Dyskeratosis Congenita

Elixirgen Therapeutics is currently enrolling patients in its Phase 1/2 trial evaluating EXG-34217 in patients with telomere biology disorders at Cincinnati Children’s Hospital Medical CenterBALTIMORE, Sept. 25, 2024 (GLOBE NEWSWIRE) -- Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery, development and commercialization of therapies using its mRNA platforms, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to EXG-34217 for the treatment of patients with dyskeratosis congenita and related telomere biology disorders (DC/TBD). EXG-34217 is comprised of autologous CD34+ hematopoietic stem cells (HSCs) that have been treated ex vivo with EXG-001, a non-integrating, non-transmissible, temperature-sensitive Sendai virus vector encoding human ZSCAN4. The company is currently enrolling patients at Cincinnati Children’s Hospital Medical Center for a Phase 1/2 trial (NCT04211714). “We’re pleased with the FDA’s decision to grant EXG-34217 RPDD for the treatment of DC/TBD, a rare group of diseases that result in abnormally short telomeres that can lead to bone marrow failure and other serious health conditions,” said Aki Ko, chief executive officer of Elixirgen Therapeutics. “The RPDD is a recognition of the significant unmet need in this space and the potential of EXG-34217, and we look forward to working with the FDA as we continue to advance this program.” The FDA grants RPDD for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and primarily affect patients less than 18 years of age. If a marketing application for EXG-34217 for the treatment of DC/TBDs is approved by the FDA, Elixirgen Therapeutics may be eligible to receive a Priority Review Voucher (PRV) that could be used to receive accelerated review of any subsequent application. About Elixirgen Therapeutics, Inc. Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of therapies using its mRNA platforms and ZSCAN4 technology. The company has one ongoing (NCT04211714) and one completed (NCT04863131) Phase 1/2 clinical trial. For more information, visit ElixirgenTx.com. Forward-Looking Statements This press release may contain "forward-looking" statements. Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in pharmaceutical research and development. Any forward-looking statements in this press release speak only as of the date of this press release, and Elixirgen Therapeutics undertakes no obligation to update or revise the statements in the future, even if new information becomes available. Contact Media Relations:Madelin Hawtin LifeSci Communicationsmhawtin@lifescicomms.com

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 Key Findings: 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.” About Elixirgen Therapeutics, Inc. 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. SOURCE: Elixirgen Therapeutics

Treatment with EXG-34217 increased the telomere length of CD34+ cells by 1.24-fold ex vivo Telomere flow-FISH study suggests the potential emergence of a cell population with longer telomeres in vivo No acute or long-term adverse effects were observed for 9 months BALTIMORE, Dec. 12, 2022 (GLOBE NEWSWIRE) -- 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 Key Findings: Zinc finger and SCAN domain containing protein 4 (ZSCAN4) is a protein that regulates telomere elongation and enhances genome stability. EXG-001 is a temperature-sensitive Sendai virus vector encoding for human ZSCAN4, which is non-integrating and non-transmissible. EXG-34217 is a dose of autologous CD34+ hematopoietic stem cells (HSCs) that have been treated ex vivo with EXG-001. In this Phase 1/2 trial, 2.8x106 CD34/kg were collected and treated with EXG-001 ex vivo in a functionally closed tubing system using the CliniMACS Prodigy (Miltenyi Biotec). Treatment increased the telomere length of the CD34+ cells by 1.24-fold, bringing them into the healthy control range. 0.85x106 CD34/kg autologous treated HSC (EXG-34217) were reinfused into the subject in February 2022 without a conditioning regimen. The telomere flow-FISH study suggests the potential emergence of a cell population with longer telomeres in vivo. No acute or long-term adverse effects were observed up to 9 months, and the differentiation potential of the CD34+ cells did not change after treatment. 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.” About Elixirgen Therapeutics, Inc. 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. Forward-Looking Statements This press release may contain "forward-looking" statements. Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in pharmaceutical research and development. Any forward-looking statements in this press release speak only as of the date of this press release, and Elixirgen Therapeutics undertakes no obligation to update or revise the statements in the future, even if new information becomes available. Contact Media Relations: Slavena Salve Nissan, M.D. LifeSci Communications snissan@lifescicomms.com 718-483-4440