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.
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.
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.
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.
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).
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).
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).
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).
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 Biomedical Genomics Core, and his team (March 17, 2015).
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 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).
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).
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, 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).
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.
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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).