Giving more punch to regenerative medicine and cell and gene therapies

By Anuj Kalsy

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Tissue and organ repair is a key goal of medicine, and many companies pursue this goal. By 2030, the regenerative medicine market is projected to nearly $38 billion, based on an annual growth rate of more than 15% per year. Much of this growth comes from advances in stem cells and the pathways around them.

In February, for example, researchers from the IRCCS Bambino Gesù Children’s Hospital in Rome reported about a Phase III clinical trial to add working copies of an altered gene to hematopoietic stem cells that can be used to treat some people with β-thalassemia who are dependent on transfusions. On August 17, the United States Food & Drug Administration (FDA) approved this treatment, called betibeglogene autotemcel, and described it as the “first cellular gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular transfusions of red blood cells”.

To better understand the rise of this type of research and the applications of human cells for this purpose, we spoke with Anuj Kalsy, scientific director of Precision for Medicine, Biological Sample Solutions Division.

Ready-to-use cells

“Human primary cells have been the key material for the research and production of cell therapies, but these cells have not always been easy to access,” explains Kalsy. “There is therefore an urgent need for suitable alternatives or viable, high-quality cell products, which can be used as powerful research tools by the R&D community in the life science industry.” To explore stem cells and a variety of immune system mechanisms, Precision for Medicine provides scientists with easy access to reliable, well-characterized cells.

For work in cell and gene therapies, stem cells, and regenerative medicine, Precision for Medicine offers a variety of leukopak products, which include specific cell types taken from a patient’s peripheral blood by a special form of apheresis. “Leukapheresis removes leukocytes — white blood cells — and returns the rest of the cells — granulocytes, platelets, and red blood cells — to the human donor,” Kalsy explains. Therefore, a leukopak provides a rich source of white blood cells – an average of around 73% in each leukopak. Of these cells, about 38% are CD8+ T cells and 18% are CD4+ T cells. “T cells have gained popularity in the cell and gene therapy markets due to many life science industry players focusing on the development of CAR-T cell therapies,” says Kalsy.

Precision for Medicine can provide fresh, cryopreserved and mobilized research-grade leucopaks that can be used in basic and clinical research. Mobilized leucopaks are made using FDA-approved granulocyte colony-stimulating factor (G-CSF) drugs, such as 5-day filgrastim, to mobilize and acquire the highest number of CD34+ stem cells.

“Leucopaks are the preferred choice in the life science industry for large-scale research and development efforts, especially early discovery and preclinical efforts, instead of the typical choice of PBMCs or blood mononuclear cells. device,” says Kalsy. “Research studies require consistency from trial to trial and large cell counts.” Leukopaks meet both of these requirements. For example, Kalsy notes that compared to whole blood products, “leukopheresis can provide up to 100 times more cells from the same donor than a blood draw.” Therefore, “leucopaks are gaining popularity in the cell and gene therapy niche, as they are an excellent source of CD4 and CD8 T cells, B cells, and natural killer or NK cells, all of which are used as therapeutic modalities. . to target human diseases,” says Kalsy.

Precision for Medicine continues to expand its line of leucopaks. For example, the company is developing leukopak enriched with CD34+ stem cells using in vivo mobilization procedures. These will be extremely useful to scientists studying the basic biology and potential therapeutic applications of these hematopoietic stem cells.

Selection of the best cells

For a specific project, Precision for Medicine can create custom leucopaks from people with certain HLA types, particular ethnicities, or even a specific disease. “The selection process involves proper screening and a pool of well-qualified human donors, and this is achieved through our professional staff, which consists of veterans trained by the American Red Cross, as well as our infrastructure which uses a modern and reference equipment for treatment. products of the highest quality,” says Kalsy.

Using research-only leucopaks, Precision for Medicine can acquire a customized collection of cells that meet a customer’s needs for cell type, optimal yield, viability and quality. “Our cell subset products are manufactured using industry standard reagents and qualified protocols, along with rigorous quality control analysis in the workflow,” Kalsy explains. “This ensures high product performance in the hands of our customers.”

To meet so many crucial criteria, Precision for Medicine oversees the entire process. “We have an end-to-end chain of custody across the entire workflow, and we collect these leucopaks from our in-house CLIA-certified blood donation and apheresis facility,” Kalsy says. Then the samples are sent directly to Precision for Medicine’s processing labs.

However, collecting the right cells is not enough. “Our leucopaks are prepared from well-characterized donors,” notes Kalsy. “We have a large database of recallable donors with different HLA types, and we collect demographic information, medical history and IRB consent.”

Explore new interests

As noted, many scientists are investigating treatments developed with CAR-T cells, but other immune cells are also gaining traction. One is NK cells, which Precision for Medicine can supply. “NK cell therapies have shown promising results in very early-stage oncology studies around the world,” says Kalsy. “Several companies are using NK cell therapies to enhance ADCC – antibody-dependent cellular cytotoxicity – in hematological and solid tumors.”

NK cell-based therapies could offer valuable benefits. “The manufacturing cost of NK cell therapies is much, much lower than a CAR-T cell therapy,” says Kalsy. “Some people in the research industry call this therapy ‘the rising star of cancer treatment’.”

Precision for Medicine provides the tools scientists need to find even more rising stars in healthcare. “We are always looking to provide innovative solutions to our life science customers,” says Kalsy. “We are constantly expanding the scope of cases and specimen types for all human diseases, including parallel cohorts of non-diseased or at-risk specimens, to provide scientists with one-stop service, from discovery to translation, to truly applying biomarker-driven healthcare.

Learn more about Precision for medicine and his leucopak offerings.

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