News and Announcements
Technology Development Fund / Technology Validation and Start-up Fund Awards
Spring 2023 Awards
| Applicant | IDF | Category | Title |
| Sriram Vaidyanathan | 2022-060 | TDF | Chemically Modified Single Stranded DNA to Improve Gene Editing of Hematopoietic Stem Cells and T-Cells |
| Kathrin Meyer | 2020-028 | TDF | Virtual Surgical Planning 3D Modeling and 3D Printing in Treatment of Epilepsy |
| Zarife Sahenk | 2022-066 | TDF | NT-3 Gene Therapy for Multiple Sclerosis Using Experimental Autoimmune Encephalomyelitis Mouse Model |
| Gail Besner | 2023-005 | TDF | Software Augmented Morbidity and Mortality Conferencing |
| Vanessa Olbrecht | 2022-015 | TDF | Biofeedback-Based Virtual Reality for Pain Management |
| Karen Diefenbach | 2022-043 | TDF | Gastrostomy Stabilization Device for Low-Profile Gastrostomy Tube or Gastrojejunostomy Tube |
| Sudarshan Jadcherla | 2022-044 | TDF | Parent-Implemented Oral Nutrition, Eating and Esophageal Regulation (PIONEER) Products |
| Allen Kadado | 2022-071 | TDF | A Biomechanical Model for Surgical Correction of Scoliosis |
| James Reinhardt | 2019-051 | TVSF | Prototype development of a closed device for seeding tissue engineered vascular grafts with cells |
Fall 2023 Awards
| Applicant | IDF | Category | Title |
| Paul Martin | 2023-043 | TDF | Sialylation and Modified Sialic Acids on AAV Capsids as Mechanisms to Improve Tissue Transduction and Avoid Host Immune Responses |
| Nicolas Wein | 2016-069 | TDF | AAVs mediating exons 6, 7 and 8 skipping as a personalized medicine for DMD patients |
| Tendy Chiang | 2022-020 | TDF | Modular Adjustable Bioreactor For Partial Decellularization |
| Jenna Merandi | 2023-019 | TDF | Enhanced IV Line Clamp |
| Marcelo de Souza Fernandes Pereira | 2023-052 | TVSF | Engineering CAR NK Cells to Target Stromal Cells in Cancer and Fibrotic/Aging Diseases |
| Meisam Naeimi Kararoudi | 2023-035 | TVSF | Generation of Off-The-Shelf CAR-T cells for cancer immunotherapies: A revolutionary cell therapy product |
2023 Innovator of the Year: Jerry Mendell, MD
Duchenne Muscular Dystrophy (DMD), a severe form of muscular dystrophy caused by a mutation in the DMD gene and resulting in a lack of the dystrophin protein, leads to muscle weakness and atrophy. DMD is an X linked condition and, as such, typically affects boys. In 1969, Jerry Mendell, MD, was working at the National Institute of Neurological Disorders and Stroke (NINDS) when he saw his first patient with DMD, igniting his passion to develop new treatments for patients with neuromuscular diseases.
“This is what I’ve devoted my life to; since I began investigating gene therapy as a potential treatment for children with neuromuscular disorders, it’s been my dream to develop a gene therapy for DMD,” Dr. Mendell says.
In 1999, Dr. Mendell performed the first in-human clinical trial using AAV for gene transfer to skeletal muscle for LGMD2D/R3. Today, he has been involved in two of the first five AAV-based gene therapies approved by the Food and Drug Administration (FDA).
Dr. Mendell was the principal investigator in the study of the gene transfer clinical trial for spinal muscular atrophy type 1, which led to the development of Zolgensma®, the first systemically delivered gene therapy to receive FDA approval in 2019. In June 2023, the FDA announced the accelerated approval of SRP-9001/ELEVIDYS for children 4-5 years old with Duchenne muscular dystrophy.
Over his career, Dr. Mendell has been involved with 18 different Nationwide Children’s technologies and is listed on 408 patent applications resulting in 60 issued patents to date. Dr. Mendell was elected to the National Academy of Medicine in 2021, and he was the first recipient of the American Society for Gene and Cell Therapy (ASGCT)’s Jerry Mendell Translational Medicine Award. He has published more than 400 articles and authored books on skeletal muscle disease, peripheral nerve disorders, and gene therapy.
Prior to his retirement in October 2023, Dr. Mendell was an attending neurologist, principal investigator, and the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at Nationwide Children’s and professor of Pediatrics and Neurology at The Ohio State University. He is a senior advisor in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s and an emeritus professor of Pediatrics at The Ohio State University. Dr. Mendell has also joined Sarepta Therapeutics as a senior advisor, placing him in a position that will help move gene therapy for neuromuscular disease to its full potential.
2022 Innovator of the Year: Steven Goodman, PhD
Biofilms are treatment-resistant colonies of disease-causing bacteria that adhere to each other and to the body’s inner surfaces and fuel chronic and recurrent infections, such as those of the middle ears, sinuses and urinary tract. The organisms erect protective matrices of extracellular DNA that shield them from the immune system and antibiotics.
According to the Centers for Disease Control and Prevention (CDC), bacteria in this biofilm state are responsible for approximately 80% of human bacterial infections and up to 1,000 times more resistant to antibiotics than those that are “free-living.”
Biofilms often need to be collapsed before the bacteria within them can be completely cleared from the body.
Steven Goodman, PhD, a principal investigator in the Center for Microbial Pathogenesis at Nationwide Children’s Hospital, has been studying biofilms — investigating how proteins and DNA create the extracellular structures that protect bacteria and identifying ways to help the body dismantle them — for more than a decade. His research has led to the creation of innovative technologies with the potential to transform care.
A biofilm-disrupting technology developed by Dr. Goodman and his collaborators is licensed by Clarametyx Biosciences. In October 2022, Clarametyx announced that the U.S. Food and Drug Administration (FDA) accepted its Investigational New Drug (IND) application for a phase I clinical trial of its CMTX-101 antibody therapy. Dr. Goodman serves as co-chair of the Scientific Advisory Board for Nationwide Children’s startup Clarametyx.
Another area of Dr. Goodman’s research aims to capitalize on the staying power of biofilms. He helped develop a proprietary Activated Bacterial Therapeutics (ABT) platform technology, which focuses on enhancing biofilm production by beneficial bacteria to advance healing for a variety of diseases. It was licensed by Nationwide Children’s startup Scioto Biosciences, a clinical-stage biotech company devoted to innovative research and discovery in the microbiome therapeutics field for which Dr. Goodman was scientific founder. In May 2022, Scioto announced positive results in its phase Ib trial of lead asset SB-121 in patients with autism spectrum disorder, including treatment-associated improvements in behavioral assessments.
Dr. Goodman’s collaborative research embodies the spirit of innovation and discovery. To recognize his dedication, creativity and impact, on behalf of the OTC, Abigail Wexner Research Institute President Dennis Durbin, MD, presented Dr. Goodman with the 2022 Excellence in Innovation Award during the AWRI Annual Research Retreat on Nov. 16, 2022. He was also honored at the OTC’s annual Excellence in Innovation Awards Ceremony and Reception on Jan. 10, 2023.
2021 Innovator of the Year: Dean Lee, MD, PhD
Natural killer (NK) cells are lymphocytes, or white blood cells, and a component of the innate immune system. They release cytotoxic molecules to help destroy cancer cells and those that have been infected with invading pathogens. Activated NK cells can minimize the side effects and lifelong impacts of chemotherapy, improve outcomes for pediatric patients who receive bone marrow transplants and help those who are immunocompromised fight viral infections. Yet NK cells have historically been underutilized as immunotherapies. These cells are resistant to gene modification, and their clinical production has proved challenging, with existing approaches for collecting NK cells being prohibitively costly and allowing only low cell numbers to be infused.
Dean A. Lee, MD, PhD, a principal investigator in the Center for Childhood Cancer in the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital and director of the Cellular Therapy and Cancer Immunology Program at Nationwide Children’s, has aimed to combat those limitations through pioneering research and a dedication to inventing new therapies. Dr. Lee’s innovations allow NK cells to be produced as an off-the-shelf product, and his work has paved the way for ongoing translational research and improvements to care for patients with cancer.
In 2021, the Office of Technology Commercialization (OTC) at Nationwide Children’s, along with hospital and research leadership, named Dr. Lee Nationwide Children’s Hospital Innovator of the Year. Since joining Nationwide Children’s in 2016, Dr. Lee, who is also a physician in the Division of Hematology, Oncology & Blood and Marrow Transplant at Nationwide Children’s and director of the Cellular Therapy and Cancer Immunology Program at Nationwide Children’s and The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James), has filed:
- 23 Invention Disclosures with the Office of Technology Commercialization (OTC),
- 18 applications for Provisional Patents with the United States Patent and Trademark Office (USPTO),
- 12 International Phase Patent Cooperation Treaty (PCT) patent applications and
- 45 National Phase PCT patent applications.
The NK cell technologies developed by Dr. Lee’s lab were licensed by a start-up, CytoSen Therapeutics, in 2018. In 2019, CytoSen was acquired by Kiadis Pharma, and in 2020, Kiadis was acquired by Sanofi for $358M. This rapid series of acquisitions demonstrates the value of NK cell therapies. Dr. Lee was honored during the OTC’s Innovation Week with a special video presentation at the AWRI Annual Research Retreat.
Learn more about Dean Lee, MD, PhD, the 2021 Innovator of the Year
Enlighten Mobility Holds an Exclusive License Agreement with Nationwide Children’s Hospital to Develop GMat Neuromotor Screening Tool for Newborns
As the first automated non-invasive neuromotor screening for newborns, the GMat serves as an objective indicator for further evaluation by using artificial intelligence to detect patterns of variability the human eye and brain are unable to process. Studies utilizing the GMat on newborns are currently underway with the Early Detection and Intervention network of top U.S. neonatal intensive care units.
Designed by Nathalie Maitre, MD, PhD and a team of Nationwide Children’s researchers, the GMat accelerates the path to earlier timing and targeting of diagnosis to mitigate loss of vital development opportunities for all children.
Read the full Enlighten Mobility Press Release
AveXis Reports Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1
AveXis, Inc., a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, presented an interim analysis of data as of April 1, 2016 from the ongoing Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1. Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, presented the data at the 19th Annual Meeting of the American Society of Gene & Cell Therapy in Washington, D.C.
Read the full news release on GlobeNewswire ›
Abeona Therapeutics Doses First Patient in Phase 1/2 Trial for Sanfilippo Syndrome Type A (MPS IIIA)
Abeona Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing products for severe and life-threatening genetic diseases, announced on May 20, 2016 that the first patient was enrolled in a Phase 1/2 trial for ABO-102 (AAV-SGSH), a single treatment gene therapy strategy for patients with Sanfilippo syndrome type A (also known as Mucopolysaccharidosis Type IIIA or MPS IIIA) at Nationwide Children's Hospital.
Milo Biotechnology Featured in Gene Therapy Spotlight
At the 19th Annual Meeting for the American Society of Gene and Cell Therapy in Washington, DC, Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital, presented a second study from Milo Biotechnology on AAV gene therapy for follistatin for patients with muscular dystrophy.
Read the full Milo Biotechnology press release on FierceBiotech ›
Breakthrough Discovery on Treating Resistant Biofilm Infections Presented at Medtech Showcase in San Francisco
Steven Goodman, PhD, principal investigator in the Center for Microbial Pathogenesis at The Research Institute at Nationwide Children's Hospital and member of the scientific board for ProclaRx, spoke about a new technology developed by ProclaRx, an Ohio-based biotechnology company, to break down and destroy biofilms. Biofilms are a physical barrier which protect bacteria and prevent antibiotics and the body's immune system from eradicating chronic infections.
Read the full ProclaRx news release on PR Newswire ›
Nationwide Children's Hospital Uses Spinoff Technology to Best Genetic Sleuthing Companies in Contest to Help Undiagnosed Patients
A team from Nationwide Children's Hospital was honored as the winner of the 2015 international CLARITY Undiagnosed Challenge. The CLARITY Challenge involved 26 leading genomics teams from seven countries who sought to interpret DNA sequences and provide information to five different families about their conditions that had eluded diagnosis despite multiple doctor visits, tests and genetic studies. The Nationwide Children's team - the only team from a pediatric hospital - was unanimously chosen by an independent panel of judges that included medical geneticists, computer scientists, bioinformaticians and clinical experts (November 16, 2015).
Read more in Columbus Business First ›
Read the full news release in our News Room ›
Milo Biotechnology Receives 2015 Venture NEXT Award for Spinout from Rev1 Ventures
Milo Biotechnology was recognized as the most promising company based on a technology developed at one of Rev1 Ventures' Innovation Partners, The Research Institute at Nationwide Children's Hospital. Milo Biotechnology is developing a therapy that increases muscle strength or reduces the rate of muscle atrophy for treatment of muscular disorders. The company's technology, a gene therapy-based upregulation of the muscle-strengthening follistatin protein, was developed at and is exclusively licensed from Nationwide Children's Hospital (October 22, 2015).
Learn more about the award recipients at VentureNEXT.org ›
Data from Ongoing Study of AVXS-101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress
AveXis, Inc., a clinical stage gene therapy company developing treatments for rare and life-threatening neurological genetic diseases, presented data from its ongoing Phase 1/2 trial of AVXS-101, the company's initial product candidate for the treatment of spinal muscular atrophy (SMA) Type 1, at the International Congress of the World Muscle Society in Brighton, United Kingdom. Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital and lead investigator in the study, reported preliminary observations from the ongoing study (October 5, 2015).
Read more in the AveXis press release ›
AveXis, Gene Therapy Company, Secures $65 Million in Financing
AveXis, Inc., a leaeding gene therapy company developing treatments for rare and life-threatening neurological genetic diseases such as spinal muscular atrophy (SMA), completed a $65 million Class D common stock financing. These proceeds will be used to advance the company's ongoing SMA clinical program and expand key operational capabilities (September 8, 2015).
Read more in the AveXis press release ›
GenomeNext, Powered by Amazon Web Services and Intel, Achieves Unprecendented Throughput of 1,000 Genomes Analyzed per Day, Enabling Population-Scale Genomics
GenomeNext, LLC, a leader in genomic data management and integrated analysis, has benchmarked whole genome sequencing analysis at an unprecedented 1,000 genomes per day, through the “Intel Heads In The Clouds Challenge on Amazon Web Services (AWS)” with support from JHC Technology, and in conjunction with Nationwide Children’s Hospital. GenomeNext and Nationwide Children’s were challenged to perform analysis of the complete 1000 Genomes Consortium’s sequencing dataset in one week. This dataset consists of raw genomic sequence data from 2,504 individuals sampled across 26 different populations,and results of the analysis will be published in the coming months by Peter White, PhD, director of the Genomic Services Core Laboratory, and his team (March 17, 2015).
New Company, Lattice Biotech, and Nationwide Children's Join Forces to Attack Chronic Bacterial Infections
Lattice Biotech is leveraging a discovery that disrupts bacterial biofilms, creating a more effective environment for antibiotics to eradicate chronic infections. The research comes from Lauren Bakaletz, PhD, director of the Center for Microbial Pathogenesis in The Research Institute at Nationwide Children's, and Steve Goodman, PhD, principal investigator in the center. Biofilm infections, such as pneumonia in cystic fibrosis patients, chronic wound infections, chronic draining ear infections and infections caused by inadequately sterilized medical equipment, affect millions of children and adults throughout the world each year (March 11, 2015).
Rev1 Ventures Announces Landmark Investment in Nationwide Children's Hospital Spinout
Rev1 Ventures and Nationwide Children's Hospital have combined forces to help a promising life science spinout deliver a new gene-based therapy to treat muscular dystrophy and various other muscle disorders to improve the lives of patients with neuromuscular diseases. Rev1 will invest in Milo Biotechnology, the exclusive licensee of patented therapy deveoped at Nationwide Children's by Brian Kaspar, PhD and Milo co-founder, and Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute at Nationwide Children's (January 19, 2015).
Read more in the Rev1 Ventures blog post ›
Video Game Technology Helps Upper Extremity Movement in Patients with Muscular Dystrophy
Linda Lowes, clinical therapies research coordinator for the neuromuscular program, and Lindsay Alfano, physical therapist at Nationwide Children's, are part of a research team responsible for developing a way to measure upper extremity movement in patients with muscular dystrophy using interactive video game technology. Their hope is to expand inclusion criteria for clinical trials to incorporate patients using wheelchairs (January 12, 2015).
Ohio State, Nationwide Children's Hospital Partner with ENTvantage Diagnostics to Bring Rapid Sinusitis Diagnostic Test to Physicians
The first-of-its-kind diagnostic kit for in-office, rapid diagnosing of bacterial sinusitis was developed as a result of the research collaboration between Subinoy Das, MD, former director of The Ohio State University Sinus and Allergy Center and now adjunct Assistant Professor at The Ohio State University, and Lauren O. Bakaletz, PhD, director of the Center for Microbial Pathogenesis at The Research Institute at Nationwide Children's Hospital and professor in the Departments of Pediatrics and Otolaryngology at The Ohio State University College of Medicine. The jointly-created technology will be the foundation for ENTvantage Diagnostics, Inc. to develop a diagnostic kit to better equip physicians with additional information when making a bacterial sinusitis diagnosis (August 21, 2014).
Nanofiber Solutions Receives Award from Ohio Third Frontier to Further Develop Tissue Engineered Intestine Technology
Nanofiber Solutions, LLC, in collaboration with Gail E. Besner, MD, chief of Pediatric Surgery, program director of the Residency in Pediatric Surgery and co-director of the Burn Program all at Nationwide Children’s Hospital, recently was honored with the Ohio Third Frontier Technology Validation and Start-up Fund award. Earlier this month, Nanofiber Solutions, an Ohio-based start-up company housed in the TechColumbus incubator, received a $100,000 grant to further develop the tissue engineered intestine technology developed in the lab of Dr. Besner, who also is principal investigator in the Center for Perinatal Research in The Research Institute at Nationwide Children’s (July 1, 2013).
Research Informatics Core Honored at 2012 TechColumbus Innovation Awards
The Research Informatics Core at The Research Institute at Nationwide Children’s received the 2012 Outstanding Service (+250 Employees) Award at the 2012 TechColumbus Innovation Awards announced February 7, 2013.
Nationwide Children’s Hospital Partners with Silicon Valley to Market High-End Diagnostic and Medical Research Software
Nationwide Children’s and Transformatix Technologies, Inc. have partnered to create BioLinQ, a new biomedical informatics company designed to supply advanced software solutions for disease diagnosis and medical research (October 29, 2012).