The In Vivo CAR-T Race
CAR immunotherapy has transformed outcomes in hematological cancers. A subset of patients treated with the first CAR-T therapy, Kymriah, remain completely cancer free 5 years later and may be completely “cured”.
However, access for patients remains extremely limited owing to the logistical complexity and cost of producing CAR-immune cells, which must be extracted from the patient, genetically edited, and reinfused.
To overcome this problem over 30 companies are racing to develop in vivo CAR therapies, where CAR immune cells are generated inside the patient.
Here, we present our report “The current state of in vivo CAR immunotherapy” that identifies both established and emerging companies competing in this space and analyses the current landscape, including emerging technologies and market trends.
● Autologous CAR-T therapy has transformed outcomes in haematological malignancies, with long term follow-up studies demonstrating stable remission and possible cure in some patients.
● This success has translated to significant commercial returns. Gilead acquired Kite Pharma for $11.9BN in 2017 for their CAR-T asset Yescarta, which brought in $1.5BN in revenue in 2024.
● At least 30 companies are now competing to deliver CAR transgenes in vivo, owing to the considerable logistical complexity and cost of autologous cell-therapy, which has limited access for most patients.
● Venture investment is strong in the sector. Notable examples include Capstan Therapeutics ($175M Series B in 2024) and Umoja Biopharma ($100M Series C in 2025).
● Lentivirus and LNP dominate as delivery vehicles, with no clear winner yet emerging.
● Four companies have reached the clinic with in vivo approaches, and the first clinical results are expected 1H 2025.
● CAR therapy is evolving to deliver additional modules. Beyond simple delivery of a CAR, these aim to drive immune cell persistence, tumor penetration and overcome the immunosuppressive tumor microenvironment, facilitating solid tumor targeting or removing the need for lymphodepletion.
● The field is expanding to non-oncology applications, especially autoimmune, which represents a significant market opportunity.
