Syncona chief investment officer Chris Hollowood (Syncona)

Syn­cona launch­es new gene ther­a­py play­er Pure­spring, spun out of the Uni­ver­si­ty of Bris­tol, with a rough­ly $60M Se­ries A

Long­time UK life sci­ences in­vestor Syn­cona has a new gene ther­a­py out­fit, one that the firm says is one of the first ever to fo­cus on kid­ney dis­or­ders.

Syn­cona is launch­ing Pure­spring Ther­a­peu­tics with a £45 mil­lion Se­ries A, or just shy of $60 mil­lion, in the hopes it can de­liv­er on a new class of AAV gene ther­a­pies for chron­ic kid­ney dis­eases. Pure­spring is be­ing spun out from the Uni­ver­si­ty of Bris­tol in the UK, based on the work of Moin Saleem, a pro­fes­sor for pe­di­atric re­nal med­i­cine.

“Gene ther­a­py has come of age in cer­tain ar­eas, but a ma­jor chal­lenge in com­plex sol­id or­gans is to pre­cise­ly tar­get the ge­net­ic ma­te­r­i­al to the cor­rect cell type,” Saleem said in a state­ment. “Us­ing ac­cu­mu­lat­ed ex­per­tise in the Bris­tol Re­nal re­search group we have solved this cru­cial hur­dle, putting us in a po­si­tion to de­liv­er cu­ra­tive gene ther­a­py to pa­tients with chron­ic and in­tractable kid­ney dis­eases.”

The com­pa­ny is aim­ing to de­vel­op ther­a­pies that di­rect­ly tar­get the glomeru­lus in the kid­ney, which is a clus­ter of cap­il­lar­ies around the end of a kid­ney tubule where waste prod­ucts are fil­tered from the blood­stream. Based on where the re­search cur­rent­ly stands, the pro­grams could leap from the lab in­to the clin­ic in the next three to four years, Bris­tol said.

Pure­spring will al­so have ac­cess to a new in vi­vo screen­ing plat­form called Fun­Sel, de­vel­oped by re­searchers at King’s Col­lege Lon­don. By uti­liz­ing this tech­nol­o­gy, Pure­spring can screen for cell-spe­cif­ic pro­tec­tive fac­tors de­liv­ered via gene ther­a­py that could have ap­pli­ca­tions across sev­er­al kid­ney dis­eases be­yond dis­or­ders caused by on­ly one gene vari­a­tion.

Do­minic Schmidt

This is the sixth gene ther­a­py com­pa­ny Syn­cona has found­ed since 2012, and the firm will be tak­ing an 84% stake in the biotech. Syn­cona’s chief in­vest­ment of­fi­cer, Chris Hol­lowood, will serve as chair­man and part­ner Do­minic Schmidt will be join­ing the board of di­rec­tors.

Each of Syn­cona’s com­pa­nies fo­cus on a dif­fer­ent sub­set of ge­net­ic dis­or­ders and their port­fo­lio in­cludes the com­pa­ny Night­star, which was sold to Bio­gen for $877 mil­lion in 2019. Night­star, whose re­search cen­tered on in­her­it­ed reti­nal dis­eases, saw its buy­out come amidst a wave of gene ther­a­py-re­lat­ed ac­qui­si­tions — around the same time, Roche pur­chased Lux­tur­na pro­duc­er Spark Ther­a­peu­tics for $4.3 bil­lion and J$J shelled out $440 mil­lion for the rights to MeiraGTx’s oph­thal­mo­log­i­cal gene ther­a­pies.

The oth­er play­ers un­der the Syn­cona um­brel­la in­clude Free­line (chron­ic sys­temic dis­ease), Gy­ro­scope (reti­nal in­flam­ma­tion), Or­bit Bio­med­ical (a sub-reti­nal sur­gi­cal de­liv­ery plat­form, now merged with Gy­ro­scope) and Swan­Bio (CNS gene ther­a­py).

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.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

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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News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

PhRMA sues Trump gov­ern­ment over drug im­por­ta­tion rule — days be­fore it's set to be ef­fec­tive

Ever since President Donald Trump floated the idea of using state-sponsored importation to lower drug prices, PhRMA has made its opposition abundant. Not only is the proposal dangerous and futile,  but the trade group has also argued that it may even be illegal.

Now that the FDA has issued its final rule permitting states to bring certain drugs from Canada, PhRMA is taking the government to court — just a few days before the rule is slated to take effect.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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