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.

DUBLIN – Silence Therapeutics plc entered a wide-ranging siRNA-based drug discovery and development deal with Astrazeneca plc that has propelled it toward the front ranks of siRNA platform companies. It is banking an up-front payment of $60 million, another $20 million as an equity investment and up to $400 million in option fees and milestone payments per candidate drug, in an alliance that could entail up to five active programs within three years.

HONG KONG – Singapore-based Proteona Pte. Ltd. has formed a couple of significant partnerships recently, including a Germany-Singapore partnership to develop a drug screening and discovery platform using single cell multi-omics analysis for patient-derived micro-tumors, and an international alliance to develop antibodies against coronaviruses.

Cytomx Therapeutics Inc. CEO Sean McCarthy told BioWorld that the longish hiatus between deals broken by the tie-up with Astellas Pharma Inc. was “very deliberate. Over that period of time, we were generating our first clinical data to get experience with how the technology works in cancer patients with our first two lead programs,” both of which have reached the phase II stage. A year ago in January, he said, the South San Francisco-based firm brought aboard Chief Business Officer Nick Galli to “hit the gas pedal a bit more in business development,” which led to the signing with Astellas, of Tokyo. Shares of Cytomx (NASDAQ:CTMX) closed March 24 at $6.53, up $1.60, or 32%.

Cytomx Therapeutics Inc. CEO Sean McCarthy told BioWorld that the longish hiatus between deals broken by the tie-up with Astellas Pharma Inc. was “very deliberate. Over that period of time, we were generating our first clinical data to get experience with how the technology works in cancer patients with our first two lead programs,” both of which have reached the phase II stage.

TORONTO – If Toronto’s Sunnybrook Hospital and the W. Garfield Weston Foundation get their way, a CA$33 million (US$24 million) initiative unveiled March 2 dramatically will increase patient access to made-to-measure ultrasound technology for penetrating the blood-brain barrier to treat Alzheimer's disease and brain cancer.