Senior Principal Scientist Bristol Myers Squibb Princeton
Adeno-associated virus (AAV) gene therapy has emerged as a promising therapeutic option for treating various genetic disorders, including cardiac diseases. However, the success of AAV gene therapy relies heavily on several crucial factors, including the appropriate selection of the AAV serotype, the gene expressed, the delivery site, etc.
The proposed strategy begins with a comprehensive risk assessment, considering various factors such as AAV serotype tropism, transgene function, and delivery site, etc. Based on this assessment, a tailored bioanalytical strategy was developed, which includes pre-enrollment criteria for patients based on known AAV serotype reactivity and other relevant factors.
In conclusion, a risk-based bioanalytical strategy is necessary to ensure the safe and effective application of AAV gene therapy for cardiac diseases. The proposed strategy takes into account several critical factors, and a thorough assessment of these factors is necessary to develop a tailored bioanalytical strategy that can lead to successful outcomes.
Learning Objectives:
Upon completion, participant will be able to understand how to establish a successful bioanalytical strategy to ensure the safe and effective application of AAV gene therapy for cardiac diseases.
Upon completion, participant will be able to understand how to establish a successful bioanalytical strategy to ensure the safe and effective application of AAV gene therapy for cardiac diseases.
Upon completion, participant will be able to understand how to establish a successful bioanalytical strategy to ensure the safe and effective application of AAV gene therapy for cardiac diseases.
Upon completion, participant will be able to understand how to establish a successful bioanalytical strategy to ensure the safe and effective application of AAV gene therapy for cardiac diseases.
Upon completion, participant will be able to understand how to establish a successful bioanalytical strategy to ensure the safe and effective application of AAV gene therapy for cardiac diseases.