News & Updates

World’s First Patient Treated with Personalized CRISPR Gene Editing Therapy at Children’s Hospital of Philadelphia

05/23/2025

Landmark Study from CHOP and Penn Medicine Showcases the Power of Customized Gene Editing Therapy to Treat Patient with Rare Metabolic Disease Excerpt from the Press Release: PHILADELPHIA and NEW ORLEANS, May 15, 2025 /PRNewswire/ — In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR…

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Orna Therapeutics Presents New Preclinical Data Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases at the American Society of Gene and Cell Therapy Annual Meeting

05/22/2025

Excerpt from the Press Release: WATERTOWN, Mass., May 15, 2025 /PRNewswire/ — Orna Therapeutics, a leading biotechnology company developing a proprietary pipeline of in vivo therapies across a broad range of autoimmune and oncology indications, today announced the presentation of new preclinical data supporting its in vivo CAR therapy approach in autoimmune diseases during an…

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Vaxart Publishes Positive, Complete Data from a Phase 2b Challenge Study of its First-Generation Oral Pill Norovirus Vaccine Candidate in Science Translational Medicine

05/21/2025

Challenge Study measured safety, efficacy against infection and symptomatic disease as well as viral shedding Machine learning analyses identified statistically significant correlates of protection and will be incorporated into the development of Vaxart’s second-generation norovirus vaccine candidate, which is being evaluated in a Phase 1 clinical trial Excerpt from the Press Release: SOUTH SAN FRANCISCO,…

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Ten-Year APHINITY Data Show Genentech’s Perjeta-based Regimen Reduced the Risk of Death by 17% in HER2-Positive Early-Stage Breast Cancer

05/20/2025

– Long term follow-up in this curative setting demonstrated clinically meaningful survival benefit when adding adjuvant Perjeta® (pertuzumab) to Herceptin® (trastuzumab) and chemotherapy – – 21% reduction in the risk of death was seen in the pre-specified subgroup of people with lymph node-positive disease – – Data to be presented as a late-breaking abstract at…

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Marea Therapeutics Announces Publication in The Lancet of Positive Data from Phase 2a Clinical Trial of MAR001 for Cardiovascular Disease

05/19/2025

Publication follows late-breaking oral presentation of Phase 2a results at the 93rd EAS Congress; MAR001 demonstrated approximately 53% placebo-adjusted mean reductions in remnant cholesterol and triglycerides at 12 weeks Second publication of MAR001 preclinical results in eBioMedicine that demonstrate improved plasma lipid profiles Excerpt from the Press Release: SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Marea Therapeutics, Inc.,…

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TransCode Therapeutics Successfully Completes Initial Dosing of Patients in Cohort 4 of Phase 1a Clinical Trial; No Dose Limiting Toxicities Reported

05/16/2025

Excerpt from the Press Release: BOSTON, May 8, 2025 /PRNewswire/ — TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced that the third patient in Cohort 4 of its Phase 1a clinical trial has received their initial dose of TTX-MC138. All cohorts have enrolled…

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IMUNON Announces First Site Initiated for Pivotal Phase 3 OVATION 3 Study of IMNN-001 in Newly Diagnosed Advanced Ovarian Cancer

05/15/2025

Company currently initiating additional trial sites and working with study investigators to enroll participants IMNN-001 is the first and only immunotherapy to show meaningful overall survival benefit in a Phase 2 trial in women with advanced ovarian cancer Data from Phase 2 OVATION 2 Study are encouraging, with new IMNN-001 data to be highlighted in…

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OmRx Oncology Initiates Phase 2 Trial of Oral PD-L1 Inhibitor OX-4224 in NSCLC

05/14/2025

Study will evaluate OX-4224 as a second-line monotherapy in patients with PD-L1–positive non-small cell lung cancer, expanding potential access to checkpoint inhibitors in low-resource settings Excerpt from the Press Release: SAN DIEGO–(BUSINESS WIRE)–OmRx Oncology, or “OmRx,” a clinical-stage biopharmaceutical venture dedicated to expanding access to cancer immunotherapy worldwide, today announced the initiation of a Phase…

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Quebec man among first in Canada to receive lab-grown cell transplant for Type 1 diabetes

05/13/2025

Excerpt from the Press Release: A 58-year-old Montrealer is the first patient in Quebec to receive a transplant of lab-grown cells to help treat his Type 1 diabetes — a milestone in an international clinical trial underway at the McGill University Health Centre (MUHC). Bruno Houde, who was diagnosed with the disease at age seven,…

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Corvus Pharmaceuticals Announces Data from Cohorts 1-3 of Placebo-Controlled Phase 1 Clinical Trial of Soquelitinib for Atopic Dermatitis

05/12/2025

Data continue to demonstrate favorable safety and efficacy profile, including earlier and deeper responses in cohort 3 compared to cohorts 1-2 Data to be presented in an oral session and poster at the Society for Investigative Dermatology 2025 Annual Meeting Company to discuss data today on its first quarter 2025 business update conference call and…

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