Biomere is committed to working with the scientific research community to develop webinars on various topics in preclinical drug development including gene therapy, ophthalmology and toxicology. Each webinar recording includes an abstract summary.
If you would like to share your research via a recorded webinar, please contact bd@biomere.com.
Standard in vitro genotoxicity testing approaches have limitations for testing nanomaterials (NMs). Substantial developments in new approach methodologies (NAMs) have created opportunities to overcome these issues in recent years. In this webinar, Dr. Doak reviews the applications of co-culture models and 3D liver spheroids for in vitro genotoxicity testing to better support regulatory risk decision making.
This webinar explores the role of veterinary anatomic pathologists in the pharmaceutical and medical device industries. It highlights the skills they bring to development and safety teams, as well as the ancillary technologies they recommend and interpret. The session introduces technologies such as immunohistochemistry, immunofluorescence, molecular diagnostics, and digital spatial profiling, with examples of their potential applications in the field.
In this webinar, Bella Lear discusses why animals remain key to preclinical research and why, despite aspirations for the complete replacement of animals, they are likely to remain an essential tool of drug discovery. New Approach Methodologies (NAMs) are advancing rapidly, yet in most cases, evaluating the efficacy and toxicity of new medicines still relies on animal testing. This webinar reviews the long history of animal models in science-based medicine, and discusses the future which will include both animal models and NAMs.
In this webinar, Andrea focuses on the importance of ligand binding assays in the bioanalysis of small molecule and complex biologics, and highlights the role of orthogonal assays, such as LC-MS, in complementing ligand binding assay data. She covers preclinical, nonclinical (GLP), and clinical assay development and design, including the creation of immunogenicity assays, and the detection of anti-drug antibodies (ADAs). The webinar also features a case study demonstrating the value of bioanalysis data in developing a therapy for a rare pediatric disease.
In this presentation, Dr. Kamendi shares key considerations for an IND submission, noting the lack of established best practices. The goal of nonclinical drug development is to match the right patient population with the right drug, properties, dose, and timing. The process begins with building the target product profile (TPP) sheet. Other important factors include:
Non-GLP toxicology programs typically involve clinical observations of drug effects and macroscopic examination of organs in two species. Once pharmacology and toxicology studies are complete, the IND can be filed, but nonclinical studies, such as carcinogenicity and abuse liability, must continue. For systemic and local administration of gene therapy, it is necessary to assess the systemic effect of the transgene before moving forward with full toxicology studies.
In this webinar, Jeff discusses Chameleon’s EVADER™ gene therapy platform, which is designed to evade the immune system, overcome the neutralizing immune response, expand the treatable patient population, and enable multiple dosing. The platform encapsulates AAVs in a lipid bilayer with specific immune cell inhibitors and has demonstrated increased infectivity, delivery, and transgene expression in preclinical models. The EVADER™ platform is currently being used to develop gene therapies for multiple diseases in collaboration with biotech and pharmaceutical companies.
In this presentation, Dr. Gao provides an overview of current trends in gene therapy, beginning with definitions of in vivo gene therapies and ex vivo modification of patient cells. Gene therapy includes four major approaches: gene replacement, gene therapy, gene addition, and gene activation.
An example of successful gene replacement is Canavan’s disease, where a gene therapy approach was developed to systemically deliver AAV9 across the blood-brain barrier (BBB) to deliver a transgene encoding the missing enzyme needed to break down N-acetyl aspartate in neuronal cell mitochondria. A clinical trial involving a single two-year-old child with Canavan’s disease demonstrated high efficacy, a good safety profile, and most importantly, the child is alive today with a good quality of life.
Nonhuman primates (NHPs) offer more translational value than rodents for gene therapy when using multiple AAV serotypes, including AAV3, AAV8, and AAV9. NHPs are primarily used for safety studies; however, because disease state NHP models are lacking, efficacy studies are not typically conducted.
RNA-based therapies can be broadly classified as RNA editing, RNA silencing, and readthrough therapy to reverse premature translational termination. AAV-based therapies can address premature translation termination by delivering suppressor tRNAs. Suppressor tRNA differs from normal tRNA by one nucleotide, and a library of suppressor tRNAs can be developed for different amino acids. It is important to note that efficacy is typically determined by delivery efficiency rather than tRNA binding.
In this webinar, Dr. Gao presents a comprehensive overview of the key principles, history, current challenges, and future directions of human gene therapy, with a focus on AAV gene therapy for rare diseases. The session also highlights AAV capsid engineering to modulate target tissue tropism and biodistribution, with examples of using AAV for gene replacement, gene addition, gene silencing, and in vivo gene editing in preclinical studies.
Additionally, Dr. Gao reviews strategies to overcome immunological barriers to enhance the efficacy of AAV-mediated gene therapy.
In this webinar, Franklin introduces the basic concepts of bioanalysis, including the important role of bioanalysis in successful drug development. He also reviews currently used plate-based and non-plate-based assay methods to analyze large and small molecule therapies. He highlights some of the requirements and challenges associated with the bioanalysis of large molecule therapies, including antibody-drug conjugates (ADCs), bispecific monoclonal antibodies (mAbs) and protein replacement therapies.
In this 5-part webinar, Patrick discusses several highly relevant topics in the drug discovery and development space.
In this 5-part webinar, Patrick discusses several highly relevant topics in the drug discovery and development space.
In this 5-part webinar, Patrick discusses several highly relevant topics in the drug discovery and development space.
