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


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.

1. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 8, 2023. Accessed December 8, 2023.
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
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