The Office of Technology Commercialization at Nationwide Children’s Hospital, led by Matt McFarland, RPh, PhD, facilitates the transfer of new technologies, research and innovations to outside partners to benefit pediatric care, the local community and the general public.
Abeona Therapeutics, formed in early 2013 based on gene therapy technologies developed at Nationwide Children’s Hospital, is a clinical stage company initially focused on developing a cure for Sanfilippo syndrome, MPS IIIA and MPS IIIB, rare genetic disorders caused by the body’s inability to properly break down certain sugars. These diseases lead to progressive muscular and cognitive decline in children after the age of 2 years. With no cure or approved treatments, children with Sanfilippo syndrome usually die before the age of 20. Two separate multi-site phase I/II clinical trials for MPS IIIA and MPS IIIB are underway to evaluate the safety and efficacy of the treatment.
Andelyn Biosciences is a viral vector contract and development manufacturing organization. It was established in 2020 as a spin-out of Nationwide Children’s Hospital’s manufacturing division, which has expertise in manufacturing several adeno-associated virus (AAV) serotypes of gene therapy products.
AveXis, recently renamed Novartis Gene Therapies, is a commercial gene therapy organization developing treatments for patients with neuromuscular diseases, including Nationwide Children’s Hospital’s licensed programs for Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA), a motor neuron disease that is the leading genetic cause of death of children under the age of 2. The SMA gene therapy technology allows for the delivery of a replacement gene to target motor neurons throughout the brain and spinal cord. The gene therapy product for SMA, Zolgensma®, was approved in mid-2019 by the Food and Drug Administration.
Celenex is a clinical stage gene therapy company targeting Batten diseases and other genetic diseases. In late 2018, Celenex was acquired by Amicus Therapeutics, a biotechnology company focusing on rare and orphan diseases. Phase I/II clinical trials for Batten diseases CLN3 and CLN6 are underway at Nationwide Children’s Hospital.
Clarametyx Biosciences is developing a platform therapeutic for the eradication of bacterial biofilms which are responsible for approximately 80% of human bacterial infections. Their lead composition CMTX-101 is a monoclonal antibody that causes rapid biofilm collapse, enabling host immune clearance and potentiating antibiotic activity.
Deep Lens is augmenting VIPER, one of the world’s first digital pathology cloud platforms, to include new features. For over 10 years, VIPER has allowed pathology groups to collaborate on groundbreaking cancer research across dozens of cancer types. Based on feedback from hundreds of expert global users, Deep Lens is enhancing the system to include clinical trial enrollment, AI-powered image detection and workflow support, telepathology, cloud storage and built in APIs for integration by hardware and software vendors and biopharma companies.
ENTvantage Dx provides primary care physicians and otolaryngologists with rapid, in-office diagnostic tests to determine the cause of ear, nose and throat illnesses. The technology was developed as a result of the research collaboration between The Ohio State University and Nationwide Children’s Hospital, for rapid diagnosis of bacterial sinusitis. ENTvantage Dx is currently developing this technology to be used as point-of-care for patients with symptoms of sinusitis.
Little Seed, Inc. was formed in 2018 in Powell, Ohio, with the goal of delivering clinically driven, evidence-based fun to pediatric patients. The foundational technology, Voxel Bay, was developed by a team of clinicians and game designers at Nationwide Children’s Hospital. Voxel Bay provides an interactive virtual reality platform designed to distract and calm children undergoing uncomfortable medical procedures. The Voxel Bay VR platform is being expanded to include other virtual environments and games tailored to specific needs within the pediatric environment.
LYST Therapeutics, based in Columbus, Ohio, was founded in 2017 to develop a platform technology for treatment of fibrotic diseases. The technology, invented by researchers in the Center for Tissue Engineering at Nationwide Children’s Hospital, is a novel immunomodulatory therapeutic antibody and has potential applications in treating stenosis, myocardial infarction and other conditions involving fibrosis.
Milo Biotechnology was founded in 2012 to develop a therapy that would increase muscle strength and improve the quality of life of muscular dystrophy patients and is based on a discovery by scientists at Nationwide Children’s Hospital. The therapy uses an adeno-associated virus (AAV) delivered follistatin protein, which inhibits the activity of myostatin, a protein that impedes muscle differentiation and growth. Phase I/II clinical trials evaluating the safety and efficacy of Milo’s follistatin therapy in patients with Becker muscular dystrophy, Duchenne muscular dystrophy and Inclusion Body Myositis took place at Nationwide Children’s Hospital.
Myonexus Therapeutics, a startup formed in 2017, is a clinical stage gene therapy company developing first ever treatments for limb-girdle muscular dystrophy (LGMD) types 2D, 2B, 2E, 2L and 2C based on research at Nationwide Children’s Hospital, a leader in muscular dystrophy gene therapy discovery and translational research. In early 2019, Myonexus was acquired by Sarepta Therapeutics.
Scioto Biosciences was founded in 2017 to develop treatments for diseases associated with microbial dysimbiosis. The technology platform, developed by researchers at Nationwide Children’s Hospital, is a novel formulation that primes the colony-forming mechanisms of probiotic bacteria by combining beneficial bacteria with polysaccharide microspheres. These natural mechanisms induce biofilm formation, enhance probiotic function and allow for nonspore-forming bacteria to survive passage through the gastrointestinal system. Among the first therapeutic indications being pursued is necrotizing enterocolitis, a high-morbidity disease that affects 7% of premature births.
The Tasseogen platform relies on the genome dashboard technology developed at Nationwide Children’s Hospital to allow researchers or clinicians to upload genome sequencing data and interactively explore the data to identify gene variants, if any, that may be causal for a patient’s disease. Tasseogen and Nationwide Children’s Hospital RISI team are the recipients of a 2020 Ohio Development Services Agency Technology Validation and Startup Fund (TVSF) Phase I award.
Thrive Neuromedical, an Ohio-based startup based on technology developed at Nationwide Children’s Hospital is developing the SmallTalk™ platform to enrich the neurological development of babies who don’t have regular, consistent access to their parent’s voice. The SmallTalk™ device is a unibody Bluetooth-enabled speaker that transmits the parent’s recorded voice with the appropriate sound characteristics to provide a clinical, therapeutic effect.
Zotarix, LLC, is a Columbus-based startup focused on patient safety during surgical procedures. Their first-in-kind product is a disposable medical device which provides protection against thermal and physical injury to the patient’s lips during oral surgery.