Gene Therapy
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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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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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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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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