PROCESS
VALIDATION APPROACH FOR GENE EDITING CRITICAL MATERIALS
background
The challenge
BDO's SOLUTION
The results
3
2
1
Result 3: The client and CMOs will reap long-term benefits from the process which is applicable to other materials required for manufacturing cell and gene therapy products.
3
Result 2: The process was completed in six months, enabling the client to meet its marketing application filing timeline.
2
Result 1: BDO helped the client develop and execute a risk-based and science-based approach to process validation for gene editing critical materials.
1
2
1
Solution 2
The VPPs included essential validation elements, which BDO either authored or significantly contributed to, including:
Characterization Studies
Control Strategies
Risk Assessments
Validation Plans, Protocols and Reports
2
Solution 1
BDO worked with with client and their CMOs to develop a Validation Project Plan (VPP) specific to each of their processes.
1
Challenge
Both the gene-engineering process and the manufacturing process for two critical gene editing materials, Cas9 and gRNA, needed validating.
The client’s contract manufacturing organizations had little experience with process validation and required support from both
a technical and
manpower perspective.
Company Background
A biotechnology company was preparing to file a marketing application for a patient-specific cell therapy product and needed to demonstrate their manufacturing process was robust and reproducible
before filing.
Title -
Up to 65 Characters
background
The challenge
BDO's SOLUTION
The results
3
2
1
Result 3
The client and CMOs will reap long-term benefits from the process which is applicable to other materials required for manufacturing cell and gene therapy products.
3
Result 2
The process was completed in six months, enabling the client to meet its marketing application filing timeline.
2
Result 1
BDO helped the client develop and execute a risk-based and science-based approach to process validation for gene editing critical materials.
1
RESULTS
2
1
Solution 2
The VPPs included essential validation elements, which BDO either authored or significantly contributed to, including:
Control Strategies
Characterization Studies
Risk Assessments
Validation Plans, Protocols and Reports
2
Solution 1
BDO worked with with client and their CMOs to develop a Validation Project Plan (VPP) specific to each of their processes.
1
BDO's SOLUTION
BDO's SOLUTION
Both the gene-engineering process and the manufacturing process for two critical gene editing materials, Cas and gRNA, needed validating. The client’s contract manufacturing organizations had little experience withprocess validation and required support from both a technical and manpower perspective.
challenge
A biotechnology company was preparing to file a marketing application for a patient-specific cell therapy product and needed to demonstrate their manufacturing process was robust and reproducible
before filing.
Background