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

Company’s Engenious™ Vectors Delivered without Stem Cell Collection or Myeloablative Conditioning in Outpatient and Low-resource Settings, Enabling Access Worldwide

$70 Million Series A Financing with Potential for $100 Million in Upfront and Preclinical Research Payments Resulting from Strategic Collaboration with Takeda

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 company’s 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 André 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.

Ensoma’s 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 (e.g., chemotherapy). As a result, Ensoma’s 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

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, the following:

• Genome editing (e.g., CRISPR/Cas9, ZFN, base editing)
• Targeted and random genomic integration approaches
• 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 M. Parmar, M.D., Ph.D., founding chief executive officer 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.”

Leadership & Scientific Founders

Ensoma was founded by and incubated within the 4:59 Initiative, the company creation engine of 5AM Ventures. The company’s 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. André 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:

• Paula Soteropoulos, executive chairman, brings to Ensoma more than 30 years of experience in the biopharma industry. Previously, Ms. Soteropoulos served as the founding CEO and board member of rare disease therapeutic company Akcea Therapeutics. She also serves as strategic advisor for 5AM Ventures and member of the board of directors of uniQure and Rallybio.
• Kush M. Parmar, M.D., Ph.D., founding chief executive officer, is a managing partner at 5AM Ventures and serves as a board director of multiple next-generation biotechnology companies, including Akouos, Homology, Entrada, Rallybio and Vor Biopharma.
• Stephen Knight, M.D., board member, is president and managing partner at F-Prime Capital. Dr. Knight has worked in the pharmaceutical and biotechnology industries for over 25 years and invests broadly across healthcare.
• Albert Seymour, Ph.D., board member, is chief scientific officer for Homology Medicines. Dr. Seymour has spent more than 20 years coupling the discipline of human genetics with pharmaceutical R&D, resulting in the delivery of multiple therapeutic programs into development.
• Robert Peters, Ph.D., chief scientific officer, most recently led the rare blood disorders research group at Sanofi and prior to that, served as senior vice president of research at the Biogen spinoff Bioverativ. Dr. Peters has 20 years of industry experience across a range of modalities, from biologics and small molecules to both in vivo and ex vivo gene therapies.
• Daniel Leblanc, chief technology officer, previously led manufacturing, analytical development and drug product development for Flexion Therapeutics, where he established a highly productive and reproducible manufacturing process for the company’s helper-dependent adenovirus-based gene therapy. Mr. Leblanc is an experienced engineer and scientist with broad experience in formulation development and process scale-up from research and development to commercialization.
• Naina Bhasin, Ph.D., chief business officer, has 15 years of life sciences experience in corporate and business development, strategic planning and operations. Previously, Dr. Bhasin served as the chief of staff to the CEO at Intellia Therapeutics, and prior to that, she was a director of business development at Shire.

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 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.

Contacts

Katie Engleman, 1AB

katie@1abmedia.com