Commercialisation of Cell and Gene Therapies [Whitepaper]

Whitepaper Overview:

The global market for cell – based therapies is expected to surpass the $20 billion USD mark by 2025, with an annual growth rate of 21%. The main targets for cell – based therapies are high impact disease areas with significant unmet needs, including cancer, heart disease, neurodegenerative diseases, musculoskeletal disorders and autoimmune diseases.


Examples of the landmarks attained are: 1) In order to accelerate a cure for diabetes, Harvard Stem Cell Institute, and Semma Therapeutics developed a process to generate nearly unlimited numbers of beta cells for the clinic. 2) Vital Therapies enrolled First Patient in Phase III Liver-Treatment Trial, for company’s ELAD System, an extracorporeal human allogeneic cellular liver therapy (already tested successfully in over 250 patients). 3) PCT (Caladrius Biosciences, Inc. , NJ, US) enter an agreement with Kiadis Pharma (Netherlands) on engineering and process development for a Phase III trial of Kiadis’ lead product, ATIR101™, for the treatment of blood cancers, in US and Canada. 4) A broad collaboration agreement was established between Memorial Sloan-Kettering Cancer Center (MSK) and Seres Therapeutics, Inc. to develop microbiome therapeutics for hematopoietic stem cell transplantation (HSCT) and immuno-oncology treatment in patients with dysbiotic microbiome. The FDA has granted SER-109 (Seres’ most advanced program) ‘orphan drug’ and ‘breakthrough’ therapy designations. 5) Vital Therapies bio-therapeutic company enrolled first patient in phase III liver-treatment trial. The study will exploit company’s ELAD System, an extracorporeal human allogeneic cellular liver therapy. 6) SAB Biotherapeutics with the US Navy has successfully developed a transchromosomic (Tc) bovine platform technology to produce Human Polyclonal Antibodies against Ebola Zaire Virus.

Among the promises for future medicine and the delivery of solutions today we can mention: 7) Injecting conditioned, human adult stem cells, straight into the brains of chronic stroke subjects, showed not only safe but effective in reestablishing motor function. The phase-I trial was conducted at Stanford University School of Medicine. Lead researcher calls for new thinking regarding the permanence of brain damage. 8) At Duke University’s Brain Tumor Center, in a small clinical trial, patients with brain tumors, called glioblastoma multiforme (GBM), were treated with a non-pathogenic designer Poliovirus (with “breakthrough therapy” denomination by the FDA). Thanks to the virus-triggered immune response, survival of patients exceeded the 33 months.

Gene and Cell Therapy (G&CT) opportunities arise for tools and biotech manufacturing infrastructure: 9) Eppendorf will tackle the challenges of scaling-up human induced pluripotent stem cells (hiPSCs) as part of a Horizon 2020 project to develop cell-based heart therapies (coordinated by Scientists from the Hannover Medical School). 10) Cevec Biotech from Germany accomplishes next step for gene therapy construction of viral vectors and partners up in the US for scalable viral vector production with Paragon Bioservices. Cevec has succeeded in the manufacture of safe adenoviral (AV) gene therapy vectors with a proprietary super safe cell-expression system (CAP GT). 11) GE Healthcare and FedDev Ontario committed CAD $ 40M for a new Centre for Commercialisation of Regenerative Medicine (CCRM) to solve cell therapy manufacturing challenges. Finally, (12) Bio-Techne Corporation Acquires Access to B-MoGen Biotechnologies’ Gene Editing Technology. B-MoGen has a patent filled for a novel universal technique for fast, single-step purification of gene-edited cells. Moreover, it seems that the more straightforward use of MSC and allogenic technology with more than 350 clinical trials are poised to dominate the commercialisation of cell therapies. Further R&D in those is expected to bloom given the biologically based advantages.

As seen from the biotech industry, there is a dynamic balance of the global regulatory environment. Where does the tipping point/s lay? How does the Good Manufacturing Principles (GMP) in clinical trials play in that game? G&CT executives and other decision makers strive for a new therapy to succeed and another life to be saved. What is the cost-effectiveness rationale behind those scientific and business breakthroughs?


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