September 2025 marked a clear transition from pioneering science to industrial-scale medicine. The month’s developments underscored a field focused on building the foundational pillars for future growth, with a strong emphasis on policy, manufacturing, and next-generation technology. Key events included high-level policy discussions aimed at solving critical access and production bottlenecks, strategic regulatory designations for novel platforms, and significant progress in automating manufacturing. In the lab, preclinical data highlighted new approaches for tackling solid tumors and refining the precision of gene editing. This report will dissect these developments, drawing on data from key global conferences and publications to provide an analysis of the events that shaped cell therapy in September 2025.
Regulatory and Policy Frontiers: Building the Framework for Access
September’s most significant developments were not just scientific but systemic, focused on creating the regulatory and commercial infrastructure needed to deliver advanced therapies to patients1,2.
The Regulatory Pulse: Policy Summits and FDA Designations
The rapid scientific progress in cell therapy is occurring in parallel with an intense effort to build the regulatory and commercial infrastructure needed to deliver these treatments to patients. This dual focus was evident in September 2025. The American Society of Gene & Cell Therapy (ASGCT) Policy Summit, held September 25-26 in Washington, D.C., brought together leaders from government, industry, and patient advocacy to address the critical non-scientific barriers to access.1 The agenda featured high-level discussions on regulatory harmonization, addressing chemistry, manufacturing, and controls (CMC) bottlenecks, and exploring innovative payment systems, including the Centers for Medicare and Medicaid Innovation (CMMI) CGT Access Model.
Simultaneously, the U.S. Food and Drug Administration (FDA) continued to signal its support for promising therapies through the use of expedited pathways. In September, the agency granted Fast Track designation to two notable cell therapies 3:
- CER-1236: An investigational T-cell therapy from CERo Therapeutics for the treatment of acute myeloid leukemia (AML), granted on September 5.3
- UB-VV111: Umoja Biopharma’s investigational off-the-shelf CAR T-cell therapy for relapsed/refractory B-cell malignancies, granted on September 30. This designation was particularly significant as UB-VV111 is an in vivo therapy, designed to generate CAR-T cells directly within the patient’s body, thus representing a key milestone for this next-generation platform.3
These parallel activities highlight the emergence of a sophisticated, two-track system for cell therapy regulation and policy. The first track is focused on the “here and now,” creating the commercial and logistical frameworks—such as payment models and CMC standards—for therapies nearing approval. The second track is focused on the “what’s next,” using expedited pathways like Fast Track to nurture and accelerate fundamentally new platforms, such as in vivo therapies, that promise to solve the very bottlenecks being debated in the first track.
Table 1: Key Regulatory Developments in Cell Therapy (September 2025)
| Therapy Name/Program | Company/Org | Modality | Indication(s) | Key Event/Finding | Significance |
| UB-VV111 | Umoja Biopharma | In Vivo CAR-T (CD19) | B-Cell Malignancies (LBCL, CLL) | FDA Fast Track Designation granted on Sep. 30. 3 | Key regulatory validation for the emerging field of in vivo cell engineering, which aims to eliminate complex ex vivo manufacturing. |
| CER-1236 | CERo Therapeutics | T-Cell Therapy | Acute Myeloid Leukemia (AML) | FDA Fast Track Designation granted on Sep. 5. 3 | Highlights urgent unmet need in AML and regulatory support for novel cellular immunotherapies in this space. |
The Engine of Innovation: Preclinical and Technological Advancements
Moving from policy to the laboratory, this section explores the foundational scientific breakthroughs from September 2025 that will fuel the next generation of cell therapies. Innovation in core technologies promises therapies that are more precise, potent, and capable of tackling difficult targets like solid tumors.
The CRISPR Toolkit Expands and Refines
September’s publications showcased significant enhancements to the CRISPR gene editing toolbox, moving the technology toward greater precision and control. These advancements represent a shift to “CRISPR 2.0,” where the goal is not just to cut DNA but to subtly and safely modulate gene function.
Key developments include:
- Multiplex Base Editing: On September 29, researchers in France and Italy announced the development of a strategy that uses base editing- a form of CRISPR that changes a single DNA letter without a double-strand break- to simultaneously target two distinct enhancer regions of the BCL11A gene. This approach is being developed as a potentially safer and more effective treatment for sickle cell disease.4
- Systematic Cell Engineering: In a move from bespoke engineering to data-driven design, researchers are now using genome-wide CRISPR screens to systematically identify genetic modifications that improve cell therapies. In a report from September 26, one such screen identified that knocking out genes like RHOG and CDKN1B significantly boosts CAR T-cell performance, persistence, and tumor-killing ability, providing a validated toolkit for creating superior therapeutic cells.5
New Approaches for Solid Tumors and In Vivo Delivery
A major focus of innovation is on developing therapies for solid tumors and creating new delivery technologies that can circumvent the complex manufacturing paradigm.
- Reprogramming the Tumor Microenvironment: On September 20, Ernexa Therapeutics presented promising preclinical data at the AACR Special Conference in Cancer Research for its lead program, ERNA-101. This therapy consists of engineered iPSC-derived mesenchymal stem cells (iMSCs) designed to migrate to tumors and remodel the immune microenvironment from “cold” to “hot” in high-grade serous ovarian cancer, thereby restoring immune activity.6 This represents a novel strategy for overcoming the immunosuppressive barriers that have limited cell therapy efficacy in solid tumors.
- In Vivo Cell Engineering: The ultimate goal for many in the field is to perform cell engineering directly inside the patient’s body, eliminating the costly ex vivo manufacturing step entirely. A significant milestone for this approach occurred on September 30, when Umoja Biopharma received FDA Fast Track designation for its in vivo CAR T-cell therapy, UB-VV111, based on encouraging data from its ongoing Phase 1 trial.3
Manufacturing at Scale: The Push for Automation and Accessibility
This section addresses what is arguably the single greatest challenge facing the cell therapy industry: manufacturing. The transition from manual, lab-scale processes to robust, scalable, and cost-effective commercial production is paramount for the field to realize its full potential. September 2025 saw key developments in automation and a continued high-level focus on policy and strategy to overcome these hurdles.
The Rise of the Robotic Cleanroom: Automating Cell Production
A critical industry trend is the combination of best-in-class, GMP-ready, closed-system instruments with overarching robotic automation to create fully autonomous, end-to-end manufacturing workflows. Manual processing is a major source of cost, variability, and contamination risk.7
A prime example of this trend was the announcement on September 30 that Multiply Labs was expanding its collaboration with Thermo Fisher Scientific. The partnership will focus on integrating Thermo’s closed, automated Gibco™ CTS™ DynaCellect™ Magnetic Separation System with Multiply Labs’ robotic platforms.8 The goal is to create a seamless, automated workflow that minimizes manual intervention and human error, ultimately reducing costs and getting treatments to patients faster.9 The agenda for the Cell Therapy Bioprocessing conference in Boston, held from September 16-18, further reflected this industry-wide push, with multiple sessions dedicated to “Automated Manufacturing and Testing Solutions” and strategies for scaling up production.9
Decentralizing Production and Addressing Systemic Bottlenecks
The industry recognizes that even a perfect automated manufacturing platform is useless without a reliable supply of starting materials and a viable model for deploying the therapy to patients. High-level discussions at forums like the ASGCT Policy Summit (September 25-26)1 and the Cell and Gene Therapy Exchange (mid-September) focused on these systemic challenges.2
Key topics of discussion included addressing critical supply chain vulnerabilities for essential raw materials like viral vectors, where global demand often outstrips capacity.7 The current centralized manufacturing model, particularly for autologous therapies, creates significant logistical challenges. As a result, decentralized models, where therapies are produced in automated systems at or near the hospital (point-of-care), are being seriously explored as a potential long-term solution to improve patient access and reduce complexity.7
Conclusion: Synthesis and Forward Outlook
September 2025 was a month of foundational progress for the cell therapy field. Rather than being defined by a single clinical breakthrough, the period was characterized by the steady, crucial work of building a sustainable industry. The high-level discussions at the ASGCT Policy Summit and the FDA’s strategic use of Fast Track designations for next-generation platforms like in vivo therapies demonstrate a concerted effort to clear the path for future treatments.1
In parallel, the push toward fully automated, end-to-end manufacturing, highlighted by new industry collaborations and dedicated conference sessions, signals a determined effort to solve the production bottlenecks that limit patient access.7 Preclinical advancements in CRISPR technology and novel iPSC-based strategies for solid tumors continue to feed the innovation pipeline, promising more powerful and precise therapies to come.6
Looking ahead, the groundwork laid in September will be critical. The policies discussed will shape the regulatory and reimbursement landscape, while the manufacturing solutions being developed will determine the scalability and commercial viability of the next wave of cell therapies. The field is methodically constructing the industrial and regulatory engine required to turn cutting-edge science into a pillar of modern medicine.
References
- 2025 Policy Summit. American Society of Gene & Cell Therapy
- 4th UK/Swiss Cell and Gene Therapy Event
- Oncology Drugs Granted FDA Designations in September 2025. Oncology News Central
- CMN Weekly (10 October 2025) – Your Weekly CRISPR Medicine News. CRISPR Medicine News
- CMN Weekly (26 September 2025) – Your Weekly CRISPR Medicine News. CRISPR Medicine News
- Ernexa Therapeutics to Present New Data in Oral Presentation at AACR Special Conference in Cancer Research. BioSpace.
- 2025 cell and gene challenges: Scalability, supply chain, and manufacturing. Cell & Gene Therapy Review
- Multiply Labs Expands Collaboration with Thermo Fisher Scientific to Automate Cell Therapy Manufacturing. Multiply Labs
- Agenda – Wednesday, September 17th, 2025 – Main Conference Day One. Informa Connect