An­oth­er gene ther­a­py com­pa­ny launch­es with VC cash and a CRISPR tech to tar­get tox­ic RNA

Gene Yeo Yeo Lab

A San Diego-based biotech has at­tract­ed sub­stan­tial ven­ture sup­port for its clin­i­cal for­ay in­to gene ther­a­pies that can tar­get tox­ic brands of RNA — sparked by DNA mu­ta­tions — that trig­ger ge­net­ic dis­eases. And they’ve wooed a well-known West Coast en­tre­pre­neur to the helm.

The biotech is called Lo­cana, which is work­ing on the sci­en­tif­ic ex­plo­rations done by UC San Diego pro­fes­sor Gene Yeo. Yeo’s pub­lished work in­cludes lab ex­per­i­ments that used CRISPR Cas9 tech to mod­i­fy repet­i­tive RNA —an ap­proach with im­pli­ca­tions for some tough dis­eases like my­oton­ic dy­s­tro­phy type 1 and 2, Hunt­ing­ton’s dis­ease, and C9orf72-linked amy­otroph­ic lat­er­al scle­ro­sis. That work was pub­lished in Cell.

Look­ing over their web site, the com­pa­ny plans to use a gene ther­a­py vec­tor to de­liv­er an RNA-tar­get­ing pro­tein with an en­zyme at the tip.

The Lo­cana plat­form Lo­cana

Click on the im­age to see the full-sized ver­sion

Jef­frey Os­trove Lo­cana

Rare ge­net­ic dis­eases and new gene ther­a­py ap­proach­es have been all the rage now for a few years, com­ing af­ter years of frus­tra­tion when it comes to ear­li­er at­tempts to ad­dress some of these dis­eases. So it’s no won­der that some high pro­file VCs are jump­ing in­to this for the ride.

Arch is lead­ing the way on this new A round, which weighs in at $55 mil­lion — which looks like a tidy sum to get up to proof-of-con­cept work. Google’s GV is jump­ing in along­side UCB for the first time, join­ing Temasek and Light­stone Ven­tures — which have been in­volved from the start.

David Nelles Yeo Lab

Lo­cana is now a pre­clin­i­cal com­pa­ny, so get­ting in­to hu­mans looks to be a top pri­or­i­ty.

Along with the mon­ey, Lo­cana is get­ting a CEO: Jef­frey Os­trove. Os­trove’s lengthy re­sume in­cludes an­oth­er CEO role at Gen­Stem in San Diego, which is work­ing on “lentivi­ral mod­i­fied stem cell treat­ments for lyso­so­mal stor­age and mi­to­chon­dr­i­al dis­eases.” He was al­so CEO of Ab­Vit­ro, which Juno snapped up in a $125 mil­lion deal back in 2016.

David Nelles is a co-founder.

Im­age: Shut­ter­stock

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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.

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.

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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.

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.

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Benjamin Oakes, Scribe Therapeutics CEO

CEO of Doud­na spin­out: With­in five years, genome ed­i­tors will have a 're­al­ly big im­pact' on pa­tients' lives

“CRISPR-by-design” is the idea behind Scribe Therapeutics, a company spun out from Jennifer Doudna’s Nobel-winning lab that’s competing in a closely-tracked field of genome editor companies just starting to make their way to the clinic.

After nabbing $100 million last March for its Series B funding round, Scribe is taking a different tack from some of its competitors, crafting a new enzyme isolated from bacteria called CasX, which has now been tweaked extensively and may be targeted to a range of genome-related diseases, offering a plethora of therapeutic options.

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Ab­bott pumps $450M+ in­to new Ire­land-based man­u­fac­tur­ing site project and hir­ing spree

As Ireland continues to see more investments and building projects from pharma companies, another contender is looking to place more investment in the Emerald Isle.

According to a report from The Irish Times on Friday, Abbott Laboratories is investing €440 million, or about $451 million, to build a new manufacturing plant in Kilkenny, located in the country’s southeast, to make more of its glucose monitors.

Andy Kidd, Aptinyx CEO

‘The place­bo caught up’: Months af­ter di­a­bet­ic nerve pain fail, Aptinyx sees an­oth­er pain tri­al fall through

In 2019, Aptinyx’s stock cratered after it reported that its lead candidate failed a diabetic nerve pain trial. After some ‘further analysis,’ the biotech re-upped with that same non-opioid pain drug in two more mid-stage studies — another diabetic nerve pain trial and later a fibromyalgia trial.

In April, Aptinyx reported that its second diabetic nerve pain trial also fell through. However, the Illinois-based penny stock biotech had one more shot for its NMDA-modulating drug, dubbed NYX-2925, in fibromyalgia.

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Tony Coles, Cerevel CEO

Cerev­el takes the pub­lic of­fer­ing route, with a twist — rais­ing big mon­ey thanks to ri­val da­ta

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

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