Gene Therapy

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

05/11/2022

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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PacBio transforms access to the epigenome and streamlines workflows

04/28/2022

Significant enhancements to the Sequel II/IIe platform include methylation calling in native DNA, greatly accelerated sample preparation, and support for gene therapy applications Excerpt from the Press Release: MENLO PARK, Calif., April 21, 2022 (GLOBE NEWSWIRE) — PacBio (NASDAQ: PACB), a leading developer of high-quality, highly accurate sequencing solutions, today announced the release of a transformative capability…

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Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration

04/11/2022

Announcement marks first clinical trial in humans of Ocugen’s modifier gene therapy platform Excerpt from the Press Release: MALVERN, Pa., April 01, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines, announced that the first patient has been dosed in the Phase 1/2…

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Thermo Fisher Scientific to Support National Program to Accelerate SARS-CoV-2 Variant Identification

03/29/2022

Excerpt from the Press Release: CARLSBAD, Calif.–(BUSINESS WIRE)–Thermo Fisher Scientific Inc., the world leader in serving science, today announced its collaboration with the National Institutes of Health (NIH) Rapid Acceleration of Diagnostics (RADx) Initiative, Helix, and Rosalind aimed at developing a new genotyping method for SARS-CoV-2 that could speed up the identification of variants as…

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