Manufacturing the Future: From N=1 Personalized CRISPR Therapy to Scalable Precision Genomic Medicine
Baby KJ’s landmark treatment, the first personalized mRNA base editing therapy for a life-threatening urea cycle disorder, demonstrated that precision gene editing can save a single life. But the true obligation of medicine is scale: transforming singular breakthroughs into solutions that can reach all who need them.
This scientific presentation and panel will explore the intellectual, technical, and translational journey from that Nof1 clinical triumph to a platform capable of treating many patients. Achieving this vision requires a new model of partnership, one grounded in platform thinking and shared innovation. Optimized process development frameworks, scalable manufacturing, advanced QC analytics, and modern regulatory paradigms must converge to convert bespoke interventions into reproducible, accessible, and cost-effective genetic medicines.
Learning Objectives:
- Evolve one personalized mRNA base editing therapy into scalable, platform-driven solutions.
- Leverage process, manufacturing, and QC innovation to ensure consistency and efficiency.
- Algin partnerships and regulatory strategy to accelerate market readiness and access.
Speakers
Kiran Musunuru, MD, PhD, MPH, ML, Associate Professor of Cardiovascular Medicine and Genetics in the Perelman School of Medicine at the University of Pennsylvania
Kiran Musunuru, MD, PhD, MPH, ML, is an Associate Professor of Cardiovascular Medicine and Genetics in the Perelman School of Medicine at the University of Pennsylvania. Dr. Musunuru studied and trained at Harvard University, Weill Cornell Medical College, The Rockefeller University, Brigham and Women’s Hospital, Johns Hopkins University, and University of Pennsylvania. His research focuses on the genetics of heart disease and seeks to identify genetic factors that protect against disease and use them to develop therapies to protect the entire population. In his recent work he has been using gene editing to create a one-shot “vaccination” against heart attacks.
Dr. Musunuru is an actively practicing cardiologist as well as a committed teacher. He is a recipient of the Presidential Early Career Award for Scientists and Engineers from President Barack Obama at the White House, the American Heart Association’s Award of Meritorious Achievement, the American Philosophical Society’s Judson Daland Prize for Outstanding Achievement in Clinical Investigation, the American Federation for Medical Research’s Outstanding Investigator Award, and Harvard University’s Fannie Cox Prize for Excellence in Science Teaching. He recently served as Editor-in-Chief of the scientific journal Circulation: Genomic and Precision Medicine.
Patricia L Musolino MD, PhD, Associate Professor of Neurology Harvard Medical School Neurocritical Care and Vascular Neurologist Massachusetts General Hospital Center for Genomic Medicine
Patricia Musolino MD, PhD is a critical care and vascular neurologist with expertise in neurogenetics and vascular neurology. She cares for patients in the Intensive Care Unit, the Emergency Department and the Pediatric Stroke Clinic. Dr. Musolino's research focus on understanding how brain inflammation and stroke occurs when patients carry specific genetic mutations. Dr. Musolino is also part of multiple therapeutic clinical trials in stroke and rare disorders.
As a former Mass General Brigham Neurology residency graduate, she stayed at MGH for neurocritical care fellowship and she is currently junior faculty in the divisions of Neurocritical Care, Stroke and Child Neurology. She cares for patients in the Neuroscience Intensive Care Unit, the Emergency Department and is the Co-director of the Pediatric Stroke and Cerebrovascular Service. Dr. Musolino clinical and research expertise revolve around the translation of discoveries in human genetics to clinical application in cerebrovascular and neuroinflammatory disorders. Training in neuroscience, brain imaging techniques and molecular biology acquired during her MD, PhD and post-doctoral education has equipped her with the tools necessary to translate discoveries from to the bench to the bedside. Her laboratory work focuses on leveraging insights from neuroimaging and gene-editing tools to understand how single genes mutations alter blood brain barrier and cerebrovascular function and to identify better therapies to improve the quality of life of patients with monogenic vascular disorders. This novel approach has already led to the development of a pre-clinical in-vitro high-throughput drug screening platform and the design of a phase-II clinical trial. Dr. Musolino's work has been recognized by several awards including the Child Neurology Society, the Hearst and Child Neurology Foundations and continues to be supported by NINDS through a K08 Career Development Award.
Tom Foti, Vice President, Scientific Partnerships, Aldevron
Tom Foti co‑founded Aldevron’s Protein Business Unit in 2009 and currently serves as Vice President of Scientific Partnerships. In this role, he collaborates with innovators across the biotechnology landscape to accelerate scientific programs and strategic initiatives.
Previously, Tom spent 16 years as General Manager and Vice President of Aldevron’s Protein Business Unit, where he led protein development and manufacturing services supporting research, diagnostic, and therapeutic applications. Based in Madison, WI, he helped shape the division into a global provider of high‑quality protein solutions.
Tom played a pivotal role in creating and expanding Aldevron’s CRISPR product portfolio. From off‑the‑shelf reagents to custom GMP manufacturing, he and his team have supported clients in advancing gene‑editing programs from early concept through clinical development.
Tom earned a Bachelor of Science in Biotechnology and Microbiology from North Dakota State University, where he also played collegiate basketball. He holds an MBA from Edgewood College and a Management Leadership Certificate from the Massachusetts Institute of Technology.
Sadik Kassim, Ph.D., Chief Technology Officer, Life Sciences Omics Solutions Group
Sadik Kassim, Ph.D. is a biotechnology executive and scientist specializing in cell and gene therapy, bioprocessing, and translational research. He currently serves as CSO/CTO of Genomic Medicines for Danaher's Life Sciences companies. Previously, he was CTO at Vor Bio, where he built the technical operations team and led development for a CRISPR-edited HSPC product, as well as the company’s preclinical CAR-T efforts. Before that, he was Executive Director at Kite Pharma, leading manufacturing process development for autologous CAR-T and TCR-based therapies. As CSO at Mustang Bio, he oversaw the company’s preclinical and manufacturing foundation. He also led early analytical development at Novartis’ Cell and Gene Therapies Unit.
Sadik has contributed to the development of three commercial CAR-T therapies—Kymriah, Yescarta, and Tecartus—and, most recently, to K-abe, the world’s first patient-specific, custom gene-edited drug product, developed and administered in a record-setting six months. He completed postdoctoral training at the National Cancer Institute (with Dr. Steven Rosenberg), the University of Pennsylvania Gene Therapy Program (with Dr. Jim Wilson), and Johnson & Johnson’s Immunology Discovery group. He holds a B.S. in Cell and Molecular Biology from Tulane University and a Ph.D. in Microbiology and Immunology from Louisiana State University.
Venkata Indurthi, Ph.D., Chief Scientific Officer, Aldevron
Venkata Indurthi, PhD, is Aldevron’s Chief Scientific Officer, where he provides essential leadership and structure while focusing on strategic opportunities to further strengthen our position across all platforms with a special emphasis on RNA-focused developments.
Indurthi earned a Bachelor of Science in Biotechnology from SRM University, Chennai, India, and his Ph.D. in Pharmaceutical Science from North Dakota State University, Fargo, N.D. He has published several scientific papers and has presented at multiple conferences and symposiums.
Indurthi has been a member of the Aldevron team since 2016, holding a variety of positions that increased in responsibility and focus across production, quality and R&D.
Scott Ripley, Vice President & General Manager, Nucleic Acid Therapeutics & Nanomedicine, Cytiva
Scott Ripley is a seasoned biotechnology executive with deep expertise in commercial strategy, bioprocessing, and advanced therapeutics. With over two decades in the industry, he has led global commercial teams and driven market growth across bioprocessing, nucleic acid therapeutics, and more recently, nanomedicine.
As Vice President of Nucleic Acids and Nanomedicine at Cytiva, he oversaw the integration of Precision Nanosystems into Cytiva, and is expanding solutions for mRNA, lipid nanoparticles, and advanced therapies.
Previously, he held senior leadership roles at GE Healthcare, including Global Marketing Director for Bioprocess and, where he played a key role in advancing biomanufacturing capabilities supporting both strategy and marketing demand generation.
Beyond his corporate role, he serves as Chairperson of the Board at Testa Center, fostering biotech innovation in Sweden. With a Ph.D. from the University of Cambridge, Scott is passionate about translating cutting-edge science into real-world impact, advancing next-generation therapeutics for global healthcare.
Scott is currently based in Uppsala, Sweden.
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