Sarep­ta picks up a slate of pre­clin­i­cal gene ther­a­py pro­grams, seed­ing new Stride­Bio deal with $48M up­front

Mavis Ag­band­je-McKen­na Stride­Bio

Hav­ing tast­ed ear­ly suc­cess in treat­ing a num­ber of mus­cu­lar dy­s­tro­phies with gene ther­a­py, Sarep­ta is set­ting it­self up to tack­le more rare dis­eases.

The Boston-based biotech has picked Stride­Bio for a part­ner­ship ev­i­dent­ly de­signed to run years, kick­ing things off with $48 mil­lion up­front and four cen­tral ner­vous sys­tem tar­gets. The small­er part­ner is tasked with all R&D work up to the IND stage, com­ing up with the AAV-based ther­a­pies to be test­ed in Rett syn­drome (MECP2), Dravet syn­drome (SCN1A), An­gel­man syn­drome (UBE3A) and Nie­mann-Pick (NPC1).

Co-found­ed by Mavis Ag­band­je-McKen­na and Ar­avind Asokan, Stride­Bio’s pitch cen­ters around its struc­ture-dri­ven cap­sid tech­nol­o­gy drawn from non-hu­man pri­mates, which it says can bet­ter di­rect these shells to tis­sues of in­ter­est and evade neu­tral­iz­ing an­ti­bod­ies. For Sarep­ta, the lure al­so lies in the po­ten­tial to re­dose pa­tients who have re­ceived AAV-de­liv­ered gene ther­a­pies be­fore.

Ar­avind Asokan Stride­Bio

Sarep­ta’s first for­ay in­to the field was a mi­crody­s­trophin ap­proach to Duchenne mus­cu­lar dy­s­tro­phy, an ex­ten­sion of its ef­forts with its mar­ket­ed ex­on-skip­ping drug Ex­ondys-51. In a re­cent bout, it ap­peared to score high­er than Pfiz­er in a Phase Ib, buoy­ing its prospects. Pre­lim­i­nary func­tion­al da­ta on a limb gir­dle mus­cu­lar dy­s­tro­phy ac­quired from My­onexus al­so ap­peared pos­i­tive.

Its new deal al­so pro­vides an ex­clu­sive op­tion to four ad­di­tion­al ge­net­ic tar­gets for CNS or neu­ro­mus­cu­lar ail­ments, which could cost up to $42.5 mil­lion up­front. Stride­Bio may co-de­vel­op and co-com­mer­cial­ize one of the eight to­tal prod­ucts — just the right num­ber for the small biotech, which is al­so jug­gling a pre­clin­i­cal pact with Take­da fea­tur­ing three tar­gets in­clud­ing Friedre­ich’s Atax­ia.

CEO Sapan Shah talked up the re­sources and ex­per­tise they will gain for ex­pand­ing their re­search and man­u­fac­tur­ing plat­form rapid­ly.

Sapan Shah Stride­Bio

“Our part­ner­ship with Stride­Bio ex­pands our re­search port­fo­lio by up to eight new tar­gets and, through our strate­gic part­ner­ing ap­proach that has our col­lab­o­ra­tor lead all IND-en­abling re­search and de­vel­op­ment, en­sures that we gain ac­cess to new tech­nol­o­gy and tar­gets while not dis­tract­ing Sarep­ta from its near-term pri­or­i­ties,” Sarep­ta chief Doug In­gram added in a state­ment.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Noubar Afeyan (Sebastien Micke/Paris Match/Contour by Getty Images)

As Mod­er­na rose, Flag­ship cashed in for $1.4B — with a lot more wealth still re­main­ing

For nearly a decade, Flagship poured record-setting levels of cash into Moderna, even as they faced setbacks on early programs and skeptics wondered whether the company’s science could ever match its hype.

Now that the science has delivered, Flagship is cashing in.

Over the last 13 months, since the World Health Organization declared a pandemic, Flagship has sold off Moderna shares worth $1.4 billion. The sales, first reported by Forbes, came as the Cambridge biotech’s shares soared from just under $20 per share on Jan. 3, 2020, to $169.50 when markets opened Thursday.

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