Reference Materials for Adeno-Associated Viruses

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1 Reference Materials for Adeno-Associated Viruses : Recommendations of the Quality Control Sub-Committee John Chiorini (NIH-NIDCR) Paul Husak (Cell Genesys) Gabrielle Kroener-Lux (Progen) James Marich (Cell Genesys) Scott McPhee (UMDNJ-RWJMS) Marie Printz (Ceregene) Jürg Sommer (Avigen) March 12, 2003 Quality Plan Requirements: Develop and qualify assays Issue SOPs Develop and approve test plan Set preliminary specifications Identify and coordinate testing sites Aggregate, review and apply statistical analysis to data Coordinate reference characterization, e.g. stability, sequencing Raw materials testing, characterization of intermediates

2 Quality Control Sub-Committee Focus: Reference material testing and characterization alone Group did not discuss intermediate testing or characterization, to be discussed in context of Working group Intermediate testing, characterization contingent upon: Manufacturing method chosen Source of materials Material s previous characterization by donating organization Define Testing for label claim analyses; Characterization defined as all other analyses Considerations for Reference Testing Assay(s): Relevancy to intended purpose of reference material, i.e., scientific validity Robustness (e.g., matrix independent) Ease of performance Technology or instrumentation readily available at testing sites Most laboratory-skilled personnel capable of performing assay Cost-effective General application to product class

3 Choice of Reference Testing Assays: Intended purpose of reference material: Vector Safety Reference functions to allow comparability of assay results and potential to normalize clinical dose across product class Normalized dose allows for safety assessment of acute or long-term toxicity, antibody response to vector Relevant testing assays chosen: Strength, defined as physical particle titer Activity, defined as infectious particle titer Both strength and activity comprise label claim analyses Reference Test: Strength Methods considered for particle titer: Total particles AE-HPLC Optical density measurement (OD260) Capsid ELISA EM Genome-containing particles Quantitative/conventional PCR Dot blot Pico-green assay Sub-Committee unable to reach a consensus for recommendation

4 Reference Test: Strength Issues with total physical particle assays: Advantage-measures total amount of particles dosed to patient AE-HPLC and EM require specialized instrumentation, variability of EM Optical density (OD260) method affected by purification processderived residual impurities Contingent on manufacturing method Capsid-ELISA quantitation depends on internal reference standard Issues with genome-containing particle assays: Advantage-currently used method for patient dosing Variability of dot blot method Dot blot, quantitative/conventional PCR requires internal reference standard for quantitation, differences in transgenes between reference and clinical vector necessitate different assays, normalization difficult Reference Test: Activity Methods considered for activity: Infectious titer (transgene-independent) TCID50 on rep/cap cell line Infectious center assay Transducing titer (transgene-dependent) Focus-forming assay FACS Functional titer (transgene-dependent expression) Luciferase assay Infectious titer by TCID50 on rep/cap cell line recommended

5 Reference Test: Activity Issues with Infectious titer assay: Method uses limiting serial dilutions of vector on rep/capcontaining cell line in presence or absence of helper virus in multi-well format, vector replication quantitated by qpcr, Spearman-Karber calculation of 50% infectious dose Advantage-quantitation independent of transgene Modifications to method include use of particle diffusioncoefficient in titer calculation Requirement for qpcr instrument, access to instrument may be limited, possible to out-source end-point analysis alone to service laboratory Assay will require donation of rep/cap-containing cell line and helper adenovirus (wild type, dl309) stock Reference Testing Specifications and Plan Physical particle titer proposed at >1E12 vg/ml Proposal to limit acceptable amount of empty capsids Infectious particle titer proposed at >1E10 inf. part./ml Proposal to limit ratio physical/infectious particle to 100:1 Compendial methods can be used for comparative assessment Minimum number of testing sites and replicates to be determined, contingent upon empirical determination of individual assay variance, confidence bounds required, manufacturing scale Input from statistician on design required early in planning stage

6 Reference Characterization Recommendation that complete safety panel characterization be performed Extent of characterization contingent upon manufacturing scale and material availability, may require prioritization Stage of testing dependent on manufacturing process and any previous characterization of raw materials or intermediates Characterization analyses include Identity, Contaminants (Purity), Residuals (Purity), Physical parameters, Stability Reference Characterization Identity Genotype Method: Sequencing Serotype Method: Western blot Specification: Matches predicted serotype Contaminant (Purity) rcaav (vector as reference) Method: Biological amplification assay Specification: NMT 1 rcaav in 1E9 vg (or vp) rca (production process dependent) Method: Biological amplification assay Specification: NMT 1 rca in 1E9 vg (or vp)

7 Contaminant (Purity) Sterility Method: USP, membrane filtration Specification: No growth detected Mycoplasma Method: 21 CFR Specification: No growth detected Endotoxin Method: LAL Specification: NMT 5 EU/mL Adventitious viruses Method: In vitro assay (in vivo assay not performed) Specification: None detected Contaminant (Purity) General Safety Not performed Virus Pathogens Porcine viruses Method: 9CFR, In vitro assay Specification: None detected Bovine viruses Method: 9CFR, In vitro assay Specification: None detected Human Viruses (EBV, B19, HCMV, HHV 6&7, HTLV-I&II, HIV1&2, HBV, HCV) Method: PCR Specification: None detected

8 Residuals (Purity) Adenovirus protein Method: Western blot for hexon Host cell protein Method: ELISA or Western (if available) Host cell DNA Method: Slot blot or PCR Serum-derived BSA Method: ELISA Residuals (Purity) Process impurities, detergents Method: TBD Physical Parameters Appearance (frozen, liquid) Method: Visual inspection Weight Method: Gravimetric ph Method: Potentiometric

9 Physical Parameters Particle-size distribution, aggregation Method: Dynamic-laser light scattering or particle-sizer Stability Long-term (-80 C) stability Timepoints, 0, 6, 12, 18, 24, 36, 48, and 60 months Method: Physical and infectious (TCID50) particle titer assays, and TBD Shipping configuration stability Method: Physical and infectious (TCID50) particle titer assays, and TBD Summary Multi-site testing for strength (physical particle titer) and activity (infectious particle titer) recommended Extensive characterization analysis recommended, including longterm stability study

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