The FDA is spelling out the de­tails of a new path­way to help speed per­son­al­ized cell and gene ther­a­pies to mar­ket for rare dis­eases.

Mon­day's long-await­ed draft guid­ance out­lines the agency’s “plau­si­ble mech­a­nism” frame­work, a path­way FDA Com­mis­sion­er Mar­ty Makary and vac­cines chief Vinay Prasad un­veiled last fall for drug de­vel­op­ers pur­su­ing be­spoke ther­a­pies.

The guid­ance de­tails a path to mar­ket for ther­a­pies where a ran­dom­ized tri­al is not fea­si­ble and where there's a spe­cif­ic ge­net­ic, cel­lu­lar or mol­e­c­u­lar ab­nor­mal­i­ty that can be al­tered or cor­rect­ed. Genome edit­ing and RNA-based ther­a­pies, in­clud­ing an­ti­sense oligonu­cleotides, are the fo­cus of the 22-page draft. But the agency notes that the gen­er­al con­cepts may ap­ply to oth­er types of in­di­vid­u­al­ized ther­a­pies.

Fortress Biotech and its sub­sidiary Cypri­um have agreed to sell a pri­or­i­ty re­view vouch­er for $205 mil­lion, one of the high­est pub­licly-an­nounced price tags in re­cent years for the right to a po­ten­tial reg­u­la­to­ry short­cut.

Pri­or­i­ty re­view vouch­ers, or PRVs, are award­ed by the FDA to cer­tain com­pa­nies that de­vel­op treat­ments for rare pe­di­atric dis­eases. The vouch­ers may be trans­ferred or sold, and once re­deemed, they can short­en the FDA’s stan­dard 10-month re­view pe­ri­od for a dif­fer­ent drug to a tar­get of about six months.

Cypri­um had re­ceived its vouch­er in con­nec­tion with the agency’s ap­proval of Zy­cubo to treat chil­dren with a rare neu­rode­gen­er­a­tive dis­or­der called Menkes dis­ease. Fortress didn’t dis­close a buy­er, but said 20% of the pro­ceeds will go to the Eu­nice Kennedy Shriv­er Na­tion­al In­sti­tute of Child Health and Hu­man De­vel­op­ment.

The Chi­nese DNA se­quenc­ing com­pa­ny MGI has sold its US sub­sidiary Com­plete Ge­nomics, fol­low­ing a leg­isla­tive crack­down that blunt­ed its ex­pan­sion plans.

Swiss-based life sci­ences com­pa­ny Swiss Rock­ets will buy Com­plete Ge­nomics, and in the sec­ond quar­ter it will launch a DNA se­quencer in the US and be­yond, the com­pa­nies said Mon­day. Terms of the deal were not dis­closed. It ap­pears Swiss Rock­ets hopes to elim­i­nate geopo­lit­i­cal fric­tion as it takes on mar­ket leader Il­lu­mi­na.

More than two years ago, MGI ramped up US ex­pan­sion plans, say­ing its ma­chines can de­code an en­tire genome for less than $100 — an im­por­tant bench­mark in the high-cost world of re­search. Il­lu­mi­na has said its flag­ship ma­chine put the com­pa­ny on the path to the $100 genome, but hasn’t talked about a price point less than this amount.