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.
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.
LONDON – Healx Ltd. has raised a $56 million series B after demonstrating its artificial intelligence-driven drug repurposing platform can systematically uncover treatments for rare diseases.
SUZHOU, China – The rare disease space continues to be an area of focus for across the sector worldwide, and China is no exception, with regulators rolling out long-awaited incentives and drugmakers viewing the field as a promising area to drive growth.
