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Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.
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was established in 1999 in the process of privatisation of the State Research Institute for Pharmacy and Biochemistry in the Czech Republic. Before the inception of this institute, the facility was oriented towards pre-clinical toxicology and breeding of the laboratory animals. Dr Ivana Surovà: Biotest s.r.o.
XTALKS WEBINAR: Enhance Early Biomarker Discovery: Translating In Vitro and In Vivo Multi-Omics Oncology Data Live and On-Demand: Thursday, January 16, 2025, at 11am EST (4pm GMT/UK) Register for this free webinar to learn how to harness the full potential of multi-omics data and advanced patient models to drive early biomarker discovery.
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MDC, the independent national centre for innovation in drug discovery, has a major focus in pre-clinical imaging and will now offer the design and production of radiotracers alongside its cutting-edge imaging and industrial drug discovery expertise based at nearby Alderley Park. ” Find out more about MDCs PET Imaging expertise here.
For example, there is no need for in vivo animal or in vitro pre-clinical or non-clinical studies. The area of digital therapeutics is growing rapidly, and in this piece Ben Hargreaves examines why this is the case, how broadly these therapies can be applied and why investment in the area is mounting. This equated to $21.3
Register for this free webinar to learn why the agile clinical trial model is a great fit for many rare disease categories, how to navigate the regulatory landscape for agile clinical trials in rare diseases, how to design studies and more. The cells are prepared outside of the body ( ex vivo ) and then infused into the patient.
Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases.
Drawing upon 50 years of expertise in clinicalresearch, biopharmaceuticals, biotechnology and drug discovery, Dr. Van Kampen excels as the chief executive officer of The Van Kampen Group, which assists other companies in research and liaisons with government and industry regarding licensing products for commercial use.
Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Such challenges highlight a significant unmet need and call for further advancements in the field.
EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. The FDA’s clearance for initiation for our EDIT-301 clinical trial is an exciting moment for us and the patients we hope to serve. “The Clinical trial materials are being manufactured by Editas Medicine.
a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant in Europe in its global Phase 3 clinical trial for HD. NAARDEN, Netherlands–( BUSINESS WIRE )– Prilenia Therapeutics B.V. ,
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Combined with real-world study (RWS) as well as medical theories and clinical cases, the monograph concludes, from the perspective of medical treatment, the experience of doctors in various regions of China in exploring the application of hydrogen-oxygen adjuvant therapy in mild, moderate, severe and critical patients at the baseline level.
AOP anticipates new opportunities in Austria as a hub for research and business. Priothera will use the funds to progress the clinical development of mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogeneic hematopoietic stem cell transplantation (HSCT) for treating AML.
WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation. Lin has made several transitions throughout her career, from being a clinician at an academic center, to academic research, to heading business development in industry. Key Moments.
Pharmaceutical research has long relied on non-human primate models for early-stage discoveries, but their use continues to cause controversy. Globally, non-human primates make up a relatively small percentage of the animals used in research. This was part of an investigation into an international primate smuggling ring.
Francisco “Paco” Arredondo, MD, MPH, FACOG, recently Chief Medical Officer of the United States largest network of fertility centers, overseeing more than 50 fertility specialists, and currently a partner in a joint venture developing INVOcell clinics in Mexico.
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SARASOTA, Fla. ,
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