StemCell Therapy

Retinal Disorders Treatment Market: Introduction

According to the report, the global retinal disorders treatment market was valued at US$ 9.18 Bn in 2019 and is projected to expand at a CAGR of ~7% from 2020 to 2030. Macular degeneration is of two types: wet age-related macular degeneration and dry age-related macular degeneration. Diabetic retinopathy is one of the common diabetic eye disorders characterized by damaged blood vessels in the retina. Damaged blood vessels and nerves lead to vision impairment, blurring of vision, and eye hemorrhage. If left untreated, it could lead to retinal detachment and blindness. In terms of indication, the global retinal disorders treatment market has been classified into macular degeneration, diabetic retinopathy, diabetic macular edema, and others. The macular degeneration segment has been bifurcated into dry macular degeneration and wet macular degeneration. Based on therapeutic class, the global retinal disorders treatment market has been categorized into anti-VEGF agents and others. In terms of dosage form, the global retinal disorders treatment market has been divided into gels, eye solutions, capsules & tablets, eye drops, and ointments.

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Based on distribution channel, the global retinal disorders treatment market has been segregated into hospital pharmacies, retail pharmacies, and online sales. Rise in prevalence of retinal disorders due to increase in geriatric patient population boosts the growth of the global retinal disorders treatment market. The U.S. dominated the global retinal disorders treatment market in 2019, and the trend is anticipated to continue during the forecast period. Well-established healthcare infrastructure and early adoption of advanced technologies are the factors expected to fuel the growth of the market in the region. Moreover, rise in prevalence of various types of retinal disorder leads to increase in demand for treatment.

China is likely to be a highly lucrative market for retinal disorders treatment during the forecast period. Diagnosis and treatment rates have increased due to a rise in disposable income and health awareness. This has led to an increase in the adoption of macular degeneration drugs

Rise in Prevalence of Retinal Disorders Due to Increase in Geriatric Patient Population to Drive Global Market

Age is a prominent risk factor for age-related macular degeneration. The risk of developing advanced age-related macular degeneration increases from 2% in people aged between 50 and 59 to nearly 30% for those over 75. The prevalence of other retinal disorders was 93 million people with diabetic retinopathy, 21 million people diabetic macular edema and 28 million people with vision-threatening diabetic retinopathy. Increase in R&D activities, rise in the number of patients suffering from diseases, and rapid expansion of healthcare and biopharmaceutical industries in developed and developing countries are projected to boost advancements in therapies in the AMD treatment market during the forecast period. For instance, Lucentis and Eylea accounted for 2.8% of total pharmaceutical sales in Canada in 2017.

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Macular Degeneration to Dominate Global Market

In terms of indication, the global retinal disorders treatment market has been divided into macular degeneration, diabetic retinopathy, diabetic macular edema, and others. Macular degeneration has been bifurcated into dry macular degeneration and wet macular degeneration. The macular degeneration segment dominated the market in terms of revenue in 2019. The rise in prevalence of macular degeneration is anticipated to drive the segment during the forecast period. For instance, the number of people living with macular degeneration is expected to reach 196 million globally by 2020 and increase to 288 million by 2040.

Anti-VEGF Agents to be Main Therapeutic Class

Based on therapeutic class, the global retinal disorders treatment market has been categorized into anti-VEGF agents and others. The anti-VEGF agents dominated the global retinal disorders treatment market in 2019. Major market products such as Avastin and Eylea are included in the anti-VEGF drug class. Increase in demand for these products in the treatment of retinal disorders and strong product pipeline are likely to drive the segment. However, the others segment, which includes anti-inflammatory drugs, is projected to expand at the highest CAGR from 2020 to 2030. The increase in the use of anti-inflammatory drugs for pain relief is anticipated to augment the segment.

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Eye Solutions to be Preferred Dosage Form

In terms of dosage form, the global retinal disorders treatment market has been divided into gels, eye solutions, capsules & tablets, eye drops, and ointments. The eye solutions segment dominated the global retinal disorders treatment market in 2019. However, the eye drops segment is expected to expand at the highest CAGR during the forecast period. The segment is likely to grow at a rapid pace due to increase in demand for eye drops for treatment of retinal diseases in emerging countries such have China, India, and Brazil.

Retail Pharmacies to Emerge as Major Distribution Channel

Based on distribution channel, the global retinal disorders treatment market has been segregated into hospital pharmacies, retail pharmacies, and online sales. The retail pharmacies segment dominated the market in terms of revenue in 2019 due to wide network, ease of access, and diverse product offerings, including prescription and OTC ophthalmic drugs. However, the shift toward the use of electronic payment modes is projected to boost the growth of the online sale segment during the forecast period.

U.S. to Dominate Global Market

The global retinal disorders treatment market has been segmented into five major regions/country: the U.S., Europe, China, Russia, and Rest of the World. The U.S. dominated the global market in 2019, followed by Europe. The U.S. accounted for a major share of the global retinal disorders treatment market in 2019. Well-developed healthcare infrastructure, high healthcare expenditure, and adoption of branded drugs to treat retinal disorder disorders are the key factors attributed to the countrys significant share of the global market.

The retinal disorders treatment market in China is anticipated to expand at a high CAGR from 2020 to 2030. There have been significant unmet medical needs in the region. Furthermore, healthcare expenditure is increasing in developing markets. Key players are making investments to establish their operations in China. This, in turn, is projected to augment the market in the country.

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Competition Landscape

Regeneron Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., and Novartis AG are the three major companies operating in the global retinal disorders treatment market. The global retinal disorders treatment market is fragmented in terms of number of players. Key players in the global market include Allergan plc, Bayer AG, F. Hoffmann-La Roche Ltd., Graybug Vision, Inc., Kubota Pharmaceutical Holdings Co., Ltd., Novartis AG, Pfizer, Inc., Regeneron Pharmaceuticals, Inc., Santen Pharmaceutical Co., Ltd., and Takeda Pharmaceutical Company Limited. New product development through robust R&D activities and mergers & acquisitions are key strategies adopted by these players to gain a competitive advantage in the global retinal disorders treatment market.

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Retinal Disorders Treatment Market: Advancements in Gene-therapy and Stem-cell Therapy to Bolster Market Growth - BioSpace

DetailsCategory: DNA RNA and CellsPublished on Monday, 02 November 2020 14:44Hits: 321

BOSTON, MA, USA I November 02, 2020 IDecibel Therapeutics, a clinical-stage biotechnology company developing novel restorative gene therapeutics to treat hearing loss and balance disorders, today announced exclusive license agreements with the University of Florida (UF) and the University of California, San Francisco (UCSF) for an adeno-associated virus (AAV) gene therapy technology designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene.

Otoferlin is a protein present in the inner hair cells of the cochlea that is critical for the communication between sensory cells of the inner ear and the auditory nerve by regulating release of neurotransmitters. People born with biallelic mutations in the otoferlin gene have profound hearing loss because this signal between the ear and the brain is lost. Decibel aims to restore functional otoferlin using gene therapy. A principal challenge is the size of the otoferlin gene, which is too large for the packaging capacity of AAV vectors. To overcome this challenge, Dr. William Hauswirth (UF), Dr. Omar Akil (UCSF), and collaborators employed a dual-vector approach to deliver the gene in two separate AAV vectors. This approach resulted in expression of the complete otoferlin gene, restored the signaling connection between the ear and the brain, and rescued normal hearing in a deaf, otoferlin-deficient mouse model.1

The dual-AAV gene therapy approach has shown significant promise in preclinical research as a method to deliver a large gene to the ear, said William Hauswirth, Ph.D., Professor of Ophthalmology at University of Florida. We were able to cure deafness in a mouse model and look forward to the potential of this technology in the development of a gene therapy to restore hearing in human patients.

The AAV-mediated gene therapy technology is jointly owned by UF and UCSF, and Decibel has secured exclusive licenses to the rights of the two universities in the intellectual property. The technology has been incorporated as a key component of Decibels lead gene therapy program, DB-OTO, for treatment of individuals lacking otoferlin.

Decibels precision gene therapeutic, DB-OTO, also incorporates proprietary, cell-specific regulatory control that restricts expression of the human otoferlin sequence to hair cells of the inner ear, thereby avoiding off-target expression. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals.

There is a significant unmet need for therapeutics that can restore hearing in individuals with congenital, profound hearing loss. We believe AAV-mediated gene therapy is a modality well-suited to the ear, which could potentially have a major impact for patients, said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. These important agreements support our goal to further advance our lead developmental gene therapy program, DB-OTO.

Terms of the agreement have not been disclosed.

1Akil et al. (2019) Proceedings of the National Academy of Sciences. https://doi.org/10.1073/pnas.1817537116

About Decibel Therapeutics

Decibel Therapeutics is a clinical-stage biotechnology company focused on developing treatments that restore functional hair cells within the inner ear to treat disorders of hearing and balance. Leveraging industry-leading single-cell genomics capabilities and proprietary gene therapy technologies, Decibel has established the worlds first comprehensive research, discovery and drug development platform aimed at restoring hearing and balance function. Decibels pipeline, including its lead gene therapy program (DB-OTO) to treat congenital monogenic deafness and its ototoxicity prevention program, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow @DecibelTx.

SOURCE: Decibel Therapeutics

Excerpt from:
Decibel Therapeutics Announces Exclusive Licensing Agreements for Hearing Loss Gene Therapy Technology | DNA RNA and Cells | News Channels -...

Big Pharma execs usually dont just fade away. Sometimes, they jump to the money side of the biotech business, like Chris Viehbacher and Olivier Brandicourt. Some stick with a batch of mentoring board seats. Joe Jimenez not only plans to do both, hes also going biotech entrepreneur and starting his own play in style.

A week ago, Jimenez and his close partner Mark Fishman, who got to know each other well during their respective time as CEO and early-stage NIBR chief at Novartis, filed papers with the SEC noting that their Aditum Bio Fund I had raised the $133,040,000 they had targeted more than a year ago.

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Novartis buys a new gene therapy for vision loss, and this is one preclinical venture that didn't come cheap - Endpoints News

Then, Girondi claimed, Sloan Kettering mothballed his work to favor Bluebird, whose chief executivehad a prior business relationship with the cancer center's boss.

Girondis rage has been fueled by Bluebirds trajectory since then: its thalassemia treatment was approved last year by the European Union and at $1.8 million per patient will be among the most expensive.

In my neighborhood, theyd have gotten ball batted for similar behavior, said Girondi, a self-described former street tough from the South Side of Chicago whosbeen using such talk to describe his adversaries for years.

Now, Girondi is finally getting his day in court. Having survived years of legal challenges, which have portrayed his case as absurd and Girondi himself as erratic and ill-tempered, his trial began on Thursday.Errant is seeking hundreds of millions of dollars in damages, according to court filings.

The trial promises a rare glimpse into the not uncommonly messy marriage of medical researchers and for-profit companies, and it will showcase a slew of revealingdocuments and emails that have emerged in the court file, including one that Girondis lawyers described in court as the smoking gun.

Written in June 2010 by Nick Leschly, then interim president of Genetix Pharmaceuticals, which was renamed Bluebird Bio a few months later, the emailsaid:Pat Girondineed to shut him down.

The recipient of the email, another Genetix executive, responded by saying they need to be nice, suck up, etc. to Girondi, so they can review valuable data from a Sloan Kettering scientist with whom Errant was collaborating.

Both Sloan Kettering and Bluebird deny Errants allegations.

Sloan Kettering is vigorously defending itself in court, said Jorge Lopez, executive vice president and general counsel for Memorial Sloan Kettering Cancer Center, in a statement. We also disagree with EGTs characterizations of the case and of the Courts rulings. He declined further comment.

In court filings, Sloan Kettering has argued that Girondis company, called EGT for short,wasnt tricked but rather was short of money and repeatedly failed to meet obligations outlined in its deal.

The evidence shows that EGTs case is the fantasy of a defunct company and its founder that refuse to accept responsibility for their own failure, an attorney for Sloan Kettering Institute wrotein a July 8, 2019 filing.

Bluebirds attorney, Jeffrey Eilender, said the court record has disproved Errantsclaim of a conspiracy between Sloan Kettering Institute, referred to as SKI in court filings, and Bluebird, as well as an allegation that Bluebird gleaned secrets from Girondis company.

None of the evidence relied upon by EGT shows a material issue as to the ultimate fact: none suggests in any way that there was an agreement between Bluebird and SKI to defraud EGT, Bluebirds lawyers wrotein a July 8, 2019 court filing. In fact, EGT does not even explain how or why the facts it cites are relevant here (they are not); it just throws everything at the wall to see if it sticks.

As for Leschlys email saying that Girondi needed to be shut down, Eilender said it referred to Girondi,not the company. Why? Because with all due respect to Mr. Girondi, hes a nudnik, Eilender told the court at January 2019 hearing, explaining Girondi had becomea nuisance.

Leschly wasnt available for an interview, but he has previously expressed his opinions about Girondi and his firm.

Errant is toothless and the guy behind it is completely insane, truly, Leschly wrote in a 2012 email to an investment analyst.

Thalassemia is an inherited blood disorder in which the body doesnt produce enough hemoglobin, the substance in red blood cells that carriesoxygen. Moderate and severe, or beta,cases require frequent blood transfusions and can result in early death.

In 1992, Rocco Girondi was diagnosed with a more severe form of the blood disorder. He was two years old. The next year, Girondi retired from what he describes as a lucrative trading career to devote himself to finding a cure.

Girondi, 62, isnt your typical biotech entrepreneur. A high school dropout, he was listed as one of America's most eligible bachelors in Playgirl magazine in 1988 and appeared on the Oprah Winfrey Show in an episode on male chauvinists. (A copy of the show wasnt readily available; Girondi said he had defended both a mans and a woman's right to work, but believed one should stay home if they have children).A 1987 article about him in Chicago Magazine is entitled Fonzie Gets Rich. He left Chicago decades ago for Italy, where he occasionally performs in concert, playing blues and rock andthe occasional Italian ballad. But hestill speaks in the blunt, sometimes salty, manner of the Chicago neighborhood where he grew up.

He had some money at a time when few others showed interest in gene therapy. By 2000, Girondi began providing financial support to researchers at Sloan Kettering, including Dr. Michel Sadelain, who had brought thalassemia under control in mice, according to Errants complaint. Those researchers had developed a method to replace defective genes in thalassemia patients with a healthy copy. The plan used a modified virus known as a vector to deliver the genetic material into the cells.

At that time, gene therapy was relatively new and scarred by missteps, including a patient who had died after undergoing treatment. In 2005, Sloan Kettering granted a license to develop Sadelains potential gene therapy treatment to the only interested party, Errant Gene Therapeutics, according to the complaint.

Progress was slow and hampered by delays, according to Girondi and his lawyers. But by 2010, Errant had manufactured enough of the medicine to start clinical trials, his lawyers say in court papers.

Girondi said the relationship between his company and Sloan Kettering changedsoon after Craig Thompsons hiring as president and chief executive officer of Memorial Sloan Kettering Medical Centerwas announced in August 2010. By that fall, he said it was clear his company was on the outs.

It ended very strangely, Girondi said. I think thats the best way to say it.

Sadelain didnt respond to messages seeking comment.

Thompson, 67, had previously worked at the University of Pennsylvania, where he had been director of the Abramson Cancer Center. He also co-founded a company called Agios Pharmaceuticals in 2007with the goal of unlocking a new field of discovery in cellular metabolism.

Agiosreceived an infusion of $33 million from several venture capital firms in 2008 including Third Rock Ventures, where Leschly the future Bluebird CEO -- was a partner. Leschly also served as Agioss interim chief business officer,according to a 2010 Bluebird press release. A few months later, when Thompson began his job at Sloan Kettering, he was listed as being on Agioss scientific advisory board.

In September, 2010, Sloan Kettering asked Errant for physical possession of the vector to complete a study which it said was necessary to move forward with clinical trials, according to Errants lawyers. Errant delivered the vector and never got it back, the lawyers said.

Sloan Kettering said in court documents that by 2010, Errant had defaulted on its obligations, and that following arbitration and a new deal the following year, all rights granted to Errant in the 2005 deal reverted to Sloan Kettering.

Thompson started his job at Sloan Kettering in November, 2010. That same month,Sloan Ketteringmet with Bluebird and gave them a technical demonstration on Errants vector, sharing confidential information that served as a preludeto a more formal agreement the next year, according to Errants lawyers.

In November 2010, Bluebirds board of directors in 2010 weighedthe pros and cons of collaborating withSloan Kettering, according to Errants court filings. Among the positives? Eliminates the most threatening competitor, according to thepresentation, which is part of the court record.

Andrew Maslow, a former Sloan Kettering executive, said in an interview thathe made the decision to pursue a collaboration with Bluebird, not Thompson. One reason was the improving landscape for gene therapy, and another was Bluebirds capabilities, he said.

These guys are the real thing. They are totally capable, he said. They were just the opposite of Pat.

While Errant squabbled with Sloan Kettering, Bluebird continued to move toward commercialization of its treatment.

Bluebirds treatment for transfusion-dependent thalassemia patients, Zynteglo, was approved by the European Union last year, and the company plans to apply for U.S. approval in 2021. But thats just the beginning. The gene therapy is also intended foruseas a treatment for sickle cell disease(SCD). It could ultimately generate $1 billion a year in annual sales, according to Bloomberg Intelligence.

These therapies have the potential to transform the lives of patients with thalassemia and SCD,said Marc Engelsgjerd, a biotech analyst at Bloomberg Intelligence, who called the treatments groundbeaking.

Leschly, for one, has already benefited. He has pocketed roughly $78 million from stock sales since the company went public in 2013, according to data compiled by Bloomberg. Girondi and Sadelain have been left to contemplate what might have been.

We could have gotten an incredible product and a Nobel Prize, Sadelain said, in a May 2015 phone call that Girondi recorded, also part of the court file. And right now, we have nothing.

Read the rest here:
Sloan Kettering Institute, Bluebird Bio in court trial over gene therapy deal - Crain's Chicago Business

Precision medicines, such as cell therapies, remain expensive to manufacture and hard to access by patients. For example, Kymriah, the first chimeric antigen receptor (CAR) T-cell treatment approved in the United States, can have price tags as high as $475,000. Unfortunately, precision medicines are expensive to develop and manufacture, and the costs are ultimately borne by taxpayers and patients, according to The State of Personalized/Precision Medicine a report issued last year by GlobalData.

Today, companies are developing new models to lower the costs of manufacturing and bring drugs to more patients. Among them are companies developing new business models and services, innovative equipment for on-site manufacturing in hospitals, and improved formulation technology.

A key challenge for companies is scaling up the delivery of precision medicines, notes Janel Firestein, partner and life sciences industry leader at Clarkston Consulting. Companies supplying precision medicines are harvesting material for patients in a hospital or clinic, and then freezing or shipping it fresh to a contract manufacturing organization (CMO), contract development and manufacturing organization (CDMO), or other manufacturing entity.

What were seeing with a lot of our clients leveraging contract manufacturers is theyre contracting for specific slots, she says. They have x number of slots per week or month, and the scalability of that is hard.

Precision medicines are manufactured in small batches in accordance with genetic, environmental, and lifestyle factors, that is, for patients in subpopulations that meet certain well-defined criteria. (The subset of precision medicines known as personalized medicines are even more specific; that is, they are developed uniquely for each individual patient.) If a patient doesnt pass prescreening at the scheduled time, Firestein warns, the manufacturing slot for the patients treatment is lost unless the manufacturer can find another eligible patient.

Conversely, if the company is working across multiple CMOs in different countries, it needs to schedule slots in a predictable way. You need to know which slots are open, Firestein points out. You need to leverage automation and artificial intelligence to give a manufacturing view to physicians at the patient hub, so they know which dates are available and can ensure the patients cells are viable upon receipt at the manufacturing plant.

Orgenesis is among the companies turning to localization to deliver precision medicines to patients. The companys CEO and director, Vered Caplan, is a serial entrepreneur and among the top 20 inspirational leaders in advanced medicine listed in The Medicine Makers Power List 2020. Caplan has developed a point-of-care business model for hospitals that combines technological and biological development with a business strategy.

We see that centralized processing is very costly, she explains. It can be a solution for companies working in clinical trials, butonce you get to marketit is not feasible for large numbers of patients.

The companys Cell & Gene Therapy Biotech Platform incorporates the following elements: POCare Therapeutics, a pipeline of licensed cell and gene therapies (CGTs); POCare Technologies, a suite of proprietary and in-licensed technologies; and POCare Network, a collaborative, international ecosystem of research institutes and hospitals. This platform, the company asserts, is about decentralization, enabling precision medicines to be prepared on-site at hospitals.

The platform automates the production of precision medicines by validating closed box processes to reduce cleanroom footprints once the product gets to market. Caplan works to develop and commercialize drugs that can be licensed for use by hospitals in the Orgenesis network.

What we do is offer a low-cost supply platform with processing and regulatory solutions that are validated in a harmonized fashion, she details. Essentially, we take responsibility for R&D. Our hospitals are partners, and because were working in a network, the economic burden isnt high, and we can supply the therapy at a reasonable cost.

The Orgenesis approach doesnt follow the usual approach, which involves a hospital research center licensing its drug to a pharmaceutical company, which then pays the center for clinical trials. Instead, Orgenesis works in partnership with a partner hospital throughout the commercialization process. Production of the final product is automated and supplied via an on-site point-of-care processing unitreducing the complex logistics involved in transporting cells.

Fujifilm Diosynth Biotechnologies, a global CDMO, is developing a new platform to streamline the development of adeno-associated viruses (AAVs) for gene therapies. There are three methods to make AAVs, says Steve Pincus, PhD, the companys head of science and innovation. Two of the methods use viral vectors, and a third uses plasmids.

People using the latter need a source of cells and plasmids, he notes. Unfortunately, there are few licensable cell lines and few plasmid manufacturers. Consequently, as Pincus points out, If you want to manufacture your GMP plasmids at one of these, you have to wait 6 to 12 months to get in the queue.

Fujifilm wanted to tackle these problems, so it decided to license five different Rep-Cap plasmids, an adenovirus helper plasmid, and a human embryonic kidney 293 (HEK293) cell line for AAV production by plasmid transfection from Oxford Genetics. Pincus explains that by licensing these technologies, the company means to offer an HEK293 master cell bank that is well characterized and stocks GMP-grade Rep-Cap and helper plasmids, so that people can come and use those readily available reagents without having to wait 6 to 12 months, and so that the clients pay only for what they need.

To support the production of AAVs, Pincus and his team are developing specialized upstream and downstream processes. They are also developing in-process analytics for common problems in the AAV manufacturing space, such as measuring empty and full virus capsids.

Earlier this year, on September 8, Lonza announced that in a project at Sheba Medical Center in Israel, the first cancer patient received a CAR T-cell therapy that had been manufactured using the companys Cocoon platform. Cocoon is another model for distributed manufacturinga closed, automated piece of equipment for manufacturing cell therapies at the scale of a single patient, with a custom cassette that incorporates all the media, agents, and other consumables.

When you look at the way cell therapies are manufactured, one of the costs is cleanroom space, says Matthew Hewitt, PhD, head of clinical development and personalized medicine at Lonza. A cleanroom suite graded class B for air quality is noticeably more expensive than one graded class C, and the size of the room also matters. If you move to a closed or functionally closed automated platform like the Cocoon that has integrated cell culture, then you can move to cheaper cleanroom space, Hewitt asserts. or you can increase the manufacturing density in your existing cleanroom to use the space more efficiently.

Hewitt divides CAR T-cell manufacturing into a seven-step process: 1) collecting a patient sample; 2) preparing the sample for manufacturing; 3) activating the cells; 4) modifying (transducing) the cells; 5) expanding cell populations as needed for dosing; 6) washing, harvesting, and formulating the cells; and 7) dosing the patient. According to Hewitt, the steps currently automated by Cocoon include activation, transduction, and washing/harvesting/formulation. Additional automation features, he says, will debut in the coming months. Later this year, the company will begin beta testing automatic magnetic cell separation. Next year, the company plans to incorporate automated sample preparation into the Cocoons cassette.

Speaking on the future of manufacturing for precision medicine, Hewitt says he sees a role for both distributed and centralized models. Lonzas centralized facility in Houston, TX, for example, can offer standardized and well-controlled conditions, as well as an experienced team, for process development and early-stage activities.

Once you get to later stages, he points out, manufacturing needs to be moved toward the point of care to mitigate any issues with logistics. He adds that as cell therapies become more common, building enough space to process patient therapies at a centralized facility becomes increasingly impractical. Even if your centralized location served 50,000 patients a year, he says, the logistics would be a heroic endeavor.

Gene and cell therapies dont have much going on in terms of formulation, says Maria Croyle, PhD, professor of molecular pharmaceutics and drug delivery at the University of Texas at Austin. The formulation side needs to catch up.

She argues that even though precision medicines are often formulated just by adding glycerol to the cells, preparing precision medicines to dose the patient is often a complex process. When I talk about these therapies to my students, she relates, I explain that you need to thaw them out and do complicated dilutions. Its not as simple as adding 5 mL to a flask.

Precision medicines are often stored on-site in ultra-low-temperature (80C) freezers, devices that are, Croyle notes, expensive to run. The costs are often passed onto the patient. In addition, preparing the medications often involves lengthy dilution processes. Any of these medications that arent used within a couple of hours must be discarded, pushing costs yet higher.

Although some companies are moving to freeze-drying as a way to preserve living viruses and cells, preserving a live virus can take 48 to 72 hours. I had no idea until I talked to industry how much freezer dryers were a power drain, she recalls. They use a lot of electricity for 72-plus hours, and thats added to the cost of the drug.

Croyle has developed a method for stabilizing live viruses inspired by the film Jurassic Park, which depicted the recovery of dinosaur DNA from amber. She has three patents on a peelable film, inspired by amber, into which gene therapy or vaccine products can be suspended and dried within hours. You can mix them by 8 am, peel them by 3 pm, and package them to be sent off, she asserts. Its very simple and space savingits just a flat envelope with a strip of film, and it can be used in a variety of ways.

Film-packaged doses, she says, can be rehydrated to produce nasal-sprayable vaccines or injectable gene therapy solutions, or they can be placed under the tongue and upper cheek, where dissolution of the film surface releases the vaccine, activating an immune response. To commercialize the technology, she has founded Jurata Thin Film. The company is named after a mythical Lithuanian goddess who lived in an amber castle under the sea.

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Precision Medicines That Are Tailored and Off-the-Rack - Genetic Engineering & Biotechnology News

DUBLIN, Oct. 28, 2020 /PRNewswire/ -- The "Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations" report has been added to ResearchAndMarkets.com's offering.

This research identifies some of the key developments across CAR-T cell therapies and provides insights across technological, IP, and investment landscapes. The study also provides an analysis of the competitive landscape while highlighting the key growth opportunities within the CAR-T cell therapy platform.

Key Topics Covered:

1.0 Executive Summary1.1 Research Focus: Emerging Technologies Enabling chimeric antigen receptor (CAR) T-cell Therapies1.2 Analysis Framework: The Author's Core Value1.3 Research Methodology: Five Steps Toward Success1.4 Key Findings of Technology Breakthrough Driving Sepsis Diagnosis

2.0 Technology Snapshot2.1 Rising Pace of Cell and Gene Therapy Approvals2.2 Regulatory and Ethical Perspectives on Gene Therapy2.3 Rising Demand for Precision Medicine Strategies2.4 Manufacturing Continues to be the Key Bottle Neck2.5 II Generation Chimeric Antigen Receptors Likely to Dominate the Cell Therapy Landscape in the Future2.6 CD-19 is the Most Common Target Antigen for Allogeneic CAR-T Therapies

3.0 Emerging Patent Landscape3.1 Steady Increase in Patent Grants for CAR-T Cell Therapies3.2 University of Pennsylvania and Novartis Lead the Patent Landscape for CAR-T Cell Therapies3.3 China and the US Lead the Patent Landscape for CAR-T Cell Therapies3.4 Snapshot of Key Patent Grants: Novartis3.5 Snapshot of Key Patent Grants: Cellectis and BlueBird Bio

4.0 Analysis of the Investment Landscape4.1 Key M&A Trends Across the Global Life Sciences Sector4.2 Gene Therapy - Venture Capital Funding Assessment4.3 Gene Therapy - Big Pharma In-licensing Deals Assessment4.4 Strategic Insights: Cell Therapies and Gene Therapies, Viral Vector CMOS

5. Analysis of the Competitive Landscape5.1 Allogene Therapeutics5.2 Precision BioSciences Inc.5.3 CRISPR Therapeutics AG5.4 Cellectis S.A.5.5 Celyad5.6 Bristol-Myers Squibb (BMS)5.7 Gilead5.8 Novartis5.9 BlueBird Bio5.10 Summary of the Scoring Methodology5.11 Competitive Analysis of CAR-T Participants

6.0 CAR-T Cell Therapies: Growth Opportunity Universe6.1 Growth Opportunity: CAR-T for Solid tumors, 2020

7.0 Industry Influencers

For more information about this report visit https://www.researchandmarkets.com/r/dgf6f3

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Analysis of Recent Cell and Gene Therapy Innovations: 2020 Competitive Landscape Report - CD-19 is the Most Common Target Antigen for Allogeneic CAR-T...

Belgian companies Novasep and Handl Therapeutics have signed an agreement for the development and manufacturing of an adeno associated virus (AAV)-based gene therapy drug candidate for the treatment of neurodegenerative diseases.

Handl is developing multiple therapeutic AAV-based programs in collaboration with the Katholieke Universiteit Leuven, with the Center for Applied Medical Research of the University of Navarra, Spain, and with the Biomedical Neuroscience Institute of the University of Chile. The company is currently conducting invesrigational new drug (IND)-enabling pre-clinical studies.

Under the terms of the agreement, Novasep will develop and manufacture AAV vectors designed for these programs and will supply drug substance and drug product to support Handl Therapeutics preclinical and clinical studies.

Michael Linden, co-founder and head of research and development at Handl, said: We are excited to engage with Novasep to develop GMP manufacture capabilities for our novel gene therapies and are happy to have identified an outstanding partner right on our doorstep here in Belgium.

Cedric Volanti, Novaseps president of biopharma solutions, said: This new agreement recognizes Novaseps expertise in the field of viral vectors and will contribute to the important development of the cell and gene therapy market in Belgium.

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BRIEFNovasep and Handl sign gene therapy product development and manufacturing deal - The Pharma Letter

PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Scout Bio, a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions, today announced it has initiated two pilot clinical studies to demonstrate effectiveness of an AAV expressing a GLP-1 analog (SB-009) in treating diabetic felines.

Scout Bio envisions two potential therapeutic applications for SB-009. First, to replace daily insulin injections with a single injection of SB-009 to treat feline diabetes and second to significantly increase the percentage of cats entering remission when SB-009 is given with insulin.

The two initiated pilot clinical studies will investigate each of these two potential treatment paradigms.

Mark Heffernan, Ph.D., Chief Executive Officer of Scout Bio remarked, This one-time injectable therapy for feline diabetes has the potential to provide a convenient single treatment that is an alternative to the burden of twice daily insulin injections. We believe SB-009 has the potential to be a blockbuster product for animal health and that our pipeline of gene therapy products for pets will disrupt and grow major markets.

SB-009 was developed under a collaboration between scientists at Scout Bio and the University of Pennsylvanias Gene Therapy Program, where the protein was engineered to improve potency, circulating accumulation kinetics and manufacturability. The design of SB-009 makes the clinical dose both affordable and commercially attractive.

Matthew Wilson, VP Product Discovery and External Innovation said, This is a further example of our strong collaborative relationship with UPenns Gene Therapy Program and Scouts internal capabilities of executing preclinical research to rapidly identify highly potent AAV gene therapies. In less than 12 months after initiating a discovery program, we are now in a position to transition into patients.

Scout Bio has conducted robust preclinical studies with various GLP-1 constructs in rodents and healthy cats. Key findings include:

Dr. Anne Traas, Scout Bios Chief Development Officer reflected, Owners can be devastated to learn their pet has this life-threatening disease and unfortunately, many are unable to give twice daily insulin injections and have to make the difficult choice to euthanize their beloved pet. A one-time safe and efficacious therapy, given by a veterinarian, that eliminates the need for insulin and worry of hypoglycemia, would greatly improve the current treatment paradigm and result in an improved quality of life for diabetic cats and their owners.

Feline diabetes, a severe disease lacking recent innovation, remains a major challenge for veterinarians and owners to safely and effectively manage. Diabetes in most cats is similar to type 2 diabetes in people. Insulin resistance, caused by factors such as obesity, leads to Beta-cell disfunction (the cells that produce insulin). Cats become insulin dependent when blood sugar levels rise, commonly 3-10 times normal, leading to the development of clinical signs which can seriously and negatively impact both the owner and the cat. The most common signs are increased drinking, increased urination and weight loss despite ravenous appetite.

About SB-009 SB-009 is a recombinant AAV gene therapy viral vector utilizing a novel capsid expressing an engineered feline GLP-1 agonist for the treatment of feline diabetes. GLP-1 has been shown to be a safe and highly efficacious molecule in the treatment of humans with type 2 diabetes and SB-009 is the first gene-therapy delivered GLP-1 to be studied in clinical studies in cats with a view to treat the disease.

The expressed feline GLP-1 analog protein functions by stimulating the beta-cells in the pancreas to produce more insulin and may also have an effect in decreasing insulin resistance. GLP-1 receptor agonists do not decrease glucose levels in animals with normal blood glucose, so there is expected to be a very low, or no risk of hypoglycemia.

About Feline DiabetesMost diabetic cats appear to have disease similar to human type 2 diabetes, which is primarily defined as a combined problem with insulin production by the beta-cells in the pancreas, as well as a decrease in the sensitivity to the normal action of insulin (insulin resistance). In cats, one of the most common factors contributing to insulin resistance is obesity which reduces insulin sensitivity.

Lack of insulin production and decreased sensitivity to insulin causes the glucose (sugar) in the blood to become very high leading to the clinical signs. Very high levels of blood glucose also hurt the beta-cells in the pancreas, leading to further reductions in insulin production.

Substantial progress has been made in the treatment of human type 2 diabetes, even in the early stages of the disease. However, insulin therapy remains the only FDA-approved treatment for diabetes in cats.

About Current Treatment | Feline DiabetesCurrent therapy aims to replace the insulin that the cats body no longer makes by injecting insulin twice daily. Giving insulin in the right amounts may bring the blood sugar levels down. If the blood glucose can be brought under control for the majority of a 24-hour period each day, then the clinical signs will be reduced to manageable levels. Too much insulin can cause the blood sugar to drop to dangerous levels (hypoglycemia), so there has to be a careful balance made between maintaining ideal blood glucose levels andadministering too much insulinwhich may result in life threatening low blood sugar levels.

Often owners find the prospect of administering injections to their cats daunting and the strict regimen of twice daily injections and feedings can be difficult to fit into a busy lifestyle. Unfortunately, not treating the cats is simply not a viable option and usually results in a rapid decline in physical health. Even with insulin treatment, some cats diabetes is not well controlled, resulting in the continuation of clinical signs and/or euthanasia.

About Diabetic RemissionGood control of blood glucose may also allow the beta-cells to rest. That rest may increase their capacity to regain some of their insulin-secreting ability. Insulin administration helps to decrease and control the excess blood glucose levels and complement whatever insulin producing ability the cat has left. In some cases, cats regain enough function to allow the insulin injections to stop. This is called diabetic remission. A cat is determined to be in remission when blood sugar is normal and there is complete correction of clinical signs once insulin has been discontinued.

About Scout BioScout Bio is a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions. Scout Bios therapeutics are designed to induce long-term expression of therapeutic proteins in pet patients using AAV vector technology. Scout Bio has an exclusive research and development collaboration with the University of Pennsylvanias Gene Therapy Program. Scout Bios innovative partnerships build on a 20-year history with AAV leaders and is complemented by Scout Bios global leaders in gene therapy research and development. Scout Bio is a private company headquartered in Philadelphia, Pennsylvania. For more information, see http://www.scoutbio.coFor further information, please contact:

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

Media:Fran Gaconnier214.417.4142Fran.gaconnier@scoutbio.co

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Scout Bio Advances Novel Gene Therapy for the Treatment of Feline Diabetes - BioSpace

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Key Market Insights

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40%, claims Roots Analysis - PRnews Leader

Scientists working at the University of North Carolina, Chapel Hill reported in the Oct. 21, 2020, issue of Nature on the successful development of a one-time specific sequence-directed gene therapy approach using the combination of AAV with CRISPR technology that successfully prevented the presentation of Angelman syndrome throughout the lifetime of a mouse model.

Lifelong gene therapy has held promise for decades now as one of the only approaches that could possibly address many neurodevelopmental genetic disorders. But even after decades of research, gene therapy still possesses significant risks due to untoward random genomic insertions of vectors that could ultimately cause other genetic disorders.

Meanwhile, it has been known for decades now that adeno-associated virus (AAV) is a particularly powerful potential gene therapy vector because AAV integrates into the genome so well. However, the integration of AAV has always been random and so it inherently comes with significant risk.

This is the first time that a treatment for Angelman syndrome has been shown to correct this neurodevelopmental disorder.

Principal investigator, Mark Zylka, professor of Cell Biology and Physiology in the Neuroscience Center, University of North Carolina, Chapel Hill, told BioWorld Science, "The key really from what we can tell is going early in treatment. So for the animals that have the disorder we can identify them with genotyping. If you catch it early, you can treat them one time and it lasts forever as far as we can tell.

That longevity, he said, "contrasts with treatments that are in development using antisense technologies that usually have to be injected every 4 months or so, which is not ideal for a pediatric disorder that will last a lifetime."

Angelman syndrome is caused by loss of function of the maternal Ube3a allele, while the paternal allele is normally silenced by a very long antisense noncoding RNA known as Ube3a-ATS. Previously in a 2011 Nature publication Zylka and collaborators demonstrated that a class of drugs called topoisomerase inhibitors could reactivate the paternal allele by interfering with Ube3a-ATS. So Zylka knew that if the paternal copy of Ube3a can be turned on, this will provide the possibility of treating the condition.

Topoisomerase inhibitors, which include chemotherapy agents such as irinotecan and doxorubicin, are not a therapeutic option for Angelman syndrome due to their broad-spectrum nature and toxicity. But with the development of CRISPR combined with AAV, the researchers have now developed a tool to precisely hone in on specific regions of the genome.

First, the team screened 250 different RNA guided CRISPR/Cas9 constructs in cell culture until they identified the best one (Spjw33) reactivating the Ube3a-ATS allele. These clones had the good fortune to target Snord115 genes within the large Ube3a-ATS locus. The Snord genes are functionally redundant, with over 100 of them present in both mice and humans.

Ultimately the CRISPR/Cas9 with the cloned RNA guide was used to a specific region of the DNA, where DNA was inserted into the Snord115 gene of the Ube3a-ATS locus. The inserted DNA possessed a polyadenylation signal that caused the premature termination of the Ube3a-ATS noncoding RNA such that it no longer silenced the paternal expression of Ube3a.

With the Ube3a now made in the mouse, it fully developed and no longer presented with any phenotypes resembling Angelman syndrome throughout the life of the animal.

In short, instead of deleting the gene, this approach disrupted the Ube3a-ATS gene by stopping its full production prematurely. Only a small nonfunctioning part of the noncoding RNA was still produced in treated animals.

Earlier is better

The broad implications are that the study proves that Angelman syndrome can be treated and possibly prevented, if it is done early enough.

Previous studies showed that if turning on the paternal copy later, even within just a few days after birth in a mouse, this approach does not prevent Angelman syndrome.

Zylka said, "It is like with a building. You want to make sure the foundation is done correctly. Tons of time is put into the foundation. If there is a problem with the foundation, then when building on top of it, it is very hard and next to impossible to go back and fix the foundation. When the brain is developing, it is the initial foundation upon which the brain is built that is critical and you cannot really go back and fix it. So this study now shows that you can fix the problem if you catch it early enough by administering just a single treatment."

One encouraging result was the lack of gene therapy occurring in the mother. The team injected the vector into the fetus, but no gene therapy was detectable in the mother's liver and brain. Instead, the gene therapy was restricted to only the fetus. This was remarkable and very important since AAV is well known to particularly target the liver.

The technology to identify fetuses with the mutation that causes Angelman syndrome is already available and currently used in hospitals around the world. Techniques like amniocentesis, chorionic villus sampling, and even newer noninvasive technologies involving taking extra blood from the mom can now detect fetal DNA and cells to find out if there are any Angelman syndrome mutations.

However, there has not been a strong incentive to look for Angelman syndrome given that there are no therapeutic options at this point.

Zylka hopes to ultimately test the approach in the clinic. But first-time gene therapy technologies are often only given one shot in clinical trials and safety is of primary concern. So, extensive further research will be necessary to not throw away his shot (Wolter, J.M. et al. Nature 2020, Advanced publication).

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Foundational research shows early gene therapy prevents Angelman syndrome - BioWorld Online

Dive Brief:

Sarepta is best known for its RNA technology platform, which has led to two approved though also controversial drugs for Duchenne muscular dystrophy.

Yet, Sareptais also deeply invested in gene therapy, having developed an extensive list of more than two dozen experimental treatments, six of which have reached human testing.

Rare diseases have been an early target in this rapidly growing field. The two gene therapies approved in the U.S., Roche's Luxturna and Novartis' Zolgensma, are respectively used to treat an uncommon form of blindness and a muscle disease that occurs in about 1 in every 10,000 births.

Sarepta'stherapies target a wide variety of rare diseases, including Duchennemuscular dystrophy, Pompe disease, and types of Limb-girdle muscular dystrophy. Taking a stake in AavantiBio, with its work in Friedreich'sataxia, could expand Sarepta'sreach even further. The disease affects approximately 1 in every 40,000 people, according to the National Organization for Rare Disorders, which would equate to around 8,200 patients in the U.S.

"Our equity participation in AavantiBio serves our strategy to build our gene therapy engine through targeted investment in potentially life-enhancing therapies,"Doug Ingram, Sarepta'sCEO, said in a statement Thursday.

AavantiBio joins a couple large, powerful companies in the hunt for a gene therapy to treat Friedreich's ataxia. Pfizer and Novartis are each working on their own programs.

Outside of gene therapy, Reata Pharmaceuticals disclosed last year positive datafrom a study that tested an oral drug, known as omaveloxolone,in patients with Friedreich's ataxia. Reata said it intends to file the drug for approval based on those results.

AavantiBiowill be headquartered in the greater Boston area, putting it close by Cambridge, Massachusetts-based Sarepta. Cumbo, along with his role as CEO, will take one of AavantiBio'seight board of directors seats. Cumbohas, during the span of his career, helped launch 11 specialty products across multiple drug companies, including Sarepta, Gilead and Vertex, where he built a sales team for the hepatitis C drug Incivek.

Co-founders Byrne and Corti will be on the board too, alongside two independent directors and representatives from the investor group of Perceptive, Bain and RA.Louise Rodino-Klapac, senior vice president of gene therapy for Sarepta, will serve as a board observer.

Read more here:
Sarepta, continuing its gene therapy push, helps launch a startup - BioPharma Dive

Some technologies that have emerged and altered the landscape in recent years include immunotherapy, CRISPR-Cas9 gene editing, and chimeric antigen receptor (CAR) T-cell therapies. Now, another platform technology is maturing from the research laboratory to commercial viability. In 2017, the U.S. Food and Drug Administration (FDA) approved the first directly administered gene therapy for mutations of a specific, disease-related gene. That product, Luxturna, marketed by Spark Therapeutics, delivers a functioning copy of the RPE65 gene to retinal cells using an adeno-associated virus (AAV) as a vector to treat a genetic form of blindness.

This advance has injected new energy into biotech startups seeking to capitalize on gene therapy.

As the field matures, gene therapy companies will eventually need to find a way to sell once-in-a-lifetime cures in a market built around chronic therapies. In some ways, its been easier for our European counterparts and other single-payer economies, [research analyst Mani] Foroohar comments. [They] can rest more assured that the financial benefits of reducing future hospitalizations due to expensive future illness will be captured by the same payor thats providing the upfront outlay.

Just as biotechnology has transformed every aspect of our healthcare system over the last 40 years, gene therapy will challenge, disrupt, and overturn our healthcare pricing and reimbursement paradigms as it becomes an increasingly common and routine treatment approach.

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Resurgence of gene therapy has dramatically altered the the biomedicine revolution - Genetic Literacy Project

Paris-based SparingVision, a genomic medicine company focused on ocular diseases, raised 44.5 million (approximately $52.2 million) in a financing round. Funds will be used to advance the development of the companys treatment for a genetic eye disorder that can lead to vision loss.

SparingVision is developing SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa, the most common inherited retinal degeneration that affects about two million people globally. There is currently no approved treatment to treat all genetic forms of this rare retinal disease that leads to blindness, the company said. According to SparingVision, SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting synergistically, aim to slow or stop the degeneration of photoreceptors. Loss of photoreceptors leads to blindness in retinitis pigmentosa. In June, the European Commission granted Orphan Drug designation to SPVN06

In addition to advancing its gene therapy treatment, funds from the financing round will be used to support SparingVisions GMP activities, including the manufacturing of a first clinical batch of the product, as well as regulatory activities. Funds will also be used to begin human trials of the gene therapy, which are set to begin in 2021.

Gene therapy has already been approved by regulatory to approve a type of genetic blindness. Spark Therapeutics, now a division of Roche, won regulatory approval for Luxturna (voretigene neparvovec), a gene therapy for a rare, genetic form of blindness. Luxturna is approved for the treatment of pediatric and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The disease can lead to vision loss and may cause complete blindness in certain patients. The approval marked the first time the U.S. Food and Drug Administration approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene.

In addition to developing its gene therapy for retinitis pigmentosa, SparingVision said it intends to establish a toehold in the United States and will expand its management team.

The financing round was led by 4BIO Capital and UPMC Enterprises. It was supported by Jeito Capital and Ysios Capital. Current investors Bpifrance and Foundation Fighting Blindness also participated in the round. Torreya Capital, LLC served as exclusive placement agent for the offering.

In addition to the financing, Stphane Boissel, who currently serves as chairman of the board of directors, was named chief executive officer of the company. He takes over from Florence Allouche, a cofounder of the company. Boissel, who previously served as head of corporate strategy at Sangamo Therapeutics, said the support SparingVision received in the financing round demonstrates the excitement about the potential of SPVN06.

With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals, Boissel said in a statement.

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SparingVision Nabs 44.5 Million to Support Gene Therapy, Adds New CEO - BioSpace

Luke Timmerman interviews Sana Biotechnology CEO Steve Harr at the GeekWire Summit this week.

Sana Biotechnology CEO Steve Harr shed more light on one of most secretive, heavily funded startups in Seattle and the global biotech industry detailing its plans to create tools that replace and repair human body cells, with the potential to treat various diseases and create new medicines.

Harr spoke with biotechnology journalist Luke Timmerman, founder of The Timmerman Report, this week at the GeekWire Summit. Sana raised more than $700 million this summer in one of the largest venture financing deals in the life sciences industry and one of the biggest rounds on record in Seattle.

Founded in 2019, the 250-person company has an ambitious goal of both repairing cells in the body (gene therapy) and also replacing damaged cells (cell therapy). Its led by several former executives from Juno Therapeutics, another Seattle biotech company that went public in 2014 and sold to Celgene for $9 billion in 2018.

Sana has kept a relatively low profile since launching. It is competing with much larger entities that have deeper pockets and more robust logistics capabilities. But Harr said a startup such as Sana has a key differentiator.

We have one competitive advantage: we can make faster and better decisions, he said. We get there because we have better people, we have greater focus, and we have better communication.

Read on for key takeaways from the conversation.

How Sana started:Harr and his former colleagues at Juno learned a lot about engineering cells and manipulating genes during their startup journey. Juno was among a handful of U.S. companies making cutting-edge cancer immunotherapy treatments.

But they also knew there was more opportunity in a nascent industry of gene and cell therapy.

We wanted to build the transformative or winning company of this next era, of the next 20 years, Harr said. To do that, we had to break the model of what biotech is, which is typically taking an idea and figuring out where to apply it best.

Sana instead is trying to build the platform that can engineer cells and fix them, much like building a computer.

There are a whole host of component parts that go into it, Harr said. We have to aggregate the right technologies.

Harr said too many biotech companies sell solutions in search of problems. He likened it to someone showing up with a tiny screwdriver and looking for a loose screw to fix. Harr sees Sana more as a toolbox that can help build the right medicine for the right patient.

Sana is targeting various disease areas, including cancer, diabetes, genetic disorders, and more. They are relatively diverse, but there are some really fundamental underlying platform and strategy principles that drive each of those, Harr said.

Sanas secret sauce:One key focus for the company is reimagining the delivery system for these therapies how to get DNA, RNA, proteins, etc. into a cell. Ultimately at the core, what were trying to do is really improve delivery, and really figure out how to hide cells from the immune system, Harr said.

Hiding re-engineered or replaced cells from ones immune system is important to prevent the possibility of the body rejecting the new cells.

Harr also talked about delivering therapy via injection, with the body becoming the bioreactor. Its similar to technology built by Moderna and others. You deliver the tools to enable your body to make its own medicine, Harr said.

Manufacturing:Sana is also aiming to innovate how gene and cell therapies are produced and distributed at scale. They are typically expensive Timmerman said CAR T-cell immunotherapies for cancer ran in the $300,000-to-$400,000 range per patient. Figuring out manufacturing costs at scale and making it less than current alternative methods of care for patients will be key to the strength of Sanas business. Harr added that you have to do it in a way thats constructive for the system.

Headcount: Sana employs 250 people spread across offices in Seattle, the Bay Area, and Cambridge, Mass. Having three outposts helps the company attract the best talent, Harr said. One advantage to raising so much capital is being able to hire the best folks. Last month Sana added top scientists Ed Rebar and Terry Fry to the executive team.

If you really hire one of the true world leaders in something, it is pretty amazing how quickly teams form around them, Harr noted.

Money matters: Having more than $700 million in the bank helps Sana in various other ways. Harr said a lot of biotech companies often run what amount to experiments to justify raising more capital. We have the privilege of running experiments to find truth as fast as you want to, he said. And then we want to have the balance sheet, technologies, and people to be able to grapple with whatever the truth is.

Timeline: Harr said the company is on track with its original strategy but does not plan on selling medicine in the next two years. It will progress with multiple medicines in parallel, not one at a time, Harr said.

Leadership advice:During the pandemic and remote work, Harr said hes started to reach out to four-to-six people at Sana each week that he wouldnt normally talk with. He holds half-hour meetings to chat about what they are working on, and what leadership could do to make their life or job better. I found that to be just such an invigorating way to learn whats going on, he said.

[The full interview with Harr, and other GeekWire Summit sessions, are available on-demand exclusively to attendees of the virtual event.Learn more and register here.]

Link:
Sana CEO reveals details about stealthy gene therapy startup that has raised more than $700M - GeekWire

NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that it will host a virtual R&D Day on Friday, October 30, 2020 at 11:30 AM Eastern time, to discuss the Companys AXO-Lenti-PD gene therapy for Parkinsons disease.

Axovants Parkinsons disease R&D Day will be moderated by Chief R&D Officer, Gavin Corcoran, M.D., and will feature presentations on the current treatment landscape and unmet medical need for people living with Parkinsons disease from the following key opinion leaders:

In addition, the Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including:

Drs. Adler, Palfi, and Eberling will be joined by Dr. Corcoran to answer questions following the formal presentations.

AXO-Lenti-PD is the only investigational gene therapy for Parkinsons disease that delivers three genes via a lentiviral vector to encode a set of critical enzymes required for endogenous dopamine synthesis, with the goal of improving motor function and restoring steady, tonic levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

To register for the R&D webcast, please click here.

A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.

Biographies of R&D Day Panelists:

Dr. Adler has received numerous grants to investigate experimental treatments for Parkinson's disease, essential tremor, dystonia, restless legs syndrome, and chronic traumatic encephalopathy (CTE). He serves as an advisory member to many different international medical societies such as the International Parkinson and Movement Disorder Society, MDS Industry Education and Services Committee, and the American Academy of Neurology Section of Movement Disorders. Dr. Adler has a commitment to education having trained residents, 14 fellows and graduate students, and has given many invited lectures. Dr. Adlers main research interests are investigating tissue diagnostic tests for Parkinsons disease, biomarkers for an early diagnosis of Parkinsons disease and PD with dementia, and identification of new treatments for PD and PD with dementia. He also has been investigating essential tremor, restless legs syndrome, and dystonia. He has published over 400 research papers and reviews, and edited a book entitled Parkinson's Disease and Movement Disorders: Diagnosis and Treatment Guidelines for the Practicing Physician. In 2006, Dr. Adler was awarded the Mayo Clinic Distinguished Investigator of the Year Award.

Dr. Palfi has published extensively on trophic factor- and enzyme-based gene therapy in Parkinsons disease and Huntingtons disease. He is a principal investigator on numerous preclinical and clinical studies and has been involved in studies of many novel agents including implanted brain devices, optogenetic, homeoprotein, trophic factors GDNF, CNTF and dopamine lentiviral vectors.

Dr. Eberling earned undergraduate and graduate degrees in biological psychology from the University of California at Berkeley, later moving to the Lawrence Berkeley National Laboratory where she developed expertise in neuroimaging techniques and gene therapy approaches for Parkinsons disease.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523investors@axovant.com

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.commedia@axovant.com

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Axovant Gene Therapies to Host Virtual Parkinson's Disease R&D Day on October 30, 2020 - BioSpace

Biogen is cutting its experimental multiple sclerosis (MS) drug opicinumab as well as an spinal muscular atrophy (SMA) candidate amid a third-quarter clear-out.

First, to its MS drug. In its third-quarter financials posted Wednesday morning, the biopharma said: In October 2020 Biogen announced that the phase 2 AFFINITY study of opicinumab in MS did not meet its primary or secondary endpoints and that Biogen has discontinued development of opicinumab.

The anti-LINGO monoclonal antibody has experience with failure: All the way back in 2016, it posted a similar phase 2 flop after not hitting its primary and secondary endpoints for MS in the so-called SYNERGY trial.

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Back then, opicinumab was seeing whether it could have an effect on a multicomponent primary endpoint designed to assess ambulation, upper extremity function and physical disability in patients with relapsing forms of MS. Opicinumab, however, failed to outperform placebo in this regard.

A secondary efficacy endpoint, intended to evaluate the slowing of disability progression, also came up negative. But Biogen dug through the data, and, instead of culling it then and there, decided to carry on, saying: While we missed the primary endpoint, the SYNERGY study results suggest evidence of a clinical effect of opicinumab.

RELATED: Biogen tumbles on PhII MS flop, but refuses to write off drug

Biogen believed the data showed an increased clinical effect of opicinumab versus placebo (when used at the same time as interferon beta-1a intramuscular injection).

That led to the AFFINITY study, started in 2017, which looked at opicinumab as an add-on therapy in patients who are adequately controlled on their anti-inflammatory disease-modifying therapy (DMT), versus the DMT alone.

The primary endpoint of the study, overall response score, looked to assess the improvement and worsening of disability over time. Clearly, it failed to achieve this, though in its third-quarter update it did not reveal further details.

It also quietly announced it was throwing out BIIB089, an SMA gene therapy hopeful that had been on an IND hold due to dorsal root ganglion toxicity. Again, it did not any extra color to this cull in its financials.

The biopharma is now pinning its hopes on another failed drug, aducanumab, which it hopes can get past an FDA advisory committee early next month and a potential approval next year, though this controversial Alzheimers disease asset has been given low odds by analysts of managing that feat.

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Biogen finally culls MS dud opicinumab, adds SMA gene therapy to the garbage heap - FierceBiotech

WATERTOWN, Mass. and RESEARCH TRIANGLE PARK, N.C., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations. The FDA grants Rare Pediatric Disease Designation to incentivize development of new treatments for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the U.S. The Rare Pediatric Disease designation program allows for a Sponsor who receives an approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

This Rare Pediatric Disease designation from the FDA highlights the significant unmet medical need that Selecta and AskBio are seeking to address with MMA-101 for this rare metabolic disorder, said Carsten Brunn, Ph.D., chief executive officer of Selecta Biosciences. When used with AAV gene therapy vectors, Selectas ImmTOR aims to inhibit the immune response to the AAV vector, potentially allowing re-dosing of gene therapies. Ongoing clinical programs will focus on evaluating product candidate performance in patients who may have been underdosed or those who may lose transgene expression over time. Were honored to receive this recognition and look forward to advancing this program in hopes of helping young patients affected by MMA and their families.

MMA is a serious and potentially life-threatening inherited metabolic disorder that presents in patients from newborns to adulthood, said Sheila Mikhail, J.D., CEO and co-founder of AskBio. AskBio is committed to delivering transformative genetic medicines for rare diseases like this one, and the Rare Pediatric Disease designation helps us continue development of MMA-101.

AskBio and Selecta expect to initiate a Phase 1 clinical trial of MMA-101 and ImmTOR for patients with MMA in 1H 2021.

About Methylmalonic AcidemiaMethylmalonic Acidemia (MMA) is a rare monogenic disorder in which the body cannot break down certain proteins and fats. This metabolic disease may lead to hyperammonemia and is associated with long-term complications including feeding problems, intellectual disability, chronic kidney disease and inflammation of the pancreas. Symptoms of MMA usually appear in early infancy and vary from mild to life-threatening. Without treatment, this disorder can lead to coma and in some cases death.

About Selecta Biosciences, Inc.Selecta Biosciences, Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the bodys natural self-tolerance in autoimmune diseases. The companys first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit http://www.selectabio.com. About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. Its pipeline includes clinical-stage programs in Pompe disease and congestive heart failure and a diverse preclinical portfolio of therapeutics targeting neuromuscular, CNS and other diseases, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics, acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics, acquired by Pfizer). AskBios gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the company), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy and MMA, the companys plans to initiate a clinical trial for a product candidate to treat MMA, the ability of the company and AskBio to develop gene therapy products using ImmTOR and AskBios technology, any development plans of the company and AskBio have for product candidates to treat serious and life-threatening diseases and the intention to seek regulatory approval thereof, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the companys intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AskBio Forward-Looking StatementsThis press release contains forward-looking statements regarding AskBio. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans, expects, will, intends, potential, possible and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding MMA-101, including the potential timing of the Phase 1 clinical trial for patients with MMA, AskBios pipeline of development candidates; AskBios goal of developing life-saving medicines aimed at curing genetic diseases; the potential benefits of AskBios development candidates to patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond AskBios control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

Any of the foregoing risks could materially and adversely affect AskBios business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

For more information please contact:

Selecta: For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

For Media: Meredith Sosulski, Ph.D.LifeSci Communications, LLC+1-929-469-3851msosulski@lifescicomms.com

AskBio:Robin FastenauVice President, Communications+1-984-275-2705rfastenau@askbio.com

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Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia - GlobeNewswire

In anticipation of our Pfizer Gene Therapy Career Event scheduled for Wednesday, October 28, 2020, BioSpace spoke with Alison Ricci, Pfizers Senior Business HR Partner- Global Sciences, Business HR for Bioprocess Research and Development, and HR Site Lead for the companys four locations in Chapel Hill and Morrisville, North Carolina, Chesterfield, Missouri and Lake Forest, Illinois.

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. This is in addition to the companys continuing investment into gene therapy research and development and other areas going on throughout the company, but specifically in its North Carolina sites in Chapel Hill and Kit Creek. The companys presence in North Carolina currently exceeds 3,600 people, with 650 in Sanford. The expanded facility is expected to add over 100 new jobs.

Ricci told BioSpace the company has been investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area. They have three facilities in and around the area that focus specifically on gene therapy, including a research facility in Morrisville, its existing clinical manufacturing site in Chapel Hill, and a very large manufacturing site in Sanford.

And in recognition of the continued investment and commitment to gene therapy, the company acquired a 60,000-square-feet building and 16 acres in Durham, which they are renovating into a clinical manufacturing site, essentially doubling our clinical manufacturing capacity.

*Rendering of Durham location

As such, the company has numerous positions in the area currently open with many more expected as the new site comes online.

Right now, we have 30 positions that were going to be advertising for that directly support gene therapy in those three different campuses, Ricci said.

The roles they will be looking to fill include what Ricci calls a multitude of individual contributor, team lead and management roles."

"For instance, weve got our R&D and analytical scientist roles; we have manufacturing engineering roles; we have technical specialist roles; and different types of manufacturing support and technology transfer roles, Ricci said.

They also have training specialist positions because there are a variety of supporting functions that go into the magic of making gene therapy work.

Ricci seemed excited about gene therapy and the part it is playing increasingly in Pfizers core mission. Speaking of Duchenne muscular dystrophy (DMD), increasingly a target of gene therapies, she noted how exciting and important it is to be able to provide a therapy that extends quality of life, that doesnt just treat symptoms, but that potentially marks a minimization of those symptoms and the elongation of life. Thats one of the central core components of who Pfizer is. Our patients are our North Star.

She also emphasized not only the science and motivation of Pfizer as an employer, but its culture.

People typically equate large organizations with a level of bureaucracy, that results in less risk and less innovation opportunities, Ricci said. In my five years with Pfizer Ive seen the culture shift to focus on innovation, inclusion, quality and smart resourcing figuring out how to free up resources so we can focus on bold moves and exciting new therapies allowing our scientists to stretch and grow, and make a difference for the patients that count on us.

The company has also structured rewards and recognition around those types of approaches, not only for people theyre bringing into the organization, but with its current staff.

Were still maintaining our focus on a combination of productivity and doing it right the first time while encouraging innovation, taking bold moves and trying new things," Ricci said. "Because thats the best way that good science is done, and patients win in the process.

She also notes that the Pfizer Gene Therapy Career Event is a great opportunity to have direct contact not only with Pfizer hiring managers, but with colleagues who are working in those same roles theyre hiring for.

Its a unique experience in terms of really understanding who Pfizer is and what we have to offer, Ricci said.

And, of course, the company is hiring throughout its worldwide operations in support of not only its COVID-19 clinical research trials and manufacturing processes, but in the numerous other areas the company focuses on.

The expansion in Sanford, North Carolina is only a part of the companys focus on building out capacity. It has committed about $5 billion in U.S.-based capital projects over the next several years.

But the North Carolina area is expanding end-to-end capabilities in gene therapy. The Kit Creek facility focuses on small scale production, from 2L flasks up to 250L bioreactors to develop the early processes that will later be used in larger scale manufacturing. The Chapel Hill facility is where the process is optimized, and staffers work at a 250L scale while developing and implementing quality control measures that include Good Manufacturing Practice (GMP) standards. The Sanford facility is designed for high-quality, efficient supply of gene therapies at clinical- and commercial-scale.

Ricci also points to Paul Mensah, Pfizers Vice President of Bioprocess Research and Development.

He has a nice, pragmatic style and approach in terms of how he continues to foster and leverage innovation, but also the science of how everyone works together to collaborate and deliver. He instills that in the leaders on the team. Ricci said.

Were continuing to focus in terms of career development and the aspirations of each colleague. There are opportunities for colleagues at Pfizer to do anything they want within the realms of the organization." Ricci said. "You can be on the frontlines of early stage development, you can work on the late stage, you can have opportunities for clinical manufacturing. You can run the gamut in terms of different roles with the scientific profile within Pfizer.

Originally posted here:
Pfizer: Expanding North Carolina Gene Therapy Facility and Adding Jobs - BioSpace

Irvine, Calif., October 19, 2020 A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing, illustrates the use of a new generation CRISPR technology and lays the foundation for the development of a new therapeutic modality for a wide range of inherited ocular diseases caused by different gene mutations.

In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function, said Krzysztof Palczewski, PhD, the Irving H. Leopold chair and a distinguished professor in the Gavin Herbert Eye Institute, Department of Ophthalmology at the UCI School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, said first author Susie Suh, assistant specialist in the UCI School of Medicine Department of Ophthalmology. We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, said co-first author Elliot Choi, also an assistant specialist in the UCI Department of Ophthalmology.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the UCI team successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases. Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu, PhD.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast and spatial and temporal frequency, said Palczewski. These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases.

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy. Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

This research was supported in part by grants from the National Institutes of Health, the Research to Prevent Blindness Stein Innovation Award, Fight for Sight, the Eye and Tissue Bank Foundation (Finland), The Finnish Cultural Foundation, the Orion Research Foundation, the Helen Hay Whitney Foundation, US Department of Veterans Affairs, and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

About the University of California, Irvine: Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visit http://www.uci.edu.

Media access: Radio programs/stations may, for a fee, use an on-campus ISDN line to interview UCI faculty and experts, subject to availability and university approval. For more UCI news, visit news.uci.edu. Additional resources for journalists may be found at communications.uci.edu/for-journalists.

About the UCI School of Medicine: Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI-led study reveals significant restoration of retinal and visual function following gene therapy - UCI News

MALVERN, Pa., Oct. 22, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen, will speak on the importance of strategic partnerships in gene therapy including product consistency, identifying the right partner as well as providing examples of successful partnerships at the 4th Annual Gene Therapy for Rare Disorders Europe Conference from October 26-28, 2020 presented digitally.

Presentation Details:Title:The Importance of Strategic Partnerships in Gene TherapyDate: Wednesday, October 28, 2020Time: 11:00 AM (GMT)Location: DigitalRegister: https://genetherapy-europe.com/take-part/register/

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact: LaVoieHealthScience Katie Gallagherkgallagher@lavoiehealthscience.com+1 617-792-3937

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CEO Dr. Shankar Musunuri to Discuss the Importance of Gene Therapy Strategic Partnerships at 4th Annual Gene Therapy for Rare Disorders Europe...