Ensoma Announces Strategic Collaboration with Takeda to Accelerate Next-Generation In Vivo Gene Therapies

Ensoma Grants Takeda Exclusive Worldwide License to Engenious™ Vectors for as Many as Five Rare Disease Indications

Potential for $100 Million in Upfront and Preclinical Research Payments with Additional Development and Commercialization Milestone Payments and Royalties Up to $1.25 Billion

BOSTON--(BUSINESS WIRE)--Ensoma, a company that formally launched today with the mission of expanding the curative power of genomic medicine by pioneering a next-generation in vivo approach, announced it has entered into a strategic collaboration with Takeda Pharmaceutical Company Limited (“Takeda”). The agreement grants Takeda an exclusive worldwide license to Ensoma’s Engenious™ vectors for up to five rare disease indications. Takeda will make an equity investment of $10 million in Ensoma’s Series A Preferred Stock with the potential for $100 million in upfront and preclinical research payments. In total, assuming success for five programs, Ensoma is eligible to receive up to $1.25 billion from Takeda, including additional development and commercialization milestone payments and up to low double-digit royalties on net sales of each product.

“Not only are we thrilled to be formally launching Ensoma today; we are delighted to announce our partnership with Takeda, an established leader in rare disease with significant clinical expertise and a global footprint,” said Paula Soteropoulos, executive chairman of Ensoma. “In addition to this early validation of our in vivo approach, near-term access to non-dilutive funding positions Ensoma to rapidly accelerate and actualize the broader potential of our revolutionary platform to provide life-changing, curative therapies for a vast set of therapeutic applications.”

Ensoma’s Engenious vectors are designed to deliver a diverse range of gene modification technologies – including those that require a high level of packaging capacity – directly to hematopoietic stem cells (HSCs) or the various cell types that arise from these cells, such as T cells, B cells and myeloid cells, without the need for stem cell collection or prior myeloablative conditioning (e.g., chemotherapy). As a result, Ensoma’s therapies can be delivered “off-the-shelf” in settings including doctors’ offices and mobile clinics.

“The Ensoma platform offers distinct advantages over AAV and ex vivo lentiviral gene therapy approaches with the potential to overcome some of the challenges associated with first-generation technologies,” said Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan. “Takeda continues to make focused investments in differentiated, next-generation gene therapy platforms through our network of innovative collaborators with the collective goal to deliver transformative therapies and functional cures for patients living with rare diseases.”

Under the terms of the collaboration, Takeda is granted an exclusive worldwide license to Ensoma’s Engenious vector technology platform for up to five rare genetic disease indications. Ensoma will conduct preclinical research activities for the Takeda programs, and both parties will share in responsibilities leading to submission of Investigational New Drug (IND) applications. Takeda will lead development activities thereafter.

About Ensoma

Ensoma is expanding the reach of the curative power of genomic medicine by pioneering a next-generation in vivo approach using its Engenious™ vectors. Ensoma’s vectors are designed to deliver a diverse range of gene modification technologies without the need for stem cell collection or prior myeloablative conditioning (e.g., chemotherapy). As a result, Ensoma’s therapies can be delivered as a single injection in a diverse range of settings, including outpatient and settings where access to sophisticated healthcare systems may be limited. For more information, visit www.ensoma.com.