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.”

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

It’s been a busy fall on Wall Street — and this week, we’ve got another biotech going public after Ventyx yesterday.

Massachusetts solid-tumor biotech Xilio Therapeutics made its public debut today, joining 149 other biotechs that have gone public so far this year.

Back on October 1st, Xilio filed its S-1 with the SEC to make its IPO debut for $100 million — the next step in financing after completing a $95 million Series C back in February.