Vertex ponies up $100M for CRISPR’s gene editing tech to develop new diabetes modality – Endpoints News

Ver­tex is pay­ing nine fig­ures to get its hands on CRISPR Ther­a­peu­tics’ gene edit­ing tech­nol­o­gy in a bid to make a third ap­proach for type 1 di­a­betes.

The biotech said Mon­day morn­ing that it is pay­ing $100 mil­lion up­front to nab non-ex­clu­sive rights to CRISPR Ther­a­peu­tics’ CRISPR/Cas9 gene edit­ing tech­nol­o­gy. The goal, ac­cord­ing to Ver­tex, is to move for­ward with pre­clin­i­cal de­vel­op­ment of a hy­poim­mune gene-edit­ed cell ther­a­py for type 1 di­a­betes.

The agree­ment al­so in­cludes an ad­di­tion­al $230 mil­lion for CRISPR Ther­a­peu­tics in mile­stones, as well as undis­closed roy­al­ties on any prod­uct that re­sults from the deal.

CRISPR and Ver­tex have a long his­to­ry that dates back to a 2015 deal on a gene edit­ing col­lab­o­ra­tion, go­ing af­ter sev­er­al tar­gets in dis­eases such as cys­tic fi­bro­sis.

The di­a­betes pro­gram at Ver­tex, mean­while, kicked off in 2019 af­ter the com­pa­ny ac­quired di­a­betes biotech Sem­ma Ther­a­peu­tics for $950 mil­lion in cash. That ac­qui­si­tion brought in pan­cre­at­ic islet cells that Ver­tex us­es in its two clin­i­cal-stage cell ther­a­pies for di­a­betes.

These two clin­i­cal pro­grams, VX-880 and VX-264, re­quire the use of an im­muno­sup­pres­sant or the use of a pro­pri­etary de­vice, re­spec­tive­ly, to shield the cells from be­ing rapid­ly de­stroyed by a per­son’s im­mune sys­tem.

A Ver­tex spokesper­son told End­points News that the hope for the deal with CRISPR is that Ver­tex can ed­it the islet cells us­ing CRISPR’s tech­nol­o­gy to make them in­vis­i­ble from the im­mune sys­tem, or make those cells able to evade the im­mune sys­tem, get­ting rid of any need of a de­vice or im­muno­sup­pres­sion.

The spokesper­son clar­i­fied that this pro­gram is dif­fer­ent from the one an­nounced be­tween CRISPR Ther­a­peu­tics and Vi­a­Cyte, which was ac­quired by Ver­tex in 2022. While it is the same idea in terms of ap­proach, the cells be­ing used in that col­lab­o­ra­tion are dif­fer­ent — and the ther­a­py is cur­rent­ly in the mid­dle of a Phase I/II tri­al.

This is one of the newest de­vel­op­ments for Ver­tex’s di­a­betes pipeline — with the an­nounce­ment com­ing down less than three weeks af­ter Ver­tex’s IND for VX-264 was ap­proved by US reg­u­la­tors. The ther­a­py is cur­rent­ly in a Phase I/II tri­al in Cana­da.