Gene Therapy

ReCode Therapeutics Doses First Patient in Phase 1 Clinical Study of Novel mRNA-based Therapy for the Treatment of Primary Ciliary Dyskinesia


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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UK authorises gene therapy for blood disorders in world first


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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World-first gene therapy for 2 blood disorders — sickle cell and thalassemia — approved


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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Neurophth Announces First Patient Dosed in Phase I/II Clinical Trial of Second Gene Therapy


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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New gene therapy being developed at University of Toronto could treat MS


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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Fractyl Health Unveils Groundbreaking Pre-Clinical Proof of Concept Results for its Rejuva® Pancreatic Gene Therapy Platform for Metabolic Diseases at the American Society of Gene & Cell Therapy Congress


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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Rejuvenate Bio Announces Data for Gene Therapy RJB-01 in Mitral Valve Disease at 2023 Meeting of the American Society of Gene and Cell Therapy (ASGCT)


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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Intergalactic Therapeutics to Present Updated Data From Its Non-Viral Gene Therapy Platform at the 2023 Retinal Cell and Gene Therapy Innovation Summit


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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BridgeBio Pharma Announces First Lung Cancer Patient Dosed in Phase 1 Trial for SHP2 Inhibitor BBP-398 in Combination with Bristol Myers Squibb’s OPDIVO® (nivolumab)


– 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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First AML Patient Transplanted with Vor Bio’s Trem-cel Demonstrated Durable Engraftment through Multiple Mylotarg™ Cycles at Initial Dose Level


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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