One of Ver­tex’s longest-tenured em­ploy­ees be­lieves he can take the biotech’s biggest med­ical and sci­en­tif­ic ac­com­plish­ments and use it to de­vel­op treat­ments for more than just cys­tic fi­bro­sis.

Three years ago, Jonathan Moore, a sci­en­tist and then ex­ec­u­tive at Ver­tex from 1990 to 2018, found­ed a com­pa­ny to de­vel­op treat­ments for dis­eases that, like CF, are caused by mu­ta­tions in a “su­per fam­i­ly” of pro­teins known as ABC trans­porters.

Jonathan Moore

He’s since man­aged to con­vince a few in­vestors. On Thurs­day, Moore, At­las Ven­ture and a hand­ful of oth­er blue-chip funds de­buted Rec­ti­fy Phar­ma­ceu­ti­cals, backed with $100 mil­lion in Se­ries A funds to find small mol­e­cules that can cor­rect such mu­ta­tions in a broad suite of ge­net­ic dis­or­ders. They’ll start in the liv­er, but even­tu­al­ly plan to hit dis­eases in near­ly every or­gan in the body.

“These are ex­pressed in all dif­fer­ent or­gans, liv­er, lungs, brain, and they play an im­por­tant role in the body,” CEO Ra­jesh De­vraj told End­points News. “You can imag­ine the amount of tar­gets that are avail­able and de­liv­ered for us to go af­ter.”

The new com­pa­ny builds off Ver­tex’s suc­cess in de­vel­op­ing a type of drug that es­sen­tial­ly had nev­er been built be­fore, a small mol­e­cule that can take a mu­tant, dys­func­tion­al pro­tein and morph it back in­to a func­tion­al one.

That work, con­ceived of and fund­ed in part by the CF Foun­da­tion, ul­ti­mate­ly led to four it­er­a­tions of mol­e­cules and com­bi­na­tions of mol­e­cules that over a decade turned one of the most com­mon ge­net­ic dis­eases from a like­ly death sen­tence in­to a treat­able con­di­tion for most pa­tients.

The pro­tein be­hind CF, known as CFTR, is not unique, though. It’s a trans­port pro­tein, shep­herd­ing salts in­to and out of cells. And the con­stant­ly whirling, busy, huffy place that is the hu­man body has many such trans­port pro­teins — 48 to be ex­act. Ac­cord­ing­ly, hu­mankind has many dis­eases as­so­ci­at­ed with mu­ta­tions in those pro­teins.

There are nu­mer­ous ben­e­fits to go­ing af­ter this class of dis­ease, De­vraj points out: They are mono­genic and re­searchers can be cer­tain that, if they suc­cess­ful­ly cor­rect the pro­tein, they can slow or re­verse the dis­ease; and it’s been done be­fore, in­clud­ing by Rec­ti­fy’s founder.

Small mol­e­cules are al­so eas­i­er to de­vel­op, make and de­liv­er than gene ther­a­py or oth­er bi­o­log­ic drugs, es­pe­cial­ly for rare dis­eases that of­ten get over­looked by drug de­vel­op­ers.

“We are — I think we are the foun­da­tion,” De­vraj said. “We are the lead­ers in ABC trans­porter bi­ol­o­gy.”

But there are al­so key hur­dles. Al­though Ver­tex proved that one could de­vel­op a small mol­e­cule to trans­form a mu­tant pro­tein, no one has yet done it suc­cess­ful­ly for any oth­er dis­ease. The big biotech has had its own trou­bles ap­ply­ing the strat­e­gy to a new con­di­tion.

Among the biggest chal­lenges with these dis­eases, De­vraj ac­knowl­edged, is that of­ten dif­fer­ent pa­tients with a sin­gle dis­ease have dif­fer­ent ge­net­ic mu­ta­tions. Like words, genes can be mis­pelled in any num­ber of ways.

That means Rec­ti­fy will po­ten­tial­ly need dif­fer­ent drugs or dif­fer­ent com­bi­na­tions of drugs to fix all or most of the mu­ta­tions in a giv­en dis­ease. It took Ver­tex a decade to find the right tri­ad of mol­e­cules that work for 90% of pa­tients’ CF and it could be even hard­er, both sci­en­tif­i­cal­ly and eco­nom­i­cal­ly, to do the same for dis­or­ders that are even more rare — as the ones Rec­ti­fy pur­sues will al­most cer­tain­ly be.

To over­come those chal­lenges, Rec­ti­fy is ap­ply­ing some of the same tricks Ver­tex did, in­clud­ing de­vel­op­ing tests that, in the­o­ry, could al­low them to quick­ly tell in a lab whether a mol­e­cule is hav­ing its in­tend­ed ef­fect and restor­ing ship­ping lanes in­to and out of cells. De­vraj said they have al­ready de­vel­oped the first as­says but de­clined to de­scribe them.

The com­pa­ny is keep­ing its in­di­ca­tions undis­closed for now, too, ex­cept that they will start in the liv­er, be­fore hope­ful­ly ex­pand­ing to the lung and cen­tral ner­vous sys­tem. The cur­rent fi­nanc­ing, co-led by Omega, For­bion and Long­wood, should get them through proof-of-con­cept clin­i­cal tri­als for the first mol­e­cule.

“Noth­ing is go­ing to be easy,” De­vraj said. That’s “why we do this job.”

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.