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About Orchard Therapeutics
Orchard Therapeutics is a leading global fully integrated commercial-stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies.
Orchard’s portfolio of ex vivo autologous gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first ex vivo autologous gene therapy approved by the EMA in 2016, three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline.
The company is partnered with world-leading institutions in gene therapy, 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.
Orchard is a publicly traded company (NASDAQ: ORTX) with offices in the UK and the US, including London and Boston.
128 articles with Orchard Therapeutics
The Rare Disease Company Coalition’s 10 founding members have brought 22 treatments to market and currently have more than 160 rare disease programs in the works.
Orchard Therapeutics Reports First Quarter 2021 Financial Results and Provides Recent Business Updates
Commercial Activities Advancing for Upcoming Launch of LibmeldyTM (atidarsagene autotemcel) for Eligible Patients with Early-onset MLD in Germany
Orchard Therapeutics Announces New England Journal of Medicine Publication of HSC Gene Therapy Data for ADA-SCID
100% overall survival and ≥95% event-free survival observed at two and three years following one-time treatment withlentiviral HSC gene therapy
Orchard Therapeutics Announces Multiple Presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy
Seven abstracts accepted show the potential of HSC gene therapy to transform the treatment of multiple severe rare disorders
OTL-203 Achieves Proof of Concept (POC) in MPS-I Hurler Syndrome (MPS-IH); Registrational Trial Planned to Initiate by Year End 2021
- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that members of the management team will present at the following virtual investor conferences: Cowen 41 st Annual Healthcare Conference on March 4, 2021, at 10:30 AM ET as part of a panel discussion focused on disorders of the central nervous system
2/15/2021It was a busy week for clinical trial announcements and news. Here’s a look.
Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH)
All eight patients treated with OTL-203 show stable cognitive function, motor function and growth within the normal range at multiple data points post-treatment Supportive initial OTL-201 biomarker data for mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo syndrome type A) in three patients Data in multiple posters highlight ongoing work in patient identification and access to treatment for metachromatic leukodystrophy (MLD) IR webinar today at 4:30 p.m. ET with Dr. Simon Jones
Orchard Therapeutics Announces $150 Million Strategic Financing Strengthened Financial Position Supports Execution into the First Half of 2023
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that it has executed a securities purchase agreement to raise gross proceeds of $150 million resulting from the sale of 24,115,755 shares through a private investment in public equity (PIPE) financing at a price of $6.22 per share.
Orchard Therapeutics Appoints Braden Parker as Chief Commercial OfficerCommercial Launch of Libmeldy™ in Europe On-Track for 1H 2021
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the appointment of Braden Parker to the role of Chief Commercial Officer (CCO). In this capacity, Mr. Parker will oversee all aspects of commercial strategy, planning and operations for the company.
Orchard Therapeutics today outlined nine upcoming presentations from its neurodegenerative portfolio to be featured at the 17th Annual WORLDSymposium™ being held on February 8-12, 2021
Orchard Therapeutics Secures Partnerships to Broaden Access to LibmeldyTM for Eligible Patients in Middle East & Turkey
Orchard Therapeutics, a global gene therapy leader, announced plans to extend the company’s commercial reach in the Middle East and Turkey through exclusive agreements with GenPharm Services and GEN, two leading regional specialty pharmaceutical companies with extensive experience in rare genetic disease.
Orchard Therapeutics Announces OTL-200 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation by FDA for the Treatment of Metachromatic Leukodystrophy (MLD)
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200,
Orchard Therapeutics Announces 2021 Corporate Priorities Supporting the Build-out of its Commercial Business in Hematopoietic Stem Cell (HSC) Gene Therapy and Expansion of its Clinical Applications
Preparations on Track for First Half 2021 Commercial Launch of Libmeldy™ (OTL-200), the First Approved Product for Metachromatic Leukodystrophy (MLD) in the EU
Orchard Therapeutics, a global gene therapy leader, announced that the company is scheduled to present at the 39th Annual J.P. Morgan Healthcare Conference being held virtually on Wednesday January 13, 2021 at 9:10 AM ET.
Orchard Therapeutics Reports OTL-201 Initial Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA)Preliminary results from first patient treated with OTL-201
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, yesterday reported initial data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-201, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy being studied for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A). The data were presented as part of an oral pres
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that its chief executive officer, Bobby Gaspar, M.D., Ph.D. will participate in a pre-recorded fireside chat as part of the Piper Sandler 32nd Annual Virtual Healthcare Conference. Management will also be available for one-on-one meetings on Tuesday, December 1, 2020. The fireside chat is available for on-demand viewing under "Events" in the Investors &
Orchard Therapeutics Announces FDA Clearance of IND Application for OTL-200 for Metachromatic Leukodystrophy (MLD)
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for OTL-200, an autologous, hematopoietic stem cell, lentiviral vector-based gene therapy in development for the treatment of metachromatic leukodystrophy (MLD). The company also has applied for Regenerative Medicine Advanced Therapy (RMAT) des
Orchard Laboratories, headquartered in West Bloomfield, Mich. (7901 Orchard Lake Road, Suite 100), is the first private laboratory in metro Detroit to operate the new, TaqPath Multiplex Kit to test for SARS-CoV-2, Influenza A and Influenza B.
Orchard Therapeutics Unveils Details on New HSC Gene Therapy Research Programs as Part of R&D Investor Event Tomorrow at 9:00 a.m. ET
First look at preclinical data infrontotemporal dementia with progranulin mutations (GRN-FTD)and new amyotrophic lateral sclerosis (ALS) program