Developing a groundbreaking cell-based therapy
targeting Duchenne Muscular Dystrophy.
About EIRx
Why you should know about us.
Developing a groundbreaking cell-based therapy targeting Duchenne Muscular Dystrophy, the most severe form of muscular dystrophy characterized by muscle degeneration and premature mortality. The company’s non-viral vector system is designed to deliver the full-length dystrophin gene, representing a rational, scalable approach to restoring functional dystrophin expression in affected muscle t issue.
Overview of DMD:
Individuals with DMD lack or produce insufficient dystrophin, leading to muscle cell damage and replacement with fat and fibrotic tissue over time. However, innovative therapies targeting the underlying genetic cause offer hope for improved outcomes.
These advancements hold promise for enhancing the prognosis and quality of life for individuals affected by DMD, offering newfound optimism for managing this debilitating condition.
1 in 3500 to 5000 males
Affected by DMD
Ages 3-5
Symptoms will first appear
Age 12
Many patients will need wheelchair assistance.
$18.1B
Market evaluation by 2030
https://www.coherentmarketinsights.com
Our Science
A Pre-clinical Cell Therapy Company with Advanced Capabilities.
• Caused by a variety of mutations in the dystrophin gene
• No definitive cure exists
• Current treatments have safety issues and limited efficacy
Stages
Current Landscape
Limitations in Current DMD Therapies.
Situations to address
Cell Engineering System to Address Current Treatment Limitations.
Licensed from CG Bioengineering (CGB), EIRx is Utilizing The Human Synthetic Chromosome* (hSynC)
AAV vector
May integrate into host genome
Viral/potential immune response
Limited repeated dosing
Therapeutic efficacy influenced by vector instability
hSync - Synthetic Chromosome
Carrying capacity of >300,000 base pairs
Non-integrating (safety)
Non-viral, stable
Safety, does not require repeated reintroduction
Stable gene expression
Moving forward
The Next Generation of Duchenne Muscular Dystrophy (DMD) Cell Therapy.
Demonstrated ability to carry entire DMD gene for full dystrophin (Dp427) for muscle fiber integrity AND DMD isomer (Dp71ab) for muscle satellite cell activation and proliferation
Validated expression of DMD gene (Dp427) from hSynC in vivo
Universal therapy for all dystrophin mutations
Does not require immunosuppression
Non-viral delivery
DMD is a space with great untapped potential, a fact underlined by the limited available product
it’s gratifying to see something approved after all these years, the current system is not perfect by any means
An example of a potential strategy would be to use stem cells carrying human artificial chromosomes containing multiple cDNAs encoding the missing protein Berry SE (2015) Stem Cells Translational Medicine 4:91-98
Partnership
The leaders in the development of novel hSynC-based cell therapies and seek to create transformative treatments for unaddressed or under-addressed human genetic diseases.
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Community Outreach
Learn More About Muscular Dystropy