Krystal Biotech, Inc. (Nasdaq:KRYS), a gene therapy company, announced today the ground breaking of the second commercial gene therapy facility in Findlay Township, Pennsylvania. The Findlay-based Current Good Manufacturing Practice (cGMP) facility, named ASTRA, will have the capacity to produce commercial gene therapy medicines to treat patients suffering from debilitating rare diseases.
Chimeric antigen receptor (CAR)–T cells have been clinically effective in killing certain hematological malignancies, but achieving long-term patient responses for solid tumors remains a challenge. Reinhard et al. describe a two-part “CARVac” strategy to overcome poor CAR-T cell stimulation and responses in vivo. They introduce the tight junction protein claudin 6 (CLDN6) as a new CAR-T cell target and designed a nanoparticulate RNA vaccine encoding a chimeric receptor directed toward CLDN6. This lipoplex RNA vaccine promotes CLDN6 expression on the surface of dendritic cells, which in turn stimulates and enhances the efficacy of CLDN6-CAR-T cells for improved tumor therapy.
Anemocyte, an innovative Italian company working in the field of cell and gene therapies, with special focus on plasmid production and non-viral gene modification approaches, attends Phacilitate Leaders World (Miami, 21-24 January 2020), a leading event for companies, professionals and investors working in the Advanced Therapy sector. Anemocyte is a key player and the first ever Biotech Manufacturing Organization (BMO) operating in the Life Science sector: company helps CGT developers to articulate initial ideas, perform clinical trials and engage in commercial production. The BMO also develops technological platform strategies for innovative R&D, HQ and GMP processes.
The planarian flatworm is a simple animal with a mighty ability: it can regenerate itself from nearly every imaginable injury, including decapitation. Scientists have studied these worms for decades to better understand fundamental principles of natural regeneration and repair. Specifically, Petersen and Schad discovered that a gene called mob4 suppresses tissue growth in the animals. The gene, they found, works in a rather surprising way: by preventing the descendants of stem cells from producing a growth factor called Wnt, a protein released from cells to communicate across distances. The Wnt signaling pathway is known to play a role in cancer cell regeneration.
Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug designation to ARU-1801, Aruvant’s investigational therapy for the treatment of sickle cell disease.
23-29 Sep, 2019
23-26 Oct, 2019
Feb. 5-6, 2020
Mar. 2-4, 2020
San Francisco, USA
Mar 31- Apr 2 , 2020