Cell therapy growing quickly and serving rare conditions

What is cell therapy and how is it different than gene therapy? There are just a handful of approved cell therapies so far. But many more are lined up for approvals.

What is cell therapy?

Cell therapy involves the intravenous transfer of intact or biologically modified live cells or tissues to a patient.  Cell therapy may prevent, treat, cure, diagnosis or mitigate rare cancers, diseases or injuries. It does so without altering the underlying genetic condition. The cells are cultivated or modified outside of the body before being injected into the patient. Cell therapy restores or alters certain sets of cells, or uses cells to carry a therapy throughout the body.^1,2,3 Cells or tissues used in cell-based therapies may be obtained from the patient’s own cells or tissues (autologous) or from a donor (allogeneic).

CAR-T therapy, or chimeric antigen receptor T cell therapy, uses the technologies of both gene therapy and cell therapy. For Prime’s purposes, we define CAR-T as cell therapy, not gene therapy.

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What is gene therapy? What’s the difference?

Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. Cell therapy involves the transfer of cells with the relevant function into the patient. Some protocols use both gene therapy and cell therapy.

Cells are the basic building blocks of all living things; genes can be found deep within cells. Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body.^1,2,3

Cell therapies on the market as of February 2023

There are seven cell therapy products FDA-approved to date. (See “What is gene therapy?” for a list of gene therapy products.)

An estimated five to 10 cell therapies have PDUFA dates in 2023.

FDA Approved CAR T-Cell Therapy One-Time Administration or One-Time Administration Tissue-Based Therapy with WAC of  > $399,000

Name	Manufacturer	Indication	WAC	Approval Date*
Kymriah™ (tisagenlecleucel)	Novartis	Pediatric ALL & LBCL & FL	$508,250 $399,110	August 2017
Yescarta™ (axicabtagene ciloleucel)	Kite	LBCL & FL	$399,000	October 2017
Tecartus™ (brexucabtagene autoleucel)	Kite	Mantle cell lymphoma	$399,000	July 2020
Breyanzi™ (lisocabtagene maraleucel)	BMS	LBCL	$410,300	February 2021
Abecma™ (idecabtagene vicleucel)	BMS and bluebird bio	Multiple myeloma	$419,500	March 2021
Rethymic™ (allogeneic processed thymus tissue agdc)	Enzyvant	Congenital athymia	~$2.7 million	October 2021
Carvykti™ (ciltacabtagene autoleucel)	Janssen	Relapsed and refractory multiple myeloma	$465,000	February 2022

* Anticipated approval dates are predictions made by Prime Therapeutics, based on industry information.

References

1. https://www.genemedi.net/i/ex-vivo-in-vivo-in-vitro
2. https://asgct.org/education/more-resources/gene-and-cell-therapy-faqs#:~:text=Gene%20therapy%20involves%20the%20transfer,gene%20therapy%20and%20cell%20therapy.
3. https://www.novartis.com/about/innovative-medicines/novartis-pharmaceuticals/novartis-gene-therapies/what-cell-and-gene-therapy#:~:text=Cells%20are%20the%20basic%20building,and%20maintain%20the%20body1.