Vertex partners with Entrada on new approach for muscle dystrophy treatment

Vertex Pharmaceuticals Incorporated and Entrada Therapeutics, Inc. have formed a global partnership to find and develop treatments for myotonic dystrophy type 1 (DM1), a form of muscular dystrophy, using Entrada’s Endosomal Escape Vehicle (EEV) platform, Vertex said in a press release.

Entrada is currently using EEV to develop ENTR-701, their most sophisticated treatment for DM1, the press release said. The EEV platform makes it possible to “engage intracellular targets that have long been considered inaccessible and undruggable,” the release said. “EEV therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues, resulting in an improved therapeutic index.”

Entrada said last month that ENTR-701 is in late-stage preclinical testing, and they would approach authorities in the second half of 2023 to request permission to test the medicine on humans, according to BioPharma Dive.

Under the agreement, Entrada Therapeutics will receive an upfront payment of $250 million from Vertex, which includes a $26 million equity investment for development of ENTR-701, according to the press release.

“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,” the release said. “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.”

Vertex said it will be responsible for global development, manufacturing, and commercialization of ENTR-701 and any additional programs stemming from Entrada’s DM1 research efforts.

“Our collaboration with Vertex represents an important step for Entrada as we work to make intracellular therapeutics a reality through our novel EEV approach,” Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics said, according to the 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.”

Other work by Vertex

Vertex has also made investments in Duchenne muscular dystrophy (DMD) treatments, another type of muscular dystrophy. Muscle wasting is the major symptom of Duchenne muscular dystrophy (DMD), which affects the muscles and worsens with time, according to the National Institutes of Health (NIH). DMD typically affects boys, however, it can occasionally affect girls as well, NIH said.

To help raise awareness about DMD, as well as other rare and debilitating diseases, Vertex is collaborating with Bloomberg Media Studios, to bring a new, educational podcast.

Targeting the Toughest Diseases looks at some of humanity’s most challenging diseases and how Vertex Pharmaceuticals is using innovative tools, methods, and a unique philosophy to search for treatments and cures,” Bloomberg says.

Vertex’s early work involved developing therapies for cystic fibrosis, a different genetic condition, and from there it grew to become one of the largest biotech companies in the world, according to Biopharma.

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