Take a look at the stories that stood out as pillars of progress and success in hematology gene and cell therapy development in 2023.
For all of 2023, the CGTLive™ team was following along the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.
From major data publications and presentations to FDA decisions and major medical meetings, the team spent all year bringing the latest information to the website's front page.
Among our areas of focus in 2023 has been hematology, a field that saw several key approvals, as well as other treatments move through the pipeline. The major news items appeared among the top pieces our team produced—but sometimes smaller stories reach those heights as well because of their clinical impact, their inventive mechanisms, or otherwise. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in hematology over the course of 2023.
Here, we'll highlight some of the most-read content on CGTLive's hematology page this year. Click the buttons to read further into these stories.
December 8, 2023 — On Friday, December 8, 2023, the FDA made a landmark decision to approve a pair of gene therapies for the treatment of sickle cell disease (SCD)—bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia; and Vertex and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel), which is to be marketed under the name Casgevy.
"Both therapies that were approved today, through slightly different mechanisms, do decrease the number of vaso-occlusive events and have the potential to take a [patient with sickle cell] who has had several of these events to not have any of these events."
– Nicole Verdun, MD, of FDA CBER
April 19, 2023 — Among individuals with relapsed and lenalidomide-refractory multiple myeloma (MM), ciltacabtagene autoleucel (Carvykti; Johnson & Johnson/Legend Biotech) has outperformed expectations compared with standard chemotherapy regimens—such as pomalidomide, bortezomib and dexamethasone; or daratumumab, pomalidomide and dexamethasone—according to leaked data from the phase 3 CARTITUDE-4 trial (NCT04181827) obtained by STAT News.
April 17, 2023 — The FDA has approved Gamida Cell's allogeneic cell therapy, omidubicel, to reduce the risk of infection in patients aged 12 years and older undergoing stem cell transplantation for hematologic cancer. Omidubicel, which will be marketed as Omisirge, is delivered as a single intravenous infusion composed of patient-specific human allogeneic cord blood-derived stem cells that have been processed and cultured with nicotinomide, a form of vitamin B3.
“Today’s approval is an important advance in cell therapy treatment in patients with blood cancers. Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation."
– Peter Marks, MD, PhD, of the FDA
October 20, 2023 — Lovo-cel therapy 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. It has been evaluated in the phase 1/2 HGB-205 (NCT02151526) and HGB-206 (NCT02140554) clinical trials, the former evaluating the therapy for both SCD and beta-thalassemia and the latter only involving participants with SCD. It is currently being evaluated in the phase 3 HGB-210 clinical trial (NCT04293185) for treating SCD.
“I think it'll be a little bit complicated as we wander into this new territory of high-priced therapies... but I think the patients that I have taken care of would tell you it is absolutely worth it. It is truly a transformative therapy."
– Julie Kanter, MD, of UAB
August 28, 2023 — The FDA has approved Bristol Myers Squibb’s first-in-class erythroid maturation agent luspatercept-aamt (Reblozyl) as a first-line treatment for anemia in adult patients with very low, low-, or intermediate-risk (IPSS-R) myelodysplastic syndromes (MDS) who may require red blood cell (RBC) transfusions. The decision was based on data from the randomized, open-label phase 3 COMMANDS clinical trial (NCT03682536.
"Results from the COMMANDS study showed nearly twice as many patients treated with Reblozyl achieved transfusion independence of at least 12 weeks and concurrent hemoglobin increase compared to epoetin alfa. Today’s approval represents an important advancement for patients with lower-risk MDS.”
– Andrew Guillermo Garcia-Manero, MD, of MD Anderson