gene 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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Leading Clinical Experts Across the U.S. Unite to Support Universal Genetic Testing for all Patients with Cancer


– Clinicians agree that evidence supports testing benefits for all – Excerpt from the Press Release: SAN FRANCISCO, Sept. 15, 2022 /PRNewswire/ — Invitae ( NYSE: NVTA), a leading medical genetics company, joined other clinical experts in releasing a new commentary in Journal of Clinical Oncology Precision Oncology, underscoring the importance of universal germline testing for…

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Graphite Bio Doses First Patient with Investigational Gene Editing Therapy GPH101 for Sickle Cell Disease


GPH101, now called nulabeglogene autogedtemcel (nula-cel), designed to directly correct the genetic mutation that causes sickle cell disease Initial proof-of-concept data from Phase 1/2 CEDAR trial anticipated in mid-2023 Excerpt from the Press Release: SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision…

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Alnylam Uncovers Genetic Mutations in INHBE That Protect Against Abdominal Obesity


– Publication in Nature Communications Reports that People with Loss of Function Mutations in the INHBE Gene Have Reduced Abdominal Fat, a Favorable Metabolic Profile, and are at Lower Risk of Cardiovascular Disease and Type 2 Diabetes – – Alnylam to Pursue INHBE as a Therapeutic Target for Cardiometabolic Disease – Excerpt from the Press…

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The Genomic Test Demonstrated 100 Percent Sensitivity and 100 Percent Specificity for MTC Excerpt from the Press Release: SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Jul. 20, 2022– Veracyte, Inc. (Nasdaq: VCYT) today announced that new clinical validation data published online in Thyroid show that the company’s RNA sequencing-based Afirma Medullary Thyroid Cancer (MTC) Classifier demonstrated high sensitivity and specificity in identifying…

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ROME Therapeutics Announces Publication of First Crystal Structure of a Human Endogenous Reverse Transcriptase in PNAS


Study reveals first-ever structure of a non-viral endogenous reverse transcriptase (eRT) Human endogenous retrovirus-K (HERV-K) RT shows striking similarity to HIV RT Findings enable structure-based drug discovery for eRTs Excerpt from the Press Release: CAMBRIDGE, Mass.–(BUSINESS WIRE)–ROME Therapeutics, a biotechnology company harnessing the power of the dark genome for drug development, today announced a new…

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


– 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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Gamida Cell Presents New Data from NAM-Enabled Genetically Modified Natural Killer (NK) Pipeline at International Society for Cell & Gene Therapy 2022


Poster selected for inclusion in conference’s Elevator Pitch Session: GDA-301 produces enhanced potency and persistence with combined genetic manipulation of CISH gene editing and the engineered expression of membrane-bound IL-15 for targeting hematologic malignancies and solid tumors GDA-601 generates promising immunotherapeutic potential to target multiple myeloma cells Company plans to select a genetically modified NK…

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