Capricor Therapeutics today announced that the U.S. Patent and Trademark Office has issued U.S. Patent 9,828,603, which includes composition of matter claims covering cardiosphere-derived cell exosomes. This patent, entitled “Exosomes and Micro-ribonucleic Acids for Tissue Regeneration,” is expected to run until at least 2033.
Exosomes are nano-sized, membrane-enclosed vesicles that are secreted by cells and contain bioactive molecules, including proteins, RNAs and microRNAs. They act as messengers to regulate cellular function. Their size, ease of crossing cell membranes and ability to communicate in native cellular language makes them an exciting class of potential therapeutic agents. CAP-2003 comprises exosomes isolated from the company’s proprietary cardiosphere-derived cells and is being developed as a potential next-generation therapeutic for diseases of fibrosis and inflammation.
“The issuance of this patent is an important milestone as Capricor plans to extend the scope of its clinical programs to the development of CAP-2003, which has demonstrated the ability to profoundly modulate the immune response, decrease inflammation, and stimulate cell growth in a variety of disease models,” said Linda Marbán, Ph.D., Capricor’s president and CEO. “Capricor plans to initially develop CAP-2003 for hypoplastic left heart syndrome, a congenital heart defect that affects about 960 newborns in the U.S. each year and is associated with high rates of mortality and heart failure secondary to the condition. Pending regulatory clearance, we expect to commence a clinical trial of CAP-2003 in patients with hypoplastic left heart syndrome in 2018.”
The therapeutic prospect of CAP-2003 in hypoplastic left heart syndrome (HLHS) is supported by preclinical studies in which this candidate was evaluated in a model of pressure-induced right ventricular dysfunction, representative of the disease present in many single ventricle congenital heart conditions such as HLHS. These studies, conducted by Sunjay Kaushal, M.D., associate professor of surgery and director of pediatric and adult congenital surgery at the University of Maryland, showed that, while untreated study animals developed right ventricular dilation and systolic dysfunction, intramyocardial injection of CAP-2003 significantly preserved right ventricular fractional area change (p=0.02 at one month of study follow-up).
U.S. Patent 9,828,603 is assigned to Cedars-Sinai Medical Center, which has granted Capricor worldwide rights to the technology under an exclusive license agreement.
Source – Capricor Therapeutics