Code Bio to collaborate with Takeda to develop gene therapies for rare diseases

Code Biotherapeutics (Code Bio), a biotechnology company pioneering targeted non-viral delivery of genetic medicines, has announced a collaboration and option agreement with Takeda to leverage Code Bio’s proprietary targeted 3DNA® non-viral genetic medicine delivery platform to design and develop gene therapies for rare disease indications.

Through the agreement, Takeda and Code Bio will design and develop a targeted gene therapy leveraging Code Bio’s 3DNA platform for a liver-directed rare disease program, plus conduct additional studies for central nervous system-directed rare disease programs.

Takeda has the right to exercise options for an exclusive license for four programs. Under the terms of the agreement, Code Bio will receive double-digit million dollars in upfront, near-term milestone and research funding payments. Code Bio is also eligible to receive future development and commercial milestone payments plus tiered royalties with a potential total deal value over the course of the partnership of up to $2 billion if milestones for all four programs are achieved.

Takeda and Code Bio will collaborate on research activities up to candidate selection. After option exercise, Takeda will assume responsibility for further development and commercialization.

“We are proud to collaborate with Takeda, a world leader in the fields of genetic medicine and rare diseases,” said Code Bio Chairman, Chief Executive Officer, and Co-Founder Brian P. McVeigh. “This collaboration will further enhance our capabilities by bringing together Takeda’s expertise in gene therapy and rare diseases with our expertise in delivering genetic medicines as we drive forward on our mission to bring meaningful treatments and cures to patients suffering from serious and life-threatening genetic diseases.”

Code Bio’s targeted 3DNA non-viral genetic medicine delivery platform is designed to overcome key limitations of other genetic medicine delivery approaches and fully unlock the potential of genetic medicines. The synthetic, multivalent design of the 3DNA platform enables cell specific targeting, the delivery of large genetic payloads, and the potential for re-dosability. Extensive preclinical data have demonstrated that targeted formulations are highly specific and effective at delivering genetic cargo to targeted cells and tissues with no evidence of off-target effects or safety concerns related to the platform seen.

“We aim to provide functional cures to patients with rare genetic and hematologic diseases through next-generation gene therapy programs,” said Madhu Natarajan, Head of the Rare Diseases Drug Discovery Unit at Takeda. “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.”

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