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. These concepts will be discussed in much more detail at the 2019 PDA Cell and Gene Therapy Conference. The conference will provide insight on process development, supply chain, validation, new advances in gene therapy, the patient experience and more!
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.
Immuno-oncology is a unique approach to cancer treatment that leverages the body’s immune system to help fight cancer. In recent years, immune checkpoint inhibitors have changed the landscape of immunotherapy, and emerging therapies such as chimeric antigen receptor T-cells (CAR-T), dendritic cell vaccines and bi-specific T-cell engager (BiTE) antibodies are pushing the envelope even further.
Today’s competitive oncology drug development space is evolving rapidly, with high stakes for sponsors and participants alike. An increasing number of novel new oncology drugs are in development—precision medicines and immunotherapies such as immune checkpoint inhibitors, CAR-T cell therapies, and oncolytic viruses—that leverage genomic insights. Many more applications than in the past are also qualifying for the FDA’s expedited approval programs. Needless to say, in this race for novel drug approvals, the complexity of oncology trial design and execution has increased dramatically.
The recent FDA approval of Sarepta’s eteplirsen (Exondys 51), while generally welcomed in the Duchenne muscular dystrophy (DMD) community, was not without controversy. Eteplirsen is a gene therapy drug for use in DMD patients whose dystrophin mutation is amenable to exon 51 skipping. It was approved after a study of 12 patients, four of whom received placebo for 24 weeks before being re-randomized to one of two dosages. Questions have arisen regarding the use of historical data as a control in the absence of a concurrent placebo arm of the latter part of the study, the efficacy measures used, and the use of post-hoc analyses conducted by the study sponsors. But in the end, Eteplirsen was approved by the FDA, and the process, regardless of attendant controversy, illustrated the potential for innovation in clinical trial design and methods of analysis.
Novel therapeutics such as gene and cell therapies, nanoparticles, and combination products requiring targeted delivery, novel treatments for wound healing, cardiovascular disease, and bone regeneration, and new medical device technologies call for innovative surgical procedures in preclinical trials to determine safety and efficacy.
Immunotherapies harness the power of the body’s own defense mechanism, the immune system, to combat disease. They were initially introduced in the form of non cell-based biologics and vaccines, such as the splurge of products known as the check-point inhibitors designed to target the PD1/PD-L1 immune pathway. An individual would be injected with a weakened form of a virus, exposing the body to the disease and prompting the immune system to produce antibodies to fight the infection from the live virus.