Gene Therapy

4D Molecular Therapeutics Announces Interim Clinical Data from Phase 1/2 Clinical Trial of 4D-710 for Cystic Fibrosis Lung Disease at NACFC 2022

11/10/2022

Cohort 1 lung bronchoscopy sample results demonstrate widespread delivery and expression of the 4D-710 CFTR∆R transgene in 100% of samples from all three patients Cohort 1 safety and tolerability of 4D-710 demonstrated to date, with no drug-related adverse events following aerosol delivery Cohort 1 enrollment completed; Cohort 2 enrollment on-going Conference call & webcast to…

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T2 Biosystems Initiates Studies to Add Acinetobacter Baumannii to the FDA-Cleared T2Bacteria Panel

10/28/2022

Excerpt from the Press Release: LEXINGTON, Mass., Oct. 20, 2022 (GLOBE NEWSWIRE) — T2 Biosystems, Inc. (NASDAQ:TTOO), a leader in the rapid detection of sepsis-causing pathogens and antibiotic resistance genes, announced today that it has initiated studies to expand the number of pathogens detected on the FDA-cleared T2Bacteria® Panel to include the detection of Acinetobacter baumannii.…

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Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA

10/17/2022

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

09/28/2022

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

09/21/2022

– 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

08/17/2022

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

08/05/2022

– 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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VERACYTE ANNOUNCES NEW CLINICAL VALIDATION DATA FOR RNA SEQUENCING-BASED AFIRMA MEDULLARY THYROID CANCER CLASSIFIER PUBLISHED IN THYROID

07/29/2022

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

07/14/2022

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

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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