Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Sh­pigel­mach­er start­ed the project, he knew he’d have to fund it him­self. Every oth­er ef­fort of its kind was aca­d­e­m­ic, re­ject­ed as too risky by in­vestors.

Sh­pigel­mach­er, a ro­bot­ics geek and en­tre­pre­neur who had drift­ed in­to con­sult­ing for phar­ma, want­ed to build the re­al-life equiv­a­lent of tech­nol­o­gy from the 1960s film “Fan­tas­tic Voy­age,” the one where a sub­ma­rine crew is shrunk to “about the size of a mi­crobe” and sent on a mis­sion to re­pair a sci­en­tist’s brain. He scanned the lit­er­a­ture, found the lab that was work­ing on the most ad­vanced project — at the Max Planck In­sti­tute in Ger­many, it turned out — and start­ed fund­ing them with mon­ey from his and his co-founders’ own ac­counts, along with some seed cash from friends and fam­i­ly.

Five years lat­er, Sh­pigel­mach­er has a com­pa­ny, Bio­naut Labs, and even a few in­vestors will­ing to take a shot on what they pro­duced: a mil­lime­ter-sized, screw-shaped, cell-pen­e­trat­ing ro­bot, de­signed to drop bi­o­log­i­cal bombs on com­mand to pre­cise spots in the body. That in­cludes Khosla Ven­tures, the promi­nent Sil­i­con Val­ley tech VC, who is lead­ing a $20 mil­lion Se­ries A round for the biotech, help­ing push them and their re­mote-con­trolled ther­a­peu­tic to­ward the clin­ic for brain dis­or­ders, ide­al­ly by 2023.

“As far as I know, there is no oth­er com­pa­ny that does re­mote-con­trolled mi­cro-ro­bot­ics in the in­dus­try,” Sh­pigel­mach­er told End­points News. “We got it to the point where VCs were able to step in.”

The new ef­fort has pass­ing sim­i­lar­i­ties to the “ro­bot­ic pills” at de­vel­op­ment at Rani Ther­a­peu­tics and oth­er star­tups, but those, like any pill, can on­ly go to the gut, Sh­pigel­mach­er said. Bio­naut will try to re­mote guide their drug to or­gans through­out the body, de­vel­op­ing a se­ries of ro­bots with dif­fer­ent shapes and fea­tures for dif­fer­ent tasks and tis­sues.

The “bio­naut”

The goal for Bio­naut is broad­en­ing the so-called ther­a­peu­tic win­dow for drug de­vel­op­ment. For most drugs, de­vel­op­ers have to bal­ance a mol­e­cule’s abil­i­ty to, say, kill a tu­mor with the po­ten­tial harm it can do to all the oth­er cells ex­posed to it, cre­at­ing a nar­row win­dow for vi­able drugs. That win­dow ex­pands if you can de­posit the drug di­rect­ly in­to the part of the body where the dis­ease is based.

The biotech chose to fo­cus first on neu­rol­o­gy be­cause it’s an area where com­pa­nies have re­peat­ed­ly failed, and where the fail­ure of­ten traces back to how well they can de­liv­er to the brain, or spe­cif­ic parts of the brain. Their first pro­gram is for a rare and fa­tal child­hood tu­mor that ap­pears on the brain stem.

Es­sen­tial­ly, the com­pa­ny will load chemother­a­py on­to the mi­ni-ro­bots, in­ject them in­to the cen­tral ner­vous sys­tem and use a mag­net­ic wire to guide it to the tu­mor, where it drops the pay­load.

“We don’t want to start from con­cepts that sound too risky or untest­ed,” he said. “We know that if we get this thing to the tar­get, it’s go­ing to work.”

Even­tu­al­ly, though, the com­pa­ny al­so wants to de­liv­er var­i­ous oth­er tech­nolo­gies, in­clud­ing gene ther­a­pies, on­colyt­ic virus­es and an­ti­sense oligonu­cleotides. Ide­al­ly, they could more pre­cise­ly tar­get drugs al­ready be­ing de­vel­oped for neu­ro­log­i­cal dis­eases. For ex­am­ple, a Parkin­son’s drug might be more ef­fec­tive if it were de­liv­ered di­rect­ly in­to the sub­stan­tia ni­gra, a re­gion at the cen­ter of the brain where much of the de­gen­er­a­tion plays out.

Sh­pigel­mach­er said the com­pa­ny raised their Se­ries A in mid-2019 and wait­ed un­til they had suf­fi­cient proof-of-con­cept da­ta to be con­fi­dent they were on track for the clin­ic. Still, it re­mains ear­ly stage and the com­pa­ny’s in­vestors come en­tire­ly from the tech world, with a cou­ple, such as Khosla, that have been re­cent­ly inch­ing in­to biotech.

“We are thrilled to bring Bio­naut Labs out of stealth mode as it typ­i­fies the type of new im­pact­ful tech­nol­o­gy com­pa­nies we like to help build,” said Vin­od Khosla, founder of Khosla Ven­tures, which has been in­creas­ing­ly inch­ing in­to biotech over the past few years. “Bio­nauts hold great promise as a new tar­get­ed treat­ment modal­i­ty for se­vere brain dis­or­ders for which there are few, if any, ef­fec­tive treat­ment op­tions. More­over, the broad ther­a­peu­tic po­ten­tial of Bio­naut ex­tends to many dis­eases where con­ven­tion­al ther­a­pies are lim­it­ed or lack­ing.”

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

From left: James Brown, Michael Chambers, John Ballantyne

Alde­vron founders back a biotech start­up that's look­ing to end the moral de­bate over cell lines once and for all

For millions of Catholics around the world, the development of new vaccines to combat Covid-19 has sparked a moral dilemma. All the approved vaccines in use relied — in some fashion — on cell lines that were derived from aborted fetal tissue.

While church leaders accepted the vaccines and recommended their use to end the pandemic, a number also highlighted their preference for the mRNA vaccines from Pfizer/BioNTech and Moderna over the J&J and AstraZeneca shots, which they noted were more heavily dependent on cell lines that they found morally objectionable.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 107,500+ biopharma pros reading Endpoints daily — and it's free.

Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.