In Vivo Gene Articles & Analysis
15 news found
It describes the potential of VG801 for the functional transfer of the ABCA4 gene in a Stargardt disease mouse model. Because the ABCA4 gene exceeds the packaging capacity of AAV vectors, the gene was transferred using the REVeRT technology. ...
Agreement strengthens Bayer’s new cell and gene therapy platform further Supports Mammoth’s vision of unlocking the full potential of novel CRISPR systems Mammoth to receive upfront payment of USD 40 million, and potential future milestone payments of more than one billion USD upon successful achievement Bayer AG and Mammoth Biosciences, Inc., which is ...
(NYSE: CRL) and ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in vivo gene replacement, gene editing and allogeneic cell therapies, today announced they have agreed to manufacture ASC618, a second-generation gene therapy for hemophilia A. ...
ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies has joined as a full partner the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) including the National Institutes ...
“The MSUD gene therapy development leverages the combined AAV gene therapy expertise at UMass Chan Medical School, such as AAV vector design, rodent and large animal modeling, large-scale vector production, and in vivo pre-clinical testing,” Drs. ...
” Interim Results From an Ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I) The oral presentation includes efficacy and safety data (cut-off March 9, 2022) at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for ...
Podium presentation highlights significant transduction and transgene expression in pharmacokinetic studies in mice, non-human primates and a humanized liver model Poster presentation focuses on pharmacology, toxicology and safety studies Poster presentation focuses on the development of a novel transduction assay to evaluate in-vitro relative Infectivity ASC Therapeutics, a privately held ...
ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic, metabolic, and other rare diseases has received from the United States (U.S.) ...
Zisoulis brings a wealth of experience in drug development in the gene therapy space. Most recently, he was Vice President of Drug Discovery at Locanabio, where he helped develop new RNA-targeting therapeutics. At Mammoth, he will lead the discovery and development of next-generation in vivo gene-editing therapeutics using Mammoth’s ...
(Nasdaq: SGMO), a genomic medicines company, today announced that Sanofi will be transitioning its rights and obligations related to SAR445136, a zinc finger nuclease gene-edited cell therapy candidate in development by Sangamo and Sanofi for the treatment of sickle cell disease (SCD), back to Sangamo over the first half of 2022. ...
NEW YORK, December 17, 2021 – Brooklyn ImmunoTherapeutics, Inc. (Nasdaq: BTX) (“Brooklyn”), a biopharmaceutical company focused on exploring the role that cytokine and gene editing/cell therapy can have in treating patients with cancer, blood disorders, and monogenic diseases, today announces that Brooklyn has been added to the ICE Biotechnology Index (NYSE:ICEBIO) following the ...
(Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary proof-of-concept clinical data from the Phase 1/2 PRECIZN-1 study of SAR445136, a zinc finger nuclease gene-edited cell therapy candidate in development with Sanofi for the treatment of sickle cell disease (SCD). ...
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Mammoth Biosciences, a biotech company building the next generation of CRISPR products to cure and detect disease, today announced a new partnership to develop in vivo gene-editing therapies for two genetic diseases using Mammoth’s next-generation CRISPR systems. “We believe our novel ultra-small ...
Our mission is to build the ideal capsid and solve the challenges of vivo gene delivery. We call ourselves AAViators and are engaged in Collective Innovation aswe aim to maximize our impact on patients through the development of groundbreaking technologies. ...
Enables expansion of novel CRISPR platform, including proprietary ultra-small Cas14 and Cas? CRISPR systems for in vivo gene-editing therapeutics Makes key appointment of biotech veteran Gary Loeb as general counsel Mammoth Biosciences, the biotech company leveraging the diversity of life to build the next generation of CRISPR products to cure and detect ...