FEATURE STORY ● Impact of gene therapy for canine monogenic diseases on the progress of preclinical studies GENE THERAPY ● Permanent inactivation of HBV genomes by CRISPR/Cas9-mdediated non cleavage base editing DISRUPTIVE TECHNOLOGIES ● Hydroxybutyl chitosan centered biocomposites for potential curative applications ● CAR-T treatment for hematological malignancies ● Anti-CRISPRs :…..
Trend Chart on Orphans - February 15th, 2020 FEATURE STORY ● The potential of gene therapy for mucopolysaccharidosis type I GENE THERAPY ● Repair of retinal degeneration following ex vivo Minicircle DNA gene therapy and transplantation of corrected photoreceptor progenitors RARE DISEASES ● Genetics and other omics in pediatric pulmonary…..
Trend Chart on Innovative Biotherapies - January 15th, 2020 FEATURE STORY ● The National Gene Vector Biorepository. Eleven years of providing resources to the gene therapy community GENE THERAPY ● Recommendations for the development of cell-based anti-viral vector neutralizing antibody assays DISRUPTIVE TECHNOLOGIES ● A small molecule-controlled Cas9 repressible system…..
Morpholino technology could allow control of gene therapy doses Scientists at Scripps Research, led by principal investigator Michael Farzan, have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing. The feat, reported in Nature Biotechnology, offers gene therapy designers what may…..
FEATURE STORY ● Artificial intelligence approach to create AAV capsids for gene therapies DISRUPTIVE TECHNOLOGIES ● Nanoparticles as carriers for drug delivery of macromolecules across the blood-brain barrier CLINICAL TRIALS - DATAS ● Libella gene therapeutics to run a patient paid trial of telomerase gene therapy M&A - AGREEMENTS ●…..
Two Chinese companies invest in Vigeneron, a German firm developing ophthalmological gene therapy programs The German gene therapy company Vigeneron has closed a series A financing round led by WuXi AppTec, a Chinese provider of R&D and manufacturing services and Sequoia Capital China, a venture capital firm based in Beijing.…..
More than 580 gene therapy products are under development in biotech and pharmaceutical companies for the treatment of more than 240 diseases. More than 200 of these products are now at the clinical stage. After releasing our « Landscape in… Gene Therapy Companies » in September, we now offer you an opportunity…..
New R&D agreement to expand Ziopharm's TCR-T Program Ziopharm Oncology, an immuno-oncology company focused on developing end-to-end cost-effective solutions using its non-viral Sleeping Beauty platform for TCR and CAR T-cell therapies and immune-stimulating gene therapy, has concluded a new R&D agreement with MD Anderson Cancer Center. This agreement is related…..
Fourth FDA approval for Vertex's cystic fibrosis medicines The FDA has approved Trikafta™ (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of…..
New version of SOPHiA Whole Exome Solution to accelerate rare disease characterization SOPHiA GENETICS, a leader in Data-Driven Medicine, has announced at the American Society of Human Genetics Annual Meeting (ASHG) the release of the new version of its SOPHiA Whole Exome Solution. This advanced genomic application is reducing the…..
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