Last update 01 Nov 2024
Orchard Therapeutics Plc

Orchard Therapeutics Plc

Public Company | Subsidiary Company|
2015|
United Kingdom
|
100-250
| NASDAQ: ORTX|

Overview

Basic Info

Introduction
Orchard Therapeutics is a commercial-stage company that specializes in developing innovative gene therapies to improve the lives of patients with rare diseases. With a portfolio of autologous ex vivo gene therapy programs, the company has demonstrated sustained clinical benefits in over 150 patients across five disease areas. These programs include Strimvelis, the first autologous ex vivo gene therapy approved by the EMA in 2016, as well as advanced registrational studies in MLD, WAS, and ADA-SCID. Orchard Therapeutics also has two clinical programs in X-CGD and beta-thalassemia, and an extensive preclinical pipeline. The company collaborates with top institutions in gene therapy research, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele.

Tags

Other Diseases
Congenital Disorders
Endocrinology and Metabolic Disease
Gene therapy
Hematopoietic stem cell therapy
Stem cell therapy

Disease domain score
A glimpse into the focused therapeutic areas
Technology Platform
Most used technologies in drug development
Targets
Most frequently developed targets
Top 5 Drug TypeCount
Gene therapy6
Hematopoietic stem cell therapy6
Stem cell therapy1

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Pipeline

Pipeline Snapshot as of 19 Nov 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3
Preclinical
3
1
Phase 2 Clinical
Phase 3 Clinical
1
1
Approved
Other
7
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Profit

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