Gene therapy experts TJ Cradick and Kate Excoffon discuss using AAV gene therapy for cystic fibrosis
Dr. Excoffon describes her academic experience that led her to the field and Spirovant Sciences, discusses interesting data from last month’s North American Cystic Fibrosis Conference, and then highlights Spirovant’s program and why using a small molecule modulator like doxorubicin might be the key to success.
The Experts
TJ Cradick, PhD is a CRISPR and gene editing expert with over 22 years of experience in cell and gene therapy research and management. In 2015, he joined CRISPR Therapeutics as the second employee and Head of Genome Editing. Subsequently, he served as the Chief Scientific Officer (CSO) at Excision BioTherapeutics, expanding his expertise in IND, regulatory affairs, intellectual property, and clinical trials.
Educated at MIT and UCSF, Dr. Cradick joined Sangamo Biosciences in 2000, designing Zinc Finger proteins and Zinc Finger Nucleases (ZFNs). At the University of Iowa, he developed the first nucleases that specifically cleaved Hepatitis B virus DNA, created bioinformatics tools, and published the Surveyor gene editing assay. As a faculty member at Georgia Tech and Emory, he conducted pioneering research on ZFNs, TAL effector nucleases (TALENs), and CRISPR, co-authoring highly cited publications and developed bioinformatic and machine learning tools such as PROGNOS, SAPTA, and COSMID. Additionally, the group initiated the hemoglobinopathies experiments with CRISPR Therapeutics that led to the development of Casgevy. Dr. Cradick's extensive experience also includes developing assays and computational methods for IND submissions and clinical trials. For more information and access to his talks, papers, and consulting projects, please visit GeneEditingFrontiers.com.
Kate Excoffon, PhD, molecular virologist and gene-therapy drug developer. With over 25 years of experience in gene therapy in the academic and biotech space, Kate’s passion is creating, building, and advancing viral vector-mediated gene therapy.
Kate is the VP of Research at Spirovant Sciences, Inc, a company focused on using AAV and lentivirus gene therapy for respiratory diseases, including SP-101, a novel AAV expressing the human CFTR gene plus doxorubicin. Kate oversaw the development of SP-101 from her initial discovery of the novel AAV capsid as a postdoc to the recently announced cystic fibrosis clinical trial (NCT06526923). Non-clinical studies associated with this clinical trial were recently published (see links below) which highlight the importance of enhancing expression from AAVs using doxorubicin, resulting in lower AAV doses. Spirovant is the first biotech company to include doxorubicin in their AAV clinical trial design.
Prior to Spirovant Sciences, Kate was a full professor at Wright State University, teaching numerous courses, discovering other novel AAVs, including ones able to transduce CD4+ T cells, understand the Coxsackievirus and adenovirus receptor (CAR), identify and patent anti-adenovirus molecules, and study adipose-derived stem cells for wound healing.
Kate started her career as a PhD student where she studied multiple different gene delivery modalities and including discoveries that led to Glybera, the first approved AAV gene therapy.
With over 50 publications, hundreds of presentations, numerous grants, patents, and trainees, she is a recognized expert in AAV, adenovirus, and lentivirus-based vectors and molecular virology.
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