The U.S. Food and Drug Administration (FDA) has announced that they are adding staff and rolling out policy changes aimed at advancing the development of safe and effective cell and gene therapies. [1] The announcement came in the form of a press release on January 15th, citing that the new policies are a response to the current surge in cell and gene therapy products that the agency is handling. Based on the number of investigational new drug (IND) applications being submitted, the FDA projects a significant rise in the number of therapies that will be approved over the next few years:
HemaCare Corporation’s new global headquarters is officially open for business! The company started 2019 in a brand new 40,000 square foot space in a newly modernized 44-acre corporate campus located in the heart of the San Fernando Valley at Northridge. The design and construction of the cutting-edge facility provide a spacious workspace that promotes the well-being of its occupants, while significantly expanding the company’s on-site capabilities.
It’s getting cold out there! And while winter temperatures may have us yearning for a warm spot by the fireside, cold is a good thing for cell and gene therapies—in many cases, the colder the better. Logistics dictate that international shipping of cellular therapeutics often means freezing them first. Cryopreservation protects fragile cellular material from loss of viability or functionality during transit and storage, by halting chemical and biological degradation processes. To understand how important that is, it is worth noting that two of the hottest cell therapy drugs on the market this year (Yescarta® and Kymriah®) are both cryopreserved prior to infusion into the patient. But how cold does it really need to be to protect the efficacy of these cellular therapeutics? We decided to put that question into perspective.
Cryopreservation of therapeutic cells allows the long-term storage of functional cells and prevents degradative processes. Fresh cells are also used for cellular therapy approaches; however, there are many circumstances that make the availability of cryopreserved cells a must for repeated and on-hand use of cells. Cell cryopreservation provides a reliable stock of cells, which reduces the variability of cell therapy product. This translates to a more standardized product that can be used on a larger scale and without constraints on optimal availability for clinical use.
The leading cause of death for U.S. citizens is cardiovascular disease, which affects nearly a third of the U.S. population. There is an active and dedicated search for effective therapies to address this serious medical issue. The use of immunotherapy approaches for a number of diseases and conditions is continuously gaining momentum. Included in this is the use of CD34+ stem cells from a patients’ own blood (autologously derived) to treat cardiovascular disease. CD34+ is derived from bone marrow and other tissue types. However, CD34+ stems cells are more widely known for their hematopoietic origin.
