Cell Therapy's Next Chapter: Scaling Innovation and Navigating Emerging Challenges

NEW YORK, NY – November 22, 2025

The Promise of In Vivo Approaches

GenScript Biotech’s recent Global Industry Forum in London underscored a critical shift within the cell and gene therapy (CGT) landscape: a growing focus on in vivo CAR-T therapies. While existing CAR-T treatments demonstrate remarkable efficacy in blood cancers, they rely on a complex and expensive ex vivo process – removing a patient’s T cells, engineering them to target cancer, and then reinfusing them. In vivo approaches aim to simplify this process by directly delivering engineered T cells within the patient's body, potentially reducing manufacturing costs, streamlining treatment timelines, and broadening access.

“The biggest bottleneck right now isn’t necessarily the science, it’s the manufacturing,” explained one industry analyst attending the forum. “If in vivo can deliver on its promise, it could be a game-changer in terms of scalability.”

However, the path to realizing this potential is not without significant technical and regulatory hurdles. Ensuring precise targeting of cancer cells, minimizing off-target effects, and achieving sufficient T cell persistence remain key challenges. Companies like Umoja Biopharma and Interius BioTherapeutics are pioneering different in vivo approaches, utilizing lentiviral vectors and mRNA delivery systems to engineer T cells directly within the patient. The success of these early clinical trials will be crucial in determining the viability of this approach.

Investment and Scaling: Navigating a Complex Ecosystem

The forum also highlighted the significant investment flowing into the CGT sector, but also the inherent risks associated with commercializing these complex therapies. While venture capital funding for gene and oligonucleotide therapies reached $3 billion in 2022, scaling manufacturing capacity to meet projected demand remains a significant constraint.

“Everyone is excited about the potential of CGT, but turning that excitement into a sustainable business is a different story,” stated a biotech executive. “The infrastructure just isn’t there yet to support widespread commercialization.”

Catalent’s recent acquisition by Novo Holdings and Thermo Fisher Scientific’s expansion of their Advanced Therapies Collaboration Centers demonstrate a growing recognition of the need for specialized manufacturing facilities and expertise. However, the limited number of qualified personnel and the complexities of validating CGT manufacturing processes pose ongoing challenges. The success of companies like Legend Biotech, with its CARVYKTI treatment for multiple myeloma, provides a proof-of-concept for commercial viability. As of March 2025, over 6,000 patients have been treated with CARVYKTI globally, with that number exceeding 9,000 as of November, demonstrating growing demand and the potential for market expansion.

Beyond the Hype: The Need for Pragmatism

While the optimism surrounding CGT is palpable, speakers at the GenScript forum also cautioned against unrealistic expectations. Dr. Miguel Forte emphasized the importance of robust clinical validation, meticulous safety monitoring, and a pragmatic approach to addressing manufacturing complexities.

“We need to move beyond the hype and focus on delivering therapies that are not only effective but also accessible and affordable,” stated a researcher involved in mRNA vaccine development. “It's easy to get caught up in the science, but we can’t lose sight of the patients we're trying to help.”

Addressing accessibility remains a critical concern, as the high cost of CGT therapies – often exceeding $300,000 per treatment – limits access for many patients. Innovative financing models, such as outcomes-based pricing and risk-sharing agreements, are being explored to mitigate this challenge. Furthermore, the long-term durability of CGT therapies remains an area of active investigation. While early clinical trials have shown promising results, longer-term follow-up studies are needed to assess the durability of responses and identify potential late-onset toxicities.

The expanding portfolio of mRNA therapies also presents unique challenges. While the success of mRNA vaccines during the COVID-19 pandemic has demonstrated the platform’s potential, translating this success to other disease areas requires overcoming hurdles related to mRNA stability, delivery, and immunogenicity. The development of novel lipid nanoparticles and targeted delivery systems is crucial for maximizing the efficacy and safety of mRNA therapies.

Ultimately, realizing the full potential of cell and gene therapy requires a collaborative effort between researchers, clinicians, manufacturers, regulators, and payers. By fostering innovation, addressing manufacturing challenges, and prioritizing patient access, the CGT sector can deliver on its promise of revolutionizing the treatment of a wide range of diseases.