Reimagine the Power of RNA
We develop life-changing therapies for patients in need using our cutting-edge RNA technologies.
- Our Bobcat mRNA™ technology uses a proprietary mRNA design to deliver large payloads such as full-length dystrophin, which is mutated in Duchenne muscular dystrophy.
- Our controllable self-replicating (c-srRNA™) technology controls the powerful gene expression of self-replicating RNA to reduce off-target effects and improve efficacy.
- Our ZSCAN4 technology, delivered via the RNA-based Sendai virus, is currently in clinical trials to extend telomeres in patients with telomere biology disorders, a rare disease with significant unmet need.
Bobcat mRNA™ overcomes existing vector payload size limits
Existing gene therapy vectors face payload size limits limits (e.g., the adeno-associated virus (AAV) can only carry a maximum payload of up to 4.7 kb). The proprietary design of Bobcat mRNA™ allows our mRNA to carry payloads over 13 kb on a single strand, opening the door to treating several target indications with an unmet need.
Lead Indication: Duchenne Muscular Dystrophy (DMD)
- People with DMD have mutations in dystrophin (11 kb), the largest gene in the human genome, leading to progressive muscle degeneration.
- Due to dystrophin’s large size, DMD therapies currently under development focus on delivering or restoring an incomplete dystrophin.
- Bobcat mRNA™ can encode full-length, normal dystrophin in a single mRNA strand, which may lead to improved stability and efficacy.
- Preclinical data has demonstrated restoration of muscular function in DMD model mice with no safety findings associated with administration or treatment.
Future Therapeutic Candidates:
- Future therapeutic candidates using Bobcat mRNA™ may address diseases involving other large genes including Ehlers-Danlos disease, Stargardt disease, von Willebrand disease, and Hemophilia A.
- Other future candidates using Bobcat mRNA™ may include long-acting mRNA therapeutics for targets such as GLP-1, FGF21, EPO, and G-CSF.
c-srRNA™ (controllable self-replicating RNA) controls the powerful expression of self-replicating RNA
Challenges in uncontrolled gene expression can limit the potential of self-replicating RNA
- Being a self-replicating RNA variant, c-srRNA allows for elevated and extended expression compared to traditional mRNA.
- However, c-srRNA has an “off switch” to control gene expression and prevent off-target effects.
Future Therapeutic Candidates:
c-srRNA is being investigated for use across multiple therapeutic targets.
ZSCAN4 extends telomeres in stem cells
ZSCAN4’s features were originally discovered at the National Institute on Aging (NIH) by our Chief Scientific Officer, Minoru Ko, and his team. ZSCAN4 extends the telomeres of stem cells through a telomerase-independent mechanism and potentially has additional therapeutic features.
Lead Indication: Telomere Biology Disorders (TBDs)
- People with TBDs have abnormally short telomeres, which are the protective end-caps of DNA.
- They are born with or develop short telomeres and have accelerated shortening, which can lead to serious conditions, such as bone marrow failure.
- The existing treatment, allogeneic hematopoietic stem cell transplant (HSCT), has potential issues such as immune reactions and an increased cancer risk.
- Our Phase I/II clinical trial (NCT04211714) is in progress at Cincinnati Children’s Hospital Medical Center to study ZSCAN4 delivered by the RNA-based Sendai virus as an autologous cell therapy in TBDs.
- Initial data has demonstrated evidence of ex vivo telomere elongation in TBD patients for the first time ever and suggests the potential emergence of a cell population with longer telomeres in vivo.
Future Therapeutic Candidates
Future candidates using ZSCAN4 may address diseases such as myelodysplastic syndromes (MDS), idiopathic pulmonary fibrosis (IPF), and aging-related diseases.
A Pipeline With Expansive Potential
We develop life-changing therapies for patients in need using our cutting-edge RNA technologies.
Bobcat mRNA™
c-srRNA™
ZSCAN4
LEADERSHIP
Our team comprises determined and experienced leaders who have a history of building companies and seeing candidates through commercialization.
Executive Management
Board of Directors
& Co-Founder, Board Chairman
& Co-Founder, Board Member
Careers
Interested in joining the best and the brightest in the industry as we charter the development of the next-generation of cell therapies using advanced RNA technology? Join us.
We are actively looking for new members to join our incredible team. We are located in the Science + Technology Park at Johns Hopkins in Baltimore, an ideal research environment that allows our scientists to conduct research at state-of-the-art facilities.
Interested in learning more? We’d love to hear from you –info@elixirgentherapeutics.com