Newborn screening is the only practical means of diagnosing MLD prior to the onset of symptoms, which is key to achieving optimal outcomes for children and their families with this rapidly progressive disease As of today, 14 states—representing more than 50 percent of U.S. births—have RUSP-aligned legislation intended to expedite the addition of newly approved RUSP conditions to their respective NBS panels Other community-led efforts to enable newborn screening for MLD advancing globally TOKYO, LONDON and B ...

Completion of enrollment achieved nearly one year ahead of schedule Primary analysis anticipated two years post-treatment TOKYO and LONDON and BOSTON, July 07, 2025 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial (NCT0614 ...

Core Insights - Orchard Therapeutics has secured an agreement with the Spanish National Health System (SNS) for reimbursed access to Libmeldy® for eligible children with early-onset metachromatic leukodystrophy (MLD) [1][3] - MLD is an ultra-rare and fatal neurometabolic disease, with severe forms leading to rapid neurological decline and high mortality rates [2][4] - The agreement follows similar reimbursement arrangements in multiple European countries, enhancing treatment access for patients [3] Company Overview - Orchard Therapeutics, a Kyowa Kirin company, specializes in gene therapy aimed at treating severe genetic diseases through hematopoietic stem cell (HSC) gene therapy [8][9] - The company was founded in 2015 and has been pivotal in advancing HSC gene therapy from concept to clinical application [9] Product Information - Libmeldy® (atidarsagene autotemcel) is designed to correct the genetic defect causing MLD by inserting functional copies of the ARSA gene into the patient's own stem cells [5][6] - The treatment requires high-dose chemotherapy prior to the infusion of genetically modified cells, which can potentially halt or slow disease progression with a single administration [6] Market Context - MLD occurs in approximately one in every 100,000 live births, with an estimated two to three new cases annually in Spain, highlighting the rarity of the condition [4] - Early detection and diagnosis are critical for improving patient outcomes, prompting Orchard Therapeutics to support expanded newborn screening initiatives [4]

Core Insights - Orchard Therapeutics, recently acquired by Kyowa Kirin, aims to accelerate the delivery of new gene therapies for rare neurometabolic diseases and beyond [1][8] - The company presented ten data presentations at the ESGCT 2024, showcasing the potential of its hematopoietic stem cell (HSC) gene therapy portfolio [2][3] - A significant study published in the New England Journal of Medicine details the world's first prospective, population-based newborn screening for metachromatic leukodystrophy (MLD), indicating a new diagnostic and treatment paradigm [6][7] Group 1: Data Presentations - Ten presentations, including three oral and seven poster presentations, were featured at the ESGCT 2024, focusing on clinical outcomes from the company's neurometabolic portfolio [2] - Key presentations include topics on HSC gene therapy for conditions such as MLD, Hurler syndrome, and NOD2-deficient Crohn's disease [2][3] - The presentations highlight the potential of HSC gene therapy to deliver therapeutic antibodies across the blood-brain barrier and provide targeted immunotherapy for autoimmune disorders [2][3] Group 2: Research Findings - A Nature manuscript discusses the long-term durability and safety profile of genetically corrected hematopoietic stem and progenitor cells (HSPCs), emphasizing sustained lineage commitment and absence of harmful mutations [4][5] - The study analyzed 53 patients treated with Orchard's HSC gene therapy programs, showing a polyclonal repertoire and no increase in somatic mutations over time [4][5] - The findings support the long-term safety and efficacy of Orchard's HSC gene therapy portfolio [5] Group 3: Newborn Screening Study - The newborn screening study for MLD involved testing dried blood samples from 109,259 newborns, establishing a comprehensive care pathway for early diagnosis and treatment [6][7] - Three newborns were identified as positive for MLD and referred for presymptomatic treatment, showcasing the potential to reduce morbidity and mortality [7] - This study represents a significant advancement in the diagnostic and treatment approach for MLD, potentially transforming clinical practice [6][7]

Core Insights - Orchard Therapeutics has entered into an exclusive distribution agreement with Er-Kim to commercialize Libmeldy® (atidarsagene autotemcel), the only approved therapy for children with early-onset metachromatic leukodystrophy (MLD) [1][3] Company Overview - Orchard Therapeutics, a Kyowa Kirin company, focuses on developing and commercializing gene therapies aimed at treating severe genetic diseases [10][11] - Er-Kim Pharmaceuticals, established in 1981, partners with over 40 global leaders and has a revenue exceeding EUR 200 million, serving over 600 million patients [9] Product Information - Libmeldy is designed to correct the genetic cause of MLD by inserting functional copies of the ARSA gene into a patient's hematopoietic stem cells [6][8] - The therapy requires patients to undergo high-dose chemotherapy prior to treatment to prepare for the infusion of genetically modified cells [7] Market Potential - MLD is an ultra-rare disease affecting approximately one in 100,000 live births, with a higher incidence in Turkish and Eurasian populations [2][5] - The partnership aims to enhance access to Libmeldy in Turkey, Russia, Ukraine, and the Commonwealth of Independent States, addressing a significant unmet medical need in these regions [3][4]

Company Overview - Orchard Therapeutics, recently acquired by Kyowa Kirin, aims to accelerate the delivery of new gene therapies globally [1] - The company focuses on hematopoietic stem cell (HSC) gene therapy to treat genetic and severe diseases [23][24] - Orchard has a pipeline of HSC gene therapies targeting serious diseases with limited treatment options [24] Upcoming Presentations - Orchard will present nine studies from its late-stage neurometabolic HSC gene therapy portfolio at the SSIEM 2024 Annual Symposium in Porto, Portugal, from September 3-6 [1] - Key presentations include a proof-of-concept study on OTL-203 for Hurler syndrome and an encore presentation on atidarsagene autotemcel (Lenmeldy) for metachromatic leukodystrophy (MLD) [2][3] Specific Therapies - OTL-203 is an investigational therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), currently in a randomized clinical trial [22] - Lenmeldy (atidarsagene autotemcel) is the only approved therapy in the U.S. for treating children with pre-symptomatic and early-symptomatic forms of MLD [5][7] Regulatory Designations - OTL-203 has received Rare Pediatric Disease and Fast Track designations from the U.S. FDA, as well as PRIME status from the EMA [22] - Lenmeldy has been approved in Europe as Libmeldy and is recognized by various regulatory agencies [6]

TOKYO, LONDON and BOSTON, May 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, today announced four oral and four poster presentations from across its hematopoietic stem cell (HSC) gene therapy platform will be featured at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place May 7-11, 2024, in Baltimore. Featured data include several accep ...

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 trib ...

Financial Performance - For the nine months ended September 30, 2023, the company reported net losses of $65.0 million compared to $142.8 million for the same period in 2022, with an accumulated deficit of $965.9 million as of September 30, 2023[125]. - Product revenue for Q3 2023 was $5,559 million, an increase of $182 million (3.4%) compared to Q3 2022's $5,377 million[151]. - Collaboration revenue rose to $743 million in Q3 2023 from $400 million in Q3 2022, marking a significant increase of $343 million (85.8%)[151]. - Total revenues for Q3 2023 reached $6,302 million, up $525 million (9.1%) from $5,777 million in Q3 2022[151]. - For the nine months ended September 30, 2023, total revenues were $14,853 million, a decrease of $816 million (5.2%) compared to $15,669 million in the same period of 2022[161]. - Net product revenues for the nine months ended September 30, 2023, decreased by $1,473 million (10.4%) to $12,744 million from $14,217 million in 2022[162]. - Net loss attributable to ordinary shareholders improved to $(35,319) million in Q3 2023, a reduction of $12,246 million (25.7%) from $(47,565) million in Q3 2022[151]. Expenses and Costs - Total costs and operating expenses for the nine months ended September 30, 2023, were $85,278 million, down $25,887 million (23.3%) from $111,165 million in 2022[161]. - Research and development expenses decreased by $3,550 million (19.6%) from $18,103 million in Q3 2022 to $14,553 million in Q3 2023[155]. - Selling, general and administrative expenses slightly increased by $113 million (1%) from $11,496 million in Q3 2022 to $11,609 million in Q3 2023[157]. - Total direct research and development expenses decreased from $32.2 million in 2022 to $23.3 million in 2023, a reduction of $8.8 million or -27%[165]. - Total indirect research and development expenses decreased from $36.1 million in 2022 to $23.9 million in 2023, a reduction of $12.2 million or -34%[166]. - Selling, general and administrative expenses decreased from $38.5 million in 2022 to $33.7 million in 2023, a reduction of $4.8 million or -12%[167]. Cash and Financing - The company had cash, cash equivalents, and marketable securities totaling $125.4 million as of September 30, 2023[125]. - Net cash used in operating activities was $81.2 million in 2023, compared to $63.9 million in 2022[177]. - Net cash used in investing activities was $24.5 million in 2023, primarily due to purchases of marketable securities totaling $146.7 million[180]. - Net cash provided by financing activities was $60.1 million in 2023, mainly from private placement financing of $68.0 million[182]. - The company expects existing cash and marketable securities will be sufficient to fund operations for at least twelve months from the filing date[184]. - The company has borrowed $33.0 million under its credit facility, with a carrying value of term loans at $25.6 million and a variable interest rate of 5.95% plus SOFR[191]. Strategic Developments - The company entered into a Transaction Agreement with Kyowa Kirin on October 5, 2023, for the acquisition of its entire issued and outstanding share capital, with a cash payment of $16.00 per ADS and contingent value rights[127][128]. - The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of March 18, 2024, for the biologics license application (BLA) of OTL-200, which is currently under review[127][131]. - The marketing authorization for Strimvelis was transferred to Fondazione Telethon on July 17, 2023, and the company will no longer recognize revenue from Strimvelis sales[132][135]. - The company expects to continue incurring significant expenses and increasing operating losses for the foreseeable future, necessitating substantial additional funding if the transaction with Kyowa Kirin does not close[126]. - The company is advancing a pipeline of HSC gene therapies aimed at addressing serious diseases with limited treatment options[123]. - The company plans to expand its global footprint by qualifying leading centers with transplant and disease area expertise and enabling patient identification through diagnostics initiatives[127]. - The company aims to grow its sales, marketing, and distribution infrastructure for the commercialization of Libmeldy in Europe[39]. - The company plans to develop and implement in-house manufacturing operations and facilities[39]. - The company is focused on expanding its intellectual property portfolio and complying with public company obligations[39]. Currency and Interest - The company recorded net realized and unrealized foreign currency losses of $4.5 million for the three months ended September 30, 2023, compared to $22.8 million for the same period in 2022[192]. - For the nine months ended September 30, 2023, the company recorded net realized foreign currency gains of $3.0 million, contrasting with losses of $47.1 million for the same period in 2022[192]. - The company does not currently engage in currency hedging activities but may consider using foreign currency forward and swap contracts in the future[194]. - Interest income increased significantly to $3,925 million for the nine months ended September 30, 2023, compared to $686 million in 2022, an increase of $3,239 million (472.5%) due to rising interest rates[161]. - Interest expense increased to $2.9 million in 2023 from $2.1 million in 2022, while interest income rose to $3.9 million from $0.7 million[169]. Accounting Policies - There have been no material changes to the company's critical accounting policies since December 31, 2022, except for the addition of the derivative liabilities policy[186]. - The company has classified PIPE Warrants and PIPE Units as liability instruments, impacting the financial statements based on fair value assessments[187].

Financial Performance - For the six months ended June 30, 2023, the net losses were $29.7 million, compared to $95.2 million for the same period in 2022, indicating a significant reduction in losses [120]. - The company reported a net loss attributable to ordinary shareholders of $29.719 million for the six months ended June 30, 2023, compared to a net loss of $95.212 million in 2022, a reduction of $65.493 million or -68.8% [153]. - Other (expense) income improved from a loss of $25.3 million in the first half of 2022 to a gain of $19.1 million in the first half of 2023, driven by reduced foreign currency transaction losses [161]. Revenue - Total revenues for the three months ended June 30, 2023, were $7.3 million, an increase of $2.9 million from $4.4 million in the same period in 2022 [140]. - Product revenue, net for Libmeldy was $6.7 million for the three months ended June 30, 2023, up from $3.1 million in 2022, reflecting a $3.5 million increase [141]. - Collaboration revenue for the three months ended June 30, 2023, was $0.7 million, compared to $0.6 million in the same period in 2022 [142]. - Product revenue, net for the six months ended June 30, 2023, was $7.185 million, a decrease of $1.655 million or -18.7% compared to $8.840 million in 2022 [153]. Expenses - Research and development expenses for the three months ended June 30, 2023, were $16.7 million, down from $22.0 million in 2022, a decrease of $5.3 million [140]. - Selling, general and administrative expenses decreased to $11.0 million for the three months ended June 30, 2023, from $13.7 million in 2022, a reduction of $2.7 million [140]. - Total research and development expenses for the six months ended June 30, 2023, were $32.688 million, down $17.511 million or -34.8% from $50.199 million in 2022 [158]. - Total selling, general and administrative expenses decreased from $27.029 million for the six months ended June 30, 2022, to $22.127 million for the same period in 2023, a decrease of $4.902 million or -18.1% [153]. Cash Flow and Financing - Net cash used in operating activities was $57.3 million for the first half of 2023, primarily due to a net loss of $29.7 million and unrealized foreign currency transaction gains of $9.8 million [170]. - Net cash used in investing activities was $36.0 million in the first half of 2023, mainly due to purchases of marketable securities totaling $114.6 million [173]. - Net cash provided by financing activities was $62.4 million in the first half of 2023, primarily from a private placement financing of $68.0 million [175]. - The company completed a private placement in March 2023, raising gross proceeds of $34.0 million from the initial closing and an additional amount in June 2023 [125]. Market and Product Development - The marketing authorization for Strimvelis was transferred to Fondazione Telethon in July 2023, resulting in no future revenue from this product [123]. - The company plans to continue developing its product candidates and expanding its sales and marketing infrastructure for Libmeldy [176]. - The company generated its first commercial sale of Libmeldy in Europe in the first quarter of 2022, following its marketing authorization in December 2020 [164]. Foreign Currency and Interest Rates - For the three months ended June 30, 2023, the company recorded net realized and unrealized foreign currency gains of $1.9 million, compared to losses of $18.2 million for the same period in 2022 [185]. - For the six months ended June 30, 2023, the company recorded net realized foreign currency gains of $7.5 million, compared to losses of $24.3 million for the same period in 2022 [185]. - The company does not currently engage in currency hedging activities but may consider using foreign currency forward and swap contracts in the future [187]. - The company does not expect immediate changes in interest rates to materially affect its operating results or cash flows [183].