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  • OCUGEN GRANTED FDA ORPHAN DRUG DESIGNATION FOR OCU400 GENE THERAPY FOR THE TREATMENT OF CEP290 MUTATION ASSOCIATED RETINAL DISEASE

    Ocugen, a biopharmaceutical company focused on discovering, developing and commercializing of innovative therapies that address rare and underserved eye diseases, has announced the U.S. FDA granted the second orphan drug designation for OCU400, Ocugen’s novel gene therapy, for the treatment of CEP290 mutation associated retinal diseases; mutations in CEP290 have been associated with different diseases including Leber Congenital Amaurosis, Bardet-Biedl syndrome, Joubert syndrome, Senior-Loken syndrome and Meckel-Grüber syndrome

    Date: Sep 18, 2019
  • ASKBIO ACQUIRES ASSETS FROM ROVERMED BIOSCIENCES, ADDS NANOTECHNOLOGY PRECISION TO DELIVERY OF AAV GENE THERAPEUTICS

    Asklepios BioPharmaceutical, a fully integrated Adeno-Associated Virus gene therapy platform company focused on providing curative therapeutics for genetic disorders, has acquired the technology assets of RoverMed BioSciences. RoverMed developed nanotechnology cargo delivery of therapeutics into the nucleus of diseased cells without affecting healthy cells. Under the terms of the agreement, AskBio will integrate the company's technology and assume all assets of RoverMed

    Date: Sep 17, 2019
  • GENE EDITING ENABLES RESEARCHERS TO CORRECT MUTATION IN MUSCLE STEM CELLS IN DMD MODEL

    Researchers at the University of Missouri School of Medicine have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation, that causes a deficiency of dystrophin, responsible for the disorder. With more study, the researchers hope this cell-targeted CRISPR approach may one day lead to longlasting therapies for children with DMD

    Date: Sep 17, 2019
  • TRANSPLANTED BRAIN STEM CELLS SURVIVE WITHOUT ANTI-REJECTION DRUGS IN MICE

    In experiments with mice, researchers have developed a way to successfully transplant certain protective brain cells without the need for lifelong anti-rejection drugs; the idea behind the experiments, developed by Johns Hopkins Medicine, was to exploit the natural tendencies of costimulatory signals to train the immune system to accept transplanted cells as self-permanently. To do that, they used two antibodies, CTLA4-Ig and anti-CD154, which keep T cells from beginning an attack when encountering foreign particles by binding to the T-cell surface. The study was published in the journal Brain

    Date: Sep 17, 2019
  • INDUCTION RADIATION THERAPY BEFORE CAR-T REDUCES TREATMENT-RELATED TOXICITIES

    A new study, presented at the American Society of Radiation Oncology Annual Meeting, shows that patients who received radiation therapy before chimeric antigen receptor T-cell therapy for relapsed or refractory non-Hodgkin lymphoma were less likely to have severe treatment-related toxicities; none of the patients who received induction radiation therapy less than 30 days before CAR T-cell infusion experienced high-grade cytokine release syndrome or neurotoxicity, two of the common treatment-related toxicities associated with CAR T-cell therapy

    Date: Sep 17, 2019

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