NewsEnvironmentResearchers at Queen Mary University of London and the Royal Botanic Gardens, Kew, discovered genes that create chemicals harmful to insects

Monday, 25th May 2020, 11:22 pm

A set of genes has been identified that could protect ash trees from a deadly beetle, which attacks by burrowing into their stems.

Named the Emerald Ash Borer (EAB), the killer pest is expected to destroy hundreds of millions of trees worldwide in the years to come without any intervention.

Researchers at Queen Mary University of London and the Royal Botanic Gardens, Kew, discovered genes that create chemicals likely to be harmful to the insects.

They sequenced the genomes of 22 types of ash tree and used this information to analyse how the different species are related to each other.

Help trees fight deadly beetles

Meanwhile, the US Department of Agriculture Forest Service, in Ohio, tested the resistance of more than 20 ash species to EAB by hatching eggs on the bark of trees, and following the fate of the beetle larvae.

Resistant ash trees killed the larvae when they burrowed into their stems, but susceptible ones did not.

The scientists discovered 53 candidate resistance genes, several of which are involved in making chemicals that are likely to be harmful to insects.

The findings suggest that breeding or gene editing could be used to place these resistance genes into ash species currently affected by EAB.

Dr Laura Kelly, an academic visitor at Queen Mary and lead author of the study, published in the journal Nature Ecology & Evolution, said: Knowledge of genes involved in resistance will...help efforts to identify trees that are able to survive the ongoing threat from EAB, and in turn, could facilitate restoration of ash woodlands in areas which have already been invaded."

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Gene therapy could help millions of ash trees fight deadly beetle Emerald Ash Borer - inews

When a field that depends on scientific benchwork suddenly moves online, what happens to collaborations and the innovation supply chain? We are seeing the answer to this question play out in real time during the Covid-19 crisis. Across the United States and abroad, measures to safeguard against this serious disease have created an unplanned, massive test for remote work in biotechnology.

Scientific companies face a unique challenge during the shutdown: continuing their research. Lab-based work requires in-person, human attention, as many experiments are complex and require specialized expertise. These projects are often achieved through research collaborations between organizations, requiring meetings to share data, shared work environments, and deep discussions. And their results are priceless these collaborations have the potential to generate therapies that save or improve millions of lives in the future. This is why biotechnology companies have been deemed an essential industry and continue to operate in some form during the shutdown.

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European regulators accept FibroGen's anemia drug for review; Passage Bio's lead gene therapy gets more love from the FDA - Endpoints News

NEWARK, Delaware, May 19, 2020 /PRNewswire/ --QPS, a global contract research organization (CRO) that provides discovery, preclinical, and clinical drug development services, is reinforcing its focus on qualitative and quantitative bioanalysis of biotherapeutics. QPS announces an expanded and upgraded fleet of high-resolution mass spectrometers (HRMS), with the addition of three new TripleTOF HRMS systems for GLP quantitation, two in the Newark, Delaware facility and one in the Suzhou, China laboratory. As part of this expansion, QPS has hired Larry Mallis, Ph.D., Director of Bioanalysis, to lead the newly merged biotherapeutics and biomarkers Liquid ChromatographyMass Spectrometry (LC-MS) quantitation team in Delaware.

The QPS facility in Newark, Delaware, now has four high-resolution mass spectrometers: two 6600+, one 6600, and one 5600, all of which are being used for Good Laboratory Practice (GLP) quantitation and metabolite identification.

"QPS has been closely watching the trends in the market and we are committed to responding to the needs of our clients by dedicating four of the five TripleTOF Ultra-Performance Liquid ChromatographyHigh-Resolution Mass Spectrometry (UPLC-HRMS) systems to quantitation of oligonucleotides and intact proteins," said John Kolman, VP, Global Head of Translational Medicine, QPS LLC.

"This increase in capacity and capability comes at a pivotal moment for QPS in China, as we continue our two-decades-long global effort to support the pharma industry in developing antiviral therapeutics and/or vaccines. This has become a higher priority in view of the new public health concerns due to the novel coronavirus," said Yondong Zhu, VP, Head of Bioanalytical Services, QPS China.

Larry Mallis, Ph.D., leader of the newly formed team in Newark, Delaware, built his career in the pharmaceutical industry (BMS, Wyeth, and Merck), before moving into the CRO industry, most recently as Director of Bioanalytical Operations at Lovelace Biomedical Research Institute. This new group now has all the necessary LC-MS and other chromatographic technology for PK/PD bioanalysis to support clients in drug discovery and development of rare diseases. This group's expertise lies in the quantitation of oligonucleotides, peptides, intact proteins, and highly hydrophilic low-molecular-weight metabolite biomarkers by UPLC-HRMS, or by immunoaffinity UPLC-MS/MS (tandem mass spectrometry), or by hybridization-LC-fluorescence.

ABOUT QPS HOLDINGS, LLC

Since 1995, QPS has provided discovery, preclinical, and clinical drug development services. An award-winning leader focused on bioanalytics and clinical trials, QPS is known for proven quality standards, technical expertise, a flexible approach to research, client satisfaction, and turnkey laboratories and facilities. QPS has CLIA-certified and GLP-compliant laboratories ready to fast-track your novel coronavirus and COVID-19 RT-qPCR/QPCR and Serological Assays and vaccine development programs. For more information, visitwww.qps.comor email[emailprotected].

QPS CONTACT:

Name: Gabrielle Pastore Phone: 1-302-635-4290 Email: [emailprotected]

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QPS Continues to Expand UPLC-HRMS Quantitation Capabilities to Support Gene Therapy and Protein Drug Development - PRNewswire

FILE PHOTO: The logo of Swiss drugmaker Novartis is pictured at the French company's headquarters in Rueil-Malmaison near Paris, France, April 22, 2020. REUTERS/Charles Platiau

ZURICH (Reuters) - Novartis won European approval for its gene therapy Zolgensma for the hereditary disease spinal muscular atrophy (SMA), the Swiss drugmaker said on Tuesday, adding it is in talks over price with countries in hopes of a quick launch.

The European Commission gave conditional approval to the therapy, whose U.S. price is $2.1 million, for patients with a clinical diagnosis of SMA Type 1, the most severe form of the disease, or SMA patients with up to three copies of a specific gene that helps doctors predict how severe the disease will be.

The EU approval covers babies and young children with SMA up to 21 kilograms. The medicine also has approval in Japan.

Novartis got Zolgensma with its $8.7 billion takeover of U.S.-based AveXis in 2018 and has forecast more than $1 billion in sales for the treatment, which in trials has been shown to significantly improve survival and motor function of babies with SMA, in particular those treated before symptoms develop.

Novartis said it is in talks with nations over what it calls its Day One access program, which the Basel-based drugmaker said is aimed at speeding up treatment by dealing with payment issues up front, even before national pricing and reimbursement agreements with individual countries are in place.

The Day One access program ensures the cost of patients treated before national pricing and reimbursement agreements are in place align with the value-based prices negotiated following clinical and economic assessments, Novartis said, adding the medicine will be immediately available in France.

Drug pricing in Europe varies from country to country, often relying on individual negotiations with regulators and pricing watchdogs that can slow down access, including in instances where officials conclude companies are seeking too much money for their medicines relative to the value they bring.

Reporting by John Miller; editing by Thomas Seythal and Brenna Hughes Neghaiwi

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Novartis wins conditional EU approval for gene therapy Zolgensma - Reuters

By 2027, the global gene therapy market is estimated to reach a staggering value of $6.6B. With the number of successfully approved gene therapies increasing, the sector has moved from hype to hope. At the heart of gene therapy lie viral vectors, which are used to transport a gene into a target cell. Here we explore the current bottleneck in viral vector production, why viral vectors still outshine non-viral vector solutions, and what we can expect in the future.

The gene therapy field is gaining momentum. Investments are pouring in. The FDA is estimating that by 2025 it will approve between 10 and 20 cell and gene therapy products every year. This shows that a treatment, which started out as a hype, is now a real hope, says Ratish Krishnan, Associate Director of Cell and Gene Therapy Bioprocessing at MilliporeSigma*.

Today, a number of treatments have been approved, such as Spark Therapeutics LUXTURNA, the first FDA approved in vivo gene therapy, or Novartis Zolgensma, which gained US approval in 2019 and European approval in March 2020.

One of the key ingredients of gene therapies is the viral vector, which is used to transfer a gene of interest into a target cell. The most commonly used vector is that of the adeno-associated virus (AAV). But the manufacturing of viral vectors and scaling up their production remain difficult.

In upstream development, one challenge is the way viral vectors are produced. In a process called transient transfection, plasmids carrying the DNA of interest are introduced into host cells that will produce the viral vectors.

But host cells are commonly grown in adherent cell cultures, which are usually harder to reproduce at a large scale. So to scale-up and achieve high titers of virus particles, researchers are working on growing cells in suspension using large bioreactors instead.

We are facing several challenges at the moment and that is what keeps us on our toes, Krishnan adds. In upstream development, there is a desire to move towards suspension. Most processes use transient transfection methods using plasmids and the transfection step at a production scale of 500L or 2000L is extremely challenging.

In downstream development, researchers are studying the purity of capsids. The capsid is the protective shell of the virus enclosing the gene of interest. Related to this is an important discussion on the purity of viral vectors.

When you produce vectors, you will generally have a population of empty capsids, which is the viral AAV assembly without the genetic material inside, or partially-full capsids with only part of the DNA inside, Krishnan explains. We have to better understand the role of empty capsids inside the body. Are they needed as immune decoys or are they strictly considered impurities? In theory, you only need the ones with all the DNA inside, the full capsids.

But researchers have yet to discover the correct percentage of full capsids in a drug substance or product, Krishnan says. We dont have the answer yet. Typically, the strategy leans towards the enrichment of a percentage of full capsids as high as possible, while taking into account the data from clinical trials.

Questions about purity are difficult to answer because there is little or no regulatory guidance. For instance, compared to monoclonal antibodies (mAbs) for which the regulatory environment is well understood, the regulatory landscape for gene therapies remains largely unclear.

Through decades of research, mAb production also works through well-established and standardized platforms, whereas viral vectors are still in their infancy and cannot be produced using platform technologies yet although much is being done in this field and they are catching up fast.

Because of their longstanding history, we already have a lot of knowledge and research about mAbs, Krishnan explains. Lets say I want to start a biotech company or a CDMO that develops mAbs. I can rely on existing templates and get up and running much quicker than if I were developing viral vectors.

AAVs come in many different serotypes distinguishable strains which impact platform development. The scale-up is also challenging since the indications can be strikingly contrasting. For example, ocular indications need smaller viral drug substances compared to a muscular indication.

Manufacturing mAbs is better understood than manufacturing viral vectors. Viruses are a whole lot bigger and can be more complex than antibodies. While antibodies usually have a size around 10 nm, AAVs measure around 20nm and Lentiviruses around 100nm in size. Not only are antibodies produced at a larger scale than viral vectors, but their scalability is also more predictable due to the established platform technologies.

Despite these challenges, the increase in popularity, investments flooding in, and the promise of essential cures have led to a bottleneck in viral vector production for gene therapies. But many companies working in the gene therapy field are small or emerging biotechs that do not have the necessary resources and expertise in-house to tackle the challenges of viral vector production.

Lacking the facilities to do it themselves, small and emerging biotechs therefore turn to experienced contract development and manufacturing organizations (CDMOs), such as Merck BioReliance, to produce their gene therapies.

There are only a handful of CDMOs that have the capability and expertise to take on the complexities of gene therapy projects, Krishnan says. But there is currently a bottleneck in manufacturing slots. Manufacturing facilities can only work 24 hours a day, and if you are a small company and a CDMO has other clients waiting in line before you to have their therapies manufactured, you have no other option but to wait.

The problem with waiting, of course, is that the biotech runs the risk of falling behind its competition. Krishnan emphasizes, time is of the essence in gene therapy. There is no silver medal for developing a therapy for the same indication.

The key, says Krishnan, lies in much planning and close engagement with the CDMO partner. Biotechs should do an analysis of what they can perform in-house versus what they have to outsource early on. Engaging a CDMO is the route you want to take.

With decades of experience, Merck can support its biotech sponsors all the way to the clinic. We are big on the concept of integrated solutions, Krishnan explains. From clone to clinic to commercialization, Merck has the expertise, knowledge network, product, and services to help guide any customer to the finish line. We have the CDMO expertise with BioReliance, with testing services, gene therapy expertise, and regulatory support.

To circumvent the manufacturing bottleneck for viral vectors, some biotechs are looking at non-viral vector solutions for gene therapies. While traditional gene therapies use a viral vector, like AAV, to transfer a gene of interest into the patient, non-viral gene therapy deploys an alternative delivery system for the gene of interest.

Examples for non-viral delivery systems include physical force to deliver the gene through the cell membrane; injecting the gene with a needle into the target region; electroporation, which uses an electric current to produce pores in the cell membrane through which the gene can be inserted into the cell; and chemical vectors, such as lipid-, polymer-, or peptide-based particles.

Nevertheless, viral vectors, such as the AAV, remain the preferred path for most companies. The efficiency of delivery for non-viral vectors remains questionable. This means that there might be reactions in the immune system that get triggered, eliciting a dangerous, adverse response. AAVs, on the other hand, are well-engineered and safe, despite being novel.

Viral vectors have recently demonstrated success, Krishnan adds. Scientists are making advances in the non-viral area of gene therapy but they also come with a unique set of challenges. Questions, such as how do they interact with serum components in the body, how do they involve the immune system before reaching the target tissue, how do they interact with the surfaces of cells, remain.

Despite unaddressed challenges, gene therapy has definitely shifted from being a hope to carrying an expectation. This is reflected in the number of investments pouring into the sector.

Big pharma and biotech companies are heavily investing their resources into gene therapy, Krishnan says. Typically, companies have vaccines or mAbs in their portfolio. Now, gene therapy is becoming a major modality of interest as well.

While we are still far away from reaching the smooth manufacturing processes we have in place for antibodies, many companies are also looking into platform approaches for gene therapy. We would take a quantum leap if we developed a platform approach for upstream and downstream processes. That would significantly reduce the time to the clinic. Platform approaches are definitely being explored, Krishnan adds.

Vendors like Merck are playing a big role in developing fit-for-purpose products for gene therapies, Krishnan says. At the R&D level, researchers are also working on advances in capsid engineering. We already have synthetic capsids, and there are other tremendous advancements in capsid engineering, design, and purity, which are going to continue to evolve.

But, as Krishnan puts it, We are running a marathon at sprint speed. The journey is exciting and challenging, identical to the ride on a rollercoaster, but we are barely even at the tip of the iceberg. There are patients waiting for life-saving treatment, so gene therapies will definitely continue to be in the limelight, and for good reason.

Are you fighting to solve the bottleneck in viral vector production? Get in touch with the expert team at Merck and view their webinar on this topic!

*The life science business of Merck operates as MilliporeSigma in the U.S. and Canada.

Images via Shutterstock.com and Elena Resko

Originally posted here:

The Roller Coaster of Gene Therapy... - Labiotech.eu

18 May 2020

A novel gene therapy prevents obesity and builds muscle without the need for additional exercise or dieting.

Researchers at Washington University School of Medicine in St Louis discovered that a novel gene therapy in mice can build strength and muscle mass quickly, resulting in a reduction in the symptoms of osteoarthritis. In addition, the same gene therapy also prevented obesity even when the mice were fed a high-fat diet and not exercising any more than usual.

'We've identified here a way to use gene therapy to build muscle quickly,' said senior investigator Professor Farshid Guilak, who is also director of research at Shriners Hospitals for Children in St. Louis. 'It had a profound effect in the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity.'

The team gave eight-week-old mice a single injection of the gene that makes follistatin,a protein that controls metabolism, enables muscle growth and boosts fertility. Follistatin normally blocks another protein called myostatin, which regulates muscle growth, so by using this therapy, mice were able to build significant muscle mass without gaining additional weight. Specifically, mice more than doubled their muscle mass whilst also nearly doubling their strength.

With obesity being the most common risk factor for osteoarthritis, this new research aimed to investigate whether this therapy could help treat osteoarthritis by increasing muscle mass and reducing the metabolic inflammation linked to obesity.

Despite being fed a high-fat diet, the researchers found that the treated mice had fewer metabolic problems, stronger hearts, and healthier joints with less cartilage damage and inflammatory markers than their untreated counterparts. In addition, these mice were less sensitive to pain.

However, the researchers noted that they are still far off from a human clinical treatment and longer-term studies will be needed to determine the gene therapy's safety. For example, some of the muscle growth caused by gene therapy can often be harmful, such as the thickening of the heart's wall. Still, they believe that follistatin gene therapy could be a promising approach to treating degenerative muscle diseases, including muscular dystrophy.

'Something like this could take years to develop, but we're excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity,' said Professor Guilak.

The study was published in the journal Science Advances.

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Gene therapy reduces fat and builds muscle in mice - BioNews

Trying to hack fitness is a multi-million-dollar industry; weve all seen at least one ad featuring a purported miracle product that claims it can make people lose weight and look greatwithout even trying. From low-effort exercise machines to strange-ingredient diets to fat-burning belts and bands, theres no shortage of attempts to make it easy to be fit.

A gene therapy trial performed on mice may foreshadow yet another way to hack fitness. In a study done by a team at Washington University in St. Louis medical school, mice quickly built muscle mass and reduced obesity after receiving the therapy, even while eating a diet high in fat and not exercising. The results were published last week in a paper in Science Advances.

Sound appealing? Heres how it worked.

The gene targeted was FST, which is responsible for making a protein called follistatin. In humans and most other mammals, follistatin helps grow muscle and control metabolism by blocking a protein called myostatin, which acts to restrain muscle growth and ensure muscles dont get too big.

The researchers injected eight-week-old mice with a virus carrying a healthy FST gene (gene therapy involves adding healthy copies of a gene to cells, usually using a virus as a deliveryman).

Over a period of 18 weeks, or about 4 months, the team observed that the muscle mass of the treated mice more than doubled, as did their strength level. They also experienced reduced damage related to osteoarthritis, less inflammation in their joints, and had healthier hearts and blood vessels than mice that didnt receive the gene therapyeven though all the mice ate the same high-fat diet and did the same amount of exercise.

Going into the study, the researchers worried the muscle growth catalyzed by the gene therapy could harm the heart, mainly through thickening of the hearts walls. Surprisingly, though, heart function and cardiovascular health of the treated mice actually improved. In subsequent studies, the team will continue to monitor the treatments effect on the heart, as complications could emerge over time.

Talk about a fitness hack; imagine being able to build muscle and maintain a healthy metabolism while lounging on the couch eating burgers and fries. There have been similar studies to replicate the effects of exercise by commandeering the genetic instructions that control the way cells interact with proteins; though various exercise pills have successfully mimicked the effects of regular cardiovascular activity in mice, scientists still dont fully understand how, at a molecular level, exercise has the effects it does on the human body.

This may change in the next couple years, though; a National Institutes of Health consortium called the Molecular Transducers of Physical Activity is in the midst of an in-depth study on the molecular effects of exercise on tissues and organs in 3,000 people.

If the muscle-building gene therapy eventually reaches a point where it can be used in humans, though, the research team isnt viewing it as a quick-fix health hack. Rather, it would be used to help get people with conditions like muscular dystrophy or severe obesity to a baseline from which they could adopt tried-and-true muscle-building practices like weight lifting or physical therapy.

In cases of severe obesity or muscle loss, it is extremely difficultif not impossibleto lose weight or improve muscle strength through normal exercise and diet, said Farshid Guilak, orthopedic surgery professor and director of research at Shriners Hospitals for Children in St. Louis. The goal of this study was to show the importance of muscle strength in overriding many of the harmful effects of obesity on the joint.

If every condition, process, and trait in our bodies is tightly linked to our genes, its conceivable that almost any aspect of our health could be manipulated using gene therapy and related tools. Maybe one day there will indeed be a pill we can take or a shot we can get to give us svelte, muscular bodies without any of the effort.

The fact that this would ruin the pleasure and satisfaction of a good workout is another conversationand one not everybody would be interested in having. But even if genetic or chemical exercise-replacement tools become safe to use in humans in the foreseeable future, theyll likely be limited, at least at first, to those who need them due to debilitating health conditions.

That saidfor the time being, keep hitting the treadmill, the weight room, or your other off-the-couch, effort-intensive workout of choice.

Image Credit: Aberro Creative from Pixabay

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Forget ExerciseThese Mice Got Ripped With Gene Therapy - Singularity Hub

After beefing up its pipeline last year with an in-licensing deal from Novartis, San Diego-based Amplyx has lined up an extension of their C round, designed to pay their way through a slate of mid-stage trials putting their experimental drugs to the test in humans. And this time theyre getting some bucks from Pfizer to pay for the next leg in the development process.

The new money a $53 million add to the first tranche in 2017 brings their C round total to $90 million-plus, with Pfizer joining a syndicate that includes Sofinnova Venture Partners, with participation from existing investors including New Enterprise Associates, Lundbeckfonden Ventures, Arix Bioscience, Pappas Capital, RiverVest Venture Partners, 35 Partners and BioMed Ventures. Adage Capital was another new investor in the round, with Pfizer, which brings the biotechs total raise to date to $140 million.

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UPDATED: Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial but another safety setback mars results - Endpoints News

Dive Brief:

Like other CDMOs, Thermo sees a massive opportunity in the field of gene therapy.

Thermo paid $1.7 billion last year to buy viral vector contract manufacturer Brammer Bio. The company later opened a $90 million manufacturing facility in Lexington, Massachusetts, and it's expanded capacity at sites in Cambridge, Massachusetts, and Alachua, Florida, as well.

Thermo's new space in Massachusetts will rival a 300,000-square-foot plant operated by Swiss drug manufacturer Lonza in Pearland, Texas, outside of Houston. Lonza called the plant the world's largest dedicated to cell and gene therapy production when it opened in 2018.

Lonza has also been steadily making deals in an effort to support biotech and pharma customers through every step of the process in gene therapy. As of April 2019, the company had worked with more than 45 viral vector customers and expected that number to keep climbing.

Other companies are expanding, too. Fujifilm in November announced plans to spend about $120 million in the gene therapy field, including a new innovation center in Texas. Catalent last year paid $1.2 billion for Paragon Bioservices to bolster its manufacturing capacity for gene therapies.

Thermo said its new plant will take advantage of the latest technology, with digital connectivity and advanced operator training. The company said it chose to construct the site in Plainville to take advantage of nearby Thermo facilities and draw on the Boston-area talent pool

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Thermo Fisher to invest $180M in new gene therapy plant - BioPharma Dive

Shares of a Cambridge, Massachusetts-based biotechnology company that is taking a different approach to gene editing have gained more than 25% since its early February initial public offering.

Beam Therapeutics Inc.'s (NASDAQ:BEAM) stock traded near $32 just days after it went public at an offering price of $17, but has since receded to $21.49. At its current price, many think the company is a bargain.

Among the optimists are the analysts at several firms that underwrote the offering, reported 24/7 Wall Street. Of course, it's little surprise that they have a buy or outperform rating for the stock.

Jefferies started coverage with a buy rating and assigned a $32 target price, as did Barclays, who gave the stock at overweight rating. Meanwhile, JPMorgan also gave Beam as an overweight rating with a $31 target price. In his report, JPMorgan's Eric Joseph noted the company is focused on multiple targets "with a first-to-market or a best-in-class potential in rare disease and oncology indications."

According to the Encyclopedia Britannica, gene editing is the ability to make highly specific changes in the DNA sequence of a living organism, essentially customizing its genetic makeup. The technology uses enzymes to target a specific DNA sequence. Typically, cuts are made into the DNA strands and existing DNA is removed and replaced.

Beam is using a different method. The company hopes to use a chemical reaction to convert one of the four building blocks of a gene into a new, functioning one. This novel approach is one reason David Nierengarten of Wedbush Securities cited for rating the firm an outperform with a $32 target price target.

Beam is working to apply its platform to treat a number of diseases, including acute myeloid leukemia, sickle cell disease and vision loss caused by a form of macular degeneration. The company is collaborating with privately held Verve Therapeutics on a treatment for coronary artery disease.

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More than half of the genetic errors associated with disease result from a single-letter change in the bases that form the human genome, according to an article in FierceBiotech. Given that there are 33,000 point mutations identified as being tied to genetic diseases, the potential application of Beam's technology is massive.

Beam is one of several companies co-founded by gene-editing scientists David Liu and Feng Zhang, as well as Harvard Medical School professor J. Keith Joung.

Another of their gene editing ventures, Editas Medicine Inc. (NASDAQ:EDIT), went public in 2016 at $16 a share and now trades at more than $26.

At the end of last year, Beam had cash, cash equivalents and marketable securities of nearly $92 million, which does not include the more than $188 million raised from the IPO. The company had a net loss of more than $78 million in 2019.

Disclosure: The author holds no positions in any of the companies mentioned in this article.

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Beam Therapeutics Taking Novel Approach to Gene Therapy - Yahoo Finance

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Cutting-edge research on rewriting the genes responsible for Tay-Sachs disease, sickle-cell anemia, and other disorders will be presented at this weeks online annual meeting of the American Society for Gene and Cell Therapy. Originally planned as a Boston gathering, the scientific groups get-together became a virtual one because of the Covid-19 pandemic.

From Tuesday through Friday, academic researchers will be presenting their latest data online, along with updates from firms like Bluebird Bio (ticker: BLUE), Voyager Therapeutics (VYGR), Fate Therapeutics (FATE), Beam Therapeutics (BEAM), Axovant Gene Therapies (AXGT), and many others. Patients and their families have found their way to clinical trials through the societys website.

Bluebird plans presentations on its cell therapy against the blood cancer known as multiple myeloma. Using a technology known as CAR-T, the company creates supercharged versions of a patients immune cells that have halted disease progression in some of the 18 patients enrolled in a continuing Phase 1 trial.

Featured on Friday will be reports on the first babies treated with gene therapy for the debilitating neurodegenerative disorder Tay-Sachs. The treatment is being developed by the London-based Axovant under license from the University of Massachusetts Medical School.

Voyager will discuss its preclinical mouse studies on treating neurological disorders like amyotrophic lateral sclerosis and Huntingtons disease by using techniques that block the rogue signals generated by defective genes.

Fate Therapuetics is scheduled to show a new off-the-shelf CAR-T technology that it hopes will allow the immune system to target a broad range of solid tumors as well as multiple myeloma. The approach is licensed from Harvard Universitys Dana-Farber Cancer Institute.

Beam, meanwhile, will detail success it has shown in preclinical editing of the genetic defect that causes sickle-cell anemia. The company is developing a sharper-edged way of rewriting faulty genes than the widely used Crispr technology that Beam licensed from researchers at the Broad institute of Harvard and MIT. Beam founder and Crispr pioneer Feng Zhang will give a featured lecture as part of the online meeting on Thursday.

Write to Bill Alpert at william.alpert@barrons.com

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Gene-Therapy Treatments for Tay-Sachs, Sickle Cell to Be Featured in Online Gathering - Barron's

Sarepta Therapeutics Inc. andDyno Therapeutics Inc., have announced an agreement to develop next-generation Adeno-Associated Virus (AAV) vectors for muscle diseases, using Dynos CapsidMap platform. Dynos proprietary CapsidMap platform opens up new ways to identify novel capsids the cell-targeting protein shell of viral vectors that could offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing.Our agreement with Dyno provides us with another valuable tool to develop next-generation capsids for gene therapies to treat rare diseases, saidDoug Ingram, Sareptas president and CEO. By leveraging Dynos AI platform and Sareptas deep expertise in gene therapy development, our goal is to advance next-generation treatments with improved muscle-targeting capabilities.Under the terms of the agreement, Dyno will be responsible for the design and discovery of novel AAV capsids with improved functional properties for gene therapy and Sarepta will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids. If successful, Dyno could receive over$40 millionin upfront, option and license payments during the research phase of the collaboration. Additionally, if Sarepta develops and commercializes multiple candidates for multiple muscle diseases, Dyno will be eligible for additional significant future milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the collaboration.This agreement is a major step forward in our plan to realize the potential of Dynos AI platform for gene therapies to improve patient health. We are excited to work with Sarepta to create gene therapies with improved properties to address a range of muscle-related diseases, stated Dynos CEO and co-founderEric D. Kelsic, Ph.D. The success of the gene therapies developed through this collaboration with Sarepta will rely on AI-powered vectors that allow gene therapies to be safely and precisely targeted to the muscle tissue.

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV)vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies DNA library synthesis and next-generation DNA sequencing to measureinvivogene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

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Sarepta And Dyno Therapeutics Will Develop Next-Gen Gene Therapy Vectors - Contract Pharma

Researchers at Washington University School of Medicine in St. Louis have demonstrated a new gene therapy that builds strength and muscle mass quickly while preventing obesity even when the recipient is eating a high-fat diet. This isnt some crazy new bodybuilding tool, though, but rather something that could be used as a physical therapy aid for people with muscle-weakening arthritis or those suffering from osteoarthritis-related joint pain.

We know that obesity and joint injury are the primary risk factors for osteoarthritis, Farshid Guilak, professor of orthopedic surgery and director of research at Shriners Hospitals for Children in St. Louis, told Digital Trends. However, in cases of severe obesity or muscle loss, it is extremely difficult if not impossible to lose weight or improve muscle strength through normal exercise and diet. The goal of this study was to show the importance of muscle strength in overriding many of the harmful effects of obesity on the joint.

So far, the gene therapy approach has only been demonstrated in mice. The researchers delivered the gene for a molecule called follistatin to the muscle of eight-week-old mice, via injection. The protein blocks myostatin, a molecule normally responsible for stopping muscle growth. The mice in the experiment gained around twice their normal muscle mass and were able to completely burn off all the extra energy from an unhealthy high-fat diet. This prevented almost all the metabolic complications of obesity, such as systemic inflammation and high blood sugar, while also reducing arthritis and pain significantly.

The researchers worried that the gene therapy could potentially have a negative effect on the mices hearts. However, these fears proved unfounded and heart function in the mice actually improved, along with overall cardiovascular health.

These first studies in mice have shown that the procedure has excellent efficacy and safety, Guilak said. The next steps will be to do longer-term studies in mice and possibly larger animals to ensure safety of this procedure.

Guilak said that, should these steps prove successful, the researchers would consider testing the approach in humans with the initial trials in those with severe, possibly life-threatening diseases of the muscles, such as muscular dystrophy.

A paper describing the work was recently published in the journal Science Advances.

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New gene therapy cuts fat and builds muscle with ease. But theres a catch - Digital Trends

Everybody wants to be fit. But not everybody wants to put in the effort to get there.

Who has time to spend long hours at the gym and eating right is such a bore. What if we told you that you could get ripped without exercising and eating whatever you wanted.

RELATED:EXERCISE IS THE BEST WAY TO KEEP THE POUNDS OFF, SAYS NEW STUDY

This is whata team at Washington University in St. Louis medical school achieved with mice in test trials. They created a gene therapy that when given to mice allowed them to build muscle mass and reduce obesity even while eating a diet high in fat and not exercising.

How did it work? The therapytargeted a gene called FST, which makes a protein called follistatin. Follistatin blocks a protein called myostatin, which stops muscle growth to ensure muscles dont get too large.

The researchers injected a virus carrying a healthy FST geneintoeight-week-old mice. They then observed the mice over a period of 18 weeks.

What they found was nothing short of impressive. The mice's muscle mass and strength more than doubled and they experienced reduced damage related to osteoarthritis and less inflammation in their joints.

"Regardless of diet, mice receiving FST gene therapy were protected from post-traumatic OA and bone remodeling induced by joint injury. Together, these findings suggest that FST gene therapy may provide a multifactorial therapeutic approach for injury-induced OA and metabolic inflammation in obesity," wrote the researchers in their paper.

Last but not least, the researchers were worried that the muscle growth caused by the therapy could hurt the heart. However, the study revealed that the heart function and cardiovascular health of treated mice actually improved.

If the therapy can be adapted to work for humans both its appeal and its applications would be limitless. The research was published in Science Advances.

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New Gene Therapy Promises You Will Get Ripped Without Stepping in a Gym - Interesting Engineering

Add a new, 100 million California facility to the growing gene therapy infrastructure.

Merck KGaA announced they are opening a second gene therapy and viral vector factory in Carlsbad, California. The new center will be just one point in the global supply chain Big Pharma is rapidly erecting to keep manufacturing capacity for the new technology at pace with clinical development. Over the past year, Novartis, PTC Therapeutics, Pfizer and Vertex each announced or opened new facilities in Switzerland, North Carolina and New Jersey that will help build gene therapy.

In November,Reutersreported that 11 drugmakers had set aside $2 billion for the manufacturing effort. They were led by Novartis, the giant behind the second FDA-approved gene therapy in Zolgensma which planned to spend $500 million, and Pfizer, which has yet to get a gene therapy approved but will spend $600 million. Catalent and Thermo Fisher, meanwhile, each spent over a billion dollars acquiring companies involved in gene therapy or viral vector manufacturing.

Merck KGaA last shored up its gene therapy manufacturing in 2016 the year before the first gene therapy was approved in the US when they expanded their original Carlsbad facility from 44,000 to 65,000 feet. At 140,000 feet, the new facility will be more than double the size of its older neighbor. Gene therapies and the viral vectors used to deliver them will be cooked up in 1,000 liter bioreactors metal vats roughly the size of 260 gallons of milk. Merck has not said when it is scheduled for completion.

Although Merck KGaA lists no gene therapies in its most recent pipeline, the German drugmaker uses its facilities to help biotechs focused on the emerging modality to manufacture their products. The list of approved gene therapies can still be counted on one hand, but hundreds are now in clinical trials.

Manufacturing emerged as a pressing bottleneck almost as soon as Spark Therapeutics got Luxturna, a treatment for a form of inherited blindness, approved as the US first gene therapy. In an op-ed in STAT last year, Sparks head of technical operations, Diane Blumenthal, described the process of building in Philadelphia one of the first in-house gene therapy manufacturing facilities. With regulators increasingly open to approving the treatments quickly, she encouraged other companies to invest in manufacturing in advance of even knowing if the therapy works, particularly because gene therapy requires customization in ways few other modalities do.

And she warned the problem will only get more pressing.

There isnt a gene therapy manufacturing playbook yet to guide the development of gene therapies, Blumenthal wrote. Manufacturing a gene therapy is only half the battle. The other half is making enough of it, doing that as efficiently as possible, and getting it to the patients who need it. These challenges become even more urgent to tackle as the industry shifts to the next chapter in gene therapy development, from treatments made in small batches for small patient populations to bigger volumes for larger rare-disease populations and commercial scale.

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Merck KGaA adds to gene therapy manufacturing boom with 100M facility in California - Endpoints News

Japans national health insurance will cover a gene therapy drug for a rare childhood genetic disorder that costs 167 million per treatment, making it the most expensive medication funded by the public system, government officials said Wednesday.

An advisory panel to the health minister approved provision of Swiss pharmaceutical giant Novartis AGs drug Zolgensma for spinal muscular atrophy patients under the age of 2 starting as early as May 20, the officials said.

The drug, which costs over 200 million in the United States, is known as the worlds most expensive medication.

It is a one-time therapy for the genetic disorder, which causes motor neuron loss and muscle wasting, and its coverage by insurance will offer hope for patients of the disease and their families.

With Japans social security expenses ballooning amid the rapid aging of its population, some experts have expressed concerns over the burden on the insurance system if the treatment is included. They also note that new drugs have tended to be highly expensive in recent years.

But a senior health ministry official said that given the small number of patients of the rare illness, the fiscal impact is going to be limited.

The previous most expensive drug in Japan was Kymriah, approved last May to treat leukemia and other hematologic cancers, at 33.49 million.

Novartis Pharma K.K., a Tokyo-based unit of the Swiss pharmaceutical company, produces and sells both Kymriah and Zolgensma.

Spinal muscular atrophy affects one or two infants out of every 100,000 and can lead to severe respiratory problems and early death. Without use of an artificial respirator, it is said that most die within 18 months.

Zolgensma will be given as a one-time infusion into the vein, which can introduce normal genes into human cells to recover motor function.

Novartis expects that the drug will be administered to about 25 patients per year in Japan, estimating annual sales of 4.2 billion.

Under the Japanese insurance system, out-of-pocket medical expenses for those under 2 are set at 20 percent of the total. But the real payment is minimal as the central and municipal governments cover almost all the expense under subsidy programs.

Excerpt from:

Japan's health insurance to cover 167 million gene therapy drug - The Japan Times

The Gene Therapy report provides independent information about the Gene Therapy industry supported by extensive research on factors such as industry segments size & trends, inhibitors, dynamics, drivers, opportunities & challenges, environment & policy, cost overview, porters five force analysis, and key companies profiles including business overview and recent development.

Gene Therapy MarketLatest Research Report 2020:

Download Premium Sample Copy Of This Report: Download FREE Sample PDF!

In this report, our team offers a thorough investigation of Gene Therapy Market, SWOT examination of the most prominent players right now. Alongside an industrial chain, market measurements regarding revenue, sales, value, capacity, regional market examination, section insightful information, and market forecast are offered in the full investigation, and so forth.

Scope of Gene Therapy Market: Products in the Gene Therapy classification furnish clients with assets to get ready for tests, tests, and evaluations.

Major Company Profiles Covered in This Report

Pfizer Inc.,Novartis AG,Bayer AG,Sanofi,GlaxoSmithKline plc.,Amgen Inc.,Boehringer Ingelheim International GmbH,uniQure N.V.,bluebird bio, Inc.,Celgene Corporation

Gene Therapy Market Report Covers the Following Segments:

Application: By Disease Indication,Cancer,Genetic disorders,Cardiovascular diseases,Ophthalmology,Neurological conditions,Others,,By Type of Vectors,Viral vectors,Non-viral vectors,,By Type of Cells,Somatic cells,Germline cells

North America

Europe

Asia-Pacific

South America

Center East and Africa

United States, Canada and Mexico

Germany, France, UK, Russia and Italy

China, Japan, Korea, India and Southeast Asia

Brazil, Argentina, Colombia

Saudi Arabia, UAE, Egypt, Nigeria and South Africa

Market Overview:The report begins with this section where product overview and highlights of product and application segments of the global Gene Therapy Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company:Here, the competition in the Worldwide Gene Therapy Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data:As the name suggests, this section gives the sales data of key players of the global Gene Therapy Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the global Gene Therapy Market.

Market Status and Outlook by Region:In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Gene Therapy Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User:This section of the research study shows how different end-user/application segments contribute to the global Gene Therapy Market.

Market Forecast:Here, the report offers a complete forecast of the global Gene Therapy Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion:This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

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COVID 19 PANDEMIC: Gene Therapy Market Is Set To Experience Revolutionary Growth By 2025 - Cole of Duty

MGTA-145 was shown to be a rapid, reliable, efficient and G-CSF-free method to obtain high numbers of functional HSCs in a Phase 1 trial; the cells could be gene modified and engraft in animals. MGTA-145 could be used to improve collection and gene therapy outcomes

Additional preclinical data show MGTA-145 serves as efficient, same-day mobilization regimen for in vivo HSC gene therapy in animals, which could be applicable in treating sickle cell disease and other genetic disorders

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics(Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of blood and immune reset to more patients, presented preclinical data on its stem cell mobilization therapy clinical candidate, MGTA-145, this week at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT).

Magenta is developing MGTA-145 as a first-line standard of care for hematopoietic stem cell (HSC) mobilization in a broad range of diseases, including autoimmune diseases, blood cancers and genetic diseases, such as sickle cell disease. MGTA-145, a CXCR2 agonist, acts in combination with plerixafor, a CXCR4 antagonist, and met all endpoints in a Phase I trial showing reliable same-day mobilization and collection of HSCs for genetic modification and transplant. MGTA-145 has been dosed in more than 100 healthy volunteers.

Magenta intends to initiate multiple Phase 2 trials of MGTA-145 and generate initial Phase 2 data in 2020. These trials, which will include both allogeneic and autologous transplant settings, will evaluate mobilization and collection of functional HSCs and their engraftment in patients after transplant to rebuild the blood and immune systems.

MGTA-145 has the potential to fundamentally transform the standard of care for stem cell mobilization, collection and engraftment for patients and donors, said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics. These data provide further confirmation that cells obtained with MGTA-145 can be used in gene therapy and gene editing settings across various genetic diseases. These are encouraging findings for the breadth of applications for MGTA-145, showing safe and robust mobilization of functional cells that can be used for stem cell transplant, as well as for gene therapy applications, expanding the programs potential for even more patients beyond the 150,000 patients presently eligible in the U.S. and Europe.

MGTA-145 Preclinical Data

These data demonstrate that MGTA-145, in combination with plerixafor, enables the same-day mobilization of sufficient functional HSCs that can be gene modified and engrafted.

Title: MGTA-145, in Combination with Plerixafor, Rapidly Mobilizes Large Numbers of HSCs in Humans That Can Be Gene Edited with CRISPR/Cas9 and Mediate Superior Engraftment to Standard-of-Care (Abstract #123)Presenter: Kevin Goncalves, Ph.D., Magenta Therapeutics, Cambridge, Mass.Date and Time: Tuesday, May 12, 2020 3:45-5:30pm

In a limit dilution study using CD34+ cells from a Phase 1 healthy volunteer study, same-day, single-dose mobilization with MGTA-145, in combination with plerixafor, led to 10x higher numbers of engrafting human HSCs in NSG mice, as compared to current standard-of-care approaches. Higher engraftment was confirmed by congenic mouse transplant models in primary and secondary recipients, indicating durable engraftment with MGTA-145 plus plerixafor mobilized blood.

To determine whether MGTA-145 plus plerixafor mobilized blood CD34+ cells could be efficiently gene-modified for use in a variety of therapeutic applications, CD34+ cells from two healthy donors were edited with CRISPR/Cas9 targeting beta-2-microglobulin. Ninety percent editing was achieved, and these cells were successfully engrafted in an NSG mouse model.

This same-day mobilization and collection regimen could potentially offer a significant improvement of cell collection protocols and autologous gene therapy outcomes for a variety of genetic diseases.

Title: MGTA-145/Plerixafor-Mediated HSC Mobilization and Intravenous Gene Therapy in Mice Allows for Efficient in vivo HSC Transduction and Stable Gene Marking in Peripheral Blood Cells (Abstract #810)Presenter: Chang Li, Ph.D., Division of Medical Genetics, Department of Medicine, University of WashingtonDate and Time: Wednesday, May 13, 2020 5:30-6:30pm

These results show, for the first time, that MGTA-145 plus plerixafor can enable robust, same-day mobilization of large numbers of stem cells in animal models that can be efficiently modified in vivo by gene therapy without transplant, which could be applicable in patients with sickle cell disease or other genetic disorders.

The data show that the one-hour MGTA-145 + plerixafor mobilization regimen was superior compared to the five-day G-CSF + plerixafor approach, yielding less leukocytosis, lower cytokine release after virus delivery, better cost effectiveness and, potentially, improved performance in models of hemoglobinopathies.

About Magenta Therapeutics

Headquartered in Cambridge, Mass., Magenta Therapeutics is a clinical-stage biotechnology company developing novel medicines for patients with autoimmune diseases, blood cancers and genetic diseases. By creating a platform focused on critical areas of unmet need, Magenta Therapeutics is pioneering an integrated approach to allow more patients to receive one-time, curative therapies by making the process more effective, safer and easier.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Magentas future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting pre-clinical and clinical data, the anticipated timing of our clinical trials, and the development of our product candidates. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials or to market products; risks, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on Magentas business, operations, strategy, goals and anticipated timelines; and other risks concerning Magenta are described in additional detail in its risks set forth under the caption Risk Factors in Magentas most recent Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200514005469/en/

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Magenta Therapeutics Presents Data at Annual Meeting of American Society of Gene and Cell Therapy Demonstrating Cells Mobilized with MGTA-145 in a...

PHILADELPHIA, May 07, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders and the Gene Therapy Program (GTP) at the University of Pennsylvania (UPenn) today announced the expansion of their collaboration agreement to include an additional five programs and extending Passage Bios period to exercise new programs for an additional three years (through 2025). Additionally, Passage Bio will fund discovery research at GTP and will receive exclusive rights, subject to certain limitations, to technologies resulting from the discovery program for Passage Bio products developed with GTP, such as novel capsids, toxicity reduction technologies and delivery and formulation improvements.

Our collaboration with the GTP gives us access not only to the best discovery, technology, and research available but also to pioneering expertise in the field of gene therapy, including pre-clinical development and manufacturing experience that will help guide our programs as we move into clinical development, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. Expanding this collaboration provides us with the opportunity to not only deepen our pipeline but also strengthen our own expertise and capabilities as we strive to develop transformative gene therapies for patients. We are tremendously proud of the progress we have accomplished to date through this partnership and look forward to continuing this momentum in the years to come.

This expansion builds upon the original collaboration, which successfully established a strong partnership between Passage and GTP. Under the expanded agreement, Passage will pay $5 million annually to Penn to fund research across numerous technology applications for gene therapy. In addition to five additional program options and an extension of the relationship through 2025, Passage will receive exclusive rights, subject to certain limitations, to IP arising from this research and related indications that are applicable to the products it develops with GTP.

The partnership between GTP and Passage Bio continues to be extremely strong and productive as we collaborate to bring our gene therapy products to patients. We are extremely excited to expand the reach of our CNS products and discovery research through this continued collaboration, said James Wilson, M.D., Ph.D. director of the Gene Therapy Program at the University of Pennsylvania and chief scientific advisor of Passage Bio. As a co-founder of the company, I am also deeply committed to the growth and success of Passage. I believe that the expansion of this strong collaboration further establishes Passage Bios leadership in gene therapy and I look forward to continuing to work with our dedicated teams to reach these shared goals of helping patients with rare, monogenic CNS disorders.

About Passage Bio Passage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.

Forward Looking StatementThis press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about our collaborators and partners ability to execute key initiatives and the benefits and obligations associated with our arrangements with our collaborators and partners; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize our product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabeStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.com

Media:Emily MaxwellHDMZ312-506-5220emily.maxwell@hdmz.com

Financial Disclosure: The University of Pennsylvania and Dr. James Wilson are both co-founders of Passage Bio and hold equity interests in the company. Dr. Wilson is also the chief scientific advisor of the Company. Penn and GTP are the recipients of significant sponsored research support from the Company under research programs directed by Dr. Wilson. Penn has licensed or optioned numerous technologies to Passage Bio under an existing license and these ongoing sponsored research activities, and both Penn and Dr. Wilson stand to receive additional financial gains in the future under these licensing arrangements.

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Passage Bio Announces Expansion of Gene Therapy Collaboration with University of Pennsylvania - GlobeNewswire

For its first quarter ended March 31, 2020, the Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million a year earlier

Inc (), a global biotech company focused on accelerating and transforming the delivery of cell and gene therapies, posted first-quarter results on Monday that saw its revenue soar 348% year-over-year driven by its Cell and Gene Therapy (CGT) Biotech platform.

For its first quarter ended March 31, 2020, the Germantown, Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million in the first quarter of 2019.

Orgenesis achieved net income of $75.6 million, or $4.23 per share, reflecting the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO).

READ:Orgenesis boss Vered Caplan makes top 20 list of inspirational leaders in advanced medicine

On February 11, Orgenesis completed the successful sale of its CDMO business to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

As a result, Orgenesis reported cash and equivalents of $107.1 million as of March 31, 2020.

In a statement accompanying the numbers, Orgenesis CEO Vered Caplan said: Step by step, our CGT Biotech Platform is gaining traction within the market, as illustrated by the year-over-year growth.

In the first quarter of 2020, revenue increased to $1.9 million, or nearly an $8 million revenue run rate compared to $3.1 million for all of 2019. We believe our CGT Biotech Platform is poised for growth this year through industry partnerships that are currently underway with leading research institutes and hospitals around the world, she added.

The companys CGT Biotech platform consists of three core elements:point-of care Therapeutics, point-of care Technologies, and point-of care Network.

Caplan also noted that earlier this year, the company struck collaboration agreements with two leading healthcare research institutes in the US.

We plan to utilize our point-of-care Network to support their growing development and processing needs in order to advance and accelerate cell and gene-based clinical therapeutic research, said Caplan.

Orgenesis is using the Masthercell sale proceeds to expand the companys point-of-care cell therapy business. The biotech is currently focused on therapies which span a wide range of treatments.

In addition to our POCare Network, we are building our pipeline of POCare Therapeutics and Technologies, with an ultimate goal of providing life-changing treatments to large numbers of patients at reduced costs within the point-of-care setting, said Caplan.

Specifically, we are focusing on immune-oncology, metabolic and autoimmune diseases, as well as anti-viral therapies.

Orgenesis also recently completed the acquisition of Tamir Biotechnology and its broad-spectrum antiviral platform, ranpirnase in a cash and stock deal for roughly $21 million. The company will use ranpirnase to target human papillomavirus (HPV), which causes genital warts.

Ranpirnase has demonstrated clinical efficacy against HPV and other hard to target viruses based on its unique mechanism of action of killing the virus and modulating the immune system, said Caplan.

Going forward, Orgenesis plans to move the program through a Phase 2b trial in the US.

Meanwhile, the Orgenesis boss said the company has received a nod from regulators to keep research alive at its labs during the coronavirus (COVID-19) pandemic.

We are leveraging all our knowledge and expertise in the field of cell and gene therapy, including anti-viral technologies, in an attempt to find potential COVID-19 cures and therapies, said Caplan.

Importantly, we have a strong balance sheet and are strategically positioned to bring a variety of therapies to market in a cost-effective, high-quality and scalable manner.

At the start of April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous CGT Biotech platform to advance clinical trials.

Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and various other cellular products.

Our plan is to combine RevaTis patented technique to obtain mdMSCs through a minimally invasive muscle micro-biopsy with our own automated/closed-systems, 3D printing, and bioreactor technologies, said Caplan.

The goal of this JV is to lower the costs and accelerate the timeline of bringing these innovative therapies through the clinic and into commercialization.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Orgenesis sees 1Q revenue rocket driven by its Cell and Gene Therapy Biotech platform - Proactive Investors USA & Canada