Therapies, Editing, Manufacturing

July 16, 2025 by Michele Cheng

Pointing to a bright, yet complex, future at ASGCT

When the brightest minds in gene therapy, genome editing, and biomanufacturing come together, there is always a wide range of views—from expert talks and poster presentations to panel discussions—providing insights that not only reflect the state of the industry but also highlight strategic opportunities.

That was the case at the ASGCT Annual Meeting in New Orleans a few weeks ago, where the topics were as diverse as the city itself, with numerous opportunities to see how the various fields in the industry are progressing.

Aldevron and IDT were in the spotlight for that progress this year, with the announcement of the successful manufacture of the world’s first mRNA-based personalized CRISPR therapy. This announcement shows just how the potential for genomic medicines discussed at ASGCT each year can become the groundbreaking news at future events. As for the topics for this year, below is a summary of those that were discussed the most.

Showcasing gene therapy
Gene therapy continues to advance with a strong emphasis on precision, long-term efficacy, and scalable delivery systems. Key themes discussed were:

• In Vivo Editing and RNA Therapeutics
  There is growing momentum behind in vivo gene editing and RNA-based treatments, with promising data on editing efficiency and immune modulation.
• Emerging Vector Platforms
  Novel vector systems are being explored for their potential to reduce costs and improve safety. Aldevron’s flexible manufacturing platforms were noted as being well-positioned to support these innovations.

Enhancing quality and efficiency with AAV
AAV vector production remains central to gene therapy, and this year’s discussions highlighted several areas that show growing opportunities, such as:

• Plasmid Design Optimization
  The importance of helper plasmid design in improving yield and quality was a recurring theme, as it can offer enhanced solutions for therapeutic development.
• Scalable, Consistent Manufacturing
  As therapies move toward commercialization, the need for robust, reproducible, and high-purity manufacturing processes is more critical than ever. This is also critical in dealing with regulatory

Broadening the genome editing toolkit
The genome editing landscape is expanding beyond traditional tools, with new approaches offering greater precision and flexibility:

• Programmable Recombinases and Large DNA Insertions
  Innovative editing strategies are enabling more targeted and efficient genome modifications.
• Delivery and Cell-Type Specificity
  Effective delivery remains a challenge, particularly for difficult-to-transfect cell types. Aldevron’s work in optimizing plasmid formats and delivery conditions is essential to helping clients overcome these barriers.

Where are we headed?
Several emerging trends are poised to shape the future of gene and cell therapy, with some exciting developments in the works, including:

• Increased Focus on Pediatric and Neonatal Applications
  Regulatory agencies are showing greater openness to early intervention in infants and children, which may shift development priorities and reduce material requirements.
• Demand for Cost-Effective Therapies
  As gene therapies expand globally, there is growing pressure to reduce costs and improve accessibility.
• Integration of AI in Therapeutic Design
  Artificial intelligence is playing a larger role in protein engineering, vector optimization, and editing tool discovery. Aldevron’s ability to support rapid prototyping and production will be essential in this fast-moving space.
• Data-Driven Differentiation
  Clients are increasingly focused on off-target effects, durability, and cell-type specificity. Generating and sharing performance data for Aldevron technologies will be critical for differentiation and client confidence.

The ASGCT 2025 meeting reaffirmed that the future of gene and cell therapy is bright—and complex. With a continuous focus on innovation, collaboration, and a relentless focus on quality, the insights from the event will help drive more advancement, whether it’s optimizing plasmid design, supporting novel vector development, or enabling next-gen editing tools.

• Learn about modalities provided by Aldevron
• Have questions on this topic? Contact Us
• Have an idea for a topic? Let us know!

Subscribe to our blog