Two recent deals signaled to the industry that efforts to target previously undruggable RNA with small-molecule therapies may be moving from academic endeavor to fruitful application.

The turbulent financial markets that have seen the Dow Jones Industrial Average drop over 2% in August appear to have caught up with innovative mid-cap public companies engaged in exciting cancer research such as immuno-oncology. Up until now they have enjoyed strong investor support, but for the first time this year investors appear to be moving out of this sector and, as a result, share values have dipped dramatically. As a result, the BioWorld Cancer Index is trading down 11% in August. 

The turbulent financial markets that have seen the Dow Jones Industrial Average drop over 2% so far this month appear to have caught up with innovative mid-cap public companies engaged in exciting cancer research such as immuno-oncology. Up until now they have enjoyed strong investor support, but for the first time this year investors appear to be moving out of this sector and, as a result, share values have dipped dramatically. As a result, the BioWorld Cancer Index is trading down 11% in August. 

The costs associated with navigating a new therapeutic through the regulatory process to final approval and subsequent marketing continue to rise despite industry's collective efforts to speed up the process of drug discovery and development in order to rein in those burgeoning expenses.

According to a new report from the Alliance for Regenerative Medicine, there are a whopping 932 regenerative medicine companies worldwide that are in the process of developing 440 gene therapies, 587 cell therapies and 125 tissue engineering/biomaterials products.

It appeared that investors were far more interested in their vacations than in tracking the progress of blue-chip biopharma companies last month. Not helping their cause was the ongoing drug pricing debate taking place in Washington. The quest to reduce the cost of drugs moved a step closer with the Senate Finance Committee completing its markup of the Prescription Drug Pricing Reduction Act and voting 19-9 to send the legislation to the full U.S. Senate. 

“It’s a rare window into seeing how the FDA makes decisions because the proceedings are open unlike many of the other decisions they make,” Audrey Zhang, a medical student at the New York University School of Medicine, said on why she decided to embark on crunching the numbers from 376 votes by FDA advisory committee meetings from 2008 to 2015.

Every year at the Cleveland Clinic Medical Innovation Summit, the Top 10 list of technologies that are predicted to come to the market and change patient care is revealed. Last year's October event saw RNA-based therapies take the No. 10 spot. 

Despite the ups and downs of the general markets and a U.S. government shutdown at the beginning of the year that contributed to no biopharma companies graduating to the public ranks in January, the enthusiasm for biopharma IPOs has remained steady since then. In fact, a flurry of IPO listings on U.S. stock exchanges last month helped bring the total of those offerings to 30 at the halfway point in the year. 

Drugs for rare diseases now account for 31% of R&D pipelines, up from 18% in 2010 and just 11% in 2005, according to a report from the Tufts Center for the Study of Drug Development. That's currently nearly 3,500 drugs in development, more than double the 1,530 in 2010.