Orchard Therapeutics NA
Orchard Therapeutics is dedicated to transforming the lives of people with severe and rare genetic diseases through innovative hematopoietic stem cell (HSC) gene therapy. With a history rooted in pioneering research and clinical development, the company focuses on developing potentially curative therapies, expanding their pipeline to include less rare conditions with high unmet medical needs, and improving manufacturing processes to ensure global accessibility. Their mission is driven by a community of families, patients, and dedicated scientists, aiming to bring transformative treatments to patients worldwide.
Industries
Nr. of Employees
medium (51-250)
Orchard Therapeutics NA
Products
Libmeldy
A gene therapy approved in the EU for treating Metachromatic Leukodystrophy (MLD), designed to correct the underlying genetic cause with a one-time treatment.
Strimvelis
The first ex vivo autologous gene therapy approved by the EMA for ADA-SCID, using the patient's own genetically modified stem cells.
HSC Gene Therapy Platform
Orchard's technology platform that modifies a patient's own hematopoietic stem cells to treat genetic diseases.
Clinical Trials
Orchard conducts clinical trials for investigational gene therapies targeting several genetic and severe diseases.
Investigator-initiated Research
Support for external research on diagnosis, natural history, and other topics relevant to Orchard's indications.
Independent Medical Education
Funding and support for medical education on gene therapy and related therapeutic areas.
Libmeldy
A gene therapy approved in the EU for treating Metachromatic Leukodystrophy (MLD), designed to correct the underlying genetic cause with a one-time treatment.
Strimvelis
The first ex vivo autologous gene therapy approved by the EMA for ADA-SCID, using the patient's own genetically modified stem cells.
HSC Gene Therapy Platform
Orchard's technology platform that modifies a patient's own hematopoietic stem cells to treat genetic diseases.
Clinical Trials
Orchard conducts clinical trials for investigational gene therapies targeting several genetic and severe diseases.
Investigator-initiated Research
Support for external research on diagnosis, natural history, and other topics relevant to Orchard's indications.
Independent Medical Education
Funding and support for medical education on gene therapy and related therapeutic areas.
Services
Clinical development and trial operations
Protocol design, patient identification and trial execution support for gene therapy clinical programs.
Viral vector manufacturing and process development
Process development for lentiviral vector production including use of stable producer cell lines and scale‑up strategies.
Automated cell processing and closed‑system solutions
Implementation and evaluation of automated closed manufacturing workflows for cell handling to increase consistency and scalability.
Patient engagement and education
Development of educational materials and collaboration with advocacy groups to support patient education, trial recruitment and access planning.
Regulatory and access planning
Regulatory filing support and participation in multi‑stakeholder initiatives for reimbursement and health‑system readiness for one‑time gene therapies.
Clinical development and trial operations
Protocol design, patient identification and trial execution support for gene therapy clinical programs.
Viral vector manufacturing and process development
Process development for lentiviral vector production including use of stable producer cell lines and scale‑up strategies.
Automated cell processing and closed‑system solutions
Implementation and evaluation of automated closed manufacturing workflows for cell handling to increase consistency and scalability.
Patient engagement and education
Development of educational materials and collaboration with advocacy groups to support patient education, trial recruitment and access planning.
Regulatory and access planning
Regulatory filing support and participation in multi‑stakeholder initiatives for reimbursement and health‑system readiness for one‑time gene therapies.
Expertise Areas
- Hematopoietic stem cell (HSC) gene therapy development
- Lentiviral vector process development
- Clinical trial management for rare disease gene therapies
- Viral vector manufacturing scale‑up
Key Technologies
- Ex vivo HSC gene therapy
- Integrating lentiviral vectors
- Stable producer cell lines for viral vector production
- Transduction enhancers