Astrazeneca plc’s Alexion rare diseases unit has taken an option to buy all remaining equity in Caelum Biosciences Inc., adding a rare disease drug to its pipeline in a deal worth $500 million. At the center of the deal is CAEL-101, a potentially first-in-class monoclonal antibody designed to tackle the toxic amyloid deposits that build up in organs of people with the rare disease light chain amyloidosis.
HONG KONG – Canbridge Pharmaceuticals Inc. signed a collaboration and licensing agreement that could be worth $581 million, gaining global rights to develop, manufacture and commercialize gene therapy candidates from Logicbio Therapeutics Inc. for the treatment of Fabry and Pompe diseases. The candidates are based on Logicbio’s adeno-associated virus (AAV) sL65, the first produced from its Saavy capsid development platform.
BEIJING – Rare disease specialist Canbridge Pharmaceuticals Inc., of Beijing, said it is collaborating with the Horae Gene Therapy Center at the University of Massachusetts (UMass) Medical School to conduct gene therapy research with a focus on neuromuscular conditions.
BEIJING – Rare disease specialist Canbridge Pharmaceuticals Inc., of Beijing, said it is collaborating with the Horae Gene Therapy Center at the University of Massachusetts (UMass) Medical School to conduct gene therapy research with a focus on neuromuscular conditions. The move is expected to add new assets to the company’s portfolio.
San Francisco-based RDMD Inc. netted $14 million in series A money fueling an approach designed to generate clinical evidence that will speed drug development in rare diseases.
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.
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.
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