BALTIMORE, Feb. 13, 2025 (GLOBE NEWSWIRE) -- Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on treating rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (TBDs). EXG-34217 is currently being studied in an ongoing Phase 1/2 trial (NCT04211714) for the treatment of patients with TBDs with bone marrow failure. Enrollment is currently open for participants aged 12 and older, regardless of gender or ethnicity.
RMAT designation, established under the 21st Century Cures Act, is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene and cell therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for that disease. A treatment with RMAT designation receives all the benefits of Fast Track and Breakthrough Therapy designation programs, including early interactions with the FDA, which may be used to discuss potential surrogate or intermediate endpoints to support accelerated approval.
About Telomere Biology Disorders and EXG-34217
Telomere biology disorders (TBDs) are a group of rare genetic conditions characterized by very short telomeres, the protective end caps of chromosomes that shorten with age. TBDs, such as dyskeratosis congenita, can lead to serious conditions including bone marrow failure due to severely affecting the ability of hematopoietic stem cells (HSCs) to produce blood cells. EXG-34217 is a dose of autologous CD34+ HSCs that have transiently expressed Zinc finger and SCAN domain containing protein 4 (ZSCAN4), a protein responsible for regulating telomere elongation and genome stability that can lengthen telomeres independently of telomerase. TBDs affect approximately 1 in 1 million people in the U.S.
About Elixirgen Therapeutics, Inc.
Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company focused on the treatment of rare diseases and aging-associated diseases using its ZSCAN4 technology and mRNA platform technologies. 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.
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