Edg­ing to­ward a piv­otal read­out, Bio­gen picks a new dis­cov­ery part­ner for oph­thalmic gene ther­a­py

Al­most two years af­ter Bio­gen swooped again in­to the oph­thalmic gene ther­a­py field with an $800 mil­lion buy­out of eye-fo­cused Night­star Ther­a­peu­tics, it’s turn­ing to a young play­er for a new pro­gram.

Ger­many’s Vi­Gen­eron will be de­ploy­ing its next-gen­er­a­tion AAV vec­tors to gen­er­ate can­di­dates for a tar­get — Bio­gen isn’t dis­clos­ing which ex­act­ly — that would treat in­her­it­ed eye dis­ease. The big biotech part­ner has an­oth­er tar­get in mind, for which it has the op­tion to add with­in two years.

Al­though the Alzheimer’s drug ad­u­canum­ab has been shoved to a cen­tral, al­most ex­is­ten­tial po­si­tion at Bio­gen, the com­pa­ny has been plac­ing mea­sured bets on oph­thal­mol­o­gy, which CEO Michel Vounatsos has called an emerg­ing area and part of the “sec­ond wave of growth” to fol­low ad­u­canum­ab in the mid-2020s.

With the new deal, it’s buy­ing in­to a plat­form that boasts of “su­pe­ri­or trans­duc­tion ef­fi­cien­cy” in reti­nal cells and in­trav­it­re­al ad­min­is­tra­tion.

Once it’s come up with the AAV cap­sids, Vi­Gen­eron will play a role in the in vi­vo proof of con­cept, but leave it all to Bio­gen af­ter that point. Bio­gen is pay­ing an undis­closed up­front and foot­ing the R&D bill.

Car­o­line Man Xu

“This col­lab­o­ra­tion ex­em­pli­fies our strat­e­gy to de­vel­op in-house pro­grams for se­lect­ed reti­nal tar­gets, while max­i­miz­ing our pro­pri­etary tech­nol­o­gy plat­forms with ad­di­tion­al col­lab­o­ra­tion pro­grams for oth­er tar­gets in oph­thal­mol­o­gy and fur­ther in­di­ca­tions,” said Car­o­line Man Xu, co-founder and CEO of Vi­Gen­eron.

There’s no word yet on when the can­di­dates might ma­te­ri­al­ize, but they will like­ly come long af­ter Bio­gen reads out the Phase III study of BI­IB111 (tim­repi­gene em­par­vovec), the lead as­set from the Night­star buy­out. De­signed to re­place the gene that’s mu­tat­ed in pa­tients with choroi­deremia, the pro­gram “rep­re­sents our next piv­otal read­out and our next po­ten­tial com­mer­cial prod­uct af­ter ad­u­canum­ab,” Vounatsos said back in Ju­ly.

The da­ta are ex­pect­ed to come in the first half of 2021.

Aside from that and a sec­ond Night­star ex­per­i­men­tal treat­ment for re­tini­tis pig­men­tosa caused by mu­ta­tions in the RP­GR gene, Bio­gen has al­so picked up a pre­clin­i­cal gene ther­a­py from Mass­a­chu­setts Eye and Ear for in­her­it­ed reti­nal de­gen­er­a­tion due to mu­ta­tions in the PRPF31 gene.

It all marks a sec­ond try at de­vel­op­ing one-shot cures for rare eye dis­eases, af­ter pulling out from a deal with Ap­plied Ge­net­ic Tech­nolo­gies fol­low­ing a failed tri­al.

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.

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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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.”

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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Paul Wotton, Obsidian CEO

Ver­tex adds a new gene edit­ing part­ner, pay­ing Ob­sid­i­an $75M for con­trol­lable CRISPR sys­tem

When Paul Wotton became CEO of Obsidian Therapeutics two years ago, he asked his team to explore where they could apply the company’s technology beyond cancer.

Launched by Michael Gilman in 2017, the GV-backed company had been working on a way of controlling cell therapies after they’re administered to a patient, allowing doctors to modulate the potency of CAR-T and other drugs as you might volume on a television set. Wotton wanted to them look beyond cancer, into areas such as gene editing.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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