Spar­ingVi­sion rais­es $52M to kick off long jour­ney for a next-gen gene ther­a­py that goes much, much broad­er than Lux­tur­na

Un­til Spark Ther­a­peu­tics’ pi­o­neer­ing gene ther­a­py, Lux­tur­na, came along, pa­tients with re­tini­tis pig­men­tosa had few treat­ment op­tions. Even af­ter it was ap­proved, though, the ma­jor­i­ty were left with the ex­act same op­tions.

Stephane Bois­sel

Be­cause it’s tar­get­ing mu­ta­tions in a spe­cif­ic gene known as RPE65, Lux­tur­na can on­ly ad­dress 2 to 3% of the en­tire RP pop­u­la­tion, Stephane Bois­sel told End­points News.

Bois­sel is the new­ly-mint­ed CEO of Spar­ingVi­sion, a French biotech co-found­ed by José-Alain Sa­hel and Thier­ry Léveil­lard at the In­sti­tut de la Vi­sion. They have their sight set on a new kind of AAV con­struct — a next-gen­er­a­tion gene ther­a­py if you will — that can treat all pa­tients of RP in­de­pen­dent of ge­net­ic mu­ta­tions.

And they have $52 mil­lion-plus (€44.5 mil­lion) to re­al­ize that vi­sion steadi­ly over the next four or five years, with plans to com­plete both first- and sec­ond-in-hu­man tri­als.

What the found­ing sci­en­tists pin­point­ed, in­stead of a gene, was a neu­rotroph­ic fac­tor se­cret­ed by rod pho­tore­cep­tors that ap­pears to pro­tect the cones, which ex­plains why RP pa­tients see their cones die even in the ab­sence of ge­net­ic ab­nor­mal­i­ties. By wrap­ping Rd­CVF, or rod-de­rived cone vi­a­bil­i­ty fac­tor, and an ox­ida­tive stress re­duc­ing en­zyme in­to a vi­ral vec­tor, they came up with SPVN06 in hopes of re­plac­ing one key func­tion that RP pa­tients lose along­side their rods.

Owen Smith

It won’t re­verse the dis­ease, but Spar­ingVi­sion is hop­ing it can slow or stop its pro­gres­sion.

The ap­proach paves a path that reach­es all 2 mil­lion RP pa­tients — a kind of “medi­um rare” in­di­ca­tion and broad­er ap­pli­ca­tion of gene ther­a­py fa­vored by 4BIO Cap­i­tal, said di­rec­tor of in­vest­ment Owen Smith. 4BIO co-led the round with UPMC En­ter­pris­es, the ven­ture arm of Pitts­burgh-based health sys­tem UPMC, where Sa­hel chairs the De­part­ment of Oph­thal­mol­o­gy.

“One of the al­ter­na­tives is de­vel­op­ing a Lux­tur­na in every sin­gle geno­type, and we haven’t even ful­ly char­ac­ter­ized the dis­ease on a ge­net­ic lev­el,” he said.

Cur­rent­ly fo­cused on a sin­gle as­set, Spar­ingVi­sion now op­er­ates vir­tu­al­ly with­out labs or man­u­fac­tur­ing — every­thing is out­sourced and will be for a while — with all 12 of its staffers cur­rent­ly in France. Hav­ing re­cent­ly moved to Paris from the Bay Area, where he was work­ing on cor­po­rate strat­e­gy for Sang­amo fol­low­ing the sale of Tx­Cell two years ago, Bois­sel al­so plans to build a US-based team to start prepar­ing for that sec­ond-in-hu­man ef­fi­ca­cy tri­al.

“It’s eas­i­er to find tal­ents who have done it in the US than in Eu­rope, and I want­ed peo­ple who have done it,” he said. “So our CMO, our CTO, our head of trans­la­tion­al sci­ence will be based in the US.”

He en­vi­sions re­cruit­ing a lean team of very se­nior peo­ple, build­ing out to 20 or 25 as­sum­ing Spar­ingVi­sion sticks on­ly to SPVN06. With that pro­gram alone there’s po­ten­tial to go in­to dry AMD, where the neu­ro­pro­tec­tive mech­a­nism could be rel­e­vant. There is al­so a pos­si­bil­i­ty of bring­ing in new prod­ucts or tech­nolo­gies that would fit un­der ge­nom­ic med­i­cines for the eye, from gene re­place­ment to edit­ing to reg­u­la­tion. But it’s ear­ly days, and Bois­sel said the syn­di­cate — which al­so fea­tures Jeito Cap­i­tal, Ys­ios Cap­i­tal, Bpifrance and Foun­da­tion Fight­ing Blind­ness — isn’t ex­pect­ing a quick flip to the mar­ket.

It’s tak­en the com­pa­ny four years to get to where they are. The first clin­i­cal tri­al should be­gin next year in Eu­rope, while the ef­fi­ca­cy tri­al is planned for 2023.

“That is a very unique set­ting in a world where things are very of­ten go­ing too fast,” he said.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

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Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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Katie Fanning, Mozart Therapeutics CEO

Mozart Ther­a­peu­tics makes its of­fi­cial de­but, jump­ing in­to the hot Treg R&D field with some big-name in­vestors back­ing it

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few.

Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate led by ARCH.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

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