Turn on, tune in: New York biotech scores $15M+ in chemo­ge­net­ics bet

Typ­i­cal­ly, once a bi­o­log­i­cal tar­get has been iden­ti­fied, sci­en­tists work on de­vel­op­ing mol­e­cules fo­cused on that tar­get. But in an emerg­ing field called chemo­ge­net­ics, re­searchers start with a small mol­e­cule and then de­sign a tar­get for it.

Bank­ing on its chemo­ge­net­ics tech­nol­o­gy to ad­dress CNS dis­or­ders, New York-based Red­pin Ther­a­peu­tics has se­cured a $15.5 mil­lion in­jec­tion, as it shep­herds its two pre­clin­i­cal pro­grams — for chron­ic pain and re­frac­to­ry epilep­sy — in­to the clin­ic.

Ex­ist­ing gene ther­a­pies tend to re­place de­fec­tive genes with healthy ones. But Red­pin’s tech­nol­o­gy us­es AAV vec­tors to de­liv­er en­gi­neered ion chan­nels to spe­cif­ic cell pop­u­la­tions. And then these en­gi­neered re­cep­tors, once ac­ti­vat­ed or trig­gered by their lig­and or the small mol­e­cule, con­trol the func­tion­ing of that par­tic­u­lar cell type.

The small mol­e­cule cho­sen by Red­pin is Pfiz­er’s smok­ing ces­sa­tion treat­ment, Chan­tix. The drug is ap­proved in 80 coun­tries, has suit­able phar­ma­co­ki­net­ic char­ac­ter­is­tics, and can al­so pen­e­trate the blood-brain bar­ri­er, co-founder and CEO El­ma Hawkins not­ed in an in­ter­view, adding that the com­pa­ny al­so has oth­er small mol­e­cule-re­cep­tor pairs in the pipeline.

“With­out Chan­tix’s pres­ence the (en­gi­neered) ion chan­nel is com­plete­ly in­ert, and then with Chan­tix there, the ion chan­nel gets ac­ti­vat­ed,” Hawkins said. “And so ba­si­cal­ly you are turn­ing the ion chan­nel on and off with an ex­ter­nal switch. And that switch is Chan­tix.”

Since the en­gi­neered re­cep­tors on­ly re­spond to spe­cif­ic drugs, dos­es can be low, and the process lim­its the off-tar­get sys­temic ef­fects of the drug on oth­er tis­sues — the once-and-done ap­proach is al­so tun­able and re­versible.

These ad­van­tages lured Lon­don-based 4BIO Cap­i­tal, which led the Se­ries A in­jec­tion. The VC shop has a $150 mil­lion fund fo­cused on ad­vanced ther­a­pies — in­clud­ing cell and gene ther­a­pies, RNA-based ther­a­pies and the mi­cro­bio­me.

“When we do treat pa­tients with small mol­e­cules for neu­ro­science, psy­chol­o­gy, and func­tion­al neu­rol­o­gy over a long time, they do cre­ate quite sig­nif­i­cant side-ef­fects,” not­ed man­ag­ing part­ner Dmit­ry Kuzmin in an in­ter­view.

But cell and gene ther­a­pies have the edge — they are one-time treat­ments, and can be very pre­cise, he said.

“Giv­en the com­bi­na­tion of the lo­cal sur­gi­cal ad­min­is­tra­tion, the AAV ad­van­tages and the pro­mot­er con­trol we can be very, very pre­cise in the brain,” Kuzmin stressed. “We can achieve fan­tas­tic de­liv­ery to func­tion­al­ly and ge­net­i­cal­ly dis­tinct cir­cuits in the brain and else­where in the body.”

Kuzmin is set to join Red­pin’s board. The com­pa­ny al­so counts Eli Lil­ly vet­er­an David Bleak­man as its chief sci­en­tif­ic of­fi­cer.

4BIO, along with Arkin Bio Ven­tures, led the Se­ries A round. Oth­er in­vestors such as the cor­po­rate ven­ture arm of Take­da, New York Ven­tures and Alexan­dria Ven­ture In­vest­ments, al­so par­tic­i­pat­ed in the round.

Ver­sant, MPM Cap­i­tal-backed Co­da Ther­a­peu­tics, which al­so has a chemo­ge­net­ics plat­form, raised $15 mil­lion last No­vem­ber bring­ing its to­tal Se­ries A haul to $34 mil­lion — as it fur­ther de­vel­ops its lead pro­grams: chron­ic neu­ro­path­ic pain and fo­cal epilep­sy.

So­cial im­age: El­ma Hawkins via Youtube

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Look­ing for 'ex­ter­nal in­no­va­tion,' Boehringer In­gel­heim re­serves $500M+ for new Shang­hai hub

Now that Boehringer Ingelheim’s bet on contract manufacturing in China has paid off, the German drugmaker is anteing up more to get into the research game.

Boehringer has set aside $507.9 million (€451 million) for a new External Innovation Hub to be built in Shanghai over five years. The site will become one of its “strategic pillars” as the team strives to get 71 approvals — either for new products or indications — by 2030, said Felix Gutsche, president and CEO of Boehringer Ingelheim China.

Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)

No­vavax snags Ben Machielse for CMC and pro­motes a trio of staffers; Mar­ty Du­vall lands an­oth­er CEO post at On­copep­tides

Novavax has been making waves recently by securing a $384 million commitment from CEPI to cover R&D and manufacturing for its Covid-19 vaccine while also spending $167 million on a 150,000 square-foot facility. The Maryland biotech continues to shore up its leadership team as well, bringing in Ben Machielse as their EVP of CMC just a couple weeks after nabbing AstraZeneca vet Filip Dubrovsky as their new CMO. Machielse was president and CEO of Vtesse from 2014-17, and before that, he also spent more than 11 years at MedImmune and was EVP of operations for the back half of his tenure.

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

Paul Tesar (Convelo Therapeutics)

Io­n­is, lead­ing MS re­searcher throw an­ti­sense at a new type of brain cells

No matter how many molecules he threw at them, Paul Tesar couldn’t get the brain cells to survive. Or he got them to survive, but then — to everyone’s bafflement — they still couldn’t do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.