Mayflower Launches Primera with Cellectis Collaboration to Develop Gene Editing Platform to Treat Mitochondrial Diseases

Mayflower Bioventures Launches Primera Therapeutics with Cellectis Collaboration to Develop Gene Editing Platform to Treat Mitochondrial Diseases

Rare Daily Staff

Cell and gene therapy accelerator Mayflower Bioventures launched its first start-up, Primera Therapeutics, which signed a collaboration agreement with gene editing biotech Cellectis focused on editing mutations in the mitochondrial DNA in vivo to treat the root cause of associated diseases.

Inherited mitochondrial diseases caused by genetic changes in the mitochondrial DNA (mtDNA) have very limited to no treatment options, and often result in early childhood death. An estimated 1 in 5,000 people worldwide suffer from mtDNA-associated diseases. Primera is advancing pioneering mtDNA gene editing therapies developed in the Mayo Clinic lab of Steven Ekker and Karl Clark to precisely fix a patient’s mutated mtDNA and potentially achieve a cure for the disease.

Primera’s initial clinical focus is on rare mitochondrial diseases for which there are no approved treatments. Mayo Clinic is expert in delivering and developing advanced care for complex diseases including the interdisciplinary research and clinical treatment through its Mitochondrial Care Research Center.

Primera, together with Cellectis, will be co-developing a mtDNA engineering toolbox that could enable effective therapies for mitochondrial diseases. The experience Cellectis brings in gene editing research, technology, manufacturing, and clinical development will enable moving quickly to advance these programs and mitochondrial research forward.

The collaboration agreement should be supplemented by other agreements to be concluded between the parties, under which Cellectis would receive a 19 percent equity ownership stake in Primera and would take a seat on Primera’s Board of Directors.

Pursuant to the agreement, Primera has a right to exercise an exclusive worldwide option for a license from Cellectis on up to five product candidates developed under the collaboration. Upon Primera exercising the option, Cellectis will be eligible for up to $750 million of development and sales milestones for the partnership products, as well as high single-digit royalty payments on the net sales of partnership products.

Along with Cellectis and Mayo Clinic, Primera is collaborating with patient advocacy groups including the United Mitochondrial Disease Foundation (UMDF), the largest source of funding for mitochondrial disease research outside of the government, which promotes research and education for the diagnosis, treatment, and cure of mitochondrial disorders and supports affected individuals and families.

“Our partnership with Primera further showcases and expands the application of our gene-editing capabilities into a previously unexplored space. This partnership is very much in line with Cellectis’ mission to leverage its gene editing technologies to develop potentially life-saving product candidates to address unmet medical needs,” said André Choulika, CEO of Cellectis.

Photo: André Choulika, CEO of Cellectis