Cel therapy

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

08/16/2022

– 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

07/07/2022

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

07/05/2022

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

06/27/2022

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

06/27/2022

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

06/24/2022

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

06/15/2022

— 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

06/14/2022

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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Bristol Myers Squibb Data at ASCO and EHA 2022 Highlight Progress in Transforming Treatment for Patients with Cancer and Blood Disorders

06/01/2022

Long-term data from Opdivo plus Yervoy-based combinations demonstrate durable survival in metastatic non-small cell lung cancer (NSCLC) and metastatic melanoma, including results from landmark five-year analysis of CheckMate -227 in NSCLC First disclosure from PILOT study of Breyanzi in second-line large B-cell lymphoma underscores important role of cell therapy earlier in treatment paradigm New data…

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Locanabio’s Proprietary CORRECTx™ RNA-Targeting Gene Therapy Reduces Disease-Causing Hexanucleotide Repeat Expansions in a Model of Amyotrophic Lateral Sclerosis

05/30/2022

– Oral presentation of new preclinical data at American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting – Excerpt from the Press Release: SAN DIEGO, May 19, 2022 /PRNewswire/ — Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with severe neuromuscular and neurodegenerative diseases, today announced the presentation of new preclinical data on its…

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