Maximize Efficiency and Accuracy Across your Gene Therapy Development and Manufacturing Workflow
From the viral vector characterization assays that fuel discovery to potency measurement and safety assays for QC release, our gene therapy manufacturing products bring accuracy and consistency to your results.
How We Help Viral Vector Manufacturing
Drug development and manufacturing has always been a challenging process. When the drug you are developing is a new type of treatment modality, such as a gene therapy, the challenges only increase in complexity.
At Bio-Rad, our goal is to simplify your day-to-day operations and bring clarity to your decision-making by removing complexity from your gene therapy manufacturing workflows. Through products that combine high-performance with ease-of-use and a long track record of success, we make it easy to obtain absolute quantification of viral titer, check for impurities like host cell DNA and mycoplasma contamination and evaluate viral potency for developing and deploying QC release assays. To support you in viral vector manufacturing, whether it is an adeno-associated virus (AAV), adenovirus (Ad), herpes simplex virus (HSV), or something else, Bio-Rad has proven products and a responsive and knowledgeable support team to accelerate your advanced therapeutic development.
Did you know?
Results from Phase 3 clinical trials show that one-time, pre-symptomatic administration of the gene therapy Zolgensma continued to show real-world efficacy even five years post-treatment.1 In the absence of treatment, children with SMA-1 would have a life expectancy of less than two years.2
Where We Help Drive Efficiency in Viral Vector Manufacturing
Get the speed and certainty of absolute viral titer determination using Droplet Digital™ PCR (ddPCR™).
Develop and deploy robust virus potency assays for development and QC release.
Find ready-to-use assays to detect harmful contaminants such as pathogenic microbes and host cell debris.
Industry Snapshot: Globally Approved In Vivo Gene Therapies
While gene therapies in clinical trials took a pause in the late 1990s to better understand the immunogenicity and characteristics of different viral vectors,3 the past few years has seen a resurgence of interest in this promising approach. Teams around the world have developed safer and more effective vectors and, while much of the research has investigated AAV production and manufacturing, approved treatments include a range of viral vectors (Table 1).
Our specialists closely follow the latest developments in the field and are excited to support your efforts in expanding the list of approved gene therapy treatments.
Table 1. Globally Approved Gene Therapies.
|Viral Vector Type
|Approval Year and Organization
|Talimogene laherparepvec/IMLYGIC® (Amgen)
|Local recurrent unresectable cutaneous, subcutaneous, and nodal melanoma after initial surgery
|2015 (USFDA), 2015 (EMA)
|Mx‐dnG1/Rexin‐G® (Epeius Biotechnolgies)
|Soft tissue sarcoma, osteosarcoma, and pancreatic cancer
|H101/Oncorine® (Shanghai Sunway Biotech)
|Ad‐p53/Gendicine® (Shenzhen SiBiono GeneTech)
|Head and neck cancer
|Voretigene neparvovec/LUXTURNA® (Spark Therapeutics)
|Leber's congenital amaurosis (Biallelic RPE65 mutation‐associated retinal dystrophy)
|2017 (USFDA), 2020 (Health Canada), 2020 (TGA)
|Onasemnogene abeparvovec/ZOLGENSMA® (AveXis, now Novartis Gene Therapies)
|Spinal muscular atrophy (SMA) with bi‐allelic mutations in the survival motor neuron 1 (SMN1) gene in pediatric patients less than two years of age
|2019 (USFDA), 2020 (EMA), 2020 (JMHW)
|Alipogene tiparvovec/Glybera® (UniQure)
|Lipoprotein lipase deficiency
More on Gene Therapy Development and Manufacturing
Hear from industry thought leaders in cell and gene therapy on five key trends for AAV gene therapies and issues in the field today.
Discover how the latest analytical tools can achieve different assays with high reproducibility and accuracy.
Gain insights into regulatory considerations for AAV-based gene therapies from industry thought leaders in cell and gene therapy.
Find tips on meeting regulatory guidance for gene therapies from industry experts in cell and gene therapy.
Learn how ddPCR technology offers the accuracy to quantify AAV viral titers to support many aspects of quality control for AAV-based gene therapy development.
Discover different delivery systems used for advancing gene therapy, including viral and non-viral vectors, and the benefits and challenges of each approach.
Explore the common challenges, analytical tools, and importance of selecting appropriate assays for the development of potency assays for cell therapy products.
Read about the current state of the industry, challenges associated with potency assays, and measuring product potency throughout the manufacturing process.
Use this guide to approach various AAV vector challenges, AAV characterization, and address developmental hurdles with using vectors in gene therapy.
Get guidance on how to transition from qPCR to ddPCR technology and the differences to consider between the two methods based on AAV titering assay study.
Get started with this protocol designed to measure AAV vector genome titer using ddPCR technology for both in-process and purified samples.
Watch this video tutorial on how to assess AAV capsid protein ratios as a fast quality control step in gene therapy development and manufacturing.
- New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children and durability 5+ years post-treatment. Novartis. Accessed July 5, 2022. https://www.novartis.com/news/media-releases/new-zolgensma-data-demonstrate-age-appropriate-development-when-used-early-real-world-benefit-older-children-and-durability-5-years-post-treatment
- Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83(9):810-817. doi:10.1212/WNL.0000000000000741
- Zhao Z, Anselmo AC, Mitragotri S. Viral vector‐based gene therapies in the clinic. Bioeng Transl Med. 2021;7(1):e10258. doi:10.1002/btm2.10258
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