Less than two years af­ter sell­ing her first biotech to Pfiz­er for a hefty $810 mil­lion, a French re­searcher has launched a new gene ther­a­py out­fit that’s fo­cus­ing on the same tar­get — dwarfism — but tak­ing a dif­fer­ent path to treat­ment.

The sci­en­tist is Elvire Gouze and the com­pa­ny is In­noSkel, which launched with €20 mil­lion in Se­ries A fi­nanc­ing (about $24.2 mil­lion) ear­ly Mon­day. Though In­noSkel is aim­ing to treat a whole host of rare skele­tal dis­eases that af­fect the body’s col­la­gen, it’s first go­ing af­ter what’s known as spondy­loepi­phy­seal dys­pla­sia con­geni­ta (SEDc), the sec­ond-lead­ing cause of dwarfism that stunts the growth of a pa­tient’s car­ti­lage.

Mon­day’s fundraise was co-led by Jeito Cap­i­tal and Arie Bellde­grun’s team at Vi­da Ven­tures. Ré­gion Sud In­vestisse­ment and The Turenne Group al­so par­tic­i­pat­ed in the round.

Gouze had been work­ing on skele­tal dys­pla­sia at In­serm, the French equiv­a­lent of the NIH, and helped launch the new­ly Pfiz­er-owned Ther­a­chon. She kept her aca­d­e­m­ic lab while work­ing there and came up with the idea for a gene ther­a­py in type 2 col­lagenopathies, which in­cludes SEDc.

“That’s how we start­ed In­noSkel, it’s a spin-out of my In­serm lab,” Gouze told End­points News. 

There are two main caus­es of dwarfism, Gouze said, the first of which was a lead­ing tar­get for Ther­a­chon. While the biotech’s re­search cen­ters al­so re­volve around rare mus­cu­loskele­tal dis­or­ders, Ther­a­chon’s mis­sion was to treat the com­pli­ca­tions of achon­dropla­sia like those in the body’s car­dio­vas­cu­lar, neu­ro­log­i­cal and meta­bol­ic sys­tems.

Achon­dropla­sia af­fects bone growth and aris­es from a sep­a­rate ge­net­ic mu­ta­tion, re­sult­ing in a com­plete­ly dif­fer­ent mech­a­nism but sim­i­lar char­ac­ter­is­tics of dwarfism as SEDc. Ther­a­chon’s ex­per­i­men­tal drug is an FGFR3 de­coy, which they be­lieve will cor­rect a sig­nal­ing path­way that trig­gers the ab­nor­mal growth.

Though both con­di­tions re­sult in in­di­vid­u­als with dwarfism hav­ing the same phys­i­cal ap­pear­ance, Gouze says the com­pli­ca­tions in this car­ti­lage-based dis­or­der are quite dif­fer­ent. Rather than their bones grow­ing ab­nor­mal­ly, pa­tients with SEDc see ef­fects in their growth plates — the car­ti­lage “tem­plate” of bones in chil­dren that haven’t formed yet — es­sen­tial­ly ne­ces­si­tat­ing treat­ment be­fore pu­ber­ty.

This can lead to reti­nal de­tach­ments and pa­tients hav­ing trou­ble breath­ing, Gouze said. Fur­ther dif­fi­cul­ties hold­ing up their heads and necks can re­sult in paral­y­sis.

“In this case, the pa­tients are very short and have se­vere com­pli­ca­tions,” Gouze said. “What we’re try­ing to achieve is to re­pair the ex­o­cyclic ma­trix of the car­ti­lage.”

In­noSkel isn’t ready to di­vulge how their pro­gram treats the con­di­tion, nor can Gouze pre­dict when they’ll first hit the clin­ic as the com­pa­ny is still await­ing the pub­li­ca­tion of a peer-re­viewed pa­per. Mon­day’s fund­ing will go to­ward ad­vanc­ing to hu­man tri­als, how­ev­er, and any prod­uct will look like a typ­i­cal gene-ther­a­py in­jec­tion to pro­vide pro­teins that help the car­ti­lage be­have nor­mal­ly, she said.

Try­ing to treat dwarfism is a cause that’s drawn sig­nif­i­cant op­po­si­tion from some cor­ners of the com­mu­ni­ty. It’s one Gouze is all too fa­mil­iar with giv­en her pre­vi­ous role as sci­en­tif­ic founder of Ther­a­chon, and now she’s back for more with In­noSkel. There have al­so been ef­forts by a promi­nent phar­ma, Bio­Marin, to get an achon­dropla­sia drug across the fin­ish line. The FDA ac­cept­ed Bio­Marin’s pitch for the pro­gram just last month, with an ac­tion date com­ing next Au­gust.

But Gouze says she “ful­ly re­spects” the ar­gu­ment from ad­vo­cates that dwarfism is not some­thing meant to be “cured.” Mak­ing every­one taller is not Gouze’s main goal, she says, rather it’s try­ing to pre­vent the com­pli­ca­tions that re­sult from achon­dropla­sia and now SEDc.

“When you’re an adult with dwarfism you have to live with your con­di­tion any­way, and it’s not to make them like every­body; the treat­ments are not try­ing to ‘stan­dard­ize’ them,” Gouze said. “It’s not so much to make every­body look the same, which would be use­less and ab­solute­ly not in­ter­est­ing any­way. It’s try­ing to pre­vent the com­pli­ca­tions and de­crease the pain.”

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

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While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.