Graphite Bio Announces Formation of Scientific Advisory Board
March 23 2022 - 8:00AM
Business Wire
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage,
next-generation gene editing company focused on developing
therapies that harness targeted gene integration to treat or cure
serious diseases, today announced the formation of its Scientific
Advisory Board (SAB), which is initially comprised of four experts
in stem cell and cancer biology, cell and gene therapy, hematology
and immunology. The SAB will provide strategic and scientific
counsel as the company advances its next-generation gene editing
platform technology and research and development programs.
“We are honored and excited to have this group of esteemed
scientists and clinicians join us as we work to leverage our
platform technology, which harnesses the power of high-efficiency
homology directed repair, to develop potential one-time curative
therapies for genetic and serious diseases,” said Jane Grogan,
Ph.D., chief scientific officer of Graphite Bio. “These scientific
thought leaders have decades of experience in their respective
fields, and their input and feedback will be invaluable as we work
to advance our goal of transforming critical components of the gene
therapy treatment paradigm – from discovery to development and
manufacturing to delivery – so that as many patients as possible
can benefit from these individualized therapies.”
The founding members of Graphite Bio’s SAB include:
- Maria Grazia Roncarolo, M.D., SAB Chair – Dr. Roncarolo
is one of the world’s foremost experts in gene therapy and one of
Graphite Bio’s academic founders. She is recognized globally for
her leadership in translating scientific discoveries in genetic
diseases and regenerative medicine into novel patient therapies,
including the world’s first ex vivo gene therapy. The George D.
Smith Professor in Stem Cell and Regenerative Medicine, Professor
of Pediatrics and of Medicine at Stanford University, Dr. Roncarolo
established the Stanford Center for Definitive and Curative
Medicine to cure patients with currently incurable diseases through
the development of innovative stem cell and gene-based therapies.
During her earlier tenure as director of the Telethon Institute for
Gene Therapy at the San Raffaele Scientific Institute in Milan, Dr.
Roncarolo developed novel approaches in gene therapy. Her work at
the Institute led to the discovery of ex vivo gene therapies for
genetic diseases of the immune system, including ADA-SCID and WASP,
and metabolic diseases such as metachromatic leukodystrophy. The
landmark stem cell gene therapy treatment for ADA-SCID was the
world’s first to be approved by the European Medicines Agency (EMA)
under the brand name Strimvelis® in May 2016.
- John E. Dick, Ph.D. – Professor Dick is a Canadian
researcher in stem cell biology and senior scientist at the
Princess Margaret Cancer Centre and McEwen Stem Cell Institute,
University Health Network; and professor of molecular genetics at
the University of Toronto. He is recognized for identifying and
characterizing normal and leukemia human hematopoietic stem cells.
His lab provided direct evidence for the cancer stem cell
hypothesis, transforming views of the origin and nature of cancer
and laying the foundation for new approaches to cancer therapy. In
recognition of his seminal contributions to the fields of molecular
hematology, stem cell biology and oncology, he was elected as a
foreign member of the National Academy of Medicine (USA), a fellow
of the Royal Society of Canada, the Royal Society of London, UK,
and the AACR Academy. He has received numerous prestigious awards,
including the Dameshek Prize, Thomas Prize and Mentor Award from
the American Society of Hematology; the Clowes Memorial Award and
the AACR-Pezcoller Prize from the American Association for Cancer
Research; and the Keio Medical Science Prize.
- Natalia Gomez-Ospina, M.D., Ph.D. – Dr. Gomez-Ospina has
dual appointments in the Divisions of Medical Genetics and Stem
Cell Transplantation and is a faculty member in the Institute for
Stem Cell Biology and Regenerative Medicine at Stanford University
School of Medicine. She also leads the Program for Inherited
Metabolic Disorders at Stanford, which brings together basic
scientists and clinicians to facilitate the development of
therapies for metabolic diseases. A physician-scientist and medical
geneticist, Dr. Gomez-Ospina is conducting research to develop
safer, more effective therapies for lysosomal storage disorders.
Her clinical and research interests bridge genetics and
transplantation. She established an adaptable platform for treating
lysosomal enzyme deficiencies and performed first-of-its-kind
preclinical studies to support the clinical development of
autologous transplantation of genome-edited cells to treat patients
with mucopolysaccharidosis type I (Hurler syndrome) and Gaucher
disease. Dr. Gomez-Ospina led the discovery and characterization of
genetic syndromes, including the infantile cholestasis syndrome
caused by mutations in the bile acid receptor. She is the lead
author of research published in The New England Journal of
Medicine, Cell, Nature Communications and the American Journal of
Medical Genetics. She earned an M.D. and a Ph.D. in chemical and
systems biology from Stanford University School of Medicine.
- Scot A. Wolfe, Ph.D. – Dr. Wolfe is a professor in the
Department of Molecular, Cell and Cancer Biology (MCCB) at the
University of Massachusetts Chan Medical School. His research is
focused on genome editing systems and protein-DNA interactions. His
group is developing improved tools for targeted genome modification
and gene regulation, with the goal of increasing their precision
and effectiveness for therapeutic translation. This research has
spanned developing a variety of improved CRISPR-based genome
editing systems and new methods for engineering the DNA-binding
specificity of zinc finger proteins. His team is focused on the
therapeutic translation of their improved genome editing and gene
regulation systems to specific disease applications, such as sickle
cell disease, beta-thalassemia, various forms of muscular dystrophy
and Hermansky-Pudlak syndrome. He has published more than 65
research articles in peer-reviewed journals. He earned a Ph.D. at
Harvard University and completed his postdoctoral research at the
Massachusetts Institute of Technology.
About Graphite Bio Graphite Bio is a clinical-stage,
next-generation gene editing company harnessing high efficiency
targeted gene integration to develop a new class of therapies to
potentially cure a wide range of serious and life-threatening
diseases. Graphite Bio is pioneering a precision gene editing
approach that could enable a variety of applications to transform
human health through its potential to achieve one of medicine’s
most elusive goals: to precisely “find & replace” any gene in
the genome. Graphite Bio’s platform allows it to precisely correct
mutations, replace entire disease-causing genes with normal genes
or insert new genes into predetermined, safe locations. The company
was co-founded by academic pioneers in the fields of gene editing
and gene therapy, including Maria Grazia Roncarolo, M.D., and
Matthew Porteus, M.D., Ph.D.
Learn more about Graphite Bio by visiting www.graphitebio.com
and following the company on LinkedIn.
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