On the pathway to GMP manufacturing in support of clinical trials, multiple technology transfers may correlate to an increase in complexity and risk to both product and timeline. The hand-off of new technologies, protocols, and materials can lead to delays or project failures.
Developing new CRISPR/Cas9-based genome editing tools will enhance innovative therapeutic products and the pathway to clinical programs. However, current obstacles in genome editing include high error rates, toxicity from production impurities, and strict regulatory concerns.
Optimizing gene payload begins with plasmid design, and NanoplasmidsTM provide several advantages over traditional plasmids. That’s the focus of our latest webinar, done in collaboration with Phacilitate, as a panel of Aldevron experts discuss the unique opportunities associated with using nanoplasmids.
The Nanoplasmid™ vector is a high-performance <500 bp antibiotic-free bacterial backbone that, by combining the best attributes of plasmid and minicircle vectors, is designed to replace antiquated bacterial backbones that have for decades been the industry standard. The Nanoplasmid backbone is engineered to reduce plasmid associated innate immune activation which is ubiquitously associated with plasmid vectors; this translates to much lower observed toxicity and vector inactivation during primary cell transfection resulting in superior performance compared to plasmid and/or minicircle vectors with a broad range of gene and cell therapy products. This webinar will provide an overview of the Nanoplasmid technology and highlight the benefits in a variety of applications, relying on data from published studies.
A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelist.
Gene and cell therapy developers are contending with the implications of the evolving regulatory environment while trying to progress programs as quickly as possible to address unmet needs and secure first-mover advantage in competitive indications. Regulators are facilitating efforts to truncate development timelines through a series of designations that can expedite programs while also making efforts to implement standardization.
Genome editing enzymes such as Cas9 have been successfully employed to target different genetic disorders in model systems, and ex vivo approaches have demonstrated the clinical potential of this technology. However, the challenge of delivering such corrective enzymes in vivo represents a substantial barrier to therapeutic translation.
Viral vectors—refined during their use in traditional gene therapy—represent the most widely-used delivery platform in preclinical work, and nanoparticles are quickly maturing into a comparably powerful vehicle. Direct delivery of pre-formed CRISPR enzymes has recently emerged as an appealing strategy for enabling therapeutic editing in vivo.
Aldevron recently announced its upcoming virtual ribbon cutting event, which will take place on Wednesday, February 10 at 12:00 PM CST. Its focus will be the company's investments in key areas, expanding Aldevron's ability to enable clients' breakthroughs in the emerging field of genetic medicine, further impacting patients and achieving the company's mission.
The Joint Session of the State of North Dakota’s 67th Legislative Assembly held its opening sessions earlier this week, and featured Governor Doug Burgum’s 2021 State of the State address. Burgum reflected on the progress and challenges of the past year, calling for bold action and investments in infrastructure and other priorities to position the state for a bright future.
