NEW YORK, Nov. 19, 2025 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today publishes an article in Nature Communications establishing circular single-stranded DNA (CssDNA) as a highly efficient non-viral DNA donor template, for gene insertion in hematopoietic stem and progenitor cells (HSPCs). Gene editing of HSPCs offers the potential fo ...

Core Insights - Cellectis reported promising clinical data for its product candidates lasme-cel (UCART22) and eti-cel (UCART20x22), indicating their potential to improve outcomes in relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) and non-Hodgkin lymphoma (r/r NHL) respectively [3][4][11] Financial Results - As of September 30, 2025, Cellectis had $225 million in cash, cash equivalents, and fixed-term deposits, which is projected to fund operations into the second half of 2027 [4][26] - Consolidated revenues and other income for the nine-month period ended September 30, 2025, were $67.4 million, up from $34.1 million in the same period in 2024, primarily driven by activities related to the AstraZeneca collaboration [29] - Consolidated net loss attributable to shareholders for the nine-month period ended September 30, 2025, was $41.3 million, compared to a loss of $42.7 million in the same period in 2024 [34] Clinical Development - The Phase 1 BALLI-01 study of lasme-cel showed an overall response rate (ORR) of 68% with the Cellectis-manufactured product, and 100% in the target Phase 2 population [6][7] - The first interim analysis for the pivotal Phase 2 BALLI-01 trial is expected in Q4 2026, with a Biologics License Application (BLA) anticipated in 2028 [8][9] - For eti-cel, preliminary data indicated an ORR of 86% and a complete response (CR) rate of 57% in the ongoing study [11] Pipeline Highlights - Lasme-cel is positioned as a potentially transformative therapy for r/r B-ALL, with a projected peak gross sales potential of approximately $700 million by 2035, which could increase to $1.3 billion with label expansions [9] - The company plans to present further updates on eti-cel at the ASH 2025 Annual Meeting [10][14] Innovation and Collaboration - Cellectis is advancing its gene editing technology, including the use of circular single-stranded DNA (CssDNA) as a non-viral template for gene therapy, which has shown high gene insertion frequency in hematopoietic stem and progenitor cells [16][22] - A strategic collaboration with AstraZeneca aims to develop up to 10 novel cell and gene therapy products targeting high unmet medical needs [23]

Core Insights - Cellectis announced the acceptance of two abstracts for poster presentations at the ASH 2025 annual meeting, focusing on its innovative gene-editing platform for cell and gene therapies [1] Group 1: Development Updates on eti-cel - The first poster presents a development update on eti-cel (UCART20x22), an allogeneic dual CAR-T therapy targeting CD20 and CD22, currently in Phase 1 of the NATHALI-01 clinical trial for relapsed/refractory non-Hodgkin lymphoma (r/r NHL) [2] - Preliminary results for eti-cel show an overall response rate (ORR) of 86% and a complete response (CR) rate of 57% among 7 patients, indicating a strong potential to improve outcomes for r/r NHL patients [3] - The addition of low-dose interleukin-2 (IL-2) is expected to enhance the anti-tumor activity of eti-cel, with full Phase 1 dataset presentation anticipated in 2026 [4][6] Group 2: Correlation with alemtuzumab in lasme-cel - The second poster discusses the correlation between alemtuzumab exposure and response in patients treated with lasme-cel (UCART22) during Phase 1 of the BALLI-01 trial for relapsed/refractory acute lymphoblastic leukemia (ALL) [5] - Data indicates a threshold level of alemtuzumab exposure that increases the likelihood of achieving a complete response without raising toxicity levels [6][7] Group 3: Company Overview - Cellectis is a clinical-stage biotechnology company focused on developing life-saving cell and gene therapies using a pioneering gene-editing platform, with a unique allogeneic approach for CAR T immunotherapies [8] - The company maintains in-house manufacturing capabilities, positioning itself as one of the few end-to-end gene editing companies that control the entire cell and gene therapy value chain [8]

Core Points - Cellectis, a clinical-stage biotechnology company, will report its financial results for Q3 2025 on November 7, 2025, after the US market closes [1] - The company specializes in gene-editing technology to develop cell and gene therapies, particularly focusing on CAR T immunotherapies for oncology [2][3] Company Overview - Cellectis employs an allogeneic approach for CAR T immunotherapies, aiming to create off-the-shelf, ready-to-use gene-edited CAR T-cells for cancer treatment [2] - The company has in-house manufacturing capabilities, making it one of the few end-to-end gene editing companies that manage the entire cell and gene therapy value chain [2] - Cellectis is headquartered in Paris, France, with additional locations in New York and Raleigh, NC, and is listed on both Nasdaq (ticker: CLLS) and Euronext Growth (ticker: ALCLS) [2]

Market Overview - U.S. stock futures are higher, with Dow futures gaining approximately 0.2% on Monday [1] Company-Specific Movements - Olema Pharmaceuticals Inc (NASDAQ:OLMA) shares fell 18.3% to $7.64 in pre-market trading following the announcement of new data from the Phase 1b/2 trial of palazestrant plus ribociclib in ER+/HER2- metastatic breast cancer at ESMO 2025 [1] - Concord Medical Services Hldgs Ltd (NYSE:CCM) experienced a decline of 24.4% to $4.15 in pre-market trading after a 6% drop on Friday [3] - Addex Therapeutics Ltd – ADR (NASDAQ:ADXN) dipped 15.6% to $9.23 in pre-market trading after a significant gain of 28% on Friday [3] - Exelixis Inc (NASDAQ:EXEL) shares fell 8.3% to $36.00 after announcing detailed results from the Phase 3 STELLAR-303 pivotal trial evaluating zanzalintinib in combination with an immune checkpoint inhibitor in metastatic colorectal cancer, presented at ESMO 2025 and published in The Lancet [3] - Cellectis SA (NASDAQ:CLLS) shares declined 7.3% to $3.69 in pre-market trading [3] - Alumis Inc (NASDAQ:ALMS) saw a decline of 6.7% to $4.19 in pre-market trading [3] - Nanobiotix SA – ADR (NASDAQ:NBTX) fell 6.3% to $23.98 in pre-market trading after a 4% decline on Friday [3] - Genmab A/S – ADR (NASDAQ:GMAB) declined 4.1% to $31.81 in pre-market trading, with Rinatabart Sesutecan achieving a 50% objective response rate and two complete responses in advanced endometrial cancer [3] - Taysha Gene Therapies Inc (NASDAQ:TSHA) fell 4% to $4.53 in pre-market trading [3]

Core Insights - Cellectis has initiated the pivotal Phase 2 BALLI-01 study for lasme-cel (UCART22) targeting relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) patients who are ineligible for transplant, with promising clinical data from the Phase 1 study [2][3][13] Efficacy - The overall response rate (ORR) for lasme-cel was reported at 68% in Process 2 (n=22), 83% at the recommended Phase 2 dose (RP2D, n=12), and 100% in the target Phase 2 population (n=9) [1][10] - In the target Phase 2 population, the complete remission (CR) and complete remission with incomplete hematologic recovery (CRi) rate was 56%, with approximately 80% of patients achieving minimal residual disease-negative (MRD-negative) status [1][15] Safety - Lasme-cel was generally well-tolerated in the Phase 1 study (n=40), with manageable adverse events including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) [8][14] - Dose-limiting toxicities (DLTs) were uncommon, with only one case reported at Dose Level 3 [14] Durability - Patients who achieved MRD-negative CR/CRi had a median overall survival (OS) of 14.8 months [1] - Among patients who proceeded to hematopoietic stem cell transplantation (HSCT) after lasme-cel therapy, there was a trend towards longer overall survival compared to those who did not undergo transplant [12] Commercial Opportunity - Cellectis estimates potential peak gross sales of lasme-cel could reach approximately $700 million across the U.S., EU4, and UK by 2035, with an addressable market of about 1,900 patients annually [2][20] - The projected pricing for lasme-cel is approximately $365,000 in the EU4 and UK, and $515,000 in the U.S., with a potential compound annual growth rate (CAGR) of ~5% [19][20] Strategic Positioning - Lasme-cel is positioned as a potentially game-changing therapy for r/r B-ALL, offering an alternative to CD19-targeted therapies with off-the-shelf availability and deep MRD-responses [4][18] - The company aims to address the significant unmet need for heavily pretreated patients, with expectations from key opinion leaders indicating a preference for lasme-cel among eligible patients [18]

Core Insights - Cellectis has initiated the pivotal Phase 2 BALLI-01 study for lasme-cel (UCART22) targeting relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) patients who are ineligible for transplant, with promising clinical data from the Phase 1 study [2][3][13] - The company estimates potential peak gross sales of lasme-cel could reach approximately $700 million across the U.S., EU4, and UK by 2035, with a significant addressable market for heavily pretreated patients [2][19] Clinical Efficacy - In the Phase 1 study, lasme-cel demonstrated an overall response rate (ORR) of 68% with Process 2, increasing to 83% at the recommended Phase 2 dose (RP2D), and achieving 100% in the target Phase 2 population [1][10] - The complete remission (CR) and complete remission with incomplete hematologic recovery (CRi) rate was 56% in the target Phase 2 population, with approximately 80% of these patients achieving minimal residual disease (MRD)-negative status [1][15] - Among patients previously treated with all three targeted therapies, 73% responded to lasme-cel, with 64% achieving MRD-negative status [1][11] Safety Profile - Lasme-cel was generally well-tolerated in the Phase 1 study, with manageable adverse events including cytokine release syndrome (CRS) occurring in 2.5% of patients and immune effector cell-associated neurotoxicity syndrome (ICANS) in 5% [8][14] - The study reported only one case of grade 2 immune effector cell-associated hemophagocytic syndrome (IEC-HS), which resolved [1][8] Commercial Opportunity - Cellectis estimates that lasme-cel could address approximately 1,900 patients annually in the 3L+ B-ALL category by 2035, with a significant portion of the projected incident 1L treated B-ALL population expected to progress to later lines of therapy [17][18] - The company anticipates a pricing strategy that could position lasme-cel at approximately $365,000 in the EU4 and UK, and $515,000 in the U.S. by 2025, with potential for significant revenue growth if approved for commercialization [19][19] Strategic Developments - Cellectis is advancing its pivotal Phase 2 program for lasme-cel and plans to submit a Biologics License Application (BLA) in 2028 following successful meetings with the FDA and EMA [13][19] - The company is also engaged in a strategic partnership with AstraZeneca to develop up to 10 novel cell and gene therapy products, leveraging Cellectis' gene editing expertise [22]

Summary of Conference Call Notes Company and Industry Overview - The conference call primarily discusses a biotechnology company focused on developing allogeneic T cell therapies for patients with acute lymphoblastic leukemia (ALL) and other hematological malignancies. The company is transitioning from phase one to phase two of its clinical trials. Key Points and Arguments Patient Demographics and Treatment Landscape - Approximately 10,000 patients in the US, EU4, and the UK are treated annually for the relevant conditions, primarily with chemotherapy as the first line of treatment [1] - The relapse rate for CD19-directed therapies is around 50%, indicating a significant need for improved treatment options [2] - The patient population is heavily pretreated, with a median of four prior therapies, and many have high disease burden with over 60% bone marrow blast count [31] T Cell Therapy Development - The company emphasizes the importance of using high-quality, less exhausted T cells derived from healthy bone marrow, which can provide consistent treatment outcomes across patients [3] - The manufacturing process for allogeneic T cells is highlighted as critical, with the company having integrated its manufacturing capabilities to ensure quality and scalability [6][7] - The company has established manufacturing plants in Europe and the US, ready for commercial production [7] Clinical Trial Results - The current phase one trial shows a complete response rate of 57% and a partial response rate of 86% among patients at the current dose level [16] - The recommended phase two dose is set at 5 million cells per kilogram, targeting patients aged 12 to 50 [34][55] - High rates of minimal residual disease (MRD) negativity were observed among patients achieving complete remission, indicating effective treatment [35] Safety Profile and Adverse Events - The safety profile of the therapy is manageable, with most adverse events being grade one or two, and only a small percentage experiencing severe events [47][48] - The incidence of serious adverse events related to the therapy is low, with only one case of grade two graft-versus-host disease reported [49] Regulatory Path and Future Plans - The company has received positive feedback from regulatory authorities regarding the unmet need for its therapies and has a clear path for registration [52][54] - Plans for a pivotal phase two trial are underway, with a focus on expanding the patient recruitment sites to 75 centers across North America and Europe [64] - The company anticipates submitting two Biologics License Applications (BLAs) by 2028 for its investigational products [67] Market Dynamics and Competitive Landscape - The discussion highlights the competitive landscape, noting that many patients have been exposed to multiple targeted therapies, which complicates treatment options [18][19] - The company aims to address the unmet needs of patients who are refractory to existing therapies, particularly in the context of CD19 and CD22 targeted treatments [72][86] Additional Important Insights - The importance of internalizing the manufacturing process is emphasized, as it allows for better control over product quality and consistency [27] - The call also discusses the potential impact of prior exposure to CD19 therapies on patient responses to CD22-targeted therapies, indicating a need for further investigation [85][90] - The company is committed to exploring the pediatric population, with plans to include patients as young as zero to twelve years in future studies [62][63] This summary encapsulates the critical aspects of the conference call, focusing on the company's strategic direction, clinical trial progress, and the broader context of the industry.

Core Insights - Cellectis is hosting an R&D Day to present full Phase 1 data and pivotal Phase 2 strategy for lasme-cel (UCART22) in relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) [1] Company Overview - Cellectis is a clinical-stage biotechnology company focused on developing life-saving cell and gene therapies using a pioneering gene-editing platform [2] - The company employs an allogeneic approach for CAR T immunotherapies, aiming to create off-the-shelf, ready-to-use gene-edited CAR T-cells for cancer treatment [2] - Cellectis has in-house manufacturing capabilities, making it one of the few end-to-end gene editing companies that control the entire cell and gene therapy value chain [2] - The company is headquartered in Paris, France, with additional locations in New York and Raleigh, NC, and is listed on both Nasdaq and Euronext Growth [2]

Core Viewpoint - Allogene Therapeutics announced that its licensor, Cellectis, is facing a lawsuit in the U.S. regarding patent infringement related to gene editing technology [2] Company Summary - Allogene Therapeutics is involved in the development of cancer drugs and is impacted by the legal issues surrounding its licensor, Cellectis [2] - The lawsuit could have implications for Allogene's operations and its relationship with Cellectis, which is crucial for its gene editing technology [2] Industry Summary - The gene editing technology sector is facing legal challenges that may affect companies involved in this field, highlighting the importance of intellectual property rights [2]