New members sought for the Advanced Therapy Medicinal Products Working Party
Date published: 18 April 2024
The British Pharmacopoeia Commission is currently looking to appoint new professional members for two new task groups focussing on providing non-mandatory best practice guidance on the topics of “Encapsidated DNA characterisation” and “Primary protein sequence modifications of encapsidated DNA”. Interested individuals should satisfy the following criteria:
1. For Encapsidated DNA characterisation
Essential criteria
- Expertise in conducting analytics of AAV vectors, including development, validation, and routine analysis, to ensure quality and consistency.
- Ability to understand the type of DNA present within the vector particles, including packaged DNA complementary to the delivery system, and assess its impact on factors such as stability, release kinetics, and targeting efficiency.
- Expertise in implementing control strategy requirements for determining the presence and integrity/fragmentation of encapsidated DNA within AAV capsids, utilising various methodologies including NAT-based techniques, single-cell sequencing, recombination prediction, and next-generation sequencing.
Desired criteria
- Expertise in conducting analytics of AAV vectors, including development, validation, and routine analysis, to ensure quality and consistency.
- Ability to identify and evaluate mutations, deletions, or insertions within vectors and their impact on product safety and efficacy.
- Proficiency in designing vectors to reduce immunogenicity and enhance delivery efficiency.
- Experience with techniques and processes for increasing DNA fragment concentration or purity within samples.
- Familiarity with techniques for collecting distinct fractions or subsets for further analysis using PCR, NGS, or Charged Detection Mass Spec.
- Experience of using AI for in silico analysis of encapsidated DNA.
- Have a good understanding of existing guidelines and best practices related to DNA characterisation in a regulatory environment.
2. For Primary protein sequence modifications of encapsidated DNA:
Essential criteria
- Experience with one or more of the following techniques:
a) Mass spectrometry and validation for AAV analysis.
b) Gel electrophoresis, CESDS, and ICIEF for charged variant analysis.
c) Forced degradation studies and stability assessment of AAV vectors.
d) Protein sequence variant analysis for functional evaluation.
e) Multi-attribute monitoring methods for quality control. - Experience in investigating post-translational modifications and their impact on protein function and vector performance.
- Proven expertise in studying protein and vector stability, with a focus on understanding molecular interactions and cellular processes..
- Familiarity with techniques and methods for confirming protein terminal regions.
Desired criteria
- Knowledge of serotype-specific characteristics and their implications for vector optimisation.
- Experience preparing in-house standards.
- Have a good understanding of existing guidelines and best practices related to primary protein sequence modifications in a regulatory environment.
If you’re interested in becoming a member, please send an email with the subject title “Encapsidated DNA characterisation” or “Primary protein sequence modifications of encapsidated DNA” with your CV attached to BiolStandards@mhra.gov.uk
The closing date for expression of interest is midday on Friday 17 May 2024
You can also find out more about the ATMP guidance currently available