Main Product Candidates

	Target	Indication	Study	Preclinical	Phase 1 Dose Escalation	Phase 2 Pivotal	Upcoming expected milestone
Fully owned	UCART22 CD22	ALL	BALLI-01 NCT04150497	UCART22 What is UCART22?  UCART22 is an allogeneic CAR T-cell product candidate targeting CD22. UCART22 is being evaluated in patients with relapsed or refractory B- cell acute lymphoblastic leukemia (r/r B-ALL). BALLI-01 clinical study (evaluating UCART22)  BALLI-01, is a Phase 1/2a open-label dose-escalation and dose-expansion study, designed to evaluate the safety, expansion, persistence and clinical activity of UCART22 in patients with relapsed or refractory B- cell acute lymphoblastic leukemia.			Phase 1 dataset & late-stage development strategy expected in 2025
Fully owned	UCART20x22 Dual Target CD20, CD22	NHL	NatHaLi-01 NCT05607420	UCART20x22 What is UCART20x22? UCART20x22 is Cellectis’ first allogeneic dual CAR T-cell product candidate, targeting CD20 and CD22, being developed for patients with relapsed or refractory B-cell Non-Hodgkin Lymphoma (r/r B-NHL). Dual targeting is designed to enhanced tumor cell killing and to prevent immune escape due to single antigen targeting UCART20x22 is Cellectis’ first product candidate fully developed and manufactured internally. NatHaLI-01 clinical study (evaluating UCART20x22) NatHaLi-01, is a Phase 1/2a open-label dose-finding and dose-expansion study to evaluate the safety, expansion, persistence and clinical activity of UCART20x22 in patients with relapsed and refractory B-Cell Non-Hodgkin Lymphoma.			Phase 1 dataset & late-stage development strategy expected in 2025
Licensed Partners	Cemacabtagene Ansegedleucel (ALLO-501A) CD19 1	LBCL	ALPHA3 NTC06500273	Cemacabtagene Ansegedleucel The following information are based on information provided or otherwise publicly reported by our licensed partners: cemacabtagene ansegedleucel has been developed under a collaboration agreement between Servier and Allogene based on an exclusive license granted by Cellectis to Servier. Servier grants to Allogene exclusive rights to cemacabtagene ansegedleucel in the U.S., EU and UK. The ALPHA3 study targets Large B-Cell Lymphoma (LBCL). What is CEMACABTAGENE ANSEGEDLEUCEL – evaluated in the ALPHA3 study? CEMACABTAGENE ANSEGEDLEUCEL (previously known as ALLO-501A) is an anti-CD19 allogeneic CAR T (AlloCAR T™) product in development, as part of the first line treatment (1L) for newly diagnosed and treated large B-cell lymphoma (LBCL) patients who are likely to relapse following 1L of chemoimmunotherapy and need further therapy. Allogene’s AlloCAR T™ programs utilize Cellectis technologies. AlloCAR T™ is a trademark of Allogene Therapeutics, Inc.
	ALLO-316 ³ CD70	RCC	TRAVERSE NCT04696731	ALLO-316 The following information are based on information provided or otherwise publicly reported by our licensed partners: Allogene’s anti-CD70 programs are licensed exclusively from Cellectis by Allogene and Allogene holds global development and commercial rights to these programs. What is ALLO-316? ALLO-316 is an allogeneic CAR T therapy candidate targeting CD70, and Allogene’s first AlloCAR T solid tumor clinical candidate for the treatment of patients with advanced or metastatic clear cell renal cell carcinoma (RCC). TRAVERSE Study (evaluating ALLO-316) Primary endpoints: Safety and tolerability Secondary endpoints: Anti-tumor efficacy Allogene’s AlloCAR T™ programs utilize Cellectis technologies. AlloCAR T™ is a trademark of Allogene Therapeutics, Inc.
	Allogeneic CAR T	Hematological malignancies		Allogeneic CAR T Research and development activities have started for one program under our collaboration and research agreement with AstraZeneca: an allogeneic CAR T for hematological malignancies.
	Allogeneic CAR T	Solid tumors		Allogeneic CAR T Research and development activities have started for one program under our collaboration and research agreement with AstraZeneca: an allogeneic CAR T for solid tumors.
	In vivo gene therapy	Genetic disorder		In vivo gene therapy Research and development activities have started for one program under our collaboration and research agreement with AstraZeneca: an in vivo gene therapy for a genetic disorder.
	IOV-4001	Melanoma, NSCLC	IOV-GM1-201 NCT05361174	IOV-4001 Cellectis has a research collaboration and exclusive worldwide license agreement with Iovance Biotherapeutics, Inc. enabling Iovance to use certain TALEN® technology addressing multiple gene targets to modify tumor-infiltrating lymphocytes (TIL) for therapeutic use in several cancer indications. What is IOV-4001? IOV-4001 is under development for treatment of unresectable or metastatic melanoma and stage III or IV NSCLC. The therapeutic candidate is next generation tumor infiltrating cells genetically modified by transcription activator-like effector nucleases (TALENS). It acts by targeting cells expressing programmed cell death protein 1 (PD-1). It is administered through parenteral route.

(1) cemacabtagene ansegedleucel has been developed under a collaboration agreement between Servier and Allogene based on an exclusive license granted by Cellectis to Servier.

Servier grants to Allogene exclusive rights to cemacabtagene ansegedleucel in the U.S., EU and UK. The ALPHA3 study targets Large B-Cell Lymphoma (LBCL).

(2) Phase 3 may not be required if Phase 2 is registrational.

(3) ALLO-316 utilizes TALEN® gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at the CD70 target. Allogene holds global development and commercial rights for this investigational candidate.

ALLO-715 and ALLO-605 utilize TALEN® gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at the BCMA target. Allogene holds global development and commercial rights for these investigational candidates.

ALL, Acute Lymphoblastic Leukemia; NHL, Non-Hodgkin’s Lymphoma; LBCL, Large B-Cell Lymphoma; RCC, Renal Cell Carcinoma; NSCLC, Non-Small Cell Lung Cancer

Development of a product candidate takes place in several stages

Discovery: Identification of a new potential target that could lead to a future product candidate.
Product development: Engineering of “Chimeric Antigen Receptor” (CAR) T-cells is one of the technologies developed by Cellectis to construct new product candidates. This approach allows us to design allogeneic product candidates through a gene editing mechanism of T-cells derived from healthy donors. Gene editing is performed using TALEN®, which allow very precise and targeted gene modification and provide new attributes to the product such as additional levels of safety or compatibility with the standard of care.
Preclinical Studies: - In vitro studies performed on specific cell lines to have some preliminary results on the activity of a potential product candidate.
- In vivo studies performed on animal models in order to have preliminary results on the dose-dependent toxicity and on the activity of a potential product candidate before further clinical investigation.
IND filing (or foreign equivalent): The Investigational New Drug (IND) filing in the USA consists of submitting the required study documentation package to the health authority (FDA) to obtain the authorization to perform clinical investigation.
Clinical Studies: Testing of the product candidate in humans.

Phases of clinical trials

Phase I: First time an experimental drug or treatment is tested in humans to examine how well the drug is tolerated.
Phase II: Trials designed to examine if the drug or treatment has a clinical activity.
Phase III: Trials designed to assess the treatment effect on a clinically meaningful endpoint.
Phase IV: Post-marketing studies to gain additional information.

Phase I: First time an experimental drug or treatment is tested in humans to examine how well the drug is tolerated.
Phase II: Trials designed to examine if the drug or treatment has a clinical activity.
Phase III: Trials designed to assess the treatment effect on a clinically meaningful endpoint.
Phase IV: Post-marketing studies to gain additional information.

Main Product Candidates

Allogeneic CAR-T Cell Pipeline

Development of a product candidate takes place in several stages

Phases of clinical trials