Spark Therapeutics Inc said it plans to seek marketing approval in the US next year for a gene therapy following a Phase 3 trial in which the experimental treatment helped restore vision to patients with an inherited eye disorder.
This year’s Nobel Prize in Psysiology or Medicine has been awarded to three scientists who together have developed therapies to treat parasitic diseases that disproportionately affect people in the developing world. The scientists are William Campbell, Satoshi Ōmura, and Youyou Tu.
Researchers at a hospital of the Technical University of Munich in cooperation with Stanford University of the US have developed an epigenetic approach to treating chemotherapy-resistant pancreatic cancer and lung cancer.
For more than a year, the European Medicines Agency (EMA) has been meeting individually with a number of companies that are developing products for difficult-to-treat diseases to speed the path towards registration. The discussions are non-binding, which means that regulatory approval is not assured. But they give both sides a chance to discuss the requirements of a good clinical programme, including the sort of information that will eventually be needed to show effectiveness in clinical practice.
Guido Rasi, a former head of the Italian Medicines Agency, has been nominated to lead the European Medicines Agency once again after a procedural challenge to his appointment in 2011 forced him to step down in November 2014.
The Swedish specialty pharmaceutical company Pharmalink AB has raised SEK 100 million (€10.5 million) to prepare for a Phase 3 development programme of its candidate product for primary IgA nephropathy, an inflammatory disease of the kidney.
The Wales Life Science Fund has led a €12 million Series B funding round for drug developer Apitope in order to enable to company to progress its pipeline of peptide-based therapeutics directed at autoimmune and allergic diseases.
The European Medicines Agency has given a positive opinion for a new Amgen Inc medicine that has shown an ability to kill or slow down the growth of multiple myeloma cells by blocking the cells’ proteasome.
Austria-based Nabriva Therapeutics AG has raised $92.25 million gross in an initial public offering (IPO) of its shares on the Nasdaq market to finance development of its portfolio of anti-infective medicines. Leerink Partners LLC and RBC Capital Markets LLC jointly managed the offering.
Replimune Ltd, a UK start-up, has raised $30 million in Series A finance to develop therapeutics based on the use of oncolytic viruses, or viruses that infect and destroy cancer cells. The funding round is being led by Atlas Venture with participation from Forbion Capital Partners and Omega Funds.