Wednesday May 8, 2019
Emerging Techniques For The Next Generation of Candidates to Catalyze Preclinical Research in Genetic Kidney Disease
9:00am – 12:00pm
As emerging techniques and technologies gain increasing
momentum, the potential to refine drug development with
improved translation and greater efficacy is exciting for
multiple disease-specific communities, including CKD.
Hear from these world-class leaders on:
• The discovery, development and use of adeno-associated
virus vectors for gene therapy of genetic diseases
• Harnessing CRISPR/Cas9 to phenotypically screen
kidney disease and improve in vitro dish models
• Outlining the potential for kidney regenerative medicine
with stem cells
Massachusetts Medical School
Research in the Gao Lab primarily involves the discovery, development and use of adeno-associated virus vectors for gene therapy of genetic diseases and the study of miRNA functions in mammals. The lab works on isolation, characterization and vectorology of novel AAV vectors from primate tissues, molecular mechanisms of AAV evolution and diversity, and molecular interactions between endogenous AAV, AAV vector, host genomes and innate RNAi defense pathways.
Assistant Professor, Division
University of Washington
The goal of the Freedman Lab is to apply the prize-winning technologies of iPS cells and CRISPR gene editing to establish ‘clinical trials in a dish’ and regenerative therapies for kidney disease. To do this, we are combining human kidney organoids that we have invented with liquid handling robots. A major focus is polycystic kidney disease (PKD), a leading cause of kidney failure.
Initiatives to Accelerate Clinical Development Programs in
the Cardio-Renal Space
1:00pm – 4:00pm
With the prevalence of end stage renal disease in the at-risk
patient population continuing to increase; new therapeutic
options are necessary to address CKD as a prominent and
growing unmet medical need.
This expert led session will help you to:
• Overview of the current and emerging unmet medical
needs in the at risk renal patient population
• Benchmarks for drug development: time and costs
• Initiatives to accelerate renal drug development
programs: applications from translational medicine, real
world evidence and point-of-care testing
John Brennan is the Principal Consultant in a pharmaceutical industry consulting service based in Illinois. He has broad experience in all phases of of global drug development with special interest in the application of translational biomarkers, point-of-care testing and strategic study analytics. He has more than 30 years of Pharma industry experience at Johnson and Johnson (Ortho), Bristol Myers Squibb, Solvay Pharma and AbbVie.
Chief Medical Officer
Dr. Czerwiec is Chief Medical Officer of Goldfinch Bio, “the kidney company” based in Cambridge MA. In this role, he is responsible for leading all aspects of clinical development in partnership with the CEO, CSO and CBO/COO in driving the overall strategy for the company’s current and future R&D pipeline. Dr. Czerwiec comes to Goldfinch Bio from Otsuka Pharmaceutical Development & Commercialization, Inc, where Dr. Czerwiec’s team was responsible for the approval of tolvaptan (Jynarque TM), the first treatment approved to delay chronic kidney disease progression in autosomal dominant polycystic kidney disease (ADPKD) and the first US-FDA approved drug for treating an important form of CKD in the past decade.