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Orchard Therapeutics plc (ORTX) is a pioneering biotechnology company dedicated to transforming the lives of children with life-threatening rare diseases through advanced gene therapies. The company focuses on the development, manufacturing, and commercialization of ground-breaking hematopoietic stem cell (HSC) gene therapies, which hold the potential to provide one-time treatments that address the underlying causes of severe genetic disorders.
Founded in 2015, Orchard Therapeutics collaborates with prestigious research centers globally to harness the life-saving potential of gene therapy. The company’s leadership team and partners bring over a decade of experience in advancing therapies for orphan diseases.
Orchard’s flagship product, Libmeldy® (atidarsagene autotemcel), is designed for the treatment of early-onset metachromatic leukodystrophy (MLD), a rare metabolic disorder. Libmeldy has received approval from the European Commission and the UK Medicines and Healthcare products Regulatory Agency and is under Priority Review by the U.S. FDA with a decision expected by March 18, 2024. The therapy aims to halt or slow disease progression by restoring enzymatic function.
The company is also advancing OTL-203 for mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome. These programs utilize the same HSC gene therapy platform as Libmeldy.
In a strategic move, Kyowa Kirin recently acquired Orchard Therapeutics, enhancing its portfolio with Orchard's clinically differentiated gene therapy platform. This acquisition is expected to drive the development of new treatments and support ongoing and future launches, including the continued growth of Libmeldy in Europe and potential new approvals in the U.S.
Orchard Therapeutics continues its mission to deliver curative gene therapies for severe diseases by translating promising early-stage research into approved treatments available to patients worldwide. The team remains committed to leveraging its extensive experience and cutting-edge technology to address unmet medical needs in rare and underserved conditions.
InsightAce Analytic reports that the global Cell and Gene Therapy Supply Chain/Logistics market was valued at US$ 1.23 Billion in 2022 and is projected to reach US$ 3.12 Billion by 2031, with a compound annual growth rate (CAGR) of 11.2% from 2023 to 2031. The report outlines the growing demand for customizable services and advanced IT solutions in supply chain management for cell and gene therapies, while highlighting challenges such as high funding costs and lack of standard protocols. Key players include Be The Match BioTherapies and Cryoport. The market is primarily driven by North America, with Asia Pacific expected to see faster growth.
ResearchAndMarkets.com has released the report "Global Genetic Disorders Partnering 2016-2023," providing a comprehensive overview of genetic disorder deals signed worldwide since 2016. The report captures key aspects including deal trends, financial terms, and the most active dealmakers in the biopharma industry. It includes detailed analyses of deal structures, contract documents, and financial metrics, such as headline values, upfront payments, and royalty rates. Covering multiple genetic disorders like Cystic Fibrosis and Huntington's Disease, this report aims to be an essential resource for stakeholders in genetic disorder collaborations.
Orchard Therapeutics (Nasdaq: ORTX) will present virtually at the Guggenheim Genomic Medicines and Rare Disease Day on April 4, 2023, at 1:35 PM ET. The live webcast will be accessible via the company's website, with a replay available afterward. Orchard aims to revolutionize treatments for genetic diseases through HSC gene therapy, modifying patients' blood stem cells to address the root causes of conditions. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio and is now advancing a comprehensive pipeline of therapies. For updates, visit the Orchard website or follow them on social media.
The report titled 'Germany Cell and Gene Therapy Market - Focused Insights 2023-2028' reveals that the market was valued at
Key drivers include an increase in target patient populations and robust regulatory support. Zolgensma is highlighted as the first blockbuster therapy, having generated
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