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The 20 under 40 : Inside the next generation of biopharma leaders

Inside the next generation of biopharma leaders – Endpoints News

“Each gen­er­a­tion needs a new mu­sic,” Fran­cis Crick wrote in 1988, re­flect­ing back on his land­mark dis­cov­ery. Crick was 35, then, in 1953, when he be­gan work­ing with a 23-year-old named James Wat­son, and 37 when the pair un­veiled the dou­ble he­lix. Ros­alind Franklin, whose dif­frac­tion work un­der­gird­ed their met­al mod­el, was 32.


The mod­el would be­come the score for a new era in bi­ol­o­gy, one de­vot­ed to crack­ing the ba­sic struc­tures turn­ing in­side life. Sub­se­quent years would bring new con­duc­tors and new rhythms: Robert Swan­son, 29 when he con­vinced a 39-year-old Herb Boy­er to build a com­pa­ny off his work and call it Genen­tech; Phillip Sharp, 29 when he dis­cov­ered RNA splic­ing and 34 when he co-found­ed Bio­gen; Frances Arnold, 36 when she pi­o­neered di­rect­ed evo­lu­tion; Feng Zhang, 31 when he pub­lished his CRISPR pa­per.


You can think of the list be­low as a playlist of 20 songs. We’ve brought you 20 re­searchers and ex­ec­u­tives think­ing through ideas that may suc­ceed or sput­ter, but will cer­tain­ly help de­fine the sound­track for the next 20 years of bio­phar­ma: com­put­er sci­en­tists crack­ing gene net­works and de­sign­ing bi­o­log­i­cal search en­gines, ven­ture cap­i­tal­ists bur­row­ing re­search pipelines in­to uni­ver­si­ties, and mi­cro­bi­ol­o­gists cre­at­ing an­tibi­otics ‘crap­sules’ from fe­ces.


KATH­LEEN MC­CARTHY – Sky­hawk Ther­a­peu­tics – 35


A sci­en­tist cor­rals top tal­ent in her en­tre­pre­neur­ial ride


U-2 Drag­on La­dy, one of the con­fer­ence rooms at Sky­hawk Ther­a­peu­tics’ of­fice in Waltham, MA, is named af­ter both the Amer­i­can sin­gle-jet en­gine, high al­ti­tude re­con­nais­sance air­craft and founder Kath­leen Mc­Carthy.


Speak­ing from the Starfire room, Mc­Carthy told End­points News that it all arose from serendip­i­ty: They’d named the com­pa­ny Sky­hawk be­cause it sound­ed like a ma­jes­tic soar­ing bird, on­ly to re­al­ize it’s al­so a Viet­nam era plane that Sen­a­tor John Mc­Cain used to fly. So when it came time to name the con­fer­ence rooms — a team-build­ing ex­er­cise — fight­er jets be­came the ob­vi­ous theme.


From the be­gin­ning of her ca­reer, Mc­Carthy has been drawn to small or­ga­ni­za­tions that re­sem­ble a light at­tack air­craft try­ing to have a big im­pact.


Long be­fore she built Sky­hawk to its cur­rent size of over 70 — even be­fore the com­pa­ny was called Rare Genetx and con­sist­ed of just her­self work­ing out of a base­ment in the Cam­bridge Bi­o­Labs — as an un­der­grad at Welles­ley, Mc­Carthy start­ed a non-prof­it to nurse kids in a poor neigh­bor­hood in the Do­mini­can Re­pub­lic. Through that project she met Bill Haney, a neigh­bor of a doc­tor she was work­ing with, who gave them the first grant to start a preschool and would lat­er be­come Sky­hawk’s an­gel in­vestor and co-founder. It al­so reaf­firmed her in­ter­est in work­ing at the in­ter­sec­tion of ap­plied sci­ence and pub­lic health.


Af­ter com­plet­ing a Ful­bright pro­gram in Switzer­land, Mc­Carthy land­ed her first job in the Spinal Mus­cu­lar At­ro­phy Foun­da­tion as it was just be­gin­ning mouse mod­el work for a small mol­e­cule drug dis­cov­ered by part­ners at PTC Ther­a­peu­tics. As one of five em­ploy­ees, she learned to mul­ti­task dif­fer­ent types of projects, or­ga­niz­ing meet­ings with ex­perts, an­a­lyz­ing da­ta, de­sign­ing new ex­per­i­ments and even mak­ing pre­sen­ta­tion slides and eval­u­at­ing bud­gets.


“They kind of promised me that no day would be the same, and that’s ac­tu­al­ly what hap­pened,” she said.


All of that di­verse work fed in­to one ul­ti­mate goal: to ad­vance ris­diplam, a small mol­e­cule drug in­vent­ed by Sergey Paushkin, who moved from PTC to the SMA Foun­da­tion (and is now VP of bi­ol­o­gy at Sky­hawk).


So when Roche got in­ter­est­ed in the po­ten­tial of ris­diplam to in­crease the lev­els of the es­sen­tial SMN pro­tein and im­prove sur­vival, Mc­Carthy was re­cruit­ed to the phar­ma gi­ant’s rare dis­ease unit as a non-clin­i­cal phar­ma­col­o­gist. Aside from the SMA drug, which is now on the cusp of an OK, she al­so con­tributed to sev­er­al oth­er projects.


As her team be­gan to back-en­gi­neer how ris­diplam worked around 2014 and 2015, though, they came up with some sur­pris­ing find­ings. Chief among them was the re­al­iza­tion that the mol­e­cule was di­rect­ly bind­ing to RNA — or more specif­i­cal­ly, mod­i­fy­ing how pre-mR­NA is spliced, there­by spurring the pro­duc­tion of func­tion­al SMN pro­tein.


“That was enough for me to say you know, I think I have enough in­for­ma­tion,” she said. “I def­i­nite­ly don’t have the full sto­ry, I don’t know how broad­ly ap­plic­a­ble this is, but my hunch was like okay, this is re­al­ly ex­cit­ing, su­per-nov­el, I’m gonna go try and raise some mon­ey to start a com­pa­ny.”


She was ad­vised to get go­ing ear­ly, and she did. Bal­anc­ing the sci­ence (by con­tract­ing out to CROs) and busi­ness (hir­ing IP lawyers to start grab­bing patents) sides of things wasn’t as chal­leng­ing as fig­ur­ing out how much they could raise from in­vestors.


Her sis­ter, Mau­ra, who used to work in ven­ture cap­i­tal be­fore join­ing Sky­hawk’s cor­po­rate de­vel­op­ment team, served as a guide­post at those mo­ments.


“When we got re­ject­ed, which we got re­ject­ed a lot, she would say, you know, it doesn’t mat­ter, you’re gonna have to do 40 of these, or 50 of these, to even­tu­al­ly raise the mon­ey,” she re­called. Then there was the sci­ence back­ing her up: “I knew I had some re­al­ly ex­cit­ing, unique re­sults. I knew oth­er peo­ple did not have or know, or weren’t tar­get­ing RNA the same way I was think­ing about it.”


Hav­ing an ex­ten­sive net­work helped, too, with Haney and Mike Luzzio — a Bio­gen vet and Sky­hawk’s first em­ploy­ee — each bring­ing in dif­fer­ent con­tacts. Draw­ing from her li­aisons dat­ing back to the SMA Foun­da­tion days, Mc­Carthy al­so got lead­ing RNA re­searchers Adri­an Krain­er and Fred Al­lain ear­ly on a star-stud­ded sci­en­tif­ic ad­vi­so­ry board.


Sky­hawk came of age at a time enough ri­val biotechs are spring­ing up, from Ar­rakis to Ri­bometrix, to con­vince VCs that the mo­ment for RNA-tar­get­ing small mol­e­cule drugs has ar­rived, she added.


The con­stel­la­tion has en­abled Sky­hawk to fol­low up its first deal, a $60 mil­lion up­front arrange­ment with Cel­gene, with a slate of new pacts fea­tur­ing GSK, Mer­ck, Take­da and Bio­gen.


“The over­all ques­tion is how broad­ly do these things ap­ply. And then if it ap­plies every­where, how do you ef­fec­tive­ly in­ter­ro­gate each type of tar­get and fig­ure out what the list of tar­gets are re­lat­ed to those,” she said. “So that’s a re­al­ly fun dis­cov­ery ques­tion.”


It’s a ques­tion that the sci­en­tist in Mc­Carthy is ex­cit­ed to find the an­swer to. And the pub­lic health ad­vo­cate in her is itch­ing to see the ben­e­fits reach small­er and small­er pa­tient pop­u­la­tions, down to n of 1, suf­fer­ing from sin­gle-point ge­net­ic mu­ta­tions. It’s a high-in-the-sky dream, she reck­oned, but she’s ready to build the fleet need­ed to soar to those heights.

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Maura McCarthy


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