Articles from Orchard Therapeutics (Europe) Limited

Completion of enrollment achieved nearly one year ahead of schedule

LENMELDY™ is the first and only disease-modifying intravenous infusion proven to extend life expectancy in pre-symptomatic late infantile (PSLI) patients and mitigate the cognitive and/or physical impact of early-onset MLD

First U.S. patients being treated with Lenmeldy™ (atidarsagene autotemcel) in a commercial setting; other launch efforts progressing well following Food and Drug Administration approval last year

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic function in a variety of tissues and organs, including the CNS

Ten presentations at ESGCT 2024 detail the therapeutic potential and applicability of the company’s platform in rare neurometabolic diseases and beyond

ISTANBUL and LONDON, and BOSTON, Oct. 07, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe and Er-Kim, an international pharmaceutical company specializing in the commercialization of novel therapies, today announced the companies have signed an exclusive distribution agreement to commercialize Libmeldy® (atidarsagene autotemcel), the only approved therapy for eligible children with early-onset metachromatic leukodystrophy (MLD).

LONDON and BOSTON, Aug. 28, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced nine presentations from across its late-stage neurometabolic hematopoietic stem cell (HSC) gene therapy portfolio will be featured at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium taking place September 3-6 in Porto, Portugal.

A nomination to add MLD to the U.S. Recommended Uniform Screening Panel has been submitted by a multi-disciplinary expert working group

Eight presentations showcase the broad applicability of HSC gene therapy to address rare neurometabolic diseases and beyond

TOKYO, LONDON and BOSTON, May 03, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced that Bobby Gaspar, M.D., Ph.D., co-founder and chief executive officer, has been named to the inaugural 2024 TIME100 Health, an annual list recognizing the impact, innovation, and achievement of the world’s most influential individuals in health this year. The full list and related tributes will appear in the May 13, 2024 issue of TIME and is available online at https://time.com/collection/time100-health/.

Five specialized treatment centers being qualified across the U.S. to minimize travel burden on eligible patients and their families

One-time treatment with Lenmeldy has shown the potential to restore enzymatic function to stop or slow disease progression, with up to 12 years of follow-up (median 6.76 years)

Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient’s own hematopoietic stem cells

Three oral presentations and seven posters highlight transformative potential of HSC gene therapy and newborn screening to address severe neurometabolic diseases

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD). The agreement covers several member countries, including Belgium, The Netherlands, and Ireland. It follows similar arrangements made with reimbursement authorities in the United Kingdom (UK), Italy, Germany, Sweden, Iceland, Finland and Norway, and is a key component of the company’s geographic expansion plans.

Acquisition enriches Kyowa Kirin’s portfolio, enables the development of numerous promising candidates with a clinically differentiated platform, and helps to resource ongoing and future launches of Libmeldy® (atidarsagene autotemcel) in early-onset MLD

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).

Previously reported results from ongoing proof-of-concept study showed extensive metabolic correction, continued cognitive, motor, and physical development, as well as early improvements in skeletal health

Reported $5.6 million in quarterly Libmeldy net revenue and $12.7 million year-to-date

New clinical data beyond previously reported neurological and skeletal results demonstrate favorable outcomes for multiple disease manifestations not effectively addressed by the current standard of care

Eight presentations highlight the differentiated profile and broad applicability of the company’s HSC gene therapy platform

PDUFA date set for March 18, 2024

Administration of one-time gene therapy resulted in statistically significant improvement in severe motor impairment-free survival with up to 12 years of follow-up (median 6.76 years)

Reported $6.6M in Q2’23 Libmeldy net sales, representing the highest quarter to date

BOSTON and LONDON, Aug. 02, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, expresses its strong appreciation for the passage of the Newborn Metabolic Screening Act in Illinois, also known as SB67. The law, anticipated to go into effect in January 2024, mandates the expansion of the Illinois Department of Public Health’s Newborn Screening program to include metachromatic leukodystrophy (MLD) for the estimated 130,000 newborns in the state each year. Illinois becomes the first state in the U.S. to add newborn screening for MLD to its statewide panel.

BOSTON and LONDON, July 25, 2023 (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, August 3, 2023, at 8:00 a.m. EDT to review business updates and its second quarter 2023 financial results.

Funding from second closing extends cash runway to mid-2025 to advance the company’s HSC gene therapy portfolio through several potential value-creating milestones

BOSTON and LONDON, May 24, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will make a virtual presentation at Stifel’s Inaugural Genetic Medicines Day on Tuesday, May 30, 2023 at 5:00 p.m. EDT.

New OTL-203 proof-of-concept data demonstrate extensive metabolic correction in the skeletal system of patients with MPS-IH including normal growth rates, improvement in joint function and progressive acquisition of motor skills

Preclinical proof-of-concept data show the therapeutic potential of OTL-104 for NOD2 Crohn’s disease, a severe and treatment-refractory form of the disease

OTL-200 Biologics License Application (BLA) submission completion anticipated in mid-2023

Six presentations showcase the differentiated profile and broad applicability of the company’s HSC gene therapy platform

BOSTON and LONDON, March 30, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will make a virtual presentation at the Guggenheim Genomic Medicines and Rare Disease Day on Tuesday, April 4, 2023 at 1:35pm ET.

BOSTON and LONDON, March 10, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Company’s previously disclosed change to its American Depositary Share ("ADS") to ordinary share ratio has been made effective. The ratio has changed from one ADS to one ordinary share to the new ratio of one ADS to ten ordinary shares.

OTL-200 pre-BLA meeting granted for second quarter of 2023 following productive clinical Type B meeting with the U.S. Food and Drug Administration (FDA)

Proceeds to accelerate commercialization of Libmeldy and advance the company’s HSC gene therapy R&D portfolio

BOSTON and LONDON, March 03, 2023 (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:

BOSTON and LONDON, Feb. 27, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced it has reached an agreement with the New Therapies (NT) Council which will result in reimbursed access to Libmeldy® (atidarsagene autotemcel) for all metachromatic leukodystrophy (MLD) patients who fall within the scope of the European marketing authorization in Sweden. Libmeldy received the strongest possible recommendation for use by the NT Council following the completion of a health economic evaluation by FINOSE, a health technology assessment consortium between Finland, Norway and Sweden.

Administration of one-time gene therapy resulted in statistically significant improvement in severe motor impairment-free survival with up to 11 years of follow-up (median 6.15 years)

BOSTON and LONDON, Feb. 10, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that it plans to change the ratio of its American Depositary Shares ("ADSs") to its ordinary shares (the "ADS Ratio"), nominal value £0.10 per share, from the current ADS Ratio of one (1) ADS to one (1) ordinary share to a new ADS Ratio of one (1) ADS to ten (10) ordinary shares (the “ADS Ratio Change”). The Company anticipates that the ADS Ratio Change will be effective on or about March 10, 2023 ( the “Effective Date”).

Four oral presentations and 11 posters highlight transformative potential of HSC gene therapy and newborn screening to address severe neurometabolic diseases

Libmeldy® revenue totaled $5.8M in Q4 2022 and $18.8M for the full year with commercial expansion activities continuing in Europe

Global registrational trial expected to commence in the second half of 2023

All patients achieved sustained engraftment and supraphysiological SGSH enzyme levels with median 1.5 years follow-up

BOSTON and LONDON, Dec. 01, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced its marketing authorization application (MAA) for Libmeldy® (atidarsagene autotemcel) has been accepted for evaluation by the Swiss Agency for Therapeutic Products (Swissmedic) for the potential treatment of eligible patients with early-onset metachromatic leukodystrophy (MLD). The Swiss filing was based on the European Union (EU) MAA for Libmeldy, which was approved by the European Commission in December 2020. Libmeldy is registered in the EU, Iceland, Liechtenstein, Norway and the United Kingdom (UK). The company expects to receive Swissmedic’s assessment report in the first half of 2023.

Libmeldy® product sales totaled $4.8M in Q3 and $13.0M YTD with market expansion activities continuing in Europe

BOSTON and LONDON, Nov. 08, 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:

BOSTON and LONDON, Nov. 07, 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 Monday, November 14, 2022, at 8:00 a.m. EST to review business updates and its third quarter 2022 financial results.

Company to host KOL conference call and webcast on Monday, December 12 at 5:00 p.m. EST

Clinical and pre-clinical data from across the company’s portfolio will be featured in four oral and six poster presentations