Paula Cannon (Credit: Richard Carrasco)

Re­searchers teamed up to de­vel­op a 'three in one' HIV treat­ment — and the NIH is throw­ing in $14.6M

The NIH is pitch­ing $14.6 mil­lion in­to a “three for one” HIV re­search pro­gram led by USC and the Fred Hutchin­son Can­cer Re­search Cen­ter that aims to strike the need for dai­ly med­ica­tion — or even achieve a “home run” cure.

The five-year grant will back pre­clin­i­cal stud­ies that com­bine gene edit­ing with tech­nol­o­gy to im­prove bone mar­row trans­plants. The po­ten­tial ther­a­py would en­gi­neer a pa­tient’s own stem cells to fight HIV, and stim­u­late them to pro­duce new im­mune cells once rein­tro­duced to the pa­tient.

“A home run would be that we com­plete­ly cure peo­ple of HIV,” Paula Can­non, a USC pro­fes­sor of mol­e­c­u­lar mi­cro­bi­ol­o­gy and im­munol­o­gy and co-di­rec­tor of the pro­gram, said in a state­ment. “What I’d be fine with is the idea that some­body no longer needs to take an­ti-HIV drugs every day be­cause their im­mune sys­tem is keep­ing the virus un­der con­trol, so that it no longer caus­es health prob­lems and, im­por­tant­ly, they can’t trans­mit it to any­body else.”

Hans-Pe­ter Kiem

Hans-Pe­ter Kiem, the Stephanus Fam­i­ly En­dowed Chair for Cell and Gene Ther­a­py at Fred Hutch, is the co-di­rec­tor. Har­vard Uni­ver­si­ty pro­fes­sor David Scad­den and Ma­gen­ta Ther­a­peu­tics are al­so col­lab­o­rat­ing on the project.

The ap­proach was in­spired by three pa­tients who ap­pear to have been cured of the virus — all of whom re­ceived blood stem cell trans­plants from donors who car­ried a mu­ta­tion in the CCR5 gene. One of them, dubbed the “Berlin pa­tient,” has been off an­ti­retro­vi­ral drugs since 2007.

“I think of the Berlin pa­tient as proof of prin­ci­ple that re­plac­ing the im­mune sys­tem with one that’s HIV-re­sis­tant by re­mov­ing CCR5 is a pos­si­ble way to treat some­body,” Can­non said.

The pro­gram will study the use of gene edit­ing to re­move CCR5 from pa­tients’ stem cells — a process which is al­ready in clin­i­cal tri­al for HIV treat­ment at City of Hope Na­tion­al Med­ical Cen­ter in Duarte, CA. The stem cells will al­so be en­gi­neered to re­lease an­ti­bod­ies and an­ti­body-like mol­e­cules that block HIV.

In ad­di­tion, the grant will fund a Fred Hutch team’s en­deav­or to adapt CAR-T cell ther­a­py to cre­ate stem cells whose prog­e­ny tar­get HIV-in­fect­ed cells.

As for prepar­ing a pa­tient for the trans­plant, Ma­gen­ta is work­ing on an­ti­body-drug con­ju­gates to re­place mild chemother­a­py or ra­dio­ther­a­py typ­i­cal­ly giv­en be­fore the pro­ce­dure. And Scad­den is re­search­ing an in­jectable gel that could help im­mune cells re­pop­u­late more quick­ly, avoid­ing a de­lay.

HIV in­fec­tion, which cur­rent­ly af­fects about 1.2 mil­lion Amer­i­cans, has proved to be ex­ceed­ing­ly dif­fi­cult to cure. In Ju­ly, Mer­ck and Dew­point inked a deal that al­lows the phar­ma to use the Boston-based biotech’s bio­mol­e­c­u­lar con­den­sate tech­nol­o­gy to de­vel­op treat­ments, and po­ten­tial­ly a cure, for the HIV virus. The NIH-fund­ed group is hop­ing to at least con­trol the virus enough to elim­i­nate the need for dai­ly meds. But at best, they’re al­so eye­ing a long sought-af­ter cure.

“This grant funds a team with an over­ar­ch­ing goal of de­vel­op­ing what our per­fect HIV gene ther­a­py would look like,” Can­non said. “All of these pieces could hap­pen sep­a­rate­ly, but the fact that the NIH has fund­ed us as a team means that the sum will be so much big­ger than the parts.”

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

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In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

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How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

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Bio­phar­ma's suc­cess rate in bring­ing drugs to mar­ket has long been abysmal. Can new tools help rewrite that trou­bled past?

In 2011, a team of researchers at British drugmaker AstraZeneca had a problem they were looking to solve.

For years, drug discovery and development were a wasteland for innovation. Novel drugs largely fell into one of two categories — monoclonal antibodies and small molecules — and new therapeutic modalities were hard to come by. After a rush of promising approvals in the late 1990s — including then-Biogen’s CD20 targeting antibody breakthrough Rituxan — the field stagnated and attrition rates stayed sky-high. What exactly is the industry doing wrong? AstraZeneca asked itself.

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