtons disease, in clinical development with the goal of modifying the disease course by targeting mutant huntingtin levels.
- AMT-150: This therapy, currently in preclinical development, is aimed at addressing Parkinson’s disease through GDNF augmentation.
Services
In addition to developing gene therapies, uniQure offers a suite of services that facilitate the development, delivery, and implementation of their innovative treatments:
- Research and Development: A significant focus on R&D allows uniQure to explore novel therapeutic strategies and refine existing treatments. The company employs a robust team of experts specializing in gene therapy, bioprocessing, analytical development, and clinical research, enabling them to stay at the forefront of gene therapy advancements.
- Manufacturing: uniQure operates a state-of-the-art manufacturing facility located in Lexington, Massachusetts, where it produces its gene therapy products. The facility adheres to stringent quality standards to ensure the safety and efficacy of treatments. Additionally, the company collaborates with contract manufacturing organizations (CMOs) to enhance and scale up production as necessary.
- Patient Advocacy: uniQure places a strong emphasis on working with patient advocacy groups to promote awareness around genetic diseases. Their commitment ensures that patients have access to the necessary therapies and support services, enhancing the overall patient experience.
- Professional Education: To guarantee the safe and effective administration of gene therapies, uniQure provides education and training resources for healthcare professionals. This service aims to equip practitioners with the knowledge and skills needed to deliver these advanced therapies, facilitating better patient management.
Conclusion
In summary, uniQure N.V. stands out as a leader in the field of gene therapy, providing a diverse range of segments, innovative products, and comprehensive services focused on treating rare genetic diseases. The companys efforts in developing therapies for conditions like hemophilia B, hemophilia A, Huntingtons disease, and Parkinsons disease illustrate its commitment to transforming patient care and enhancing quality of life through groundbreaking gene therapies. By integrating robust research and development, advanced manufacturing capabilities, patient advocacy, and healthcare professional training, uniQure is positioned to make a significant impact in the field of genetic disease treatment.