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.”

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.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

More man­u­fac­tur­ing is­sues: Fen­nec preps for sec­ond CRL for po­ten­tial hear­ing loss drug

Shares of Fennec Pharmaceuticals stock were cut almost in half early Monday as the company said manufacturing issues caused another FDA rejection of its reformulated version of sodium thiosulfate, which is intended to help kids who lose hearing due to chemo treatment.

The biotech had resubmitted an NDA for the drug to treat platinum-based, chemo-related ototoxicity in young children earlier this year. The first NDA was denied by the FDA last year, with the agency citing manufacturing issues with the biotech’s supplier.

Like the flu vac­cine every year, the FDA could move quick­ly on a vari­ant-tar­get­ed Covid vac­cine

In the same way that the FDA signs off on flu vaccines every year without requiring large clinical trials to measure their efficacy, the FDA may employ a similar strategy in authorizing variant-focused versions of the mRNA vaccines.

As the world braces for more data on the latest variant Omicron, which may reduce vaccine efficacy, top vaccine developers like Moderna and Pfizer-BioNTech have promised they can pull together a new vaccine targeted against a specific Covid variant in about 100 days. Since Omicron emerged last week, Pfizer-BioNTech, Moderna and J&J have all said they’ve begun work on Omicron-specific vaccines, if needed.

Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; 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.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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