Gene Therapy
Eight patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable safety and tolerability observed to date Assessments of the initial 3 patients reaching their six-month follow-up showed improvements on validated functional scales and increases in brain white matter and myelin content Encouraging efficacy and safety data support further development of…
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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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– 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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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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– 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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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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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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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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– 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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