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Trend Chart On Innovative Biotherapies March 28th

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 13th, 2020

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

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…..

New R&D agreement to expand Ziopharm’s TCR-T Program

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

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…..