Gene Therapy
– Ciitizen real-world data platform expansion helps advance research and potentially improve outcomes for patients with pediatric epilepsy and/or developmental delay – Excerpt from the Press Release: SAN FRANCISCO, Dec. 1, 2022 /PRNewswire/ — Invitae (NYSE: NVTA), a leading medical genetics company, today announced the launch of the Rare Patient Network expanding Invitae’s Ciitizen platform to…
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Excerpt from the Press Release: CARLSBAD, Calif.–(BUSINESS WIRE)–Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, announced today that its partner Genentech, a member of the Roche Group, has launched a Phase 2a, multicenter, open-label, single arm clinical study of RG6501 (OpRegen), a…
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Summary: SickKids-led study sequences the entire genomes of more than 11,000 individuals, offering new insights into the genetics that underlie autism spectrum disorder. Excerpt from the Press Release: Researchers from The Hospital for Sick Children (SickKids) have uncovered new genes and genetic changes associated with autism spectrum disorder (ASD) in the largest autism whole genome…
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Excerpt from the Press Release: SAN DIEGO–(BUSINESS WIRE)–Escient Pharmaceuticals, a clinical-stage company advancing novel small molecule therapeutics for the potential treatment of a broad range of neurosensory-inflammatory disorders, today announced the initiation of a Phase 2 clinical study of EP547 in subjects with cholestatic pruritus. EP547 is a highly selective antagonist of MRGPRX4, a cell…
Read More 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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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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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…
Read More 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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