At the Bio International Convention held last year in Philadelphia, The Discovery Labs announced it was opening 1.6 million square feet of coworking space and collaborative labs for healthcare, life sciences and tech companies out of GlaxoSmithKlinesformer campus in King of Prussia.

About half a year later, nearly all its square footage has been accounted for, Audrey Greenberg, executive managing director of the Discovery Labs, told Technical.ly.

Included is a big get for the region: The Discovery Labs and Deerfield Management Company have formed The Center for Breakthrough Medicines, a contract development and manufacturing organization and specialty investment company, the Discovery Labs announced this week.

The Center for Breakthrough Medicines leased 680,000 square feet of Discovery Labs facility to provide pre-clinical through commercial manufacturing of cell and gene therapies, including process development, plasmid DNA, viral vectors, cell banking, cell processing and support testing capabilities.

Its goal is to to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies, Discovery Labs said.

Today brilliant scientists are advancing an unprecedented number of gene and cell therapy drug candidates. The real tragedy, however, is a scarcity of manufacturing know-how, which is complex and expensive, said Alex Karnal, partner and managing director of Deerfield Management and a board member of the Discovery Labs, in a statement. It is hoped that the Center for Breakthrough Medicines will help realize the promise of cell and gene therapies in time to treat the many patients who need them.

TheCenter for Breakthrough Medicines has initiated a substantial hiring effort, with plans to hire more than 2,000 team members within the next 30 months in positions like Ph.D. scientists, manufacturing experts, lab technicians and support staff.

Greenberg called the company an end-to-end solution for companies that are looking to manufacture their solutions and products.

There wasnt really space for that to happen, and were in a biotech epicenter, she said. Companies can grow within our space.

An early rendering of The Discovery Labs. (Courtesy image)

First leases within the 1.6 million square feet across campus will likely be taken up by 10 to 20 companies in the first quarter of 2020. Many, including the Center for Breakthrough Medicines, will move into the space in Q3 or Q4 of 2020, after renovations are complete and labs are outfitted to each companys needs.

The campus is also getting a biotech incubator, Unite IQ, which will offer space to emerging life sciences startups with resources needed to initiate business operations. Unite IQ tenants will be able to use the discovery, development, testing and manufacturing capabilities of the Center for Breakthrough Medicines and tech transfer from research lab to large-scale production.

Currently, Philadelphia is home to the only Discovery Labs campus, but the company is looking toward expanding with locations on the West Coast, in Asia and in Europe.

The region is continuously growing as a hub for life sciences, andGreenberg cited King of Prussias proximity to top cell and gene therapy talent, area hospitals and patient pools as important to the Discovery Labs. Also important, she noted, was access to the Philadelphia International Airport (and its supply-chain abilities with cold storage function for transporting cell therapies).

The work is extremely important to Greenberg personally, she said.

Theres an immediacy to this work, in this industry thats working on cures for patients who are dying every day, she said. To me, this work is doing well by doing good.

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A cell and gene therapy development center is taking 680K square feet at KOP's Discovery Labs - Technical.ly

Passage Bio Jim Wilsons self-described legacy company has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

He joins as the biotech preps IND filings for three lead programs in rare, monogenic diseases of the central nervous system in 2020 the lysosomal storage disorders GM1 gangliasidosis and Krabbe disease, as well as frontotemporal dementia.

Bruce is ideally suited to lead Passage Bio as chief executive officer given his strong neuroscience background coupled with his robust healthcare and biotechnology industry experience, board chairman Tachi Yamada said in a statement.

Passage launched last February with $115 million from a marquee group of Series A investors including Frazier (where Yamada is a partner), OrbiMed, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures. With an office just a 10-minute walk away from Wilsons lab at the University of Pennsylvania, the company was designed to apply the gene therapy pioneers 35-year experience into cross-correctional therapies for CNS.

According to what he calls the Jim Wilson 90/10 rule, Squinto previously told Endpoints News, AAV vectors can cover and transduce 90% of motor neuron cells but only 10% to 15% of other brain cells making it difficult to go after indications where broad transduction is needed. But it can still prove useful in disorders that result from mutations in enzymes that can be taken up by neighboring cells once secreted normally.

A close pact with Penns Gene Therapy Program and Orphan Disease Center gave Passage rights to five programs right out of the gate, with options to license seven more.

Its a very aggressive clinical development strategy across a multitude of programs, Squinto said as he closed a $110 million Series B in September. Were not gonna rely on any one program to drive the value of Passage, were gonna rely on what is a very very full pipeline of opportunities.

Goldsmith will now lead a team of about 25 to build on preclinical and IND-enabling data from Wilsons lab a company growing exercise he honed as COO of Lycera. There, he was also credited for a number of business development initiatives.

The transition into the clinic would also mean moving production from early facilities at Penn to Paragons GMP sites, and eventually to a customized suite slated for completion in the third quarter of this year.

Squinto, a rare disease expert who devoted much of his career to Alexion, will continue to help oversee all of that as a board director.

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Eyeing a trio of trial initiations, Jim Wilson's gene therapy startup woos Bruce Goldsmith from Deerfield as CEO - Endpoints News

Manufacturing issues and a scarcity of new commercial products leave predictions that 10-20 cell and gene therapy approvals each year by 2025 somewhat fanciful, says Dark Horse Consulting.

In his plenary address at the Phacilitate conference yesterday, Anthony Davies, founder of cell and gene therapy specialist firm Dark Horse Consulting, reflected on the difficulties the sector has faced since the high of 2017 when three products achieved US Food and Drug Administration (FDA) approval: Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), and gene therapy Luxturna (voretigene neparvovec).

A few years ago, I introduced this evening by saying: Finally the field has had the year that weve been saying we are going to have for years. That was a great year, he told the packed room in Miami, Florida.

Dark Horses Anthony Davies opened the Phacilitate conference in Miami, Florida

The CAR-T therapies Kymriah and Yescarta gave hope to patients who previously could measure their life expectancy in a small number of months, while gene therapy Luxturna offered hope to children whose ophthalmic deterioration was a statistical certainty, he added.

With these breakthroughs, positivity was high and in January 2019 then FDA Commissioner Scott Gottlieb predicted in an agency statement that there will be upwards of 200 regenerative medicine IND submissions from 2020, and by 2025 the agency will be approving 10 to 20 cell and gene therapy products a year.

I think 200 INDs is doable this year, but INDs do not cure patients, Davies said. And I think if weve struggled with getting three commercial approvals in the years after that first year when three commercial approvals were made, so getting 10-20 in five years from now is going to be extremely challenging.

Since that breakthrough year, the industry has been hot by bad news and a lack of commercial products. Novartis/AveXis Zolgensma (onasemnogene abeparvovec) and bluebirds Zynteglo (autologous CD34+ cells encoding A-T87Q-globin gene) were approved by the FDA last year, while Takedas allogeneic cell therapy Alofisel (darvadstrocel) has been approved to a certain extent in Europe.

While Davies described the approval of Zolgensma, at a cost of $2.1 million, as groundbreaking, he noted it has been overshadowed by a scandal involving data falsification during the approval process.

He also noted that Zynteglos success has been muted by multiple manufacturing problems which has delayed launch.

Meanwhile, pioneer product Kymriah continues to suffer from manufacturing difficulties, and Novartis seems to be struggling with fixing them, Davies suggested.

At JP Morgan [Healthcare Conference] it was announced that for 10% of patients no shipment of drug is made, and for a very significant minority of patients shipment is made with out-of-spec product for which Novartis cannot charge, he told the conference

He added that at the investor conference last week, Novartis CEO Vasant Narasimhan said that they had made great process in identifying the manufacturing issues and were negotiating their resolution with the FDA.

This was exactly the same statement he made at JP Morgan the year before that.

But despite the slowdown in commercialization and industrys challenges, Davies said there remains a lot to be positive about.

Everything that I said reflects the extreme difficulty in bringing this class of therapeutics to market. If these therapeutics were easy to develop,p they would have been developed. If diseases were easy to cure, we wouldnt need new therapeutics.

Let us just use these good pieces of news and these bad pieces of news as inspiration, lets continually remind ourselves that what we do is one of the hardest things in science or medicine at this time.

Davies was not alone in his views.

Speaking Wednesday, Robert Preti, CEO of Hitachi Chem Advanced Therapeutic Solutions, admitted the industry is behind where he thought it would be when he began his career 37 years ago, but said he was not too worried.

I want to commend this industry on what we have achieved for patients, he said, noting the difficulty in developing and making these therapies. He also highlighted that with over 1,000 regenerative therapies in development, problems will eventually be ironed out and cell and gene therapies will make the widespread impact intended.

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FDA cell and gene therapy forecast 'unlikely' - Bioprocess Insider - BioProcess Insider

MIAMI--(BUSINESS WIRE)--Anemocyte, an innovative Italian company working in the field of cell and gene therapies, with special focus on plasmid production and non-viral gene modification approaches, attends Phacilitate Leaders World (Miami, 21-24 January 2020), a leading event for companies, professionals and investors working in the Advanced Therapy sector.Phacilitate Leaders World is an opportunity to take stock of this sector, which is in constant evolution, states Marco Ferrari, CEO of Anemocyte. We need to anticipate market demands and offer innovative, effective solutions and strategies".Anemocyte is a key player and the first ever Biotech Manufacturing Organization (BMO) operating in the Life Science sector: Anemocyte helps CGT developers to articulate initial ideas, perform clinical trials and engage in commercial production. The BMO also develops technological platform strategies for innovative R&D, HQ and GMP processes.

PlasmidsAnemocyte is the leading Italian producer of gene therapy plasmids. The brand-new Plasmid Manufacturing Unit dedicated to producing plasmids for viral vectors is equipped with state-of-the-art laboratories and facilities. The company improved the production capacity (tripled the output since 2018): "In a market where production wait times are extremely long, Anemocyte is able to respond to requests quickly and with the highest quality standards, states Stefano Baila, Director of Operations and Business Development at Anemocyte. Anemocyte is a dynamic market player equipped with cutting-edge technologies and over ten years of expertise producing recombinant proteins; experience that it now exploits to produce plasmids our core business. We are able to intercept market needs and offer punctual, effective and qualitatively competitive results.

Non-viral: excellence in genetic modificationFollowing a strategic agreement with MaxCyte. Inc., Anemocyte is now a centre of excellence for electroporation technology and can support non-viral gene modification projects requiring both process development and GMP production.The ability to modify cells genetically has exponentially increased the clinical use of cell-based medicines. Non-viral gene modification is an emerging trend in the delivery of nucleic acids to cells thanks to its reliability, safety and affordability. Electroporation technology is at the forefront of non-viral gene transfer for ex-vivo cell modifications.The construction of a global non-viral gene transfer hub will see product developers exploit Anemocytes experience and MaxCytes technology.

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Anemocyte Brings Italian Excellence in the Cell and Gene Therapy Sector to Phacilitate Leaders World 2020 in Miami - Business Wire

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 its currently done.

What we saw differently here is not just the obvious that theres 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 theyre gonna have a place where they can call home.

Just how big is the demand? Days ago John Chiminski, whos 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. Thats up from 300,000 liters now.

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 Chacn 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 theyve announced the project.

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

Its 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; were 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, thats just thats 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, whats 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 theres 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.

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

REDWOOD CITY, Calif., Jan. 28, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the presentation of 24-week data from the second cohort of patients (n=6; 2 x 1011 vg/eye) as well as an update from the first cohort of patients (n=6; 6 x 1011 vg/eye) in the OPTIC Phase1 clinical trial of ADVM-022 intravitreal gene therapy in wet AMD at the Angiogenesis, Exudation, and Degeneration 2020 Meeting.

Adverum plans to issue a press release relating to the presentation and post the presentation on Adverums website at http://www.adverum.com in the Investors section under the Events and Presentations page at the beginning of the data presentation at Angiogenesis.

KOL Event Details:In addition, Adverum will host an event with expert retinal specialists to discuss the OPTIC data presented at Angiogenesis and the potential opportunity for ADVM-022. The discussion will be held on Sunday, February 9, 2020 beginning at 10:00 am EST. The event will be webcast live from Adverums website at http://www.adverum.com in the Investors section under the Events and Presentations page. A replay of the webcast will be archived and available for replay following the event.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration. For more information, please visit http://www.adverum.com.

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Adverum Biotechnologies to Present Additional Data from the OPTIC Phase 1 Trial with ADVM-022 Intravitreal Gene Therapy in Wet AMD at the...

The FDA has published their industry guidance on the topic of long term follow up (LTFU) post gene therapy. Please see a link to the full guidance below. In summary, they are stipulating that a good proportion of these therapies will require LTFU of up to fifteen years.

This presents significant challenge to manufacturers given that patients will, in that stretch of time, have multiple changes to their care settings and life circumstances. In addition to this, and if these therapies live up to their promise, treated patients will potentially become chronically well and keen to disengage from their previous disease state and their healthcare professionals.

This is a significant challenge but achievable! We have already shared our experienced thinking on this topic and a link to our article is provided below. Our recommendations focus on putting the patient at the centre of a solution and wrapping a, digitally enabled, registry solution around thoughtful patient and healthcare professional engagement.

Please read our full thinking below:

http://edition.pagesuite-professional.co.uk/html5/reader/production/default.aspx?pubname=&edid=5cf08b2f-f3d9-435a-8255-ef07542eb9a3&pnum=31

https://www.fda.gov/media/113768/download

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OPEN Health's response to the FDA's guidance on long term follow up post gene therapy - PMLiVE

Krystal Biotech and Foghorn Therapeutics are beginning 2020 with significant expansion projects.

Pittsburgh-based Krystal, a gene therapy company developing medicines to treat rare diseases, broke ground this week on its second commercial gene therapy facility in Findlay Township, Penn. The new 100,000 square-foot facility, called ASTRA, will have the capacity to produce commercial gene therapy medicines to treat patients suffering from debilitating rare diseases. The facility is being designed as a state-of-the-art cGMP manufacturing facility that will allow the in-house incorporation of raw material preparation, excipient manufacturing, testing, packaging, labeling and distribution, fully-integrating all components of the supply chain from starting materials to patient experience, the company said in an announcement.

Krystal said the ASTRA facility will initially be used as a commercial back up facility for B-VEC, previously known as KB103, which is being developed for the treatment of dystrophic epidermolysis bullosa, a rare and devastating skin disorder, and expand to produce investigational and commercial material for our pipeline products. When the site is fully operational, it will have the potential to create around 200 new jobs when at full capacity.

Krish S. Krishnan, chairman and chief executive officer atKrystal, said biologics manufacturing is a complex science and is a competitive advantage forKrystal Biotech.

We are very excited to announce the creation of ASTRA which will be a global resource for production of gene therapies with the potential to bring new treatments to rare disease patients around the world. The success with our first GMP facility, Ancoris, gives us the experience and confidence to have ASTRA be functionally ready in time for the anticipated launch of our lead therapeutic, B-VEC, Krishnan said in a statement.

In Massachusetts, Foghorn Therapeutics, launched by Flagship Pioneering in 2018, is undertaking an expansion of its headquarters, nearly tripling the space. In a brief report, the Boston Business Journal said the Foghorn headquarters will include biology labs, chemistry labs, open-space offices, huddle rooms, conference rooms and an employee cafe. The additional space provides the company with significant room for growth, but Foghorn has no specific timeline for that growth, a company spokesperson told the Journal.

"There is no specific timeframe for filling the new office to capacity but Foghorn has grown rapidly since its inception and we are regularly adding talented individuals to help us achieve our mission, the spokesperson said, according to the Journal.

Foghorns goal is to develop drugs that work in the chromatin regulatory system, which orchestrates the movement of molecules that turn genes on and off, the company said. Using its Gene Traffic Control platform, Foghorn is focusing on sarcoma, as well as prostate cancer and non-small cell lung cancer. The chromatin process has possible applications in autism, schizophrenia and some rare neurological diseases. The company is currently pre-clinical but said it is rapidly advancing more than 10 programs.

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Krystal, Foghorn Undertake Expansions that Will Lead to New Hires - BioSpace