Gene therapy may be the next revolution in healthcare. The opportunity to cure diseases through the modification of the genome was once considered science fiction, but no longer. The United States has seen a few approvals in this area, including the approval of voretigene neparvovec for a rare form of blindness. In addition, CAR-T agents, two of which are available, have demonstrated alternative ways to treat disease by manipulating genes of different cells.
Pharmaceutical companies have three targeted users: physicians, pharmacists, and patients. Various digital platforms are used to create a good customer experience for educating these end users. The core focus is on patients driving patient care, education, and adherence.
While traditional biotechnology focuses on material inventory, cell therapy products must account for the entirety of a patient’s journey in order to develop a robust supply strategy.
In today’s breakthrough medicines, personalization changes everything. Patients are used to receiving medications. Now, in the era of cell and gene therapies, patients are actually providing the key components to create them.
A quick and nimble approach is often sought after in the cell and gene therapy product space due to the likely one-time or limited patient dosing and/or rare disease conditions limiting the number of available patients, which reduces the number of clinical studies.
Nicole Gularte is currently battling cancer for the 8th time. The last time she had to do so, it seemed a CAR-T cell therapy trial might be the solution. This article is about her long battle with cancer, the struggle to get into a CAR-T cell trial, and the ups and downs she has faced along the way. Her story is an inspiration to others.
The regenerative medicine sector is at a remarkable moment. Transformative products are now on the market and accessible to greater numbers of patients every day. Dozens of additional therapies are in late stage studies. The regulatory and policy environment has evolved rapidly alongside the science, enabling a surge of incoming innovation.
A number of challenges are hindering efforts to develop cell and gene therapies to treat rare diseases. While these challenges may seem common across other drug markets, in the case of rare diseases, they are exacerbated by limited patient populations
The FDA’s RMAT and breakthrough designations have created a streamlined environment for product sponsors. To effectively leverage this regulatory policy environment, sponsors must conduct high-quality clinical trials that are often operationally complex. Clinical trial networks, capable of managing the array of regenerative medicine technologies, are well suited to manage this complexity.
Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?
