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.

Mov­ing Out of the Clin­ic with Dig­i­tal Tools: Mo­bile Spirom­e­try Dur­ing COVID-19 & Be­yond

An important technology in assessing lung function, spirometry offers crucial data for the diagnosis and monitoring of pulmonary system diseases, as well as the ongoing measurement of treatment efficacy. But trends in the healthcare industry and new challenges introduced by the COVID-19 pandemic are causing professionals in clinical practice and research to reevaluate spirometry’s deployment methods and best practices.

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Sanofi, Glax­o­SmithK­line jump back in­to the PhI­II race for a Covid vac­cine — as the win­ners con­gre­gate be­hind the fin­ish line

Sanofi got out early in the race to develop a vaccine using more of a traditional approach, then derailed late last year as their candidate failed to work in older people. Now, after likely missing the bus for the bulk of the world’s affluent nations, they’re back from that embarrassing collapse with a second attempt using GSK’s adjuvant that may get them back on track — with a potential Q4 launch that the rest of the world will be paying close attention to.

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Matt Gline (L) and Vivek Ramaswamy

In­sid­er ac­count of Roivan­t's SPAC deal — and that $7.3B val­u­a­tion — re­veals a few se­crets as Matt Gline po­si­tions the com­pa­ny as the new ‘Big Phar­ma’

It was Oct. 7, 2020, and Matt Gline wasn’t wasting any time.

The CEO of Roivant had word that KKR vet Jim Momtazee’s SPAC had priced late the night before, triggering a green light for anyone interested in pursuing a big check for future operations and riding the financial instrument to Nasdaq. So he wrote a quick email congratulating Momtazee, whom he knew, for the launch.

Oh, and maybe Momtazee would like to schedule something with Gline and his executive chairman, Roivant founder Vivek Ramaswamy, to chat about Roivant and its business?

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SCO­TUS de­clines to re­view En­brel biosim­i­lar case, tee­ing up 30+ years of ex­clu­siv­i­ty and $20B more for Am­gen’s block­buster

As the House Oversight Committee is set to grill AbbVie CEO Richard Gonzalez on Tuesday over tactics to block competition for its best-selling drug of all time, another decision on Capitol Hill on Monday opened the door for billions more in Amgen profits over the next eight years.

The Supreme Court on Monday denied Novartis subsidiary Sandoz’s petition to review a Federal Circuit’s July 2020 decision concerning its biosimilar Erelzi (etanercept-szzs), which FDA approved in 2016 as a biosimilar to Amgen’s Enbrel (etanercept). Samsung’s Enbrel biosimilar Eticovo also won approval in 2019 and remains sidelined.

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How to man­u­fac­ture Covid-19 vac­cines with­out the help of J&J, Pfiz­er or Mod­er­na? Bi­ol­yse sees the dif­fi­cul­ties up close

When Biolyse, an Ontario-based manufacturer of sterile injectables, forged a deal with Bolivia last week to manufacture up to 50 million J&J Covid-19 vaccine doses, the agreement kicked off what will prove to be a test case for how difficult the system of compulsory licenses is to navigate.

The first problem: When Biolyse asked J&J, via a March letter, to license its Covid-19 vaccine, manufacture it in Canada and pay 5% royalties on shipments to needy, low-income countries, J&J rejected the offer, refusing to negotiate. J&J also did not respond to a request for comment.

Tim Mayleben (L) and Sheldon Koenig (Esperion)

On the heels of a sting­ing Q1 set­back, Es­pe­ri­on's long­time cham­pi­on is ex­it­ing the helm and turn­ing the wheel over to a mar­ket­ing pro

Just days after getting stung by criticism from a badly disappointed group of analysts, there’s a big change happening today at the helm of Esperion $ESPR.

Longtime CEO Tim Mayleben, who championed the company for 9 years from early clinical through a lengthy late-stage drive to successfully get their cholesterol drug approved for a significant niche of patients in the US, is out of the C suite, effective immediately. Sheldon Koenig — hired at the end of 2020 with a resume replete with Big Pharma CV sales experience —  is stepping into his place, promising to right a badly listing commercial ship that’s been battered by market forces.

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No­var­tis' En­tresto takes its 2nd fail­ure of the week­end at ACC, show­ing no ben­e­fit in most dire heart fail­ure pa­tients

Novartis’ Entresto started the ACC weekend off rough with a trial flop in heart attack patients, slowing the drug’s push into earlier patients. Now, an NIH-sponsored study is casting doubt on Entresto’s use in the most severe heart failure patients, another black mark on the increasingly controversial drug’s record.

Entresto, a combination of sacubitril and valsartan, could not beat out valsartan alone in an outcomes head-to-head for severe heart failure patients with a reduced ejection fraction (HFrEF), according to data presented Monday at the virtual American College of Cardiology meeting.

A T-cell play­er with back­ing from Roche takes next big step for BiTE drugs with 'on-of­f' switch to avoid tox­i­c­i­ty

The bispecific T cell engager field is absolutely packed with big-name players who have crowded in despite some high-profile failures in the class. Now, a Bay Area biotech thinks it may have the key to tackling BiTE toxicity, using an old “on-off switch” idea to give doctors more control of the drugs’ effect on patients.

San Francisco-based Soteria Biotherapeutics uncloaked Monday with a $42 million Series A co-led by Roche Venture Fund and 5AM Ventures with participation from the Novartis Venture Fund to advance its bispecific T cell engagers with an “on-off” switch the founders think can avoid some of the dire safety flags endemic to the class.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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