As investigational cell therapies influx the clinical trial landscape, especially for oncologic indications, some companies are ceding the race to their competitors.
While 2023 may be bringing the potential for new approved gene and cell therapies across indications, several companies are reversing on their cell and gene therapy pipelines and discontinuing development or deprioritizing these programs. Two companies that have recently made such announcements are Glaxo-Smith-Kline (GSK) and Talaris.1,2
GSK’s move away from gene and cell therapies was not unexpected, as the company had terminated collaborations on T-cell receptor therapies for oncologic indications with both Immatics and Lyell Immunopharma in late 2022.3,4 The company’s direction was confirmed as it stated that it was to “end [its] investment in gene and cell therapy” in February 2023.1 GSK has instead poised itself to embrace RNA-editing therapies, namely, oligonucleotide therapies, of which the company has 2 in clinical trials for liver diseases, and just recently invested $170 million upfront to collaborate on another with Wave Life Sciences.5
In a more drastic change, Talaris Therapeutics is abandoning its lead program cell therapies intended for recipients of kidney transplants without immunosuppression as of a February 2023 announcement.2 Its lead candidate, FCR001, was being evaluated in the phase 3 FREEDOM-1 trial (NCT03995901) as well as in the phase 2 FREEDOM-2 trial (NCT01649388). Both studies have now been discontinued months after a patients died in the FREEDOM-1 study from grade 4 graft-versus-host disease (GvHD) amid multiple cases of GvHD.6 Talaris’ only clinical trial left running is FREEDOM-3 (NCT05098145) evaluating FCR001 for safety in patients with rapidly progressive diffuse cutaneous systemic sclerosis.
“It was an exceptionally difficult decision to discontinue further development of FCR001 in kidney transplantation tolerance despite the promising early data,” Scott Requadt, chief executive officer, Talaris, said in a statement.6 "I want to express our sincere thanks, first and foremost, to our patients and their donors, as well as to our investigators and collaborators for their participation in this effort, and hope to see continued improvements in kidney disease care in the future. We also want to sincerely thank all our employees, who have been supporting our mission to transform patients’ lives. While we are disappointed that our work in kidney transplantation will not continue, given the potential of FCR001 to induce durable tolerance, we intend to continue its evaluation for scleroderma, which remains a very high unmet medical need for which there are limited treatment options."
In the field of sickle cell disease (SCD), early 2023 similarly brought about discontinuations of cell therapies by Sangamo Therapeutics and Graphite Bio, which shelved development of BIVV003 and nulabeglogene autogedtemcel, respectively.7,8 Graphite’s SCD program had been paused following a serious AE in January 2023, while Sangamo simply stated that it was reprioritizing other programs, including a gene therapy for Fabry disease.