Hosted by Drug Discovery World and supported by Bio-Techne this webinar will explore the advances and challenges taking place within the cell and gene therapy field. You will hear from Hans Schambye CEO at BOOST Pharma, Lucas Arruda, CSO at CuraCell, and Brandon Cieniewicz, Director, Research & Development at CERo… Read More
Despite their challenging nature, cell & gene therapies (CGTs) are becoming ever more successful and accessible one-time treatments for haematological malignancies and genetic diseases, while researchers have recently made significant strides forward in their use to treat other cancers.
Hosted by Drug Discovery World and supported by Bio-Techne, this webinar will explore the advances and challenges taking place within the cell and gene therapy field. You will hear from Hans Schambye CEO at BOOST Pharma, Lucas Arruda, CSO at CuraCell, and Brandon Cieniewicz, Director, Research & Development at CERo… Read More
Skylark Bio has revealed it is expanding its pipeline with SKY-AS, a differentiated gene therapy candidate designed to restore UBE3A expression in individuals with Angelman syndrome (AS). SKY-AS represents the company’s first central nervous system (CNS) development programme and expands Skylark’s portfolio of precision, locally delivered… Read More
A new study has presented an innovative electromagnetic field (EMF)-responsive gene switch that enables fully reversible, safe, and precise control over gene expression. “Gene switches” hold significant potential for understanding gene expression and for therapeutic applications, particularly for the non-invasive treatment or management of genetic disorders. … Read More
Genespire has commenced patient enrolment in an observational study, InforMMA, in children with methylmalonic acidemia (MMA) caused by mutations in the MMUT gene. The study is designed to follow patients aged up to 16 years who live with severe MMA, with the aim of better characterising disease… Read More
The US Food and Drug Administration (FDA) granted accelerated approval for Ultragenyx Pharmaceutical’s Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and paediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). Dr Eric Crombez, Chief Medical Officer at Ultragenyx, commented: “As our… Read More
Skylark Bio has dosed the first patient in the SONIX Phase I/II clinical trial evaluating SKY-GJB2, its investigational inner-ear-targeted gene therapy for GJB2-mediated hearing loss. “Dosing the first patient in SONIX brings us one step closer to a future where children born with genetic hearing loss have new… Read More
Hosted by Drug Discovery World and supported by BioIVT, this webinar examines the advances, challenges and opportunities that exist in neuroscience drug discovery and development. You will hear from a number of experts who will discuss how both technology and scientific approaches are helping enable the development of… Read More
Circio Holding and Avenue Biosciences have entered a research collaboration to combine their synergistic technologies to improve long-term expression of secreted proteins, relevant for treatment of a broad range of diseases. Secreting proteins from cells into surrounding tissue, or into circulation, opens new treatment opportunities beyond classical gene therapy for… Read More
Hosted by Drug Discovery World and supported by BioIVT, this webinar examines the advances, challenges and opportunities that exist in neuroscience drug discovery and development. You will hear from a number of experts who will discuss how both technology and scientific approaches are helping enable the development of… Read More
Biotechnology company EpilepsyGTx has partnered with ClearPoint Neuro on a project that will support the targeted delivery of the biotech’s gene therapy candidate, EPY201, for focal refractory epilepsy (FRE). The agreement will see EpilepsyGTx use the ClearPoint SmartFlow Cannula and the ClearPoint Neuro Navigation system to administer EPY201 in its upcoming first-in-human Phase I/IIa clinical trial for FRE. ClearPoint’s MRI-guided stereotactic… Read More
Hosted by Drug Discovery World and supported by BioIVT, this webinar examines the advances, challenges and opportunities that exist in neuroscience drug discovery and development. You will hear from a number of experts who will discuss how both technology and scientific approaches are helping enable the development of… Read More
Genespire, in collaboration with researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), has revealed preclinical data supporting the potential of its liver-directed immune-shielded lentiviral gene therapy approach to treat metabolic disorder methylmalonic acidemia (MMA). The findings, published in the Journal of Hepatology, show that a single systemic administration of a lentiviral vector… Read More
Hosted by Drug Discovery World and supported by BioIVT, this webinar examines the advances, challenges and opportunities that exist in neuroscience drug discovery and development. You will hear from a number of experts who will discuss how both technology and scientific approaches are helping enable the development of… Read More
Hosted by Drug Discovery World and supported by BioIVT, this webinar examines the advances, challenges and opportunities that exist in neuroscience drug discovery and development. You will hear from a number of experts who will discuss how both technology and scientific approaches are helping enable the development of new therapies… Read More
