Curis [NASDAQ:CRIS]

Richard Hu

In our exciting and ever-changing industry, pharmaceutical and biotech companies must continue innovating not only their technologies but also the way they do business. Let’s review the top three trends that are “hot” in the eyes of a formulation scientist

The first thing that came to mind is the advancement of CRISPR/Cas9, a gene editing technology developed by Professor Zhang at MIT. Inspired by how living cells disable invading viruses, CRISPR (Clustered Regularly Short Palindromic Repeats) technology utilizes Cas9, a nuclease enzyme, to cut DNA strands at a precise location when the target gene is complexed with a guide-RNA. As a result, scientists can disable certain sequence in that gene and understand the function of that particular sequence. Sequentially, CRISPR also enables scientists to add a desired function to the gene by inserting a pre-fabricated sequence. We all know that genes define who we are as life-forms and individuals. There is no doubt that our genes have a profound effect on our health. While still in its infancy, gene editing certainly has the potential to completely transform not only our industry but our world as well. If something can finally bankrupt our social security, I think gene editing will be it.

For cancer patients, chemotherapy and radiation therapy have long been the only options. Recent development of immuno-oncology has changed the game. In this new hope, scientists are developing drugs that can stimulate our own immune system to recognize and fight cancerous cells. One such advancement is the chimeric antigen receptor (CAR) T-cell technology, in which a patient’s own immune cells are removed, engineered to have the ability to recognize and attack cancerous cells, then infused back to his/her body to fight cancer. While only approved to treat limited types of cancer and may have severe side effects, the approval of Kymriah^® and Yescarta^® clearly represents the future of personalized medicine.

Another trend worth mentioning in immunotherapy is the advancement of checkpoint inhibitor. These drugs are often highly targeted, highly efficient and have relatively low side effects. In recent years, antibody-based checkpoint inhibitors have dramatically improved the management of cancer. To me, developing small molecule version of such inhibitors, which are orally available and going to be much cheaper, is even more exciting.

In the foreseeable future, biopharmaceutical companies, regardless of their sizes, will continue to utilize R&D outsourcing to access innovations and expertise. According to Visiongain, the global drug discovery outsourcing market is expected to exceed $40 billion by 2026. Meanwhile, the tide of outsourcing model is certainly shifting, especially at early stage. R&D work is coming back to the States, while some companies start to run clinical trials in developing countries, often-times looking for treatment-naïve patients. I also sense a strong focus from pharmaceutical companies on partnering with academia. In the past few years, several major pharma companies moved their R&D headquarters into the most congested neighborhoods in Boston, with the intention to put their folks into the proximity of Harvard and MIT. But for God’s sake, put them in the suburb and take a subway to the “Inspiration” when needed. Haven’t they checked out the Boston morning traffic in last twenty years?