Stephanie Cherqui Discusses Clinical Development of CRISPR Therapies for Rare Diseases

Imran House, Ph.D. and Junyun Lai Ph.D. are Senior Scientists at the biotech start-up, oNKo-Innate in Melbourne, Australia that focuses on developing immuno-oncology therapies. In this episode, they discussed what they do at oNKo-innate, their career transitions from leaving academics to entering the biotech industry, and how CRISPR is impacting cancer-immunotherapy development. They also discussed overcoming common obstacles faced when developing cytokine therapies and future outlooks on clinical trials using these kinds of therapies.   Reach out and connect with both Imran and Junyun on social media platforms: Imran House, Ph.D. LinkedIn X: @ImranHouse   Junyun Lai, Ph.D. LinkedIn X: @JunyunLai

Dr. Mark DeWitt Ph.D., Associate Director at Mammoth Biosciences, and Dr. Don Kohn M.D., distinguished professor and Director of the UCLA Human Gene and Cell Therapy Program and CIRM grantee sat down with us to discuss their latest clinical trials to cure sickle cell anemia, how synthetic guides accelerated their journey from the bench to the clinic, and their experience in the cell and gene therapy space.

Dina Simkin, Professor of Neurology at Northwestern University, joins us in this episode of CRISPR Cuts. Dr. Simkin talks about her path to becoming a researcher, art, and the importance of patient and isogenic iPSC models to study genetically linked forms of epilepsies.

Dr. Sunil Sharma and Dr. Raffaella Soldi of the Translational Genomics Research Institute chat about using CRISPR arrayed screens to identify novel drug targets for Ewing's Sarcoma, outlooks for the future of cancer therapies, and the Arizona heat.

Professor Stephanie Cherqui, UCSD, has worked on the development of cell therapies for two rare genetic diseases, cystinosis and Friedreich’s ataxia. In this interview, Dr. Cherqui chats about her experience working on rare diseases, developing a CRISPR-edited therapy for Friedreich's ataxia, navigating regulatory challenges in cell and gene therapies, and more.

Tune in to hear Christian Groendahl, CEO of SNIPR Biome, talk about CRISPR editing in prokaryotes, their novel E. coli-targeting CRISPR therapy trials, and his experiences and vision leading a start up.

In this episode, Bryan Dechairo, CEO of Sherlock Biosciences, talks about the impact of technology advances in the field of CRISPR diagnostics, his experiences leading Sherlock Biosciences, and his team’s mission of accessibility. 

Dr. Ayal Hendel, a genome-editing pioneer and group leader at Bar Ilan University in Israel, was one of the first researchers to use modified synthetic sgRNA to edit primary cells with CRISPR. In this episode, Dr. Hendel shares his perspectives on the recent CRISPR advancements and his work on the bubble boy disease.

Jimi has dealt with sickle cell disease, a genetic disorder with serious implications, his entire life. Undergoing a CRISPR gene editing trial turned his hope for a cure into reality with promising results from the early phases. Tune in to learn about Jimi's journey in this episode. 

TJ Cradick, Chief Scientific Officer at Excision Bio, discusses their CRISPR HIV therapy that recently proceeded into clinical trials in this episode and their ongoing work on other viral diseases.