LONDON – Evox Therapeutics Ltd. has validated its exosome drug delivery technologies in a $882 million deal with Takeda Pharmaceutical Co. Ltd., in which the partners will develop protein replacement and messengerRNA (mRNA) therapies in five rare disease indications. As an indicator of the potential therapeutic power of exosome delivery, the lead program in the collaboration rests on the ability of exosomes to cross the blood-brain barrier and deliver a correct copy of the NPC1 gene, to treat the inherited neurodegenerative disorder, Niemann-Pick type C.
The Rare Disease Day, which takes place at the end of February each year, is designed to focus global attention on the need for therapies to treat patients suffering from devastating rare diseases. The most recent event represented the 13th year it has been held. Over that period, research and development in the area has come a long way, and there are now 420 companies around the world that are active in developing regenerative medicines and advanced therapies for the treatment of rare diseases, according to a new report released by the Alliance for Regenerative Medicine (ARM).
Palvella Therapeutics Inc. has completed enrollment of its phase II/III trial for treating adults with pachyonychia congenita, a rare disease that eventually leads patients to a life of chronic pain and severe problems with walking.
HONG KONG – Beijing-headquartered Canbridge Pharmaceuticals Inc. has completed a $98 million series D financing round to target rare genetic diseases in China, and is considering an IPO after several rounds of financing to further pursue its goal.
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.
If you can’t see the wood for the trees the common sense response is to do a little thinning and let the light shine through. But for the bogged-down-in-bureaucracy European Medicines Agency (EMA), the response last week to the need to increase transparency and streamline its procedures was to set up an expert committee to investigate the activities and operations of its expert committees. I don’t imagine this is a cynical move by the recently installed head of EMA, Guido Rasi, to kick complaints about a lack of transparency and failure to listen to the needs of patients into the...