The partnership will see Code Bio’s 3DNA platform used to design and develop gene therapies for rare disease indications.

Under the terms of the deal, Takeda will use Code Biotherapeutics’ (Code Bio’s) non-viral 3DNA platform to target a liver-directed rare disease program and central nervous system conditions across a total of four programs.

“3DNA is a synthetically made DNA that can be used as a targeted vehicle to deliver therapies.  We have a proprietary process to attach therapeutic payloads and targeting moieties to the DNA scaffold,” Brian McVeigh CEO of Code Bio told BioProcess Insider.

Brian McVeigh, CEO of Code Bio. Image c/o Code Bio

McVeigh continued: “Our 3DNA platform is non-viral so we have the potential to re-dose patients, if needed. We believe our 3DNA platform is poised to transform the field of genetic medicines. Targeting is one of the key differentiators of our platform, and we have strong data demonstrating our high degree of tissue-specific and cell-specific targeting capabilities. By limiting the need for high dosing, we are able to minimize the effects from off-targeting.”

Takeda will fork out an upfront payment worth “double-digit million dollars” to Code Bio, but specific financial details were not disclosed. Code Bio also has the potential to receive future commercial and development milestone payments of up to $2 billion if all four programs are successful.

Both firms will work together on research activities up to candidate selection. But after option exercise has occurred, Takeda will take full responsibility for development and commercialization.

Code Bio did not divulge where scientific work will be conducted or if there is a time limit to the partnership but did tell us that its “business model includes scaling up appropriately to support continued growth through both our internal pipeline and external partnerships.”