Under the direction of Arun Srivastava, PhD, the Division of Cellular and Molecular Therapy, a part of the UF Department of Pediatrics, consists of a group of investigators having shared interests in basic molecular biology of adeno-associated virus, or AAV, and the development of the next-generation of AAV vectors, eminently suitable for human gene therapy. The division serves as a reference point for collating the incoming and outgoing ideas and materials relevant to the application AAV vectors in basic science research and clinical trials at UF, as well as throughout the scientific community.
The division houses four laboratories, the Arun Srivastava Lab, the Sergei Zolotukhin Lab, the Brad E. Hoffman Lab, and the Shannon Boye lab. Dr. Hoffan’s research group is developing gene immunotherapy to treat autoimmune diseases using the AAV gene therapy platform. For the past three decades, Dr. Srivastava’s research has focused on parvoviruses, the nonpathogenic AAV, and a common human pathogen called the parvovirus B19, in addition to the development of recombinant parvovirus vectors for human gene therapy. The primary mission of Dr. Zolotukhin’s lab is to merge molecular genetics research and health care by developing new therapeutic strategies involving gene transfer. The idea of gene therapy is a logical and natural progression of the last 20 years of research in medical genetics and molecular biology. Dr. Boye’s work focuses on developing viral vector-based gene replacement strategies for the treatment of inherited ocular disease.
We are proud of the work we have been able to accomplish over the past few decades. Here are some of our biggest accomplishments over the years.
- We developed optimized AAV serotype vectors, including capsid-modified next generation, or NextGen, genome modified generation X, or GenX, and a combination of the two.
- One of the NextGen AAV2 vectors was used successfully by others in a Phase I clinical trial in gene therapy of Leber Hereditary Optic Neuropathy in 2017.
- One of the NextGen AAV3 vectors will be used in a Phase I clinical trial for gene therapy of Hemophilia B in 2020.
- One of the NextGen AAV6 vectors will be used in a Phase I clinical trial for gene therapy of hemoglobinopathies in 2023.
- Additional Phase I clinical trials are anticipated to begin in 2021-2022.
- We are the co-founder of the following AAV gene therapy companies: Lacerta Therapeutics (2017); aaVective (2018); Nirvana Therapeutics (2018); and KASHX Bio (2018).
- We developed a preclinical AAV gene immunotherapy to treat autoimmune diseases such as multiple sclerosis.
- We established immunotherapy for such therapy under UF Tech #15158, “Re-establishing Immune Tolerance to Neuro-proteins By AAV Gene Therapy as Treatment for Multiple Sclerosis.” This technology has obtained patents in Australia and Japan, with the United States, Europe and Canada still pending.
- We collaborated with Sarepta Therapeutics in a more than $62 million licensing deal for UF Tech #15158 and additional sponsored research agreements for more than $1 million to continue developing AAV technology for the clinical treatment of multiple sclerosis.
- We are in the process of working on a potential partnership with Takeda.
- Brad Hoffman, Ph.D., received the 2018 Scientific Researcher Award from the North Florida Chapter of the National Multiple Sclerosis Society.