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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Vipin Suri, Catamaran Bio CSO

Cata­ma­ran Bio sails in­to the CAR-NK wa­ters with a $42M launch round

Catamaran Bio’s founding members decided to jump into the CAR-NK game last December over drinks at a trendy bar in Boston.

They were sitting around a table, discussing an MD Anderson study which provided some of the first clinical proof that natural killer (NK) cells can be reengineered to attack tumors, much like CAR-T therapies. It was a “long and lively” discussion, COO Mark Boshar recalls. And by the time it was over, they had a starting point to launch a company.

Peter Thiel (Riccardo Savi/Sipa via AP Images)

Tech bil­lion­aire Pe­ter Thiel backs a lead­ing psy­che­del­ic drug de­vel­op­er

Right on the heels of investing in antibody drug developer AbCellera, Facebook billionaire Peter Thiel has jumped into a syndicate putting up $125 million for a company with a portfolio of psychedelic drugs in the clinic for mental health.

The C round — which includes a $32 million conversion of notes to equity — will fuel the development programs at ATAI Life Sciences, a Berlin-based biotech that has assembled a portfolio of companies with psychedelic and non-psychedilc approaches to depression, anxiety and addiction.

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Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

Simeon George, SR One CEO (SR One)

Scoop: SR One crew com­pletes a com­pli­cat­ed spin­out from Glax­o­SmithK­line. And now they have a $500M fund to in­vest on their own

It’s taken close to 2 years, but Simeon George and his team at SR One have completed their spinout from GlaxoSmithKline, ending a saga as one of the longest running venture arms of Big Pharma as they go out on their own to forge the next chapter with a new and independent $500 million fund.

GSK is sticking with the spinout, this time as a minority investor — though a big one. And I’m told that the R&D group at GSK will remain involved in evaluating their new plays, helping with the scientific due diligence involved in scouting the world for new opportunities during a period of explosive growth in biotech investing.

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