Orchard Therapeutics plc - ADR

Orchard Therapeutics Plc is a biopharmaceutical company, which engages in discovering, acquiring, developing and commercializing gene therapies for patients with rare disorders. The company focuses on its autologous ex vivo gene therapy approach on three therapeutic rare disease franchise areas: primary immune deficiencies, neurometabolic disorders, and hemoglobinopathies. Its portfolio includes Strimvelis, a commercial-stage gamma retroviral-based product for the treatment of Adenosine deaminase deficiency (ADA-SCID). The company was founded by Nicolas Koebel and Andrea Spezzi in August 2018 and is headquartered in London, the United Kingdom.

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Orchard Therapeutics is a fully integrated commercial-stage company dedicated to treating the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical benefit 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, 3 programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott-Aldrich syndrome), and ADA-SCID (adenosine deaminase severe combined immunodeficiency), 2 other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, 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.

BOSTON and LONDON, May 10, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today outlined seven presentations from across its platform to be featured at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting taking place May 16-19 in Washington, D.C. Featured presentations include updated results on the OTL-203 clinical program for mucopolysaccharidosis type I Hurler syndrome (MPS-IH), as well as several accepted abstracts highlighting preclinical work demonstrating the applicability of HSC gene therapy to potentially address other neurodegenerative and CNS-related conditions, including frontotemporal dementia (FTD).

Orchard Therapeutics Announces Presentations at ASGCT 2022 Showcasing Potential of HSC Gene Therapy in Neurodegenerative Disorders and Beyond

BOSTON and LONDON, May 05, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will host a conference call and live webcast on Thursday, May 12, 2022, at 8:00 a.m. ET to review business updates and its first quarter 2022 financial results.

Orchard Therapeutics to Webcast Conference Call of First Quarter 2022 Financial Results

BOSTON and LONDON, May 02, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced it has reached an agreement with Gesetzliche Krankenversicherung Spitzenverband (GKV-SV) which will result in reimbursed access to Libmeldy® (atidarsagene autotemcel) for all metachromatic leukodystrophy (MLD) patients in Germany who fall within the scope of the European marketing authorization.

Orchard Therapeutics Announces Agreement Enabling Access and Reimbursement for Libmeldy for All Eligible MLD Patients in Germany

BOSTON and LONDON, April 11, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced it has reached another historic reimbursement agreement with the Italian Medicines Agency, also known as Agenzia Italiana del Farmaco (AIFA), to enable access to Libmeldy® (atidarsagene autotemcel) for all children with metachromatic leukodystrophy (MLD) who fall within the scope of the European marketing authorization. The news marks the second reimbursement agreement to cover all eligible MLD patients that Orchard has reached with a European market this year.

Orchard Therapeutics Announces Reimbursement Agreement Making Libmeldy Available for All Eligible MLD Patients in Italy

Orchard Therapeutics' (ORTX) CEO Bobby Gaspar on Q4 2021 Results - Earnings Call Transcript

Libmeldy European launch momentum building with multiple MLD patients treated and strong recognition of value proposition; U.S. BLA filing of OTL-200 on track for late 2022 / early 2023

Orchard Therapeutics Extends Runway into 2024, Focusing HSC Gene Therapy Platform Exclusively on Severe Neurometabolic Diseases and Research Platform

BOSTON and LONDON, March 24, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will host a conference call and live webcast on Wednesday, March 30, 2022, at 8:00 a.m. ET to review business updates and its fourth quarter and fiscal year 2021 financial results.

Orchard Therapeutics to Webcast Conference Call of Fourth Quarter and Fiscal Year 2021 Financial Results

BOSTON and LONDON, March 03, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that members of the management team will present at the following investor conferences:

Orchard Therapeutics to Present at Multiple Investor Conferences in March 2022

BOSTON and LONDON, Feb. 07, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today outlined five upcoming presentations from across its neurodegenerative portfolio to be featured at the 18th Annual WORLDSymposium™ being held on February 7-11, 2022.

Orchard Therapeutics Announces Multiple Presentations at WORLDSymposium™ 2022

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First-ever lentiviral hematopoietic stem cell gene therapy approved for reimbursement by NHS England

Orchard Therapeutics Announces Historic Agreement Making Libmeldy Available by NHS England for MLD Patients

Administration of one-time hematopoietic stem cell (HSC) gene therapy resulted in sustained, clinically meaningful benefits by preserving cognitive function and motor development in most patients

Orchard Therapeutics Announces Publication in The Lancet of Long-term Clinical Outcomes with Libmeldy for the Treatment of Children with Early-onset MLD

Launch Momentum Building for Libmeldy® in Europe with Health Technology Assessments Progressing Favorably and Treatment Underway for Multiple Commercial Patients

Orchard Therapeutics Announces Recent Commercial and Regulatory Progress for Late-stage HSC Gene Therapy Programs and Outlines Key 2022 Milestones

BOSTON and LONDON, Jan. 04, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company is scheduled to present at the virtual 40th Annual J.P. Morgan Healthcare Conference on Thursday January 13, 2022, at 9:00 a.m. ET.

Orchard Therapeutics to Present at the 40th Annual J.P. Morgan Healthcare Conference

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100 percent overall survival with median follow-up of two years post-treatment with HSC gene therapy

Orchard Therapeutics Announces New England Journal of Medicine Publication of Interim Proof-of-concept Study Results of OTL-203 for Hurler Syndrome

BOSTON and LONDON, Nov. 08, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that members of the management team will present and be available for one-on-one meetings at the following virtual investor conferences:

Orchard Therapeutics to Present at Virtual Investor Conferences in November 2021

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent Business Updates

Nine accepted abstracts demonstrate broad potential of the company's HSC gene therapy approach to treat severe neurodegenerative diseases and immunological disorders Nine accepted abstracts demonstrate broad potential of the company's HSC gene therapy approach to treat severe neurodegenerative diseases and immunological disorders

Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress

Novel Discovery Research in HSC-generated Antigen-specific Regulatory T Cells Disclosed

Orchard Therapeutics Outlines Differentiated Profile of Its HSC Gene Therapy Approach and Discusses Potential Future Applications at Virtual R&D Investor Event

Gene therapy experts Fulvio Mavilio, Ph.D., and Nicoletta Loggia, Ph.D., join as chief scientific officer and chief technical officer, respectively

Orchard Therapeutics Bolsters R&D and Technical Operations Leadership with New Executive Appointments

BOSTON and LONDON, Sept. 08, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will webcast a virtual R&D Investor Event on Tuesday, September 14, 2021, from 7:00 a.m. to 8:00 a.m. ET.

Orchard Therapeutics to Host Virtual R&D Investor Event on Tuesday, September 14, 2021

BOSTON and LONDON, Sept. 07, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Compensation Committee of Orchard's Board of Directors approved the grant of non-statutory stock options to four new employees to purchase an aggregate of 278,500 ordinary shares under Orchard's 2020 Inducement Equity Plan. The awards were granted as an inducement material to the employees' acceptance of employment with Orchard in accordance with Nasdaq Listing Rule 5635(c)(4).

Orchard Therapeutics Announces Grant of Inducement Awards Under Nasdaq Listing Rule 5635(c)(4)

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ORTX is a developer of autologous gene therapies for rare diseases.

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