As part of the Takeda deal, Ensoma will do preclinical research for therapies in up to five rare disease indications, and both companies will work on Investigational New Drug-enabling studies.
With a Takeda collaboration already in its pocket, Ensoma announced a $70 million Series A round to help develop its platform of engineered gene therapy vectors for off-the-shelf in vivo therapeutics, starting in rare diseases.
The venture round was led by 5AM Ventures, which co-founded and seeded Ensoma. F-Prime Capital, Takeda Ventures, Viking Global Investors, Cormorant Asset Management, RIT Capital Partners, Symbiosis II, LLC and Alexandria Venture Investments also participated, with Takeda making a $10 million equity investment in the new company.
As part of the Takeda deal, Ensoma will do preclinical research for therapies in up to five rare disease indications, and both companies will work on Investigational New Drug (IND)-enabling studies. Takeda has an exclusive license for Ensoma’s vectors in the chosen indications. Upfront payments alone were not disclosed, but Ensoma could earn up to $100 million for its early-stage work and up to $1.25 billion overall in milestone payments.
Ensoma is developing helper-dependent adenoviral vectors–vectors with viral coding sequences removed–that would not be limited to ex vivo editing for autologous cell therapies or require myeloablative conditioning to prepare a patient’s immune system.
The technology is based on the work of scientific co-founders Hans-Peter Kiem and André Lieber. Kiem is director of the Stem Cell and Gene Therapy Program Clinical Research Division at Fred Hutchinson Cancer Research Center, and Lieber is a professor of medical genetics at the University of Washington School of Medicine.
In September, Lieber’s lab published preclinical findings that showed bi-modular helper-dependent adenoviral vectors could deliver a therapeutic gene therapy in vivo to hematopoietic stem cells (HSCs) in a mouse mode of sickle cell disease.
According to the company, its engineered vectors can be specifically targeted to deliver large payloads for a diverse array of genome-modifying technologies in vivo, not just to HSCs but also the cells those generate like T, B and myeloid cells. And because the vectors have been engineered to lack a viral genome, they are expected to have lower immunogenicity than lentiviral vectors typically used for in vivo genetic therapies, without the need for redosing.
“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,” said Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan.
Takeda made similar forays in search of a post-adenoviral vector delivery approach last year. In June, it partnered with extracellular vesicles (EVs) company Carmine Therapeutics to develop and commercialize non-viral gene therapies for two rare diseases. Carmine is developing EVs derived from engineered O-type blood cells as vehicles for gene therapies. And last March, Takeda also partnered with EV company Evox Therapeutics for therapies in up to five rare disease indications.
