Cell therapy

Anyone looking for evidence of genetic medicine’s enormous promise need only read of KJ Muldoon. The 10-month-old infant headed home from a Philadelphia hospital in June, dressed in a celebratory cap and gown, after his life-threatening disease was successfully treated with a bespoke CRISPR therapy.

While baby KJ is not cured, the treatment has stabilized his disease, a rare liver condition known as CSP1 deficiency, to such extent he’s able to resume eating a normal diet. Doctors, who hurriedly designed and constructed KJ’s custom therapy in a matter of months, have backed off supportive medications and hope he’ll no longer need a liver transplant.

“Each year, 10 million babies are born with one of about 10,000 known rare genetic diseases, many of which are, in principle, now treatable with genetic medicines,” David Liu, a pioneering CRISPR scientist whose laboratory helped in KJ’s treatment, said at a meeting hosted by the Food and Drug Administration.

“The opportunity created by this perfect storm moment in scientific, medical, regulatory and manufacturing innovation is to provide on-demand genetic treatments like KJ’s at scale.”

Yet Liu and 22 other gene therapy experts and advocates who attended the roundtable didn’t travel to the regulator’s headquarters in White Oak, Maryland to extol the field’s advances. By and large, they came to warn of a crisis.

There are now dozens of approved cell and gene therapies in the U.S., some of which offer near-curative potential for serious diseases like spinal muscular atrophy, sickle cell disease and acute lymphoblastic leukemia. However, the sector that’s produced these therapies is struggling.

Investors have soured on genetic medicine as developers struggle to prove they can profitably sell the complex and often hugely expensive treatments. Biotechnology companies are cutting research, laying off staff and, in some cases, shutting down. Large pharmaceutical firms are no longer willing to bet billions of dollars they can surmount the regulatory and reimbursement hurdles that stand in the way of many of these therapies. And academic labs, still bursting with promising new ideas for technologies like CRISPR, now fear their projects will wither on the vine.

“We estimate that over 100 rare disease gene therapy products that had reached clinical stage have been discontinued since 2023 — not because of treatment failure, but because of the risk of market failure,” said Terence Flotte, dean of the University of Massachusetts’ T.H. Chan School of Medicine and president of the American Society of Cell and Gene Therapy.

“The scientific advances that we have witnessed are just nothing short of spectacular. It’s not hyperbole,” said Crystal Mackall, a professor at Stanford University and founding director of the cancer cell therapy center there. “Despite this unconditional scientific success, the field is really struggling to deliver these therapies to all patients who can benefit.”

Their warnings found a receptive audience in FDA leadership. Commissioner Martin Makary and top official Vinay Prasad, who leads the office that oversees cell and gene therapies, were sympathetic to experts’ arguments and pledged to help.

“We are going to continue the successes of the FDA in facilitating the regulatory process for these conditions and these products,” said Makary. “We’re also going to try to improve by creating more efficiencies.”

Prasad, who in the past has criticized the FDA’s accelerated approval of a gene therapy for Duchenne muscular dystrophy, showed support for flexible trial designs and endpoints when appropriate for the disease or treatment.

He also noted the agency accepts that cell and gene therapies don’t always comes with transformative potential. “We understand that progress is not always made in a single leap,” he added. “We will consider incremental steps forward, because those add up.”

The assembled experts came with lists of possible solutions. Carl June, a famed immunologist and cell therapy researcher at the University of Pennsylvania, called for the U.S. to borrow from the two-tier regulatory system used in China, which allows for medical institutions to more rapidly start first-in-human trials under the supervision of local review boards.

Don Kohn, a University of California, Los Angeles scientist who has developed gene therapies, asked the FDA to reduce the requirements for “comparability” testing when companies transition production from academic to commercial settings. 

Others emphasized the importance of regulatory awards, like the priority review vouchers granted by the FDA to developers of certain therapies, who often sell them for needed capital. And many called for the agency to share more feedback and lessons learned from the applications they receive from industry. 

Behind all of their suggestions was a consistent concern: If regulators don’t help solve the field’s problems, the U.S. risks losing its leadership in developing the kinds of treatments that can cure diseases.

“If we don’t adapt, the next generation of treatments will emerge abroad,” said June. “The future of medicine with cell and gene therapy is at stake.”

Their message seemed to be heard by Makary and Prasad, who noted that many of the issues raised are on their radar at FDA. Prasad, for instance, noted that they hope to redact and make available more internal documents to aid developers’ understanding of what the FDA is looking for. 

“This is not a horse and pony show to say we did this,” added Makary. “This is an honest listening session.” 

Abbvie has agreed to acquire cell therapy developer Capstan Therapeutics in a deal worth up to $2.1 billion, the companies announced June 30. 

The acquisition will hand AbbVie access to technology developed by Capstan that uses small fatty spheres known as lipid nanoparticles to deliver into the body genetic instructions able to engineer specific cells. It’s an ambitious scientific approach that blends the science behind CAR-T cell therapy with that of messenger RNA vaccines.

At the time of the deal’s announcement, Capstan was a few weeks removed from dosing the first patient in a Phase 1 trial of its lead drug candidate, which it’s testing as treatment for B cell-mediated autoimmune diseases. Dubbed CPTX2309, the therapy is designed to reprogram immune T cells to target a protein called CD19 that’s commonly found on B cells.

While CAR-T therapies are now well established in cancer medicine, all existing treatments are constructed by removing a patient’s own cells and reengineering them in a laboratory. The complexity, expense and time involved in this process has, for the most part, kept adoption of CAR-T to just a few treatment niches within oncology broadly. 

Researchers have imagined broader usage could come if production of CAR-T was done by the body, rather than by technicians in a lab. This “in vivo” approach requires delivery of genetic instructions to the target cells.

Capstan accomplishes this by using modified lipid nanoparticles and messenger RNA, building blocks similar to the technology that enables mRNA vaccines to train the body to recognize viral proteins. Capstan engineers its lipid nanoparticles to especially seek a certain type of T cell.

In vivo CAR-T could offer a more convenient alternative in oncology, where AbbVie is already working with partner Umoja Biopharma.

But it also could be well suited for applying CAR-T to immune disease. Academic research from several years ago showed CAR-T’s potential treating diseases like lupus and kindled a frenzy of investment from small and large drugmakers alike. Capstan is one of many companies now exploring whether cell therapy might help “reset” the immune system in diseases where the body’s self regulation has come unglued. 

With its drug CPTX2309, Capstan aims to redirect T cells to hunt down B cells that express the CD19 protein. By clearing both pathogenic and normal B cells, the reprogrammed T cells will prompt this immune reset and hopefully prevent disease progression. 

And because T cell reprogramming is accomplished by mRNA instructions, Capstan believes the engineered T cells its treatment creates will be transient and clear out after accomplishing their job.

Prior to the June 30 deal, the biotech had convinced a good number of investors in its approach. Pfizer, Bayer, Eli Lilly, Bristol Myers Squibb, Novartis and Johnson & Johnson all invested in Capstan in at least one of its private funding rounds, through which the startup has raised $340 million.

The acquisition could pay Capstan’s investors up to $2.1 billion at closing. A spokesperson for AbbVie, reached via email, declined to break down the deal’s terms further.

Earlier this year, AstraZeneca paid $425 million upfront to acquire EsoBiotech, which is also working on in vivo cell therapy. 

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By ProBio

Cell and gene therapies (CGT) are redefining how we treat disease. Driven by innovations and improvements in the field of CAR-T therapies, CRISPR gene editing and mRNA technologies, combined with better ways to deliver these treatments to patients, the market is expected to achieve tremendous growth. Indeed, the global CGT industry is entering a reported scale-up phase, with market forecasts projecting growth worth billions of dollars, with a CAGR between 17% to 22%, depending on source.

This growth is reflected in an expanding pipeline, increasing approvals and maturing platforms across viral and nonviral delivery systems, particularly for CAR-T therapies that reprogram immune cells to attack disease at the biological root. While ex vivo CART has transformed outcomes for patients, its complex personalized manufacturing process, time to therapy and cost, have limited its potential.

In vivo CAR-T offers an exciting alternative path. In vivo CAR-T therapy engineers the patient’s own immune cells, to target and destroy cancer cells, inside their body. This eliminates the need for some of the complex and costly elements of ex vivo CAR-T, such as cell manufacturing, simplifying production, reducing costs and allowing patients more accessible and timely treatment options.

Strategic investment and collaborations are on the rise for vivo CAR-T therapies. Kelonia Therapeutics recently announced the first patient dosing for anti-BCMA in vivo CAR-T (KLN-1010) and Vivacta Bio announced promising in-human results for GT801, an in vivo CAR-T therapy for Non-Hodgkin’s Lymphoma. In addition, numerous companies can be seen to be investing in their pipelines, services and products to support the growing market.

Considerations for Manufacturing In Vivo CAR-T Therapies

In vivo CART delivers genetic payloads via viral vectors, such as lentivirus (LVV) and adeno-associated virus (AAV) and nonviral carriers such as targeted lipid nanoparticles (tLNPs). LNPs deliver mRNA encoding the CAR construct directly into T cells in vivo, where the mRNA is translated into CAR protein, but does not integrate into the genome. LVVs deliver DNA encoding the CAR construct, which integrates into the T-cell genome.

The consideration of which delivery system to use will depend on several factors:

Safety and Immunogenicity

tLNPs enable the integration of RNA into immune cells by transient expression, providing a shorter expression of days to weeks, depending on mRNA stability and immune clearance. Viral vector delivery enables a more stable integration, with long-term expression (years) and carries higher risks due to genomic integration. Primary investigations have suggested that tLNPs have potential for autoimmune disease, whilst LVV offers potential for treating cancer.

Targeting Efficiency

LNPs can be engineered to target specific tissues; however, this does require optimization of the LNP formulation to avoid adverse effects on non-targeted tissues. The LNP is conjugated with a targeting antibody which binds to the T cell receptor. Viral vectors are highly efficient at delivering genes into cells, with precise regulation achievable. The viral vector envelope is modified to de-target first, followed by another plasmid coding targeting antibody to retarget T cells.

Manufacturability

The LVV manufacturing process is mature but still faces CMC challenges. mRNA-tLNP is a new modality with the advanced project, in clinical stages, so it is difficult to compare at this stage. Lipid nanoparticles (LNPs) currently dominate due to scalability, faster turnaround, and consistency, particularly in oncology and immunology, though technical challenges remain around targeting, conjugation, and QC. Specialization in advanced QC testing for targeted LNPs with high-end analytics and comprehensive QC panels is critical. Assays for potency, lipid composition, and endosomal escape profiling are increasingly recognized as necessary for clinical success.

Although viral delivery markets are well established, non-viral delivery is seeing fast growth and innovation, with more standard offerings driving towards platformization and broader adoption.

The Role of Investigator-Initiated Trials for In Vivo CAR-T

In vivo CAR-T offers promise to expand into treating solid tumors and autoimmune diseases, an area where ex vivo CAR-T has not been able to fully realize potential due to scalability and accessibility issues. Investigator-initiated trials (IITs) may offer a way of helping fulfil that promise.

IITs are clinical studies conceived, designed and led by independent investigators, usually academic centers. These centers can leverage academic infrastructure and ethics processes, often reducing start-up time and cost. Because of their position outside a corporation, they can focus on exploratory work and early-stage innovation, to enable rapid testing. This offers a time advantage for emerging innovative therapies, bringing them to patients more quickly.

An example of IITs driving innovation was shown recently by EsoBiotec, a small startup working on an immune shielded lentiviral BCMA in vivo CART (ESOT01). EsoBiotec worked with Chinese doctors to run an investigator lead-initiated trial, compressing lab-to-clinic timelines, showing positive results quickly. Indeed, China is very attractive for IITs for numerous reasons such as its large patient population, regulatory flexibility at early innovation stages, academic structure and expertise, cost efficiency and government and institutional support.

The move to in vivo CAR-T is redefining the CGT landscape. To deliver on its promise, manufacturers will need to have expertise in many areas of advanced therapy manufacturing, providing flexible, scalable platforms, support from cell line development to CMC and guidance through regulatory frameworks.

The Food and Drug Administration is easing limitations it imposed around the complex cancer drugs known as CAR-T therapies, removing several onerous requirements as well as loosening restrictions on which facilities can provide treatment.

The FDA’s action applies to therapies made by Bristol Myers Squibb, Gilead Sciences, Johnson & Johnson and Novartis that are used to treat several types of blood cancer. Specifically, it removes so-called “Risk Evaluation and Mitigation Strategies” from the drugs’ labeling, which are used to help manage serious side effects of treatment.

According to the FDA, these REMS requirements are no longer necessary to ensure safe use of these CAR-T therapies as both physicians and hospitals are now well versed in managing the two syndromes most commonly associated with the drugs.

Since the FDA’s approval of Novartis’ Kymriah for leukemia in 2017, six more CAR-T therapies have reached market. These medicines are made from a patient’s own immune cells, extracted from the body and engineered in a lab to hunt down specific proteins found on the surface of malignant clones.

For some people with leukemia, lymphoma or multiple myeloma, they can prompt powerful responses and even long-lasting remission. But their administration also comes with notable risks, including a hyperactive immune response known as CRS and neurological toxicity dubbed ICANS.

When CAR-T therapies were new, physicians and the hospitals they worked at faced a learning curve handling these treatment-associated conditions, which require specific management and drugs to bring under control.

The REMS imposed by the FDA was designed to help mitigate these risks by requiring hospitals receive special certifications before they could administer treatment, and mandating specialized reporting of side effects to the agency.

“Given the established management guidelines and extensive experience of the medical hematology [and] oncology community in diagnosing and managing the risks of CRS and neurologic toxicities across products in the class of BCMA- and CD19-directed autologous CAR-T cell immunotherapies, FDA has determined that the safe and effective use of CAR-T cell immunotherapies for the indicated population can be assured without a REMS,” the agency said in a June 27 statement. BCMA and CD19 are the protein targets of the seven approved CAR-T therapies.

Reporting rates for CRS and ICANS have “remained stable,” the FDA added.

Alongside removal of the REMS, the regulator also reduced its requirement that patients remain nearby the facility they received treatment from one month to two weeks. And its limitation on how long patients must wait before they can drive or operate machinery again after treatment is lowered from two months to two weeks.

Removal of the REMS should lower the logistical hurdles patients face in receiving CAR-T treatment as well as the cost of administration, the Alliance for Regenerative Medicine, a trade group, said in a post to LinkedIn.

“We believe the reduction in CAR-T burden for patients and caregivers, enabled by FDA's new monitoring and driving requirements, will drive expansion of the U.S. cell therapy market,” Daina Graybosch, an analyst at Leerink Partners, wrote in note to clients.

In a statement, Bristol Myers, which sells the CAR-T therapies Abecma and Breyanzi, noted that only two in 10 eligible patients receive the treatments “due to the confluence of complex logistical and geographic barriers affecting patients and providers.”

The changes could help expand use of CAR-T from specialized medical institutions, where it’s most commonly given, to community health centers, Bristol Myers said.

Breyanzi, Kymriah and Gilead’s Yescarta and Tecartus are cleared to treat lymphoma, although Kymriah is more commonly used for leukemia, where it is also approved. Abecma and J&J’s Carvykti are used for multiple myeloma.

The most recently OK’d CAR-T treatment, Autolus Therapeutics’ Aucatzyl for lymphoma, was the first to be cleared without an initial REMS program.

Kyverna Therapeutics will seek clearance of what could be the first marketed cell therapy for an autoimmune condition after the treatment succeeded in a pivotal trial in a progressive condition known as stiff person syndrome.

According to Kyverna, four months after treatment with its therapy miv-cel, Phase 2 study participants had a median 46% improvement from the trial’s start on a type of timed walking test. More than four-fifths had at least a 20% improvement, a threshold that’s believed to be “clinically meaningful,” Kyverna said in December. The therapy met its key secondary study goals as well.

Kyverna said there were no “high-grade” cases of the immune or neurological side effects associated with treatments like miv-cel. There were Grade 3 or 4 instances of neutropenia, or low counts of a kind of white blood cell, but Kyverna said they were “manageable.” The company plans to ask for U.S. approval in the first half of 2026.

Kyverna has long been a front-runner in the race to transform the types of cell therapies originally developed for cancer into autoimmune disease treatments.

Originally formed to pursue so-called Treg cell therapy, the company changed direction and licensed a more conventional cell therapy technology that’s yielded what’s now known as miv-cel. Fueled by academic studies suggesting these therapies might rewire the immune systems of people with autoimmune disorders like lupus, it raised hundreds of millions of dollars in private and public funding. But mixed early results and growing competition tempered excitement about these treatments, depressing the share price of Kyverna and many of its peers. 

Kyverna swapped CEOs and, under a new leader, began developing miv-cel for stiff person syndrome, a disorder that causes intensifying muscle stiffness. While rare, the condition has less therapeutic competition than Kyverna’s other targets and, because of its fast progression, offered a quicker path to clinical results.  

Since then, Kyverna has begun clawing back some of its stock value and quickly completed enrollment in a registrational trial. Its goal in the 26-patient study was for miv-cel to help drive at least a 20% change on a timed, 25-foot walking test. Executives had pointed to case reports suggesting miv-cel could produce more powerful effects.

According to Leerink Partners analyst Thomas Smith, the fresh results are a “best-case scenario” for Kyverna, enabling the company to bring to market the first and only therapy for stiff person syndrome as well as, more broadly, the first approved cell therapy for an autoimmune disease.

In addition to clearing the important walking test threshold, miv-cel also helped many patients no longer need a walking device after 16 weeks and, so far, has freed all study participants from the immunotherapies typically used to treat their condition. 

“We believe this highlights miv-cel's potential to provide unprecedented clinical benefit, while significantly reducing or eliminating chronic treatment burden,” Smith wrote. 

Kyverna will provide detailed study results at a medical meeting in 2026. Company shares climbed by more than 25% shortly after the results were disclosed. 

Bristol Myers Squibb is joining big pharma’s rush into “in vivo” cell therapies, paying $1.5 billion to acquire Orbital Therapeutics for a technology designed to rewire the immune systems of people with inflammatory conditions.

The deal, announced in October, gives Bristol Myers ownership of a company that’s been working on ways to genetically modify immune cells inside the body. Orbital’s lead program, OTX-201, does so by sending into cells “circular” RNA instructions training them to seek out cells with a particular protein flag. OTX-201, which is envisioned as an autoimmune disease treatment, could begin human testing in 2026. 

The acquisition expands Bristol Myers’ presence in cell therapies. The company is already one of the field’s leaders, with multiple marketed medicines for blood cancers. But, like its peers, Bristol views autoimmune disorders as a way to potentially broaden use of the complex treatments.

Bristol Myers has already been working on autoimmune cell therapies that involve “ex vivo” procedures, in which technicians genetically modify patient cells in a lab. But “in vivo” technology could be a more convenient alternative and way to overcome some of the safety hazards — like a chemotherapy conditioning regimen — associated with their more complex counterparts. Those advantages may be particularly helpful in treating autoimmune conditions, which aren’t as deadly as cancer. 

Several pharmaceutical companies have already bought into the concept. In 2025 alone, Gilead Sciences, AbbVie and AstraZeneca have all acquired companies working on in vivo cell therapies.

“In vivo CAR-T represents a novel treatment approach that could redefine how we treat autoimmune diseases,” said Robert Plenge, Bristol Myers’ chief research officer, in a statement. “This acquisition enhances our robust cell therapy research platform and provides an opportunity to advance a potential best-in-class therapy designed to deplete autoreactive B cells and reset the immune system.”

Like other autoimmune cell therapies in development, OTX-201 is designed to eliminate the B cells that can malfunction in an array of autoimmune diseases, such as lupus and myasthenia gravis. It teaches cells to go after the same target, CD19, as multiple approved therapies for cancer, among them Bristol Myers’ Breyanzi.

Orbital launched in 2022 and was co-founded by longtime Alnylam Pharmaceuticals CEO John Maraganore. The company raised $270 million in an initial funding round and has a portfolio of RNA delivery technologies. In 2025, it presented data from preclinical studies showing its approach could lead to “full B cell depletion in blood, spleen, and lymph nodes, which is required for an effective immune system reset in autoimmune disease.”

The most effective drug for Parkinson’s disease hasn’t changed in 50 years. But fresh research published in one of the country’s top scientific journals is helping build the case for a more cutting-edge approach that uses stem cells to restore important brain functions.

The second most common neurodegenerative illness, Parkinson’s is caused by the loss of certain nerve cells. These cells produce a chemical messenger, dopamine, that plays a crucial role regulating movement. Since the 1970s, a drug called levodopa, which the body converts into dopamine, has been the mainstay treatment for combating the tremors, slowness, stiffness and balance issues that come with Parkinson’s.

Levodopa doesn’t stop the disease, however, so scientists have spent decades trying to find a more permanent fix. The latest development in that search comes from two clinical trials testing stem cell-derived therapies transplanted into the brains of Parkinson’s patients.

The first of those trials, conducted at the Kyoto University Hospital in Japan, evaluated seven people who received what are essentially blank slate cells that can turn into dopamine-producing neurons. These cells were created using a technology pioneered at Kyoto University, where roughly 20 years ago, scientists discovered how to reset some adult human cells to act like stem cells.

The study’s main focus was safety. Results, published in April in the journal Nature, show no serious adverse events were reported, though researchers did identify 73 mild to moderate events. Importantly, there was no evidence the introduced cells started growing in an unchecked, tumor-like fashion — a major concern with stem cell implants.

The study also explored whether this therapy had any effect on patients’ disease. Two years post-treatment, the six participants who were evaluable had gotten better on a scale clinicians use to measure the severity of Parkinson’s motor symptoms. Researchers looked at what’s known as “on” time, when medications are adequately controlling these symptoms, as well as the opposite “off” time, and found scores improved an average of 36% and 20%, respectively.

Another test indicated that dopamine-producing cell activity had increased substantially. This was determined through a technique in which researchers strap a radioactive element to a molecule of levodopa and then use a PET scan to follow its path through the nervous system, somewhat like how a submarine tracks objects on a sonar display.

The second trial took place at sites throughout the U.S. and Canada, and employed a different kind of stem cell that comes from human embryos. It enrolled a total of 12 participants and followed them for a year post-transplant.

Similar to the Japan study, there were no deaths, serious adverse events or tumor-like tissue growth related to the introduced cells. Trial runners did report two serious events overall. One participant was hospitalized with COVID-19, while another had a seizure that was attributed to the surgical procedure.

The trial also saw improvements in “off” scores on that disease severity scale and positive results from the radioactive levodopa test.

The cell therapy tested, called bemdaneprocel, is being developed by Bayer subsidiary BlueRock Therapeutics, which back in August 2023 announced some of the trial data. The company expects to start a Phase 3 experiment before the end of June.

Hideyuki Okano, a stem-cell scientist at Keio University in Tokyo, argues more research is needed to confirm these types of cell therapies are effective against Parkinson’s.

Still, in an accompanying Nature editorial, he called the new trial results “encouraging” because they suggest treating Parkinson’s patients with donor cell transplants is likely safe. That both trials “proved to be safe, and hinted at possible efficacy, is an important step towards the establishment of this cell therapy for Parkinson’s disease in wider society,” he wrote.

Cell therapy for Parkinson’s isn’t a new idea. In 1989, a team led by neuroscientist Olle Lindvall performed the first cell transplantations for Parkinson’s patients. While the landmark study didn’t show a major therapeutic benefit, a few interesting signals encouraged the research community to keep exploring.

One ethical concern from that trial was that it used a relatively large amount of tissue derived from aborted fetuses. In the decades since, Parkinson’s and cell biology experts have made significant progress identifying other, less controversial sources of stem cells that can be scaled more easily.

A milestone of that work came in 2020, when the technique invented at Kyoto University was used to turn a Parkinson’s patient’s own skin cells into the early stages of dopamine-producing neurons, which were then implanted into his brain.

According to Okano, as of December, there were 115 clinical trials testing 83 products derived from “pluripotent” stem cells. And among those targeting central nervous system disorders, the ones for Parkinson’s disease are at a more advanced stage of development.

Further behind Bayer and BlueRock, Aspen Neuroscience, a San Diego-based biotechnology company, has a Parkinson’s therapy derived from patients’ own cells currently in early-stage human testing.

Available cell therapies for cancer aren’t easy treatments to receive. Personalized to each patient, the drugs are complicated and time-consuming to manufacture, and must be delivered and administered on an exacting schedule. And because they come with serious risks, patients need to remain close to the hospital where they were treated for weeks of monitoring.

All of these characteristics make Johnson & Johnson’s choice to roll out direct-to-consumer advertising for Carvykti, a cell therapy it sells for the blood cancer multiple myeloma, somewhat unexpected. The ad, which will air on streaming services like YouTube, Paramount and Roku, is J&J’s first for the drug and a rare instance of DTC marketing for this kind of cell therapy, known as CAR-T.

According to June Lanoue, J&J’s head of U.S. hematology, the company aims to raise awareness about cell therapy, particularly among patients who may not be receiving care from the kind of specialized academic centers where CAR-T is often given.

Carvykti is currently approved in the U.S. to treat multiple myeloma in patients whose disease either relapsed or becomes resistant to treatment after an initial round of therapy. Another CAR-T therapy, called Abecma and sold by Bristol Myers Squibb, is also cleared for this use, but only after two or more prior lines of treatment.

Among these “relapsed, refractory” patients, about half are treated at community oncology centers, Lanoue said. “The actual awareness level of these options is very low,” she added. “In the community, if they’re not able to get it, doctors are not talking about and therefore they don’t have visibility to having a new option.”

J&J’s choice to advertise is a signal of confidence in its ability to manufacture enough Carvykti to meet any rise in demand. Early on in the drug’s launch, production challenges forced the company to limit orders from treating centers. (Bristol Myers ran into similar issues.)

“We feel very confident around production,” said Lanoue. Treatment sites can “order Carvykti without any constraint.”

The launch of the ad came several weeks after the Food and Drug Administration took steps to improve access to CAR-T treatment broadly. The agency eased some side effect reporting requirements and lessened the time it mandates patients stay near their treating facility for monitoring. It also said patients could resume driving or operating machinery sooner after treatment than previously prescribed.

“We think it will give more opportunity to more patients who [live at] a little bit further distance to be able to receive Carvykti,” Lanoue said.

J&J’s 120-second ad introduces a composite patient, “Marvin,” and describes the heavy burden of injections and pills typically used to control multiple myeloma. Carvykti, by contrast, is a one-time infusion that can deliver lasting benefits in some.

Recent follow-up data from an early study of around 100 patients treated with Carvykti showed that, at five years, one-third had not yet experienced disease progression. A story published in June by The New York Times that highlighted these findings led to more patients requesting information about Carvykti, according to Lanoue.

J&J did not share how much it’s spending on this marketing campaign, but Lanoue said it is “significantly less” than what a pharmaceutical company would typically spend on a regular TV advertisement. “This is not a share of voice play; this is ultra targeted,” she added.

Sales of Carvykti reached $439 million in the three months between April and June. The company expects peak annual sales to eventually eclipse $5 billion.

Iovance Biotherapeutics has laid off staff in a restructuring that followed a significant cut to its revenue forecasts earlier this year.

A spokesperson in August confirmed Iovance’s plans to reduce its workforce by “less than 20%.” A regulatory filing showed Iovance employed 838 people at the end of last year, though a company presentation last month indicated that number had grown to more than 1,000. Iovance’s workforce will still have more than 1,000 people following the job cuts, which will affect full-time employees as well as contractors, according to the spokesperson.

“After careful evaluation of our long-term goals, operational needs, and resources, we have implemented a strategic restructuring that includes a selective reduction in force to support our mission to innovate, develop and deliver TIL cell therapy to patients in need,” the spokesperson wrote in an email, referring to the type of cellular medicine Iovance specializes in. “This restructuring will extend our cash runway, and we remain on track to deliver on our financial goals.”

In a second quarter earnings report Aug. 7, the company said that 19% of its workforce would be impacted by the cuts. The restructuring will save Iovance more than $100 million annually and enable it to operate through the fourth quarter of 2026. Iovance will also “continue to optimize and refine its cost structure” through what it described as “operational excellence initiatives” over the next few quarters, it said in a statement.

Iovance had about $307 million in cash on hand at the end of June. 

The layoffs come after a slow commercial start for Amtagvi, a cell therapy Iovance developed for an advanced form of skin cancer. Amtagvi is the first marketed cellular medicine made by engineering human cells known as tumor-infiltrating lymphocytes, or “TILs.” The therapy originated from research at the National Institutes of Health in the 1980s and got to market only after decades of fine-tuning, as well as multiple regulatory delays.

Amtagvi was cleared in February 2024 for people whose melanoma progressed after a commonly used immunotherapy or targeted cancer medication. It’s derived from a patient’s tumor tissue in a complex, weekslong manufacturing process. Upon approval, analysts and investors were skeptical of its sales potential, given questions about demand and the complex logistics involved. At about $515,000, the drug’s list price was also at the time the highest of any cell-based medicine for cancer in the U.S.

Iovance initially projected product revenue — which includes contributions from a different immunotherapy called Proleukin — would reach $450 million to $475 million this year. But in May, it cut that forecast by 40%, to between $250 million to $300 million, citing “recent launch dynamics” that had changed its view. In a research note last month, Mizuho Securities analyst Salim Syed noted how prior guidance was modeled on launches from other marketed cancer cell therapies known as CAR-T treatments, whereas the updated projections rely on “real data” from centers actively administering Amtagvi.

Iovance said in its latest earnings report that 102 patients were treated with Amtagvi in the second quarter, equating to about $54 million in sales. Shares fell about 30% as the numbers “were on the lighter side” of the 100 to 110 patients Iovance had anticipated treating, and total product revenue of $60 million missed consensus estimates, wrote Syed in an Aug. 7 note to clients.

Amtagvi could be approved in Europe, the U.K. and Canada this year. It’s also in late-stage testing for non-small cell lung cancer.

Takeda will abandon all of its cell therapy work, retreating from research into a field it had made once made a strategic priority.

The Japanese pharmaceutical giant said in October that, as part of a “strategic portfolio prioritization process,” it will stop its cell therapy research. Takeda plans to seek an “external partner” to advance that work, which includes platform technologies and certain “clinic-ready programs.” It doesn’t currently have any clinical trials underway. 

Takeda will redirect near-term investments into programs that can “deliver transformative therapies to patients at increased speed and scale,” the company said. Preclinical work on biologics, small molecules and antibody drug conjugates is producing “novel and highly innovative” candidates, Takeda added. 

The move marks the end of a yearslong foray by Takeda into cell therapy. Though a late entrant, trailing early adopters like Gilead and Novartis, the company zeroed in on the field as part of a broader push into genetic medicine late last decade. Over the next few years, the company built up its manufacturing capabilities and turned to dealmaking to build out a portfolio. 

In the last year or so, however, Takeda has dialed back that work. Amid a broader profit decline catalyzed by patent losses, Takeda restructured in 2024. In the process, the company cut a series of programs for solid tumors and blood cancers and pivoted to autoimmune conditions, as many other cell therapy developers have amid a tough funding climate.

Takeda said it expects to take an impairment charge of about 58 billion yen, or roughly $394 million, associated with the “gamma delta” cell therapy technology it had been working on. The company said it will “continue to assess any other related impacts” and factor them into its next quarterly results.