Or­bimed part­ner in search of a cure for Parkin­son’s launch­es new gene ther­a­py play­er

Jonathan Sil­ver­stein is ac­tive­ly look­ing for a biotech mir­a­cle.

Jonathan Sil­ver­stein

The Or­bimed part­ner formed a non­prof­it group called the Sil­ver­stein Foun­da­tion af­ter he was di­ag­nosed with Parkin­son’s, and now he’s launch­ing a new biotech which he hopes can pro­vide just that.

The new biotech is called Pre­vail Ther­a­peu­tics. Based in New York, it will be run by Co­lum­bia Uni­ver­si­ty’s Asa Abe­liovich, look­ing to de­vel­op gene ther­a­pies that will try to cor­rect a dis­ease that has proven to be one of the tough­est tar­gets in drug de­vel­op­ment. Or­bimed and the foun­da­tion — which Sil­ver­stein is al­so fund­ing — are back­ing the start­up along with Re­genXBio, one of the lead­ers in AAV tech used in the gene ther­a­pies now in clin­i­cal de­vel­op­ment.

The key fo­cus at Pre­vail will be the GBA1 mu­ta­tion car­ried by about 1 in 10 Parkin­son’s pa­tients. Ac­cord­ing to the biotech, the link be­tween the GBA1 gene, glu­co­cere­brosi­dase (GCase) pro­tein pro­duc­tion, and clear­ing out an ac­cu­mu­la­tion of al­pha-synu­cle­in in the brain “may have im­pli­ca­tions for the broad­er Parkin­son’s pop­u­la­tion, as well as in oth­er neu­rode­gen­er­a­tive dis­ease.”

The ven­ture cap­i­tal­ist told CN­BC that he’s seen biotechs like Bio­gen de­liv­er mir­a­cles to pa­tients, adding: “And … now I’ve got to cre­ate an­oth­er mir­a­cle.”

Adds Sil­ver­stein:

We be­lieve Re­genXBio’s NAV Tech­nol­o­gy Plat­form pro­vides us with a promis­ing op­por­tu­ni­ty to de­vel­op po­ten­tial life-chang­ing ther­a­pies for pa­tients suf­fer­ing from Parkin­son’s Dis­ease and oth­er neu­rode­gen­er­a­tive dis­eases. Dr. Abe­liovich’s lead­er­ship and deep ex­per­tise in the mol­e­c­u­lar mech­a­nisms of neu­rode­gen­er­a­tion unique­ly po­si­tion Pre­vail to make a sig­nif­i­cant mark in de­vel­op­ing in­no­v­a­tive bi­o­log­ic ther­a­pies for Parkin­son’s Dis­ease and oth­er cen­tral ner­vous sys­tem dis­eases with huge un­met med­ical needs.

Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

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But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

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Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

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The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

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The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.