Session Abstract – PMWC 2022 Silicon Valley


The last 10 years have been a breakout decade for CRISPR across research and medicine, and the next decade holds the potential for CRISPR to cure the underlying cause of genetic disease. This session will discuss the latest breakthroughs in the field and address the challenges that need to be overcome to deliver on the promise of CRISPR.

 Session Chair Profile

Ph.D., CTO and Co-Founder, Mammoth Biosciences

Biography
Janice Chen is co-founder/CTO of Mammoth Biosciences, a biotechnology company based in the San Francisco Area that harnesses the diversity of nature to power the next generation of CRISPR products across diagnostics and therapeutics. Through its discovery of novel CRISPR systems, the company enables the potential of its platform to read and write the code of life. Janice received her PhD from the lab of Nobel Laureate Jennifer Doudna at University of California, Berkeley. She investigated mechanisms of CRISPR proteins and developed technologies leading to multiple papers and patents, and co-invented the programmable CRISPR-based detection technology called DETECTR®. Janice was selected as a Forbes 30 Under 30 in Healthcare, Business Insider's 30 Under 40 in Healthcare, Endpoints Top 20 Women in Biopharma, MIT Technology Review 35 Innovators Under 35, EY Entrepreneur Of The Year, SF Business Times Most Influential Women, and delivered a TEDx talk on the potential for CRISPR to democratize diagnostics.


 Speaker Profile

Ph.D., Professor, Molecular and Cell Biology Department, UC Berkeley

Biography
In his work at Sangamo Therapeutics (2000-16), Fyodor co-developed and co-named human genome editing at native loci with engineered nucleases, and co-led efforts to develop its fundamental toolbox (gene correction, knockout, and integration). Fyodor then led collaborative teams to establish at-scale applications of genome editing for human somatic cell genetics and model animal and crop reverse genetics. Fyodor was a key member of the team that developed the first-in-human application of genome editing (2009), and then led a cross-functional team through basic discovery and to IND of the first-in-human clinical trials for the hemoglobinopathies, beta-thalassemia and sickle cell disease targeting the erythroid enhancer of BCL11A (as of Nov 2021 with 4 subjects free of disease symptoms post-dosing, and 22 subjects major-symptom-free on the follow-up clinical trial for the same target using Cas9). Fyodor co-led efforts to develop the fundamental toolbox of human epigenome editing as a disease therapeutic (2000-2003), and then co-led a team that developed in vivo epigenome editors for Hungington's disease and Tau dementia (2010-2016). At the IGI, Fyodor’s focus is on establishing turnkey editability of the human genome and epigenome for clinical use, and building and leading collaborative teams to first-in-human applications of experimental CRISPR-based editing therapeutics for sickle cell disease and genetic disorders of the immune system, as well as epigenome editing therapeutics for radiation injury, neuroinflammation, and neurodegeneration. Fyodor is an author on 98 scientific publications and a named inventor on 75 issued US patents related to genome editing and targeted gene regulation technology.


 Speaker Profile

Ph.D., Co-Founder and Chief Scientific Officer, Cardea Bio

Biography
Dr. Aran’s current research focuses on creating novel electronic biosensors with true multiomic detection capabilities and applying them to solve real-world problems. She invented CRISPR-ChipTM, which utilizes CRISPR and biology-gated transistors to create the only technology that rapidly detects genes of interest without amplification. CRISPR-Chip™ was the cover of Nature Biomedical Engineering in 2019, featured in over 50 news outlets, and the most read paper that year. CRISPR-Chip™ has since been expanded to detect single-point mutations - published in Nature Biomedical Engineering in 2021. Through Cardea Bio, Dr. Aran’s inventions are being actively developed into commercial products to ensure a lasting impact beyond the academic world. Her research efforts have been recognized by Clinical OMICs 10 under 40 Award, Athena Pinnacle Award, and Nature Research Awards for Inspiring Women in Science. Dr. Aran was also the recipient of the NSF Career Award to develop the next generation of electronic sensors.


Clinical & Research Tools Showcase:
Cardea Bio

Being able to use Biology as Technology will result in a new generation of natural resources and advanced Powered by Cardea products that have until now been impossible.

All-electronic Infrastructure Leads Way to Real-time Multiomics
A critical challenge faced by systems biology is that multiomics is crippled by the current optical and static approaches all requiring numerous systems. Our all-electronic system unifies multiomics through direct and real-time capture of interactive streams of actionable multiomic data.

 Speaker Profile

Ph.D., CSO, EVP, Intellia Therapeutics

Biography
Laura Sepp-Lorenzino, Ph.D. leads Research and Early Development at Intellia Therapeutics. Before joining Intellia, Laura was at Vertex, Alnylam, Merck and Memorial Sloan-Kettering. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina and both her M.S. and Ph.D. in Biochemistry from New York University. Laura is a member of the BOD of Taysha Gene Therapies, the Alliance for Regenerative Medicine and the Oligonucleotide Therapeutics Society, and sits on the SABs of Thermo Fisher Scientific, Zymergen, and the U.K. Nucleic Acid Therapy Accelerator.


 Speaker Profile

M.D., M.Phil., CEO, Graphite Bio

Biography
Dr. Josh Lehrer is CEO of Graphite Bio, where he bridges his clinical background as a practicing physician with decades of experience at global biopharmaceutical companies leading businesses and high-functioning teams across all stages of drug development. Before joining Graphite Bio, he was CMO of Global Blood Therapeutics, served in leadership roles at Genentech in clinical development and business development, and held attending physician roles at Stanford University Medical Center and the Palo Alto Veteran’s Affairs Health System. Dr. Lehrer earned his Doctor of Medicine at the UCSF School of Medicine and completed his residency at UCSF in internal medicine. He holds an A.B. in Biochemical Sciences from Harvard University and a Master of Philosophy in Biological Sciences from the University of Cambridge. Dr. Lehrer served as a clinical and postdoctoral fellow in cardiovascular medicine at Stanford University and attended the Institute for Entrepreneurship at the Stanford Graduate School of Business.