Gene Therapy

Avacta Announces Positive New Data from the AVA6000 Phase 1 trial Demonstrating Clinically Meaningful Tumor Shrinkage in Patients with Salivary Gland Cancers

01/21/2025

Meaningful tumor shrinkage observed in five out of 10 patients with partial and minor responses and 90% disease control rate in patients with salivary gland cancers Phase 1b expansion cohorts screening patients in triple negative breast cancer, soft tissue sarcoma and salivary gland cancer Excerpt from the Press Release: LONDON and PHILADELPHIA, Jan. 16, 2025…

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First of Its Kind Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment

01/07/2025

New model and vector could be instrumental in transforming the lives of XLSA patients and those with a broad spectrum of other diseases Excerpt from the Press Release: PHILADELPHIA, Jan. 2, 2025 /PRNewswire/ — Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind…

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Senti Bio Announces First Patient Dosed in Clinical Trial of SN301A in Hepatocellular Carcinoma in Collaboration with Celest Therapeutics

12/30/2024

Dose-finding clinical trial in China designed to evaluate safety and preliminary anti-tumor activity of SN301A (SENTI-301A manufactured in China) in hepatocellular carcinoma (“HCC”) Excerpt from the Press Release: SOUTH SAN FRANCISCO, Calif., Dec. 16, 2024 (GLOBE NEWSWIRE) — Senti Biosciences, Inc. (Nasdaq: SNTI) (“Senti Bio”), a clinical-stage biotechnology company developing next-generation cell and gene therapies using…

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Scribe Therapeutics Reports Preclinical Data Validating its CRISPR Genome Editing and Epigenome Modifying Technologies for Addressing Cardiometabolic Disease at American Heart Association (AHA) Scientific Sessions 2024

12/05/2024

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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Avirmax Biopharma Announces First Patient Dosed in Clinical Trial of ABI-110, a Groundbreaking Gene Therapy for Wet AMD Including PCV

11/27/2024

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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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…

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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…

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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…

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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…

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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…

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