Startups
The Office of Technology Commercialization at Nationwide Children’s Hospital, led by Margaret Barkett, 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.
Armatus Bio is an emerging biotechnology company based in Ohio that is applying the rigor of advanced bioengineering to the complex challenge of genetic medicine. Through a dynamic collaboration with renowned gene therapy experts the company is building a pipeline of precision genetic-based therapies designed to overcome the limitations of today’s technologies and propel the next generation of medicines that could transform care for people with rare genetic neuromuscular disorders.
CARTx’s mission is to use our unique off-the-shelf CAR-T cell immunotherapy approach that genetically engineers γδT-cells using CRISPR/AAV to bring advanced therapies to people everywhere.
Cellula BioPharma is developing a game changing CAR-T platform technology for solid tumors. CAR T cell therapy has represented an exciting breakthrough in the treatment of patients with hematologic malignancies leading to the FDA approval of six CAR-T products. To date, >200 registered CAR T cell clinical trails against solid tumor showed that the antitumor activity of CAR-T in solid tumors is very disappointing.
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.
Genosera is a gene therapy biotechnology company that is revolutionizing the way diseases are treated. Founded in 2019, our mission is to develop cutting-edge gene therapy treatments for rare diseases and difficult disease applications. We believe that gene therapy is the key to unlocking better healthcare, and our team of experts is dedicated to developing safe, effective, and affordable treatments. We are passionate about our work and strive to make a positive difference in the lives of people from around the world inflicted with disease.
HyperPath Solutions, launched in 2021, is commercializing technology licensed from Nationwide Children’s to create an artificial intelligence (AI)-driven, cloud-based digital pathology whole slide imaging infrastructure to enhance research and healthcare. Their offerings include products and services to provide value chain management to academic partners and partners in the biotechnology and pharmaceutical industries.
Nationwide Children’s researchers developed a gene replacement therapy that aims to address the root cause of Type 1 galactosemia by delivering the functional GALT gene via the AAV9 vector. This investigational gene therapy is extensively licensed to Jaguar Gene Therapy.
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.
NephKey Therapeutics is focused on modifying existing therapeutics for use in the treatment of kidney disease in children. The company intends to protect development of a new formulation of an adult diabetes drug to treat patients with childhood nephrotic syndrome, using a 505(b)(2) new drug application (NDA) to the U.S. Food and Drug Administration (FDA). A large, multicenter clinical trial of this new formulation is under discussion with the FDA.
RxGames was developed collaboratively by therapists, scientists, and patients to create an engaging patient-focused model that therapists can use to deliver better outcomes, even when their in-clinic time together is limited by payor benefits, geography, and/or patient compliance. The games were developed over a period of eight years and are backed by clinical research. Our founders are proud to offer an innovative way to improve patients’ daily lives and contribute to better recoveries. The goal is to transform the way therapy is delivered - empowering the patient, enabling better treatment, and ultimately making therapists more effective.
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.
Ohio-based startup smallTalk is developing the SmallTalk™ platform to enrich the neurological development of babies who don’t have regular, consistent access to their parent’s voice. Based on technology developed at Nationwide Children’s Hospital, 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.
Storilly is a first-of-its-kind digital storybook innovation targeted toward helping children with hearing impairment develop speech and literacy skills. It is a software application, currently available in iOS and web access planned for 2025, enabling parents and speech-language pathologists (SLP) to partner together to help children who are hearing impaired achieve reading, speech and language goals. Utilizing storybook learning, the goal is to further stimulate language development at home to supplement office-based speech therapy. The startup recently received Phase II funding from the Ohio Third Frontier Technology Validation and Start-up Fund (TVSF) in October 2023. With this funding, Digital Story Therapies Inc., will continue developing the technology and incorporating digital storybooks from their partnership with Highlights for Children.
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.
Vironexis Biotherapeutics, Inc. is a preclinical stage company with clinical stage potential founded in 2021 to utilize gene therapy to achieve long-term, consistent bloodstream levels of protein therapeutics. The company is initially focused on developing immunotherapies for cancer. Their pipeline includes targeting various pediatric and adult blood and solid organ malignancies.
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.