In Vivo Therapy
June 29, 2020 9:47 amMeaning “within the living”, this type of therapy is administered directly into the patient, targeting the cells and editing the... View Article
Meaning “within the living”, this type of therapy is administered directly into the patient, targeting the cells and editing the... View Article
In a clinical trial setting, informed consent means that patients considering volunteering to enroll receive information and consent to the... View Article
Insertion of a new DNA sequence into the genome to manufacture a desired protein using a gene editing technology, such... View Article
In a research or clinical setting, investigational means that the drug has not been approved or authorized for use in... View Article
Also known as an IND or IND application. Companies like Intellia submit an IND to begin testing of investigational therapies... View Article
Reduction of protein, with the goal of achieving a therapeutic effect, as a result of a gene or genome editing... View Article
Inactivation/deletion of a DNA sequence using a gene editing technology, such as the CRISPR/Cas9 system. This is the type of... View Article
Also known as LNP. LNPs are fat-based molecules that are the basis of Intellia’s CRISPR/Cas9 delivery platform. In Intellia’s experimental... View Article
Also known as blood cancers or hematological malignancies. Cancers like leukemia, lymphoma and myeloma that originate in blood, bone marrow... View Article
Also known as mRNA. mRNA, which encodes the Cas9 protein, is one of the components comprising Intellia’s CRISPR/Cas9 LNP delivery... View Article