Gene therapy is a promising new therapeutic modality for the treatment of inherited and acquired diseases. In gene therapy, the faulty gene is modified to restore normal function. The genetic material is delivered to targeted cells via viral delivery or non-viral delivery such as peptide-based or lipid-based delivery systems. These therapies involve modified polynucleic acid chains that enhance stability and hold promise for respiratory conditions, neurodegenerative diseases and cancers.
Clinical applications utilize different types of oligonucleotides, including antisense oligonucleotides (ASo), micro-RNA (miRNA), small interfering RNA (siRNA), aptamers and CpG oligonucleotides. To deliver these oligonucleotides effectively, specialized technology like lipid nanoparticles (LPNs) acts as carriers, targeting specific cells and tissues.
Bioanalysis is the foundation of novel drug development. Although regulatory guidance exists for manufacturing, non-clinical and clinical conduct of gene therapies, there is no specific guidance on the bioanalysis of gene therapies.
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This webinar offers insights into the latest strategies and techniques that apply to bioanalysis of gene therapies. Experts will share knowledge and experience, providing valuable guidance on overcoming the challenges associated with this critical aspect of bioanalysis to support gene therapies.
In addition, a case study will be presented to illustrate the bioanalytical challenges and solutions associated with LNP encapsulated antisense oligonucleotides (ASO). Accurately isolating and measuring these ASOs from plasma presents challenges. Separating free and encapsulated oligonucleotides in plasma is complicated due to factors like oligonucleotide size, vector structure and plasma components. A specific and sensitive procedure is needed to isolate free oligonucleotides without affecting the structure of lipid nanoparticles.
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Join this webinar and learn about the bioanalytical challenges and solutions in gene therapy development, including the characterization of lipid nanoparticles and measurement of encapsulated oligonucleotides.
Speakers
Rafiq Islam, MSc, VP, Bioanalysis and Biomarker, Amador Bioscience
Rafiq Islam is the Vice President at Amador Bioscience responsible for scientific and operational leadership of Bioanalysis and Biomarker Services operation in Germantown, MD. Prior to joining Amador, he was the VP of Pharmaceutical Development Services Division at Smithers.
Previously, he served as the Executive Director at Celerion Inc, and a Scientific Director at EMD Millipore (MO, USA). He held similar positions as the head of a bioanalytical department at Covance Inc. (AZ, USA) and Huntingdon Life Sciences (NJ, USA). He has over 25 years of biotechnology and CRO industry experience. He has authored and contributed to more than 50 articles, industry whitepapers and numerous posters on the topic of regulated bioanalysis.
Message Presenter
Margaret Costanzo, PhD, Associate Principal Scientist Cell & Gene Therapy, Amador Bioscience
With 11+years of extensive experience in R&D and assay development, Margaret Costanzo excels in supporting non-clinical, preclinical and clinical studies. Her expertise lies in viral vectors (HSV2, Lentivirus, AdVs), LNP and oligonucleotides. She earned her PhD in pathobiology from the University of Texas at El Paso.
Message Presenter
Meina Liang, PhD, Chief Technology Officer & President of Bioanalysis & Biomarkers, Amador Bioscience
Dr. Liang is currently the Chief Technology Officer (CTO) and President of Bioanalysis & Biomarkers at Amador Bioscience, leading global strategy to establish cutting-edge bioanalysis capabilities and enhancing Amador’s global-standard services to biopharmaceutical companies. She joint Amador with broad experiences in drug development and in global regulatory submissions.
Prior to Amador, Dr. Liang was the Executive Director and global head of Integrated Bioanalysis at AstraZeneca. She led a centralized GxP group with 100+ scientists across 5 global sites to support AstraZeneca portfolio from discovery to post marketing across therapeutic areas and drug modalities. Her team is responsible for assessment of pharmacokinetics, immunogenicity, biomarkers and also contributed to MOA investigation, candidate drug selection, companion diagnostics development. She played a key role in a number of successful BLA submissions and market approvals, e.g. IMFINZI and LUMOXITI.
Throughout her career, she held various positions with increasing leadership responsibilities at Amgen, Abgenix and Berlex Biosciences. She is an inventor for a number of patents, an author for 60+ peer-reviewed articles and has frequently presented at international conferences. Dr. Liang holds B.S in Pharmacy from Beijing University in China and Ph.D in Pharmacology from Univ. of North Carolina at Chapel Hill.
Message PresenterWho Should Attend?
- R&D development scientists
- Biotechnology and pharmaceutical industry professionals
- Academic scientists and researchers
- Gene editing and cell & gene therapy professionals
- Biomedical engineers and biochemists
- Regulatory affairs personnel
What You Will Learn
In this webinar, attendees will learn:
- Bioanalytical strategy development to support gene therapies — The challenges associated with isolating free and encapsulated oligonucleotides
- Analytical considerations for measuring oligonucleotide using hybridization ELISAs
Xtalks Partner
Amador Bioscience
Amador Bioscience’s Bioanalysis and Biometrics team ensures compliance with international clinical trial standards. We focus on enhancing the success rate of new drugs by scientifically determining the optimal dose, target, and patient population. Our multidisciplinary experts provide comprehensive bioanalytical services, including method development, sample analysis, and result interpretation. Equipped with state-of-the-art facilities in our GLP compliant labs in the United States and China, we deliver accurate data for evaluating drug efficacy, safety, and pharmacokinetics. Our experienced biostatisticians employ innovative methodologies and trial designs to analyze data and generate meaningful insights. By partnering with us, you gain access to a dedicated team committed to supporting your innovative drug discovery projects from pre-clinical research to market approval. We combine scientific excellence, regulatory expertise, and advanced analytics to accelerate your journey towards successful clinical development and commercialization of novel therapies.
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