CRISPR

2017 was an exciting year for the cellular therapy industry and for Be The Match BioTherapies®. Hematopoietic stem cell transplants continue to be instrumental to both the past and future growth of the industry as a whole, and we’re energized by the new advancements, key stories and top trends that have shaped 2017. We recall a few of our favorites below, as we shift our focus and look forward to the year ahead. CAR-T—The biggest story of the year? No competition here. The FDA approval of tisgenlecleucel (Kymriah) to treat relapsed or refractory B-cell acute lymphoblastic leukemia in adults and children, and, fast on its heels, the approval of Gilead’s axicabtagene ciloleucel (Yescarta) to treat adults with certain types of large B-cell lymphoma. As the first genetically-modified autologous T-cell therapies to hit the market, Kymriah and Yescarta are game changers. A similar approach is now being tested in a range of disorders, including non-Hodgkin lymphoma, multiple myeloma and solid tumors, but expect to see it soon expand to autoimmune diseases and more in the coming months and years. The future is bright. CRISPR—Following the approval of Kymriah and Yescarta, the biotech industry is gearing up to create a new and improved generation of cell therapies, and version 2.0 aims to leverage the potential of CRISPR/Cas9. The gene editing technology promises to enable the development of a wide range of new therapeutic approaches, including more potent, more targeted CAR-T therapies, and researchers believe it could also be used to create so-called “off the shelf” versions of CAR-T from universal donor cells that may work for a broader range of patients. To this end, 2017 saw a number of collaborations emerge in the space, including between Mustang Bio and pioneering Harvard Stem Cell Institute scientist Chad Cowan; and between CRISPR Therapeutics and immuno-oncology researchers at Massachusetts General Hospital. We can’t wait to see the exciting new research that emerges from these partnerships. bluebird bio—It was a big year for gene therapy company bluebird bio, which is developing autologous gene therapies for adrenoleukodystrophy, β-thalassemia, and sickle cell disease, while also collaborating with Celgene to develop a BCMA-targeting CAR-T therapy for multiple myeloma. Known as bb2121, this therapy was granted accelerated review status by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency, shortly after the company reported outstanding results from its Phase 1 clinical study. Meanwhile, bluebird continues to position itself as a leader in the cell and gene therapy space. Amidst reports that manufacturing challenges are limiting the growth of the emerging CAR-T therapies, the company announced that it purchased a facility to be used for manufacturing the viral vectors used to produce its therapies. Expansion of gene therapy—Just this month, Spark Therapeutics planted their flag in new territory with the FDA approval of Luxturna, the first directly administered gene therapy in the U.S. targeting a disease that’s caused by specific gene mutations. The therapy is intended for patients with biallelic RPE65 mutation-associated retinal dystrophies, some of which can cause permanent blindness. Although approval of Luxturna came as no surprise to the industry, FDA Commissioner Scott Gottlieb still noted: “This milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases.” We’re excited about what’s next in the gene therapy realm. Partnerships—Given the promise, as well as the complexity, of cell therapy, it’s not surprising to see transplant medicine and cell therapy communities joining forces with biopharma companies. For biopharma companies, the transplant and cell therapy communities offer deep experience and expertise in the design and delivery of cell products, as well as established local and global donor inventories and networks that can help accelerate clinical development. Case in point? The recent collaboration agreement between Be the Match BioTherapies and Magenta Therapeutics, a Cambridge-based biotech developing therapies to improve and expand the use of curative stem cell transplantation. The digital revolution—Increasingly, digital tools are also revolutionizing drug development. This year, Be The Match BioTherapies announced the launch of MatchSource®, its new technology platform for the cell therapy supply chain, designed to minimize implementation risk for companies developing and delivering new autologous and allogeneic cell and gene therapies. The digital revolution is here to stay – we’re eager to see what new technologies emerge in 2018. Discover how, together, we can empower the discovery, development and delivery of next-generation therapies for patients.OUR SOLUTIONS

CRISPR

2017: cell therapy year-in-review