CRISPR pays to part­ner with Vi­a­Cyte on an off-the-shelf gene-edit­ing ap­proach to cur­ing di­a­betes

Vi­a­Cyte has been de­vel­op­ing a line of pan­cre­at­ic cells out of stem cells, look­ing for a re­gen­er­a­tive med­i­cine ap­proach to treat­ing Type 1 di­a­betes. 

But there’s a big draw­back. These cells are seen by the im­mune sys­tem as for­eign, so in or­der for it to work, di­a­bet­ics’ im­mune sys­tem has to be sup­pressed — with ob­vi­ous po­ten­tial for harm. 

Samarth Kulka­rni

Now, CRISPR Ther­a­peu­tics $CR­SP, one of the lead­ers in CRISPR/Cas9 gene edit­ing with a big in­ter­est in re­gen­er­a­tive med­i­cine, is step­ping in to use their im­mune-eva­sive tech to cre­ate a new line of cells that can do the job with­out kick­ing in the re­jec­tion re­sponse.

It’s the same ba­sic ap­proach that CRISPR Ther­a­peu­tics is al­ready us­ing for an al­lo­gene­ic — off-the-shelf — ap­proach to CAR-T, says CEO Samarth Kulka­rni. And he’s will­ing to pay a con­sid­er­able amount for a col­lab­o­ra­tion.

In the deal he’s hand­ing over $15 mil­lion up­front and an ad­di­tion­al $10 mil­lion as a con­vert­ible promis­so­ry note of­fered un­der “cer­tain cir­cum­stances.”

The two com­pa­nies are now part­ner­ing on the de­vel­op­ment of an al­lo­gene­ic pro­gram that they plan to joint­ly com­mer­cial­ize.

The last we heard from Kulka­rni the biotech was ex­plain­ing that an IND for a sick­le cell pro­gram they have with Ver­tex was put on hold. 

“We be­lieve the com­bi­na­tion of re­gen­er­a­tive med­i­cine and gene edit­ing has the po­ten­tial to of­fer durable, cu­ra­tive ther­a­pies to pa­tients in many dif­fer­ent dis­eases, in­clud­ing com­mon chron­ic dis­or­ders like in­sulin-re­quir­ing di­a­betes.” not­ed Kulka­rni. “Vi­a­Cyte is a pi­o­neer in the re­gen­er­a­tive med­i­cine field, and has built a com­pelling clin­i­cal pro­gram, ro­bust man­u­fac­tur­ing ca­pa­bil­i­ties, and as­sem­bled a strong in­tel­lec­tu­al prop­er­ty po­si­tion.  Part­ner­ing with Vi­a­Cyte will al­low us to ac­cel­er­ate our ef­forts in re­gen­er­a­tive med­i­cine, an area that we be­lieve will pro­vide a va­ri­ety of longer-term op­por­tu­ni­ties for our com­pa­ny.”

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.

Per­pet­u­al cri­sis? Phar­ma com­mu­ni­ca­tions and pub­lic re­la­tions pros just wait for the next shoe to drop

Welcome to pharma public relations where every day feels like it might be a crisis.

More than three-fourths (76%) of pharma communications leaders expect to face three or more crises this year — compared to less than half (49%) of their peers in other industries, according to new survey from risk intelligence company Crisp. The same 76% also expect that a brand new risk that is yet to be identified will crop up this year, compared to 66% of peers across other industries who worry about the same thing.

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As new C. diff treat­ments prep for mar­ket, spe­cial­ists di­vid­ed on 'poop in a pil­l' or small mol­e­cule, study finds

What do physicians think about the emerging market for recurrent C. diff? That depends on who you talk to, especially which specialists you talk to.

Fecal microbiota transplants, or FMT, are favored by gastroenterologists familiar with them, while infectious disease doctors prefer traditional small molecule therapies, according to a recent future market evaluation by Spherix Global Insights. While current FMT involves transplanting healthy stool into a C. diff patient through a colonoscopy, FMT encapsulated microbiome pills that are swallowed – referred to by doctors as “poop in a pill” in Spherix’s interviews – are likely on the way to market.

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