Biodistribution & Shedding

The biodistribution data, coupled with other preclinical safety endpoints such as clinical pathology and histopathology, help determine whether vector presence or gene expression correlates with any tissue-specific detrimental effects in animals.”*

Guidelines
FDA. EMA, ICH, MIQE

The MIQE guidelines encourage better experimental practice, allowing more reliable and unequivocal interpretation of qPCR results Bustin et. al. 2009

biodistribution throughout body

Species: Dog, Minipig, Rabbit, Rat, Sheep, Guinea Pig

Excretions: Saliva, Feces, Nasal, Tears, Urine

Organs: Kidney, Brain, Heart, Spleen, Gonads, Liver, Injection Site(s), Other

Treatment: AAV, Lentiviral, Plasmid, RNA, Oligonucleotide, Other

DNA

Vector Presence
ddPCR, qPCR

“Tissue/biological fluid analysis should be conducted at the molecular level, using a quantitative PCR (Q-PCR) assay to determine the number of vector copies per microgram of genomic DNA at specified time points  post-vector administration.”*

RNA

Gene Expression
ddPCR, RT-qPCR

“Quantitation of transgene expression using methods such as a quantitative Reverse Transcriptase PCR (RT-PCR) assay can help determine… correlation of the kinetics of transgene expression with desired activity or undesired toxicity profile.”*

Protein

Protein Expression
ELISA, Western Blot

“When determining the safety of an expressed transgene and/or translated protein, sponsors should consider the following: a) local versus systemic expression; b) level and duration of expression; and c) acute versus chronic effects.”*

Our Approach

In-life Phase

Accreditations: FDA Inspected, EMA Certified, ISO Certified, AAALAC Accredited, DEA Licensed, OLAW Assured, USDA Registered

*FDA Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products, November 2013