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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Yao-Chang Xu, Abbisko Therapeutics founder and CEO

Qim­ing-backed Ab­bisko makes $200M+ Hong Kong de­but, as a SPAC and Agenus spin­out al­so price on Nas­daq

Three new entities priced their public debuts late Thursday and early Friday, including a SPAC, a traditional Nasdaq IPO and a Chinese biotech joining the Hong Kong Index.

Shanghai-based Abbisko Therapeutics raised the most money of the triumvirate, garnering $226 million in its Hong Kong debut and pricing at HK$12.46, or roughly $1.60 in US dollars. The blank check company followed up with a $150 million raise, while MiNK Therapeutics priced on Nasdaq at $12 per share and a $40 million raise.

Paul Grayson, Tentarix CEO (Versant)

Phar­ma vet­er­ans re­group with $50M and a plan to dis­cov­er new mul­ti-specifics

While a horde of drugmakers develops bispecific antibodies to more directly target tumor cells — there were about 100 programs in or nearing clinical trials back in May — a new company is emerging to go one step further.

On Thursday, Tentarix Biotherapeutics unveiled a $50 million Series A round to support its next-gen multi-specifics platform. While the field has largely focused on bispecifics, which engage two targets, Tentarix believes its multifunctional programs have the potential to be even more specific, since more conditions must be met for potent activity to occur.

Tillman Gerngross, Adagio CEO

Q&A: Till­man Gern­gross ex­plains why his Covid mAb will have an edge over an al­ready crowd­ed field

If anyone knows about monoclonal antibodies, it’s serial entrepreneur, Adimab CEO, and Dartmouth professor of bioengineering Tillman Gerngross.

Even the name of Gerngross’ new antibody startup Adagio Therapeutics is meant to reflect his vision behind the development of his Covid-19 mAb: slowly, he said, explaining that “everyone else, whether it’s Regeneron, Lilly, or AstraZeneca, Vir, they all valued speed over everything.”

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