While many companies use viruses and viral vectors to deliver gene therapy and to modify cells for CAR T treatments, others have shunned adeno-associated viruses (AAV) and lentiviral vectors for other methods to deliver DNA and RNA into the cells.

"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.

“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.

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.

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.

In late May, Novartis AG's Avexis Inc. unit gained FDA approval for Zolgensma (onasemnogene neparvovec) to treat spinal muscular atrophy, and other companies are looking to follow suit developing drugs to treat a variety of diseases of the central nervous system (CNS).

Adding on to its $14 million series A round in April 2018, TFF Pharmaceuticals Inc. has secured another $8.17 million for the round to support development of its namesake thin film freezing (TFF) technology that was developed to increase the solubility of drugs.

Despite the strong growth in the number of drugs approved by the FDA over the last few years, clinical trial productivity fell 27% from 2013 to 2018, according to a report from the Iqvia Institute for Human Data Science.