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 firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Getting a foot in the door in Boston’s bustling biopharma hub is a rite of passage for many companies, but it comes with a steep price tag. Lithuanian CDMO Northway — now with a new moniker — will set up a new plant in close proximity, and it’s hoping its biologics focus will find a willing customer base.

Northway Biotech (formerly Northway Biotechpharma) on Wednesday held a virtual grand opening ceremony for its $40 million Waltham, MA facility — a 30,000 square-foot cGMP manufacturing and process development plant that will widely expand on the company’s previous capabilities.

The wave of biotech IPOs we’ve been seeing in the last few days underscores that the public markets remain one of the key channels for fresh investments in drug R&D. And that trend was in full view this week as a slate of biotechs nailed down hundreds of millions of dollars in fresh funds.

One of the big winners of the week is Editas $EDIT, which nailed $231 million to back its pioneering work on a gene editing platform. The biotech sold 3.5 million shares at $66 each.

About 15 months since closing a $28 million Series A, a San Diego protein-degradation upstart returned to the venture well Thursday with an extension of that round and some new hires, including one of the city’s best-connected biotech execs.

Plexium has bagged an additional $35 million in financing, the biotech said, money that will push undisclosed oncology and immuno-oncology programs into the clinic. In addition, longtime industry vet Mike Grey is jumping on as chairman of the board, and two others from Thursday’s leads — Adam Goulburn from Lux Capital and Rob Hopfner from Pivotal BioVentures — joined the board too.

Seven years after the FDA first approved Esbriet, the blockbuster Roche IPF drug is getting an expedited review for a second indication.

On Thursday, the agency gave Esbriet priority review for unclassified interstitial lung diseases, or forms of pulmonary inflammation and scarring that don’t fit easily into the over 200 known types of ILD. The move comes 10 months after Esbriet received breakthrough designation and sets Roche up for a decision by May.