Utilizing transformative cell-based medicine for the treatment of Duchenne Muscular Dystrophy
Utilizing transformative cell-based medicine for the treatment of Duchenne Muscular Dystrophy
A NEXT GENERATION CELL THERAPY APPROACH
A NEXT GENERATION CELL THERAPY APPROACH
Develop a groundbreaking cell-based therapy program designed to treat Duchenne muscular dystrophy (DMD), the most severe form of muscular dystrophy, which results in muscle degeneration and premature death. Our innovative technology utilizes a non-viral vector system carrying the full dystrophin gene designed to treat DMD. Working in collaboration, our translational approach to product development is based on the strong research foundation provided by our key partnerships.
Develop a groundbreaking cell-based therapy program designed to treat Duchenne muscular dystrophy (DMD), the most severe form of muscular dystrophy, which results in muscle degeneration and premature death. Our innovative technology utilizes a non-viral vector system carrying the full dystrophin gene designed to treat DMD. Working in collaboration, our translational approach to product development is based on the strong research foundation provided by our key partnerships.
PROGRESS
OVERVIEW OF OUR WORK
Everything you need to know…
Pushing Limits
Addressing the limitations of current Duchenne Muscular Dystrophy (DMD) treatments with the next generation of DMD cell therapy technology.
Pushing Limits
Addressing the limitations of current Duchenne Muscular Dystrophy (DMD) treatments with the next generation of DMD cell therapy technology.
Pushing Limits
Addressing the limitations of current Duchenne Muscular Dystrophy (DMD) treatments with the next generation of DMD cell therapy technology.
Measurable Growth
A non-integrating, non-viral vector known as the hSynC—a human synthetic chromosome—has been utilized to deliver the entire DMD gene via induced pluripotent stem cells (iPSCs), enabling full dystrophin expression and offering the potential for a universal therapy applicable to all Duchenne muscular dystrophy (DMD) mutations. As a rational and tractable engineered multigene expression system, the DMD-hSynC also carries additional genomic elements, including safety switches and cell differentiation factors, enhancing its therapeutic versatility and safety profile.
Measurable Growth
A non-integrating, non-viral vector known as the hSynC—a human synthetic chromosome—has been utilized to deliver the entire DMD gene via induced pluripotent stem cells (iPSCs), enabling full dystrophin expression and offering the potential for a universal therapy applicable to all Duchenne muscular dystrophy (DMD) mutations. As a rational and tractable engineered multigene expression system, the DMD-hSynC also carries additional genomic elements, including safety switches and cell differentiation factors, enhancing its therapeutic versatility and safety profile.
Measurable Growth
A non-integrating, non-viral vector known as the hSynC—a human synthetic chromosome—has been utilized to deliver the entire DMD gene via induced pluripotent stem cells (iPSCs), enabling full dystrophin expression and offering the potential for a universal therapy applicable to all Duchenne muscular dystrophy (DMD) mutations. As a rational and tractable engineered multigene expression system, the DMD-hSynC also carries additional genomic elements, including safety switches and cell differentiation factors, enhancing its therapeutic versatility and safety profile.
Seamless Collaboration
Seeking co-development/out-license opportunities: Partnering with pharmaceutical, biotechnology, and patient advocacy groups to advance our DMD-hSynC program for the treatment of DMD.
Seamless Collaboration
Seeking co-development/out-license opportunities: Partnering with pharmaceutical, biotechnology, and patient advocacy groups to advance our DMD-hSynC program for the treatment of DMD.
Seamless Collaboration
Seeking co-development/out-license opportunities: Partnering with pharmaceutical, biotechnology, and patient advocacy groups to advance our DMD-hSynC program for the treatment of DMD.
PROCESS
How itWorks
Steps One through Six…
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STEP 3
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STEP 5
STEP 6
STEP 4
STEP 5
STEP 6
STEP 4
STEP 5
STEP 6
CONTACT
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Addressing the limitations of current Duchenne Muscular Dystrophy (DMD) treatments with the next generation of DMD cell therapy technology.
Looking for endless possibilities
Reach out anytime
Addressing the limitations of current Duchenne Muscular Dystrophy (DMD) treatments with the next generation of DMD cell therapy technology.
Looking for endless possibilities
Reach out anytime
Addressing the limitations of current Duchenne Muscular Dystrophy (DMD) treatments with the next generation of DMD cell therapy technology.
Looking for endless possibilities