A Company with a Rich History

The road to the formation of Andelyn Biosciences started more than 15 years ago on the third floor of a research building at Nationwide Children’s Hospital.

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 houses Andelyn Biosciences.

In 2020, Andelyn was spun out of the Nationwide Children’s Hospital as a for-profit Contract Development and Manufacturing Company.

The First Gene Therapy Approved by the FDA

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.

Andelyn Launch; Expansion into Phase III and Commercial

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 for profit Cell and Gene CDMO in January 2020.

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 make today’s gene therapies possible and reminding us that patients are at the heart of everything we do.

In November 2020, Andelyn broke ground on a new 185,000 square-foot commercial scale gene therapy manufacturing plant in the Ohio State University’s Innovation District in Columbus.

This new facility, opening in 2022, will allow Andelyn to expand its services to include Phase III and commercial manufacturing and become a full spectrum CDMO. Clients can now stay with Andelyn experts through all phases of development and commercialization, eliminating the need for tech transfers. This saves clients time to market, and shortens the wait for patients who have run out of options and are hoping for a breakthrough treatment to enhance or extend their lives.

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 lab expansion in Dublin – called the Andelyn Development Center – to help meet the growing demands of our clients.

The Evolution of Andelyn

NCH builds a vector manufacturing core facility and begins investigational gene therapy for Duchene Muscular Dystrophy.
Release of first gene therapy product for clinical use.
Experimental gene therapy for spinal muscular atrophy.
9000 sq. foot cGMP clinical manufacturing facility opened.
Expansion of offering to include research-grade plasmids.
Andelyn announced the addition of a 200L bioreactor to further enhance manufacturing capabilities and that it is expanding to phase III manufacturing capabilities.
Begin offering clinical grade plasmids.
Andelyn executes the completion of a new state-of-the-art cGMP manufacturing facility to fulfill client demand and become a full, turn-key gene therapy CDMO from conceptual development through commercial production. Andelyn will also have commercial grade plasmids capabilities.

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