3D rendered Illustration, visualisation of a anatomically correct Mitochondrion, a organelle of most eukaryotic and other cells - to illustrate mitochondrial DNA changes in cancer
Credit: Westend61/Getty Images

Primera Therapeutics, the first launch from cell and gene therapy (CGT) accelerator Mayflower BioVentures, announced it has signed a collaborative agreement with Cellectis that will see the two companies will work to develop gene editing methods for mutations in mitochondrial DNA (mtDNA) as potential in vivo therapies to treat mitochondrial disease.

Primera employs a TALE-based gene editing system purpose built to target mutant DNA in the mitochondrial genome developed in the Mayo Clinic lab of Steven Ekker and Karl Clark. The focus of the company is to develop treatments for rare mitochondrial diseases for which there are not approved therapies. Together with Cellectis, the two companies will co-develop a mtDNA engineering toolbox to help create effective therapies for mitochondrial diseases. Cellectis will add its proven abilities to gene editing research, manufacturing, and clinical development to help advance the research and cell editing capabilities applicable to mitochondrial diseases.

“It requires a team effort to drive this program forward into the clinic and for patients who are suffering with these dreadful diseases,” noted Ekker, Dean of the Mayo Clinic Graduate School of Biomedical Sciences and a scientific inventor for Primera. “We are thrilled to launch Primera through Mayflower BioVentures and power it with the research and treatment expertise at Mayo Clinic and through collaborations with leading industry partners such as Cellectis, alongside a wonderful group of academic centers and nonprofit partners.”

Under the terms of the agreement, Primera has the exclusive option rights to license up to five therapeutic candidates developed as part of the Cellectis collaboration. Upon exercise of the option, Cellectis will be eligible to receive up to $750 million in development and sales milestones. Additionally, it would receive high single-digit royalty payments on the net sales of ensuing products.

“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, PhD, CEO of Cellectis.

As Primera begins work with Cellectis, it it also working with patient advocacy groups of mitochondrial diseases and the as well as the United Mitochondrial Disease Foundation (UMDF), which funds mitochondrial disease research and promotes research and education for the diagnosis, treatment, and cure of mitochondrial disorders.

Philip Yeske, PhD, Science and Alliance Officer for UMDF noted that UMDF and Primera are “closely aligned in our vision of driving the development and application of technologies to address the needs of those currently suffering from mitochondrial genetic diseases.”

According to information from the Children’s Hospital of Philadelphia (CHOP), mitochondrial disease affects roughly one in 43,000 people in the United States and encompass a group of disorders including fatigue, weakness, metabolic strokes, seizures, cardiomyopathy, arrhythmias, developmental or cognitive disabilities, diabetes mellitus, impairment of hearing, vision, growth, liver, gastrointestinal, or kidney function, among many others.

As there currently are no highly effective treatments, we are largely limited to offering supportive therapy,” noted clinical geneticist Marni Falk, MD, executive director of the Mitochondrial Medicine Frontier Program and a clinical and scientific research advisor to Primera. “We see over 1,300 patients from around the world in our mitochondrial medicine clinic each year, including both adults and children suffering from these devastating multi-system diseases. Through an interdisciplinary approach, we collaborate in performing groundbreaking research and development of technologies, such as Primera’s, that may have the potential to be disease-modifying and address the needs of those currently battling energy deficiency from genetic-based primary mitochondrial diseases.”

Mayflower BioVentures, which supported the launch of Primera was created in September by the Mayo Clinic, Hibiscus BioVentures, and Innoforce as a cell and gene therapy accelerator to help launch companies addressing unmet medical needs. The academic-industry partnership seeks to establish independent cell and gene therapy companies to advance the development of Mayo Clinic technologies through preclinical and early feasibility studies.

Also of Interest