There’s no shortage of local biotechs working in the field of genomic medicine, but something still keeps investor Kush Parmar up at night: The managing partner at Boston venture capital firm 5AM Ventures knows the emerging treatments won’t be widely accessible to patients.
Some forms of gene therapy and gene editing require a patient’s cells to be removed and reinserted into the body. Others involve cell donors, or initial steps such as chemotherapy — treatments that can only be done in specialized centers or that require days of hospitalization.
“These are not small-molecule pills that can go generic and be on the shelves of every pharmacy,” Parmar said. “Our industry needs to develop the technologies and approach that make them accessible. It’s not going to be someone else’s problem, it is our problem.”
After 18 months of incubation within 5AM, Parmar’s startup Ensoma came out of stealth on Thursday with $70 million in venture capital and a partnership with Japanese pharmaceutical giant Takeda, one of the largest biopharmaceutical employers in Massachusetts. Ensoma has developed vectors, or carriers, that can deliver genes or gene-editing tools directly to certain cells, including the stem cells that make blood cells, as well as T cells and B cells of the body’s immune system.
What sets the company apart, at least in theory, is that Ensoma’s medicine could be administered through a single intravenous injection, meaning it would be accessible in outpatient centers or less sophisticated health care systems.
Paula Soteropoulos, the executive chairman of Ensoma and a strategic advisor at 5AM, said more complicated delivery methods for gene therapy are “not going to work for everyone.” They could require a patient to take time off of work for a lengthy procedure, or they may not be available in a region or country without access to specialized centers, she said.
“Even now at the onset, we are thinking about access and partnering because we want to deliver our medicines globally,” Soteropoulos said.
Parmar, who is Ensoma’s founding chief executive, said the partnership with Takeda “speaks to the vast potential of our starting point.” The drug giant signed a deal to license up to five rare disease targets, with the potential for $100 million in upfront and preclinical research payments. Takeda also made a $10 million investment in the biotech. The overall funding round was led by 5AM Ventures with participation from F-Prime Capital, Viking Global Investors, Cormorant Capital, RIT Capital Partners, Alexandria Venture Investments, and Symbiosis.
The company plans to grow its 20-person team and find additional lab space in the city this year.
“To execute on the ambition we have, a company like this should be well-resourced out of the gate,” Parmar said. “Folks are fired up about the kind of impact we are looking to make.”