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.

First Primary Outcome Measure Met with All Eight Patients Achieving Hematologic Engraftment

Orchard Therapeutics Presents New Interim Data from OTL-203 Proof-of-concept Study for MPS-I

Orchard Therapeutics: A Leading Gene Therapy Company For Your Watchlist

Orchard Therapeutics' (ORTX) CEO Bobby Gaspar on Q1 2020 Results - Earnings Call Transcript

Shares of Orchard Therapeutics (NASDAQ:ORTX) fell 1.5% after the company reported Q1 results.

Orchard Therapeutics: Q1 Earnings Insights

Orchard Therapeutics: Updating The Investment Thesis

BOSTON and LONDON, April  27, 2020  (GLOBE NEWSWIRE) --  Orchard Therapeutics  (Nasdaq: ORTX), a global gene therapy leader, today announced that the first patient has been dosed in an open-label, proof-of-concept investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). The study is designed to evaluate safety, tolerability and clinical efficacy and is intended to enroll up to five patients between three months and 24 months of age who will be followed for three years. The study also contains a number of key secondary outcome measures such as overall survival, cognition and behavior to help inform future clinical development of HSC gene therapy in this indication.

Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome (MPS-IIIA)

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The COVID-19 pandemic has changed normal operations for companies across the world as they cope with resource limitations, health precautions and other challenges.

What Wedbush Is Watching in Biotechs Amid Coronavirus Crisis

BOSTON and LONDON, March  31, 2020  (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global leader in gene therapy, today provided an update on its business and operations, outlining how the COVID-19 pandemic has and may continue to impact the company’s development programs and timelines, product supply, commercial operations and financial position. Orchard has taken important steps to help ensure the safety of employees and their families and reduce spread of the virus in the communities where the company operates.

Orchard Therapeutics Outlines Business Impact of COVID-19

-- Mark Rothera steps down as President and CEO --    -- Frank Thomas takes on additional responsibilities as President and Chief Operating Officer --

Orchard Therapeutics Appoints Company Founder and Gene Therapy Pioneer Bobby Gaspar, M.D., Ph.D., as New Chief Executive Officer

Orchard Therapeutics PLC (ORTX) CEO Mark Rothera on Q4 2019 Results - Earnings Call Transcript

BOSTON and LONDON, Feb.  03, 2020  (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the appointment of Steven Altschuler, M.D., to its board of directors. Dr. Altschuler brings nearly 20 years of experience building and leading world-class, integrated, translational, value-based health care systems and transformational biotechnology companies. He currently serves as managing director of Healthcare Ventures at Ziff Capital Partners and was previously chair of the board of directors at Spark Therapeutics (now part of Roche) for six years, from its founding until it was acquired in 2019.

Orchard Therapeutics Appoints Industry Veteran Steven Altschuler, M.D. to its Board of Directors

Early Clinical Data Support ex vivo Hematopoietic Stem Cell Gene Therapy as a Potentially Promising Treatment Option for X-CGD Early Clinical Data Support ex vivo Hematopoietic Stem Cell Gene Therapy as a Potentially Promising Treatment Option for X-CGD

Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (X-CGD)

Application Being Evaluated Under Accelerated Assessment Application Being Evaluated Under Accelerated Assessment

Orchard Therapeutics Announces the Filing and Validation of Marketing Authorization Application by European Medicines Agency for OTL-200 for the Treatment of Metachromatic Leukodystrophy

BOSTON and LONDON, Nov.  18, 2019  (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted an accelerated assessment for OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

European Medicines Agency Grants Orchard Therapeutics Accelerated Assessment of OTL-200 for Patients with Metachromatic Leukodystrophy

Orchard Therapeutics' (ORTX) CEO on Q3 2019 Results - Earnings Call Transcript

Registrational Trial for Wiskott-Aldrich Syndrome Met Key Primary and Secondary Endpoints at Three Years; Data from Integrated Analysis Reinforce Treatment Benefits of Gene Therapy and Durability of Effect in Additional Patients

Orchard Therapeutics to Present New Registrational Data of Investigational Gene Therapies at the 61st American Society of Hematology Annual Meeting

Stable Levels of Engraftment and Reconstitution of Enzyme Activity  in Patients with Up to 12 Months of Follow-up Indicate Similar Profiles Between Cryopreserved and Fresh  Formulations

Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation

BOSTON and LONDON, Oct.  02, 2019  (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that the company’s president and chief executive officer, Mark Rothera, has been appointed to The Alliance for Regenerative Medicine’s (ARM) 2020 Board of Directors. In this role and in tandem with the executive committee and board of directors, Mark will oversee the formation and execution of ARM’s strategic priorities and focus areas over the coming year.

Orchard Therapeutics Announces Appointment of CEO Mark Rothera to The Alliance for Regenerative Medicine’s 2020 Board of Directors

Initial Results from Cryopreserved Formulation Clinical Trial Accepted as Poster Presentation Initial Results from Cryopreserved Formulation Clinical Trial Accepted as Poster Presentation

Orchard Therapeutics Announces Presentation of New Clinical Data for OTL-200 for Metachromatic Leukodystrophy at Upcoming European Society of Gene & Cell Therapy Annual Congress

BOSTON and LONDON, Sept.  25, 2019  (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that president and chief executive officer, Mark Rothera, will present at the annual Cell & Gene Meeting on the Mesa at 5:00pm PT on October 2, 2019 in Carlsbad, California.

Orchard Therapeutics to Present at the 2019 Cell & Gene Meeting on the Mesa

BOSTON and LONDON, Sept.  13, 2019  (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that chief commercial officer Jason Meyenburg is leaving the company effective today to take on a chief executive role in the industry. In the interim Mark Rothera, the company’s president and chief executive officer, will assume global commercial leadership responsibilities. Mr. Rothera has driven the transition of multiple emerging biotechnology companies from R&D stage to commercialization, with a special focus on bringing novel therapies to market for patients with rare diseases, having launched seven orphan drugs over the course of his 30-year career in the biopharmaceutical industry. Orchard has initiated a global search for Mr. Meyenburg’s permanent replacement.

Orchard Therapeutics Announces Departure of Chief Commercial Officer

Six of Eight Patients Treated to Date with Follow-up Out to 12 Months in First Patient to Receive Treatment

Orchard Therapeutics Announces Encouraging Update from Proof-of-Concept Study of OTL-203 for the Treatment of Mucopolysaccharidosis Type I (MPS-I)

Further Demonstration that OTL-200 Provides Meaningful Clinical Benefit on Cognitive and Motor Function

Orchard Therapeutics Presents an Integrated Data Analysis Demonstrating Sustained Clinical Benefit of OTL-200 for the Treatment of Metachromatic Leukodystrophy

BOSTON and LONDON, Aug.  30, 2019  (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced the appointment of John Curnutte, M.D., Ph.D., to its board of directors. Dr. Curnutte brings more than 26 years of research and development expertise in both the biotechnology and pharmaceutical sectors.

Orchard Therapeutics Further Strengthens Board of Directors with Appointment of John Curnutte, M.D., Ph.D.

Mucopolysaccharidosis Type I and Metachromatic Leukodystrophy Abstracts Accepted for Oral Presentations Mucopolysaccharidosis Type I and Metachromatic Leukodystrophy Abstracts Accepted for Oral Presentations

Orchard Therapeutics Announces Presentation of Data from Neurometabolic Portfolio at Upcoming Society for the Study of Inborn Errors of Metabolism (SSIEM) 2019 Annual Symposium

News for Orchard Therapeutics plc - ADR Stock (ORTX)