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.<\/p>\n
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.<\/p>\n
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 Ensomas 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.<\/p>\n
Ensoma is developing helper-dependent adenoviral vectorsvectors with viral coding sequences removedthat would not be limited to ex vivo editing for autologous cell therapies or require myeloablative conditioning to prepare a patients immune system.<\/p>\n
The technology is based on the work of scientific co-founders Hans-Peter Kiem and Andr 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.<\/p>\n
In September, Liebers 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.<\/p>\n
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.<\/p>\n
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.<\/p>\n
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.<\/p>\n
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Ensoma Launches with $70 Million Series A and Takeda Licensing Deal - BioSpace<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" 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. Continue reading