The US biotech’s lead candidate missed the mark in a Phase III hospital-related pneumonia trial due to low recruitment affected by macroeconomic headwinds but analysts retain high hopes for another upcoming study.

Private Company Edition: 5AM revealed two funds totaling $450m and $300m, V-Bio raised a €110m venture capital fund and AXA IM Alts committed $500m for health care private equity deals. Also, TRIANA launched with $110m and the AMR Action Fund backs Adaptive Phage Therapeutics.

After a decade of doubts, fears, and scares, advances in gene therapy have brought one drug close to approval, and a few venture-backed start-ups are gaining momentum. Is it enough to persuade more VCs to jump in?

When the European Medicines Agency (EMA) declined to approve Amsterdam Molecular Therapeutics’ (AMT’s) Glybera (alipogene tiparvovec) for the rare genetic disease lipoprotein lipase deficiency (LPDL) back in June (scripintelligence.com, 24 June 2011), the CEO of AMT was ‘convinced and confident’ of the approval of Europe’s first gene therapy within five months. At the end of this week, AMT completed the first, and easiest part of that process by filing an appeal against the EMA’s rejection and stated that they expected that the re-examination of the dossier would be completed by the end of 2011 (scripintelligence.com, 8 July 2011). Behind the backdrop of the appeal for re-examination which, history has shown does not have a high chance of success, the AMT annual report for 2010 reveals that the regulatory path for Glybera has been far from smooth.