Ed­i­tas and Cel­gene sub Juno are tack­ling hottest im­munother­a­py cells

As the first CRISPR-edit­ed can­cer pa­tients watch their treat­ments un­fold, one of the first CRISPR com­pa­nies is re­jig­ging a ma­jor on­col­o­gy deal.

Ed­i­tas Med­i­cine is amend­ing its long-run­ning col­lab­o­ra­tion with Cel­gene and their sub­sidiary Juno Ther­a­peu­tics. The new deal will ex­pand the fo­cus of their work to cov­er a sub­set of im­mune cells that have be­come an in­creas­ing­ly hot tar­get for im­munother­a­py: gam­ma-delta cells.

The deal will make Ed­i­tas el­i­gi­ble for a $70 mil­lion pay­ment along with oth­er pos­si­ble mile­stones and roy­al­ties.

“It’s a sig­nif­i­cant ex­pan­sion” of the deal, Ed­i­tas CSO Char­lie Al­bright told End­points News. These cells are part of the im­mune sys­tem and have sig­nif­i­cant po­ten­tial to treat sol­id tu­mors.

Char­lie Al­bright

Since it be­gan in 2015, the Juno-Ed­i­tas col­lab­o­ra­tion has fo­cused large­ly on al­pha-be­ta cells, the ones out­fit­ted with the spe­cial re­cep­tors in cur­rent CAR-T treat­ments. Sci­en­tists at those com­pa­nies and else­where have most pub­licly tried to ap­ply CRISPR to im­prove CAR-T, which now work sole­ly through vi­ral gene trans­fer.

But they have al­so worked on ex­pand­ing the ap­proach to oth­er im­mune cell types in hopes of mak­ing the treat­ment more ef­fec­tive, more ac­ces­si­ble or — as is the case with some of the gam­ma delta re­search — ex­pand it in­to oth­er can­cer types, es­pe­cial­ly sol­id tu­mors.

Ed­i­tas has been slow­ly build­ing their gam­ma-delta base through­out the year, Al­bright said. In April, they signed an agree­ment with Blue­Rock, in part to ac­cess pluripo­tent stem cells they hope to make in­to en­gi­neered gam­ma-delta cells that can be de­liv­ered to a pa­tient. (Es­sen­tial­ly a form of off-the-shelf CAR–T).

Sev­er­al com­pa­nies are now pur­su­ing gam­ma-delta im­munother­a­pies, in­clud­ing Gam­maDelta Ther­a­peu­tics and its new spin­off Adap­tate and Re­gen­eron-backed Adicet Bio. They’re bet­ting chiefly on these cells’ abil­i­ty to pen­e­trate the sol­id tu­mors that have been so re­sis­tant to the first wave of CAR-T treat­ments.

Al­bright ar­gued, though, that for these tech­niques to work you need gene edit­ing. In­nate abil­i­ties in the cells have to be tuned up, he said. You have to in­crease cells’ per­sis­tence and en­hance their abil­i­ty to sur­vive in a tu­mors’ mi­cro-en­vi­ron­ment. Ide­al­ly, he said, you even give it new abil­i­ties, such as the pow­er to cat­alyze the body’s in­nate im­mune sys­tem.

“You can’t do that with vi­ral trans­duc­tion,” Al­bright said. “You need gene edit­ing.”

Spe­cial re­port: Meet 20 ex­tra­or­di­nary women who are su­per­charg­ing bio­phar­ma R&D

Even though many biopharma leaders have come together in recent years to address its gender gap, the consensus is clear: We still have a long way to go.

Companies this year were 2.5 times more likely than last year to have a diversity and inclusion program in place, according to a recent BIO survey, but women are still largely absent from executive roles. Getting women to enter the industry isn’t the problem — studies show that they represent just under half of all biotech employees around the world. But climbing through the ranks can be challenging, as women still report facing stereotypes, and, unfortunately, harassment.

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Op­ti­miz­ing Oral Drug De­liv­ery us­ing Zy­dis® Oral­ly Dis­in­te­grat­ing Tablet Tech­nol­o­gy to Ad­dress Pa­tient Chal­lenges


Patients prefer oral dosing, but swallowing tablets can be a challenge for many patients.
The Zydis® orally disintegrating tablet (ODT) platform addresses challenges associated with oral dosing, expanding benefits for patients and options for healthcare providers.
A strong growth trajectory is expected for ODTs given therapeutic innovation and continued technology development.

Many patients prefer conventional tablets for the administration of medications, but some geriatric and pediatric patients and those with altered mental status and physical impairments find swallowing tablets to be difficult. Orally disintegrating tablets (ODTs), which dissolve completely without chewing or sucking, offer a patient-friendly dosage form for the administration of small-molecule drugs, peptides and proteins. With the potential for multiple sites of drug absorption, often faster onset action for the active pharmaceutical ingredient (API), and potentially greater bioavailability, ODTs are an attractive option for drug developers considering first-to-market formulations or product line extensions of existing drugs with compatible API. In this report, we look at how innovation in the industry-leading Zydis ODT platform is expanding oral formulation options and bringing benefits to patients.

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Geoffrey Porges (SVB Leerink)

The 2022 wave com­ing? Top an­a­lyst says Big Phar­ma will have more than $1T avail­able to sat­is­fy its grow­ing ap­petite for biotech M&A

All through this year you could practically feel the frustration of the biotech investor class as M&A activity continued to drag behind expectations — or desires. Buyouts of public companies provide the essential juice for keeping stocks lively, and there’s been a notable lack of juice in 2021.

So is all that about to change, big time?

SVB Leerink’s Geoffrey Porges, a longtime student of biotech M&A, thinks so. In a lengthy analysis he put out last week, Porges totted up the cash flow of the major pharmas and determined that there was a good long list of industry buyers who would have around a half trillion dollars of cash to play with in 2022. Leverage that up with added debt and you could get that deal cache to $1.6 trillion.

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Mer­ck pumps the brakes on two more PhI­II tri­als for its lead an­ti-HIV drug

After trial investigators flagged a drop in immune cell counts that an external committee determined was related to treatment last month, Merck has been pausing HIV-related Phase II and III trials ever since.

On Monday, the biopharma company announced it’s pausing enrollment in two of its Phase III trials evaluating its leading anti-HIV drug candidate, which is the once-monthly, oral islatravir.

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Bolt Bio CEO Randy Schatzman

Bolt Bio goes bust as in­vestors boo sin­gle re­sponse in ear­ly test against HER2-ex­press­ing tu­mors

Bolt Bio’s BDC-1001, an antibody conjugate drug designed to amp up the body’s innate immune system response to tumors, posted a single partial response in a Phase I/II study in patients with HER2-expressing solid tumors after a year of dosing. Just 13 of 40 evaluable patients showed any signs of “clinical activity,” the biotech said Monday.

BDC-1001 links a HER2-targeting biosimilar of Herceptin with a TLR7/8 agonist, which is designed to activate myeloid cells in the innate immune system and drive tumor cytotoxicity, Bolt said. But the early results paint the picture of a drug with little effect on HER2 tumors, one of the most highly validated tumor targets in drug development.

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Biohaven CEO Vlad Coric (Photo Credit_ Andrew Venditti)

Bio­haven shakes up lead­er­ship team as it feels the heat from mi­graine com­peti­tors

As Biohaven Pharma comes off a rollercoaster pipeline year, its CEO will take on more responsibility in a full C-suite makeover.

Vlad Coric was unanimously elected to the role of chairman of the board of directors, after Declan Doogan’s retirement. Matthew Buten will take over the role of CFO after James Engelhart’s retirement, and director Michael Heffernan has been appointed lead independent director. All of the appointments are effective immediately, a company press release said.

Chen Schor, Adicet CEO

Adicet un­veils ear­ly re­spons­es for off-the-shelf drug lever­ag­ing rare T cells. Will dura­bil­i­ty hold up?

On the hunt for the next generation of “off-the-shelf” cell therapies, biotech players like Adicet Bio have looked to leverage some of the less-obvious members of the immune system as potent cancer fighters. In Adicet’s case, scarce gamma delta T cells are on the menu, and an early cut of data is showing some promise.

Adicet’s AD-001, an off-the-shelf cell therapy developed by engineering a CD20-targeting chimeric antigen receptor (CAR) onto a donor’s gamma delta T cells, posted two complete responses across four patients in an early Phase I study testing the drug in patients with heavily pretreated B cell non-Hodgkin’s lymphoma, the biotech said Monday.

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Mar­ket­ingRx Matchup: How Ab­b­Vie and Bio­haven ads rank in head-to-head mi­graine chal­lenge

Are you ready to rumble? DTC brands that is. MarketingRx is launching a new monthly feature today called MarketingRx Matchup. We’re pitting two pharma brands’ DTC advertising in the same therapeutic category against each other to find out what consumers and patients really think.

Market research company Leger is handling the polling and analysis each month, and I’ll be writing up the results — along with my own take — inside MRx on the first Tuesday of the month.

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Wendy Lund, Organon chief communications officer

Q&A: Organon chief com­mu­ni­ca­tions of­fi­cer Wendy Lund talks about the Mer­ck spin­off, women’s health and why it mat­ters

One of Wendy Lund’s earliest jobs was head of marketing at Planned Parenthood. As the youngest person on its management team, she introduced them to emerging new technologies, and in return, she learned the importance of fighting for what you believe in.

Now as chief communications officer at Organon, the women’s health company recently spun off by Merck, Lund is keeping that point top of mind. That’s in part because women’s health hasn’t been a spotlight therapy area for Big Pharma in years. Several companies have spun off, sold or at least considered selling women’s health assets to focus on “core” products.

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