We’re a gene editing company committed to cures

Our vision: A future where the word ‘incurable’ no longer exists.

Striving to make ‘one dose, one cure’ a reality

We are a next-generation gene editing company driven to discover and develop cures for patients living with serious diseases. In our pursuit of this better future for patients, their loved ones, and our communities, we aren’t afraid to take smart risks and push past obstacles.

Our novel approach to gene editing has the potential to go beyond personalized medicine and deliver individualized therapies with the potential to cure—ultimately helping patients live their best lives possible. We’re building on CRISPR technology and harnessing the cell’s natural DNA repair processes to rewrite genes, so patients can write the next chapter of their life story.

A strong scientific foundation

Our gene editing technology is rooted in the extensive work and expertise of our academic co-founders, including Matthew Porteus, M.D., Ph.D., and Maria Grazia Roncarolo, M.D. Dr. Porteus is considered to be one of the scientific pioneers and founders of the genome editing field. He has dedicated years of research to moving the use of CRISPR technology beyond cutting and gene knockouts towards precise DNA repair to cure genetic diseases.

Dr. Maria Grazia Roncarolo is one of the world’s foremost experts in gene therapy. She is globally recognized for her work in translating scientific discoveries in genetic diseases and regenerative medicine into novel therapies for patients, including the world’s first ex vivo gene therapy.

Remarkable science, with life-changing potential

Our UltraHDR platform is designed to enable incredible precision in the gene-editing process, providing the potential to correct, replace, and insert genes anywhere in the genome. We continue to push ourselves and science forward to fulfill our mission to develop one-time cures for millions of patients around the world.

Determined not just to treat, but to cure

‘One dose, one cure’ isn’t a dream. It’s our mission. We’re not afraid to take on hard problems because we know in the end, solving them will be worth it. And we won’t stop until we help create a future where incurable diseases no longer exist.


Our team

Smart. Scrappy. Roll-up-your-sleeves types. Committed to changing lives by developing cures. We believe we can make what was once considered impossible a reality. By working together, we can turn our diverse backgrounds and experiences into smarter thinking that gets us closer to curing seemingly incurable diseases.

Meet the people who inspire the big ideas, fresh thinking, and bold action that help us get over challenges today that will lead to cures tomorrow.

josh lehrer
Josh Lehrer, M.D., M.Phil., FACC
Chief Executive Officer

Dr. Josh Lehrer is the chief executive officer at Graphite Bio. In this role, Dr. Lehrer 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.

Previously, Dr. Lehrer served as chief medical officer at Global Blood Therapeutics (GBT), where he led the innovative clinical development strategy for sickle cell disease treatment Oxbryta® (voxelotor) from pre-IND stages through commercial launch, including playing an integral role in raising over $1 billion to fund the product’s development and launch.

At GBT, Dr. Lehrer also built a 45-member development organization and oversaw all clinical development and operations for the company during a period of rapid growth and pipeline advancement. Dr. Lehrer has also served in leadership roles at Genentech in clinical development and business development, as well as 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 University of California, San Francisco (UCSF), School of Medicine, and completed his residency at UCSF in internal medicine. He holds a Bachelor of Arts degree 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.

josh lehrer
Josh Lehrer, M.D., M.Phil., FACC
Chief Executive Officer

Dr. Josh Lehrer is the chief executive officer at Graphite Bio. In this role, Dr. Lehrer 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.

Previously, Dr. Lehrer served as chief medical officer at Global Blood Therapeutics (GBT), where he led the innovative clinical development strategy for sickle cell disease treatment Oxbryta® (voxelotor) from pre-IND stages through commercial launch, including playing an integral role in raising over $1 billion to fund the product’s development and launch.

At GBT, Dr. Lehrer also built a 45-member development organization and oversaw all clinical development and operations for the company during a period of rapid growth and pipeline advancement. Dr. Lehrer has also served in leadership roles at Genentech in clinical development and business development, as well as 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 University of California, San Francisco (UCSF), School of Medicine, and completed his residency at UCSF in internal medicine. He holds a Bachelor of Arts degree 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.

jerry cacia
Jerry Cacia
Chief Technical Officer

Jerry Cacia is the chief technical officer at Graphite Bio. In this role, Mr. Cacia is responsible for leading the overall manufacturing strategy and technical operations in support of Graphite Bio’s gene editing programs as they advance to the clinic. Mr. Cacia is advancing the development and implementation of a robust manufacturing strategy to ensure the advancement of Graphite Bio’s clinical programs and develop a clear and scalable path to patients.

In his most recent position, Jerry served as head of global technical development at Roche during which time he supported a pipeline that included over 80 new molecular entities and more than 100 development projects in various stages, including a number of cell, gene, and individualized neoantigen therapy programs.

Mr. Cacia holds a Bachelor of Science degree in biological sciences from the University of California at Santa Cruz.

Christine Garrett, Ph.D., PMP
Chief of Staff and Senior Vice President of Operations

Dr. Christine (Chris) Garrett is chief of staff and senior vice president of operations at Graphite Bio. In this role, Dr. Garrett is responsible for providing strategic guidance to successfully execute key business initiatives to optimize and support the growth of the company.

Dr. Garrett brings more than 20 years of industry experience leading high-performing, cross-functional teams at global biopharmaceutical companies to advance the development and life-cycle management of pipeline and approved therapies. She most recently worked at Bristol Myers Squibb (BMS), overseeing late development project management for the global R&D organization, and significantly contributed to the integration of development programs from Celgene and Myokardia into the BMS portfolio.

Dr. Garrett has also held senior leadership roles in global R&D project and portfolio management at Leo Pharma and Celgene. Earlier in her career, she worked at Novartis in various program management roles of increasing responsibility. Prior to joining the biopharmaceutical industry, she led and executed projects to develop antimicrobial preservatives for a specialty chemicals company.

Dr. Garrett received her Bachelor of Science in chemistry from the Rochester Institute of Technology and her Ph.D. in organic chemistry from Massachusetts Institute of Technology. She also holds certificates in project management and pharmaceutical business development.

jane grogan
Jane Grogan, Ph.D.
Chief Scientific Officer

Dr. Jane Grogan is the chief scientific officer at Graphite Bio. In this role, she oversees Graphite Bio’s clinical research and development efforts, leveraging the company’s targeted gene-integration platform technology in new therapeutic areas to impact patients and support the continued growth of the company’s world-class research group.

Prior to joining Graphite Bio, Dr. Grogan served as chief scientific officer and a member of the executive leadership team at ArsenalBio, a programmable cell therapy company, where she established the research organization and preclinical pipeline of cell therapies for solid tumors. From 2004-2019, Dr. Grogan distinguished herself as an influential scientist throughout her tenure at Genentech, a member of the Roche Group.

As head of adaptive tumor immunity and principal scientist of cancer immunology discovery research at Genentech, Dr. Grogan oversaw discovery research programs from target identification through clinical development, and she managed in-licensing business partnerships and external development collaborations. She is widely recognized as the founder and original host of the popular podcast series, “Two Scientists Walk into a Bar.”

During her career, Dr. Grogan has published over 60 papers in a wide array of journals and is the inventor on over 20 patents. She obtained her Ph.D. from Leiden University, The Netherlands, and her BSci (Hons) degree from the University of Melbourne, Australia. Dr. Grogan completed her post-doctoral training as an Alexander von Humboldt Fellow at the German Rheumatism Research Centre Berlin (DRFZ) and as a Howard Hughes Fellow at the University of California, San Francisco (UCSF).

Philip Gutry
Philip P. Gutry
Chief Business Officer

Phil Gutry serves as chief business officer at Graphite Bio. Mr. Gutry brings to this role extensive business development, strategy, finance, and investor relations experience with a successful track record of raising capital and establishing partnerships for biotech companies.

Mr. Gutry most recently worked at Kronos Bio, a clinical-stage oncology company focused on targeting dysregulated transcription, where he served as chief business officer. In this role, he led the acquisition of Gilead’s spleen tyrosine kinase inhibitor portfolio, which transformed Kronos into a clinical-stage company and was the primary catalyst for raising $500 million in equity capital, including the company’s initial public offering.

Previously, Mr. Gutry served in senior business development and finance roles at Regeneron Pharmaceuticals, MPM Capital, and Gilead Sciences, during which he led or managed over 30 transactions raising over $2 billion in equity, debt, and R&D funding.

Mr. Gutry received his Bachelor of Arts degree in Earth sciences from Dartmouth College and his MBA in healthcare management from The Wharton School.

julia tran
Julia Tran
Chief People Officer

Julia Tran is the chief people officer at Graphite Bio. Ms. Tran brings to Graphite Bio more than 20 years of experience building and growing passionate, mission-driven organizations that are committed to developing innovative solutions to tackle some of the biggest global challenges in biotech, cleantech, and cybersecurity.

As a people executive for biotech companies like Amyris, Ms. Tran played a pivotal role in scaling them from early-stage development through commercialization to IPO. Amyris, in partnership with the Bill and Melinda Gates Foundation, developed artemisinic acid-producing yeast strains through their novel synthetic biology platform and made it available to Sanofi on a royalty-free basis via OneWorld Health, which distributed it to malaria patients in the developing world.

The company went on to deliver impactful cleantech products such as second-generation biofuels, chemicals, and consumer healthcare products. Additionally, Ms. Tran worked on the commercial organizational buildout for Millennium Pharmaceuticals’ launch of Velcade for the treatment of multiple myeloma and Integrilin for acute coronary syndrome. Notably, at CV Therapeutics, she led the recruitment efforts for the first national sales force for their flagship product, Ranexa, for chronic angina. Ms. Tran brings an incredibly diverse background to Graphite Bio. In addition to her biotech background, she brings tech executive leadership experience. Most recently, as a Silicon Valley entrepreneur, she co-founded a venture-backed cybersecurity company, Blue Lava Inc, where she served as both chief operating officer and chief community officer from concept to commercialization.

Ms. Tran holds a Bachelor of Arts degree in political science from Boston College.

alethia young
Alethia Young
Chief Financial Officer

Alethia Young is the chief financial officer at Graphite Bio. In this role, Ms. Young oversees the company’s finances, investor relations, and corporate communications functions and plays a key role in overall corporate strategy.

Previously, Ms. Young served as senior biotech analyst and head of research at Cantor Fitzgerald, managing the equity research department covering large-cap and small- to mid-cap biotechnology companies. Prior to joining Cantor Fitzgerald in 2018, Ms. Young held senior biotech analyst positions at Credit Suisse and Deutsche Bank. Earlier in her career, she was a research policy analyst and president at Marwood Group, providing healthcare-focused advisory services to institutional investors. She began her career at JP Morgan in the investment banking and asset management divisions.

Ms. Young earned a Bachelor of Arts degree in economics and Spanish from Duke University. She currently serves on the board of directors for BUILD NYC and PTC Therapeutics.

Perry Karsen, Board Chair

Perry Karsen brings decades of proven executive leadership and board experience to the board of directors at Graphite Bio. Mr. Karsen served as the chief executive officer of the Celgene Cellular Therapeutics division of Celgene Corporation and as executive vice president and chief operations officer of Celgene Corporation. Previously, he was president and CEO of Pearl Therapeutics, Inc, a privately held biotechnology company subsequently acquired by AstraZeneca. He also has held executive positions at Human Genome Sciences, Inc, a biopharmaceutical company subsequently acquired by GlaxoSmithKline, Bristol-Myers Squibb Co., Genentech, Inc, and Abbott Laboratories. Currently, Mr. Karsen is board chair of Jounce Therapeutics, executive chair of Nitrome Biosciences, and on the board of ESCAPE Bio. Mr. Karsen has also served on the Board of Intellia Therapeutics through 2020. He also serves on the Gladstone Foundation Board. In 2019, Mr. Karsen was appointed as a fellow in the Stanford University Distinguished Careers Institute.

Perry Karsen, Board Chair

Perry Karsen brings decades of proven executive leadership and board experience to the board of directors at Graphite Bio. Mr. Karsen served as the chief executive officer of the Celgene Cellular Therapeutics division of Celgene Corporation and as executive vice president and chief operations officer of Celgene Corporation. Previously, he was president and CEO of Pearl Therapeutics, Inc, a privately held biotechnology company subsequently acquired by AstraZeneca. He also has held executive positions at Human Genome Sciences, Inc, a biopharmaceutical company subsequently acquired by GlaxoSmithKline, Bristol-Myers Squibb Co., Genentech, Inc, and Abbott Laboratories. Currently, Mr. Karsen is board chair of Jounce Therapeutics, executive chair of Nitrome Biosciences, and on the board of ESCAPE Bio. Mr. Karsen has also served on the Board of Intellia Therapeutics through 2020. He also serves on the Gladstone Foundation Board. In 2019, Mr. Karsen was appointed as a fellow in the Stanford University Distinguished Careers Institute.

Abe Bassan

Abe Bassan has extensive experience in the gene therapy arena as an operator and investor. He currently is a vice president at Samsara BioCapital. Mr. Bassan previously served as director of program biology at Revolution Medicines, where he co-led the initial stages of the company’s 4EBP1/mTORC1 cancer program. Prior to that, he was associate director of program development at bluebird bio, where he was the project manager for the company’s beta-thalassemia, sickle cell anemia, and ALD gene therapy programs. Earlier in his career, Mr. Bassan was an associate at Third Rock Ventures, where he played a leading role in the firm’s investment in bluebird bio, as well as the ideation of Blueprint Medicines, a precision medicine oncology company. Mr. Bassan was also the founder of Aurora Medical, a molecular diagnostics testing services company.

Jerel Davis, Ph.D.

Dr. Jerel Davis brings a distinguished track record as an investor and advisor to his role as a board member of Graphite Bio, with proven expertise in company creation, R&D alliances, and corporate partnerships. He currently serves as managing director of Versant Ventures. During his 9-year tenure at Versant, Dr. Davis has helped propel the growth strategies of a multitude of the firm’s portfolio companies, including BlueRock Therapeutics, CRISPR Therapeutics, Repare Therapeutics, Chinook Therapeutics, Tentarix Biotherapeutics, Turnstone Biologics, Ventus Therapeutics, and several others. Prior to joining Versant, Dr. Davis was an associate principal at McKinsey & Company, where he advised a broad range of pharmaceutical, biotechnology, medical device, and molecular diagnostics companies. He has worked in a number of healthcare markets globally, including the US, Canada, Europe, China, Russia, and India.

Kristen M. Hege, M.D.

Dr. Kristen Hege is a renowned physician-scientist and a pioneer in advancing cell therapies through clinical development to regulatory approval. She currently serves as senior vice president, early clinical development, hematology/oncology and cell therapy at Bristol Myers Squibb (BMS), where her team is responsible for advancing the pipeline from first-in-human studies to clinical proof-of-concept. Notably, Dr. Hege led the development of Abecma (idecabtagene vicleucel; ide-cel), the first BCMA-directed CAR T cell immunotherapy for the treatment of adult patients with relapsed or refractory multiple myeloma, from product concept to approval in partnership with bluebird bio. Prior to BMS, Dr. Hege held multiple executive roles at several biotech companies, including Celgene, Cell Genesys, Cellerant Therapeutics, and Theraclone Sciences. Dr. Hege, a hematologist-oncologist, is also a clinical professor of medicine at the University of California (UCSF), San Francisco, where she continues to manage patients with benign and malignant blood disorders. Dr. Hege also serves on the board of directors of Mersana Therapeutics and was elected to the board of directors for the Society for Immunotherapy of Cancer (SITC) for a 3-year term from 2016-2019.

Joe Jimenez

Joe Jimenez is the former chief executive officer of Novartis, one of the world’s leading pharmaceutical companies. Mr. Jimenez joined Novartis in 2007 to lead the pharmaceuticals division and was named CEO in 2010. He navigated the company through a period of change following patent expirations of its 2 largest products. Under his stewardship, Novartis developed one of the largest pipelines of self-originated drugs in the industry, driven by a strong commitment to research and development. Currently, Mr. Jimenez is co-founder and managing director of Aditum Bio, an investment firm focused on developing biotechnology assets that will benefit patients. He also serves as a board member of General Motors Company and The Proctor & Gamble Company.

Josh Lehrer, M.D., M.Phil., FACC

Dr. Josh Lehrer is the chief executive officer at Graphite Bio. In this role, Dr. Lehrer 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. Previously, Dr. Lehrer served as chief medical officer at Global Blood Therapeutics (GBT), where he led the innovative clinical development strategy for sickle cell disease treatment Oxbryta® (voxelotor) from pre-IND stages through approval and commercial launch, including playing an integral role in raising over $1 billion to fund the product’s development and launch. Dr. Lehrer has also served in leadership roles at Genentech in clinical development and business development, as well as attending physician roles at Stanford University Medical Center and the Palo Alto Veterans Affairs Health System.

Matthew Porteus, M.D., Ph.D.

Dr. Matthew Porteus is an academic founder of Graphite Bio and a pioneer in the field of gene editing. Currently, he is a professor of pediatrics and stem cell transplantation and the co-director of the Center for Definitive and Curative Medicine at Stanford University. His work in gene editing spans more than 2 decades, during which he was an academic founder of CRISPR Therapeutics and an advisor to several other gene editing companies, as well as the National Heart, Lung, and Blood Institute on Sickle Cell Disease. As a trained pediatric hematologist, Dr. Porteus now cares for children who are on the Pediatric Stem Cell Transplant service.

Carlo Rizzuto, Ph.D.

Dr. Carlo Rizzuto is a seasoned biopharmaceutical business executive and investor with over 2 decades of diverse leadership experience. He currently is a partner at Versant Ventures based in New York and has played an instrumental role in launching and growing multiple companies, including Pandion Therapeutics, Repare Therapeutics, Century Therapeutics, and Jnana Therapeutics, among others. Earlier in his career, he led global product development teams at Novartis AG and was an associate principal with McKinsey & Company, advising US and European clients in the pharmaceutical, payor, and provider sectors.

Smital Shah

Smital Shah brings proven executive leadership expertise to her role as a board member of Graphite Bio, including over 2 decades of senior management experience in the biopharma and investment banking industries. She currently serves as chief business and financial officer of ProQR Therapeutics, a biotechnology company focused on rare genetic diseases. Previously, Ms. Shah managed the multi billion-dollar debt, cash, and investment portfolios of Gilead Sciences. Her proven management and leadership experience in the biopharma industry is augmented by her earlier career in investment banking, where she has particular expertise in financial strategy, capital markets, and business development. Prior to Gilead, Ms. Shah spent several years in investment banking at Leerink Partners and JP Morgan focused on the biotech sector. Earlier in her career, she held various research and development positions at Johnson & Johnson. Ms. Shah is a board member and chair of the audit committee at Pliant Therapeutics.

Jo Viney, Ph.D.

Dr. Jo Viney brings a distinguished track record in drug discovery and development to the board of directors at Graphite Bio. She currently serves as president, chief scientific officer, and co-founder of Pandion Therapeutics, now a wholly owned subsidiary of Merck & Co., Inc. Prior to founding Pandion, Dr. Viney served most recently as senior vice president, drug discovery, at Biogen. Throughout her career, Dr. Viney has been an advocate for workplace inclusiveness. From 2015–2020, she served on the board of WEST, a nonprofit organization supporting early to mid-career women, and was WEST’s president for the 2017–2018 term. Dr. Viney has maintained longstanding memberships with the Society for Mucosal Immunology (SMI) and the American Association of Immunologists (AAI). She has served numerous terms on the SMI Board and was president from 2011–2013. In 2020, Dr. Viney was chosen as one of FiercePharma’s Fiercest Women in Life Sciences. Dr. Viney is also a member of the board of directors for Finch Therapeutics and Harpoon Therapeutics, and she serves on the Scientific Advisory Board for HotSpot Therapeutics.

Maria Grazia Roncarolo, M.D., SAB Chair

Dr. Maria Grazia 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.

Maria Grazia Roncarolo, M.D., SAB Chair

Dr. Maria Grazia 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.

Prof. John 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) and a fellow of the Royal Society of Canada; the Royal Society of London, UK; and the American Association for Cancer Research (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 AACR and the Keio Medical Science Prize.

Natalia Gomez-Ospina, M.D., Ph.D.

Dr. Natalia Gomez-Ospina has dual appointments in the Divisions of Medical Genetics and Stem Cell Transplantation and is a faculty member at 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. Scot 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.

Matthew Porteus, M.D., Ph.D.

Dr. Matthew Porteus’ visionary gene editing research led to the formation of Graphite Bio and our mission to develop best-in-disease therapies for patients with serious genetic disorders. Professor of Pediatrics and Stem Cell Transplantation and the Co-Director of Center for Definitive and Curative Medicine at Stanford University, Dr. Porteus is considered to be one of the scientific pioneers and founders of the field of genome editing. At his renowned Stanford Lab, he was first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients. His research is now the gold standard in gene editing, blazing the trail for medical innovation in a field that holds bold promise for addressing the underlying causes of diseases in order to save lives. In addition to his role as a scientific advisor, Dr. Porteus is a member of Graphite Bio’s Board of Directors.

Matthew Porteus, M.D., Ph.D.

Dr. Matthew Porteus’ visionary gene editing research led to the formation of Graphite Bio and our mission to develop best-in-disease therapies for patients with serious genetic disorders. Professor of Pediatrics and Stem Cell Transplantation and the Co-Director of Center for Definitive and Curative Medicine at Stanford University, Dr. Porteus is considered to be one of the scientific pioneers and founders of the field of genome editing. At his renowned Stanford Lab, he was first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients. His research is now the gold standard in gene editing, blazing the trail for medical innovation in a field that holds bold promise for addressing the underlying causes of diseases in order to save lives. In addition to his role as a scientific advisor, Dr. Porteus is a member of Graphite Bio’s Board of Directors.

Maria Grazia Roncarolo, M.D.

Dr. Maria Grazia 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.

Daniel Dever, Ph.D.

Dr. Daniel Dever is a co-founder and the head of discovery research at Graphite Bio. As a post-doctoral scholar working in the laboratory of Dr. Matthew Porteus at Stanford, Dr. Dever demonstrated an increase in site-specific DNA integration efficiency from less than 1% to greater than 50% across diverse genetic lesions in a wide range of cell types, including hard-to-edit hematopoietic stem cells. This work forms the foundation of our next-generation, high-efficiency DNA-targeted integration platform. Dr. Dever’s research has been published in an array of peer-reviewed journals, including Nature, Cell Stem Cell, Molecular Therapy, Nature Medicine, Nature Protocols, Nucleic Acids Research, and other high-impact journals.

A name with purpose

As our team pursues a bold mission to advance gene editing to cure disease and transform lives, we are proud to recognize a scientific hero who blazed the path for our important work.

The name Graphite Bio is a nod to Dr. Rosalind Elsie Franklin, an English chemist and X-ray crystallographer. Dr. Franklin was the first to illustrate the atomic structure of graphite. Even more impactful was her work that became central to the understanding of the molecular structures of DNA, RNA, and viruses. Her X-ray diffraction images led to the understanding of the DNA double helix.

While Dr. Franklin’s discoveries are foundational to the field of gene editing, it’s her personal story that truly inspires us. She lived a life of extreme clarity and purpose. Science was always her singular focus and calling. While her work was groundbreaking, her contributions were largely overlooked and under-appreciated by the scientific community for decades. She faced many professional and personal challenges in her work. Undaunted, she always marched forward, persevering where many others would not, to overcome seemingly insurmountable barriers. She believed that advancing science for the greater good is what mattered most. Through her dedication, desire, and deeds, Dr. Franklin embodies the hero of science that we honor and celebrate by linking her remarkable life story to our company name.

What’s the use of doing all this work if we don’t get some fun out of this?”

Dr. Rosalind Elsie Franklin — As quoted by Aaron Klug, interview, June 17, 2005

Our platform is driving the future of gene editing

The science behind our promising therapy candidates is what connects our diverse backgrounds and experience to the impact we strive to have on the lives of patients.

Explore our science