Ensoma emerges with Series A funding and a collaboration deal with Takeda for its vector-delivered treatments.

Ensoma was backed in its Series A financing by 5AM Ventures and Takeda, amongst other investors, with $70 million (€57 million).

Takeda’s investment occurs alongside an exclusive worldwide license to develop Ensoma’s ‘Engenious’ vectors for up to five rare disease indications.

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The agreement includes a potential $100 million investment in upfront and preclinical research payments. Should the candidates progress past the preclinical stage, Ensoma is in line to receive up to $1.25 billion in additional development and commercialization milestones.

At the heart of the deal are Ensoma’s vectors, which the company states are designed to deliver genome modification technologies, such as genome editing through CRISPR/Cas9 or zinc finger nuclease.

This would allow Ensoma’s vectors to potentially treat rare monogenic diseases but, according to the company, also the possibility for broader disease indications in oncology or infectious diseases.

The vectors are engineered adenovirus vectors that do not contain any viral genome, allowing for ‘storage space’ to deliver therapeutic cargo.

A spokesperson for Ensoma confirmed that it is currently working with unspecified contract manufacturing partners but that there are plans to bring manufacturing inhouse.

“We are launching with significant process development and manufacturing expertise,” the spokesperson added.

The company arrives with a chief technology officer in Daniel Leblanc, who previously led manufacturing, analytical development and drug product development for Flexion Therapeutics, a company developing an adenovirus-based gene therapy.

Regarding the complexity of producing Engenious vectors, the spokesperson stated that the process is ‘much simpler and scalable’ compared to adeno-associated virus and lentiviral vector manufacturing.

Executive chairman of Ensoma, Paula Soteropoulos, announced on the company’s launch that the plan is to deliver any treatments ‘off-the-shelf’, as no stem cell donors are required in their production and with no ‘prior conditioning’ needing to take place.

The conditioning refers to patients who receive certain ex vivo gene therapy via lentiviral vectors undergoing chemotherapy prior to delivery.

Ensoma plans to administer its therapies through a single injection, potentially allowing the treatment to be delivered “where access to sophisticated healthcare systems may be limited”, the company states.

In terms of the next steps for the company, the spokesperson outlined that the Series A financing would allow the company to reach the investigational new drug (IND)-candidate nomination stage before it would need to raise additional equity. Ensoma plans to advance its own internal programs alongside those developed with Takeda.