ADDED
Risks related to serious adverse events (SAEs) in the discontinued fludarabine, cyclophosphamide, and ALLO-647 (FCA) arm of our ALPHA3 trial, including the Grade 5 SAE, could lead to regulatory actions, negative perceptions, and potential product liability claims.
Reduced manufacturing operations may limit our ability to timely support our development programs.
Disruptions to the operations of the FDA, the SEC and other government agencies, including comparable foreign regulatory authorities, resulting from funding shortages, policy initiatives, staffing reductions or related uncertainty, could impair their ability to perform regulatory functions and negatively impact our business.
There is uncertainty regarding whether the use of fludarabine and cyclophosphamide (FC) without ALLO-647 will achieve sufficient lymphodepletion to support the efficacy of our allogeneic CAR T product candidate in the ALPHA3 trial.
We are heavily reliant on our partners, Cellectis and Servier, for access to TALEN gene editing technology for the manufacturing and development of our oncology product candidates.
After nearly eight years of platform development and treatment of more than 200 patients across six clinical studies, multiple anticipated clinical readouts are expected in the second quarter of 2026.
These readouts could begin to validate several key scientific and clinical assumptions underlying off-the-shelf CAR T therapy, including biologic activity, safety, and the feasibility of standardized, readily available cell therapy across oncology and autoimmune indications.
Renal Cell Carcinoma (RCC) : TRAVERSE trial with ALLO-316 seeks to advance scientific innovation underlying the Dagger technology to optimize CAR T cell expansion and persistence, thereby maximizing the potential of allogeneic CAR T in solid tumors.
Although we are currently focusing on our three core development programs noted above, we continue to build a pipeline to further the research and development of allogeneic CAR T cell product candidates in both hematological malignancies and solid tumors, as well as in autoimmune diseases.
(Cellectis), TALEN gene-editing technology and Arbor Biotechnologies CRISPR-based gene-editing technology in our oncology and autoimmune programs, respectively, to limit the risk of GvHD by engineering T cells to lack functional T cell receptors (TCRs), thereby preventing them from recognizing a patient s normal tissue as foreign.
REMOVED
Changes in funding for the FDA, the SEC and other government agencies including comparable foreign regulatory authorities could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal functions on which the operation of our business may rely, which could negatively impact our business.
Risks Related to the Development of Our Product Candidates Our engineered allogeneic T cell product candidates represent a novel approach to cancer treatment and treatment of autoimmune diseases, which creates significant challenges for us.
Last year we executed our 2024 Platform Vision, which we believe will redefine the future of chimeric antigen receptor (CAR) T therapy by leveraging the unique attributes of allogeneic CAR T products.
Renal Cell Carcinoma (RCC) : TRAVERSE trial with ALLO-316 seeks to advance scientific innovation underlying the Dagger technology to optimize CAR T cell expansion and persistence, thereby maximizing the potential of allogeneic CAR T in solid tumors while mitigating treatment-associated inflammatory response.
Although we are currently focusing on our three core development programs noted above, we continue to have a deep pipeline to further the research and development of allogeneic CAR T cell product candidates in both hematological malignancies and solid tumors.
(Cellectis), TALEN gene-editing technology to limit the risk of GvHD by engineering T cells to lack functional T cell receptors (TCRs), thereby preventing them from recognizing a patient s normal tissue as foreign.
With the goal of enhancing the expansion and persistence of our engineered allogeneic T cells, we use TALEN technology to inactivate the CD52 gene in donor T cells and an anti-CD52 monoclonal antibody to deplete CD52 expressing T cells in patients while sparing the therapeutic allogeneic T cells.
We believe this enables a window of persistence for the infused allogeneic T cells to actively target and destroy cancer cells.
We are currently utilizing CF1 for clinical manufacturing of our product candidates.
Finally, we plan to leverage next generation technologies to develop more potent product candidates and to develop product candidates to overcome premature rejection of allogeneic CAR T cells by the patient s immune system.