USA – Takeda has formed a new partnership with Code Biotherapeutics to gain opt-in rights for four rare disease candidates in a deal worth up to US$2 billion.

These treatments will be developed using Code Bio’s targeted 3DNA non-viral genetic medicine delivery platform.

The companies will use the 3DNA platform to design and develop targeted gene therapy for a liver-directed rare disease program, according to the agreement.

Takeda and Code Bio will also conduct additional research on central nervous system-directed programs.

Takeda has the option to exercise options to obtain an exclusive license for four programs under the terms of the agreement.

Takeda is entitled to pay Code Bio upfront, near-term milestones, and research funding payments in the double digit million-dollar range.

In the future, Takeda will make development and commercial milestone payments in addition to tiered royalty payments.

This raises the potential deal value of the collaboration to up to US$2 billion if all four programs meet their milestones.

Initially, the companies will work together on research activities till the selection of the candidates.

Takeda will oversee the development and marketing of the programs following the subsequent option exercise.

​Takeda Pharmaceutical Rare Diseases Drug Discovery Unit head Madhu Natarajan said: “We aim to provide functional cures to patients with rare genetic and hematologic diseases through next-generation gene therapy programmes.

Code Bio’s 3DNA platform will allow us to build upon the foundation we have established through our internal capabilities and external partnerships and will hopefully enable us to develop re-dosable and durable gene therapies that will be superior to current approaches.”

The 3DNA platform can address critical shortcomings of other genetic medicine delivery methods, allowing genetic therapies to reach their full potential.

Takeda has expanded its pipeline over the last two years, signing numerous deals, with gene and therapies and rare diseases at the top of the list.

Takeda signed a US$3.6 billion deal with Poseida Therapeutics on gene therapies for six to eight programs in October 2021.

These included liver and hematologic diseases, while another deal with Selecta Biosciences focused on lysosomal storage disorders and was worth around US$1.1 billion.

Takeda and Immusoft signed a research and licensing agreement in October of last year to develop cell therapies for rare neurometabolic disorders.

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