Blog | HemaCare

An independent publication cites the use of bone marrow and mobilized peripheral blood sourced from HemaCare in a study aimed at improving the efficiency of resource consumption in gene therapy.

The publication, published in the journal Molecular Therapy, focuses on the use of lentiviral vector (LV) to introduce corrected versions of defective genes into stem cells. The goal of gene therapy is to transplant these stem cells into people suffering from genetic disease, in hopes of restoring the function of the faulty gene. One of the main challenges in this clinical strategy is the cost; substantial amounts of LV are needed to modify a therapeutically effective dose of stem cells.

“As the industry and regulatory landscape continues to evolve, it’s vital that as an industry, we take a lifecycle approach to risk management –to ensure that we mitigate as much risk as possible all the way from discovery through to commercialization.” - Dr. Dominic Clarke, Global Head of Cell Therapy, HemaCare

Cell and Gene Therapy Insights has just published HemaCare’s white paper on how to manage starting material quality and stability to maximum effect during cell therapy manufacturing. ^[1] Quality cell therapies can only be created from quality starting materials, yet paradoxically, starting materials are the single greatest source of variability in the cell therapy manufacturing process. Mitigating risk requires minimizing that variability, and also managing shelf-life limitations and biopreservation logistics.

In an independent publication ^[1] researchers at UCLA cited using leukopaks obtained from HemaCare in order to investigate a new gene editing method.

The group is researching strategies to treat genetic blood disorders such as sickle cell anemia. While there has been a great deal of research focused on using CRISPR/Cas9 gene therapy to treat these illnesses ^[2], current strategies for gene editing in hematopoietic stem cells are inefficient, and thus costly.

An independent publication in Nature: Scientific Reports cites using fresh leukopaks sourced from HemaCare in their T cell cryopreservation study. The authors, who work at the Cell and Gene Therapy Catapult in London, are studying the impact of cooling and thawing rates on cryopreserved human peripheral blood-derived T cells. In order for the emerging cell and gene therapy industry to effectively ship and store cell-based therapies to global markets, it will be necessary to cryopreserve cell therapy starting materials, as well as the final therapeutic products. To retain peak cellular function, and therefore therapeutic efficacy upon patient administration, it is necessary to understand how to optimize the cryopreservation process.

Melanoma is the most serious form of skin cancer and more likely to grow and metastasize. It has a high response rate to checkpoint inhibitor therapy compared to other cancers; however, about 60% of patients treated do not respond well or relapse. Immune checkpoints are proteins expressed on T cells and function to ensure self-tolerance, but they are also used by tumor cells to limit anti-tumor immune function.