Gene Therapy
Excerpt from the Press Release: ALAMEDA, Calif.–(BUSINESS WIRE)–Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, presented preclinical data on its CRISPR X-Editor (XE) and Epigenetic Long-Term X-Repressor (ELXR) technologies at the American Heart Association’s Scientific Sessions 2024. The new data described in Scribe’s late-breaking, oral, and…
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We are thrilled to announce this significant milestone in the clinical investigation of ABI-110″ said Shawn Liu, Ph.D., Chief Executive Officer of Avirmax Biopharma Inc. “ABI-110 has the potential to revolutionize the treatment landscape for Wet AMD and PCV.” Excerpt from the Press Release: HAYWARD, Calif., Nov. 21, 2024 /PRNewswire/ — Avirmax Biopharma, Inc., a leader in the…
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Excerpt from the Press Release: CAMBRIDGE, Mass., Sept. 13, 2024 /PRNewswire/ — Marengo Therapeutics, Inc., a clinical-stage biotech company pioneering a new way to activate T cells targeting the Vβ chain of the T cell receptor to select the optimal T cell subsets against cancer, today announced that it has entered into a clinical study collaboration…
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Excerpt from the Press Release: – Preclinical data demonstrate pharmacodynamic activity of KRIYA-586, an investigational gene therapy which encodes for an anti-IGF1R antibody – – Kriya anticipates advancing KRIYA-586 into the clinic in 2025 to evaluate its safety and efficacy in people suffering from TED – Click the button below to read the entire Press…
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Excerpt from the Press Release: MALVERN, Pa., Aug. 28, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that dosing is complete in the third cohort of its Phase 1/2 GARDian clinical trial for…
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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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