Editas signs genome-edited stem cell pact with GSK, Biogen biotech partner

Gene editing biotech Editas Medicine ($EDIT) has signed a three-year R&D collab deal with Milan-based San Raffaele Telethon Institute for Gene Therapy to develop next-gen stem and T-cell therapies.

The Italian institute (also known as SR-TIGET)--which is the result of joint collab between Fondazione Telethon and Ospedale San Raffaele--will work with Cambridge, MA-based CRISPR biotech Editas to research and develop genome edited hematopoietic stem cell and T-cell therapies.

The institute already works with GlaxoSmithKline ($GSK) to develop its recently approved (in Europe) stem cell therapy Strimvelis for the incredibly rare condition known colloquially as “bubble boy syndrome.” Last January, SR-TIGET also signed a collab with Big Biotech Biogen ($BIIB) to jointly develop gene therapies for the treatment of both hemophilia A and B.

The scientific work at SR-TIGET in Milan, Italy, will be led by institute director Dr. Luigi Naldini, a world-renowned expert in lentiviral gene therapy and hematology. Lentiviral vectors are engineered viruses used to deliver working versions of defective genes responsible for causing certain diseases into the patients’ cells. 

Stated goals of the tie-up--financial details of which were not revealed--include the development of gene correction strategies for the treatment of rare diseases, with two specified indications in the blood and bone marrow. Further details were not shared.

“Dr. Naldini and SR-TIGET are world leaders in gene therapy,” said Katrine Bosley, CEO at Editas Medicine. "They have pioneered many important scientific advancements working with cells of the immune system and have extensive expertise in translating that work into cell-based therapies. We believe there will be great synergy across our scientific teams through this collaboration.”

Editas pulled off a $94.4 million IPO earlier this year, coming despite strong headwinds in the market that blew so many other biotechs off course. Its primary focus has been on the gene-editing technique CRISPR-Cas9 and is slated to begin human trials of its tech next year.

It has however fallen behind the University of Pennsylvania in being the first to trial CRISPR in the clinic, as it gained the NIH go-ahead for a study last month, with the FDA now to make the final decision on whether it can start this year.

Chinese researchers however last week announced they will likely pip Penn’s academics to the post, as they have been given the green light as well--but could start as soon as next month.

Editas is currently working on a number of projects, including a Duchenne muscular dystrophy program, as well as on researching hematopoietic stem/progenitor cells (HSPCs).

In its latest HSPC data, released in May, the biotech showed long-term engraftment in vivo after Cas9-mediated genome editing. These data also showed that editing did not alter hematopoietic reconstitution or differentiation properties in comparison to untreated control HSPCs.  

“My team and I believe genome editing is a promising answer to advance medicines to treat technically challenging diseases,” added Dr. Naldini. “We at San Raffaele Telethon Institute for Gene Therapy have worked for years to develop targeted integration of therapeutic genes into T cells and HSCs, and the collaboration with Editas Medicine represents an important opportunity to develop more effective and safe therapies for patients in the years ahead.”

Editas' shares ended more than 4% up at close yesterday (before the institute deal was announced). 

- check out the release

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