Sarep­ta snags its part­ner's CSO, Louise Rodi­no-Kla­pac, to lead new gene ther­a­py unit


Putting one more ar­row in its mus­cu­lar dy­s­tro­phy quiver, Sarep­ta has snagged a top ex­ec­u­tive from one of its part­ners to lead the com­pa­ny’s new­ly-cre­at­ed gene ther­a­py unit. Louise Rodi­no-Kla­pac is com­ing on board as vice pres­i­dent of gene ther­a­py.

Rodi­no-Kla­pac is com­ing from Sarep­ta’s part­ner Na­tion­wide Chil­dren’s Hos­pi­tal, where she was head­ing up the lab­o­ra­to­ry for gene ther­a­py re­search for mus­cu­lar dy­s­tro­phies. Her work has in­volved 11 in­ves­ti­ga­tion­al new gene ther­a­py drugs, and six that have reached hu­man tri­als. Sarep­ta is part­nered with the hos­pi­tal on a mi­cro-dy­s­trophin Duchenne gene ther­a­py that got IND clear­ance last No­vem­ber.

Doug In­gram

But Rodi­no-Kla­pac al­so has ties to an­oth­er Sarep­ta part­ner — she’s CSO of My­onexus, the com­pa­ny Sarep­ta li­censed five gene ther­a­pies from ear­li­er this month. The deal al­lowed Sarep­ta to edge in­to Limb-gir­dle mus­cu­lar dy­s­tro­phies, pay­ing My­onexus $60 mil­lion up­front with $45 mil­lion in po­ten­tial mile­stones — and snar­ing a buy­out op­tion at proof-of-con­cept.

She’s keep­ing her gig at My­onexus, too, ac­cord­ing to Sarep­ta’s state­ment.

“Mak­ing an im­pact on the lives of pa­tients af­fect­ed by mus­cu­lar dy­s­tro­phies has been cen­tral to my pro­fes­sion­al work,” said Rodi­no-Kla­pac. “Sarep­ta’s RNA plat­forms and mul­ti­ple gene ther­a­py pro­grams pro­vide sig­nif­i­cant op­por­tu­ni­ty to make a pro­found dif­fer­ence for pa­tients with neu­ro­mus­cu­lar dis­eases. I’m ex­cit­ed to join the Sarep­ta team lead­ing its gene ther­a­py de­vel­op­ment ef­forts, and share the or­ga­ni­za­tion’s ur­gency to ad­vance its ro­bust pipeline of po­ten­tial ther­a­pies.”

Sarep­ta’s CEO Doug In­gram had this to say:

Her ap­point­ment to lead our gene ther­a­py di­vi­sion per­fect­ly ex­em­pli­fies our strat­e­gy of quick­ly be­com­ing among the most mean­ing­ful glob­al ge­net­ic med­i­cine com­pa­nies by re­tain­ing and nur­tur­ing the field’s best and bright­est gene ther­a­py and neu­ro­mus­cu­lar sci­en­tists. It would be chal­leng­ing to find a sci­en­tist that has matched Dr. Rodi­no-Kla­pac’s record of ad­vanc­ing our un­der­stand­ing of gene ther­a­py and its ap­pli­ca­tion to neu­ro­mus­cu­lar con­di­tions while ac­cel­er­at­ing bench-side re­search in­to hu­man clin­i­cal tri­als.


Im­age: Louise Rodi­no-Kla­pac. NA­TION­WIDE CHIL­DREN’S HOS­PI­TAL

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis chips in $10M for IPO-bound part­ner Pli­ant; Tenax shares soar on heart drug da­ta

Novartis is coming in with $10 million to help support the looming IPO of a partner. Pliant Therapeutics posted a new filing with the SEC showing that Novartis is buying the shares at $15, the mid-point of the range. It’s adding several million shares to the offering, bringing the total to around $135 million. Biotech companies have been enjoying quite a run on virtual Wall Street, with investors boosting new offerings to some big hauls.

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bris­tol My­ers Squib­b's just-launched MS drug Zeposia makes the cut in key ul­cer­a­tive col­i­tis tri­al

In March, Zeposia became the third oral S1P modulator to secure US approval for multiple sclerosis. Now, the drug has succeeded in a key ulcerative colitis study.

The immunomodulator, akin to others in its class, controls lymphocyte trafficking by limiting the white blood cells to the lymphatic system, in the lymph nodes, and thwarting their ability to jam up lymph nodes — precluding their ability to penetrate the bloodstream and the central nervous system.

Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.