ZSCAN4

Bobcat mRNATM technology is a novel linear mRNA platform able to encode large proteins Data showed restoration of muscular function in DMD mouse model BALTIMORE, March 04, 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 presented preclinical data on its proprietary Bobcat mRNATM technology in a poster presentation at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place March 3-6, 2024, in Orlando, Florida. Bobcat mRNATM is designed to deliver large protein payloads (over 13 kb), and results show that a Bobcat mRNATM encoding a full-length dystrophin protein can restore muscular function in a mouse model for Duchenne muscular dystrophy (DMD). “We’re excited to be presenting these promising data on our novel Bobcat mRNATM technology. Dystrophin, the largest known human gene, is mutated in DMD, which is a rare disease that causes progressive muscle weakness and atrophy,” said Aki Ko, Co-Founder and Chief Executive Officer of Elixirgen Therapeutics. “Our Bobcat mRNATM full-length dystrophin approach may be complementary to others currently approved or in development, or may even mitigate some of their issues. We’re pleased to add the Bobcat mRNATM technology to our pipeline of RNA technologies and look forward to sharing updates in the months ahead.” Presentation Title:Development of mRNA Therapeutic Delivering Full-length Dystrophin for Duchenne Muscular Dystrophy Poster Number: T428 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: Slavena Salve Nissan, M.D. LifeSci Communications snissan@lifescicomms.com

No safety concerns were observed with EXG-5003 administration The data suggest a priming effect by EXG-5003 on the long-term cellular immunity of approved SARS-CoV-2 mRNA vaccines The Company is exploring the use of the c-srRNA platform for the development of vaccines, cell therapies and immunotherapies for several indications BALTIMORE, Oct. 16, 2023 (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 promising data from a Phase 1/2 trial (NCT04863131) evaluating EXG-5003, a SARS-CoV-2 vaccine developed with the Company’s temperature controllable self-replicating RNA (c-srRNA) technology. The data, published on medRxiv, demonstrate no safety concerns with EXG-5003 and induction of cellular (T cell-mediated) immunity. With clinical evidence that the c-srRNA platform is safe and can produce a T cell-mediated response, the Company is exploring its potential for the development of vaccines, cell therapies and immunotherapies for several indications. “Current RNA vaccines have demonstrated clear efficacy benefits, but challenges remain with the need for multiple booster doses due to neutralizing antibodies with limited cross-reactivity across variants,” said Yohei Doi, a Principal Investigator of this clinical trial and Professor of Fujita Health University School of Medicine and University of Pittsburgh School of Medicine. “Importantly, it’s been demonstrated that T cell-mediated immunity results in strong cross-reactivity across variants, but the T cell response by approved mRNA vaccines wanes rapidly. The results from this trial suggest a priming effect by EXG-5003 on the long-term cellular immunity of approved SARS-CoV-2 mRNA vaccines. Altogether, data show the unique and beneficial features of EXG-5003.” Akihiro Ko, chief executive officer of Elixirgen Therapeutics, added, “Our self-replicating RNA platform allows for expression to be temperature-controllable, which helps to improve efficacy while reducing off-target effects. EXG-5003 is active at around 33°C (skin temperature) but does not function at 37°C (core body temperature). This allows for the vaccine to be delivered intradermally, which is a method that’s been shown to induce strong T cell-mediated immunity. Additionally, EXG-5003 is delivered without lipid nanoparticles (LNPs), which may reduce local reactogenicity in the skin. We are pleased with the safety and immune data from this study and are exploring the potential of the c-srRNA platform for other indications.” A Phase 1/2 clinical trial of intradermal, controllable self-replicating RNA vaccine EXG-5003 against SARS-CoV-2 This was a double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety, tolerability and immunogenicity of EXG-5003.EXG-5003 is a vaccine that encodes the receptor binding domain (RBD) of SARS-CoV-2.Forty (40) healthy participants were enrolled in Cohort 1 (5 µg per dose, n = 16; placebo, n = 4) and Cohort 2 (25 µg per dose, n = 16; placebo, n = 4). The participants were followed for 12 months.No safety concerns were observed with EXG-5003 administration. It is noteworthy that the reactogenicity and systemic adverse events associated with EXG-5003 were much lower than the approved mRNA vaccines and other srRNA/saRNA vaccines.SARS-CoV-2 RBD antibody titers and neutralizing antibody titers were not elevated in either cohort by EXG-5003 alone.Elicitation of antigen-specific cellular immunity was observed in the EXG-5003 recipients in Cohort 2.At the 12-month follow-up, participants who had received an approved mRNA vaccine (BNT162b2 or mRNA-1273) >1 month after receiving the second dose of EXG-5003 showed higher cellular responses compared to equivalently vaccinated participants in the placebo group.Data suggest a priming effect by EXG-5003 on the long-term cellular immunity of approved SARS-CoV-2 mRNA vaccines. 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 trials. 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:Slavena Salve Nissan, M.D.LifeSci Communicationssnissan@lifescicomms.com718-483-4440

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