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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

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

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.