Andelyn Biosciences Observes Rare Disease Day

BlogFebruary 28, 2022

Observed every year on the last day in February, Rare Disease Day works to raise awareness for rare diseases and improve access to treatment and medical representation for the 300 million people living with rare disease, their families, and loved ones. Here at Andelyn Biosciences, Rare Disease Day holds particular importance. Manufacturing innovative therapies to treat rare diseases is at the heart of our mission. Even our name, “Andelyn,” was inspired by two young gene therapy pioneers living with rare diseases.

Our commitment to a patient-first mentality is rooted in our origins at Nationwide Children’s Hospital as their cell and gene therapy development and manufacturing. Today as Andelyn Biosciences, a standalone entity, our dedication to the entire rare disease research community has never wavered. We now support clients across the country and around the globe developing gene therapy indications preclinical concept to commercialization. Adherence to excellence and prioritizing patient safety are at the core of our every decision—families around the world count on our team.

As a for-profit manufacturer, our unique business model supports reinvestment into the pioneering research that continues at the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s. This research ranges from basic discovery science to clinical studies to population health research – all to address rare disease cures and challenges affecting children’s health worldwide.

“Andelyn Biosciences has been and continues to be an incredible partner in producing many high-quality small-scale vector preps for my discovery research with an incredible turnaround time. Hopefully, some of these small projects will develop into next-generation treatments for rare diseases. Moreover, they have produced material for safety studies needed to move gene therapy for rare disorders forward towards the clinic,” said Kathrin Meyer, Assistant Professor and Principal Investigator with AWRI at Nationwide Children’s. “We also recently made two clinical vector batches at Andelyn that will ideally help change the life of many rare disease patients in the near future. It is an incredible, highly motivated team with sincere compassion towards the rare disease community; they truly care and put their heart into the production of these therapeutics.”

“Over the past two years, Andelyn Biosciences has produced over a dozen research-grade vectors used in preclinical gene therapy studies for rare diseases in my laboratory,” added Allison Bradbury, Assistant Professor and Principal Investigator with AWRI at Nationwide Children’s. “As next steps, lead candidate vectors will be manufactured by the equivalent processes for the production of clinical-grade vectors allowing for methodical translation of a therapy from the bench to bedside. This partnership has been instrumental in the development of therapies for devastating, rare pediatric inherited disorders.”

Currently, Andelyn leases space and continues to operate out of AWRI while awaiting the completion of its state-of-the-art commercial manufacturing and development sites. The 185,000 square foot Andelyn Corporate Center located on The Ohio State University’s Innovation District will open for production in mid-2022. The Dublin-based Andelyn Development Center is set to open in May 2022 and will house Andelyn’s preclinical and development services.

By observing Rare Disease Day each year, we at Andelyn strive to shine a light on people living with a rare disease. In partnership with the AWRI and all our clients, we are Pioneering Solutions that Turn Hope Into Reality™ as we work to change and improve the lives of millions of people worldwide living with genetic disease.