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) announced participation in two upcoming virtual investor conferences. The Barclays Gene Editing & Gene Therapy Summit will be held on November 15, 2021, at 1:00 p.m. ET, where Orchard will join a panel discussion. The following day, November 16, 2021, at 9:20 a.m. ET, the Stifel 2021 Virtual Healthcare Conference will take place. Live webcasts of both presentations will be available on the company's website, with replays archived after the events.
Orchard Therapeutics (Nasdaq: ORTX) reported Q3 2021 results, revealing a net loss of $36.4M, up from $20.3M in Q3 2020. Revenue from Strimvelis was $0.7M, down from $2.0M, while collaboration revenue from Pharming Group was $0.5M. R&D expenses rose to $20.8M due to increased costs in neurometabolic programs. Cash and investments stand at approximately $254M, supporting operations into H1 2023. Updates on gene therapy programs, including OTL-201 and OTL-204, show promising clinical results. The company is advancing Libmeldy launches across Europe.
Orchard Therapeutics (Nasdaq: ORTX) has announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress, scheduled from October 19-22, 2021. This event will showcase critical clinical and pre-clinical data related to the company’s hematopoietic stem cell (HSC) gene therapy portfolio. Key presentations include updates on the ongoing study of OTL-201 for Mucopolysaccharidosis type IIIA and pre-clinical findings from OTL-204 targeting frontotemporal dementia. Notably, expert Alessandra Biffi will share insights on HSC gene therapy's potential for neurodegenerative diseases.
Orchard Therapeutics (Nasdaq: ORTX) presented its research in HSC gene therapy, focusing on its OTL-104 program for Crohn's disease and new applications involving antigen-specific regulatory T cells and monoclonal antibodies. The company emphasizes its differentiated gene therapy platform, which has demonstrated significant safety and efficacy across over 160 patients. The OTL-104 program is advancing towards IND-enabling studies, and the potential for partnerships in new therapeutic areas is highlighted. Orchard aims to expand its HSC gene therapy approach beyond rare diseases.
Orchard Therapeutics (Nasdaq: ORTX) has appointed Fulvio Mavilio, Ph.D., as Chief Scientific Officer and Nicoletta Loggia, Ph.D., as Chief Technical Officer. Leslie Meltzer, Ph.D., has been promoted to Chief Medical Officer. These changes aim to enhance the company's strategic focus on gene therapy innovation, especially in hematopoietic stem cell (HSC) therapies. The executives bring extensive expertise in their fields, with planned advancements in clinical development and manufacturing capabilities. Mavilio will start full-time in January 2022, while the other appointments are effective immediately.
Orchard Therapeutics (Nasdaq: ORTX) will host a virtual R&D Investor Event on September 14, 2021, from 7:00 a.m. to 8:00 a.m. ET. The management team will provide updates on their HSC gene therapy initiatives, particularly the OTL-104 program targeting NOD2 Crohn's disease, and discuss advancements in developing antigen-specific regulatory T cells.
Presenters include CEO Bobby Gaspar, M.D., Ph.D., and Director Piv Sagoo, Ph.D. The presentation will be streamed live and archived on Orchard's website.
Azura Ophthalmics Ltd., a clinical-stage company focused on therapies for Meibomian Gland Dysfunction (MGD), has appointed Dr. Steven M. Altschuler as chair of its board of directors. Dr. Altschuler, who has over 20 years of experience in healthcare and biotechnology, expressed confidence in Azura's potential to transform ocular surface disease treatment. He previously co-founded Spark Therapeutics and held leadership roles at major healthcare institutions. Azura aims to advance its lead candidate AZR-MD-001 while addressing various ocular diseases.
Orchard Therapeutics (Nasdaq: ORTX) announced the grant of stock options to four new employees, totaling 278,500 shares, under its 2020 Inducement Equity Plan. These options, exercisable at $3.09 per share, will vest over four years, providing 25% on the first anniversary of employment and the remainder monthly over three years. This grant aligns with Nasdaq Listing Rule 5635(c)(4) and aims to incentivize new talent in the company’s gene therapy initiatives.
Orchard Therapeutics (Nasdaq: ORTX) announced that its management team will present at key investor conferences in September 2021. These events include the Goldman Sachs Biotech Symposium on September 7, Morgan Stanley Healthcare Conference on September 15, and Oppenheimer Life Sciences Summit on September 20, among others. Live webcasts will be available on the company's website, enhancing accessibility for investors. Orchard Therapeutics specializes in innovative gene therapies aimed at treating severe diseases, utilizing a unique ex vivo approach with genetically modified blood stem cells.
Orchard Therapeutics (Nasdaq: ORTX) reported financial results for Q2 2021, highlighting significant progress in its gene therapy programs. Regulatory feedback received on OTL-200 for metachromatic leukodystrophy and OTL-203 for mucopolysaccharidosis type I is paving the way for pivotal trials. The company has secured a $17.5 million upfront payment from a collaboration with Pharming Group for OTL-105, aimed at treating hereditary angioedema. Cash and investments total approximately $270 million, providing operational runway into H1 2023.
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