Andelyn Biosciences Enhances Gene Therapy Platform Options

Andelyn Biosciences Expands Gene Therapy Offerings

Andelyn Biosciences, Inc., a prominent player in the cell and gene therapy sector, has made a significant enhancement to its AAV Curator™ Platform. This development comes through a licensing agreement with the Broad Institute of MIT and Harvard, incorporating the highly anticipated Stanton Lab CNS (Central Nervous System) capsids.

What Are AAV Curator™ Platform and CNS Capsids?

The AAV Curator™ Platform is designed to bolster the development of adeno-associated viruses (AAVs), leveraging cutting-edge plasmids that facilitate improved transduction, especially in neural tissues. The addition of the Stanton Lab CNS capsids is poised to enhance the efficacy of these therapies by increasing brain transduction rates while minimizing the likelihood of peripheral organ engagement.

Research and Development Benefits

Under the terms of this expanded agreement, Andelyn is granted the authority to utilize the Stanton Lab CNS capsids in various research and development endeavors. This includes rigorous screening of potential gene therapy candidates and facilitating scale-up projects alongside pre-clinical development steps in readiness for IND-enabling studies. Notably, the agreement also allows Andelyn to sublicense these innovative capsids, offering its clients broader access for internal research applications.

Quotes from Leadership at Andelyn

"Integrating the Stanton Lab CNS capsids into our AAV Curator™ Platform is a significant step forward for gene therapies addressing nervous system disorders," shared Matt Niloff, the Chief Commercial Officer at Andelyn. "This expansion empowers our clients in the pursuit of developing more effective treatments for neurological conditions, ensuring patients receive safer options when dealing with rare diseases."

Focus on Improved Treatment Efficacy

The inclusion of these CNS capsids has shown promising data, indicating that they can be significantly more effective in targeting various regions of the brain. For instance, these capsids could be six times as efficient in targeting most areas of the cerebrum and up to 13 times stronger in neuroretinal transduction. Such advancements are crucial in enhancing treatment precision and diminishing unwanted off-target effects, which can complicate therapies.

Andelyn's Commitment to Patients

Andelyn remains dedicated to the advancement of transformative gene therapies for both rare and widespread diseases. With a steadfast commitment to quality, the organization ensures that it meets the highest standards throughout its development and manufacturing processes. With facilities designed to support scalable end-to-end operations, Andelyn is fully equipped to meet the growing demands of gene therapy.

About Andelyn Biosciences, Inc.

As a full-service cell and gene therapy CDMO, Andelyn Biosciences specializes in producing and characterizing viral vectors for gene therapy applications. Their extensive experience spans over 20 years, including the successful production of cGMP material for more than 450 clinical batches across 75 global trials. Operating from their advanced manufacturing facilities, Andelyn facilitates the development of cell and gene therapies from initial concept through plasmid design, process optimization, and full-scale commercial manufacturing.

In addition, with the robust features of the AAV Curator™ Platform, Andelyn is capable of accelerating project timelines and consistently delivering high-quality products. The organization also upholds a stringent quality framework, alongside regulatory support and supply chain integration, to assist clients in efficiently bringing their critical therapies to the marketplace.

Frequently Asked Questions

What is the AAV Curator™ Platform?

The AAV Curator™ Platform is a sophisticated framework that helps develop adeno-associated viruses for gene therapies, enhancing their efficacy and precision.

What are CNS capsids, and why are they important?

CNS capsids are specialized viral capsids designed to target the central nervous system. Their inclusion in gene therapies can significantly improve treatment outcomes for neurological disorders.

How does Andelyn support its clients?

Andelyn offers comprehensive services from initial concept to commercial manufacturing, ensuring high-quality standards throughout the development lifecycle.

What is Andelyn's commitment to quality?

With a focus on high-quality production and stringent regulatory support, Andelyn maintains an exceptional quality system to guarantee the safety and effectiveness of its therapies.

What is the company’s experience in gene therapy?

With over 20 years of experience, Andelyn has successfully supported numerous clinical trials, highlighting their expertise in the production of gene therapy materials.

About The Author

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