Cellectis
ENXTPA:ALCLS
€
3,09
€
+
€0,20 (6,92%)
3,09
€
+€0,20 (6,92%)
End-of-day quote: 04/08/2026
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Cellectis Company Info
EPS Growth 5Y
3,95%
Market Cap
€0,22 B
Long-Term Debt
€0,04 B
Annual earnings
N/A
Dividend
€0,00
Dividend Yield
0,00%
Founded
1999
Industry
Country
Website
ISIN Number
Website
Analyst Price Target
The Analyst Price Target shows the analysts’ low, high, and average target at a glance.
€6,50
110.36%
Last Update: 04/09/2026
Analysts: 1
Highest Price Target €6,50
Average Price Target €6,50
Lowest Price Target €6,50
In the last five quarters, Cellectis’s Price Target has risen from €10,60 to €10,60 - a 0,00% increase. One analysts predict that Cellectis’s share price will increase in the coming year, reaching €6,50. This would represent an increase of 110,36%.
Top growth stocks in the health care sector (5Y.)
What does Cellectis do?
Cellectis S.A. operates as a clinical stage biotechnological company.
The company is employing its core proprietary technologies to develop products based on gene-editing, with a portfolio of allogeneic Chimeric Antigen Receptor T-cells, or Universal Chimeric Antigen (UCART), product candidates in the field of immuno-oncology and gene-edited hematopoietic stem and progenitor cells, or hematopoietic stem and progenitor cells (HSPC), product candidates in other therapeutic indications.
The compa...
Cellectis S.A. operates as a clinical stage biotechnological company.
The company is employing its core proprietary technologies to develop products based on gene-editing, with a portfolio of allogeneic Chimeric Antigen Receptor T-cells, or Universal Chimeric Antigen (UCART), product candidates in the field of immuno-oncology and gene-edited hematopoietic stem and progenitor cells, or hematopoietic stem and progenitor cells (HSPC), product candidates in other therapeutic indications.
The company’s UCART product candidates, based on gene-edited T-cells that express chimeric antigen receptors, or CARs, seek to harness the power of the immune system to target and eradicate cancer cells. The company is designing next-generation immunotherapies that are based on gene-edited CAR T-cells. The company’s gene-editing technologies allow it to create allogeneic CAR T-cells, meaning they are derived from healthy donors rather than the patients themselves. The company’s gene-editing expertise also enables it to develop product candidates that feature certain safety and efficacy attributes, including control properties designed to prevent them from attacking healthy tissues, to enable them to tolerate standard oncology treatments, and to equip them to resist mechanisms that inhibit immune-system activity.
Together with its focus on immuno-oncology, the company is using, through its HEAL platform, the company’s gene-editing technologies to develop HSPC product candidates for genetic diseases. HEAL is a new gene-editing platform developed by Cellectis that leverages the power of TALEN technology to allow highly efficient gene inactivation, insertion and correction in hematopoietic stem and progenitor cells. The company initial focus is on developing allogeneic treatments.
Products Candidates
The company is directly developing product candidates internally and has also enters licensing relationships with AstraZeneca, Allogene, and Servier. The company’s agreements with AstraZeneca, Allogene, and Servier have validated its technology platform, its strong expertise in the allogeneic CAR T-cells field, in the field of gene editing and the strength of its intellectual property portfolio.
Under the AstraZeneca Joint Research and Collaboration Agreement (AZ JRCA), AstraZeneca (AZ) Ireland has an exclusive right to pursue the development and commercialization of products for a total of 10 selected candidate products.
Under the License Agreement dated March 8, 2019 between Allogene and the company (the Allogene License Agreement), Allogene has exclusive rights to pursue development and commercialization of products for a total of fifteen selected targets, including BCMA (targeted by the Allogene’s product candidates named ‘ALLO-715’ and ‘ALLO-605’), FLT3 (targeted by the Allogene’s product candidate named ‘ALLO-819’), CD70 (targeted by the Allogene’s product candidate named ‘ALLO-316’), DLL3 (targeted by Allogene’s product candidate named ‘ALLO-213’), and claudin 18.2 (targeted by Allogene’s product candidate named ‘ALLO-182’).
The company has a license agreement with Iovance Biotherapeutics, Inc. (Iovance). In addition, under the license, development, and commercialization agreement dated March 6, 2019, between Servier and it, and as amended on March 4, 2020 (as so amended, the Servier License Agreement), Servier has an exclusive worldwide license to develop and commercialize ALLO-501, in the field of anti-tumor adoptive immunotherapy (Allogene’s product candidate developed pursuant to a sublicense by Servier to Allogene).
The exclusive rights for the development and commercialization of UCART19 in the United States have been sublicensed by Servier to Allogene.
UCART19
In 2016, Servier commenced two Phase 1 clinical studies for the first version of UCART19, one in adult Acute Lymphoblastic Leukemia (ALL), referred to as the CALM study, and one in pediatric ALL, referred to as the PALL study. The company refers to the CALM and the PALL Studies, collectively as the UCART19 Studies.
In November 2020, the Phase 1 of the UCART19 Studies were completed. Servier has informed us that no additional patients are planned for enrollment.
ALLO-501 and cemacabtagene ansegedleucel or cema-cel
In January 2019, Allogene announced, in collaboration with Servier, that the U.S. Food and Drug Administration (FDA) approved the Investigational New Drug (IND) for a Phase 1 clinical study for ALLO-501, in relapsed or refractory Non-Hodgkin Lymphoma (NHL), which is referred to as the ALPHA Study. The ALLO-501 candidate product is similar to UCART19 and is licensed to Allogene, pursuant to the sublicense from Servier.
In February 2020, Allogene announced that the FDA had approved the IND for a Phase 1/2 clinical study for ALLO-501 in NHL, which is referred to as the ALPHA2 Study. The ALLO-501 candidate product was created to omit the rituximab recognition domains originally added in ALLO-501, allowing for use in a broader patient population, including those NHL patients with recent rituximab exposure.
In February 2021, Allogene announced that the FDA had granted fast track designation to ALLO-501 for r/r LCBL.
In October 2021, Allogene announced that the FDA had placed a hold on all Allogene’s CAR T clinical trials based on a report of a chromosomal abnormality detected post-Allo CAR T administration in a single patient treated with ALLO-501 in the ALPHA2 Study. In January 2022, Allogene announced that the FDA removed the clinical hold on all of its AlloCAR T clinical trials.
In June 2022, Allogene announced that the FDA granted Regenerative Medicine Advance Therapy (RMAT) designation to ALLO-501 in relapsed or refractory diffuse large B cell lymphoma (LBCL).
In October 2022, Allogene announced the initiation of the potentially pivotal Phase 2 clinical trial of ALPHA2 trial in patients with relapsed or refractory large B-cell lymphoma. Allogene also announced that it was in the process of initiating the EXPAND trial, which is a separate potentially registration trial for ALLO-647—Allogene’s anti-CD52 monoclonal antibody. Allogene has stated that, assuming favorable outcomes and subject to FDA discussions, Allogene plans to seek FDA approval of ALLO-501 and ALLO-647 based on the ALPHA2 trial and the EXPAND companion trial.
In January 2024, Allogene announced it will focus on the development of its investigational product cemacabtagene ansegedleucel, or cema-cel), as part of a first line treatment plan for newly diagnosed and treated LBCL patients who are likely to relapse and need further therapy (ALPHA3 pivotal Phase 2 clinical trial). As a result, Allogene announced it will deprioritize the currently enrolling third line (3L) LBCL ALPHA2 and EXPAND trials. Furthermore, Allogene announces a new Phase 1b ALPHA2 cohort of up to 40 relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) patients to be treated with the investigational product cema-cel.
ALLO-316
In December 2020, Allogene announced that the FDA had approved the IND for a Phase 1 clinical study for ALLO-316, in Renal Cell Carcinoma (RCC), which is referred to as the TRAVERSE Study. ALLO-316 is a gene-edited allogeneic CAR T-cell product targeting CD70 and is licensed to Allogene pursuant to the Allogene License Agreement.
In October 2021, Allogene announced that the FDA had placed a hold on all Allogene’s AlloCAR T clinical trials based on a report of a chromosomal abnormality detected post-Allo CAR T administration in a single patient treated with ALLO-501 in the ALPHA2 study. In January 2022, Allogene announced that the FDA has removed the clinical hold on all its AlloCAR T clinical trials.
In March 2022, Allogene announced that the FDA has granted fast track designation to ALLO-316.
Allogene - Other Programs
Allogene has additional programs in its pipeline under license, including ALLO-715 and ALLO-605, which target BCMA for the treatment of Multiple Myeloma (MM), ALLO-819, which targets FLT3 for the treatment of acute myeloid leukemia, ALLO-213, which targets DLL3 for the treatment of small cell lung cancer, and ALLO-182, which targets Claudin 18.2 for the treatment of gastric and pancreatic cancer.
UCART Product Candidates
UCART123
In December 2016, the company submitted an IND application for UCART123 with respect to two proposed Phase 1 studies to be conducted, one in Acute Myeloid Leukemia (AML).
In June 2019, the company submitted a new IND application with respect to a proposed Phase 1 study to be conducted in relapsed/refractory Acute Myeloid Leukemia (r/r AML) with a new version of the UCART123 product candidate. In July 2019, the FDA approved the IND, and the first patient was dosed in January 2020 at MD Anderson Cancer Center (Houston, Texas).
UCART22
In April 2018, the company submitted an IND application with respect to a proposed Phase 1/2 study to be conducted in relapsed or refractory B-cell Acute Lymphoblastic Leukemia (r/r B-ALL). In May 2018, the FDA approved the IND, and the first patient was dosed in November 2019 at MD Anderson Cancer Center (Houston, Texas).
UCARTCS1
In December 2018, the company submitted an IND application with respect to a proposed Phase 1 study to be conducted in r/r MM. In January 2019, the FDA approved the IND, and the first patient was dosed in October 2019 at MD Anderson Cancer Center (Houston, Texas).
On July 6, 2020, the company announced that the MELANI-01 Study was placed on clinical hold by the FDA, which hold has been lifted by the FDA on November 17, 2020. In May 2023, the company announced its decision to stop enrollment and treatment of patients with UCARTCS1 under the MELANI-01 Study.
UCART20x22
In June 2022, the company submitted an IND application with respect to a proposed Phase 1/2a study to be conducted in relapsed or refractory B-Cell Non-Hodgkin’s Lymphoma (B-NHL). In August 2022, the FDA approved the IND, and the trial is enrolling patients.
Strategy
The company strategy is to leverage the transformative potential of its unique gene-editing technologies and expertise through its cell therapy platform.
The key elements of its strategy are to Advance its self-owned allogeneic UCART portfolio of product candidates up to the Biologics License Application (BLA) and commercialize them; continue to utilize its self-owned manufacturing network to produce commercial-grade UCART products for clinical use, as well as critical raw and starting materials of the UCART product candidates; Structure a commercial launch plan for its self-owned product candidates; and continue the research and development of its hematopoietic stem and progenitor cells (HSPC) platform (named .HEAL).
UCART Pipeline
The company is developing a series of product candidates for advanced hematologic cancers. The company’s lead immuno-oncology product candidates, which it refers to as Universal CAR T-cells (UCARTs), are allogeneic CAR T-cells engineered to be used as an off-the-shelf treatment. Each UCART product candidate is designed to target a selected antigen expressed on tumor cells and bears specific engineered attributes, such as inhibition of alloreactivity and compatibility with specific medical regimens that cancer patients may undergo. UCART is the first therapeutic product line that the company is developing with its gene-editing platform to address unmet medical needs in oncology. The company focuses its initial internal pipeline in the hematologic cancer space, targeting diseases with high unmet needs, such as ALL, AML, and NHL.
UCART123 for AML
UCART123 is an allogeneic engineered T-cell product designed for the treatment of hematologic malignancies expressing the alpha chain of the interleukin-3 receptor (IL3RA), or CD123, and is being developed for the treatment of AML.
UCART123 is designed to become active, proliferate, secrete cytokines and kill CD123 expressing cells. UCART123 bears a CAR targeting the CD123 antigen, providing specificity for CD123 expressing cells. In addition, as with all UCART products, UCART123 lacks the TCR and is intended to be used in an allogeneic context. UCART123 activity could potentially lead to eradication of CD123-expressing cancer cells through T-cell mediated killing, pro-inflammatory cytokine production, as well as CAR T-cell amplification. The current version of UCART123 has, in addition of the suppression of the TCRa gene, the suppression of the CD52 gene in order to potentially induce resistance to an anti-CD52 monoclonal antibody, such an alemtuzumab, as part of the preconditioning.
The AMELI-01 Study is an open-label, Phase 1, single arm, multicenter clinical trial designed to evaluate the safety, expansion, persistence and clinical activities of UCART123 in patients with r/r AML. This trial is a dose-escalation study for UCART123 with 4 separate dose cohorts across different lymphodepletion regimens—lymphodepletion with either fludarabine and cyclophosphamide (FC) or FC with alemtuzumab (FCA).
In March 2020, the company filed an amendment to the protocol of the AMELI-01 Study to evaluate the addition of an anti-CD52 antibody to the lymphodepletion regimen compared to the pre-amendment fludarabine (F)-cyclophosphamide I lymphodepletion regimen. An anti-CD52 antibody (alemtuzumab, A)-based lymphodepletion regimen is evaluated in separate cohorts of patients, to guide the future development of UCART123 in AML. The optimal lymphodepletion regimen prior to the administration of CAR-T product candidates remains an area of investigation in the field of CAR T-cell therapy. The AMELI-01 Study is open for patient recruitment at University of Texas, MD Anderson Cancer Center (Houston, Texas), H. Lee Moffitt Cancer Center & Research Institute (Tampa, Florida), Dana Farber/ Partners Cancer Care, Inc (Boston, Massachusetts), New York Presbyterian / Weill Medical College of Cornell University (New York, New York), Northwestern University (Chicago, Illinois), University of Miami (Miami, Florida), the Regent of the University of California on behalf of its San Francisco Campus (San Francisco, California), The Trustee of University of Pennsylvania (Philadelphia, Pennsylvania), and Roswell Park Cancer Institute Corporation doing business as Roswell Park Comprehensive Cancer Center (Buffalo, New York).
After a protocol-based pause in patient recruitment following a grade 5 event related to CRS, the protocol treatment strategy was modified and as of December 31, 2023, AMELI-01 had commenced enrolling patients in the FCA 2-dose regimen arm at DL2. The arm incorporated the use of prophylactic tocilizumab, which is associated with reduced incidence of CRS.
In December 2022, the company reported positive preliminary safety and efficacy clinical data from the Phase 1 AMELI-01 Study, at the American Society of Hematology annual meeting. This preliminary clinical data was collected prior to the protocol amendment. The preliminary safety data showed that the FCA lymphodepletion regimen resulted in robust lymphodepletion for greater than 28 days in all patients.
UCART22 for B-ALL
UCART22 is an allogeneic engineered T-cell product candidate designed for the treatment of CD22-expressing hematologic malignancies and is being developed for the treatment of B-ALL.
UCART22 is an allogeneic engineered T-cell product candidate intended for the treatment of CD22-expressing hematologic malignancies. UCART22 is designed to become active, proliferate, secrete cytokines and kill CD22 expressing cells (i.e. either CD22 positive tumor cells or non-malignant CD22-positive B lineage cells). UCART22 bears a CAR targeting the CD22 antigen, providing specificity for CD22 expressing cells. As with all UCART products, UCART22 lacks the TCR and is intended to be used in an allogeneic context. In addition, UCART22 has undergone the suppression of the CD52 gene in order to potentially induce resistance to an anti-CD52 monoclonal antibody, such as alemtuzumab, as part of the preconditioning.
UCART22 activity could potentially lead to eradication of CD22-expressing cancer cells through T-cell mediated killing, pro-inflammatory cytokine production, as well as CAR T-cell amplification.
The BALLI-01 Study is an open-label, Phase 1/2, single arm, multicenter clinical trial designed to evaluate the safety, expansion, persistence, and clinical activities of UCART22 in patients with r/r ALL. This trial is a dose-escalation and expansion study for UCART22 with 4 separate dose cohorts. The clinical study protocol allows for up to 40 patients to enroll in the dose escalation period and 18-53 patients in the dose expansion period of the Phase 1/2 study.
In April 2020 and December 2020, the company filed amendments to the protocol of the BALLI-01 Study to open the study to young adults and adolescents and to evaluate the addition of an anti-CD52 antibody (alemtuzumab) to the lymphodepletion regimen with this FCA lymphodepletion regiment compared to the pre-amendment FC lymphodepletion regimen.
BALLI-01 Study is open to patient recruitment at Memorial Sloan Kettering Cancer Center (New York, New York), Children’s Hospital of Philadelphia (Philadelphia, Pennsylvania), the University of Chicago (Chicago, Illinois), University of Texas, MD Anderson Cancer Center (Houston, Texas), The Regents of the University of California on behalf of its Los Angeles campus (Los Angeles, California), Dana Farber/Mass General Brigham Cancer Care, Inc. (Boston, Massachusetts), HÔpital Saint-Louis AP-HP (Paris, France), HÔpital Robert Debre AP-HP (Paris, France), CHU de Nantes (Nantes, France), and CHU de Rennes (Rennes, France). As of December 31, 2023, the company was enrolling patients of the BALLI-01 Study at dose level 2 with an FCA lymphodepletion regimen, and the BALLI-01 Study is being conducted with UCART22 product candidate that has been fully manufactured in-house at its facility in Raleigh, North Carolina.
In December 2022, the company presented positive preliminary clinical data from the Phase 1 BALLI-01 Study at a Live Webcast during the American Society of Hematology annual meeting.
UCART20x22 for NHL
UCART20x22 is an allogeneic engineered T-cell product candidate targeting CD20 and CD22, both of which are expressed in B-cell malignancies and developed for the treatment of relapsed or refractory B-cell NHL.
UCART20x22 is a derivative of UCART22, that includes an additional CAR targeting CD20 to increase breadth of antigen targeting. The company targets both CD20 and CD22 is more likely to prevent tumor escape. As all its UCART product candidates, UCART20x22 lacks the TCR and is intended to be used in an allogeneic context. In addition, UCART20x22 has the suppression of CD52 gene in order to potentially induce resistance to an anti-CD52 monoclonal antibody, such as alemtuzumab, as part of the preconditioning.
In April 2022, the company presented positive preclinical data of UCART20x22 at the American Association for Cancer Research 2022 annual meeting. These data showed that UCART20x22 has a strong activity against tumor cell lines expressing either a single antigen, CD20 or CD22, or both simultaneously. The in vivo pre-clinical models demonstrated that UCART20x22 efficiently eradicates tumors expressing both or either antigen, and sustained presence of UCART20x22 cells was observed in the bone marrow after tumor clearance. in vitro assays against primary cells from non-Hodgkin lymphoma patients with diverse CD22 and CD20 antigen levels demonstrate that UCART20x22 has potent and specific cytotoxic activity.
The NATHALI-01 Study is open to patient recruitment at Dana-Farber/Mass General Brigham Cancer Care, Inc (Boston, Massachusetts), and Sarah Cannon Research Institute, LLC and St. David’s Healthcare Partnership, LP., LLP doing business as St. David’s South Austin Medical Center (Austin, Texas).
As of December 31, 2023, the company presented preliminary results of the Phase I NaThaLi-01 clinical trial evaluating UCART20x22 in patients with relapsed or refractory non-Hodgkin lymphoma (r/r NHL) at the American Society of Hematology Annual Meeting.
CLLS52 (alemtuzumab) as anti-CD52 monoclonal antibody
Following the execution of the supply agreement the company entered into with Genzyme relating to the supply of alemtuzumab, it is implementing the use of alementuzumab as a Cellectis investigational medicinal product, coded as CLLS52, in the clinical protocols BALLI-01, MELANI-01, and NATHALI-01 in the United States and in the relevant European Union member states.
Self-owned UCART programs for solid tumors
The company is applying is UCART platform to develop CAR-T candidates targeting solid tumors. Its self-owned UCART programs for solid tumors are in the preclinical phase of development.
UCARTMESO
UCARTMESO is an allogeneic CAR T-cell product candidate targeting Mesothelin.
In November 2021, the company presented the first preclinical data on UCARTMESO at the annual meeting of the Society for Immunotherapy of Cancer (SITC). UCARTMESO product candidate is composed of allogeneic non-alloreactive T cells edited with TALEN-encoding mRNAs to disrupt TRAC, CD52 and TGFBR2 genes, and transduced ex vivo with a recombinant lentiviral vector to express a second-generation CAR targeting Mesothelin. It is the first TALEN-induced triple knock out (KO) product candidate in the allogeneic CAR-T space. The preclinical data demonstrated potent activity of UCARTMESO in vitro and in vivo against MSLN expressing cell lines, and in vivo activity in pancreatic and pleural mesothelioma mouse models. Due to TGFBR2 KO, UCARTMESO was shown to restore IL2RA upregulation upon in vitro activation, even in media rich in TGFB1, which contributes to the immune suppressive microenvironment in tumors.
UCARTFAP
UCARTFAP is an allogeneic CAR-T cell targeting Cancer Associated Fibroblasts (CAFs) in the tumor microenvironment. CAFs secrete a number of factors amounting to physical and chemical barriers preventing T-cell activity. Reducing the amount of CAFs, will, in turn reduce the immunosuppressive signals emitted from the tumor and hopefully convert ‘cold’ tumors into ‘hot’ tumors that can then be targeted with checkpoint inhibitor therapy. The TCR knockout is to prevent GVHD and the beta-2 microglobulin knocked out to provide resistance to the patient’s own T-cells.
UCARTMUC1
UCARMUC1 is an allogeneic CAR T-cell targeting Mucin 1 for triple negative breast cancer and a variety of epithelial cancers. As other solid tumor targets can be plagued by safety concerns due to off-tumor expression, MUC1 is of high interest as its expression in normal epithelium is restricted to apical membranes. Additionally, its heavy glycosylation in normal tissue renders MUC1 undetectable by Cellectis’ MUC1 CAR that only recognizes hypoglycosylated MUC1 present in cancer cells. UCARTMUC1 incorporates four TALEN knockouts (TCR, B2M, PD-1, and TGFBR2) with two knockins (IL-12 and HLA-E). In lieu of the deleted beta-2 microglobulin gene (part of MHC-1 complex), Cellectis has inserted the HLA-E gene to cloak the cells from immune detection by NK cells, thus increasing CART persistence. In lieu of the PD-1 gene, Cellectis has inserted the IL-12 gene to enhance tumor cell killing and attract other pro-inflammatory cells when induced by the MUC1 CAR binding tumor cells. Preclinical data indicates that UCARTMUC1 shows strong intratumoral expansion translating into promising preclinical anti-tumor activity in vivo.
Programs Under Licensing Agreements
ALLO-501 and cema-cel, for NHL and chronic lymphocytic leukemia (CLL)
ALLO-501 (which we exclusively license to Servier pursuant to the Servier License Agreement, and which has been sublicensed to Allogene by Servier in the United States) is an allogeneic engineered T-cell product intended for the treatment of CD19-expressing hematologic malignancies.
ALLO-501A was created as a second-generation version of ALLO-501, designed to omit the rituximab recognition domains originally added in ALLO-501. Because rituximab is a typical part of the treatment regimen for a patient with NHL, this change is intended to facilitated treatment of a broader patient population.
In January 2019, Allogene announced, in collaboration with Servier, that the FDA approved the IND for Phase 1 clinical study for ALLO-501 in relapsed or refractory NHL (the ALPHA Study). The ALPHA Study is an open-label, Phase 1, single arm, multicenter clinical trial evaluating the safety and tolerability of ALLO-501 in adult patients with the most common r/r NHL subtypes, including r/r large B-cell lymphoma, including DLBCL, and r/r follicular lymphoma (FL). The trial is a dose-escalation study for ALLO-501 with three separate dose cohorts. Prior to ALLO-501 treatment, all patients undergo lymphodepletion with a regimen of fludarabine, cyclophosphamide and ALLO-647 (an anti-CD52 monoclonal antibody).
In February 2020, Allogene announced that the FDA had approved the IND for a Phase 1/2 clinical study for ALLO-501A in relapsed or refractory NHL (the ALPHA2 Study). The ALPHA2 Study is an open-label, Phase 1/2, single arm, multicenter clinical trial of ALLO-501A in adult patients with R/R large B-cell lymphoma, including DLBCL, or transformed FL. The Phase 1 portion of the ALPHA2 Study is designed to assess the safety and tolerability at increasing dose levels of ALLO-501A and identify the recommended doses of ALLO-501A and ALLO-647 (an anti-CD52 monoclonal antibody) for use in the Phase 2 portion of the trial. Allogene initiated the ALPHA2 Study in the second quarter of 2020.
In February 2021, Allogene announced that the FDA had granted fast track designation to ALLO-501A for relapsed or refractory diffuse large B cell lymphoma (LCBL).
In June 2022, Allogene announced that the FDA granted Regenerative Medicine Advance Therapy (RMAT) designation to ALLO-501A in relapsed or refractory large B cell lymphoma (LBCL).
In October 2022, Allogene announced the initiation of the Phase 2 clinical trial of ALPHA2 trial in patients with relapsed or refractory LBCL. Allogene also announced that it was in the process of initiating the EXPAND trial, which is a separate potentially registration trial for ALLO-647—Allogene’s anti-CD52 monoclonal antibody that Allogene is developing with the goal of potentially enabling expansion, persistence and improved clinical outcomes of allogeneic CAR T cell product candidates, including ALLO-501A. Allogene has stated that, assuming favorable outcomes and subject to FDA discussions, Allogene plans to seek FDA approval of ALLO-501A and ALLO-647 on the basis of the ALPHA2 trial and the EXPAND companion trial.
In January 2024, Allogene announced it will focus development of cema-cel (or ALLO-501A), as part of the first line (1L) treatment plan for newly diagnosed and treated LBCL patients who are likely to relapse and need further therapy. Allogene announced it will deprioritize the currently enrolling third line (3L) ALPHA2 and EXPAND trials. Furthermore, Allogene announced a new Phase 1 ALPHA2 cohort of 12 CLL patients treated with the investigational product cema-cel.
In December 2021, Allogene, in collaboration with Servier, reported Phase 1 data on ALLO-501 and ALLO-501A r/r NHL at the annual meeting of the American Society of Hematology. As of the October 18, 2021 data cutoff, 50 patients were enrolled in the ALPHA study, of whom 49 were evaluable for safety and 40 were evaluable for efficacy, and 29 patients were enrolled in the ALPHA2 study, of whom 28 were evaluable for safety and 25 were evaluable for efficacy. ALLO-501 and ALLO-501A therapy was associated with consistent and manageable safety with no DLTs or GvHD; low rates of Grade 3 ICANs and CRS. In November 2022, Allogene presented an update on clinical data for the Phase 1 ALPHA Studies.
In November 2022, Allogene presented an update on clinical data for the Phase 1 ALPHA Studies. Allogene reported that the Phase 1 ALPHA Studies support the ability of a single administration of CAR T cells to generate responses similar to approved autologous CAR T therapies and that the ALPHA Studies demonstrated a manageable safety profile.
In June 2023, Allogene presented long-term follow up data from the Phase 1 ALPHA/ALPHA2 trials of ALLO-501/501A in patients with r/r LBCL at the American Society of Clinical Oncology Annual Meeting.
ALLO-316, for Renal Cell Carcinoma (RCC)
ALLO-316, which the company exclusively license to Allogene pursuant to the Allogene License Agreement, is an allogeneic engineered CAR T-cell product targeting CD70.
In December 2020, Allogene announced that the FDA had approved the IND for a Phase 1 clinical study for ALLO-316, in RCC, which is referred to as the TRAVERSE Study.
In March 2022, Allogene announced that the FDA has granted fast track designation to ALLO-316.
In November 2022, Allogene presented initial data from TRAVERSE Study and reported that observed anti-tumor activity was largely confined to patients with CD70 expressing tumors. In April 2023, Allogene presented interim data from its Phase 1 TRAVERSE trial of ALLO-316, in an oral presentation at the American Association for Cancer Research Annual Meeting. As of the March 23, 2023, data cutoff, 19 patients were enrolled in the Phase 1 trial, 10 of whom had RCC confirmed to express CD70.
Other Gene Editing Programs
Beyond its CAR-T programs, the company leverages its TALEN gene editing platform to pursue additional development opportunities, both internally and in collaboration with third party companies and academic centers. The company aims to enter the clinic with one or more gene editing programs beyond UCARTs in the future.
HEAL, the hematopoietic stem and progenitor cells platform for genetic diseases
The company is developing a gene editing platform that leverages the power of TALEN technology, to allow highly efficient gene inactivation, insertion and correction in hematopoietic stem and progenitor cells (HSPCs). It is used this platform to develop programs in sickle cell disease (SCD), lysosomal storage disease (LSD) and primary immunodeficiencies.
TalGlobin is developed with TALEN technology intended to induce a double DNA strand break at the HBB gene causing SCD, and AAV particles containing a DNA repair template designed to correct the faulty HBB gene via endogenous homology directed repair (HDR).
In December 2021, the company presented initial pre-clinical data from TalGlobin at the American Society of Hematology Annual Meeting. Initial pre-clinical data from TALGlobin show that TALEN is specific and efficient in correcting the mutated beta-globin gene, the underlying cause of SCD. The data also demonstrate that TALEN-based engineering could be used to correct the beta-globin gene mutation in HbSS patient-derived hematopoietic stem and progenitor cells. The data show up to 70% of HBB allelic correction, with only 9% of HBB biallelic inactivation and a low level of TALEN off-target cleavage. Genetic correction of HBB translates into high level of hemoglobin A expression (up to 47% HbA detected among total hemoglobin) and reversion of the sickling phenotype in differentiated red blood cells. Preclinical data show the capacity of TALGlobin01 edited cells to engraft in vivo using an immunodeficient mouse model. Collectively, the preclinical data demonstrate efficiency and safety of TALEN treatment in SCD patient-derived hematopoietic stem and progenitor cells.
In October 2022, the company presented encouraging pre-clinical data from TalGlobin at the European Society of Gene and Cell Therapy 29th Congress. Overall, the results showed that non-viral DNA delivery associated with the company’s TALEN gene editing technology reduces the toxicity observed with viral DNA delivery and allows high levels of HBB gene correction in long-term repopulating hematopoietic stem cells.
ArtEx
The company has also developed an artificial exon (ArtEx) strategy to introduce a corrected gene copy coding for a relevant LSD enzyme into the intronic region of a gene expressed in myeloid cells. This approach would avoid the potential collateral effect of knocking out the endogenous gene without a correct replacement. This editing strategy opens new avenues for the treatment of LSDs, as it would allow to address the systemic lack of lysosomal enzyme activity, including in the brain, and could be used to produce virtually any defective LSD enzyme. It represents a new platform, in which a single safe and well characterized TALEN could be used to treat different LSDs.
In October 2022, the company presented a TALEN-based gene editing approach that reprograms HSPCs to secrete alpha-L-iduronidase (IDUA), an enzyme missing in Mucopolysaccharidosis type I (MPS-I). MPS-I is associated with severe morbidity representing a significant unmet medical need. The company established a TALEN-based ex vivo gene editing protocol to insert an IDUA-expression cassette into a specific locus of HSPC.
Licensing Relationships
In addition to the development of the company’s own portfolio of product candidates targeting tumor-associated antigens, it has pursued a strategy of forging strong relationships with pharmaceutical or clinical stage biopharmaceutical companies.
License Agreement with Allogene
In June 2014, the company entered into a research collaboration and license agreement with Pfizer, Inc. (‘Pfizer’) pursuant to which it agreed to collaborate to conduct discovery and pre-clinical development activities to generate CAR T-cells directed at Pfizer- and Cellectis-selected targets in the field of human oncology. It granted Pfizer an exclusive, worldwide, royalty-bearing, sublicensable license, on a target-by-target basis, under certain of our intellectual property to make, use, sell, import, and otherwise commercialize products directed at the Pfizer-selected targets in the field of human oncology.
On April 3, 2018, Pfizer and Allogene Therapeutic, Inc. (‘Allogene’) announced that they entered into an asset contribution agreement, pursuant to which Allogene purchased Pfizer’s portfolio of assets related to allogeneic CAR T-cell therapy (the ‘Asset Contribution Transaction’). Pursuant to the Asset Contribution Transaction, effective as of April 6, 2018, Allogene purchased Pfizer’s portfolio of assets related to allogeneic CAR T-cell Therapy.
License, Development and Commercialization Agreement with Servier
In February 2014, the company entered a research, product development, option, license, and commercialization agreement (the Prior Servier Agreement) with Servier. Pursuant to the Prior Servier Agreement, the company was responsible for the research and development up to and, including the Phase 1 clinical trial of candidate products directed against five targets, including the UCART19 product candidate. Pursuant to the Prior Servier Agreement, the company grants Servier the right to exercise an exclusive option to obtain an exclusive, worldwide license, on a product candidate-by-product candidate basis, with respect to each product candidate selected by Servier and developed under the agreement.
On March 6, 2019, the company and Servier entered into a new License, Development and Commercialization Agreement (the March Servier License Agreement). The March Servier License Agreement superseded and replaced the Prior Servier Agreement in order to modify the targets covered by the company’s license to Servier, to establish the terms of its and Servier’s collaboration and to reflect the status of products in development.
On February 18, 2020, the company and Servier entered into a binding term sheet to enter into an amendment to the March Servier License Agreement to grant to Servier an exclusive license limited to CD19 target, but extended to all gene-edited allogeneic CAR T-cell products targeting CD19 and gene edited exclusively by Cellectis’ TALEN. On March 4, 2020, the company and Servier entered into the amendment to the March Servier License Agreement contemplated by this term sheet (such March Servier License Agreement as amended on March 4, 2020, the Servier License Agreement).
Under the Servier License Agreement, Cellectis grants to Servier, an exclusive worldwide, royalty bearing license with sublicensing rights under certain conditions, under certain of its patents and know-how to develop, manufacture and commercialize gene-edited allogeneic CAR T-cell products targeting CD19 and gene edited exclusively by Cellectis’ TALEN. Servier, directly or through its sublicensees, will be solely responsible for the research, development and commercialization of these products. In addition, Servier confirms it will not pursue the development of five other targets for products using Cellectis technology and consequently Cellectis retains control over them.
On September 15, 2022, Servier sent to the company and Allogene a notice of discontinuation of its involvement in the development of the CD19 Products and purported to provide Allogene with the ability to elect to obtain a license to the CD19 Products outside of the United States.
Research Collaboration and Exclusive License Agreement with Iovance Biotherapeutics
On December 30, 2019, the company entered into a research collaboration and exclusive worldwide license agreement with Iovance Biotherapeutics. Iovance licensed its TALEN technology in order to develop tumor infiltrating lymphocytes (TIL) that have been genetically edited to create more potent cancer therapeutics. The worldwide exclusive license enables Iovance to use TALEN technology to address multiple gene targets to modify TIL for therapeutic use in several cancer indications.
Collaboration and License with Cytovia Therapeutics
On February 12, 2021, the company entered into a research collaboration and non-exclusive license agreement with Cytovia Therapeutics, Inc., or Cytovia to develop induced Pluripotent Stem Cell (iPSC) iPSC-derived Natural Killer (NK) and CAR-NK cells edited with its TALEN (the Cytovia Agreement).
Pursuant to the Cytovia Agreement, as expanded in November 2021 to include a new CAR target and development in China by Cytovia’ joint venture entity, CytoLynkx Therapeutics. Cellectis also received an option to participate in certain future financing rounds by Cytovia.
In April 2022, in connection with Cytovia’s entering into a definitive business combination agreement with a publicly traded Special Purpose Acquisition Company (SPAC), the company entered into an amendment to the Cytovia Agreement.
Collaboration and Investment Agreements with AstraZeneca
On November 1, 2023, the company and AstraZeneca announced that they entered into a Joint Research and Collaboration Agreement, an Initial Investment Agreement and the Subsequent Investment Agreement.
Joint Research and Collaboration Agreement
On November 1, 2023, the company entered into a Joint Research and Collaboration Agreement with AZ Ireland (the AZ JRCA). Pursuant to the AZ JRCA, the parties will collaborate to develop up to 10 novel cell and gene therapy candidate products, selected from a larger pool of potential targets identified by AZ Ireland, for human therapeutic, prophylactic, palliative, and analgesic purposes. Each party will be responsible for performing research and development activities based on research plans to be agreed upon throughout the initial five-year collaboration term under the AZ JRCA.
Intellectual Property
The most relevant issued patents in the company portfolio consists approximately 66 company-owned and 12 in-licensed U.S. patents, 49 company-owned and 5 in-licensed European patents, and 214 company-owned and 26 in-licensed patents in other jurisdictions, such as Australia, Canada, China, Hong Kong, India, Israel, Japan, Korea, Mexico, and Singapore.
The most relevant pending patent applications in the company portfolio consists of 31 company-owned and 2 in-licensed U.S. patent applications, 41 company-owned and 1 in-licensed European patent applications, 141 company-owned and 7 in-licensed patent applications pending in other jurisdictions, such as Australia, Brazil, Canada, China, Hong Kong, India, Israel, Japan, Korea, Mexico, and Singapore.
The company’s most relevant portfolio includes a total of 372 owned and in-licensed granted patents, and 223 owned and in-licensed patent applications.
The company’s UCART product candidates rely for each product candidate upon one or more patent rights protecting various aspects of the technologies, including rights relating to: The genetic editing of T-cells, using TALEN technology, covers approximately twelve company’s owned patent families and three in-licensed patent families; the insertion of transgenes into T-cells using electroporation of mRNA, covered by approximately five company-owned patent families; the appending of attributes to T-cells, covered by approximately eight company-owned patent families and one in-licensed patent family; the molecular structure of CARs, covered by approximately six company-owned patent families; and specific CARs that target selected antigen markers are covered by approximately fifteen company-owned patent applications and one in-licensed patent family.
Gene Editing Platform
The company’s UCART product candidates rely upon its gene-editing platform and T-cell and CAR technology platforms. The patent portfolio covers these platforms and technologies, includes approximately 214 patents or pending patent applications in various countries, comprising 43 in-licensed and 98 the company owns issued patents among which 26 are US granted patents and 13 European granted patents. Certain of these issued patents and pending patent applications, which expire between 2031 and 2041, cover product claims or process claims relevant to each of its product candidates, including UCART19, UCART123, UCART22, UCARTCS1 and UCART20x22.
The company’s gene-editing platform and each of its UCART product candidates benefits from the protections conferred by several patents and patent applications in its patent portfolio. As a result of this broad range of patent protection, very few individual patents in the company’s portfolio are critical to its ability to effectively conduct its product development activities. Although certain patents relating to the company’s electroporation technology have expired, other patents and patent applications covering this technology remain in force, and additional patents protect the nucleases delivered by the company’s electroporation technology, as well as the methods to modify the cells by use of such nucleases. Among the company’s main patents EP3189073, EP3126390, EP3008186, EP3004349 and EP3116902 are under opposition before the European Patent Office. JP6810685, which was opposed before the Japanese patent office has been maintained under amended form.
UCART19
In addition to the patent portfolio relating to its platform and technologies, the company’s patent portfolio relating specifically to UCART19 includes granted patents and pending patent applications from the patent family WO2014184143 (CD19 Specific Chimeric Antigen Receptor and Uses Thereof).
UCART123
In addition to the patent portfolio relating to its platform and technologies, the company’s patent portfolio relating specifically to UCART123 includes granted patents and pending patent applications from the patent family WO2018178377 (CD123 Specific Chimeric Antigen Receptors for Cancer Immunotherapy). These patent and patent applications, which, if issued, would expire in 2035, include claims to cover the composition of matter of UCART123, methods of manufacture of UCART123, and methods to use UCART123 in cancer treatment.
UCART22
In addition to the patent portfolio relating to its platform and technologies, the company’s patent portfolio relating specifically to UCART22 includes pending patent applications from the families WO2018173878 and WO2028278377. These patent applications, which if issued, would expire in 2038, include claim directed to the composition of matter of UCART22, methods of manufacture of UCART22, and methods to use UCART22 in cancer treatment.
UCART20x22
In addition to the patent portfolio relating to its platform and technologies, the company’s patent portfolio relating specifically to UCART20x22 includes pending patent applications from the family WO2022023529. These patent applications, which if issued, would expire in 2041, include claims directed to the composition of matter of UCART20x22, methods of manufacture of UCART20x22, and methods to use UCART20x22 in cancer treatment.
Material Exclusive Licenses Granted to Cellectis
License from Regents of the University of Minnesota
In January 2011, the company entered into an exclusive license agreement with Regents of the University of Minnesota, or UMN. Pursuant to this agreement, as amended in 2012, 2014, 2015 and 2022 the company and its affiliates were granted an exclusive, worldwide, royalty-bearing, sublicensable license, under certain patents and patent applications owned by UMN, to make, use, sell, import, and otherwise dispose of products covered by the licensed patents, for all fields of use. These licensed patents relate to TALEN technology. Pursuant to the agreement, the company is required to achieve certain specified research- and sales-related milestones.
Intellectual Property Strategy
The company’s strategy is also to develop and obtain additional intellectual property covering innovative manufacturing processes and methods for genetically engineering T-cells expressing new constructs and for genetically engineering plants expressing new traits. To support this effort, the company has established expertise and development capabilities focused in the areas of pre-clinical research and development, manufacturing and manufacturing process scale-up, quality control, quality assurance, regulatory affairs and clinical trial design and implementation. Thus, the company expects to file additional patent applications to expand this layer of its intellectual property estate.
Competition
The company’s competitors include:
Autologous and Allogeneic CAR T-cell Space: Juno Therapeutics, Inc. (in collaboration with Editas Medicine Inc.), acquired by Celgene Corporation and acquired since by Bristol-Myers Squibb; Bluebird bio, Inc. (in collaboration with Celgene Corporation, acquired since by Bristol-Myers Squibb); Ziopharm Oncology Inc. (in collaboration with Intrexon Corporation); Kite Pharma Inc. (in collaboration with Amgen Inc. and with Sangamo Therapeutics Inc.), acquired by Gilead Sciences Inc.; Novartis AG (in collaboration with Intellia Inc.); Johnson & Johnson (in collaboration with Transposagen Biopharmaceuticals Inc.); Precision Biosciences (in collaboration with Servier); Regeneron Pharmaceuticals Inc. (in collaboration with Adicet Bio Inc); Fate Therapeutics Inc. (in collaboration with Janssen); CRISPR Therapeutics Inc.; Takeda Pharmaceutical Company Limited; Tmunity Therapeutics Inc.; Mustang Bio; Atara Biotherapeutics Inc., (in collaboration with Bayer); Adaptimmune (in collaboration with Astellas); Poseida Therapeutics Inc.; BioNTech SE; Vor Therapeutics Inc.; Autolus Therapeutics plc.; Bellicum Pharmaceuticals, Inc.; and Celyad S.A.
Gene-editing Space: CRISPR Therapeutics Inc. (in collaboration with Bayer AG and Vertex Inc.); Editas Medicine, Inc. (partnered with Allergan and Celgene); Intellia Therapeutics, Inc. (partnered with Novartis); Precision BioSciences, Inc.; Sangamo BioSciences, Inc. (partnered with Kite/Gilead and Pfizer); Vertex/Exonics Therapeutics (partnered with CRISPR Therapeutics); Graphite Bio Inc.; and Beam Therapeutics Inc.
Cell-therapy Space: Adaptimmune Ltd; Iovance Biotherapeutics; Unum Therapeutics, Inc.; NantKwest, Inc.; Cytovia Therapeutics, Inc.; Atara Biotherapeutics, Inc.; and Immunocore Ltd.
The company also faces competition from non-cell based treatments offered by companies, such as Amgen Inc.; AstraZeneca plc; Bristol-Myers Squibb Company; Incyte Corporation; Merck & Co., Inc.; and F. Hoffman-La Roche AG. Immunotherapy is further being pursued by several biotech companies, as well as by large-cap pharmaceuticals.
Government Regulation and Product Approval
The company’s product candidates must be approved by the FDA before they may be legally marketed in the United States and by the appropriate foreign regulatory agencies before they may be legally marketed in foreign countries.
The company’s biological product candidates must be approved by the FDA through the Biologics License Application, or BLA, process before they may be legally marketed in the United States.
The company relies on third parties for the production of clinical and commercial quantities of its products in accordance with current Good Manufacturing Practices (cGMP) regulations.
In the United States, the company’s activities are subject to regulation by various federal, state and local authorities in addition to the FDA, including but not limited to, the Centers for Medicare and Medicaid Services, or CMS, other divisions of the U.S. Department of Health and Human Services (e.g., the Office of Inspector General), the U.S. Department of Justice, or DOJ, and individual U.S. Attorney offices within the DOJ, and state and local governments.
In addition to the foregoing, state and federal laws regarding environmental protection and hazardous substances, including the Occupational Safety and Health Act, the Resource Conservancy and Recovery Act, and the Toxic Substances Control Act, affect the company’s business.
Research and Development.
For the year ended December 31, 2023, the company spent $87.6 million on research and development.
History
Cellectis S.A. was founded in 1999. The company was incorporated as a societe anonyme, under the laws of the French Republic in 2000.
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