Dhvanit Shah, Garuda CEO

Look­ing to make donor cells ob­so­lete, an ex-Har­vard re­searcher gets off-the-shelf stem cell biotech off the ground

A stem cell re­searcher who spent time at the Har­vard Stem Cell In­sti­tute, the Broad In­sti­tute and Brigham and Women’s Hos­pi­tal launched a new biotech Thurs­day, and he’s at­tract­ed sig­nif­i­cant blue-chip in­vest­ment to get things up and run­ning.

Dhvan­it Shah un­veiled a $72 mil­lion Se­ries A for Garu­da Ther­a­peu­tics with the goal of de­vel­op­ing off-the-shelf blood stem cell ther­a­pies. Shah, who found­ed Garu­da and serves as CEO, told End­points News that while he rec­og­nizes oth­er com­pa­nies are re­search­ing off-the-shelf cell ther­a­pies, Garu­da dif­fer­en­ti­ates it­self by try­ing to elim­i­nate the need for donor cells en­tire­ly.

“We can’t choose who our donors will be; the age, qual­i­ty and quan­ti­ty of cells from an­oth­er al­ways varies,” Shah told End­points. “There’s been noth­ing new in the last 50 years, and the promise is un­matched here. If some­one can come up with a way to make off-the-shelf hematopoi­et­ic stem cells, to me that’s the biggest break­through in the field.”

Some promi­nent in­vestors are back­ing the biotech, with Ais­ling Cap­i­tal, North­pond Ven­tures and Or­biMed lead­ing the Se­ries A.

The field of hematopoi­et­ic stem cells has pro­gressed to the point where some­thing like an off-the-shelf ther­a­py is pos­si­ble, Shah added. He de­scribed a steady rise af­ter the No­bel Prize-win­ning re­search of Shinya Ya­mana­ka and John Gur­don changed the game in 2006, when the pair showed how ma­ture stem cells could be re­pro­grammed to be “pluripo­tent,” or can di­vide in­to more stem cells.

It’s es­sen­tial­ly a way for pluripo­tent stem cells to be­come self-re­new­ing hematopoi­et­ic stem cells, Shah said, and it proves the ba­sis of Garu­da’s un­der­ly­ing the­o­ry. Af­ter re­cruit­ing a se­lect group of vol­un­teers to help gen­er­ate a “bank” of pluripo­tent stem cells, Garu­da re­searchers then con­vert these cells to the hematopoi­et­ic stem cells nec­es­sary for its ther­a­pies.

And Shah hopes hav­ing that bank on hand makes donor cells — and what can be a dif­fi­cult search to find them — com­plete­ly un­nec­es­sary.

“I’m done with peo­ple lin­ing up to be a donor,” he said. “We can do the job once in a life­time and no one ever has to look back.”

What goes in­to the tech­nol­o­gy be­hind this con­ver­sion, or how Garu­da picked the most suit­able vol­un­teers, Shah isn’t say­ing. But he claims the biotech has some stel­lar pre­clin­i­cal da­ta show­ing the tech­nol­o­gy works, ev­i­denced by the hefty Se­ries A and blue-chip syn­di­cate.

But he likened the po­ten­tial of Garu­da’s plat­form to pro­vide broad ac­cess to cu­ra­tive treat­ments to the ex­per­i­men­tal stem cell trans­plants giv­en to two HIV-pos­i­tive in­di­vid­u­als. In these in­stances, the pa­tients were giv­en new cells do­nat­ed from some­one who had a ge­net­ic mu­ta­tion re­sis­tant to HIV, al­low­ing them to stop tak­ing an­ti­retro­vi­ral drugs and be de­clared virus-free, per a 2019 Na­ture ar­ti­cle.

Those treat­ments were un­able to be brought in­to the larg­er HIV/AIDS pop­u­la­tion, Shah not­ed, but when build­ing Garu­da’s cell bank, he said the biotech is look­ing for peo­ple whose ge­net­ic make­up is suit­able for sim­i­lar ther­a­pies. If suc­cess­ful, Garu­da will be able to bring stem cell ther­a­pies to pa­tients who nor­mal­ly strug­gle to ac­cess them, such as racial and eth­nic mi­nori­ties.

Garu­da is de­vel­op­ing a pipeline of drugs as well, with ini­tial fo­cus­es in hema­to­log­ic ma­lig­nan­cies, sick­le cell dis­ease, be­ta-tha­lassemia and bone mar­row fail­ure dis­eases. But Shah said the im­me­di­ate next step is to be­gin build­ing out man­u­fac­tur­ing ca­pa­bil­i­ties to en­sure Garu­da can meet what he ex­pects will be sig­nif­i­cant de­mand.

If every­thing works out, the biotech ex­pects to launch its first in-hu­man stud­ies with­in the next two to three years, Shah said. But he cau­tioned that the es­ti­mate is just a ball­park fig­ure.

“I don’t want to over­promise and un­der­de­liv­er,” he said.

In ad­di­tion to the lead in­vestors, Thurs­day’s Se­ries A saw fund­ing from Cor­morant As­set Man­age­ment, Ridge­back Cap­i­tal In­vest­ments, Monashee In­vest­ment Man­age­ment, Sec­toral As­set Man­age­ment, Na­tion­al Re­silience, Inc. (Re­silience) and Mass Gen­er­al Brigham Ven­tures, among oth­ers.

Clar­i­fi­ca­tion: This ar­ti­cle has been up­dat­ed to clar­i­fy how pluripo­tent stem cells be­come self-re­new­ing.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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‘Catchy’ de­sign tops big ad buys on­line for grab­bing on­col­o­gists’ at­ten­tion — sur­vey

The cancer drug ads that get oncologists’ attention online are informative and use clear, eye-catching designs. That’s ZoomRx’s assessment in its most recent tracking survey, and while not necessarily surprising, the details in the research do break a few common misconceptions.

One of those is frequency, also known as the number of impressions an ad gets. No matter how many times oncologists saw a particular cancer drug ad, effectiveness prevailed in the survey across five drug brands. ZoomRx measured effectiveness as a combination of most attention-getting, relevant information and improved perception as reported by the doctors.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

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Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.