Bob Cuddihy, Capsida CEO

CRISPR Ther­a­peu­tics takes a swing at ALS gene edit­ing in dis­cov­ery deal with AAV up­start Cap­si­da

When small biotech Cap­si­da launched just a few months ago, it hit the scene with back­ing from drug gi­ant Ab­b­Vie to chase AAV-de­liv­ered drugs for CNS. Now, the team has earned the sup­port of one of the biggest names in gene edit­ing — and it’s tak­ing on a big chal­lenge right away.

CRISPR Ther­a­peu­tics has signed a deal with Cap­si­da Bio­ther­a­peu­tics, a gene ther­a­py play­er spe­cial­iz­ing in AAV en­gi­neer­ing, to carve out a de­liv­ery mech­a­nism for the com­pa­ny’s gene edit­ing tech in ALS and rare neu­rode­gen­er­a­tive dis­or­der Friedre­ich’s atax­ia, the com­pa­nies said Tues­day.

As part of their pact, Cap­si­da will pick up R&D re­spon­si­bil­i­ties for the nascent ALS pro­gram as well as chip away at cap­sid de­sign for both pro­grams. CRISPR, mean­while will man­age R&D for the FA pro­gram and de­vel­op gene edit­ing can­di­dates for both, the com­pa­nies said.

In a re­lease, CRISPR tout­ed Cap­si­da’s work in cre­at­ing “high-through­put” ade­no-as­so­ci­at­ed vi­ral vec­tors op­ti­mized to tis­sue tar­get with lim­it­ed off-tar­get side ef­fects. Both com­pa­nies will hold op­tions to co-de­vel­op­ment and -com­mer­cial­iza­tion rights for the pro­gram which the part­ner com­pa­ny leads, CRISPR said.

The fi­nan­cial terms of the deal were not dis­closed.

If one of the part­ners does opt-in, the two firms would share R&D and com­mer­cial­iza­tion costs and prof­its on that giv­en pro­gram. Cap­si­da, with its ex­pe­ri­ence in AAV pro­duc­tion, will han­dle clin­i­cal and com­mer­cial man­u­fac­tur­ing, if it reach­es that point.

Samarth Kulka­rni

“The com­bi­na­tion of Cap­si­da’s AAV en­gi­neer­ing plat­form and CRISPR Ther­a­peu­tics’ gene-edit­ing plat­form has the po­ten­tial to en­able trans­for­ma­tive gene-edit­ed ther­a­pies for pa­tients with neu­ro­log­i­cal dis­eases,” CRISPR CEO Samarth Kulka­rni said in a state­ment. “This new part­ner­ship is one more step in our over­all strat­e­gy of bring­ing to­geth­er in­no­v­a­tive and com­ple­men­tary tech­nolo­gies to un­lock the full po­ten­tial of our core plat­form.”

Kick­start­ed on work by CRISPR/Cas9 in­no­va­tor Em­manuelle Char­p­en­tier back in 2014, CRISPR is per­haps best known for its sick­le cell and be­ta tha­lassemia pro­grams with Ver­tex, which re­cent­ly read out win­ning da­ta. Late last week, the part­ners read out da­ta from two stud­ies show­ing pa­tients dosed with ex­per­i­men­tal drug CTX001 in­di­cat­ed a “con­sis­tent and sus­tained” re­sponse for at least three months af­ter dos­ing. All 15 pa­tients with trans­fu­sion-de­pen­dent be­ta tha­lassemia did not need fur­ther blood trans­fu­sions and all sev­en with se­vere sick­le cell dis­ease were pain free, the biotechs an­nounced.

There was one pa­tient not in­clud­ed in the da­ta cut­off of March 30, how­ev­er, who ex­pe­ri­enced a cere­bel­lar he­m­or­rhage less than three months af­ter be­ing treat­ed. The se­ri­ous side ef­fect was re­lat­ed to the busul­fan con­di­tion­ing gene ther­a­py pa­tients un­der­go be­fore re­ceiv­ing treat­ment, Ver­tex said, and has since re­solved.

Cap­si­da, mean­while, on­ly launched in April with a siz­able $50 mil­lion check and $90 mil­lion in go­ing-out mon­ey from Ab­b­Vie for its own dis­cov­ery pact. The biotech is aim­ing ini­tial­ly at broad rare and neu­ro dis­eases, and Ab­b­Vie came on board with the goal of iden­ti­fy­ing three CNS tar­gets.

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

KEY POINTS

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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James Sabry

'We're in': Roche and Genen­tech join forces on a multi­bil­lion-dol­lar dis­cov­ery pact with a brash AI up­start

Over the past couple of years, the top execs at Roche and Genentech have inked a flurry of deals aligning the global pair with several of the new players that have emerged in the booming AI and machine learning world. That strategy was supercharged in the spring of 2020 by their decision to recruit Aviv Regev out of the computational world she occupied at the Broad. And today they’re taking that computational approach in R&D to a whole new level.

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Brett Monia, Ionis CEO

As­traZeneca grabs PhI­II AT­TR drug from Io­n­is — in­fus­ing $200M cash in­to strug­gling part­ner

AstraZeneca is plucking another antisense drug out of Ionis’ prolific pipeline.

Paying $200 million in cash, AstraZeneca has inked a development and commercialization deal around eplontersen — the Phase III TTR amyloidosis drug formerly known as IONIS-TTR-LRX. On top of the upfront and $485 million worth of conditional payments to follow regulatory approvals, the pharma giant is promising $2.9 billion in sales-related milestones should the drug reach megablockbuster status, plus royalties.

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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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Mar­ket­ingRx roundup: Pfiz­er re-ups pneu­mo­nia ads as Mer­ck threat looms; Re­al Chem­istry founder CEO Jim Weiss steps back

Every autumn, leaves fall from the trees and people start holiday shopping – and for the last few years Pfizer debuts a new “Know Pneumonia” awareness TV ad. This year the commercial, launched a week ago, features different people who talk about why they got vaccinated against pneumococcal pneumonia. Actors portray a young female firefighter with asthma, a mechanic with heart disease and an older woman with her grandchild. A Pfizer spokesperson declined comment on the latest iteration of the long-running campaign.

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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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