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.

Regulatory feedback obtained on OTL-200 (MLD) and OTL-203 (MPS-IH) programs

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

Orchard Therapeutics plc (NASDAQ: ORTX) and Pharming Group NV (NASDAQ: PHAR) have collaborated to research, develop, manufacture and commercialize OTL-105 for hereditary angioedema (HAE). OTL-105 is an investigational autologous hematopoietic stem cell (HSC) gene therapy designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks.

Orchard Therapeutics, Pharming Ink Development Pact For Gene Therapy

Highlights the broader potential of Orchard's ex vivo HSC gene therapy platform approach in new and larger indications

Orchard Therapeutics and Pharming Group Announce Collaboration to Develop and Commercialize ex vivo autologous HSC Gene Therapy for Hereditary Angioedema

Orchard Therapeutics plc (NASDAQ: ORTX) has provided updates on the progress of its lead gene therapy programs targeting metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I Hurler syndrome (MPS-IH), and Wiskott-Aldrich syndrome (WAS). After receiving feedback from the FDA, the company expects to file a marketing application for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023.

Orchard Therapeutics Issues Updates On Gene Therapy Programs

Expected clinical package for OTL-200 BLA for MLD using data from existing patients clarified with FDA

Orchard Therapeutics Announces Regulatory and Clinical Updates for Lead Gene Therapy Programs

Commercial Activities Advancing for Upcoming Launch of Libmeldy TM (atidarsagene autotemcel) for Eligible Patients with Early-onset MLD in Germany

Orchard Therapeutics Reports First Quarter 2021 Financial Results and Provides Recent Business Updates

Three years after receiving gene therapy from Orchard Therapeutics (NASDAQ: ORTX) in an early-stage clinical trial, 100% of patients with an inherited disease that damages the immune system were still alive. Data from a study that used Orchard's gene therapy OTL-101 to treat adenosine deaminase severe combined immunodeficiency (ADA-SCID) were published in the New England Journal of Medicine.

Orchard Therapeutics Posts Long-Term Data For Gene Therapy In Inherited Immune System Disorder

100% overall survival and ≥ 95% event-free survival observed at two and three years following one-time treatment with lentiviral HSC gene therapy

Orchard Therapeutics Announces New England Journal of Medicine Publication of HSC Gene Therapy Data for ADA-SCID

Seven abstracts accepted show the potential of HSC gene therapy to transform the treatment of multiple severe rare disorders

Orchard Therapeutics Announces Multiple Presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy

Orchard Therapeutics is the category leader for engineering and developing hematopoietic stem cell medicines. The company's platform has been validated with 2 approvals in Europe and a diverse pipeline of drug candidates.

Orchard Therapeutics: Curative Medicines For Immune-Related Diseases

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Regenerative Medicine Attracted Record $20 Billion in Funding in 2020

Orchard Therapeutics: Slow And Steady, But Lacking Major Near-Term Catalysts

MINNEAPOLIS--(BUSINESS WIRE)--Be The Match BioTherapies announced an expansion of their multi-year partnership with Orchard Therapeutics.

Be The Match BioTherapies® Announces Expansion of Multi-Year Strategic Alliance with Orchard Therapeutics to Support European Commercial Launch of Libmeldy™

Shares of Orchard Therapeutics (NASDAQ:ORTX) remained unaffected after the company reported Q4 results. Quarterly Results Earnings per share were up 26.09% year over year to ($0.34), which beat the estimate of ($0.35).

Recap: Orchard Therapeutics Q4 Earnings

$150M Strategic Financing Supports Execution into the First Half of 2023

Orchard Therapeutics Reports 2020 Financial Results and Reviews Recent Accomplishments

BOSTON and LONDON, Feb. 25, 2021 (GLOBE NEWSWIRE) -- 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:

Orchard Therapeutics to Present at Multiple Investor Conferences in March 2021

Orchard Therapeutics (ORTX) will provide updates on earnings, revenues of Strimvelis and the pipeline when it releases results for fourth-quarter 2020.

What's in Store for Orchard Therapeutics (ORTX) Q4 Earnings?

The company released some promising data from a pair of proof-of-concept studies.

Why Orchard Therapeutics Stock Is Jumping Today

Orchard Therapeutics Plc (NASDAQ: ORTX) gains in premarket in reaction to positive interim data from a proof-of-concept study evaluating its gene therapy OTL-203 in Mucopolysaccharidosis type I (MPS-I). All eight patients treated with OTL-203 showed stable cognitive function, motor function, and growth within the normal range at multiple data points post-treatment.

Orchard Therapeutics' MPS-I Gene Therapy Shows Encouraging Action In Early Study, Shares Spike

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

Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH)

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Strengthened Financial Position Supports Execution into the First Half of 2023 Strengthened Financial Position Supports Execution into the First Half of 2023

Orchard Therapeutics Announces $150 Million Strategic Financing

Commercial Launch of Libmeldy™ in Europe On-Track for 1H 2021 Commercial Launch of Libmeldy™ in Europe On-Track for 1H 2021

Orchard Therapeutics Appoints Braden Parker as Chief Commercial Officer

Nine abstracts accepted demonstrating potential of HSC gene therapy to treat multiple neurodegenerative disorders

Orchard Therapeutics Outlines Comprehensive Presence at 2021 WORLDSymposium™

BOSTON and LONDON, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today 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. GenPharm and GEN will collaborate with Orchard to facilitate access to treatment with Libmeldy in European-based qualified treatment centers for eligible patients living in the Middle East and Turkey.

News for Orchard Therapeutics plc - ADR Stock (ORTX)