Media Center

Andelyn Biosciences’ unique business model leads to reinvestment into pioneering research conducted at the Abigail Wexner Research Institute (AWRI) at Nationwide Children's, from basic discovery science to population health research on a broad range of challenges affecting children’s health.

Look for more news about Andelyn Biosciences and Nationwide Children's research on this page in the future.

Press Releases

Andelyn Biosciences Logo

Nationwide Children’s Hospital Announces Plans for Andelyn Biosciences

Central Ohio’s first commercial-scale gene therapy Good Manufacturing Practices (cGMP) clinical manufacturing facility slated for 2023.

Gene Therapy Research at AWRI

Long-Term Follow Up of Patients Receiving Novel Gene Therapy for SMA Type I

Researchers recently published the long-term outcomes of patients who received the investigational drug AVXS-101 – an adeno-associated virus serotype 9 mediated gene replacement therapy.

Test tubes

Preventing the Development of Muscular Dystrophy Through Surrogate Gene Therapy

Researchers identify a key regulatory protein implicated in Galgt2 overexpression and begin to elucidate its protective mechanism against muscular dystrophy.

Heart Function

Preclinical Gene Therapy Study Shows Muscle Restoration in Charcot-Marie-Tooth Disease Model

Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.

Big Gains in FSHD Research: A Newly Published Model of FSHD and a Potential Gene Therapy to Improve Functional Outcomes

The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.

spinal muscular atrophy SMA

Can Gene Therapy Treat Dominantly Inherited Disorders?

Recent applications of adeno-associated virus (AAV) mediated gene therapy have focused mainly on correcting recessively inherited diseases. But what about dominantly inherited disorders? It looks like AAV could be a delivery mechanism for treating those genetic disorders, too.

Nationwide Children's Hospital

Nationwide Childrens Hospital Leads Effort for Gene Therapy Treatment for Duchenne Muscular Dystrophy

Sarepta Therapeutics announced yesterday that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). Nationwide Children's Hospital was the sole original site for the clinical study that resulted in this approval, led by Dr.

Jerry Mendell, MD

Phase 1 Study Shows Encouraging Data for Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published today in the New England Journal of Medicine. The study was conducted by Researchers from Nationwide Children’s Hospital in collaboration with AveXis, Inc. and The Ohio State University College of Medicine.

Breakthrough of the Year logo

Science Breakthrough of Year

Visitors to Science’s website were offered the chance to vote on a list of candidates for Breakthrough of the Year. The first round of voting narrowed the field to four, and a second round, in which more than 12,000 votes were cast, determined the People’s Choice.

Nationwide Children's Hospital

Nationwide Children's Hospital Study Named Among Top Ten Clinical Research Achievement Awards by the Clinical Research Forum

The first clinical trial to test the functional replacement of the mutated gene responsible for SMA1 will be recognized at the 2018 Top Ten Clinical Research Achievement Awards at the National Press Club in Washington, D.C., on April 18, 2018.

Nationwide Children’s Hopes Gene Therapy Factory Makes Columbus a Major Biotech Hub

“We have a vision to help Central Ohio to become more of a biotechnology hub in the gene therapy space,” Dr. Dennis Durbin, chief scientific officer of the hospital’s Wexner Research Institute. “We’re really optimistic and ambitious about what this might mean for Central Ohio over the long term.”

This is how you Spell Hope

Nationwide Children’s Hospital spinoff Andelyn Biosciences takes its name in part from Andrew Kilbarger, who volunteered as a child in gene therapy research that won’t save his life, but could cure others.