Danuta Jeziorska, Nucleome Therapeutics CEO

Drug dis­cov­ery biotech look­ing in­to the ‘dark’ re­gions of the hu­man genome picks up a $40M+ Se­ries A

While the com­bo of ma­chine learn­ing and drug dis­cov­ery has man­aged to rake in a lot of cap­i­tal for com­pa­nies this year, one biotech is look­ing to the dark side of the genome to stand out.

Nu­cle­ome Ther­a­peu­tics, a UK-based com­pa­ny found­ed in 2019 and spun off from Ox­ford Uni­ver­si­ty, is seek­ing to cre­ate pre­ci­sion med­i­cines by de­cod­ing “dark mat­ter” from the hu­man genome to find tar­gets no one else has gone af­ter. The com­pa­ny has now gar­nered a £37.5 mil­lion ($42.4 mil­lion) Se­ries A to ad­vance its dis­ease pro­grams and build out its plat­form.

In an in­ter­view with End­points News, Nu­cle­ome CEO and co-founder Danu­ta Jeziors­ka said when the hu­man genome was se­quenced, sci­en­tists found that around 98% of the DNA that we have did not con­tain genes. While they didn’t ful­ly ap­pre­ci­ate what it was, the sci­en­tif­ic com­mu­ni­ty has come to un­der­stand that with­in this space there is an “in­struc­tion man­u­al of sorts.” The area es­sen­tial­ly does not en­code pro­teins but con­tains a large num­ber of ge­net­ic changes as­so­ci­at­ed with dis­ease.

“So, if you think in each cell, you have the same DNA, you have two me­ters of DNA, but then you have hun­dreds of cell types,” she said. “So how the cell knows that from the 22,000 genes that are there, which ones to turn on and which ones to turn off, and al­so in which amounts? And this is the dark genome — it’s like the in­struc­tion man­u­al.”

If this area is af­fect­ed there could be ei­ther not enough or too much pro­tein pro­duced, or an onco­gene can be turned on in the wrong place. Jeziors­ka of­fers the dis­ease al­pha tha­lassemia as an ex­am­ple of the role the dark genome can play when af­fect­ed.

One of the rea­sons that oth­ers have not pur­sued the dark genome, Jeziors­ka said, is that pre­vi­ous tools were not present to un­der­stand its func­tion and were not pre­cise enough, but that us­ing ma­chine learn­ing and the com­pa­ny’s 3D tech­nol­o­gy might get the job done.

The com­pa­ny uti­lizes “3D genome tech­nol­o­gy” in its plat­form along with ma­chine learn­ing to dis­cov­er genes that are linked to dis­eases, with the even­tu­al goal of find­ing drug­gable can­di­dates. This tech­nol­o­gy was in­vent­ed by the found­ing team at Nu­cle­ome and al­lows the team to look at how DNA is fold­ed in the cell from a 3D per­spec­tive and help to un­lock the ge­net­ic vari­ants in the “dark” ar­eas, which will help elu­ci­date their func­tion, Jeziors­ka ex­plained.

By us­ing this tech­nol­o­gy com­bined with ma­chine learn­ing, the com­pa­ny is cur­rent­ly look­ing at tar­gets in the au­toim­mune dis­ease space, how­ev­er, the com­pa­ny does not have a can­di­date pre­pared yet.

So far, the com­pa­ny has raised £5.2 mil­lion ($5.8 mil­lion) in seed fund­ing and cur­rent­ly has a team of around 20. This lat­est in­jec­tion will give the com­pa­ny around three years of run­way to build out its plat­form and get a can­di­date to­geth­er. The com­pa­ny will al­so look to dou­ble its head­count and will look to build part­ner­ships with larg­er phar­ma com­pa­nies.

The round was led by M Ven­tures, the VC arm of Mer­ck KGaA. John­son and John­son In­no­va­tion, Pfiz­er Ven­tures, British Pa­tient Cap­i­tal and Ox­ford Sci­ence En­ter­pris­es all par­tic­i­pat­ed in the round.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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John Flavin, Portal Innovations CEO

Por­tal In­no­va­tions takes its Chica­go biotech in­vest­ing, lab space mod­el to Boston with Bob Langer start­up in tow

Life sciences investor and lab operator Portal Innovations is taking the blueprint it set in Chicago and expanding to Massachusetts, where it will deploy $25 million, and help house new startups in the former Boston Globe headquarters.

Portal CEO and founder John Flavin told Endpoints News the company will open its Boston-area operations in the coming weeks, and its first lab occupant will be Syntis Bio, an early-stage biotech out of Moderna co-founder Bob Langer, MIT professor Giovanni Traverso and Sherlock Biosciences co-founder Rahul Dhanda.

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Deborah Waterhouse, ViiV Healthcare CEO

MPP re­cruits three gener­ic man­u­fac­tur­ers to ex­pand use of Vi­iV's in­jectable PrEP drug

ViiV Healthcare has teamed up with the UN-backed Medicines Patent Pool and three generic manufacturers to expand access to medicine that can prevent HIV.

ViiV and the Medicines Patent Pool jointly announced Thursday that the MPP signed sub-licensing agreements with Aurobindo, Cipla and Viatris to manufacture generics of a long-acting form of cabotegravir for HIV pre-exposure prophylaxis (PrEP). As a result of the agreement, the manufacturers will be able to develop, manufacture and supply generic versions of cabotegravir LA in 90 countries — subject to regulatory approvals.