Jennifer Doudna and Jonathan Weissman. BARBARA RIES for UCSF

GSK's Bar­ron joins hands with Doud­na, Weiss­man to de­ploy CRISPR tech in drug dis­cov­ery

When Hal Bar­ron un­veiled his grand plan to turn Glax­o­SmithK­line’s R&D group around, he cham­pi­oned ge­net­ics as one of two key fo­cus­es for the UK phar­ma gi­ant, an­nounc­ing a dis­cov­ery deal with 23andme and pledg­ing to fo­cus more on CRISPR tech as well as ma­chine learn­ing. A year lat­er, the R&D chief is build­ing on that promise as he seals a new pact with two promi­nent CRISPR re­searchers in his neigh­bor­hood in San Fran­cis­co.

Jen­nifer Doud­na and Jonathan Weiss­man, both in­ves­ti­ga­tors at Howard Hugh­es Med­ical In­sti­tute, are throw­ing their weight be­hind a new col­lab­o­ra­tion space dubbed the Lab­o­ra­to­ry for Ge­nomics Re­search.

“LGR is about build­ing that space where cre­ative sci­ence is part­nered with the de­vel­op­ment of ro­bust tech­nol­o­gy that will help de­vel­op to­mor­row’s drugs,” Doud­na, who pi­o­neered the use of CRISPR gene edit­ing tool in hu­mans, said in a state­ment. “I think we’re go­ing to be able to do sci­ence that none of us can even imag­ine to­day.”

Hal Bar­ron GSK

Click on the im­age to see the full-sized ver­sion

Un­like the CRISPR biotechs that Doud­na has helped found, though, LGR will use the tools not to cre­ate ther­a­peu­tics or di­ag­nos­tics, but to un­cov­er how ex­act­ly gene mu­ta­tions cause dis­ease. By tin­ker­ing with one gene at a time and then com­par­ing the da­ta on a mas­sive scale, the goal is to iden­ti­fy po­ten­tial tar­gets for ge­net­ic dis­eases.

In par­tic­u­lar, the lab will fo­cus on im­munol­o­gy, on­col­o­gy and neu­ro­science.

This marks the sec­ond ini­tia­tive that Weiss­man — a func­tion­al ge­nomics ex­pert at UCSF— is di­rect­ing with fel­low Howard Hugh­es Med­ical In­sti­tute in­ves­ti­ga­tor Doud­na, who’s based out of UC Berke­ley. The duo are co-di­rec­tors of the In­no­v­a­tive Ge­nomics In­sti­tute, which has a broad mis­sion to im­prove pub­lic health with CRISPR.

Be­tween the two UC in­sti­tu­tions 24 full-time staffers will be hired at the LGR, work­ing along­side 14 GSK em­ploy­ees in a space near UCSF’s Mis­sion Bay cam­pus and run­ning on a $67 mil­lion bud­get over five years. They will get a hand from GSK’s ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing group on build­ing the tools need­ed to an­a­lyze all the da­ta. Chris Miller, the new head of func­tion­al ge­nomics GSK scooped from Ab­b­Vie, will al­so help steer the ship.

The an­nounce­ment to­day is a cul­mi­na­tion of dis­cus­sions and ne­go­ti­a­tions be­tween GSK and the two uni­ver­si­ties for over a year, Bar­ron told STAT. Not long af­ter be­gin­ning his cur­rent CSO role, he sat down with Doud­na about har­ness­ing the lat­est CRISPR ad­vances for drug dis­cov­ery, and with­in min­utes there was “in­cred­i­ble ex­cite­ment” around the project.

For the drug­mak­er, it is an op­por­tu­ni­ty to test a hy­brid mod­el bring­ing in­dus­try and acad­e­mia un­der one roof, in­stead of sim­ply li­cens­ing tech or fos­ter­ing biotech spin­outs. Aside from cer­tain ex­clu­sive li­cens­es for GSK, the tools cre­at­ed in the lab will be ac­ces­si­ble to oth­er re­searchers.

“One of our key goals is to ad­vance the field over­all and make these tools as broad­ly avail­able as pos­si­ble,” Weiss­man said. “The LGR screen­ing cen­ter will en­able labs at UCSF and Berke­ley. Hav­ing ac­cess to it will give our sci­en­tists op­por­tu­ni­ties to ad­vance their re­search in ways that would be very hard for them to do in their own labs.”

Im­age: Jen­nifer Doud­na Jonathan Weiss­man by Bar­bara Ries for UCSF

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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