CTMC创始人Jason Bock谈细胞治疗：第十个IND获批与商业化可扩展性瓶颈

What was your biggest professional highlight of the past 12 months?

The biggest highlight has been seeing the novel CTMC model we created repeatedly translate scientific innovation into real patient impact – while also building the foundation for products to scale.

We recently cleared our tenth IND in collaboration with our biotech and academic partners – a milestone that reflects the model we set out to build at CTMC. The real highlight is not just clearing ten INDs. It is seeing those INDs become first-in-human studies where patients with few or no remaining options are already achieving complete responses.

That is what makes cell therapy so powerful. In many areas of drug development, early clinical studies are primarily about safety, dose, and pharmacology. Those are still critical in cell therapy, but in several of the programs we have supported, the first patients treated have also experienced meaningful clinical responses. For patients with advanced cancers and limited options, that is incredibly motivating.

At the same time, our goal is not to create one-off academic successes that help a small number of patients. The goal is to build a development and manufacturing foundation that allows the most promising therapies to keep advancing – into later-stage trials, broader access, and ultimately commercialization. Speed matters, but speed only matters if it is paired with rigor, reproducibility, and scalability.

That is why I founded CTMC. We are not simply trying to move programs quickly into the clinic. We are trying to help build products that can survive the full development journey: from translational insight, to IND, to first-in-human testing, to manufacturing scale-up, to regulatory maturity, to commercial readiness.

One example of that model is Obsidian’s OBX-115 program. CTMC and MD Anderson supported the initial IND and first-in-human clinical translation of this engineered TIL therapy. Since that initial clinical translation, Obsidian has raised substantial capital to advance OBX-115, including its Series B, its oversubscribed Series C, and its recent PIPE and reverse merger. Taken together, Obsidian has raised more than $600 million since beginning its collaboration with us to support the continued development and scaling of the program.

That is the kind of arc we wanted CTMC to enable: take strong science into humans quickly, learn from the earliest patients, and build a product and process foundation that can attract capital, scale responsibly, and continue moving toward patients at much larger scale.

For me, the professional highlight is seeing that original CTMC experiment work. We set out to test whether a different development model could help advanced medicines move faster without sacrificing rigor – by integrating clinical insight, industrial-grade manufacturing, analytics, regulatory strategy, and biotech partnership from the beginning. The answer increasingly appears to be yes. We are pushing the science forward, generating the safety and clinical data required in early development, and in some cases already seeing complete responses in patients who urgently need new options. But just as importantly, we are doing it in a way that is intended to support the long-term scalability of the therapies that show those early signs of promise.

That is the CTMC model in action: speed, rigor, scalability, and patient impact together.

What is the biggest bottleneck slowing progress in the cell and gene field right now – and how do we fix it?

The biggest bottleneck is commercial scalability.

The field has no shortage of innovation. We have extraordinary biology, creative engineering, new targets, new constructs, new editing tools, and new ways of thinking about immune cells as medicines. The problem is that too many of these innovations still do not have a clear path to becoming scalable commercial products.

That is especially true in cell therapy. A therapy can show remarkable clinical activity and still face enormous challenges if it is too slow, too expensive, too variable, too labor-intensive, or too difficult to deliver reliably across many treatment centers. In cell therapy, manufacturing is not a back-office function. It is part of the therapeutic hypothesis. If the process cannot scale, the product cannot fully realize its clinical potential.

The field cannot solve this just by building more cleanrooms and hiring more operators. That may increase capacity, but it does not fundamentally change the scalability equation. To make cell therapy broadly accessible, we need to move from artisanal manufacturing toward industrialized manufacturing – closed systems, robotics, automation, integrated analytics, digital batch records, and fit-for-purpose process control.

The monoclonal antibody field offers a useful analogy. Antibodies did not become one of the dominant therapeutic classes simply because the biology was compelling. The field developed powerful discovery tools, learned to engineer antibodies for manufacturability, and built industrial manufacturing systems that could deliver products consistently at global scale. Cell therapy needs to go through a similar maturation, but faster.

For cell therapy, that means designing both the product and the process for scalability from the beginning. It means asking not only, “Can this work in a patient?” but also, “Can we make it consistently? Can we reduce labor and variability? Can robotics replace manual steps? Can we automate the highest-risk parts of the process? Can we measure what matters in real time? Can we shorten vein-to-vein time? Can we support hundreds or thousands of patients without recreating an artisanal process at every site?”

At CTMC, this is central to how we think about the field. The goal is not just to manufacture cell therapies. The goal is to help build the next generation of scalable cell therapy manufacturing: where product design, process development, analytics, automation, robotics, regulatory strategy, and clinical delivery are developed together.

So the bottleneck is scalability, but not in the narrow sense of bigger facilities. It is scalability of the entire therapeutic model: the product, the process, the labor model, the analytics, the regulatory strategy, the clinical workflow, and the economics.

That is what the field needs to solve next. 

Where is the cell and gene industry making real progress that isn’t getting enough attention?

One area where the field has made real progress – and I do not think it gets enough attention – is that cell therapies are becoming part of mainstream oncology practice.

A few years ago, CAR-T therapy was still often viewed as an extraordinary intervention reserved for the sickest patients, usually after multiple prior lines of therapy. That is changing. In several indications, cell therapies have become standard of care, and they are moving into earlier lines of treatment where patients may be healthier, their immune cells may be more functional, and the potential for durable benefit may be even greater.

That shift matters enormously. The earlier we can use these therapies appropriately, the broader their impact can be.

We are also seeing progress in the care model around cell therapy. The field is becoming more sophisticated about how patients are monitored, how toxicities are managed, and how these therapies can be delivered in a more fit-for-purpose way. The FDA’s 2025 decision to eliminate REMS requirements for approved autologous CAR-T therapies was an important signal that the clinical community now has substantially more experience managing these products.

Similarly, post-infusion requirements are evolving. For CARVYKTI, current prescribing information instructs patients to remain near the treatment center for at least two weeks and avoid driving for at least two weeks after infusion. Earlier materials referenced substantially longer restrictions, so this is a meaningful reduction in patient burden.

We are also seeing more outpatient administration models. CARVYKTI, for example, has been studied and described in outpatient lymphodepletion and outpatient infusion settings with structured monitoring, and expert groups have discussed models where a substantial fraction of patients can be treated outpatient.

Taken together, these changes lower the practical barriers to cell therapy. Manufacturing innovation is critical, but access is not only a manufacturing problem. It is also a clinical operations problem, a regulatory problem, a monitoring problem, and a patient-burden problem. The field is making real progress on all of those fronts.

The broader story is that cell therapy is moving from being a heroic, highly specialized intervention toward becoming a more routine part of cancer care. That transition may be less flashy than a new construct or target, but it is one of the most important developments in the field. 

What’s one widely held belief in your field that you disagree with – and why?

One belief I disagree with is that in vivo CAR-T will simply replace autologous ex vivo cell therapy.

I understand why the field is excited. In vivo CAR-T is an elegant idea: deliver the genetic payload directly into the patient and turn their own immune cells into therapeutic cells inside the body. If it works safely, reproducibly, and at scale, it could remove some of the most challenging parts of today’s cell therapy model: leukapheresis, individualized manufacturing, chain of identity, complex logistics, and long vein-to-vein timelines.

But I think it is too simplistic to assume that this will replace autologous cell therapy.

Ex vivo manufacturing gives us something very powerful: control. We can select, activate, engineer, expand, characterize, release, and cryopreserve the product before it goes back to the patient. We can measure identity, potency, phenotype, vector copy number, viability, sterility, and other critical quality attributes. We can intervene in the process. We can learn from the process. And we can link product attributes to clinical outcomes over time.

In vivo approaches will need to solve a different set of hard problems: targeting the right cells in the right tissues, controlling the level and duration of expression, managing biodistribution, avoiding off-target transduction, achieving consistency across very different patients, and understanding how to regulate and reverse the therapy if something goes wrong.

So I do not think the future is “in vivo replaces autologous.” I think the future is more nuanced. Some diseases, targets, and clinical settings may be ideal for in vivo engineering. Others may continue to benefit from the control, characterization, and flexibility of ex vivo manufacturing. The winning approach will depend on the biology, the patient population, the safety requirements, and the product attributes needed for clinical benefit.

The broader lesson is that we should not confuse convenience with therapeutic superiority. The goal is not simply to make cell therapy easier to administer. The goal is to make therapies that are safe, potent, durable, controllable, scalable, and accessible. In vivo CAR-T may become an important part of that future, but it will not eliminate the need for highly engineered, well-characterized autologous cell therapies.

What’s the smartest way to navigate today’s funding and commercial landscape in cell and gene therapy?

The smartest strategy is to be capital-efficient, clinically grounded, and brutally honest about what creates value.

The funding environment has forced the field to mature. That is painful, but it is not all bad. Investors are no longer rewarding cell therapy companies simply for having an exciting construct or a plausible platform. They want to see a credible path to clinical proof of concept, manufacturability, regulatory execution, and commercial differentiation.

For emerging companies, that means they should not try to recreate every capability internally. They should preserve capital for the experiments that create value: demonstrating that the therapy can help patients, identifying the right dose and product attributes, and building the evidence package needed for the next stage of development.

That is one reason CTMC was created. Our role is to help partners de-risk development by giving them access to integrated CMC, manufacturing, analytics, quality, regulatory, and clinical translation capabilities without forcing them to build a full infrastructure prematurely.

In today’s environment, the winners will be the companies that can generate high-quality clinical and CMC evidence quickly, make disciplined decisions, and avoid spending years and enormous amounts of capital on infrastructure before they know whether the product works.

If you could introduce one policy change to accelerate progress in your field, what would it be – and why?

I would create a more explicit regulatory framework for iterative manufacturing improvement in cell therapy.

Everyone agrees that cell therapy manufacturing needs to become faster, more automated, more consistent, and less expensive. But the regulatory risk of changing a process during development – and especially later in development – can be substantial. That creates a paradox: the field needs innovation in manufacturing, but companies are often afraid to introduce improvements because comparability can be difficult, expensive, and uncertain.

I would like to see clearer, more predictable pathways for platform-based manufacturing changes, especially when companies can demonstrate strong analytical understanding and product comparability. This does not mean lowering standards. Patients need safe, potent, well-controlled products. But we need regulatory mechanisms that encourage continuous improvement rather than locking early, imperfect processes in place.

A better framework would accelerate the adoption of automation, improved analytics, closed processing, shorter culture systems, and lower-cost manufacturing. That would help patients because it would make therapies faster to deliver, easier to scale, and more economically sustainable.

What will look completely different about drug development in 10 years?

In 10 years, I think drug development for advanced medicines will be much more integrated, much more data-rich, and much faster-cycle.

Today, the industry often treats discovery, process development, clinical translation, manufacturing, and commercialization as sequential steps. For complex biologics and especially cell therapies, that model is too slow. The future will be more iterative. We will design the product and the process together. We will use richer analytics to understand the relationship between starting material, manufacturing conditions, product attributes, and clinical outcomes. We will use digital systems and AI not as buzzwords, but as tools to connect development decisions across the full lifecycle.

The analogy I often think about is monoclonal antibodies. Early antibodies were powerful but difficult to develop and manufacture. Over time, the field learned to engineer not just for binding or potency, but also for manufacturability, stability, expression, delivery, and commercial scalability. Cell therapy will go through a similar evolution. We will engineer the product for the patient, but also for the process.

That shift will change everything. The best cell therapies of the future will not just have better biology; they will be designed from the beginning to be made, measured, delivered, and scaled.

Do you think your younger self would be surprised to see the career you’ve had?

Yes and no. My younger self would probably be surprised by the path. I started as a scientist and spent much of my career in therapeutics development, moving products from research toward the clinic and commercialization. I do not think I would have predicted that I would one day be building a company focused on manufacturing as a central lever for therapeutic impact.

But the motivation would feel very familiar. I have always been drawn to the point where science becomes medicine. Cell therapy is one of the most compelling examples of that. You can take living cells, engineer or expand them, return them to a patient, and in some cases produce a response that changes the course of that person’s life.

What would not surprise my younger self is that I ended up working on hard problems with practical consequences. That has always been the exciting place for me: where biology, engineering, clinical need, and execution all have to come together. 

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