After challenging year, cell and gene therapy industry poised for further growth in 2021
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Challenges presented by the COVID-19 pandemic did not stop progress in research or clinical development of gene and cell therapies, according to a state-of-the-industry briefing by Alliance of Regenerative Medicine.
Growth — driven primarily by oncology indications — should continue this year, Janet Lambert, CEO for the international advocacy group, said during the briefing.
The cell and gene therapy field was not immune to the effects of the COVID-19 pandemic, according to Lambert.
“Regulators were slammed with new and urgent COVID-related demands and, as a result, had less bandwidth for cell and gene therapy,” Lambert said. “This was on top of the growing industry demands of a fast-moving science.”
There are 1,220 active worldwide clinical trials in regenerative medicine and advanced cell/gene therapies, Lambert said. The majority are in oncology (n = 554), followed by indications for neurodegenerative diseases (n = 94), monogenetic diseases (n = 87) and infectious diseases (n = 73).
More than half (n = 685) are phase 2 trials, 383 are phase 1 trials and 152 are phase 3 trials.
“Cell-based immunotherapy trials make up almost a third of all regenerative medicine clinical trials and almost 50% of phase 1 trials,” Lambert said.
Clinical, regulatory milestones
Several “life-changing” cell and gene therapies received regulatory approval in 2020, according to Lambert. They include:
- brexucabtagene autoleucel (Tecartus; Kite Pharma/Gilead), the first CAR T-cell therapy for mantle cell lymphoma, which the FDA approved in July;
- onasemnogene abeparvovec-xioi (Zolgensma, Novartis Gene Therapies) — an adeno-associated virus vector-based gene therapy — which was approved in Europe, Japan and Canada for spinal muscular atrophy in children. The therapy received approval in the U.S. in 2019; and
- voretigene neparvovec (Luxturna, Spark Therapeutics), approved in Canada as a directly administered gene therapy for RPE65 mutation-associated retinal dystrophy.
The most promising clinical data in 2020, according to Lambert, came from trials of cell therapies that target B-cell maturation antigen (BCMA) in multiple myeloma, as well as new trials exploring antigen targets other than CD19, multiple antigen targets, allogeneic cell-based therapies and gene-edited cell therapies.
New milestones were established for gene-edited therapies that continue to advance in clinical testing, Lambert said. Last year, researchers reported data on the first patients treated with CRISPR-based gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia.
“By the end of 2020, there was no doubt that gene therapy was an established modality, with commercial products, a deep pipeline, strong support from large bio and pharma, and consistent investor enthusiasm,” Lambert said. “Gene therapies represent almost half of the phase 3 pipeline, so we should expect the industry to continue to mature and learn as more products move into the commercial marketplace.”
Twelve gene or cell therapies received the FDA’s regenerative medicine advanced therapy (RMAT) designation in 2020, as the pipeline of therapies continued to grow and advance through clinical trials.
“Policymakers and regulators were rightly distracted by COVID-19 and its associated challenges, but nevertheless the [cell and gene therapy] sector actually managed a couple of significant policy wins in this tumultuous year,” Lambert said.
Two major regulatory developments in the U.S. related to public payer reimbursement of these therapies. Medicare approved a new diagnostic-related group for CAR T-cell therapies that will significantly improve reimbursement for providers, Lambert said.
In addition, CMS finalized a new rule allowing state-based Medicaid programs to engage in innovative payment models, including outcomes-based arrangements for gene therapies.
Looking ahead to 2021
The regulatory landscape in 2021 is poised to be busy in Europe, where a handful of gene therapies are expected to receive approval over the next year.
Meanwhile, Lambert said she anticipates the FDA will render decisions on two CAR T-cell therapies this year: idecabtagene vicleucel (Bristol Myers Squibb, bluebird bio) for relapsed or refractory multiple myeloma, and lisocabtagene maraleucel (Bristol Myers Squibb) for relapsed or refractory large B-cell lymphoma.
“All indications are that 2021 will be a fantastic year in science, technology and clinical progress in [the cell and gene therapy] sector,” she said.
The industry also will “address the social context of [its] work” and focus on treatments for diseases that may not affect large numbers of people but will have a significant impact on improving the lives of those who are affected, Lambert said.
“I think we will see increased public-private efforts to enable N-of-1 and ultra-rare therapies to safely and efficiently reach patients,” she said.
For more information:
Janet Lambert can be reached at Alliance for Regenerative Medicine, 1015 18th St. NW, Suite 1102, Washington, DC 20036; email: jlambert@alliancerm.org.
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