Mark Walters, MD, on Lessons Learned With Investigating Lovo-Cel for Sickle Cell

“It's spectacular when it works, and it's disappointing when it takes a longer period of time to generate a benefit. And that's kind of the way medicine is, a lot of the time once the glow of success wears off a bit, then reality sinks in and you realize that this is a great treatment, but it's not going to work for everybody.”

The field of sickle cell disease (SCD) is nearing 3 months since the landmark FDA approvals of bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia and Vertex and CRISPR Pharmaceuticals' exagamglogene autotemcel (exa-cel; Casgevy) in December, the first approved gene therapies for treating sickle cell disease (SCD).

Lovo-cel, which, along with exa-cel, is approved for patients aged 12 years and older with SCD, 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, to learn more about his experiences with and lessons learned while investigating lovo-cel. Walters served as an investigator on the phase 1/2 HGB-205 (NCT02151526) and HGB-206 (NCT02140554) clinical trials evaluating lovo-cel. He also shared some early research with SCD in vivo gene editing therapy that is excited to see more of.

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 Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up. Blood. 2023;142(suppl 1):1051.doi:10.1182/blood-2023-174229