TOKYO, LONDON and BOSTON — Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The...
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TOKYO, LONDON and BOSTON — Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, for the treatment of children...
BOSTON and LONDON — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I...
BOSTON and LONDON — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, announced various business accomplishments along with its financial results for the quarter ended March 31, 2023. “Following multiple productive interactions this year with the FDA on our clinical and CMC packages, I am delighted to announce the...
Boston and London — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, announced that the company will make a virtual presentation at the Guggenheim Genomic Medicines and Rare Disease Day on Tuesday, April 4, 2023 at 1:35pm ET. A live webcast of the presentation will be available under “News...
TARRYTOWN, N.Y. — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Commission (EC) has approved Ordspono™ (odronextamab) to treat adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), after two or more lines of systemic therapy. This marks the first...
PORTLAND, Oregon — An artificial intelligence technology can accurately and independently detect 100% of severe cases of a blindness-causing condition that affects prematurely born babies, according to new research from Oregon Health & Science University and collaborators that was published today in the journal JAMA Ophthalmology. The technology has the potential...
CORVALLIS, Ore. – A study spearheaded by Oregon State University has shown why certain polyunsaturated fatty acids work to combat a dangerous liver condition, opening a new avenue of drug research for a disease that currently has no FDA-approved medications. Scientists led by Oregon State’s Natalia Shulzhenko, Andrey Morgun and...
Researchers funded by the National Institute of Neurological Disorders and Stroke (NINDS) have found that an organ transplant drug might one day be used to treat meningioma, a type of brain tumour. The drug also could be used to treat neurofibromatosis type 2, a rare disease associated with meningiomas and...
Los Angeles, Calif. – A traumatic brain injury (TBI) can quadruple your risk for developing dementia and increase your chances of developing neurodegenerative diseases such as ALS. In a new study published in Cell Stem Cell, USC scientists use lab-grown human brain structures known as organoids to offer insights into...