We are pleased to announce that we have published encouraging early data from our Phase 1/2 #ClinicalTrial evaluating our #GeneTherapy for the treatment of #TelomereBiologyDisorders (TBDs) in NEJM Evidence today. The study demonstrates the first-ever successful sustained telomere elongation and increased ANC in two patients with TBDs following treatment with EXG-34217, with no treatment-related adverse events observed. The data demonstrates the potential of our candidate as a therapeutic intervention for preventing or treating bone marrow failure for those with TBDs. We thank Dr. Kasiani Myers and Cincinnati Children's for their collaboration on this study. Learn more in our press release: https://bit.ly/4gUvylj
关于我们
Elixirgen Therapeutics is a clinical-stage RNA therapeutics company focused on the discovery and development of innovative therapies, including cell and gene therapies. The Company is advancing three technologies to develop therapeutics for a broad spectrum of diseases including rare and genetic diseases, and aging-related disorders. Bobcat mRNA? technology uses a proprietary mRNA design to deliver large payloads such as full-length dystrophin, which is mutated in Duchenne muscular dystrophy. Preclinical data have demonstrated restoration of muscular function in DMD model mice with no safety findings associated with administration or treatment. Controllable self-replicating (c-srRNA?) technology controls the powerful gene expression of self-replicating RNA to reduce off-target effects and improve efficacy. ZSCAN4 technology, delivered via the RNA-based Sendai virus, is currently in a Phase 1/2 clinical trial to extend telomeres in patients with telomere biology disorders (TBDs), a rare disease with significant unmet need. Initial data have demonstrated ex vivo telomere elongation in TBD patients for the first time ever and suggest the potential emergence of a cell population with longer telomeres in vivo. Elixirgen Therapeutics is located in the Science + Technology Park at Johns Hopkins in Baltimore.
- 网站
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https://ElixirgenTx.com/
Elixirgen Therapeutics, Inc.的外部链接
- 所属行业
- 生物技术
- 规模
- 11-50 人
- 总部
- BALTIMORE，Maryland
- 类型
- 私人持股
- 创立
- 2017
- 领域
- Therapeutics、Drug Development和Biotechnology
地点
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主要
获取路线
US，Maryland，BALTIMORE，21205
Elixirgen Therapeutics, Inc.员工
动态
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We’re happy to announce that the FDA has granted Orphan Drug Designation to our gene therapy for treatment of #Telomere Biology Disorders (#TBD). The FDA also recently granted EXG-34217 both Rare Pediatric Disease Designation and Regenerative Medicine Advanced Therapy (RMAT) Designation. We look forward to continuing the advancement of our program to address the significant unmet need in TBD. Read the full press release here:?https://lnkd.in/egtHdwmk
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We’re pleased to announce that the FDA has granted Regenerative medicine Advanced Therapy (RMAT) Designation to our gene therapy for treatment of #Telomere Biology Disorders (TBD). We look forward to continuing the advancement of our program to address the significant unmet need in TBD. Read the full press release here: https://lnkd.in/et4surw2
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In case you missed it, this Bioprocess Online article highlights how we're pioneering a decentralized approach to manufacturing and delivery, offering potential logistical and efficacy benefits in #CellTherapy. Read the article here: https://lnkd.in/g5-4Sbhn
How Elixirgen Manufactures Hematopoietic Stem Cells At The Bedside
bioprocessonline.com
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?? November is #TBDMonth! We're developing a therapy to treat #TelomereBiologyDisorders, a #RareDisease characterized by shortened #telomeres. Join us in raising awareness & supporting innovative solutions for those affected. Learn more from Team Telomere here: https://bit.ly/40oUc9d
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Did you know that while #DuchenneMuscularDystrophy is a rare muscle disorder, it is one of the most frequent genetic conditions, affecting 1 in 3,500 male births worldwide? At Elixirgen Therapeutics, we're exploring the potential of our Bobcat mRNA? in treatment for #DMD. Learn more here: https://bit.ly/4euOyXI
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#RNA plays a crucial role in biology and is a powerful tool in modern medicine. At Elixirgen Therapeutics, we are reimagining the power of RNA to develop various technologies for the treatment of #RareDiseases. Learn more here: https://bit.ly/3MZIFVK
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Living with a #TelomereBiologyDisorder can deeply impact patients and their families. As we work to develop a #CellTherapy for TBDs, we are proud to partner with Team Telomere, an organization that is fostering a strong patient community. Read some of the inspiring patient stories here: https://bit.ly/3XAIPYB
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We are pleased to announce that the FDA has granted Rare Pediatric Disease Designation to our candidate for the treatment of #DyskeratosisCongenita and related #TelomereBiologyDisorders. We look forward to working with the FDA as we continue the advancement of our program to address the unmet need in the TBD space. Read the full press release here: https://bit.ly/3XVi9mF
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