Cel therapy

Neurona Therapeutics Presents Clinical Case Study from First Human Trial of Regenerative Cell Therapy, NRTX-1001, in Adults with Drug-Resistant Focal Epilepsy


Clinical assessments three-months post-treatment report no serious adverse events and reduced seizure frequency to date in first trial participant Data Safety Monitoring Board clears advancement of trial to continue enrollment Pioneering cell therapy approach could provide a disease-modifying treatment for drug-resistant focal epilepsy Data presented at ISSCR-ASGCT Conference: Emerging Therapies at the Intersection of Genetic…

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Geron Announces First Patient Dosed in IMproveMF Phase 1 Combination Study in Frontline Myelofibrosis


Preclinical data showed synergistic and additive effects of combination imetelstat and ruxolitinib Study intended to explore potential for disease modification with imetelstat in earlier, frontline myelofibrosis setting Single-agent imetelstat currently being studied in a separate Phase 3 trial designed to confirm clinically meaningful benefits observed in relapsed/refractory MF patients in Phase 2 study Excerpt from…

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Agios Announces Publication of Phase 2 Data in The Lancet Demonstrating Safety and Efficacy of PYRUKYND® (mitapivat) in Non-transfusion-dependent α- and β-Thalassemia


– In Adults with Non-transfusion-dependent α- or β-Thalassemia, PYRUKYND® Induced ≥1.0 g/dL Hemoglobin Increase from Baseline in 16 of 20 (80%) Patients Between Weeks 4-12 – – PYRUKYND® Safety Profile Consistent with Label for FDA-approved Indication in Pyruvate Kinase Deficiency – – Actively Enrolling Phase 3 ENERGIZE and ENERGIZE-T Studies Evaluating PYRUKYND® in Adults with Non-transfusion-dependent and Transfusion-dependent α-…

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Ventyx Biosciences Announces Positive Topline Phase 1 Data for its Peripheral NLRP3 Inhibitor VTX2735


Excellent safety, tolerability and pharmacokinetic profile Robust dose-dependent target engagement as measured by ex vivo IL-1β release assay Phase 2 trial planned in CAPS patients to efficiently establish clinical proof of concept Clinical update in Q3 from Phase 1 trial of VTX958, our oral, selective allosteric TYK2 inhibitor Excerpt from the Press Release: ENCINITAS, Calif., June…

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Poseida Therapeutics Announces Publication Highlighting Potential of Cas-CLOVER™ as an Efficient and Robust Gene Editing System for Developing Allogeneic CAR-T Products


Publication highlights ability of Cas-CLOVER to perform multiplexed gene editing to produce allogeneic products with a high percentage of Tscm cells that may result in better tolerability and deeper clinical responses Cas-CLOVER has demonstrated lower off-target and translocation activity than other published technologies including CRISPR, TALENs and Base Editors Excerpt from the Press Release: SAN…

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Affini-T Therapeutics & Metagenomi Announce Next-Generation Gene Editing Partnership to Advance Cell Therapies for Solid Tumor Patients


Excerpt from the Press Release: BOSTON & SEATTLE & EMERYVILLE, Calif.–(BUSINESS WIRE)–Affini-T Therapeutics, Inc., a biotechnology company unlocking the power of T cells against oncogenic driver mutations, and Metagenomi, Inc., a genetic medicines company with a versatile portfolio of next-generation, wholly-owned gene editing tools, today announced a partnership to enable Affini-T’s next generation ex vivo…

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BioCardia Announces US Patent on Imaging System for Targeting Cardiac Therapies


Excerpt from the Press Release: SUNNYVALE, Calif., June 15, 2022 (GLOBE NEWSWIRE) — BioCardia®, Inc. [Nasdaq: BCDA], a developer of cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, today announces that the United States Patent Office has granted Patent Number 11,357,463: titled, “Target Site Selection, Entry and Update With Automatic Remote Image…

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SQZ Biotechnologies Announces Generation of Dopaminergic Neurons from Human Pluripotent Stem Cells Through Single-Step Delivery of Six Cell-Fate Encoding RNAs


Pre-Clinical Research Could Lead to Rapid Development of Dopamine-Producing Neurons; Part of Company’s NIH-Funded Program Seeking Scalable Cell Replacement Therapy Approaches for Parkinson’s Disease Use of Non-Viral, Self-Amplifying RNA Enhanced and Lengthened Transcription Factor Expression, Driving the Signaling Required for Dopamine-Producing Neuron Development Findings Presented at 2022 International Society for Stem Cell Research Annual Meeting…

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Wugen Presents New Preclinical Data Demonstrating Enhanced Anti-Tumor Activity of WU-NK-101 at the European Hematology Association (EHA) 2022 Hybrid Congress


— WU-NK-101 exhibited enhanced metabolic flexibility and resistance to tumor microenvironment (TME) immunosuppression relative to conventional natural killer (NK) cells — — WU-NK-101 demonstrated robust anti-tumor activity in vitro and in vivo in preclinical models of acute myeloid leukemia (AML) — — Data support the development of WU-NK-101 for AML and solid tumor indications —…

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Resilience and MD Anderson Launch Joint Venture to Accelerate Development and Manufacturing of Innovative Cell Therapies for Cancer


Excerpt from the Press Release: SAN DIEGO & HOUSTON–(BUSINESS WIRE)–National Resilience, Inc. (Resilience) and The University of Texas MD Anderson Cancer Center today announced the launch of a joint venture, the Cell Therapy Manufacturing Center, to accelerate the development and manufacturing of innovative cell therapies for patients with cancer. Uniting the strengths of Resilience and…

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