Takeda Taps Code Biotherapeutics to Develop Non-Viral Gene Therapies in $2 Billion Biobucks Deal

Takeda is dangling $2 billion on the line to access Code Biotherapeutics’ 3DNA non-viral gene delivery platform. The Japanese drug giant aims to use the platform to develop up to four rare disease programs. 

The first program will target the liver, while the two companies will also conduct additional studies for central nervous system-directed rare disease programs. 

Upon closing of the deal, Code Bio will receive “double-digit million dollars” in upfront, near-term milestone and research funding payments. The biotech could also receive up to $2 billion if milestones for all four programs are met, as well as royalties on product sales. 

Takeda and Code Bio will jointly research and select candidates, which Takeda has the option to license for further development and commercialization.  

The Advantages of Non-Viral Gene Delivery

Many high-profile gene therapies use viral vectors, such as AAVs, to deliver their genetic payload. While this method sees relatively high uptake by cells, it has the potential to stimulate unwanted immune responses. Off-target effects are another risk, as the gene may insert itself in a random location in the genome, possibly causing gene mutations that may lead to cancer. 

More than a few AAV gene therapies have landed in hot water over the potential risks. In recent years the FDA had slapped clinical holds on AAV gene therapies developed by Astellas, Biomarin, Homology Medicines, bluebird bio, and UniQure. Some of these holds have yet to be resolved. 

Code Bio is one of the emerging companies that are working on non-viral gene delivery. Its vector consists of strands of synthetic DNA, linked together to form a scaffold. 

The company believes its platform can overcome the limitations of most gene delivery methods, and says that its vector allows specific cell targeting, is able to deliver large genetic payloads, and can be re-dosed, unlike AAV therapies. 

Preclinical studies indicate that the platform is specific and effective at delivering genes without causing off-target effects or observable safety concerns, the company said. 

Besides the Takeda deal, Code Bio has a program for Duchenne muscular dystrophy in the discovery pipeline. Another program takes aim at type 1 diabetes. 

Related Article: BIO CEO: Overcoming the Obstacles in Developing Gene Therapies

Companies Developing Non-Viral Gene Therapies

Philadelphia-based Code Bio launched last April to a $10 million seed funding round which saw participation from the venture arm of Takeda. It faces competition from a number of companies already working on non-viral gene therapies. 

Last October, Takeda inked a deal worth $3.6 billion with Poseida to develop up to eight non-viral gene therapies. 

And Generation Bio clinched $230 million in a Nasdaq IPO in 2020 to fund its lead hemophilia gene therapy, which uses close-ended DNA strands and liquid nanoparticle delivery. 

Related Article: Takeda Inks $3.6 Billion Deal with Poseida to Advance Non-Viral Gene Therapies and Overcome Safety Issues

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Joy Lin

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