Advanced Therapy Medicinal Products (ATMP) Flashcards
(30 cards)
What are ATMPs?
ATMPs are biological medicines classified into gene therapy, somatic cell therapy, and tissue engineered products.
What is Gene Therapy?
Gene therapy modifies DNA to treat or cure diseases.
What is Somatic Cell Therapy (SCT)?
SCT involves manipulating or using cells for different functions.
What are Tissue Engineered Products (TEP)?
TEPs regenerate, repair, or replace tissues and may contain additional substances.
What are the sources of ATMPs?
Sources include autologous (self), allogeneic (donor), xenogeneic (animal), and combined products (with devices).
What is the pharmacy’s role in ATMPs?
The pharmacy is responsible for governance, quality assurance, supply, training, and collaboration with the Human Tissue Authority.
What are the steps in the pharmacy process for initiating Gene Therapy?
The steps include protocol, risk assessment, technical feasibility assessment, Genetic Modification Safety Committee approval, confirmation of class and containment, and SOPs.
What are the routes for Gene Therapy administration?
Routes include inhalation, oral, intramuscular injection, subcutaneous injection, and intranodular injection for in vivo; and ex vivo involves modifying cells outside the body.
What is the aim of Gene Therapy?
The aim is to replace faulty genes, produce beneficial proteins, and correct mutations.
What vectors are used in Gene Therapy?
Vectors include plasmids, retroviruses, adenoviruses, adeno-associated vectors, vaccinia/fowlpox vectors, poxviruses, lentiviruses, and herpes simplex.
What are the risks associated with viral vectors in Gene Therapy?
Risks include infecting healthy cells, incorrect gene insertion, immune reactions, and insertional mutagenesis.
What are the storage requirements for ATMPs?
ATMPs must be stored in -80°C freezers or vapor-phase nitrogen at -150°C.
What handling precautions are necessary for ATMPs?
Handling requires risk assessment, PPE, and adherence to COSHH category 2 biohazards.
What are the key regulatory bodies for ATMPs?
Key bodies include MHRA, HSE, and HTA.
What is the role of the Genetic Modification Safety Committee (GMSC)?
The GMSC assesses risks to human health and the environment before any contained use of GMOs.
What are the waste management requirements for Class 2 GMOs?
Contaminated waste must be inactivated using validated methods such as disinfection, incineration, or autoclaving.
What is required for staff training in Gene Therapy?
Staff must be trained on risks, have the option not to participate, and should not be pregnant, breastfeeding, or immunosuppressed.
What is the process for CAR-T Cell Therapy?
The process involves harvesting patient T-cells, engineering them with CAR receptors, expanding, and infusing modified cells back.
What are examples of ATMPs?
Examples include Talimogene Laherparepvec for melanoma, Voretigene Neparvovec for inherited blindness, and Strimvelis for ADA-SCID.
What are the risks associated with CAR-T Cell Therapy?
Risks include cytokine release syndrome and neurotoxicity.
What are the advantages of retroviruses?
Well characterised
Efficient gene transfer
Long-term expression of therapeutic gene
