Syn­cona dives deep in­to dry AMD R&D, up­ping Gy­ro­scope bet to $100M

The ex­plo­sion of gene ther­a­pies in the clin­ic promis­es to trans­form the treat­ment of eye dis­eases. And Syn­cona thinks one of their port­fo­lio com­pa­nies may have found the an­swer to dry AMD.

The life sci­ences in­vest­ment group sunk an ad­di­tion­al $57.7 mil­lion in Se­ries B fund­ing in­to the reti­nal gene ther­a­py group Gy­ro­scope Ther­a­peu­tics, bring­ing their to­tal in­vest­ment to just un­der $100 mil­lion. Syn­cona main­tains an 80% stake in the UK-based out­fit. An ad­di­tion­al $2.9 mil­lion will be pro­vid­ed by Cam­bridge In­no­va­tion Cap­i­tal.

A host of gene ther­a­pies have been re­leased or are in de­vel­op­ment for rare eye dis­or­ders – most promi­nent­ly Spark’s cost­ly Lux­tur­na, the first gene ther­a­py for in­her­it­ed blind­ness. But no ef­fec­tive treat­ment ex­ists for dry, age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD) — the most com­mon cause of blind­ness in the in­dus­tri­al world. AMD af­fects 170 mil­lion peo­ple glob­al­ly, 85-90% of whom have the dry type.

The new dol­lars will al­low Gy­ro­scope to com­plete its Phase I/II study of its GT005 ther­a­py, which works by in­ject­ing a virus-em­bed­ded so­lu­tion be­neath the reti­na to de­liv­er a gene se­quence cod­ing a pro­tein that pre­vents “com­ple­ment” cells from at­tack­ing healthy reti­nal cells. The mon­ey will al­so al­low it to start Phase II and ex­pand its sur­gi­cal plat­form to en­sure safe de­liv­ery of the gene to the reti­na.

In Feb­ru­ary, Gy­ro­scope dosed its first pa­tient, an 80-year-old Ox­ford woman, with the ther­a­py.

Two forms of AMD ex­ist: “dry” and “wet.” Sev­er­al drugs for the wet type ex­ist and more are un­der de­vel­op­ment, but be­cause the dry form re­mains un­treat­able by con­ven­tion­al means, it presents a ma­jor op­por­tu­ni­ty for de­vel­op­ers. A Ju­ly re­port from mar­ket in­tel­li­gence firm In­fini­ti Re­search es­ti­mates that “huge un­met med­ical need” for dry-AMD will dri­ve the mar­ket by 8% CA­GR.

Khurem Fa­rooq

The mar­ket’s first big dry-AMD hope, Roche’s lam­pal­izum­ab was dropped af­ter fail­ing both Phase III tri­als in 2017. Gy­ro­scope is com­pet­ing against J&J, Parisian biotech Bio­phytis and Ken­tucky-based biotech Apel­lis, among oth­ers, to be the first to in­tro­duce a dry–AMD ther­a­py. Re­searchers at NIH and in Chi­na are al­so test­ing stem-cell-based mod­els, while in Ju­ly the bio­elec­tron­ics com­pa­ny Pix­i­um Vi­sion an­nounced suc­cess in the 12-month fea­si­bil­i­ty study for their reti­nal im­plant.

Gy­ro­scope ac­quired US-based de­vice mak­er Or­bital Bio­medic in April. It is run by for­mer Genen­tech ex­ec­u­tive Khurem Fa­rooq, who over­saw com­mer­cial ac­tiv­i­ties for the wet-AMD drug Lu­cen­tis and pre-launch work for Roche’s lam­pal­izum­ab.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Fol­low­ing US, Chi­na hos­pi­tal ef­forts, Gilead plots its own PhI­II tri­als for close­ly watched Covid-19 drug

Gilead is launching its own Phase III trials of remdesivir, the repurposed antiviral that a WHO official called the “one drug right now we think may have real efficacy” against Covid-19 as the novel coronavirus originating from Wuhan, China ravages the world.

Announced just a day after the NIH and the University of Nebraska Medical Center registered their US-based trial online, Gilead’s program will comprise two studies enrolling around 1,000 patients beginning in March. They will recruit primarily in Asian countries but will also include patients from other locations with “high numbers of diagnosed cases,” the company said.

Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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In at least one life-sci hub, gen­der and di­ver­si­ty ini­tia­tives haven’t made a dent

Gender and racial diversity at the top of UK life science companies has hardly budged over the last seven years despite repeated advocacy efforts, according to a new report.

The report, from the recruiting firm Liftstream, found that 14.8% of directors on life sciences boards were women and 21.1% of top executives were women. That’s a modest bump from the 9.8% of directors and 18.1% of executives Liftstream identified in their last report from 2014. The percentage of women CEOs moved from 8% to 9.8%.

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Will a 'risk-of­f' mind­set has­ten cell ther­a­py M&A? Io­vance surges on buy­out chat­ter

Is it time for some cell therapy M&A?

Investors of Iovance Biotherapeutics certainly thought so, sending its stock $IOVA up as much as 40% after Bloomberg reported that the cancer-focused biotech is talking to potential buyers.

While 2019 saw a number of high-profile gene therapy company takeovers — led by Roche’s $4.3 billion bid of Spark as Astellas went for Audentes, Biogen snapped up Nightstar and Vertex absorbed Exonics — large players appeared to prefer partnering on the cell therapy front, particularly when it comes to cancer. Hal Barron put his weight behind Rick Klausner’s startup as he rebuilt GlaxoSmithKline’s cancer pipeline. Takeda turned to MD Anderson to license their natural killer cell therapy.

One less ri­val for Im­muno­vant, as Alex­ion aban­dons FcRn in­hibitor

Less than one year after Alexion parted with $25 million upfront to secure access to a second anti-FcRn asset, it is abandoning the experimental drug. The discontinuation, disclosed at the SVB Leerink Global Healthcare Conference in New York during a fireside chat, bodes well for rival Immunovant.

The drug (ABY-039), partnered for development with Sweden’s Affibody, was forsaken on the basis of early-stage data that was not viewed favorably, Baird and SVB Leerink analysts noted.

Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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