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Highlights the broader potential of Orchard’s ex vivo HSC gene therapy platform approach in new and larger indications Reinforces Pharming’s commitment to the HAE community and utilizes its relevant clinical expertise and global commercialization infrastructure ...
Vor has a lead asset entering phase 1 clinical trials for AML that has the potential to become the standard of care for stem cell transplants. The company also has a platform and process to expand the use of their lead asset, VOR33, and design other cell therapies for beyond AML. ...
Ole Schwander/iStock via Getty Images Orchard Therapeutics ([[ORTX]] -2.3%) is trading lower for the second straight session today after the company issued an update on the progress of its lead gene therapy programs. In reaction, Cantor Fitzgerald analyst Emma Nealon has lowered the pric...
metamorworks/iStock via Getty Images Orchard Therapeutics (ORTX) is trading marginally higher in the pre-market with a ~6.0% gain after the company provided updates on the progress of its lead gene therapy programs. The experimental treatments OTL-200, OTL-203, and OTL-103 targ...
Expected clinical package for OTL-200 BLA for MLD using data from existing patients clarified with FDA Guidance obtained on design of OTL-203 global registrational trial for MPS-IH Enrollment goal met in OTL-201 POC trial for MPS-IIIA Updates for OTL-103 MAA and BL...
Orchard Therapeutics (ORTX): Q1 GAAP EPS of -$0.31 beats by $0.03.Cash, cash equivalents and investments as of March 31, 2021, were $298.4 million compared to $191.9 million as of December 31, 2020, with the increase primarily driven by net proceeds of $143.7 million from the February 2021 pr...
Commercial Activities Advancing for Upcoming Launch of Libmeldy TM (atidarsagene autotemcel) for Eligible Patients with Early-onset MLD in Germany Update on OTL-200 U.S. BLA Filing Strategy for MLD on Track for Mid-2021 Following Type B RMAT Meeting with FDA ...
InvestorPlace - Stock Market News, Stock Advice & Trading Tips Good morning, investors, and get ready for another busy day of trading! We’re back at it again with a look at the biggest pre-market stock movers for Wednesday. Expect a lot of earnings news to be affecting stock as ...
Orchard Therapeutics (ORTX) announces data published in the New England Journal of Medicine evaluating the safety and efficacy of investigational gene therapy products, including OTL-101, for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).Fifty ADA-SCID patie...
100% overall survival and ≥ 95% event-free survival observed at two and three years following one-time treatment with lentiviral HSC gene therapy 50 total participants represent largest published dataset of gene therapy-treated patients with a monogenic condit...
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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, ...
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Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient’s own hematopoietic stem cells HURCULES study to enroll 40 patients at clinical sites across the U.S....