.png)
CGT Watch Newsletter: March'25 Edition
- Oncofocus Team
- Mar 20
- 6 min read
Dear Readers,
Welcome to our inaugural CGT Watch newsletter! We’re excited to bring you monthly highlights on novel targets, innovative therapeutic approaches, and emerging companies in the cell and gene therapy space.
Since we’re keen to keep things concise and focused, this issue will exclusively cover cell therapies. We’re using a 45-day window for these highlights; from our next issue onward, we’ll transition to a monthly update schedule.
Section 1: Key Monthly Updates
🎯 Regulatory Events
⭐ Bristol Myers Squibb’s Breyanzi EU Approval for Follicular Lymphoma (FL) (Ref)
The European commission granted approval to BMS’ Breyanzi (lisocabtagene maraleucel; an autologous, anti-CD19 CAR-T) for the treatment of adult patients with ≥3L FL.
👉 Why it matters: Breyanzi demonstrated higher ORR (97.1%) compared to the other two CAR-Ts that are approved for the treatment of FL in Europe. This positions Breyanzi as a leading CAR-T option for the patients with R/R FL.
⭐FTD Designation for Azer-cel (Ref)
The US FDA granted Fast Track Designation (FTD) to Imugene Limited’s azercabtagene zapreleucel (azer-cel; an allogeneic, anti-CD19 CAR-T) for the treatment of R/R DLBCL patients who progressed on prior therapies, including autologous CAR-T.
👉 Why it matters: Azer-cel is the only active allogeneic CAR-T with FTD for DLBLC patients who progressed on prior treatments, including autologous CAR-T. If azer-cel can reproduce the strong efficacy data in a large trial, it can become the first approved allogeneic CAR-T for these patients.
⭐ NMPA Grants BTD to CARsgen’s Satri-Cel (Ref)
China’s NMPA granted Breakthrough Therapy Designation (BTD) to CARsgen Therapeutics’s satricabtagene autoleucel (satri-cel; CT041; an autologous, anti-CLDN18.2 CAR-T) for the treatment of Claudin18.2-positive ≥3L G/GEJ cancer.
👉 Why it matters: Satri-cel achieved a statistically significant improvement in the primary endpoint of PFS in the Phase 2/NCT04581473 trial for this patient population. With this designation, satri-cel takes a significant step forward, and the company aims to submit an NDA to the NMPA in the H1’25.
🤝 Mergers & Acquisitions
⭐ BMS to Acquire 2Seventy Bio (Ref)
Bristol Myers Squibb is set to acquire 2seventy bio in an all-cash transaction valued at approximately $286 million. This acquisition gives BMS full control of Abecma (idecabtagene vicleucel)—an autologous, anti-BCMA CAR‑T therapy indicated for the treatment of adult patients with relapsed/refractory multiple myeloma (MM) who have undergone at least two prior lines of therapy.
👉 Why it matters: After factoring in 2Seventy Bio’s cash reserves, the net acquisition cost for BMS is around $102 million, making it a highly cost-effective investment. This deal allows BMS to fully leverage Abecma's potential without any profit-sharing obligations, potentially yielding returns within two years.
⭐ AstraZeneca's $1B Move in In Vivo Cell Therapy (Ref)
AstraZeneca concluded a $1 billion agreement to acquire EsoBiotec, a company specializing in in vivo cell therapies. AstraZeneca will be paying $425 million upfront with an additional $575 million in milestone-based payments. The EsoBiotec Engineered NanoBody Lentiviral (ENaBL) platform employs highly targeted lentiviruses to deliver genetic instructions directly to specific immune cells—such as T cells—programming them to recognize and destroy tumor cells.
👉 Why it matters: This acquisition will enhance AstraZeneca's cell therapy capabilities by leveraging the ENaBL platform to modify immune cells directly within the body, thereby reducing complexity and eliminating treatment delays.
🔬 Clinical Events
⭐ NexCAR19 Achieves 72.5% ORR (Ref)
The Phase 1/2 results of ImmunoACT’s NexCAR19 (talicabtagene autoleucel; an autologous, anti-CD19 CAR-T) were published, showcasing an ORR of 72.5% (37 out of 51 efficacy-evaluable patients; 36 with B-cell Lymphoma and 15 with B-ALL). These results led to the approval of NexCAR19 in India in Oct 2023.
👉 Why it matters: NexCAR19 is India's first homegrown approved CAR-T therapy, developed by ImmunoACT in collaboration with IIT Bombay and Tata Memorial Centre. With its impressive ORR and lower cost, NexCAR19 represents an effective and accessible treatment option for patients.
⭐ First Fast-Manufactured CEA CAR-T (Ref)
Long manufacturing time has been a bottleneck in providing quick access to approved CAR-Ts to eligible patients. The biopharma companies have been exploring various strategies to manufacture CAR-Ts rapidly. Chongqing Precision Biotech initiated a Phase 1 trial for the first fast-manufactured CAR-T targeting carcinoembryonic antigen (CEA) in CEA-positive advanced malignant solid tumors.
👉 Why it matters: Because CEA-positive cancers are especially aggressive, faster manufacturing can significantly improve timely and effective treatment.
⭐ AND-gated RevCAR-T Platform: Next-Level Precision (Ref)
The Helmholtz-Zentrum Dresden-Rossendorf (HZDR), in collaboration with the German Cancer Consortium (DKTK) and the DKFZ German Cancer Research Center , continues to advance its dual RevCAR-T platform. The dual RevCAR-T platform uniquely allows precise control over CAR-T cell activation through reversible targeting modules. Recently, they introduced a novel AND-gated antigen combination of PD-L1 & PSCA for their dual RevCAR-T which demonstrated significant tumor size reduction in Prostate Cancer preclinical models.
👉 Why it matters: This AND-gated RevCAR-T system aims to increase specificity and reduce off-target effects—a major step in making CAR-T therapies safer.
⭐ Tackling Off-Target Toxicity in AML (Ref)
In Acute Myeloid Leukemia (AML), off-target toxicity affecting hematopoietic stem cells remains a critical challenge. To mitigate this, the University of Pennsylvania has refined its bispecific split CAR-T (bissCAR) cells—comprising a nanobody targeting CD13 and an scFv targeting TIM-3 by developing novel cognate nanobody-directed bispecific anti-CD13/TIM3 nbiCARTs, demonstrating significant promise in preclinical AML models.
👉 Why it matters: Off-target toxicity is often a deal-breaker in AML CAR-T therapies. Positive early findings could pave the way for safer treatments.
⭐ EU’s Clinical Trial Authorisation for Non-viral Edited TCR-T (Ref)
Anocca received Clinical Trial Application (CTA) authorisation from the regulatory authorities in four European countries under the EU harmonised framework, for Phase 1/2/VIDAR-1 clinical trial evaluating ANOC-001 (KRAS G12V targeting non-viral gene edited TCR-T) for the treatment of PDAC.
👉 Why it matters: This CTA approval represents the first regulatory authorisation in Europe to study a non-viral gene-edited TCR-T therapy in clinical practice, representing a significant milestone in advancing innovative cell therapy technologies.
Shanghai Vitalgen Bio initiated a Phase 0 trial for its VGO-Cs01p, a CAR-NK therapy targeting CD7 in relapsed/refractory (R/R) T-cell Acute Lymphoblastic Leukemia.
Xinxiang Medical University is exploring CAR-NK92 therapy with a dual-targeted approach (PD-L1 and MICA/B) for lung cancer, a heterogenous cancer that demands multitargeted approach. Preclinical studies reported tumor regression and enhanced cytotoxic activity, suggesting dual antigen targeting could disrupt resilient tumor microenvironments.
👉 Why it matters: CAR-NK therapies are gaining momentum, offering new avenues where CAR-T might face challenges, especially in solid tumors and certain hematologic malignancies.
🛑 Discontinuations
⭐ Atara’s Discontinuation of Two Allogeneic CAR-Ts (Ref)
Atara Biotherapeutics discontinued all development activities for ATA3219 (an allogeneic, anti-CD19 EBV CAR-T) currently in Phase 1 for the treatment of B-NHL and ATA3431 (an allogeneic, anti-CD19/CD20 bispecific EBV CAR-T) in preclinical development for the treatment of B-cell malignancies.
👉 Why it matters: The decision could be attributed to regulatory challenges, including the FDA's clinical hold on ATA3219, as well as manufacturing compliance issues that have presented considerable difficulties for Atara.
Section 2: Cell Therapies Pipeline Landscape – An Overview
As an oncology-focused business consulting firm, we closely monitor emerging modalities in the oncology space. We weekly track over 4,500 preclinical and clinical CGT programs, covering 75 critical parameters—ranging from basic details and molecular characteristics to clinical outcomes.
CAR-T therapies, with over 3,700 programs (2,000+ for the treatment of Haematological Cancers), including 30 programs in the regulatory phase (refer exhibit 1a). dominate the CGT landscape. Globally, 13 unique CAR-T therapies have received approval for treating various hematological malignancies, while two more are currently under regulatory review. Notably, more than 2,000 programs are being developed for the treatment of Haematological Cancers.
Moving to TCR-T therapies, a different type of engineering observed on T-cells, TCR-T therapies are steadily gaining prominence, with around 380 programs in development (refer exhibit 1b). Notably, one TCR-T therapy has been approved for the treatment of synovial sarcoma, marking a significant milestone in the field.
Beyond CAR-T and TCR-T therapies, CAR-NK therapies are emerging as a promising alternative, leveraging the natural cytotoxic capabilities of NK cells for therapeutic applications. Currently, 330 CAR-NK programs are under development, with 217 still in the early stages, reflecting strong research interest in this modality.
Other cell therapies, such as CAR-macrophages and CAR-NKTs, are also being explored, but their numbers remain limited.
That’s all for our first edition of CGT Watch! We will update these charts / this space every month and hope our efforts help you keep a pulse on the ever-changing competitive landscape.
A Request
We’d love your feedback—what did you find most interesting? If you have any questions or insights, feel free to hit reply or reach out. We look forward to exploring more breakthroughs with you in our next newsletter.
Until next time,
CGT Watch Team
Comments