Michael Yang, ViaCyte CEO

CRISPR, Vi­a­Cyte beat Ver­tex to the clin­ic with di­a­betes cell ther­a­py that evades the im­mune sys­tem

The race to de­vel­op a cell ther­a­py for type 1 di­a­betes is heat­ing up, and the team at Vi­a­Cyte and CRISPR Ther­a­peu­tics ap­pears to have pulled ahead by a hair.

Vi­a­Cyte an­nounced Tues­day it’s putting a new ther­a­py in the clin­ic de­rived from “off-the-shelf,” gene-edit­ed, im­mune-eva­sive stem cells, the first time such a treat­ment will be test­ed in hu­mans. The move comes af­ter its clin­i­cal tri­al ap­pli­ca­tion was ac­cept­ed by Cana­di­an reg­u­la­tors, and pa­tient en­roll­ment is ex­pect­ed to start by the end of the year.

“This ap­proach builds on pre­vi­ous ac­com­plish­ments by both com­pa­nies and rep­re­sents a ma­jor step for­ward for the field as we strive to pro­vide a func­tion­al cure for this dev­as­tat­ing dis­ease,” Vi­a­Cyte CEO Michael Yang said in a state­ment.

Both squads had test­ed T1D cell ther­a­pies in hu­mans be­fore, but there’s a par­tic­u­lar dis­tinc­tion with Vi­a­Cyte and CRISPR’s new pro­gram. Pre­vi­ous­ly, the in-hu­man treat­ments had used in­fused, naked, lab-grown islet cells that stim­u­late an im­mune re­sponse, with the body usu­al­ly killing the ther­a­peu­tic cells be­fore they can pro­duce in­sulin.

Those ther­a­pies, there­fore, like­ly would have been lim­it­ed on­ly to pa­tients with se­vere dis­ease, giv­en the po­ten­tial need for ad­di­tion­al im­muno­sup­pres­sive med­i­cines. How­ev­er, Tues­day’s news in­volves cells en­gi­neered to be bet­ter at nat­u­ral­ly avoid­ing the im­mune sys­tem’s trig­gers.

It’s one of a few dif­fer­ent ap­proach­es the Vi­a­Cyte/CRISPR and Ver­tex teams are at­tempt­ing. Vi­a­Cyte had pre­vi­ous­ly tried en­cap­su­lat­ing the cells in a com­pan­ion de­vice, but that method failed mis­er­ably — most pa­tients didn’t see the cells en­graft and al­most no change to their dis­eases.

The im­mune-eva­sive stem cell plan al­so comes af­ter Vi­a­Cyte re­vealed da­ta from a stem cell trans­plant pa­tient, who re­quired im­muno­sup­pres­sive treat­ment, ear­li­er this year. Though ex­treme­ly lim­it­ed, ex­perts said at the time they pro­vid­ed the first proof-of-con­cept for that ap­proach, po­ten­tial­ly pro­vid­ing a path to a func­tion­al cure for both type 1 and type 2 di­a­betes.

It re­mains to be seen how Vi­a­Cyte and CRISPR’s new study will turn out, but Ver­tex isn’t sit­ting idly by. Last month, the biotech re­leased its own da­ta for the im­muno­sup­pres­sive-re­quired method in one pa­tient, re­port­ing their need for in­sulin in­jec­tions van­ished al­most en­tire­ly af­ter suf­fer­ing five near­ly life-threat­en­ing low blood sug­ar episodes in the year be­fore treat­ment.

And though Ver­tex and CRISPR are long­time col­lab­o­ra­tors, they’re com­pet­ing against each oth­er in this race. CRISPR orig­i­nal­ly part­nered with Vi­a­Cyte in 2018 to de­vel­op off-the-shelf gene and cell ther­a­pies for di­a­betes, with Tues­day’s news com­ing from their first pro­gram to­geth­er.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi snaps up new vac­cine can­di­date and de­vis­es mR­NA game plan around it — but not for what you think

Paul Hudson has spotlighted vaccines, immunology and dermatology as some of the top R&D focuses at Sanofi. His latest deal brings all of them together.

The French pharma giant isn’t sharing any financial details about the buyout of Origimm, a low-profile, private Austrian biotech whose technology promises to identify antigens causing skin disease and build vaccines against them. Their lead candidate targets acne vulgaris.

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As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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