On the heels of the approval of the first CAR-T therapies, last month saw a flood of news stories about exciting breakthroughs in the cell therapy space, from the remarkable, life-saving, genetically modified skin transplant of a young boy to the launch of a new company. Check out our favorite stories from the month of November:
Researchers at Cardiff University used CRISPR genome editing to develop a new method for boosting the cancer-destroying ability of T-cells. The scientists edited out non-cancer specific receptors of killer T-cells and replaced them with ones that would recognize and destroy cancer, an adjustment they believe will make immunotherapy treatments available to wider cohorts of patients. “The T-cells we made using genome editing do not have any of their own T-cell receptors left, and therefore the only receptor they can use is the one specific for cancer. As a result, these cells can be a thousand times better at seeing and killing cancer than the cells prepared using the current methodology,” said Dr. Mateusz Legut, who led the study, in a statement issued by the university. The preclinical research, funded by Cancer Research UK and the Wellcome trust, was published in Blood.
Flagship Pioneering announced the launch of Torque Therapeutics, a biotech promising to revolutionize the cell therapy space with highly targeted, next generation treatments. The company has three programs in its pipeline which aim to arm the immune system to target blood cancers and solid tumors. We’ll be following closely as the company progresses.
Meanwhile, the extraordinary story of a boy whose life was saved with a transplant of genetically modified skin cells took the internet by storm. In a Nature editorial, a group of European scientists reported that they had used stem cell therapy to grow replacement skin for a 7-year-old boy suffering from a serious, sometimes fatal genetic disease known as junctional epidemolysis bullosa (JEB), which causes severe skin lesions and blisters all over the body. At the time of the treatment two years ago, the boy had suffered “complete epidural loss” on about 60 percent of his body. He made a rapid recovery, and his doctors report that he is now living a normal life. Researchers believe this achievement could ultimately lead to new and revolutionary approaches to treating burn victims and others with skin diseases.
Also last month, The New York Times and several other industry news sources, including Genetic Engineering & Biotechnology News, reported on one of the key challenges facing companies developing cell and gene therapies: potential manufacturing capacity shortages. One particularly vexing issue, the Times notes, is the challenge of obtaining a key component of the CAR-T therapies – the disabled viruses used to deliver normal cells for the patient’s benefit. With CAR-T research booming, firms capable of creating these viruses are struggling to keep up with demand. But, companies are thinking creatively to solve manufacturing problems, as Endpoints reports – bluebird bio recently purchased its own manufacturing facility, and has partnered with a network of suppliers worldwide.
Speaking of bluebird bio, the company recently announced that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted its lead oncology program, bb2121, accelerated review status. Together with Celgene, Bluebird is evaluating bb2121, a CAR-T therapy targeting b-cell maturation antigen (BCMA), in a phase 1 trial of patients with relapsed or refractory multiple myeloma. Congrats, bluebird and Celgene