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Tuesday, 05/09/2023 1:46:38 PM

Tuesday, May 09, 2023 1:46:38 PM

Post# of 8922

Earlier this week, I had the honor of presenting to
@Capricor
and meeting with their CEO, Dr. Linda Marbaán. Being able to tour the lab and meet many of their scientific staff was nothing short of fascinating and informative.

Learning more about their CAP-1002 program was intriguing, as it has already shown many advantageous benefits (stimulating muscle tissue growth, retaining muscle function, decreasing inflammation, and preventing scarring) for patients with Duchenne muscular dystrophy in the clinic and is now in Phase III of development – meaning this drug is close to coming to market for patients. CAP-1002 is made up of CDCs (cardiosphere-derived cells or simply parts of donor heart cells) and holds the potential of being a meaningful treatment for DMD patients. I foresee this as a life-extending synergistic treatment that addresses the cardiac issues that patients with DMD unfortunately succumb to. It’s also noteworthy that this treatment should work for all DMD patients regardless of their mutation type, exon region, or antibody status.

Additionally, Capricor’s exosome program is very exciting as it has the potential to address the current gene-therapy and gene editing delivery problems associated with viral vectors, as a non-viral vector. These problems include not being able to redose patients (which is critically important for durability purposes) as they develop immunity to the viral vector, and patients already having preexisting antibodies to current viral vectors, making the vector ineffective. Having a vector that is safe, redose-able, and can deliver the genetic payload to its intended location will allow for great strides in gene therapy. Exosomes hold this potential and I’m excited to see where the data leads us.

Thank you, Capricor, for a spectacular day and for your contribution to the overall scientific community! #StayAmbitious



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