List of Gene Therapy Companies in Ohio - 15

Andelyn Biosciences is a gene therapy CDMO dedicated to Turning Hope into Reality™. Our capabilities span viral vector process and analytical development, adherent and suspension based GMP drug substance manufacturing up to 2,000L, as well as GMP plasmid process development and GMP manufacturing. We are located in Columbus, OH, USA. With more than 20 years of pioneering experience in viral vector and vaccine manufacturing, Andelyn offers its clients direct access to globally recognized thought leaders with extensive expertise. We have produced GMP material for over 75 worldwide clinical trials in addition to over 450 cGMP clinical batches and more than 2,000 research-grade productions. Andelyn provides research and tox grade plasmids with an additional offering of GMP plasmid manufacturing, advanced quality systems, full regulatory support, semi-automated to automated fill stations, and supply chain vertical integration. Our 180,000 sq ft clinical and commercial manufacturing facility comprises 16 upstream production suites for customization of new and tech transferred programs. Andelyn offers clinical through commercial-scale capabilities that will help accelerate the development and manufacturing of innovative therapies to bring more treatments to more patients.

Armatus Bio is a biotechnology company that specializes in precision gene therapy for raising expectations in FSHD.

Athersys is a clinical stage biopharmaceutical company with a growing pipeline of highly differentiated, potential best in class therapeutics to treat significant and life-threatening diseases. We are engaged in the discovery and development of innovative new medicines designed to extend and enhance the quality of human life. Through the application of our proprietary technologies, we have established a pipeline of therapeutic product development programs in multiple disease areas. This pipeline includes both stem cell therapy and pharmaceuticals.

Avantor® is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. Our global footprint enables us to serve more than 300,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries. We set science in motion to create a better world. More than 14,500 strong, our associates are passionate about our mission to set science in motion to create a better world. We share enthusiasm for innovation, excellence, and achievement. Whether we are collaborating with our customers to advance science or solve multifaceted problems, we help them reach their goals more efficiently and effectively. Visit our website to learn more about Avantor.

BioHeights LLC specializes in business strategy for novel cell and gene therapies. We help companies, startups, and academic institutions.

Case Western Reserve University is a Higher Education institution that offers research and academic programs.

One of the few FDA-licensed cord blood centers in the U.S., the Cleveland Cord Blood Center (CCBC) collects, processes, stores and distributes stem-cell rich umbilical cord blood for transplantation in patients with life-threatening disorders.

Eikonoklastes Therapeutics is an early stage biopharmaceutical company focused on rapidly advancing innovative medicines for numerous areas of significant unmet clinical need, starting with neurodegenerative diseases. We are iconoclasts who are passionate about finding new and better ways to treat disease. Our first-in-class gene therapy moves beyond single defective gene replacement. It is a transformational, disease-modifying gene supplement therapy with a unique mechanism of action that provides extra copies of a critical gene to cause overexpression of a critical neurotrophic factor that is essential for neuromuscular signaling and function. Our initial focus is on Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s Disease), a rare, yet debilitating nerve disease that causes progressive muscle degeneration and eventual death. An estimated 5,000 new patients in the U.S. are diagnosed each year with ALS, and there are few treatment options beyond supportive care. Our dynamic discovery and development approach allows us to focus on accelerated approvals and outsized returns for ALS and potentially other neurodegenerative diseases such as Parkinson’s, Huntington’s, Alzheimer’s, traumatic brain injury, and more.

We are a specialty CDMO for LV vector manufacture & use. Our three platforms: Expression Cassette Optimization (ECO), LentET (3rd gen system), & LentET Express (cGMP mfg.) enable GMP LV manufacture 10 months from sequence submittal. ECO increases expression up to 8 fold. LentET plasmids are ECO optimized, have improved safety, produce potent LVs & may be available royalty/licensing free depending upon the specifics. With full CMC & regulatory support, Expression is your preferred CDMO of choice.

Forge is a gene therapy development engine, focused on enabling access to life changing gene therapies and helping bring them from idea into reality. We partner with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups. We bring expertise in gene therapy manufacturing and therapeutics development to the table, helping champion teams that are navigating the long road from the lab bench to the bedside. With a patients-first approach, we are forging new models for working together to better accelerate these transformative medicines to reach those who need them most.

Genosera is revolutionizing the gene therapy field with its next generation gene therapy treatments that can help stop the progression of diseases and allow patients to regain their health. Founded in 2019, Genosera is a privately held, gene therapy biotechnology company located in Columbus, Ohio.

Heidelberg University is a higher education institution offering a variety of academic programs and research opportunities.

Orange Grove Bio was established with a clear vision: to bridge the gap between promising research programs and the successful development and commercialization of transformative therapies. Our company is driven by the recognition of the disparity in venture capital investment across different regions of the United States, often resulting in groundbreaking discoveries going unexplored and potentially life-changing treatments failing to reach the patients who need them most. Based in Cincinnati, we strategically partner with prominent institutions and researchers across the Midwest, Southeast, and Mid-Atlantic regions. By fostering strong partnerships built on a shared commitment to innovation, our aim is to ensure that discoveries from these regions translate into life-changing treatments for patients, unlocking the full potential of the most promising research programs.

University Hospitals is a healthcare provider that offers medical services and patient care.