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) and Pharming Group N.V. have formed a strategic collaboration to develop OTL-105, an ex vivo gene therapy for hereditary angioedema (HAE). OTL-105 aims to enhance C1 esterase inhibitor production in patients, potentially providing a long-lasting treatment solution. Pharming will handle clinical development and commercialization, while Orchard oversees manufacturing. Orchard receives an upfront payment of $17.5 million and could earn up to $189.5 million in milestones. The deal represents significant potential in a $2 billion HAE market.
Orchard Therapeutics (Nasdaq: ORTX) announced significant updates regarding its gene therapy programs following regulatory interactions with the FDA. The company aims to submit a Biologics License Application for OTL-200 for MLD by late 2022 or early 2023, leveraging existing patient data. Guidance received concerning the OTL-203 trial for MPS-IH will shape its clinical protocol, while the OTL-201 trial for MPS-IIIA has met its enrollment goal. OTL-103's submission timelines are adjusted, with MAA expected in 2022. A conference call is scheduled for today at 8:00 a.m. ET for further discussion.
Orchard Therapeutics (Nasdaq: ORTX) reported significant advancements in its gene therapy programs, notably the upcoming launch of Libmeldy for early-onset MLD in Germany. A submission for pricing and reimbursement is underway, with initial treatment center qualification in progress. The company anticipates regulatory updates on OTL-200 by mid-2021. Recent financial results show a reduced net loss of $35.2 million for Q1 2021 and cash reserves of approximately $300 million, ensuring operational support into H1 2023.
Orchard Therapeutics (Nasdaq: ORTX) announced compelling results for OTL-101, a gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID), published in the New England Journal of Medicine. Among 50 patients treated, there was a 100% overall survival rate, with ≥95% event-free survival at two and three years. The study, the largest of its kind, also noted significant improvement in immune function and high rates of immunoglobulin replacement therapy discontinuation. Safety data indicated no serious adverse events associated with the therapy.
Orchard Therapeutics (Nasdaq: ORTX) announced the acceptance of seven abstracts at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), highlighting their advancements in hematopoietic stem cell (HSC) gene therapy. Key programs include OTL-203 for mucopolysaccharidosis type I and OTL-101 for adenosine deaminase severe combined immunodeficiency. The presentations underscore the potential for HSC gene therapy to transform treatments for rare diseases and the company’s innovations in improving manufacturing efficiency.
Orchard Therapeutics (Nasdaq: ORTX) has expanded its partnership with Be The Match BioTherapies to enhance supply chain services for the upcoming commercial launch of Libmeldy, a gene therapy approved in Europe for early-onset metachromatic leukodystrophy (MLD). This collaboration aims to streamline the onboarding and training of apheresis centers and ensure effective logistics throughout the treatment process. With approximately 1 in 100,000 births affected by MLD, Orchard's efforts are critical to delivering this innovative therapy to patients in need.
Orchard Therapeutics (Nasdaq: ORTX) announced a $150 million PIPE financing to support its gene therapy strategies. The company reported financial results for 2020, showing R&D expenses of $22.6 million, down from $30.9 million the previous year. A net loss of $33.6 million in Q4 2020 was noted, an improvement from $45.4 million in Q4 2019. Key milestones include initiating an OTL-203 registrational trial for MPS-I Hurler Syndrome by year-end 2021 and expected U.S. filing for OTL-200 for Metachromatic Leukodystrophy by mid-2021. Cash reserves of $191.9 million are projected to finance operations until mid-2023.
Orchard Therapeutics (Nasdaq: ORTX) announced its participation in three upcoming virtual investor conferences. The events are as follows:
- Cowen 41st Annual Healthcare Conference on March 4, 2021, at 10:30 AM ET.
- Barclays 2021 Global Healthcare Conference on March 11, 2021, at 8:35 AM ET.
- Oppenheimer 31st Annual Healthcare Conference on March 17, 2021, at 8:00 AM ET.
Live webcasts of the Barclays and Oppenheimer presentations will be available on the company's website, with replays archived afterward.
Orchard Therapeutics (ORTX) announced promising interim results from its gene therapies, OTL-203 for MPS-I and OTL-201 for MPS-IIIA, presented at the 17th Annual WORLDSymposium. All eight patients treated with OTL-203 showed stable cognitive and motor functions, with normal growth metrics at multiple follow-ups. Preliminary data for OTL-201 indicated successful engraftment and improved biomarker levels. The company is pursuing regulatory feedback from the FDA and EMA for OTL-203, aiming to initiate a registrational study by year-end 2021.
Orchard Therapeutics (Nasdaq: ORTX) announced a successful $150 million private investment in public equity (PIPE) offering, issuing 24,115,755 shares at $6.22 each. This financing includes participation from notable healthcare investors like RA Capital Management and Avidity Partners.
The funds will support the launch of Libmeldy in Europe, advance regulatory and clinical activities, and expand gene therapy approaches. The financing is expected to close on February 9, 2021.
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