It was a big year for research industries.
Biotech. Pharma. Healthcare. Government. Every corner of the market was shaped by the rapid pace of research and development.
In genetic engineering, we saw the first U.S. approvals of two entirely new drug categories: CAR-T and gene therapy.
Major mergers and acquisitions signaled the changing landscape of healthcare and precision medicine. International players like CVS and Gilead made big moves just to stay ahead of the curve.
And finally, in the political realm, a change in administration prompted one of the largest demonstrations by the scientific community in the nation's history.
While its impossible to incorporate the vastness of this year's news into a single post, the following list provides a small glimpse of the tectonic shifts that accompanied 2017:
1. CRISPR expands to new frontiers: human gene-editing trials
Since 2015, when scientists in China first deployed the gene-editing technology known as CRISPR to edit a human embryo in a laboratory setting, there has been mixed opinions in the international scientific community over how the tool should be regulated. Fast forward to 2017, several gene-editing trials are now in the early phases with human patients, though most are still taking place in China. The principal U.S. policymakers — namely the National Academy of Sciences and National Academy of Medicine, have been more cautious with their recommendations. They have, however, issued a "yellow light" for U.S. researchers, ruling that clinical trials "might be permitted, but only following much more research."
2. FDA approves the first (and second) CAR-T therapy
Following years of research and development, the U.S. saw the first (and then second) approval for a genetically-modified immunotherapy known as CAR-T: chimeric antigen receptor, T-cell therapy. In short, the therapy works by taking immune cells from a patient, editing them in a laboratory, and then reinserting them to fight cancer. Because the cells originate from the patient, they can fight disease without the body attacking them first. CAR-T drew praise for its clinical success (up to 80% of patients cancer free after three months!) along with concern regarding its hefty price tag ($475,000 per patient!).
3. FDA approves the first viral gene therapy for rare vision disorder
If one new drug category wasn't enough, try another. In October, the FDA approved the first U.S. gene therapy in humans. The viral-based therapy targets a rare genetic mutation which causes major vision problems. While the concept of viral delivery of gene therapy has been discussed for decades, the approval was "a validation" long awaited by many researchers. It seems this may only be the start of larger efforts as well: viral vectors from some suppliers are on back order for up to two years following the announcement, Nature News reported.
4. Gilead acquires Kite Pharma for $11.9B
Things weren't looking all that great for pharmaceutical giant Gilead before August. The drugmaker's aging portfolio relied primarily on its blockbuster hepatitis C and HIV medications, which were facing increased market pressure and declining revenue. Then, they signed an $11.9B deal — the biggest in company history — to acquire the Los Angeles-based Kite Pharma, which subsequently won FDA approval for its CAR-T therapy, Yescarta. Now, the behemoth is positioned to redefine itself as a central player in the cancer therapy market for years to come.
5. Opioid crisis declared a public health emergency, but still lacks funding
Following record numbers of opioid deaths in the country in 2016 and the early months of 2017, the Trump administration took the formative step of declaring the crisis a public health emergency, a move that generally accompanies organized efforts by the federal government to combat such health disasters. While many saw it as a step in the right direction, the administration did not immediately allocate funds to support recovery efforts, drawing criticism from some officials that the move was, therefore, an empty gesture. The crisis rages on today with little relief in sight.
6. Denali Therapeutics tops the charts with $250M IPO
Neurological and neurodegenerative diseases have been notoriously difficult for drugmakers in recent years. The blood-brain barrier presents unique physiological challenges, while the progression of these diseases — slowly, over decades — means that treatments aren't often administered until late stages of the disease. All the more surprising then that the largest entry in the therapeutics market was Denali, whose portfolio includes two candidates with potential benefits for patients with Alzheimer's, Parkinson's, and ALS. The company netted $250M in its December IPO, breaking the 2017 record formally held by Ablynx. Denali was founded by a team of former Genentech scientists, which may have inspired confidence among investors despite early clinical issues with drug toxicity.
7. Congress looks to raise NIH budget, despite administration
When the Trump administration presented its initial budget proposal back in May, it called for a nearly 22% cut — $7.7B — in the NIH budget. That didn't sit well with the scientific community or Congress. All but a few lawmakers (on both sides of the aisle) support the work done by the research institutes, which is why its no surprise that both the Senate and House budget drafts defied the President's request and called for moderate increases to research funding. While the budget negotiations are ongoing (and still contentious), it looks like the institutes will be well situated moving into 2018.
8. CVS buys Aetna (as Amazon looms large)
The healthcare market moves like a tectonic plate — usually, so slow that you'd never notice. Then, once in a while, it unleashes an earthquake. The $70B purchase of Aetna by CVS was a 7.0 on the Richter scale and may signal more shifts on the horizon. The national retail chain has already taken steps to streamline pharmacy and primary care options for many customers, and signing the biggest health insurance deal in the nation's history shows they have no signs of slowing down anytime soon. However, as they break into new territory, they may face new entries — Amazon has not-so-quietly been positioning itself as a potential supplier of healthcare products and services.
9. Researchers defy the status quo with preprints
Journal reviews are a painfully slow and laborious process, though many researchers consider them integral to maintaining scientific integrity. For early career scientists, however, the push to publish quickly and get noticed has sparked a record number of preprint publications in 2017. While these works still only constitute a small fraction of the total literature, current trends suggest more and more researchers may look for alternative publication methods moving forward.
10. Scientists get up and march
The biggest science event of 2017 didn't involve breakthroughs, publications, or investors. On Earth Day, hundreds of thousands of researchers gathered in major American cities in the first-ever March for Science. Inspired largely by actions from the Trump administration to curb climate change policies and decrease government support for scientific programs, the marches garnered international media attention and inspired a new wave of activism within the scientific community.