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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.