Vertex and Entrada Therapeutics to discover and develop Endosomal Escape Vehicle therapeutics for myotonic dystrophy type 1

Vertex Pharmaceuticals and Entrada Therapeutics have established a global collaboration focused on discovering and developing intracellular Endosomal Escape Vehicle (EEV) therapeutics for myotonic dystrophy type 1 (DM1). The collaboration includes Entrada’s program for DM1, ENTR-701, which is in late-stage preclinical development.

“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, M.D., Ph.D., executive vice president, Global Research, and chief scientific officer of Vertex.

“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 Chief Executive Officer of Entrada Therapeutics.

“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.”

Under the terms of the agreement, Entrada will receive an upfront payment of $224 million, as well as an equity investment of $26 million. Entrada is eligible to receive up to $485 million for the successful achievement of certain research, development, regulatory and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement.

The agreement includes a four-year global research collaboration whereby Entrada will continue to advance and receive payments for certain research activities related to ENTR-701, as well as additional DM1-related research activities. Vertex will be responsible for global development, manufacturing and commercialization of ENTR-701 and any additional programs stemming from Entrada’s DM1 research efforts.

The collaboration is subject to review under the Hart-Scott-Rodino Antitrust Improvements Act.

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