BOSTON—A new company opened its doors on Feb. 11, as Ensoma launched with a $70-million Series A financing and a focus on expanding the curative power of genomic medicine by pioneering a next-generation in-vivo approach. The financing was 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. Ensoma was founded by and incubated in the 4:59 Initiative, 5AM Ventures' company creation engine.
“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 platform is based on its Engenious vectors, the result of more than 20 years of research by scientific co-founders Dr. Hans-Peter Kiem of Fred Hutchinson Cancer Research Center and Dr. André Lieber of the University of Washington School of Medicine. The vectors are specially engineered adenovirus vectors that lack any viral genome and minimal pre-existing immunity, which not only minimizes the chance of an immune response, but also makes up to 35 kilobases of DNA packaging available. The Engenious vectors are engineered to deliver their payloads directors to hematopoietic stem cells, T cells, B cells, or myeloid cells, and work without requiring stem cell collection or myeloablative conditioning such as chemotherapy. Ensoma reports that its vectors have applications in areas such as genome editing (such as CRISPR/Cas 9, ZFN, or base editing), targeted and random genomic integration approaches, and regulatory elements for cell type-specific gene expression.
“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,” Dr. Kush M. Parmar, founding CEO of Ensoma, commented in a press release. “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.”
On the same day as its launch, Ensoma also shared news of a strategic collaboration with Takeda Pharmaceutical Company Limited that grants the latter an exclusive worldwide license to Ensoma's Engenious vectors for up to five rare disease indications. In addition to making an equity investment of $10 million in Ensoma's Series A financing round, Takeda will also pay up to $100 million in upfront and preclinical research payments. If all five programs are successful, Ensoma could receive up to $1.25 billion from Takeda, including development and commercialization milestone payments and up to low double-digit royalties on net sales of any resulting products. Ensoma will be responsible for conducting preclinical research activities for the Takeda programs, and both companies will share responsibilities related to submission of IND applications, with Takeda leading development activities after that point.
“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,” stated 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.”