Ascendis Pharma A/S of Denmark has issued 6.9 million shares in the United States at $18 per share, generating net proceeds of $111.5 million. In early trading on 3 February 2015 the shares traded at $19.08 on the Nasdaq, representing a 6.0% premium.
The buzz at the Phacilitate Cell & Gene Therapy Forum in Washington DC on 26 to 28 January was that gene therapy will become a mainstream activity in the global pharmaceutical industry over the next five years, driven in part by the new gene-modified cell therapies and genome editing.
AstraZeneca Plc is to work with four research institutes in the UK and the US to apply a genome editing technology known as CRISPR across its drug discovery platform. The collaborations are expected to yield new drug targets in preclinical models of disease.
Canada-based EnGene Inc, which has developed a gene-based technology to treat autoimmune diseases, has raised $11.5 million in a Series B investment round. The funds will be used to advance the lead product which is intended to treat ulcerative colitis.
NightstaRx Ltd, a one-year old gene therapy company, has received £5 million in new venture finance to expedite the development of a treatment for choroideremia, an inherited cause of progressive blindness. This brings total funding for the company to £17 million.
Roche stepped up its acquisition activity in 2014 with the goal of broadening its pharmaceutical base and augmenting its diagnostics business with new gene sequencing technologies. Sales of oncology drugs were a key growth driver, but at least one late-stage cancer trial failed to show statistical superiority.
Novartis expects that group net sales will continue to grow this year despite an increase in generic competition for its off-patent medicines. Cost savings, and the completion of an asset swap with GlaxoSmithKline Plc that will narrow the company’s focus to three global businesses, is expected to drive profitability.
GlaxoSmithKline Plc said a Phase 3 trial of its candidate vaccine against the Ebola virus is set to start in Liberia in the coming weeks, subject to regulatory approval. The trial is being led by the US National Institutes of Health, which is a co-developer of the vaccine.
About 70% of the targets for approved medicinal products are proteins that are either secreted from cells or directed to the membranes cells while 30% of these proteins are widely distributed in the body, something that could explain the incidence of drug side effects, according to a Swedish study.
A new technique for culturing human liver and pancreatic stem cells could pave the way for improved therapies for treating both liver disease and pancreatic cancer. This is one of the conclusions from two studies published in the journal Cell on 15 January.