Vertex and Entrada Therapeutics Enter Endosomal Escape Vehicle Collaboration

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Under the collaboration between Vertex and Entrada, the companies will work on discovering and developing intracellular endosomal escape vehicle therapeutics for myotonic dystrophy type 1.

Vertex Pharmaceuticals, a biotechnology company based in Boston, Mass., and Entrada Therapeutics, a biopharmaceutical company also based in Boston and specializing in therapeutics derived from its endosomal escape vehicle (EEV) platform, announced a collaboration on Dec. 8, 2022. Under this $735 million collaboration, the companies will jointly work on the discovery and development of EEV therapeutics for myotonic dystrophy type 1 (DM1). The research will include ENTR-701, Entrada’s program for DM1 that is in late-stage preclinical development.

Under the agreement, Entrada will receive an upfront payment of $224 million from Vertex, as well as an equity investment of $26 million. Entrada is also eligible to receive an additional $485 million per the completion of certain research, development, regulatory, and commercial milestones, as well as tiered royalties on any net sales from products that result from this collaboration.

Also under the agreement, the companies will conduct a four-year global research collaboration in which Entrada will receive payments for research associated with ENTR-701 and other DM1-related research activities. Vertex will be responsible for global development, manufacturing, and commercialization of ENTR-701 and any additional programs stemming from the agreement.

ENTR-701 is a proprietary EEV-conjugated phosphorodiamidate morpholino oligomer designed to address the underlying cause of DM1. It achieves this through allele-specific targeting of the disease-associated trinucleotide repeats in dystrophia myotonica protein kinase transcripts. The goal of this mechanism of action is to restore the function of muscle blind-like proteins, correct the mis-splicing and aberrant expression of downstream transcripts, and restore normal muscle function.


“Vertex’s strategy is to discover and develop transformative medicines for people with serious diseases, and DM1 has therefore been a disease area of interest to Vertex for some time,” said David Altshuler, executive vice-president, Global Research, and chief scientific officer, Vertex, in a company press release. “Entrada’s innovative EEV approach, the significant progress in their DM1 program, and the potential for it to reach the clinic in the near-term hold exciting potential for patients. Working together, we believe we have the opportunity to develop a transformative treatment for this devastating disease.”

“Our collaboration with Vertex represents an important step for Entrada as we work to make intracellular therapeutics a reality through our novel EEV approach,” said Dipal Doshi, president and CEO, Entrada Therapeutics, in the press release. “DM1 is a progressive disease with no treatment options available. Working with Vertex will enable us to expeditiously move this program forward, while focusing the majority of our internal resources on advancing new therapeutic options for patients living with Duchenne and expanding our commitment to non-neuromuscular disease programs.”

Source: Vertex Pharmaceuticals