year in review Archives

For many in the field of cell and gene therapy, 2020 had a singular theme: COVID-19. Dozens of clinical and preclinical trials were launched in labs around the world to test the efficacy of cell therapies to target complications from COVID-19 infection. But while the pandemic drew most of the media attention, it wasn’t the only area of focus this year. After an initial stalling of research pipelines due to pandemic lockdowns, 2020 proceeded with a flurry of other key developments on everything from supply chain management to the health of the broader industry. Cornering cancer with CAR-T therapies Continuing a trend from recent years, exciting gains were made in cell therapies for treatment of cancer, with chimeric antigen receptor T-cell (CAR-T) therapies leading the way. In July, the FDA approved TecartusTM, the first CAR-T therapy designed to treat relapsed or refractory mantle cell lymphoma, an area of high unmet medical need. The approval represents the advancement of CAR-T therapy as a broadly applicable cancer treatment. It also made Kite Pharma the first company to have multiple approved CAR-T therapies. At the American Society of Hematology (ASH) annual meeting in December 2020, Johnson & Johnson reported results from its Phase 1b/2 CARTITUDE-1 study. The study found that 89% of patients with severe multiple myeloma treated with an experimental CAR-T therapy were alive one year later, and most were still responding to treatment. CAR-T therapies were also a topic of interest at The ONE Forum® 2020, which was hosted online by the National Marrow Donor Program®/Be The Match® in November. During the meeting, Carl June, MD, of the University of Pennsylvania shared an overview of recent advances in CAR-T cell therapy for treating hematologic malignancies. It has now been a decade since CAR-T cells were first used to treat leukemia. A follow-up session with one of the first patients treated by Dr. June’s team revealed that CAR-T cells still persist 10 years after treatment. Related content Transforming patient care through cell and gene therapy (blog post) Meanwhile, China continued to set the pace for CAR-T trials. According to clinicaltrials.gov, there were 357 CAR-T trials registered in China in mid-2020, the most of any country. Exuma Biotech CEO Gregory Frost told STAT in October that China’s advantage in biotech lies in its “industrial infrastructure, large scale manufacturing capability, and overall accessibility.” Still, Chinese researchers have identified a number of improvements that are needed to China’s CAR-T therapy pipeline, such as expanding trial sizes and strengthening oversight. China’s challenges were echoed across the global cell therapy landscape this year. For instance, patient wait times for treatment remained long as production was upended during the early days of the coronavirus pandemic. Throughout 2020, Be The Match BioTherapies® made Herculean efforts to enable the delivery of cell therapy treatments even as wait times stalled during the pandemic. In addition, our teams continued to support efforts to increase Medicare and Medicaid reimbursement rates to make these life-saving treatments more affordable. A year of unexpected growth in biotech investments Despite the cloud of uncertainty cast by COVID-19, investments in cell, gene and tissue-based therapies far exceeded expectations. In fact, financing hit a record high during the first three quarters of 2020, with biotech companies attracting $15.9 billion, surpassing the previous record of $13.5 billion in 2018. According to the Alliance for Regenerative Medicine, the bulk of this new funding was public financing to support development of cancer-related treatments. The investment helped fuel a surge in biotech innovation. Dozens of new companies were launched in the U.S. alone. Among the target research areas were therapies for the treatment of drug-resistant tumors, new gene-editing techniques and tools to trigger innate immune responses. One example: Atsena Therapeutics. The Durham, North Carolina-based startup that launched in December 2020 is pioneering new treatments for a leading cause of blindness. Another relative newcomer, Qihan Biotech, from Hangzhou, China, announced progress this year in the development of immunologically privileged human stem cells for allogeneic cell therapies. New funding also flowed into cell and gene therapies this year. Funding included a $110 million Series C funding round for Cambridge firm Generation Bio, which is developing a new generation of liver-targeted gene therapies, and $115 million in a second-round financing for neurological gene therapy research company Neurogene. Cell therapy supply chain improvements Last year also saw important developments in supply chain management and manufacturing, gaps that were laid bare by the coronavirus crisis. In November, Resilience Inc., backed by $800 million in fresh financing, pledged to “reimagine” how therapies are brought to market. One of the major challenges manufacturers like Resilience are up against is cold storage of cells, which require sub-Arctic temperatures for optimal performance. Contract manufacturing also came of age in 2020. COVID-19 made it clear how important service firms are to the increased scale and swift manufacturing of cell and gene therapies. Along the same lines, Be The Match BioTherapies continued to engage with our partners around the world in efforts to strengthen supply chains and starting material quality. In January 2020, we led discussions at the Phacilitate Leaders World conference to share supply chain best practices. We also announced a key partnership with Lonza in November 2020 to support our critical mission to standardize and streamline the cell therapy supply chain. As the severity of COVID-19 grew throughout the year, we also applied renewed industry focus to overcoming autologous cell therapy logistics challenges and scaling donor sourcing efforts. The elephant in the room While 2020 brought significant advancements in cell and gene therapies, the most prominent news story was the novel coronavirus – SARS-CoV-2 – which to date has infected over 100 million people and killed more than two million worldwide. In response to these tragic numbers, biotech firms around the world have launched or continued clinical trials to investigate how cell therapies can treat patients with severe COVID-19 complications. Some of the most encouraging gains were made in research of mesenchymal stem cells (MSCs). These multipotent stem cells derived from bone marrow and umbilical cords have shown promise initiating immune responses to tame the cytokine storm, one of the biggest drivers of COVID-19 mortality. Our partnership with NantKwest, announced in May, facilitates this field of research by providing donor material to support the company’s Phase 1b clinical trial of BM-Allo.MSC, which uses MSCs to reduce the lung inflammation associated with acute respiratory distress syndrome (ARDS). Although most MSC trials in 2020 involved older patients, some began assessing treatments for children and adolescents. Another promising development was in the use of cells from perinatal sources, such as cord blood. These sources which have been shown to eliminate many of the logistical challenges associated with sourcing starting material. For instance, biotech companies like Houston-based Cellenkos and Galway, Ireland-based Orbsen Therapeutics are engaged in trials to treat ARDS in COVID-19 patients with cells derived from cord blood, which has the added benefit of not being dependent on adult donors who need to travel to an apheresis center to donate their cells. Cellenkos announced encouraging early data from its initial studies in July 2020. COVID-19 impacted every aspect of the cell and gene therapy industry in 2020 – from donor collection to therapy production. No matter what 2021 brings, there is little doubt that 2020 is one for the history books – in our industry, and many others. Never miss a story like this one. Subscribe to our Cell Lines blog and get updates delivered to your inbox. Subscribe to the blog>

Advancements in cell and gene therapy