Ensoma Launches with $70 Million for In Vivo Gene Therapy, Partners with Takeda

Rare Daily Staff

Ensoma has launched with $70 million in a series A venture financing to develop off-the-shelf, next-generation genetic medicines that can be injected directly into the body.

Co-founder and seed investor 5AM Ventures led the financing with participation from F-Prime Capital, Takeda Ventures, Viking Global Investors, Cormorant Asset Management, RIT Capital Partners, Symbiosis II, and Alexandria Venture Investments.

In addition to an equity investment of $10 million in the series A financing, Takeda Pharmaceutical and Ensoma entered into a strategic collaboration with the potential for upfront and preclinical research payments totaling $100 million, and up to $1.25 billion in additional development and commercialization milestone payments.

Under the terms of their 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 applications. Takeda will lead development activities thereafter. Ensoma will be entitled to low double-digit royalties on net sales of any products arising from the collaboration.

Ensoma’s platform—Engenious vectors—is based on more than two decades of academic and clinical research generated by scientific co-founders and renowned experts, Hans-Peter Kiem of Fred Hutchinson Cancer Research Center, and André Lieber of University of Washington School of Medicine.

Engenious vectors are designed to deliver a diverse range of genome 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. The company’s vectors are optimized to work without the need for stem cell collection or prior myeloablative conditioning. As a result, Ensoma can design its therapies to be delivered via single injection in diverse environments, including outpatient and areas where access to sophisticated healthcare systems may be limited.

“With the launch of Ensoma, we aspire to bring innovative new treatments to patients in a way that is accessible for all,” said Paula Soteropoulos, executive chairman of Ensoma. “Because our in vivo therapies do not require prior conditioning or stem cell donors, we hope to deliver them as ‘off-the-shelf’ treatments to address diseases—both rare and common—dramatically simplifying the logistics of scaling production and reducing patient and healthcare-system burden. Every person, no matter where they are in the world, should have access to the innovative technologies that are changing the way we treat disease.”

Ensoma’s Engenious vectors are specially engineered adenovirus vectors devoid of any viral genome and minimal pre-existing immunity, thus minimizing the chance of an immune response and freeing up ample storage space—up to 35 kilobases (kb) of DNA packaging capacity—to deliver a diverse range of genome modification technologies. Also known as therapeutic cargo, these technologies may include, separately and in combination: genome editing (e.g., CRISPR/Cas9, ZFN, base editing); targeted and random genomic integration approaches; and regulatory elements for cell type-specific gene expression.

These approaches enable Engenious vectors to engineer various erythroid, lymphoid (e.g., T cells, B cells) and myeloid (e.g., macrophages, microglia) cell types, with great precision and vast therapeutic potential. Addressable indications range from rare monogenic diseases to broader diseases such as oncology, autoimmunity and infectious diseases via precision, “off-the-shelf” engineering of the immune system.

Given the highly specific nature of these technologies, Ensoma’s Engenious vectors enable preferential targeting of HSCs inside the body. Additionally, Ensoma’s founders have developed an in vivo selection system that can increase the population of genetically modified HSCs, if needed. This proprietary approach enables precise titration to lasting therapeutic levels without the need to re-dose patients, bypassing the immunogenic challenges associated with re-dosing for some other gene therapy modalities.

Ensoma’s Engenious platform has been extensively validated in numerous preclinical models with a range of genome editing technologies, demonstrating robust genetic modification of bone marrow HSCs and stable long-term expression of therapeutic proteins in small and large preclinical models.

“There have been tremendous advancements in technologies to precisely target, genetically edit and modify human disease. However, many of these tools pose delivery challenges; some lack the ability to reach the right cells within the body, while others lack the ability to broadly reach significant numbers of patients due to complex procedures and supply chain challenges,” said Kush Parmar, founding CEO of Ensoma. “Ensoma’s scientific approach allows us to do what hasn’t been done before—to make the curative power of genomic medicine and stem cell technology portable so they may be administered in low-resource and outpatient settings for the very first time.”

Photo: Kush Parmar, founding CEO of Ensoma