BOSTON--(BUSINESS WIRE)--Ensoma, a company expanding the curative power of genomic medicine by pioneering a next-generation in vivo approach, today launched with a $70 million Series A financing led by co-founder and seed investor 5AM Ventures, with participation from F-Prime Capital, Takeda Ventures, Viking Global Investors, Cormorant Asset Management, RIT Capital Partners, Symbiosis II, LLC, and Alexandria Venture Investments. In addition to an equity investment of $10 million in the Series A financing, Takeda Pharmaceutical Company Limited (Takeda) and Ensoma have entered into a strategic collaboration with the potential for upfront and preclinical research payments totaling $100 million as part of a strategic collaboration worth up to $1.25 billion, announced in a separate press release this morning.

The foundation of the companys platform its Engenious vectors is based on over two decades of academic and clinical research generated by scientific co-founders and renowned experts, Hans-Peter Kiem, M.D., Ph.D., of Fred Hutchinson Cancer Research Center, and Andr Lieber, M.D., Ph.D., of University of Washington School of Medicine. The company will be led by biotechnology industry veterans with demonstrated track records in innovative therapeutic modalities, including gene therapy and editing, across an array of disease areas, including rare disease, hematology and oncology.

Ensomas 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 companys vectors are optimized to work without the need for stem cell collection or prior myeloablative conditioning (e.g., chemotherapy). As a result, Ensomas therapies will be designed 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.

Engenious Vectors

Ensomas 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, the following:

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, Ensomas Engenious vectors enable preferential targeting of HSCs inside the body. Additionally, Ensomas 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.

Ensomas 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 M. Parmar, M.D., Ph.D., founding chief executive officer of Ensoma. Ensomas scientific approach allows us to do what hasnt been done beforeto 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.

Leadership & Scientific Founders

Ensoma was founded by and incubated within the 4:59 Initiative, the company creation engine of 5AM Ventures. The companys scientific co-founders include Dr. Hans-Peter Kiem, an oncologist and world-renowned pioneer in gene-editing technologies, including stem cell and gene therapies, from Fred Hutch, who also serves as vice president of the American Society of Gene & Cell Therapy and chief scientific and clinical advisor for Ensoma; and Dr. Andr Lieber, an accomplished academic researcher and professor of medicine, Division of Medical Genetics, UW School of Medicine, who has studied the biological and translational aspects of human adenoviruses for more than two decades. Ensoma is based on an exclusively licensed portfolio of technologies developed by the Fred Hutch lab of Dr. Kiem and the University of Washington lab of Dr. Lieber that enable in vivo genome engineering and gene therapy advances of HSCs for therapeutic use in blood diseases.

Following more than 20 years of academic and clinical research, Ensoma has assembled an exceptional team to boldly forge a new era of genomic medicine in vivo, said Bihua Chen, founder and portfolio manager at Cormorant Asset Management. The company is moving swiftly to accelerate and broaden the therapeutic potential of its approach, and I am confident they have the right team and the right technology to potentially bring life-changing, curative therapies within reach for people all over the world.

Additional details surrounding company leadership, including its board of directors, are as follows:

Ensoma has also named its scientific advisory board, which may be viewed here.

About 5AM Ventures

Founded in 2002, 5AM actively invests in next-generation biotech companies. With approximately $1.5 billion raised since inception, 5AM has invested in 89 companies. For more information, please visit http://www.5amventures.com.

About the 4:59 Initiative

The 4:59 Initiative is the internal company creation engine at 5AM Ventures that helps discover, incubate, and fund breakthrough science. The 4:59 team provides hands-on scientific, strategic, and operational support, working closely with academics and entrepreneurs to advance breakthrough science and establish proof-of-concept data to enable a clear path to transformative therapies for patients.

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Ensoma Launches to Pioneer Next-Generation In Vivo Approach to Deliver First Off-the-shelf Genomic Medicines - Business Wire