Recently there has been a significant resurgence in the use of gene and cell therapies thanks to improving efficiencies and safety of these methodologies. As we described in last week's issue of BioWorld Insight, with a number of gene therapies advancing into later-stage trials, the field has begun to attract significant amounts of both private and public capital investments once again.

Ariad Pharmaceuticals Inc. provided positive clinical data for Iclusig (ponatinib). The drug had a rocky start after receiving a December 2012 approval for use in patients with chronic, accelerated and blast phases of chronic myelogenous leukemia (CML) and Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) whose disease is resistant or intolerant to tyrosine kinase inhibitors (TKIs).

With a number of gene therapies advancing into later-stage trials the field has begun to attract significant amounts of both private and public capital investments once again. According to BioWorld Snapshots, more than $200 million in venture capital has been invested into companies developing gene therapies this year alone.

"Sell in May and go away" is an oft-quoted investment strategy for equities but it seems that biotech investors are bucking that trend big time. The BioWorld Blue Chip Biotech Index has jumped 5.2 percent in value for the month by the close of the markets last Thursday, thanks to stellar performances from many of the leading biotech companies in the group.

The concept is compellingly attractive: replace a faulty, disease-promulgating gene with a normal one and, voila, a cure should be initiated. This has been the hope for gene therapies for many years. Unfortunately, everything has not gone according to plan, and the technology fell out of favor until comparatively recently. Confidence is now returning and expectations are once again high that the gene therapy field can finally make good on its promise to transform 21st century medicine.

With the general markets sliding last week over concerns about an uncertain economy, biotech companies followed suit and by close of market Thursday, the BioWorld Blue Chip Biotech Index had dropped almost 1 percent since the start of the month. This was, however, slightly better than the 1.5 percent decline in the Nasdaq Composite Index over the same period, while the Dow Jones Industrial average has so far held its own and remained unchanged.

Despite the prevailing uncertain financial markets, the lure of going public has not diminished for biotech companies, with New Haven, Conn.-based Marinus Pharmaceuticals Inc. joining a list of 18 other companies currently lining up on the initial public offering (IPO) runway.

With first quarter earnings season in full swing investors have not been overly impressed with what biotech companies are reporting. As a result, the overall sector’s performance on the capital markets has dipped so far this month. There have been a number of stocks that beat earnings estimates but were still hit by selling.

There has been a great deal of discussion lately about productivity. Namely, the overarching theme is on how to bring new medicines to patients faster and at less cost. It’s no secret that R&D productivity has been challenged over the past few years despite the availability and implementation of technology designed to screen for promising molecules.