The Evolution Of Cell And Gene Therapy: Pioneering Breakthroughs For A New Era In Medicine
By Vishal Mehta, Founder & CEO, Ubuntu Research

The world of cell and gene therapy (CGT) stands at a crossroads, poised for a revolution that fills us with excitement for the future and promise for patients who are awaiting life-saving therapies. Having achieved remarkable success in hematology and recently in some oncology indications, we are now witnessing a bold expansion into areas like autoimmunity, senescence, and cardio-metabolic conditions, to name a few. The not-so-distant future promises a wave of rapid advancement and the introduction of new personalized cell and gene based therapies. With 23 CGT products having received FDA approval since 2017 and a significant increase of new therapies projected to enter the clinic each year, we are witnessing a remarkable evolution from cautious beginnings to substantial growth.
Dr. Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER) at the FDA, recently reaffirmed his commitment to this continued growth at ARM’s 2025 Cell & Gene State of the Industry Briefing, stating, “The CBER will continue to actively advance the science and regulatory framework for gene therapy as well as expedite the review process to help bring the promise of genetic therapies to the broadest number of people globally.” We are excited to see the promise Marks speaks of come to life, including via additional therapies as well as a future focused on moving from immunosuppression to immune regeneration (a future that could have a profound impact on geriatric patients, among others).
Lessons from the Past
As we approach this future, it is essential for the industry to support and fully embrace the innovations to come. By drawing on our previous successes deploying CGT as an innovative therapeutic in hematology and oncology, we can strategically position the industry to harness this momentum and better tackle forthcoming challenges. As we imagine the pivotal moments to come, it’s critical that we remember – and carry forward – learnings from our past.
Consider the story of Emily Whitehead, a six-year-old girl whose battle with acute lymphoblastic leukemia (ALL) led her family to seek cutting-edge solutions. Diagnosed with ALL at a young age, Emily faced a dire prognosis and extremely limited treatment options. When she became the first pediatric patient ever to receive autologous CD 19 CAR-T cell therapy, her story became a watershed moment in the industry. This therapy not only saved Emily’s life but also showcased the transformative power of CGT and its potential to safely and effectively offer long-term remission in cancers that had long been considered fatal.
Yet if Emily’s treatment had not been successful, the progress we've made in CGT could have been significantly stunted (or perhaps halted altogether), and we wouldn’t be anywhere near where we are today in realizing the benefits of this innovation. The uncertainty surrounding the feasibility of using modified T-cells to combat cancer might have discouraged further investment and research in this promising field.
A Future Focused on Safety and Patient Access
Emily's case galvanized excitement and support for CGT, leading to increased funding, research, and development across a range of diseases. Today it serves as a reminder of the profound impact that safe and effective therapies can have on patients’ lives; her story laid the foundation for the rigorous safety standards and collaborative frameworks that guide current CGT initiatives.
The safety of cell and gene therapies is paramount, particularly given the complex nature of treatments that harness and modify patients' immune cells. However, it's important as we look to future innovations to recognize that safety thresholds vary significantly across different diseases. For instance, in cancer, patients often accept higher risks and less pleasant side effects due to the life-threatening nature of their condition. In contrast, when dealing with chronic but manageable conditions such as lupus or type 1 diabetes, the tolerance for risk is much lower. This necessitates a more nuanced approach to safety planning from the outset. There is a tremendous amount of knowledge sitting within the industry, and we can effectively collaborate and apply that knowledge – for the benefit of our patients.
Drawing from experiences in hematology, where safety signals emerged during early trials, it’s critical for CGT companies to build robust safety monitoring protocols into their trials that can promptly detect and address emerging adverse events. Establishing specialized oversight committees to continuously evaluate patient responses during clinical trials ensures real-time adjustments to treatment protocols as needed. Additionally, by integrating advanced analytics and biomarker identification into clinical trial designs, researchers can anticipate potential safety concerns before they manifest.
The ultimate goal of an early-stage clinical development program is to construct a comprehensive safety framework that not only safeguards patients but also builds trust with stakeholders – including regulatory authorities, healthcare providers, and patients themselves. This proactive approach enables us to expand CGT into broader therapeutic areas and additional care settings while fostering a culture of safety as an integral part of the development process.
Equally important is the issue of patient access and health equity. Historically, these advanced therapies have been primarily accessible through large academic medical centers, creating geographical and socioeconomic barriers for many patients seeking treatment. To combat this challenge, strategies must be implemented to widen the reach of CGT, including incorporating community hospitals and outpatient facilities into the treatment network. Establishing satellite centers that are equipped to deliver CGT safely and effectively will facilitate local access, reducing travel burdens on patients and their families.
In essence, it’s important as we move forward to focus on not only safety and efficacy, but on therapy administration that comfortably fits into patients’ routines, rather than expecting patients to mold their lives to fit their care. Moreover, we must consider how effectively we can integrate long-term safety follow-up efforts, to ensure we stay informed of new safety signals and patterns, as part of a product’s life cycle management.
Additionally, recruiting diverse populations into clinical trials is essential to ensure that the effectiveness and safety of therapies is validated across different demographics, including various ethnicities, ages, and health backgrounds. This inclusivity not only addresses health disparity concerns but also enriches the overall understanding of treatment responses, enabling personalized care that considers the unique characteristics of various patient groups. By prioritizing access and diversity, we position CGT as a lifeline for a wider array of patients, ensuring that groundbreaking advancements translate into tangible health benefits for all.
A Paradigm Shift: Knowledge Sharing is the Way Forward
As we look to the future and remember the important lessons of the past, the thread that carries through is the value of sharing knowledge to ensure we arrive at our shared goal – necessary therapies for patients who, like Emily Whitehead, are counting on us. The complexities of bringing groundbreaking therapies to market will demand not only a continued focus on safety and patient access, but a clinical development model that is anchored on agile infrastructure. This infrastructure must enhance timeline efficiency, promote cross-functional collaboration, keep costs in check, and foster meaningful stakeholder engagement. This is a mindset shift, from in-house clinical development infrastructure and transactional CRO utilization to a “Knowledge Research Organization” (KRO) approach.
At its core, a KRO is about knowledge-sharing and collaboration. It means bringing in the right people, processes, and technology at the right moment in a cell therapy’s lifespan to optimize and enhance efficiency and productivity and streamline the clinical development processes. By aggregating digital tools and technological enablers, a KRO not only simplifies the development journey but also makes it easier for all stakeholders – patients, founders, investors, clinical vendors, and regulators – to navigate complex processes. This approach breaks down silos and allows innovation to thrive.
As companies consider how to bring the KRO mindset to life within their organizations, there are five key identifying attributes to strive for:
- Rich clinical research domain expertise: The goal is to design, embed, and accentuate efficiency in drug development programs.
- A purpose-driven mission: Above all else, the goal is to positively impact patient outcomes.
- Fiduciary and business stewardship: Competent capital allocation, single asset to portfolio maturation, and an enterprise risk management framework are essential.
- Delivery of unrealized value across stakeholder groups (patients, innovators, and investors): The formula for unprecedented value creation is simple: time and cost efficiency + enriched quality attributes demonstrable across all phases of clinical development.
- A continuous improvement mindset: CGT companies must emulate complementary behaviors and engender “intellectual humility.”
This focus on collaboration and knowledge sharing is particularly crucial in a dynamic environment where swift and informed decision-making is paramount. We’ve reached a pivotal moment – the future is no longer about whether these therapies work; it's about ensuring that their benefits reach the many who are in need.
In this moment, the stars are aligned for cell and gene therapy development, allowing us to harness our collective knowledge to provide faster access to life-saving therapies. The KRO approach to clinical development brings more than hope – it brings a clear path forward, with actionable steps that all CGT developers can implement today. We already possess the tools and strategies needed to guide the next wave of innovation.
The future begins today.
About the Author
Vishal Mehta founded Ubuntu Research Inc. with a focused mission to bring highly innovative therapies to patients in an expedient, sustainable and contemporaneous manner. With more than two decades of experience in therapeutics development across pharmaceutical, biotechnology, and contract research organizations (CRO), Vishal has developed deep insights in research and drug development strategies, clinical program execution frameworks, and curated business operations.
Vishal is honored to have played a key role in the development of many impactful patient treatments, examples of which include CAR T cell therapies BREYANZI®, ABECMA®; targeted therapies KISQALI®, AFINITOR®; Immunotherapy YERVOY®, as well as biosimilars and multiple life cycle management programs in oncology and other therapeutic areas.
Vishal earned his B.S. in Pharmaceutical Sciences from Univ. of Mumbai (India) and MS in Clinical Research & Administration from George Washington University (USA).