On May 12, 2025 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, reported financial results for the first quarter 2025, ending March 31, 2025, and provided a business update (Press release, Cellectis, MAY 12, 2025, View Source [SID1234652871]).
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« We are making progress in our wholly-owned clinical studies and in the three programs under our strategic partnership with AstraZeneca. We will continue to focus our efforts and resources on advancing these core programs and are looking forward to the results expected over the next few months » said André Choulika, Ph.D., Chief Executive Officer at Cellectis.
Pipeline Highlights
UCART Clinical Programs
BALLI-01 study evaluating lasme-cel (UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)
On April 15, 2025, the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) selected lasmecabtagene timgedleucel (lasme-cel) as recommended international non-proprietary name of UCART22 drug substance.
Cellectis continues to focus on the enrollment of patients in the BALLI-01 study and expects to present the Phase 1 dataset and late-stage development strategy for lasme-cel in r/r B-ALL in the third quarter of 2025.
NATHALI-01 study evaluating eti-cel (UCART20x22) in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL)
On April 15, 2025, the INN Expert Committee of the WHO selected etivelcabtagene erigedleucel (eti-cel) as recommended international non-proprietary name of UCART20x22 drug substance.
Cellectis continues to focus on the enrollment of patients in the NATHALI-01 study and expects to present a Phase 1 readout for eti-cel in r/r NHL in late 2025.
Research Data & Preclinical Programs
Novel Non-Viral Gene Editing and Base Editing Research
On April 28, 2025, Cellectis announced the presentation of research data on TALEN-mediated non-viral transgene insertion for advancing cellular and gene therapies, and advancements in genetic editing using TALE base editors (TALEB), at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual meeting that will be held on May 13-17, 2025 in New Orleans. The data will be presented in two posters:
TALEN- Mediated non-viral Transgene Insertion for the Advancement of Cellular and Gene Therapies
In this work, Cellectis combines TALEN-mediated gene editing with non-viral transgene insertion for advancing cellular and gene therapies and explores gene insertion-efficacy and cellular health using single-stranded DNA (ssDNA) for payload delivery in different cell types.
This innovative approach has the potential to address the challenges associated with traditional lentiviral viral methods or AAV-mediated transgene insertion such as manufacturing constraints, potential genomic toxicities or limited payload size.
High fidelity C-to-T editing with TALE base editors
TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits double-stranded DNA by converting a cytosine (C) to a thymine (T) and does not involve a DNA strand nick. Cellectis has developed a method to evaluate TALEB activity, analyzing factors affecting its efficiency. Using precise ssODN knock-in in primary T cells, the method assesses how target sequence composition and spacer variations impact TALEB performance.
Overall, the results of this study enhance the control and use of TALEB, allowing for the design of highly efficient and specific TALEB compatible with future potential therapeutic applications.
The abstracts are live on the ASGCT (Free ASGCT Whitepaper) website. The posters will be available on Cellectis’ website the first day of the event.
Partnerships
AstraZeneca Joint Research and Collaboration Agreement
Research and development activities are ongoing under three cell and gene therapy programs under the joint research and collaboration agreement entered into by Cellectis and AstraZeneca in November 2023: one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder.