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