A Gene Therapy Company with a Rich History in Viral Vector Production
The road to Andelyn Biosciences started decades ago with ground-breaking research at Nationwide Children’s Hospital.
Our Name
The name “Andelyn” was chosen as a hybrid of two patients who participated in pivotal Phase I clinical trials at Nationwide Children’s Hospital – Andrew and Evelyn. Andrew received the first U.S. investigational gene therapy for Duchenne muscular dystrophy in 2006. Evelyn received experimental gene therapy for spinal muscular atrophy in 2015. “Andelyn” combines their names, representing all the families who have courageously participated in the research that makes today’s gene therapies possible and reminding us that patients are at the heart of everything we do.
When the idea of Andelyn was born, GMP facilities were unheard of at children’s hospitals, and gene therapy research was a rarity. Nationwide Children’s physician-scientists collaborated with hospital and research institute leadership to create a small GMP facility to produce viral vectors for gene therapy clinical trials. Having this resource accelerated the hospital’s gene therapy program and helped bring some of the brightest minds to Nationwide Children’s.
The Abigail Wexner Research Institute at Nationwide Children’s opened a larger cGMP Clinical Manufacturing Facility in 2017, which initially housed Andelyn Biosciences.
In 2020, Andelyn was spun out of the Nationwide Children’s Hospital as a Contract Development and Manufacturing Company.
In a key milestone toward advancing cell and gene research, Brian Kaspar, PhD, during his tenure with Nationwide Children’s, discovered that the AAV9 vector was capable of crossing the blood brain barrier when injected into the vascular system to deliver genes directly to motor neurons.
A few years following that discovery, Jerry Mendell, MD, principal investigator in the Abigail Wexner Research Institute at Nationwide Children’s, led the Phase 1 clinical trial that was the first to study gene therapy for spinal muscular atrophy type 1, the most severe form of SMA. Based on that clinical research, Zolgensma (Onasemnogene abeparvovec), a one-time infusion for pediatric patients less than 2 years of age with spinal muscular atrophy, was the first systemic gene therapy approved by the FDA. The gene therapy was originally licensed to AveXis, which was acquired by Novartis in 2018.
Other notable gene therapy spin-outs from Nationwide Children’s are Celenex, developing gene therapies for lysosomal storage disorders (acquired by Amicus Therapeutics in 2018) and Myonexus, acquired by Sarepta Therapeutics in 2019, which is developing the first corrective gene therapies for Limb-girdle muscular dystrophies. Many more gene therapies, and now cell-based therapies, have progressed into clinical trials.
Realizing the potential of both cell and gene therapy, and the value of the experience and capabilities developed over the years, Nationwide Children’s Hospital spun off Andelyn as a separate cell and gene therapy CDMO in January 2020.
In November 2020, Andelyn broke ground on a new 185,000 square-foot commercial scale gene therapy manufacturing plant on the Ohio State University’s Innovation District.
In 2021, Andelyn received a significant Investment and entered into a strategic partnership with Pall Corporation and Cytiva, strengthening its ability to serve more clients and expand its offerings. Also in 2021, the company signed a lease for a 42,000 square foot process development lab expansion in Dublin, OH, called the Andelyn Development Center, to help meet the growing demands of our clients.
In 2022, our manufacturing facility, the Andelyn Corporate Center (ACC) was opened and allowed Andelyn to expand its services to include late-stage and commercial manufacturing and become a full spectrum CDMO. Today, the facility has released many GMP matches, boasts many client programs and is producing PPQ batches. Clients continue to partner with Andelyn to leverage its deep expertise for all phases of viral vector gene therapy development through to commercialization.
Key Moments in Our History
News & Press Releases
July 10, 2024
Andelyn Biosciences Selected by UMass Chan Medical School to Manufacture Clinical Grade AAV9-CSA Vector to Treat Cockayne SyndromeAndelyn Biosciences Selected by UMass Chan Medical School to Manufacture Clinical Grade AAV9-CSA Vector to Treat Cockayne SyndromeJune 11, 2024
UMass Chan Receives $2.2 Million to Fund Gene Therapy for Cockayne SyndromeUMass Chan Receives $2.2 Million to Fund Gene Therapy for Cockayne SyndromeMay 23, 2024
Andelyn Biosciences Selected as Viral Vector Manufacturing Partner for the California Institute for Regenerative Medicine (CIRM) Accelerating Medicines Partnership (AMP) Bespoke Gene Therapy Consortium (BGTC)Andelyn Biosciences Selected as Viral Vector Manufacturing Partner for the California Institute for Regenerative Medicine (CIRM) Accelerating Medicines Partnership (AMP) Bespoke Gene Therapy Consortium (BGTC)March 25, 2024
Andelyn Biosciences and Grace Science, LLC Partner to Tech Transfer Phase I/II/III Manufacturing of a Suspension Process Adeno-Associated Virus (AAV) Gene Therapy for NGLY1 DeficiencyAndelyn Biosciences and Grace Science, LLC Partner to Tech Transfer Phase I/II/III Manufacturing of a Suspension Process Adeno-Associated Virus (AAV) Gene Therapy for NGLY1 DeficiencyEmpowering Our Community Through Science and Service
At Andelyn Biosciences, we believe in giving back and forging strong community connections. Our dedication extends beyond the lab, as we actively participate in community service, educational outreach, and partnerships that promote public health and scientific literacy. Discover how we’re making a difference in communities locally and globally.
Shape the Future of Gene Therapy with Us
Join a team where innovation meets purpose. At Andelyn Biosciences, we’re not just developing life-changing therapies; we’re cultivating a culture of excellence, diversity, and continuous learning. If you’re passionate about making a meaningful impact in the biotech industry, explore our career opportunities and become part of our dynamic team.