Chris Garabedian (Forge Biologics)

Chris Garabe­di­an's in­cu­ba­tor leads Se­ries A fund­ing for gene ther­a­py man­u­fac­tur­er/de­vel­op­er com­bo

Back in De­cem­ber, long­time in­dus­try in­vestor Chris Garabe­di­an helped launch a $210 mil­lion VC fund for ear­ly-stage biotechs. On Tues­day, Garabe­di­an and the Per­cep­tive Xon­toge­ny Ven­ture Fund in­tro­duced its lat­est start­up.

Tim Miller

The ex-Sarep­ta CEO has teamed with for­mer Abeona chief Tim Miller to cre­ate Forge Bi­o­log­ics, a gene ther­a­py com­pa­ny fo­cus­ing on both man­u­fac­tur­ing and de­vel­op­ment of GMP ade­no-as­so­ci­at­ed virus­es, with $40 mil­lion in Se­ries A fund­ing. Forge is the eighth biotech in which the PXV Fund has in­vest­ed — but on­ly the sixth an­nounced — and the first in the gene ther­a­py area, Garabe­di­an told End­points News.

Dri­ve Cap­i­tal al­so par­tic­i­pat­ed in the fund­ing. Be­cause Forge will serve as both a con­tract de­vel­op­ment and man­u­fac­tur­ing or­ga­ni­za­tion (CD­MO) and a pro­duc­er of its own pipeline, Garabe­di­an sees up­side here.

“That com­bi­na­tion CD­MO and pro­pri­etary pipeline is rare to find, that ca­pa­bil­i­ty in one shop,” Garabe­di­an, who will serve as Forge’s chair­man, said.

Own­ing and con­tract­ing out its man­u­fac­tur­ing fa­cil­i­ty, a 175,000-square-foot for­mer ware­house space dubbed “The Hearth” in Colum­bus, Ohio, will al­low Forge to cre­ate a net­work of clients en­com­pass­ing not on­ly biotechs but aca­d­e­m­ic in­sti­tu­tions and re­search foun­da­tions. There is huge de­mand for such spe­cial­ty sites in the gene ther­a­py field, and Miller said Forge has al­ready se­cured some po­ten­tial clients.

The high de­mand, com­bined with hav­ing the ca­pac­i­ty to de­vel­op AAVs on-site, makes for “a very com­pelling busi­ness mod­el,” Garabe­di­an said. Ear­li­er this year, Deer­field Man­age­ment in­vest­ed $1.1 bil­lion in a new CD­MO fa­cil­i­ty while two oth­er big play­ers, Catal­ent and Ther­mo Fish­er, had pre­vi­ous­ly spent over $1 bil­lion each on gene ther­a­py man­u­fac­tur­ers.

Forge’s chal­lenge in meet­ing that de­mand will be up­scal­ing its pro­duc­tion ca­pa­bil­i­ties, and the com­pa­ny plans to have ser­vices at the 500-liter scale by mid-2021, up from 50 liters cur­rent­ly.

“We’re us­ing what we con­sid­er to be a rel­a­tive­ly stan­dard way of ap­proach­ing us­ing a sus­pen­sion cul­ture, but with an ex­pe­ri­enced team who can take those cul­tures and scale those for the mass­es,” Miller said.

In terms of its own pipeline, Forge is ini­tial­ly re­search­ing an AAV in com­bi­na­tion with um­bil­i­cal cord bone mar­row (UCBM) trans­plants as a treat­ment for Krabbe dis­ease, a ge­net­ic dis­or­der that in many cas­es caus­es death by age 2. The pro­gram, la­beled FBX-101, is ex­pect­ed to be in ear­ly clin­i­cal tri­als by the end of 2020.

Miller specif­i­cal­ly men­tioned Philadel­phia-based Pas­sage Bio, whose Krabbe dis­ease treat­ment will hit Phase I/II clin­i­cal tri­als in the first half of 2021, as a po­ten­tial com­peti­tor. While Pas­sage Bio fo­cus­es sole­ly on AAV de­liv­ery, Forge aims to prove that its UCBM and AAV com­bo will ul­ti­mate­ly be safer and ef­fi­ca­cious.

“What this [com­bi­na­tion] does is it gives the pa­tients a re­set, or a new im­mune sys­tem,” Miller said, “so they don’t have an im­mune re­sponse against the pro­tein that would be ex­pressed dur­ing this gene ther­a­py, and it sup­press­es any im­mune re­sponse to the ac­tu­al AAV.”

Of course, Krabbe dis­ease treat­ments are just the first in what Forge hopes is a long fu­ture of ser­vic­ing and de­vel­op­ing gene ther­a­py. The biotech has two oth­er in-house projects it’s cur­rent­ly work­ing on, one of which is re­lat­ed to on­col­o­gy.

Look­ing for­ward, Forge in­tends to be a “de­vel­op­ment en­gine” that us­es Garabe­di­an’s seed fund back­ground with Miller’s vast net­work to ser­vice ear­ly-stage man­u­fac­tur­ing while al­so tak­ing on new projects it­self.

“Be­tween the two of us, we’re pret­ty plugged in to the emerg­ing gene ther­a­py re­search projects,” Garabe­di­an said.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Quince Ther­a­peu­tics faces takeover bid from share­hold­er Echo Lake Cap­i­tal

A bid to take over the biotech Quince Therapeutics has been put forward by one of its shareholders.

On Tuesday, Echo Lake Capital sent a letter to Quince’s board of directors putting forth a proposal to acquire all the biotech’s stock for $1.60 per share, which would value a takeover at around $58 million.

In the letter, Echo Lake said that it believes Quince’s stock is severely undervalued and that no drugs are being actively marketed or developed that require cash expenditures. It’s trading below the value of its assets, Echo Lake said.

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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