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Biotech startup Ray raises $100M for vision-restoring gene therapies

Bio Pharma Dive

Founded in early 2021, the company is using optogenetics to design treatments that could work across disease genotypes and in the later stages of vision loss.

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Gender and age differences found in gene expression related to circadian clock

Medical Xpress

A trio of bioengineering scientists at Ecole Polytechnique Fédérale de Lausanne has found age and gender differences in gene expression related to the circadian clock. For their paper published in the journal Science, Lorenzo Talamanca, Cédric Gobet and Felix Naef analyzed data obtained for the Genotype-Tissue Expression project.

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betibeglogene autotemcel (beti-cel) One-Time Gene Therapy for ?-thalassemia Continues to Demonstrate Durable Efficacy Across Pediatric and Adult Patient Populations and All Genotypes in Data Presented at EHA2021 Virtual

BioTech 365

betibeglogene autotemcel (beti-cel) One-Time Gene Therapy for ?-thalassemia thalassemia Continues to Demonstrate Durable Efficacy Across Pediatric and Adult Patient Populations and All Genotypes in Data Presented at EHA2021 Virtual betibeglogene autotemcel (beti-cel) One-Time Gene Therapy for ?-thalassemia

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Long-Term Data for bluebird bio’s betibeglogene autotemcel (beti-cel) Gene Therapy Show Patients Across Ages and ?-thalassemia Genotypes Achieve Transfusion Independence and Remain Free from Transfusions Up to Six Years Presented at 62nd ASH Meeting

BioTech 365

Long-Term Data for bluebird bio’s betibeglogene autotemcel (beti-cel) Gene Therapy Show Patients Across Ages and ?-thalassemia

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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New approach successfully traces genomic variants back to genetic disorders

Medical Xpress

National Institutes of Health researchers have published an assessment of 13 studies that took a genotype-first approach to patient care. This approach contrasts with the typical phenotype-first approach to clinical research, which starts with clinical findings. The study was published in the American Journal of Human Genetics.

Genome 75
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US FDA approves Vertex’s Orkambi for cystic fibrosis in children

Pharmaceutical Technology

The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It enrolled 46 children aged one to under two years with the F/F genotype. According to the findings, Orkambi was found to be well tolerated.