Welcome to our dedicated page for Orchard Therapeutics plc news (Ticker: ORTX), a resource for investors and traders seeking the latest updates and insights on Orchard Therapeutics plc stock.
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.
Orchard Therapeutics (Nasdaq: ORTX) previewed investigational HSC gene therapy programs targeting frontotemporal dementia with progranulin mutations (GRN-FTD) and Crohn's disease (NOD2-CD), alongside a new program focused on amyotrophic lateral sclerosis (ALS). The GRN-FTD program aims to introduce the GRN gene into HSCs, potentially impacting 2,500 patients yearly in the U.S. and Europe. The ALS program seeks to restore healthy microglia to improve symptoms. New manufacturing innovations, including transduction enhancers, support larger indications. A webcast on these developments is scheduled for November 13, 2020.
Orchard Therapeutics (Nasdaq: ORTX), a leader in gene therapy, will present at the Barclays Gene Editing & Gene Therapy Summit on November 16, 2020, at 11:15 AM ET. A live webcast will be available on the company's website, with an archived replay following the presentation.
Orchard aims to revolutionize treatment for rare diseases through innovative gene therapies, utilizing genetically modified blood stem cells. The company has a strong product pipeline and acquired GSK’s rare disease gene therapy portfolio in 2018.
Orchard Therapeutics (Nasdaq: ORTX) announced preliminary results from the first patient in an ongoing proof-of-concept trial for OTL-201, aimed at treating MPS-IIIA, a rare metabolic disease. The results indicate engraftment of gene-modified cells and elevated enzyme expression over a three-month follow-up. The findings will be presented at the 62nd ASH Annual Meeting on December 7, 2020. MPS-IIIA affects about 1 in 100,000 births, with no approved treatments available. This trial could pave the way for addressing critical unmet needs for these patients.
Orchard Therapeutics (ORTX) announced a positive opinion from the CHMP regarding its gene therapy, Libmeldy, for early-onset metachromatic leukodystrophy (MLD). This therapy, if approved by the European Commission by year-end 2020, would be the first commercial ex vivo HSC lentiviral gene therapy for this condition. The company reported Q3 2020 revenue from product sales of $2.0 million, a slight increase from $1.9 million in Q3 2019, and a net loss of $20.3 million compared to $36.7 million in the same period last year. Cash reserves stand at $201.3 million, providing a runway into 2022.
Orchard Therapeutics (Nasdaq: ORTX) will host a virtual R&D event titled ‘New Horizons in Gene Therapy’ on November 13, 2020, from 9:00 a.m. to 11:00 a.m. ET. The event will feature presentations on hematopoietic stem cell gene therapy targeting frontotemporal dementia and Crohn’s disease. Key speakers include CEO Bobby Gaspar and leading experts from prestigious institutions. The event can be accessed via their website, and a replay will be available after the event. Orchard focuses on developing gene therapies for rare diseases, leveraging innovative genetic modification techniques.
Orchard Therapeutics (Nasdaq: ORTX) announced that CEO Bobby Gaspar has been appointed to the Alliance for Regenerative Medicine's 2021 board of directors. This role will see Dr. Gaspar guiding ARM's strategic priorities at a crucial time for cell and gene therapies. He emphasized the importance of collaboration to address the challenges posed by transitioning to one-time, potentially curative treatments. ARM is a leading organization advocating for the cell and gene therapy sector worldwide, promoting initiatives that advance patient access to innovative therapies.
Orchard Therapeutics (Nasdaq: ORTX) announced that the CHMP has recommended full marketing authorization for Libmeldy, the first gene therapy for metachromatic leukodystrophy (MLD) in eligible patients. Effective for late infantile and early juvenile forms, Libmeldy demonstrates potential to preserve cognitive and motor functions. Data from 35 patients highlights significant improvements in motor function and enzyme activity post-treatment, with follow-ups up to 8 years revealing durability in effects. A final decision by the European Commission is expected by year-end 2020.
Orchard Therapeutics (Nasdaq: ORTX) announced the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to its investigational gene therapy OTL-203 for treating mucopolysaccharidosis type I (MPS-I). This designation indicates the potential for significant therapeutic benefits over existing treatments. The company anticipates advancing a registrational trial in 2021, building on promising early data from an ongoing proof-of-concept study involving eight patients. MPS-I is a rare inherited disease, affecting approximately one in 100,000 live births.
Orchard Therapeutics (Nasdaq: ORTX) announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo gene therapy for treating mucopolysaccharidosis type I (MPS-I). This designation follows a clinical assessment highlighting the therapy's potential benefit compared to existing treatments. In 2021, Orchard plans to build on early study data and commence a registrational trial. MPS-I affects 1 in 100,000 live births, with severe cases often leading to early mortality.
Orchard Therapeutics (Nasdaq: ORTX) will present at two upcoming virtual investor conferences: the Morgan Stanley 18th Annual Global Healthcare Conference on September 18, 2020, at 8:45 AM ET and the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit on September 23, 2020, at 9:10 AM ET. Live webcasts will be accessible on Orchard's website under 'News & Events' and will be archived for later viewing. Orchard focuses on innovative gene therapies for rare diseases, leveraging an ex vivo autologous gene therapy approach to potentially provide curative treatments.
FAQ
What is the market cap of Orchard Therapeutics plc (ORTX)?
What does Orchard Therapeutics specialize in?
What is Libmeldy?
Which regulatory approvals has Libmeldy received?
Who recently acquired Orchard Therapeutics?
What are Orchard Therapeutics' other clinical-stage programs?
When was Orchard Therapeutics founded?
What is the mission of Orchard Therapeutics?
How does Orchard Therapeutics' HSC gene therapy work?
Who is leading Orchard Therapeutics post-acquisition?
Where can I find more information about Orchard Therapeutics?
