Eight CAR-T cell therapy companies you should know about

Chimeric antigen receptor (CAR)-T cell therapies have gone down in history as a major breakthrough in cancer treatment. Although more advanced cell therapies attempt to overcome the limitations of CAR-T, it has been a significant advancement in cancer research, and more companies are joining the pursuit of new and innovative approaches in the field. 

CAR-T typically works by collecting a patient’s T cells and engineering them to recognize and target a specific protein on cancer cells. Once these engineered cells are grown in the lab, they are sent back into the patient’s bloodstream, where the cells then go on to attack cancer cells. 

In this article, we look at eight promising biotech companies that are advancing their CAR-T cell therapy candidates in the clinic that have grabbed media attention this past year. 

Cabaletta Bio 

Having appointed a new chief commercial officer (CCO) last month, Pennsylvania-based startup Cabaletta Bio has made heads turn this past year.  

The company’s platform technology CABA has been designed to create CAR-T therapies specifically tuned to address autoimmune diseases. Its lead candidate CABA-201, is a fully human CD19-CAR T cell therapy, which is in early stages of clinical trials for systemic lupus erythematosus – an autoimmune disease wherein the body’s immune system mistakenly attacks healthy tissue – the muscle inflammation condition myositis, systemic sclerosis, a condition wherein the overproduction of the collagen protein results in the thickening of organs, and generalized myasthenia gravis, another rare autoimmune disorder characterized by muscle weakness. 

The candidate is said to employ specific targeting domains, custom-made for each autoimmune disease, and designed to permanently eliminate only the disease-causing B cells and sparing normal B cells. It is an autologous treatment where a patient’s T cells are extracted and edited in a lab to target B cells before being reinfused into the patient. 

Recently, Cabaletta revealed that CABA-201 was successful in depleting harmful B cells and lower autoantibodies in the body, and managed to do so in patients without preconditioning, which is often a chemotherapy administered to patients to the prepare the body for CAR-T as well as to boost the effect of the CAR-T therapy itself. In fact, the treatment was found to eliminate all problematic B cells in two out of the three patients with various autoimmune diseases, and in the third patient, it still decreased the number of these cells. 

Lyell Immunopharma 

California-based company Lyell Immunopharma has a pipeline dedicated to next-generation CAR-T cell therapies. What makes them next generation? 

The T cells are being reprogrammed to maintain its cancer cell-killing ability while resisting exhaustion to treat solid tumors. Typically, CAR-T therapies are directed towards blood cancer, and so far, they’ve not made significant progress in treating solid tumors because the dysfunctional state that T cells enter hinder their ability to respond to cancer cells and kill them. So, targeting T-cell exhaustion seems like the right thing to do to tackle solid tumors, which represent around 90% of all cancers. 

Lyell’s lead candidate is ronde-cel, also called LYL314, is designed to target two antigens present on B-cell lymphoma, namely CD19 and CD20. B-cell lymphoma is a type of blood cancer that originates in the B-cells. It is currently in the clinic being tested in a phase 3 trial that will enrol 120 patients. The company plans to submit a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) in 2027 for approval.  

To add to its clinical CAR-T pipeline, Lyell bought Maryland-based Innovative Cellular Therapeutics’ GCC19CART, which has reaped positive results in patients with metastatic colorectal cancer (mCRC) who don’t respond well to currently available treatments, last week. It demonstrated a 67% overall response rate and an 83% disease control rate at the highest dose level. Now dubbed LYL273, Lyell will take over the reins further into the clinic. 

Kyverna Therapeutics 

California-based Kyverna Therapeutics is getting closer to FDA approval for one of its CAR-T candidates to address autoimmunity. 

Based on its T-cell cell engineering platform, cells are programmed to kill disease-causing cells. As with any autologous cell therapy, Kyverna’s approach is to collect the white blood cells of patients where the T cells are separated and then altered to recognize, attack, and destroy the problematic B cells implicated in the development of the disease. Through intravenous infusion (IV), these T cells are injected back into the patient’s body to kill said B cells. 

Its candidate KYV-101 is in the lead to treat two autoimmune conditions, stiff person syndrome, a rare, neurological disorder that causes muscle stiffness and painful spasms, and myasthenia gravis, which causes weakness in the muscles. For both these conditions, KYV-101 has achieved the Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. RMAT is assigned to cell therapies and regenerative medicines to speed up their development in the clinic. 

In patients with myasthenia gravis, the medicine hit all primary and secondary endpoints, according to interim results from a phase 2 trial. All the patients experienced reductions in the MG-ADL and QMG scores – both scales used to measure the severity of the disease. Moreover, minimal symptom expression (MSE) was achieved in two out of three patients over six months of follow-up. MSE is a score that records the symptoms associated with the muscle weakness condition in patients and is regarded as a major treatment goal for many patients. A phase 3 study is expected to begin soon. 

Meanwhile, for stiff person syndrome, Kyverna is continuing with phase 2 trials and claims it’s on track to submit its first Biologics License Application (BLA) with the FDA to potentially market the drug early next year. KYV-101 is also in the early stages of the clinic for autoimmune conditions like lupus nephritis and multiple sclerosis, among others. 

To support the clinical development of KYV-101, Kyverna nabbed $25 million from Oxford Finance two weeks ago. This is part of a larger financing of up to $150 million in non-dilutive funding, as the startup gears up to potentially become the first company to have CAR-Ts that address autoimmunity on the market. 

Carsgen Therapeutics 

The Chinese-American biotech company Carsgen Therapeutics has been locked into the CAR-T space for more than a decade. It ushered in 2025 with promising phase 2 results for its stomach cancer-addressing cell therapy. 

Its autologous CAR-T therapy satri-cel was found to improve progression-free survival – the length of time a patient lives with cancer during and after treatment without their condition worsening – in patients whose stomach cancers failed other treatments. Satri-cel targets a protein called Claudin18.2, which is abnormally expressed in tumors, particularly stomach cancers. 

Since then, satri-cel’s new drug application (NDA) has been accepted by regulators and having been granted priority review, it is en-route to approval in China.  

Carsgen also has other autologous CAR-Ts in the clinic, but in their early stages in China and the U.S., namely CT071 and CT011 in blood cancers. Plus, it is advancing allogeneic CAR-Ts in clinical trials in China. These therapies use cells from healthy donors, which are engineered and injected into patients to treat cancers.  

The biotech already has one approval in the bag. Its autologous CAR-T therapy zevor-cel was cleared in China to treat people whose multiple myeloma, a blood cancer that originates in white blood cells, has progressed even after three lines of therapies last year. 

Wugen  

Missouri-based Wugen is another CAR-T therapeutics startup that drew attention this year when it scooped up $115 million to power the clinical development of its CAR-T candidate WU-CART-007. 

The allogeneic candidate is in phase 2 trials to treat T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (LBL). T-ALL is an aggressive blood cancer that stems from the T-cells in the bone marrow and LBL is another aggressive type of blood cancer that begins in the thymus, an organ located in the upper chest. Designed to target abnormal T-cells in the body, WU-CART-007 disrupts the T-cell receptors.  

Wugen dosed its patients in the pivotal trial in March, and the study is ongoing. This was following a phase 1/2 study where WU-CART-007 exhibited anti-tumor activity. A 91% overall response rate – a measure of how effective a treatment is in shrinking tumor size – was achieved and in patients who had undergone treatment prior to the study, and a 73% composite complete remission rate was observed. The latter is a measure of remission, when all signs and symptoms of cancer have disappeared following treatment. 

As allogeneic cell treatments have a heightened risk of triggering graft vs. host disease, a complication linked to cell transplants when a donor’s foreign cells attack a patient’s cells, WU-CART-007 is specifically tailored to evade this side effect. With the help of gene editing technology, Wugen deleted certain proteins that set off these unwarranted side effects in the body. 

The American startup is fully focused on WU-CART-007. With no other candidates in its lineup, WU-CART-007 is being tested preclinically in T-cell non-Hodgkin’s lymphoma, a rare blood cancer, acute myeloid leukemia, which is characterized by the growth of abnormal cells that build up in the bone marrow and blood, and undisclosed autoimmune conditions. 

Cartesian Therapeutics 

Situated in Maryland, Cartesian Therapeutics is another CAR-T developer, but unlike most others advancing research and development (R&D), Cartesian’s platform involves mRNAs. 

Combining mRNA technology and cell therapy research, Cartesian wants to take on autoimmune diseases. The CAR-T cells have mRNA molecules that have been engineered to temporarily modify the T cells without the genome integrating into the cells. When the CAR-Ts encounter the disease-causing cells, they bind to these cells and further divide into more CAR-Ts with fewer mRNA present in the newly formed CAR-Ts to reduce toxicity but improve efficacy. 

Emerging from this platform, Cartesian’s lead candidate is Descartes-08, an autologous therapy that targets the protein B-cell maturation antigen (BCMA) involved in aiding hyperactivity in the immune system. It is in phase 3 trials to treat myasthenia gravis – the first patient was enroled in May – and in phase 2 studies for systemic lupus erythematosus (SLE) and myositis, another autoimmune disease driven by inflammation and causing muscle weakness. 

Phase 2 results from an SLE study demonstrated that the candidate had 100% LLDAS response rates. LLDAS is a treatment target that represents low disease activity and is linked to a decreased risk of organ damage and flares – periods when lupus symptoms worsen. Two out of three patients were in remission after three months, according to results published two weeks ago. 

Kite 

At the forefront of CAR-T cell therapeutics is California-based Kite Pharma. Known for its autologous CAR-T drug Yescarta to treat LBCL, which was approved by the FDA in 2017, Gilead-owned Kite is now testing the drug in the clinic to address other forms of lymphoma.   

The drug, which contains the active ingredient axicabtagene ciloleucel, is in two phase 3 trials for patients with high-risk follicular lymphoma and high-risk lLBCL, and in another high-risk LBCL phase 2 trial. The drug, however, does come with a boxed warning for side effects such as cytokine release syndrome. 

Second in line is its other phase 3 CAR-T candidate anitocabtagene autoleucel, which targets BCMA in multiple myeloma. Kite is currently recruiting participants who have already received other lines of therapy, including treatments like immunomodulators or antibodies, in collaboration with fellow California-based CAR-T developer Arcellx. The candidate is an up-and-coming rival of Legend Biotech’s CAR-T Carvykti, anticipated to topple the latter’s market position. 

It bought Pennsylvania-based Interius BioTherapeutics for $350 million in August, with the goal to up its in vivo cell therapy game. Unlike ex vivo therapies like Yescarta where cells are harvested and engineered outside the patients’ body and reinfused, in vivo therapies involve modifying the genetic material directly inside the patients’ bodies, making them a more personalized approach. Moreover, this process does away with preconditioning the body with chemotherapy, and according to Kite, it is supposed to offer a more durable and long-lasting therapeutic effect. 

Zeroing in on vivo therapies, Kite is putting aside $1.6 billion for Guangdong-based Pregene Biopharma, which is developing CAR-Ts, CAR-NKs, and nanobodies. The deal saw the Chinese biopharma snag $120 million upfront and aims to advance CAR-Ts against cancer and autoimmune diseases. 

Legend Biotech 

Yet another pioneer in the CAR-T cell therapies field, New Jersey-based company Legend Biotech is best known for co-developing the CAR-T therapy for multiple myeloma, Carvykti, along with Johnson & Johnson. It was greenlit by the FDA more than three years ago and was the first of its kind to score FDA approval for the blood cancer indication. Now, with Kite and Arcellx’s anitocabtagene autoleucel closing in on the CAR-T multiple myeloma space, it looks like Legend is only doubling down. 

Carvykti is being tested in different cohorts of patients with multiple myeloma across phase 1,2, and 3 trials. These patients include those who have not responded to other therapies, people whose cancer has come back after a period of remission, patients who are eligible for a stem cell transplant as well as those who are not. 

A study found that a third of the patients with relapsed/refractory multiple myeloma who took Carvykti and had been heavily pretreated have remained progression-free for five years, Legend revealed in June. 

Beyond Carvykti’s growing momentum in the clinic, Legend also has CAR-T candidates being tested to treat autoimmune conditions and other cancers such as gastric, pancreatic, and small cell lung cancer – for which it has teamed up with Swiss giant Novartis for. Some of the drug targets include Claudin 28, CD19, CD20, CD22, all associated with disease development. 

CAR-T cell therapies: an evolving space 

Research and development in the CAR-T sector has seen mixed results this past year, with several biotechs announcing layoffs and slashing pipelines but also therapies climbing up the clinical ladder. California-based Caribou Biosciences let go of a third of its employees earlier this year, and so did another California-based biotech Allogene Therapeutics, which also reported a patient death linked to one of its cell therapy programs. Moreover, Belgian company Galapagos has closed the shutters of its cell therapy wing, having failed to find a buyer. 

None of this has put a stopper on progress at large. As seen with the aforementioned young startups and biotechs, there is a lot going on in the field. University College London-spinout Autolus Therapeutics has got obe-cel up its sleeve, which targets the CD19 protein and has the potential to address a number of cancers, such as non-Hodgkin lymphoma, chronic lymphocytic leukemia – another cancer of the blood and bone marrow – primary central nervous system lymphoma, which is a rare non-Hodgkin lymphoma that begins in the lymph tissue, as well as the autoimmune condition systemic lupus erythematosus. Autolous is pushing through amid BioNTech’s forfeiture of its option to co-develop a CAR-T therapy along with it. 

Similarly, London-based Leucid bio’s CAR-T candidate for solid tumors debuted in the clinic earlier this year and Texas-based March Biosciences’ MB-105 hit phase 2 studies in February and just last week, it was awarded the RMAT designation by the FDA to treat CD5-positive T-cell lymphoma. Besides, California-based Anixa Biosciences is working with the Moffitt Cancer Center in Florida to continue the development of its CAR-T candidate for ovarian cancer, which is being evaluated in phase 1 trials. And Umoja Biopharma recently racked up the fast-track designation for its CAR-T candidate from the FDA.  

Although CAR-T technology was invented nearly forty years ago, it has since evolved. And as tackling side effects remain a priority along with addressing diseases apart from cancer like autoimmune conditions, new forms of this technology aim to address patients’ needs.