Mark Walters, MD, on Moving Toward In Vivo Approaches for SCD Gene Therapy

Commentary
Video

The professor in residence of pediatrics at University of California San Francisco discussed trends in research for SCD gene therapy.

“I think [in vivo approaches are] the holy grail to making this widely accessible, that gets away from the high dose chemotherapy. It makes it much more accessible, even in areas of the world where the technological aspects of performing a bone marrow transplant will be challenging. And it should lower the cost. So, that's where the research is going. And I'm hopeful it will have an impact in the next decade or so.”

The FDA approved the first gene therapies for treating sickle cell disease (SCD) in December 2023. These were bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia and Vertex and CRISPR Pharmaceuticals' exagamglogene autotemcel (exa-cel; Casgevy), both approved for patients aged 12 years and older with SCD.

Lovo-cel consists of autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and apheresis, transduced with BB305 lentiviral vector (LVV) encoding the human beta-A-T87Q globin gene.

CGTLive spoke with Mark Walters, MD, professor in residence, pediatrics, Sickle Cell Center of Excellence, University of California San Francisco, an investigator on the phase 1/2 HGB-205 (NCT02151526) and HGB-206 (NCT02140554) clinical trials evaluating lovo-cel, to learn more about trends in research for gene therapy in SCD. He discussed how in vivo approaches to gene therapy may be the next step to helping address concerns with clonal hematopoiesis and myeloablation.

REFERENCES
1. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 8, 2023. Accessed December 8, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
2. Kanter J, Thompson AA, Kwiatkowski JL, et al. Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received LovotibeglogeneAutotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up. Blood. 2023;142(suppl 1):1051.doi:10.1182/blood-2023-174229
Recent Videos
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira
Jeffrey Chamberlain, PhD
Tami John, MD
Tami John, MD
Tami John, MD
Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital
Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Related Content
© 2025 MJH Life Sciences

All rights reserved.