EXG-34217

BALTIMORE, Dec. 21, 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 the appointment of Jeffrey L. Cleland, Ph.D., to the Board of Directors to support the advancement of its technology in developing therapies for a broad spectrum of diseases including rare diseases, aging-related disorders and cancer. "We are pleased to welcome Dr. Cleland to our Board as we continue to design next-generation RNA therapeutics with our proprietary technologies. His decades of expertise across the scientific and financial aspects of the industry will be invaluable to our clinical and business development strategies," said Akihiro Ko, chief executive officer of Elixirgen Therapeutics. "We look forward to working with Dr. Cleland as we continue to advance the EXG-34217 program in telomere biology disorders with bone marrow failure and explore other ways our technology can be harnessed to develop therapies for patients in need.” Dr. Cleland has 30 years of industry experience and currently serves as chairman, president and chief executive officer of Ashvattha Therapeutics. He also serves on the boards of BIO, Exicure and Zylem, and holds advisory roles with small emerging biotechnology companies. Previously, Dr. Cleland worked at Genentech, Inc. for more than a decade, where he served in product development and manufacturing roles. He has also served as CEO of Versartis and Graybug Vision. He has held executive management positions at several companies and brings to the team experience in drug development and late-stage research, including the successful approval of two drugs, Herceptin® and Nutropin Depot®, as well as early work on Lucentis®, Avastin® and Kadcyla®. He has authored more than 100 articles and four books and holds several issued patents. Dr. Cleland earned a B.S. in chemical engineering from the University of California, Davis, and a Ph.D. in chemical engineering from the Massachusetts Institute of Technology. Dr. Cleland added, "I’m eager to join the Board as Elixirgen Therapeutics continues to demonstrate how their novel technology has the potential to be a key player in the rapidly-evolving RNA space. Designed with safety in mind and with several key benefits including better control of gene expression, the platform has the versatility to be used across multiple therapeutic areas. I look forward to working with the Elixirgen Therapeutics team at this pivotal time in the Company’s growth." 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

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