Tami John, MD, on the FDA Approvals of Exa-cel and Lovo-cel

This is the second part of an interview with Tami John, MD. For the first part, click here.

“We're gonna be in a fortunate place to have to figure out which patient does better with which therapy; but to have 2 available therapies is really quite exciting. The next step is really going to be: How can we expand this to all the patients that need it? I think it's a wonderful first step to have 2 available products plus [allogeneic] transplant for sickle cell disease as transformative therapies. And now we get to focus on how we can treat patients all across the world in regions that have higher prevalence than in the United States or in the EU and UK.”

With the recent approvals of Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia) on December 8, 2023, patients with sickle cell disease (SCD) now have 2 new potentially transformative options for the treatment of their disease, in addition to the established standard of care option, allogeneic hematopoietic stem cell transplantation (HCT).¹ These new approvals are part of a broader trend of recent activity on the development of genetic medicines for SCD and other inherited hematological diseases.

In order to gain more insight into the importance of these new approvals, CGTLive™ sat down with Tami John, MD, a clinical associate professor at Stanford Medicine, who helped to conduct research focused on assessing red cell function in patients with SCD who received HCT or gene therapy for their condition. The work was presented at the the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California.²

In addition to the new FDA approvals, John discussed other research she was excited to see at ASH’s 2023 conference, as well as the big trends in SCD research in 2023. She specifically highlighted the conversation in the field about in vivo approaches to gene therapy, which have seen success in hemophilia and may be able to be translated to red cell disorders.

REFERENCES
1. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 7, 2023. Accessed January 8, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
2. Patel AP, Kanne CK, Stenger EO, John TD, Sheehan VA. Red cell rheology and blood viscosity in pediatric individuals having received allogenic hematopoietic stem cell transplantation or ex vivo autologous gene therapy for sickle cell disease. Presented at: ASH 2023 Annual Meeting & Exposition. December 9-12; San Diego, CA. Abstract #1076