Kristin Brooks, Managing Editor, Contract Pharma02.04.21
Capricor, a clinical-stage biotech company focused on developing cell- and exosome-based therapeutics for treating and preventing a variety of diseases, is developing two COVID-19 vaccines, marking the first time that exosomes (as opposed to liposomes) have ever been used to develop an mRNA vaccine.
Additionally, Capricor’s cardiac-derived cell therapy, CAP-1002, has treated several critically ill COVID compassionate care patients under the thesis that it can reduce inflammation related to cytokine storm.
Dr. Linda Marbán, President, Chief Executive Officer and Director of Capricor Therapeutics discusses the future of vaccines and biologics, as well as advances in responding to infectious diseases. –KB
Contract Pharma: What do you anticipate for the future of vaccines and biologics?
Dr. Linda Marbán: These are the most exciting times since the beginning of our understanding of infectious diseases, vaccines and biologics. The concept that we can take an mRNA and engineer it based on what we believe is an appropriate sequence to address a pathogen and thus, having the ability to create that sequence in such short order, suggests that we will be able to be very fluid in our response to infectious diseases in the future.
For instance, up until the current vaccine strategies for COVID-19, the flu strains are typically predicted out of Southeast Asia. As such, the flu vaccines would be built based on those predictions. However, if the virus happened to mutate along the way, the vaccine developed would only be partially effective or sometimes not effective at all, there was no ability to address that. Now, with this new technology of harnessing the power of mRNA, we will have the ability to quickly change for rapid adaptations, such as a change in the strain of these viruses.
CP: What are the key capabilities needed to support advancing biologic development and manufacturing?
LM: This will be one of the key issues we will be facing. We will need to build plants that are able to make mRNA on a sustainable, quality-controlled basis. We will have to be able to adequately package these types of biologics appropriately.
CP: With the advances in cell and gene therapies, what type of growth do you anticipate? Is the industry positioned to accommodate manufacturing?
LM: I think that we will continue to see growth in cell and gene therapies. There is now an understanding that we can take the power of a cell and use it to correct defects in human manufacturing. Therefore, we are in a position to start building larger plants, and quantify these quality systems to then be built more efficiently.
CP: What are the current challenges developing and manufacturing these therapies and where are advances being made?
LM: The biggest challenges are in the actual manufacturing of the biologics themselves. There are also packaging steps that must be done exactly right, and issues with lipid nanoparticles in current vaccines, which require very precise measurements, and building them so these doses are accurate and efficient. The net-net is that we must continue to build out quality systems in order to ensure the efficacy of these therapies.
Thankfully, the field is moving very rapidly and there is a push to identify and build out appropriate facilities.
CP: What are some of the latest technology platforms being leveraged for today's specialized drug products?
LM: The field of genetics has allowed for this type of expansion. We now understand what causes diseases and learned how to manufacture every component of that - from DNA to RNA, to regulatory suppressors. The technologies that are being developed are all of these bioreactors and plants that allow for biologics and data from human beings.
Dr. Linda Marbán is Capricor Therapeutics' President, CEO and Director. From 2003 to 2009, Dr. Marbán was with Excigen, Inc., a biotech start-up, where she was responsible for business development, operations, pre-clinical research and supervising the development of gene therapy products in a joint development agreement with Genzyme Corp. While at Excigen, she also negotiated a joint development and sublicense agreement with Medtronic Corp. utilizing Excigen’s technology. Dr. Marbán began her career in academic science, first at the Cleveland Clinic Foundation working on the biophysical properties of cardiac muscle.
Additionally, Capricor’s cardiac-derived cell therapy, CAP-1002, has treated several critically ill COVID compassionate care patients under the thesis that it can reduce inflammation related to cytokine storm.
Dr. Linda Marbán, President, Chief Executive Officer and Director of Capricor Therapeutics discusses the future of vaccines and biologics, as well as advances in responding to infectious diseases. –KB
Contract Pharma: What do you anticipate for the future of vaccines and biologics?
Dr. Linda Marbán: These are the most exciting times since the beginning of our understanding of infectious diseases, vaccines and biologics. The concept that we can take an mRNA and engineer it based on what we believe is an appropriate sequence to address a pathogen and thus, having the ability to create that sequence in such short order, suggests that we will be able to be very fluid in our response to infectious diseases in the future.
For instance, up until the current vaccine strategies for COVID-19, the flu strains are typically predicted out of Southeast Asia. As such, the flu vaccines would be built based on those predictions. However, if the virus happened to mutate along the way, the vaccine developed would only be partially effective or sometimes not effective at all, there was no ability to address that. Now, with this new technology of harnessing the power of mRNA, we will have the ability to quickly change for rapid adaptations, such as a change in the strain of these viruses.
CP: What are the key capabilities needed to support advancing biologic development and manufacturing?
LM: This will be one of the key issues we will be facing. We will need to build plants that are able to make mRNA on a sustainable, quality-controlled basis. We will have to be able to adequately package these types of biologics appropriately.
CP: With the advances in cell and gene therapies, what type of growth do you anticipate? Is the industry positioned to accommodate manufacturing?
LM: I think that we will continue to see growth in cell and gene therapies. There is now an understanding that we can take the power of a cell and use it to correct defects in human manufacturing. Therefore, we are in a position to start building larger plants, and quantify these quality systems to then be built more efficiently.
CP: What are the current challenges developing and manufacturing these therapies and where are advances being made?
LM: The biggest challenges are in the actual manufacturing of the biologics themselves. There are also packaging steps that must be done exactly right, and issues with lipid nanoparticles in current vaccines, which require very precise measurements, and building them so these doses are accurate and efficient. The net-net is that we must continue to build out quality systems in order to ensure the efficacy of these therapies.
Thankfully, the field is moving very rapidly and there is a push to identify and build out appropriate facilities.
CP: What are some of the latest technology platforms being leveraged for today's specialized drug products?
LM: The field of genetics has allowed for this type of expansion. We now understand what causes diseases and learned how to manufacture every component of that - from DNA to RNA, to regulatory suppressors. The technologies that are being developed are all of these bioreactors and plants that allow for biologics and data from human beings.
Dr. Linda Marbán is Capricor Therapeutics' President, CEO and Director. From 2003 to 2009, Dr. Marbán was with Excigen, Inc., a biotech start-up, where she was responsible for business development, operations, pre-clinical research and supervising the development of gene therapy products in a joint development agreement with Genzyme Corp. While at Excigen, she also negotiated a joint development and sublicense agreement with Medtronic Corp. utilizing Excigen’s technology. Dr. Marbán began her career in academic science, first at the Cleveland Clinic Foundation working on the biophysical properties of cardiac muscle.
