Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the be­gin­ning of 2015, Deer­field Man­age­ment co-led a $10 mil­lion Se­ries C for a pri­vate gene ther­a­py start­up, re­shap­ing the com­pa­ny and bring­ing in new lead­ers to pave way for an IPO just a year lat­er.

Fast for­ward four more years and the start­up, AveX­is, is now a sub­sidiary of No­var­tis mar­ket­ing the sec­ond-ever gene ther­a­py to be ap­proved in the US.

For its part, Deer­field has al­so grown more com­fort­able and am­bi­tious about the nascent field. And the in­vest­ment firm is now putting down its biggest bet yet: a $1.1 bil­lion con­tract de­vel­op­ment and man­u­fac­tur­ing fa­cil­i­ty to pro­duce every­thing one needs for cell and gene ther­a­py — faster and bet­ter than how it’s cur­rent­ly done.

“What we saw dif­fer­ent­ly here is not just the ob­vi­ous — that there’s such a out­sized lev­el of de­mand for the amount of sup­ply — but we al­so saw the need to cre­ate an en­tire ecosys­tem” for gene ther­a­py play­ers, Alex Kar­nal, part­ner and man­ag­ing di­rec­tor at Deer­field, told End­points News. “This is the first time they’re gonna have a place where they can call home.”

Just how big is the de­mand? Days ago John Chimin­s­ki, who’s lead­ing Catal­ent in bulk­ing up its own gene ther­a­py unit, told an End­points au­di­ence that the num­ber of projects in the pipeline is ex­pect­ed to surge from 300 to­day to 1,100 by 2026 — trans­lat­ing to a vol­ume of 2.5 mil­lion to 4 mil­lion liters of vi­ral vec­tors need­ed. That’s up from 300,000 liters now.

Tony Khoury

Deer­field is build­ing its new Cen­ter for Break­through Med­i­cines at a for­mer Glax­o­SmithK­line cam­pus at King of Prus­sia, PA, now run by The Dis­cov­ery Labs. Span­ning 680,000 square feet, the site will con­sist of some­where be­tween 75 to 100 suites spread around 26 in­ter­con­nect­ed build­ings.

Start­ing from an old lab space with much of the equip­ment still in­tact means the cen­ter can hit the ground run­ning, with the site ex­pect­ed to be par­tial­ly func­tion­al by the end of this year and ful­ly up and run­ning in 2021. And do­ing so on the out­skirts of Philadel­phia — where some of the ear­li­est work in cell and gene ther­a­py were done by pi­o­neers such as Carl June and Jim Wil­son — on a sprawl­ing 1.6 mil­lion square feet com­plex de­signed to oth­er biotech star­tups should make it ap­peal­ing to the 2,000 sci­en­tists, man­u­fac­tur­ing ex­perts, tech­ni­cians and sup­port staff Deer­field plans to re­cruit.

With Tony Khoury, a key con­sul­tant for AveX­is, on board as a di­rec­tor and Paragon Bio founder Mar­co Chacón as chair at the Dis­cov­ery Labs, Kar­nal feels con­fi­dent about cast­ing a wide net and train­ing a whole co­hort of cell and gene ther­a­py spe­cial­ists on site. In fact, Deer­field has seen con­sid­er­able in­ter­est in the 24 hours they’ve an­nounced the project.

Which is good, be­cause the King of Prus­sia site is on­ly step 1. Deer­field is plot­ting three more across the US.

Mar­co Chacón

It’s not just about ex­pand­ing ca­pac­i­ty or even hav­ing the first end-to-end pro­duc­tion fa­cil­i­ty. Ac­cord­ing to Kar­nal, they will in­vest in op­ti­miz­ing the process of pro­duc­ing vi­ral vec­tors — from the most com­mon AAV to lentivirus — to both ad­dress the po­ten­cy and yield.

“The tragedy in the mar­ket­place is that the pu­rifi­ca­tion process­es are still in their in­fan­cy; we’re on­ly get­ting yields that are 10 to 20% on av­er­age from a good run,” he said. “You start with 10 to the X virus­es but then af­ter you pu­ri­fy it you lose 70 to 80% of that batch, that’s just — that’s like liq­uid gold be­ing wast­ed.”

The hope is to dou­ble the cur­rent num­bers.

Most of the ini­tial set­up will be geared to­wards gene ther­a­py — one out of 26 build­ings will be re­served for cell ther­a­py — but Kar­nal said they can keep it flex­i­ble for cus­tomers’ needs, re­gard­less of com­pa­ny lo­ca­tion, tar­get tis­sues, or de­liv­ery meth­ods. Each suite can pro­duce around 8 to 12 batch­es every year, and com­pa­nies will have the op­tion to take one whole build­ing for them­selves. The to­tal num­ber of cus­tomers will de­pend on all those vari­ables.

“Gen­er­al­ly speak­ing a batch is pret­ty con­sis­tent­ly priced, what’s not con­sis­tent is how many pa­tients you can serve,” he said.

A num­ber of oth­er com­pa­nies, both drug­mak­ers and con­trac­tors, are rush­ing to serve the same, ever-ex­pand­ing pa­tient pool. No­var­tis and Pfiz­er have com­mit­ted $500 mil­lion and $600 mil­lion on their own pro­duc­tion ca­pa­bil­i­ties, re­spec­tive­ly, and then there’s the CD­MOs like Catal­ent and Ther­mo Fish­er, which have grown their gene ther­a­py teams through bil­lion-dol­lar ac­qui­si­tions of Paragon and Bram­mer Bio. Biotechs big and small are jump­ing in­to the game; Pitts­burgh-based Krys­tal Biotech has just bro­ken ground on a sec­ond com­mer­cial fa­cil­i­ty in Find­ley Town­ship near Ohio.

“We want all the play­ers in the mar­ket­place to make it and be wild­ly suc­cess­ful be­cause the re­al­i­ty of it is even with us and every­body that ex­ists to­day, the de­mand far ex­ceeds the sup­ply still,” Kar­nal said.

Key Avance Clinical executives and CEO Yvonne Lungershausen (centre) at their offices in Australia

How In­no­v­a­tive Tech­nolo­gies and a Ded­i­cat­ed Site Net­work is Dri­ving Pa­tient Re­cruit­ment for Biotech Clin­i­cal Tri­als in Aus­tralia

Australia’s CRO for biotechs, Avance Clinical, deep dives into the patient recruitment landscape identifying the mechanisms and processes that deliver exceptional patient recruitment for early phase trials in Australia.

Avance Clinical also offers models for later phase pivots to large patient populations in Central and Eastern Europe and the US via exclusive collaborations with more than 3,500 sites. Learn more here.

Jim Wilson (WuXi Global Forum at JPM20)

Jim Wil­son of­fers a safer so­lu­tion to some of the in­dus­try's gene ther­a­py woes

Jim Wilson has been warning for a while about the dangers of the viral vectors used in most gene therapies. Two years ago he walked away from Solid Biosciences as he published a paper showing the potential adverse neurological effects of AAV vectors. Last year, he called the field’s progress on vectors “embarrassing.”

Now the gene therapy pioneer, whose tragic 1999 trial showed the deadly risks of the first generation of vectors, is offering a solution. On Wednesday, he and a team of researchers at the University of Pennsylvania published a paper in Science Translational Medicine showing a way of modifying the vectors so they correct genes without hitting a delicate clusters of neurons that, when disturbed, can cause dangerous side effects.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 93,800+ biopharma pros reading Endpoints daily — and it's free.

UCB is beef­ing up its gene ther­a­py ops with back-to-back deals to buy a start­up and sign up a uni­ver­si­ty spin­out for dis­cov­ery work

Belgian pharma player UCB is adding AAV gene therapy programs to the R&D menu through a pair of deals struck in Europe and the US.

The Brussels-based company has acquired a fledgling gene therapy startup in nearby Leuven — Handl Therapeutics — that was launched by longtime Novartis vet Florent Gros, one of two companies he’s co-founded in the last 2 years. And UCB inked a separate research deal with University of Florida spinout Lacerta.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 93,800+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

As Pfiz­er's stock surged Mon­day, Al­bert Bourla net­ted $5.6M in stock sales

On Monday morning, Pfizer announced they had developed the first effective Covid-19 vaccine, the world celebrated, their stock jumped 13% and their CEO became $5.6 million richer.

In an SEC filing Tuesday, Pfizer CEO Albert Bourla disclosed he sold off 132,508 shares at the peak of the Monday surge for $41.92 a piece. Bourla’s sale is kosher because it was planned and pre-approved in August for when the stock hit a certain price, but it adds the Big Pharma chief to the list of executives who have drawn scrutiny for selling shares as their company’s Covid-19 efforts progressed and stocks rose.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 93,800+ biopharma pros reading Endpoints daily — and it's free.

Franz-Werner Haas, CureVac CEO

Pfiz­er’s cel­e­brat­ed Covid-19 vac­cine freezes many peo­ple out — but a ri­val mR­NA team says they thawed out a so­lu­tion

Once the euphoria began to wane a bit following Pfizer’s dramatic unveiling of a 90%-plus effectiveness rate from its pivotal Covid-19 vaccine study earlier this week, less affluent countries and regions around the world began to wonder if they would ever be able to get in line for it.

The vaccine has to be shipped in -70 degrees celsius temps and then used within 5 days of being thawed. For distributors in China, that means building a unique distribution system to deliver the first dose and then a booster. And there are plenty of countries that say they can’t afford to go that route — adding a big obstacle in front of a huge logistical challenge.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 93,800+ biopharma pros reading Endpoints daily — and it's free.

Tom Hochuli (Vibalogics)

Un­der a new CEO, Ger­man CD­MO Viba­log­ics bets $150M and 110,000 square feet on the gene ther­a­py rev­o­lu­tion

Vibalogics started out 18 years ago making vaccines for chickens. Now they’re looking to drive the gene and cell therapy revolution — and make a few Covid-19 shots on the way.

On Wednesday, the German CDMO announced they’re investing $150 million into a 110,000 square foot virus and viral vector manufacturing facility in Boston, joining a wave of companies trying to build the infrastructure that can one day mass produce the hundreds of gene and cell therapies now in biotech and Big Pharma pipelines across the globe.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 93,800+ biopharma pros reading Endpoints daily — and it's free.

Bay­er fronts $65M Se­ries A for Metageno­mi, a gene ther­a­py biotech aim­ing to find new CRISPR en­zymes

Gene editing is all the rage in biotech these days, with the field growing in prominence especially after this year’s Nobel Prize in Chemistry was awarded to two pioneering CRISPR scientists in Emmanuelle Charpentier and Jennifer Doudna. And now, Bayer’s investment arm is going all-in on a new gene editing outfit.

Leaps by Bayer is co-leading a $65 million Series A round for Metagenomi, a biotech launched by UC Berkeley scientists that emerged from stealth on Thursday. Humboldt Fund is co-leading the round with Leaps. Metagenomi, which will be run by Berkeley researcher Brian Thomas, is looking to develop a “toolbox” of both CRISPR- and non-CRISPR-based gene editing systems beyond the Cas9 protein typically seen in the field these days.

Stéphane Bancel, Moderna CEO (JPM20/Jeff Rumans)

Mod­er­na says it's reached the re­quired num­ber of Covid-19 cas­es for its first in­ter­im analy­sis. When is the read­out com­ing?

Pfizer and BioNTech came out with the first Phase III interim analysis of their Covid-19 vaccine earlier this week, and it appears Moderna is right on their heels.

The Cambridge, MA-based biotech announced Wednesday afternoon that it has reached the threshold required to conduct its own initial Phase III interim analysis. And as a result of a “significant increase” in trial participants contracting confirmed cases of Covid-19, Moderna also expects the analysis will include “substantially more” than the 53 cases originally set out for the analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 93,800+ biopharma pros reading Endpoints daily — and it's free.

News brief­ing: Small merg­er to ad­vance an­ti-ag­ing pro­gram; Sanger In­sti­tute spin­out nabs $50M from Se­ries C

Small-cap player Akers Biosciences $AKER has engineered a reverse merger with private company MyMD Pharmaceuticals.

The new combined biotech, which will retain the name MyMD, is expected to trade under the new ticker $MYMD once the transaction closes. MyMD will obtain an 80% stake in Akers, and the deal comes with an $18 million private placement that values the Akers at $1.85 per share, a roughly 7.5% premium above Wednesday’s closing price.