Elixirgen Therapeutics Presents New Clinical Data Demonstrating Ex Vivo Telomere Elongation with EXG-34217 in Dyskeratosis Congenita at the 64th ASH Annual Meeting

13 Dec 2022
pipelinereview.com
Clinical StudyASHGene Therapy
BALTIMORE, MD, USA I December 12, 2022 I Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery, development and commercialization of therapies using its controllable self-replicating RNA (c-srRNA) platform, today announced new data from a Phase 1/2 trial (NCT04211714) showing successful ex vivo telomere elongation with lead asset, EXG-34217 (autologous CD34+ hematopoietic stem cells that have been treated ex vivo with EXG-001), in a patient with dyskeratosis congenita, a telomere biology disorder (TBD) that can lead to bone marrow failure. In addition, the telomere flow-FISH study suggests the potential emergence of a cell population with longer telomeres in vivo. The data were presented in an oral presentation at the 64th American Society of Hematology (ASH) Annual Meeting, taking place December 10-13, 2022 in New Orleans, Louisiana.
“The encouraging data in this study represent the first time that ex vivo telomere elongation of CD34+ cells has been demonstrated in a patient with TBD. Dyskeratosis congenita has historically been characterized by a triad of abnormalities in the skin, nails and oral mucosa but can also include pulmonary and liver disease,” said Kasiani C. Myers, M.D., associate professor, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, and principal investigator in the study. “In addition, due to the shortened telomeres, most patients develop bone marrow failure, which requires the need for hematopoietic stem cell transplantation (HSCT), a procedure that is accompanied with potentially life-threatening complications. The data presented here provide promising early signs of the potential of EXG-34217 to provide an alternative treatment option for bone marrow failure in these patients and their families.”
Presentation Title: Successful ex vivo telomere elongation with EXG-001 in a patient with a dyskeratosis congenita
Session Name: Gene Therapies: Advances in Clinical Gene Therapy for Hematological Disorders
Publication Number: 781
Presenter: Kasiani C. Myers, M.D., Associate Professor, Department of Pediatrics, Cincinnati Children's Hospital Medical Center
Akihiro Ko, chief executive officer of Elixirgen Therapeutics, added, “We’re pleased to share these early positive data from our lead indication at a prestigious conference like ASH. This is a critical milestone in a process that started with the identification of ZSCAN4 by our Chief Scientific Officer, Dr. Minoru Ko, at the NIH. Our nuanced understanding of this protein has allowed us to create a novel potential treatment approach for TBD that is genotype and mutation independent. In addition, the absence of a conditioning regimen or immunosuppression is of significant importance in this radiation and chemotherapy sensitive population. We look forward to sharing additional data as we continue to enroll patients with our partners at Cincinnati Children's Hospital Medical Center.”
Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of therapies using its controllable self-replicating RNA (c-srRNA) platform. The company has two products in Phase 1/2 clinical trials (NCT04211714 and NCT04863131). For more information, visit ElixirgenTherapeutics.com.
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