Darrin Disley (L) and Pierre-Louis Joffrin

A UK-based start­up looks to beat Spark's Lux­tur­na at its own game with pro­gram­ma­ble cell plat­form for eye dis­ease

Spark’s Lux­tur­na made waves in 2017 when it was ap­proved to treat in­her­it­ed reti­nal degra­da­tion by re­plac­ing the mu­tat­ed RPE65 gene with a nor­mal copy. But in­stead of re­plac­ing a faulty gene to re­gain func­tion, what if sci­en­tists could re­pro­gram whole cells in vi­vo?

On Tues­day, a UK-based start­up reeled in $17 mil­lion to pur­sue just that.

Mo­gri­fy set out in 2016 to pi­o­neer a new class of in vi­vo re­pro­gram­ming ther­a­pies in oph­thal­mol­o­gy and use that same tech­nol­o­gy to trans­form the way ex vi­vo cell ther­a­pies are de­vel­oped. The com­pa­ny first closed a Se­ries A round in 2019. And now, in­vestors are tack­ing on an­oth­er $17 mil­lion, bring­ing the ex­pand­ed round to $33 mil­lion in to­tal.

While the ini­tial fund­ing helped the young com­pa­ny set up op­er­a­tions, cor­po­rate de­vel­op­ment of­fi­cer Pierre-Louis Jof­frin says this next wave will be used to es­tab­lish proof-of-con­cept.

“In the case of oph­thal­mol­o­gy, this re­gen­er­a­tive ap­proach of ac­tu­al­ly bring­ing back some of the cells that have dis­ap­peared as a re­sult of dis­ease would be trans­for­ma­tive,” he told End­points News. “Most ap­proach­es at the mo­ment, ei­ther ap­proved or in the pipeline, are on­ly try­ing to slow down the pro­gres­sion of the dis­ease.”

Mo­gri­fy’s name is de­rived from the word “trans­mo­gri­fy,” which Mer­ri­am Web­ster de­fines as  “to change or al­ter great­ly and of­ten with grotesque or hu­mor­ous ef­fect,” and es­sen­tial­ly sums up what the com­pa­ny is try­ing to do. The team us­es tran­scrip­tion fac­tors — small pro­teins that reg­u­late gene ex­pres­sion — to dri­ve the con­ver­sion of cells, es­sen­tial­ly al­low­ing sci­en­tists to “switch” a cell from one state to an­oth­er.

The idea of cell re­pro­gram­ming isn’t new. In fact, Tenaya Ther­a­peu­tics scored a $106 mil­lion crossover round back in March for its heart fail­ure pro­grams, in­clud­ing one that us­es a sin­gle AAV vec­tor to de­liv­er com­bi­na­tions of mul­ti­ple genes that dri­ve in vi­vo re­pro­gram­ming of car­diac fi­brob­lasts to cre­ate new heart mus­cle cells.

In im­muno-on­col­o­gy, Mo­gri­fy us­es tran­scrip­tion fac­tors to con­vert in­duced pluripo­tent stem cells (IP­SC) in petri dish­es or test tubes, which are then de­liv­ered as a ther­a­peu­tic through trans­fu­sion. The com­pa­ny re­cent­ly struck a part­ner­ship with Sang­amo Ther­a­peu­tics that will look to de­rive Tregs from IP­SCs us­ing Mo­gri­fy’s plat­form.

“The use of tran­scrip­tion fac­tors in the dif­fer­en­ti­a­tion from IP­SC to spe­cif­ic cells, means that we can dri­ve the ef­fi­cien­cy up, as well as the time of dif­fer­en­ti­a­tion down,” Jof­frin said.

Mo­gri­fy has a sec­ond part­ner­ship it hasn’t dis­closed yet, ac­cord­ing to Jof­frin.

The re­newed Se­ries A — led by Park­walk Ad­vi­sors with help from Astel­las Ven­ture Man­age­ment, 24Hay­mar­ket, Ab­cam co-founder Jonathan Mil­ner and Mo­gri­fy CEO Dar­rin Dis­ley — will pro­vide a run­way through 2022, Jof­frin said. The com­pa­ny will look to pull in a “sig­nif­i­cant” Se­ries B by 2023, he added.

“The most ex­cit­ing thing about the com­pa­ny is its abil­i­ty to re­al­ly com­plete­ly dis­cov­er new bi­ol­o­gy,” he said.

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Roger Perl­mut­ter lines up deals, fresh fund­ing at Eikon; Sec­ond RSV vac­cine ap­proved; Sev­er­al biotechs flash­ing red; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you come back to our website this weekend for ASCO news, don’t forget to check out our updated event lineup at BIO, which will cover everything from the current state of VC investing in biotech to top pharma R&D chiefs discussing how to make pipeline decisions.

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Roger Perlmutter, Eikon Therapeutics CEO

Roger Perl­mut­ter builds Eikon's pipeline with deal-mak­ing flur­ry, rais­ing $106M more

Eikon Therapeutics announced three business development deals on Thursday, effectively dropping in a pipeline of cancer drugs alongside more than $100 million in fresh funding.

The Hayward, CA-based company has become one of biotech’s richest startups since its 2019 founding, having raised nearly $775 million. It’s developing a massive, automated research approach built around Nobel Prize-winning microscope science to peer inside cells and watch proteins in action. After its Series B last year, PitchBook reported a $3.02 billion valuation. And while CEO Roger Perlmutter declined to comment on that figure, he said its first tranche of nearly $106 million in Series C funding is a “meaningful step-up to our Series B valuation.”

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Grail’s blood test charts path for di­ag­nos­ing pa­tients sus­pect­ed of hav­ing can­cer in large study: #AS­CO23

Grail’s vision is simple but bold. The blood testing company has long held that people are often diagnosed with cancer too late. If seemingly healthy people were screened for early signs of the disease before symptoms appear, they may be able to get more effective treatments that nip cancer in the bud.

That premise is the basis of Grail’s commercial blood test, Galleri, which searches for the genetic fingerprints of cancer in the blood. The test, launched in 2021, reaped $55 million in sales last year, but now the company is setting its sights on a new market: patients suspected of having cancer due to symptoms such as abdominal pain, rectal bleeding or unexplained weight loss. Rather than administering expensive scans or conducting invasive biopsies right away, Grail hopes doctors will consider a simple blood test.

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Bala Venkataraman, Avego founder and managing partner (L), and Bruno Paquin, AtomVie CEO

Cana­di­an CD­MO se­cures more fund­ing to get its man­u­fac­tur­ing site up and run­ning

AtomVie Global Radiopharma Inc, a Canadian radiopharmaceutical contract manufacturer, has received additional funds to get its manufacturing facility up and running.

The manufacturer announced that it has raised an additional 90 million Canadian dollars ($66.9 million) in a “Tranche 2 Series A round,” led by the healthcare investment firm Avego Management. The company previously announced a $40 million Series A round last year, which contributed to the construction of a new 64,000-square-foot facility.

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GSK pro­motes rou­tine im­mu­niza­tions for adults amid post-pan­dem­ic vac­cine back­slide

GSK launched a new initiative on Thursday and committed up to $1 million in grant funding to improve adult routine vaccination rates.

While the pandemic spotlight was trained on the race for novel Covid-19 vaccines, other routine vaccination rates plummeted, raising concerns that missed doses may put children and even some adults at risk of preventable diseases such as measles or shingles. The World Health Organization last year reported the largest drop in childhood vaccinations in roughly three decades.

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Tammie Denyse speaks up about Black women and breast cancer inequity in Gilead's first TikTok campaign. (Gilead Sciences)

Gilead joins Tik­Tok with on­col­o­gy aware­ness cam­paign fea­tur­ing di­verse group of can­cer ad­vo­cates

Gilead Sciences is taking over the opening page on TikTok for the next two weeks. A Gilead-sponsored video, featuring cancer advocates talking about equity and other issues, will show up as the landing page, called the “For You” page, for millions of TikTok watchers.

The cancer awareness campaign will begin on Monday and run for two weeks, a Gilead spokesperson told Endpoints News. The TikTok ad debut is timed around the ASCO medical conference, but the work is aimed more broadly at healthcare professionals, as well as people touched by cancer and people interested in advancing Black and general health equity.

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Dol­lars flow with three new pub­lic of­fer­ings, two pri­vate place­ments

A handful of biotechs announced plans to raise money this week.

First up is Hookipa Pharma, which announced Wednesday night that it is looking to raise $50 million in gross earnings in a public offering — by selling 22.9 million shares of common stock at $1.31 a share. The biotech, which is developing immuno-oncology treatments and infectious disease programs, is also offering roughly 15,000 shares of non-voting preferred stock, which could be converted into 1,000 shares of common stock for a price of $1,310 each.

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Stephen MacMillan, Hologic CEO (Photo by Riccardo Savi/Getty Images for Concordia Summit)

Il­lu­mi­na names Ho­log­ic CEO as new board mem­ber and chair

Illumina’s board appointed two new members, including Hologic CEO Stephen MacMillan as the non-executive chair, a move that followed a proxy fight that saw shareholders oust the company’s board chair.

The DNA sequencing company also appointed Scott Ullem, the CFO of Edwards Lifesciences, to the board, according to a company statement.

Illumina’s plans to add two new board members came as Carl Icahn waged a board proxy campaign culminating with shareholders electing his candidate, Andrew Teno, over board chair John Thompson. Illumina CEO Francis deSouza survived a threat to his board seat by securing more than twice the shareholder votes than his challenger. Another Illumina candidate, Robert Epstein, was also elected and remained on the board.

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