Gene Therapy
Phase 1 study aims to evaluate safety and tolerability of a single ascending dose of RCT1100 in patients with PCD caused by mutations in the DNAI1 gene RCT1100 is the first clinical evaluation of an inhaled mRNA-based therapy designed to restore ciliary function in people with PCD Excerpt from the Press Release: MENLO PARK, Calif.–(BUSINESS…
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Excerpt from the Press Release: LONDON, Nov 16 (Reuters) – Britain has authorised a gene therapy that aims to cure sickle-cell disease and another type of inherited blood disorder for patients aged 12 and over, the country’s medical regulator said on Thursday, becoming the first in the world to do so. Casgevy is the first…
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Casgevy the 1st medicine licensed using the gene editing tool CRISPR Excerpt from the Press Release: Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the disease in the U.K. In a statement on Thursday, the Medicines…
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Excerpt from the Press Release: WUHAN, China and SAN DIEGO, Aug. 17, 2023 /PRNewswire/ — Neurophth Therapeutics, Inc. (“Neurophth”) announced today that the first patient has been dosed in the international multi-region, multi-center Phase I/II clinical trial for the treatment of Leber hereditary optic neuropathy caused by ND1 mutation (ND1-LHON). Neurophth is conducting a Phase I/II,…
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Excerpt from the Press Release: Canada has one of the highest rates of Multiple Sclerosis (MS) in the world — a chronic condition for which there is no cure. But a new research breakthrough at the University of Toronto has the potential to improve the lives of those living with the disease far beyond current…
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Rejuva device & procedure reliably and successfully targets pancreas with Adeno Associated Virus (AAV), potentially enabling gene therapy to address pancreatic diseases for the first time Rejuva platform and genetic medicines offer potential for reversal of Type 2 Diabetes with a single point-in-time local administration of durable GLP-1-based genetic medicines Excerpt from the Press Release:…
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Excerpt from the Press Release: SAN DIEGO–(BUSINESS WIRE)–Rejuvenate Bio, today announced new data for its gene therapy RJB-01 from a pilot study in canines with myxomatous mitral valve disease (MMVD), at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), held May 16-20, 2023, in Los Angeles, California. RJB-01, expresses…
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Lead program, IG-002 targeting ABCA4 retinopathies, demonstrates durable 12-month expression of human ABCA4 protein using a single subretinal dose of DNA payload Excerpt from the Press Release: CAMBRIDGE, Mass., April 20, 2023 /PRNewswire/ — Intergalactic Therapeutics, a company focused on transforming medicine through non-viral gene therapy, announced today that Gayathri Ramaswamy, Ph.D., Vice President, Drug Discovery…
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– BBP-398, an investigational SHP2 inhibitor, is a potentially best-in-class therapy for use in combination approaches, which is shown by preclinical findings demonstrating its safety profile, continuous, once-daily dosing regimen and synergistic efficacy to treat cancers driven by KRAS mutations – If successful, the combination of investigational therapy BBP-398 and OPDIVO has the potential to…
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Excerpt from the Press Release: CAMBRIDGE, Mass., Feb. 16, 2023 (GLOBE NEWSWIRE) — Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, presented clinical data from VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (previously VOR33) in patients with acute myeloid leukemia (AML). In the first patient, trem-cel maintained hematopoiesis through three…
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