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Biotechs Join Forces for Off-the-Shelf Stem Cells

Crispr genome edits illustration

(NIH.gov)

3 April 2019. Two biotechnology enterprises working in genome editing and regenerative medicine are sharing their technologies to develop off-the-shelf cell therapies. Financial aspects of the licensing deal and research collaboration between Editas Medicine Inc. and BlueRock Therapeutics LP, both located in Cambridge, Massachusetts, were not disclosed.

Editas Medicine, founded in 2013 by geneticist Feng Zhang and colleagues from Broad Institute, a genomics research center affiliated with Harvard University and MIT, commercializes research from their labs on genome editing known as Crispr, short for clustered regularly interspaced short palindromic repeats. Crispr is based on bacterial defense mechanisms that use ribonucleic acid or RNA to identify and monitor precise locations in DNA. The actual editing is done by enzymes such as Cas9 or Cpf1.

Since its founding, Editas is advancing several therapies with genome editing to treat inherited diseases, with a therapy for leber congenital amaurosis, a rare inherited eye disease, which received clearance to begin clinical trials. But the company is also collaborating with Juno Therapeutics on genome editing of T-cells used in cancer therapy.

BlueRock Therapeutics develops treatments with engineered induced pluripotent stem cells, also known as iPSCs or adult stem cells since they’re derived from existing human rather than embryonic tissue. A spin-off company founded in 2016 by Versant Ventures and Bayer, BlueRock licenses stem cell technology from the lab of Kyoto University cell biologist and 2012 Nobel laureate Shinya Yamanaka, a pioneer in the reprogramming of induced pluripotent stem cells.

BlueRock’s technology uses gene editing to engineer adult stem cells for treatments in neurology, cardiology, and immunology. The company says its processes make it possible to produce stem-cell treatments with high therapeutic payloads and low toxicity to patients. One of Bluerock’s therapies, a treatment for Parkinson’s disease, expects to begin a clinical trial this year.

The companies plan to develop allogenic stem cell treatments that provide the same regenerative benefits, but are produced with stem cells from any donors. The deal calls for BlueRock to gain a non-exclusive license to Editas Medicine’s Crispr genome-editing processes, with Editas gaining access to BlueRock’s adult stem cell and cell transformation technologies. The collaboration is expected to enable Editas to develop new treatments for solid tumor and blood-related cancers, while BlueRock extends its work in neurology, cardiology, and immunology.

The agreement calls for each company to make developmental, regulatory, and commercial milestone payments, as well as royalties on sales, to the other party for all licensed therapies created from the collaboration. The dollar amounts of those payments were not disclosed.

“BlueRock and Editas share a common belief in the disruptive potential of utilizing an engineered cell as a therapeutic,” says BlueRock CEO Emile Nuwaysir in a joint statement. “We are equally committed and passionate about bringing these new treatment options to those living with diseases where the unmet medical need is high and growing.”

“We believe combining CRISPR-based genome editing with cell therapy has the potential to deliver game-changing allogeneic medicines,” notes Editas’s interim CEO Cindy Collins, “and we are excited to work with the team at BlueRock to develop genome-edited iPSCs with the potential to enable and accelerate the development of numerous, transformative medicines for people with many serious diseases.”

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