Subscribe

|Videos|April 16, 2022

Building on CRISPR and Striving for Definitive Cures: Josh Lehrer, MD

Author(s)CGTLive Staff

The chief executive officer of Graphite Bio discusses the phase 1/2 CEDAR trial of GPH101, an investigative Cas9-edited autologous CD34+ cell therapy for the treatment of sickle cell disease.

"Patients are trusting us to come in and go through a procedure where we're manufacturing their own cells to potentially cure them. It's a big commitment."

Graphite Bio expects to treat its first patient with sickle cell disease later this year with GPH101, its investigational next-generation gene-edited autologous hematopoietic stem cell product candidate.

Josh Lehrer, MD, MPhil, FACC, chief executive officer at Graphite Bio, joins CGTL to share updates on the phase 1/2 CEDAR trial (NCT04819841), his company's approach and platform, and other investigational therapies in the pipeline, including for β-thalassemia and Gaucher disease.

Graphite announced the first patient had been enrolled¹ in the CEDAR trial at the 63rd Annual American Society of Hematology (ASH) Meeting back in December and now expects to treat its first patient by the end of the year.

GPH101 was developed using Graphite’s next-generation targeted gene integration platform, which relies on Cas9 technology and a non-integrating DNA template to correct the mutation in the β-globin gene through the homology directed repair cellular pathway.

Reference

1. Graphite Bio enrolls first patient in phase 1/2 clinical trial of GPH101 for sickle cell disease. News release. Graphite Bio. November 17, 2021. https://www.businesswire.com/news/home/20211117005527/en/Graphite-Bio-Enrolls-First-Patient-in-Phase-12-Clinical-Trial-of-GPH101-for-Sickle-Cell-Disease

Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Related Articles

Top News in Lymphoma Cell Therapy for World Lymphoma Awareness Day 2025
September 15th 2025
Duchenne Action Month 2025: Looking Back at News and Expert Insights
September 14th 2025
CGTLive®’s Weekly Rewind – September 12, 2025
September 12th 2025
First Patient Treated in Capsida Biotherapeutics’ Trial for Encephalopathy Gene Therapy CAP-002 Passes Away
September 11th 2025
Some Patients Treated in Anixa's Ovarian Cancer CER T Trial Show Longer-Than-Expected Survival
September 11th 2025
Patients Treated With REGENXBIO's MPS II Gene Therapy RGX-121 Sustain 82% Median Reduction in CSF HS D2S6 Levels Through 1 Year
September 10th 2025

Latest CME

$New Horizons in Multiple Myeloma Treatment – Selection and Sequencing From Newly Diagnosed to Relapsed/Refractory Disease$

New Horizons in Multiple Myeloma Treatment – Selection and Sequencing From Newly Diagnosed to Relapsed/Refractory Disease

Amrita Krishnan, MD; Suzanne Lentzsch, MD, PhD; Sagar Lonial, MD, FACP

Exploring the Dynamic Shifts in the MDS Treatment Paradigm: Unveiling Advances in Disease Modifying Therapies, Spotlight on Cutting-Edge Clinical Trials, and the Latest Approvals

Exploring the Dynamic Shifts in the MDS Treatment Paradigm: Unveiling Advances in Disease Modifying Therapies, Spotlight on Cutting-Edge Clinical Trials, and the Latest Approvals

Amer Zeidan, MBBS, MHS; Michael R. Savona, MD

Medical Crossfire®: Uncovering The Clinical Utility of Combination Therapies and MRD Assessment in Newly Diagnosed Multiple Myeloma

Medical Crossfire®: Uncovering The Clinical Utility of Combination Therapies and MRD Assessment in Newly Diagnosed Multiple Myeloma

Ajai Chari, MD; Adriana Rossi, MD; Anupama Kumar, MD

BURST Expert Illustrations and Commentaries™: Exploring the Mechanistic Rationale for CSF-1R– Directed Treatment in Chronic GVHD

BURST Expert Illustrations and Commentaries™: Exploring the Mechanistic Rationale for CSF-1R– Directed Treatment in Chronic GVHD

Corey Cutler, MD, MPH, FRCP(C); Carrie L. Kitko, MD

Community Practice Connections™: 8th Annual School of Nursing Oncology™

Community Practice Connections™: 8th Annual School of Nursing Oncology™

Beth Sandy, MSN, CRNP, FAPO; Laura Wood, RN, MSN, OCN; Michael Lai, ARNP; Tiffany Richards, PhD, ANP-BC, AOCNP®; Lauren Verity Moore, DNP, MSN, AGACNP-BC; Megan Wanca, APRN, DNP, FNP-C

SimulatEd™: Patient-Centered Care to Improve Outcomes in Chronic Lymphocytic Leukemia

Case-based Simulation

SimulatEd™: Patient-Centered Care to Improve Outcomes in Chronic Lymphocytic Leukemia

Catherine C. Coombs, MD

(COPE) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

(COPE) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

(CME) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

(CME) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

Community Practice Connections™: 6th Annual Precision Medicine Symposium – An Illustrated Tumor Board

Community Practice Connections™: 6th Annual Precision Medicine Symposium – An Illustrated Tumor Board

Andre H. Goy, MD; Monique Hartley-Brown, MD, MMSc (Guest Co-Chair); Chiara Battelli, MD, PhD; Marios Giannakis, MD, PhD; Shail Maingi, MD; Aparna Parikh, MD

Addressing Pivotal Updates in AML – Expert Perspectives on Individualizing Management for Special Populations

Addressing Pivotal Updates in AML – Expert Perspectives on Individualizing Management for Special Populations

Eytan M. Stein, MD; Marina Kremyanskaya, MD, PhD; Hannah Levavi, MD

Innovations in MDS Care: Navigating New Treatment Paradigms, Breakthrough Therapies, and Emerging Clinical Evidence

Innovations in MDS Care: Navigating New Treatment Paradigms, Breakthrough Therapies, and Emerging Clinical Evidence

Rami Komrokji, MD; Maen Hussein, MD

Annual Hematology Meeting: Preceding the 67th ASH Annual Meeting and Exposition

In-Person + Virtual Event

Annual Hematology Meeting: Preceding the 67th ASH Annual Meeting and Exposition

December 5, 2025

Addressing Racial Disparities in Multiple Myeloma: Forging a Path Towards Health Equity in Clinical Trials and Everyday Practice

Addressing Racial Disparities in Multiple Myeloma: Forging a Path Towards Health Equity in Clinical Trials and Everyday Practice

Craig Emmitt Cole, MD; Sikander Ailawadhi, MD; Irene Ghobrial, MD; Monique Hartley-Brown, MD, MMSc

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Elias Jabbour, MD

How We Do It®: New Combinations Reshaping Treatment Decision-Making in Newly Diagnosed Multiple Myeloma

How We Do It®: New Combinations Reshaping Treatment Decision-Making in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Cristina Gasparetto, MD; Katja Weisel, MD

Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET

Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET

Rami S. Komrokji, MD; Ghaith Abu-Zeinah, MD; Gabriela Hobbs, MD; Barbara Mora, MD

From Barriers to Breakthroughs in Multiple Myeloma: How Trials Compare to the Real-World Management of Patients in Your Practice

From Barriers to Breakthroughs in Multiple Myeloma: How Trials Compare to the Real-World Management of Patients in Your Practice

Krina Patel, MD, MSc; Alfred L. Garfall, MD; Ajay K. Nooka, MD, MPH, FACP

Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?

Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?

Corey Cutler, MD, MPH, FRCPC; Amin M. Alousi, MD; Yi-Bin Chen, MD; Carrie Lynn Kitko, MD

Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead

Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead

Alexey V. Danilov, MD, PhD; Leidy L. Isenalumhe, MD, MS; Lori A. Leslie, MD; Mazyar Shadman, MD, MPH; Jason R. Westin, MD, MS, FACP

Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios

Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios

Elias Jabbour, MD; Hagop M. Kantarjian, MD; Aaron Logan, MD, PhD, MPhil; Rob Pieters, MD, PhD; Claire Roddie, MD, PhD

SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients

Case-based Simulation

SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients

Amrita Krishnan, MD

30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

In-Person Event

30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

February 26-28, 2026

Medical Crossfire®: Expert Interpretations of the Latest Data in CLL Management – Understanding the Impact of Optimal Treatment Selection on Patient Outcomes

Medical Crossfire®: Expert Interpretations of the Latest Data in CLL Management – Understanding the Impact of Optimal Treatment Selection on Patient Outcomes

Jennifer R. Brown, MD, PhD; Alexey V. Danilov, MD, PhD; Matthew S. Davids, MD, MMSc

Community Practice Connections™: Tailored Treatment Approaches for Older Patients With Advanced HR+/HER2– Breast Cancer

Community Practice Connections™: Tailored Treatment Approaches for Older Patients With Advanced HR+/HER2– Breast Cancer

Hope S. Rugo, MD, FASCO; Neil M. Iyengar, MD; Komal Jhaveri, MD, FACP

29th Annual International Congress on Hematologic Malignancies: Focus on Leukemias, Lymphomas, and Myeloma

29th Annual International Congress on Hematologic Malignancies: Focus on Leukemias, Lymphomas, and Myeloma

André H. Goy, MD; Jorge E. Cortes, MD; Sagar Lonial, MD, FACP

Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights

Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights

Gail J. Roboz, MD; Hagop Kantarjian, MD; Catherine Lai, MD, MPH; Amer Zeidan, MBBS, MHS

Medical Crossfire®: Harnessing the Power of Modern Therapies in Newly Diagnosed Multiple Myeloma

Medical Crossfire®: Harnessing the Power of Modern Therapies in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches

Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches

Rami Komrokji, MD; Gabriela Hobbs, MD; John Mascarenhas, MD; Naveen Pemmaraju, MD

$Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma$

Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

10th Annual Live Medical Crossfire®: Hematologic Malignancies

In-Person Event

10th Annual Live Medical Crossfire®: Hematologic Malignancies

July 11, 2026

Expert Perspectives on Applying Recent Evidence to Practice in the Management of Newly Diagnosed Multiple Myeloma

Expert Perspectives on Applying Recent Evidence to Practice in the Management of Newly Diagnosed Multiple Myeloma

Amrita Krishnan, MD; Meletios Athanasios (Thanos) C. Dimopoulos, MD; Shaji Kumar, MD; Katja Weisel, MD

Imaging Matters: Decoding PSMA PET for Better Decision-Making

Imaging Matters: Decoding PSMA PET for Better Decision-Making

Jeremie Calais, MD, PhD; Tanya B. Dorff, MD; Scott T. Tagawa, MD, MS, FACP, FASCO

Go To PER in Chicago

In-Person + Virtual Event

Go To PER in Chicago

On-Demand Sessions Available Now!

Trending on CGTlive®

Top News in Lymphoma Cell Therapy for World Lymphoma Awareness Day 2025

Duchenne Action Month 2025: Looking Back at News and Expert Insights

© 2025 MJH Life Sciences^®

All rights reserved.