Gene Therapy

Marengo Therapeutics Announces Clinical Study Collaboration with Gilead Sciences to Evaluate Invikafusp alfa (STAR0602) and Trodelvy® in both Metastatic TNBC and Metastatic HR+/HER2- Breast Cancers

09/23/2024

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…

Read More

Kriya Highlights Positive Preclinical Data for KRIYA-586, a Gene Therapy Product Candidate for Thyroid Eye Disease (TED), at ESOPRS Annual Meeting

09/18/2024

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…

Read More

Ocugen Announces Completion of Dosing in Subjects with Stargardt Disease in High Dose Cohort of Phase 1/2 GARDian Clinical Trial of OCU410ST—A Modifier Gene Therapy

09/11/2024

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…

Read More

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

01/05/2024

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…

Read More

UK authorises gene therapy for blood disorders in world first

12/11/2023

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…

Read More

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

11/22/2023

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…

Read More

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

08/24/2023

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,…

Read More

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

05/30/2023

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…

Read More

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

05/29/2023

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:…

Read More

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)

05/25/2023

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…

Read More