Nationwide Children's Hospital, a hot spot for gene therapy research, this week launched a biotech spinout dedicated to constructing the complex one-time treatments.
In creating Andelyn Biosciences, as the new company is called, Nationwide aims to build on its decade-long experience in developing, testing and manufacturing gene therapies for deadly inherited diseases like spinal muscular atrophy and Duchenne muscular dystrophy.
Andelyn will operate as a for-profit subsidiary of the Columbus, Ohio-based hospital, producing gene therapy components for biotech and pharmaceutical companies running clinical trials. By 2023, Andelyn also plans to contract with drugmakers to make commercial products from a larger facility that's yet to be built.
As the gene therapy field has surged forward — yielding two pioneering drug approvals and at times remarkable clinical results — manufacturing has emerged as a persistent challenge.
Current treatments are delivered via hollowed-out, inactivated viruses that must be made in enormous quantities to deliver sufficient copies of the target gene. Scaling up from small studies to late-stage tests and commercial marketing, meanwhile, requires a company either own a dedicated manufacturing plant or turn to existing contract manufacturers, many of which currently have long wait times for production.
"We're tracking the growth, maturation and evolution of the field," said Dennis Durbin, chief scientific officer of the Abigail Wexner Research Institute at Nationwide, in an interview. "Commercial-scale manufacturing represents one of the next significant barriers to the continued development of the field."
For years, Nationwide has produced gene therapies for its research at a small clinical facility. As Nationwide's work expanded, the hospital added space, increasing the number of production suites dedicated to viral vector manufacturing from one to three.
More recently, Durbin said, Nationwide's industry partners began asking the hospital if it could make preclinical or early-stage clinical product, requests that spurred Nationwide to consider launching Andelyn.
"Part of the reason we feel we can do this is because of our track record of success in doing it at the scale we've been doing it for several years," said Durbin, who joined Nationwide two years ago from the University of Pennsylvania. "We don't feel we're starting out from scratch here."
That track record includes a Phase 1 study led by researcher Jerry Mendell that proved the promise of a gene therapy for spinal muscular therapy, later taken forward by the biotech AveXis and approved as Zolgensma.
Other notable examples include two spinouts, Celenex and Myonexus, later acquired by Amicus Therapeutics and Sarepta Therapeutics, respectively. Sarepta has also licensed from Nationwide a Duchenne muscular dystrophy treatment that has since become the company's leading experimental drug.
Select biotech spinouts from Nationwide Children's
|Spinout||Disease focus||Lead drug||Bought buy|
|AveXis||Spinal muscular atrophy||Zolgensma||Novartis, for $8.7 billion|
|Celenex||Lysosomal storage disorders||Two programs in Batten disease||Amicus Therapeutics, for $100 million|
|Myonexus||Limb-girdle muscular dystrophy||MYO-101||Sarepta Therapeutics, for $165 million|
With Andelyn, Nationwide expects to offer manufacturing support from the start of clinical testing all the way through to a gene therapy's potential approval — something its current clinical facility can't do.
Durbin also hopes Andelyn will be able to keep pace with a field that's unlikely to look the same in several years' time.
"The manufacturing methods in this space are still what I would consider barely first-generation," he said. "So we anticipate continued improvements and innovation and we want Andelyn to be on the forefront."
Nationwide isn't the only academic center investing in gene therapy manufacturing. The Children's Hospital of Philadelphia, another focal point in gene therapy research, opened in late 2018 a clinical production site to make viral vectors. The facility, which accepts outside projects from industry as well as academia, is slated to begin full operations this year, a spokesperson for CHOP confirmed to BioPharma Dive.
But structuring Andelyn as a for-profit contract manufacturer appears unique, particularly from a not-for-profit hospital.
Durbin, though, doesn't see a conflict between the two, noting that Nationwide will reinvest some of the revenues from Andelyn back into its own research programs.
Additionally, by helping gene therapies advance, Andelyn could usher in new treatments for children, Durbin said. That would be in keeping with the company's name, which honors Andrew Kilbarger and Evelyn Villarreal, two children who participated in Phase 1 gene therapy studies at Nationwide.