Promise of genome and cell therapies move closer to reality

June 16, 2016 Erin Righetti



Stem cell technologies and adoptive immunotherapies are big buzzwords in the cell therapies world, with the promise of personalized therapies driving interest. Boston is a hotbed for cell and gene therapies potential. STATnews reported today that “several Boston-area hospitals have teamed up with the Harvard Stem Cell Institute and biotech Semma Therapeutics to make personalized cell-based therapies and organize clinical trials.”

Boston companies are known for their innovation, and this is no less apparent in the cell therapies space. According to the article, there are many initiatives in Boston and across the country focused on manufacturing cell-derived treatments. The Boston initiative, called the Boston Autologous Islet Replacement Program, is focus on type 1 diabetes, and manufacturing the beta cells critical to reversing the effects of the disease. The body’s immune system is less likely to attack transplanted cells because they are derived from the patient’s own blood cells. Hurdles remain. Manufacturing cells is a very expensive endeavor, and the initiative, like all stem cell therapies companies, is eager to find funding.

Still, like a sci fi movie, these new technologies are poised to change genetic and rare disease treatment options as we know it, once the bumps get smoothed out. Despite challenges with delivery mechanisms, safety and precision of the technology, according to Andre Choulika, Chairman and CEO of Cellectis, as quoted in the Alliance for Regenerative Medicine’s (ARM) Annual Report 2015, says, “With more scientists working on gene editing around the globe, we will hopefully see more viable targets to go after, as there is a huge unmet medical need in genetically-based diseases.”

Want to know how to stake a claim in this paradigm-changing space? The Partnerships in Cell and Gene Therapies track at BioPharm America™ this September will highlight advances in CRISPR technologies, immunotherapies,  pathways to commercialization and the use of regenerative medicine in the treatment of rare diseases and cancer. Program offerings will soon be online. Find out more here.




About the Author

Erin Righetti

Editor-in-Chief, Insight

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