Rare Daily Staff

iECURE, a mutation-agnostic in vivo gene editing company striving to cure genetic diseases with high unmet need, said it closed a $50 million series A financing to advance the company’s pipeline of up to 13 programs.

Versant Ventures and OrbiMed Advisors led the financing of the company, which is commercializing in vivo delivery approaches being developed in the laboratory of James Wilson of the University of Pennsylvania Gene Therapy Program.

iECURE’s approach focuses on the mutation-agnostic in vivo insertion of healthy copies of disease-causing genes, with an initial focus in liver disorders. This strategy has the potential to provide durable benefits for a variety of diseases, particularly in dividing tissues such as the livers of young children. The initial developmental programs rely on the use of an endonuclease-based gene editing technology delivered via AAV. Wilson’s laboratory has been studying this approach in several model systems to be further developed via the partnership with iECURE and has generated initial data that supports this approach.

In addition, since a complete gene is inserted into the patient’s chromosome, the treatment approach is independent of the specific disease-causing mutation. In primate models, iECURE’s programs have shown the ability to integrate wild-type genes into the genome, leading to long-term expression.

“Through our foundational collaboration with Penn, we are gaining access to a comprehensive, liver-focused, in vivo gene editing program that Wilson and his team have been developing in his labs for several years,” said Joseph Truitt, CEO of iECURE.

The Penn collaboration provides iECURE with exclusive licensing rights to three liver disorder programs under current study at GTP and the option to license over 10 additional pipeline assets in the future. Under the joint research and development initiative, the Gene Therapy Program will continue to lead preclinical research activities in collaboration with iECURE until company sponsored Investigational New Drug Application submissions have been filed. Post-IND, iECURE will execute the clinical studies and prepare for potential commercialization.

Photo: Joseph Truitt, CEO of iECURE

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