Gene Therapy

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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Kura Oncology Announces First Patients Dosed in Phase 2 Registration-Directed Trial of Ziftomenib in NPM1-Mutant Acute Myeloid Leukemia


– Phase 1 trial showed 30% CR rate among 20 NPM1-mutant AML patients treated at recommended Phase 2 dose – – Phase 2 registration-directed trial expected to enroll 85 patients in the U.S. and Europe – Excerpt from the Press Release: SAN DIEGO, Feb. 09, 2023 (GLOBE NEWSWIRE) — Kura Oncology, Inc. (Nasdaq: KURA), a…

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Center for Breakthrough Medicines Launches Precision Plasmids™ Manufacturing to Accelerate Advanced Therapies into the Clinic


Superior quality Precision Plasmids™ expedite clinical trial timelines with no wait time to start manufacturing custom plasmids Excerpt from the Press Release: KING OF PRUSSIA, Pa., Feb. 2, 2023 /PRNewswire/ — The Center for Breakthrough Medicines (CBM), a contract development, and manufacturing organization (CDMO), has launched its plasmid manufacturing offering, Precision Plasmids™ to provide phase-appropriate…

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Frontera Therapeutics Doses First Patient in a Clinical Trial of FT-003 Gene Therapy for the Treatment of Wet AMD


Excerpt from the Press Release: BEDFORD, Mass. and SHANGHAI, China, Feb. 02, 2023 (GLOBE NEWSWIRE) — Frontera Therapeutics, a global clinical-stage biotechnology company that seeks to develop novel and best-in-class gene therapy medicines to improve the lives of patients across multiple disease areas, announced that it has dosed the first patient in a clinical trial…

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Cell BioEngines Enters into an Exclusive Worldwide Agreement to Develop and Commercialize Potent Immune Cell States to Cure Cancer


Excerpt from the Press Release: BOSTON and NEW YORK, Jan. 25, 2023 (GLOBE NEWSWIRE) — Cell BioEngines Inc. (the “Company”), a cell and gene therapy company focused on developing an allogenic cell-based product for solid tumors, today announced that it has entered into an exclusive license agreement with Icahn School of Medicine at Mount Sinai…

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Jasper Therapeutics Announces New Positive Briquilimab Data to be Presented at the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR


Three abstracts highlight the safety and efficacy of briquilimab combined with low-toxicity radiation conditioning to achieve full donor engraftment and leukemia disease eradication Excerpt from the Press Release: REDWOOD CITY, Calif., Jan. 13, 2023 (GLOBE NEWSWIRE) — Jasper Therapeutics, Inc. (Nasdaq: JSPR) (“Jasper” or the “Company”), a biotechnology company developing novel antibody therapies addressing chronic…

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Tempero Bio Announces FDA Clearance of Investigational New Drug (IND) Application for TMP-301 for the Treatment of Alcohol and Other Substance Use Disorders


Excerpt from the Press Release: OAKLAND, Calif., Jan. 4, 2023 /PRNewswire/ — Tempero Bio, Inc, a clinical-stage biopharmaceutical company focused on developing transformative therapies for the treatment and prevention of alcohol and other substance use disorders, today announced the U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application for TMP-301.…

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