Nordic Life Science 1
retinal cells, is the first gene therapy for a ge
netic disease approved in both the U.S. and EU. “We hope the historic approval of Luxturna continues to serve as a catalyst for the field of gene therapy, improving our collective capabilities so that we may together, someday, conquer the realm of inherited disease,” said Katherine A. High, president and head of R&D at Spark Therapeutics after the approval. Another innovative cell and gene therapy company in Philadelphia is Imvax. The company’s initial objective is to define the treatment for Glioblastoma multiforme (GBM), the most common and lethal malignant brain tumor in adults. The company has demonstrated safety and proof of concept for an autologous cell vaccine in patients with recurrent and newly diagnosed GBM thus far, and its ongoing clinical trial serves to establish the recommended treatment regimen for future clinical trials. A third example is Adaptimmune, a company that has developed a cell therapy platform that engineers T-cells to target and destroy solid tumors. The company, who employs around 150 people in Philadelphia and 400 worldwide, has ongoing early clinical trials that address solid tumors, liver cancer and synovial sarcoma and the company plans to launch an inaugural commercial treatment for sarcoma patients in 2022. Other examples are Cabaletta Bio, aiming to treat autoimmune diseases, Carisma Therapeutics, engineering white blood cells to fight back against cancer, Parkinson’s and autoimmune diseases and Passage Bio, focusing on gene therapy for nervous-system diseases. One of the shifts in the sector over the past five to ten years has been the explosion of new businesses, according to Molineaux. While Philadelphia long has been home to large pharmaceutical companies, over the past decade, 58 NORDICLIFESCIENCE.ORG many of them had to downsize and assign tasks to other companies. “There has been enormous growth of start-ups founded by former pharmaceutical executives,” Molineaux explains. “They took their expertise with them. Between 2014 and 2019, there were 478 new life science companies in Pennsylvania. Now there are more than 2,800, and about half have ten employees or fewer. This is very promising for the growth of the life sciences ecosystem.” he start-up boom began when pharmaceutical pipelines were not keeping up with patent expirations, so some of the company functions were outsourced, according to Molineaux. “Processes that every pharmaceutical company followed, such as flow cytometry – a system used to screen cells to identify healthy and diseased cells to develop strategies to attack diseased cells – were outsourced. Johnson & Johnson opted to outsource flow cytometry, and the head of flow cytometry went out and started his own group, and was hired by Johnson & Johnson as a contractor.” For the past two or three years, one of the biggest challenges to developing the sector was lack of capital, but recently that has turned around, stated Molineaux in February. “In the last 18 months, there has been an opening of pocketbooks for investment in life science research, an influx of capital we haven’t seen in years.”