Worldwide Clinical Trials
NOVEMBER 27, 2023
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.
Worldwide Clinical Trials
OCTOBER 24, 2023
Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”
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Drug Discovery World
MAY 2, 2024
Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. Perhaps most significantly, Casgevy (or exa-cel) from Vertex Pharmaceuticals and CRISPR Therapeutics, was approved to treat sickle cell disease and became the first gene therapy that uses CRISPR gene editing.
Advarra
NOVEMBER 29, 2022
The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.
Drug Discovery World
AUGUST 11, 2023
T-MAXIMUM Biotech hopes to progress multiple products to the Phase II clinical research stage and deliver products to market within the next five years. Dr Shang Xiaoyun, CEO of T-MAXIMUM, said: “Gene editing reagents are crucial raw materials for the quality and efficacy of our products.
Drug Discovery World
MARCH 10, 2023
Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). News round-up for 6-10 March by DDW Digital Content Editor Diana Spencer.
WCG Clinical
NOVEMBER 17, 2023
It also establishes the role of the Institutional Biosafety Committee (IBC) in providing local oversight of such research. At the time this change was implemented, methods used to deliver rsNA to cells or research participants typically involved the use of viral vectors that were subject to the NIH Guidelines.
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