Will This CRISPR/Cas9 Technology Agreement Help Duchenne Patients?


CRISPR Therapeutics and Anagenesis Biotechnologies collaborate to produce CRISPR/Cas9-based cell therapies for muscle diseases.

crispr-therapeutics-anagenesis-biotechnologies-collaborationThis agreement will grant CRISPR Therapeutics exclusive worldwide license to AnagenesisParaxial Mesoderm Multipotent Cells (P2MCs) technology to develop cell therapies for all human muscle diseases starting with Duchenne Muscular Dystrophy (DMD).

The will provide platform for “the efficient, reproducible and chemically defined differentiation” of those master cells which can potentially develop into any cell or tissue of the body (pluripotent) into skeletal muscle stem cells. This “strategic in-licensing” agreement will allow the hottest genome-editing tool, CRISPR based cellular therapies to carve out musculoskeletal disease treatment.

P2MC technology anagenesis

crispr p2mc anagenesis

anagenesis p2mc technology

Figure 1. Paraxial Mesoderm Multipotent Cells (P2MC) technology of Anagenesis Biotechnologies (Photo credit: Anagenesis Biotechnologies). For detail, please visit Anagenesis Biotechnologies.

DMD is a genetic, fatal disorder which leads to progressive muscle disorder due to absence of a protein, dystrophin required to keep the muscle cells intact. Approximately 1 in 3,500 newborn boys suffer from this disease.

duchene muscular dystrophy cause

Figure 2. Cause of DMD (Photo credit: “Designer” genes correct Muscular Dystrophy, //

DMD treatment development is currently going through a bad phase. Recently, BioMarin stopped the development of its Duchenne drug, Kyndrisa after some regulatory issues which it acquired from Prosensa Holding in a $680 million deal. Sarepta Therapeutics’ Duchenne drug, eteplirsen is also waiting for its FDA decision for a long time.

CRISPR Therapeutics is a revolutionary gene editing company striving to develop gene based medicines using its own CRISPR/Cas9 gene editing platform. It is well known for developing accurate alterations in genomic DNA and the well equipped workforce relentlessly developing therapeutics for rare diseases.

Anagenesis Biotechnologies found by Dr. Olivier Pourquiéis is developing new therapies for diseases such as DMD, sarcopenia and cachexia. The team led by President & CEO, Dr. Jean-Yves Bonnefoy offers key opinion in musculoskeletal development and stem cells.

Jean-Yves Bonnefoy, PhD, President and Chief Executive Officer of Anagenesis announced, “We are excited to partner with CRISPR Therapeutics, a leader in the field of CRISPR gene editing, to advance therapeutic development for musculoskeletal diseases, including DMD, where there is a high unmet medical need”.  

The P2MC technology developed by AFM-telethon, INSERM-Transfert, CNRS and Université de Strasbourg offers efficient, reproducible and chemically defined differentiation of pluripotent cells into satellite cells (skeletal muscle stem cells). Combining this technology with CRISPR/Cas9 gene editing platform will enhance ex vivo therapeutic approaches to treat muscular disorders.

The Chief Business Officer of CRISPR Therapeutics, Samarth Kulkarni announced “We are highly committed to finding new treatments for DMD and this collaboration is an important building block in support of our broader research strategy within DMD and other musculoskeletal diseases.”

This collaboration is the third most prominent one for CRISPR Therapeutics projecting it towards a leading growth in the global pharmaceutical sector.

Featured image credit: human anatomy 3D head model with face muscles © ronstik (Stock Photo ID: 129303044). Graphics re-mixed by Medgenera.

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