Andelyn Biosciences AAV Curator® Platform Used to Manufacture Clinical Grade Viral Vector for FDA-Authorized First-In-World Universal Donor CRISPR/AAV Transduced CAR-NK Cells

COLUMBUS, Ohio, May 6, 2025 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has manufactured viral vector with its AAV Curator^® Platform for Nationwide Children's Hospital's Cellular Therapy and Cancer Immunology Program. The Adeno-Associated Virus (AAV) will be utilized in a novel cell therapy for Universal-Donor CD38KO CD33CAR-NK cells, to be studied in a forthcoming clinical trial. The Phase 1 trial will study the safety of the novel therapy in patients with advanced, high-risk acute myeloid leukemia (AML).

Within the field of cell therapy, there is a consistent need from researchers to manufacture cGMP virus to support the advancements of these critical therapies. While many AAV manufacturing platforms are specifically designed to be scaled to support large scale indications, Andelyn's AAV Curator^® Platform utilizes Optimization-by-Design^™ to be correctly sized for scale and speed to the clinic. The well-established platform allows for the ability to support the exacting viral vector manufacturing needs of the NCH NK cells created on the hospital's patented universal-donor NK cell platform. 

Matt Niloff, Chief Commercial Officer at Andelyn said, "We are excited to be playing a key role in this very promising Universal Donor CD38KO CD33CAR-NK cell therapy technology by NCH. We look forward to the clinical trial and ushering in a new era for patients with AML and other blood cancers."

Andelyn is enabling the progression of life-altering cell and gene therapies for both rare and prevalent diseases with the highest quality standards and scalable end-to-end development and manufacturing capabilities at its Columbus, Ohio facilities.

About Andelyn Biosciences, Inc.
Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of its development and manufacturing facilities in Columbus, Ohio, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn can accelerate programs and deliver high quality products by developing and manufacturing processes on its configurable, data-driven AAV Curator^® Platform, or tech transferring in an established client program. Capabilities include cGMP manufacturing for suspension processes up to a 2,000-liter or scaling-out with adherent processes. A rigorous quality system, regulatory support, and supply chain vertical integration further support clients in bringing their critical therapies to market. For more information, visit andelynbio.com.

About The Abigail Wexner Research Institute at Nationwide Children's Hospital.
Named to the Top 10 Honor Roll on U.S. News & World Report's 2024-25 list of "Best Children's Hospitals," Nationwide Children's Hospital is one of America's largest not-for-profit free-standing pediatric health care systems providing unique expertise in pediatric population health, behavioral health, genomics and health equity as the next frontiers in pediatric medicine, leading to best outcomes for the health of the whole child. Integrated clinical and research programs are part of what allows Nationwide Children's to advance its unique model of care. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children's faculty train the next generation of pediatricians, scientists and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Children's Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational, behavioral and population health research. The AWRI is comprised of multidisciplinary Centers of Emphasis paired with advanced infrastructure supporting capabilities such as technology commercialization for discoveries; gene- and cell-based therapies; and genome sequencing and analysis. More information is available at NationwideChildrens.org/Research.

SOURCE Andelyn Biosciences