List of Adeno-associated viral vector (AAV) Companies in Ohio - 7

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.

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.