StemCell Therapy

When it comes to advancing gene therapy products in the United States and Europe, the key is early consultation with regulators and identification of potential problems, experts said at the Euro Convergence 2020 virtual meeting.

It is better to do it earlier and establish a collaborative and educational approach to discussions, advised Mridula Shukla, director of global regulatory affairs at Arcutis Biotherapeutics in Palo Alto, Calif. Patrick Celis, PhD, head of the scientific secretariat of the Committee for Advanced Therapies at the European Medicines Agency (EMA), echoed that advice.

Please talk to EMA and National Authorities early and frequently so that we can help you as much as possible to get these innovative products to the patients as soon as possible, Celis said.

There are currently more than 10 gene therapy products approved globally and momentum has been building in this area since around 2015, Shukla said.

Gene therapy seems to have potential as it offers the possibility of an actual cure instead of chronic treatment for rare diseases, hereditary diseases and cancer, Shukla said.

Despite the promise of the therapies, more than half of gene therapy clinical trials are in phase 1. Shukla said this is due to a combination of factors including the complexity of the therapies, the rare indications, the high price of the therapies and reimbursement issues, unique manufacturing and supply chain challenges and controversies surrounding the ethnics of the techniques.

There is guidance from the US Food and Drug Administration (FDA) to assist developers. In July 2018, FDA issued draft guidance on Human Gene Therapy for Rare Diseases, which was finalized in January 2020. The document offers advice on some common manufacturing challenges, including encouraging companies to establish critical process parameters (CPP) early on and establish and qualify a potency assay prior to conducting clinical trials.

The FDA recommends communication with its Office of Tissues and Advanced Therapies early in product development, even before submission of an investigational new drug (IND) application. The agency also offers an Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) meeting, which can be used to discuss specific product issues, Shukla said.

Overall, the recommended approach is to front-load as much development as possible, preferably prior to Phase 1, and to hold early interactions and frequent interactions with FDA or EMA, Shukla said.

In the US, there are also multiple options for expedited review of products, including:

These expedited processes are similar to programs available for advanced therapy medicinal products (ATMPs) in Europe, such as the priority medicines (PRIME) scheme, which offers early consultation and the potential for accelerated assessment.

Since 2009, the EMA has approved 17 ATMPs, of which 10 are gene therapy products, Celis said.

The common issues that come up with ATMP applications include a lack of experience with commercial manufacturing processes, finding a relevant potency assay, ensuring product consistency and challenges with consistent starting materials, according to Celis. Other problems that can lead to delays in authorization include a lack of randomization in trial design, an indication that doesnt reflect the patients in the trial, and limited data on safety and durability of response.

The nature of gene therapies and the small number of patients in clinical trials mean that sponsors often have difficulty showing long-term safety and efficacy in a robust patient population. While regulators dont expect the same level of data as with other traditional medicines, there is a need for early planning of post-authorization studies, Celis said.

The EMA is asking for a detailed post-authorization plan that can include follow-up of patients in the clinical trials, post-marketing trials, and post-authorization registry-based studies.

If a sponsor is using a registry, Celis said it is better to go with an established disease registry and to contact the registry early to find out what data is collected. Additionally, registries may not be a good basis for safety reporting, Celis said.

There are support mechanisms in place for ATMP developers including scientific advice, a meeting with the EMAs Innovation Task Force, and the PRIME program for medicines addressing unmet needs.

We can help, Celis said. Make sure that you make best use of what we can do.

For sponsors looking to market gene therapies globally, Celis said that the EMA and FDA are in frequent contact and are seeing many of the same issues as they review products. He encouraged developers to make use of this interaction between European and US regulators either formally, through parallel scientific advice, or informally.

Euro Convergence 2020

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Euro Convergence: Early consultation critical in overcoming gene therapy hurdles - Regulatory Focus

Pharmaceutical companies with gene therapies in their pipeline need to establish scalable production processes that can keep up with the increasing demand in terms of volume, quality and robustness, says CEVEC.

Gene therapies are seen as one of the most promising approach to treat life-threatening diseases with no therapeutic options and high medical needs. In addition, gene therapies are now advancing also into indications with larger numbers of patients, including Alzheimers and Parkinsons, thereby dramatically increasing the demand for viral vectors. As a result, pharmaceutical companies with gene therapies in their pipeline need to establish scalable production processes that can keep up with the increasing demand in terms of volume, quality and robustness.

CEVEC Pharmaceuticals, a German biotechnology company focusing on viral vector technologies, has developed and commercially launched ELEVECTA, a unique technology that enables fully scalable AAV manufacturing providing unprecedented process robustness and consistency in vector yield and quality.

One of the major bottlenecks in AAV vector production is volumes. Standard technologies have proven to be very hard or impossible to scale, as they are often based on adherent cell lines or require transient transfections or helper viruses.

While suspension cell lines are getting more and more common now, most processes still rely on transient transfection to bring the different AAV genes into the producer cells. The typical transient transfection process is complex and requires three different GMP-grade plasmids driving up the production costs. This is where ELEVECTA stableAAV producer cell lines come into play.

Figure 1: ELEVECTA, a revolutionary technology that enables fully scalable, stable viral vector manufacturing with unprecedented process robustness and consistency in vector yield and quality.

Based on a stable human suspension cell line, ELEVECTA enables full scalability in all types and formats of suspension bioreactors, from laboratory to commercial scale. ELEVECTA Producer Cell Lines reach titers of 1*1014 vg/l and more during a typical single-use suspension bioreactor run. CEVEC has now successfully scaled-up from 15 ml to 50 L bioreactors in a single step without any productivity loss, demonstrating the scalability and robustness of the process.

ELEVECTA Producer Cells contain all components required for AAV stably integrated into the cell. There is no need for cGMP plasmids, special transfection reagents and difficult-to-scale-up transfection protocols. Just one cell line which, after propagation and expansion to the desired cell titers, produces AAV vectors at high yields a process that resembles well-established production methods for recombinant proteins and monoclonal antibodies.

Figure 2: Cryo TEM picture from AAV particles derived from ELEVECTA Producer Cell Lines. The viral particles display an approximate size of 22 nm. Full particles are indicated by a black arrow, empty particles by a white arrow

AAV vectors can have different surface structures, so called serotypes, which in nature allow the virus to target specific cells in a body. For gene therapy applications viral serotypes can further be engineered to improve their tissue-specificity and safety. Every ELEVECTA producer cell line is custom-made and employs a specific natural or engineered capsid gene and the therapeutic gene of interest.

With this newly introduced technology, CEVEC is the leading technology provider for stable AAV producer cell lines. Its inherent scalability, process robustness and consistency in yield and quality provide the features that are needed to pave the way for the success of modern gene therapies in a broad range of indications.

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ELEVECTA for AAV gene therapy vector production - Bioprocess Insider - BioProcess Insider

- By Barry Cohen

Novartis AG (NYSE:NVS) is expanding its gene therapy footprint by acquiring a venture capital-backed company focusing on treating vision loss that can lead to blindness.

The Swiss pharmaceutical giant will pay shareholders of Vedere Bio $150 million upfront, which could be boosted by $130 million more if the Cambridge, Massachusetts-based company's programs achieve several development milestones.

Novartis has made huge strides aimed at beefing up its gene therapy program. In 2018, the company bought AveXis for $8.7 billion, inheriting its spinal muscular atrophy therapy Zolgensma, and this year established partnerships with Dyno Therapeutics and Sangamo Therapeutics Inc. (NASDAQ:SGMO).

Vedere is trying to take naturally occurring, light-sensing proteins and use a special gene therapy method to inject them into a certain part of the eye. Once there, they will be sent to retinal cells that are still healthy to help improve the vision of patients, according to an article in BioPharma Dive.

Novartis thinks this approach has much broader uses. The company said the proteins, in conjunction with Vedere's delivery tools, could "vastly expand" the number of treatable patients with vision loss from photoreceptor death.

Investors should keep in mind that the Vedere treatments are still in the pre-clinical stage so a great deal of development still needs to be done. Working with the acquisition's scientists, Novartis is preparing to promptly begin human testing.

There could be an even bigger population the Vedere platform could address, beyond inherited eye disorders. That would be a new way to treat geographic atrophy, one of the more advanced forms of an age-related vision loss called dry age-related macular degeneration. The American Academy of Ophthalmology notes an estimated 15 million people in North America have AMD, the vast majority with the dry form.

Story continues

Following the close of the deal, Vedere Bio II will operate as a wholly independent entity from Novartis and Vedere Bio.

Novartis decided to get out of one area of the eye-care business when it spun off Alcon Inc. (NYSE:ALC) last year. The company said it wanted to concentrate on treating diseases of the eye, not eye-care products.

Novartis' eye drugs include Xidra, treating dry eye disease, and Beovu, which was recently approved for the wet form of AMD. Its third eye drug and the biggest producer is Lucentis, which had sales of $515 million in the third quarter. Xidra and Beovu combined to bring in revenue of about $151 million. Novartis' biggest competitor is the Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) drug Eylea, a multibillion-dollar annual seller.

The Novartis faithful are hoping for better days ahead. At about $90, the company's shares are pretty much where they were five years ago. On the plus side, its dividend yields nearly 4%.

Despite the stock being stuck in neutral, Wall Street seems to think Novartis is a good investment, with three recent buy ratings and just one hold. Analysts give it an average target price of more than $106, according to TipRanks

UBS analyst Laura Sutcliffe last month upgraded the stock to buy from hold, calling its valuation "compelling." She thinks new products in the pipeline will boost the company's annual earnings growth to a healthy 9%.

Disclosure: The author has no positions in any of the stocks mentioned in this article.

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Novartis Expands Presence in Gene Therapy With Acquisition - Yahoo Finance

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.

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Sloan Kettering Institute, Bluebird Bio in court trial over gene therapy deal - Crain's Chicago Business

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.

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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...

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

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

Early in the coronavirus pandemic, management at the North Hill Retirement Community a sprawling high-end campus for older adults in suburban Boston kept a wary eye on how the virus was sweeping through similar facilities with devastating, deadly consequences.

It took precautions to protect its residents, and when the order came down that by mid-July, all of its 400 staff members had to be tested for the virus every week, administrators scanned a list of approved tests provided by the Massachusetts health department and selected a vendor a Boston-based DNA lab named Orig3n that had developed its own test for the novel coronavirus.

North Hill was soon to learn, not all Covid-19 tests are created equal. In a vast, confusing landscape populated by hundreds of different private companies and labs, some tests are proving problematic, especially when used to screen people who have no symptoms of Covid-19. A STAT investigation found that top officials at the U.S. Department of Health and Human Services ended already-minimal oversight of these so-called laboratory developed tests in mid-August, despite being informed that the tests were plagued with quality issues.

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It was just two weeks earlier that North Hills administrators, after going months with only one positive test for Covid-19, had been informed that 19 staff members tested positive for the virus at the same time.

It was really out of the blue, said Ted Owen, North Hill president and CEO. The numbers looked strange to us, that suddenly there would be this big thing.

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State public health investigators were called in to verify the test results, and meanwhile, Owens said, There was a week of absolute pandemonium. Staffers were sent home to nervous families, waiting for symptoms to emerge, while residents and their families waited to see if the virus had spread among the vulnerable, older population of North Hill. In the end, the problem was not a viral outbreak but a testing issue. All 19 of the results were false positives; none of the staffers actually had Covid-19.

North Hills false positives were not isolated. A state investigation in August found 383 false coronavirus positives in that period from Orig3n tests, which had been used by about 60 nursing homes in the state. The state health department suspended Orig3ns testing, demanding the company address contamination and documentation issues. Orig3n told STAT it faults human error for its testing problems.

The Orig3n test is just one of dozens that hospitals, university researchers, and private labs rushed to make as the pandemic spread across the U.S. All these laboratory developed tests, known as LDTs, use the same basic PCR technology to identify genetic material from the coronavirus in patient swabs, but their performance can vary widely. They are being used across the country by states, private employers, sports leagues, and schools to try to manage the pandemic, yet they are something of a black box. Because they fall into a regulatory gray area, nobody outside the labs themselves knows how accurate they are not even the health care providers who use them.

Nor is it clear how many LDTs for Covid-19 are on the market. The Food and Drug Administration doesnt know for sure, but acknowledges the number is not small; the agency has granted emergency authorization to more than 200 diagnostic tests, and LDTs may account for more than half of tests in use, an FDA official estimated. These lab PCR tests are essentially the same as ones sold by manufacturers like Abbott and Roche, but theyre regulated differently and are exempt from oversight by the FDA.

In February, as the coronavirus was starting to spread across the U.S., the FDA began requiring these labs to apply for an emergency use authorization, as it had in previous public health emergencies like the H1N1 flu pandemic in 2009. The bar was set low to get tests on the market fast, labs were allowed to start using them without waiting for FDA sign-off and the standards for approval were far less than would normally be required. Labs merely had to show reasonable accuracy detecting the virus on contrived test samples that werent obtained from actual patients.

Then this summer, the Department of Health and Human Services directed the FDA to stop requiring emergency authorization for this segment of the testing market. The Centers for Medicare and Medicaid Services retained oversight of the labs, but it ensures only that labs meet quality standards, not that specific tests work. The decision meant the FDA has no way to track problems with Covid-19 LDTs including false-positive or false-negative results or even which labs are marketing these tests.

An HHS spokesperson said the decision was made for purely legal reasons, based on its Office of General Counsels determination that the FDA does not have jurisdiction over lab-developed tests.

This deregulatory action ensures compliance with law, is responsive to multiple Trump Administration Executive Orders and better prepares us for future pandemics while maintaining regulatory safeguards for quality and accuracy, HHS chief of staff Brian Harrison said in a statement.

But STAT has learned that before the decision, HHS and its general counsel, Robert Charrow, were alerted to major concerns about many of the LDTs for detecting the coronavirus, which had been identified during the FDAs review process.

Another FDA official, speaking on background, told STAT that data on problems with LDT accuracy and performance were sent by the FDA to HHS at the beginning of August, and were seen by Charrow before the decision to revoke FDA authority over LDTs, announced on Aug. 20. This officialwas on multiple phone calls with FDA colleagues and scientists in which the submission of data to HHS was discussed, including with people who had gathered and compiled the data and who were expressing extreme frustration with HHS for, as they described it, suppressing the data.

The data became public in a September article in the New England Journal of Medicine, in which two FDA officials outlined how the emergency use authorization process enables the agency to track the safety and problems with tests, but HHS earlier knowledge of the data has not been previously reported.

In the first months of the pandemic and testing rollouts, the FDA analyzed 125 EUA requests from laboratories, and found design or validation problems with 82, while several have been denied authorization, wrote FDAs Jeffrey Shuren, director of the Center for Devices and Radiological Health, and Timothy Stenzel, director of the FDA Office of In Vitro Diagnostics and Radiological Health. In most cases, the EUA process allowed the agency to work with labs to correct problems, they noted.

No test is 100 percent accurate and performance can vary within populations, they wrote. Covid-19 diagnostic tests may be less accurate in asymptomatic or low-risk populations and in persons who shed little virus or are early or late in the course of illness.

In a later interview, Shuren said the FDAs oversight role is critical in monitoring tests for safety and accuracy and keeping problematic tests off the market.

The value of FDA oversight of tests is that we are able to review and rely on the science to better understand whether or not a product works and how well it works, he said. If were not reviewing something, we cant do that.

Responding to criticism of the HHS decision, Charrow, the general counsel, said in a statement,It is false to say that this will lead to unregulated, low-quality COVID-19 tests. Every single COVID-19 test in the United States will still be regulated at the federal level. This simply removes an additional regulatory barrier that at most other times is not required.

Alberto Gutierrez, former director of the FDA Office of In Vitro Diagnostics and Radiological Health, who was with the agency for 25 years, said the current chaos in testing could have been prevented if the FDA and HHS were working together to track problems and safety and get the best tests to market.

The problem with LDTs is there are a lot of different laboratories and there are no real standards because the tests are developed by the laboratories, he said. Theres no bar to meet. You have very good laboratories that are very thorough, and you have some that cut corners.

In early October, three Democratic members of the U.S. House Reps. Frank Pallone Jr. of New Jersey, the chairman of the Energy and Commerce Committee, Anna Eshoo of California, and Diana DeGette of Colorado wrote to HHS Secretary Alex Azar, demanding answers about the agencys decision to end FDA oversight and tracking of lab-developed tests. During the Covid-19 pandemic, they said, FDA jurisdiction over LDTs is warranted because as former FDA Commissioner Scott Gottlieb recently wrote, in public health emergencies, diagnostic tests not only diagnose diseases that arent completely understood, but help guide analyses of disease progression and risks to public health.

The testing company at the center of this summers debacle in Massachusetts where it was also deployed to test people who are homeless says it is working to improve its Covid-19 test, which remains off the market there and in other states where it was used, including North Carolina. Weve taken extraordinary steps to work through this challenging time and hope to be able to bring our capacity carefully back online to help address this pandemic, said Orig3n CEO Robin Smith.

Orig3n was granted emergency authorization in April, before HHS dropped the requirement. Under an EUA, test makers are supposed to report to the FDA when things go wrong including false results. But few do. A review of the agencys public database for testing issues shows only 657 reports related to Covid-19 diagnostic tests as of Sept. 30, out of more than 100 million tests performed by that point. This means the vast majority of false Covid-19 results including all of the Orig3n testing errors are simply never reported.

The FDA acknowledges underreporting is rampant, limiting the databases usefulness for spotting problematic tests. Especially for LDTs, it relies on essentially an honor system, expecting labs to report on themselves. Without better tracking and oversight from the federal government, some agency officials told STAT, they fear testing problems will continue as the pandemic worsens through the fall and winter, with patients and medical providers flying blind on which tests can be trusted.

More:
HHS eased oversight of Covid-19 tests though it knew of problems - STAT

Global Cell Banking Outsourcing Market: Overview

The global cell banking outsourcing market is likely to be driven by the rising demand for biopharmaceutical production targeting novel active sites, stem cell therapy, and gene therapy. A cell bank is a facility storing cells extracted from various organ tissue and body fluids so as to cater to the needs of the future. The cell banks make storage of cells with an elaborate characterization of the entire cell line as it reduces the possibilities of cross contamination. These benefits are estimated to fuel expansion of the global cell banking outsourcing market over the timeframe of assessment, from 2020 to 2030.

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Cell banking outsourcing industries engage testing, characterization, storage, and collection of tissues, cell lines, and the cells. These activities are done to assist in the production of biopharmaceuticals and in the research and development activities so as to ensure minimum adverse effects and high effectiveness. The procedure of the cell storage involves first proliferation of cells, which then multiplies in a huge number of identical cells and is then put inside cryovials safety for use in future. Cells are primarily utilized in the production of regenerative medicine. A surge in the number of cell banks together with the high demand for stem cell therapies is likely to work in favor of the global cell banking outsourcing market over the tenure of analysis, from 2020 to2030.

The global cell banking outsourcing market has been segmented on the basis of four important parameters, which are bank type, phase, cell type, and region.

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Global Cell Banking Outsourcing Market: Notable Developments

The global cell banking outsourcing market is considered a fairly competitive market and is marked with the presence of many leading market players. The companies in this market are forging mergers, partnerships, and collaborations so as to gain larger revenue and market share. The following development is expected to play an important role in the market:

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Global Cell Banking Outsourcing Market: Key Trends

The global cell banking outsourcing market is characterized by the presence of the following restraints, drivers, and opportunities.

High Demand for Stem Cell Therapies to Trigger Growth of the Market

The rising number of stem cell therapies across the globe primarily influences the global cell banking outsourcing market. According to a survey conducted by World Network for Blood and Marrow Transplantation (WBMN), nearly 1 million hematopoietic stem cell transplantation processes were conducted in between 2006 to 2014. These figure comprised removal of stem cells procedures from peripheral blood or bone marrow, proliferating, and then finally storing them cell banks for future use by patients. Stem cell therapies are able to multiple disease, such as amyotrophic lateral sclerosis, type 1 diabetes, cancer, Alzheimer's disease, Parkinson's disease, and so on. Ability to cure such a wide variety of diseases is expected to propel growth of the global cell banking outsourcing market in the years to come.

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Global Cell Banking Outsourcing Market: Geographical Analysis

North America is expected to dominate the global cell banking outsourcing market throughout the timeframe of analysis, from 2020 to 2030. Such high growth of the North America market is ascribed to the increased production of antibiotics, therapeutics protein, and vaccines. In addition, presence of several biopharmaceutical companies in the region is anticipated to foster growth of the cell banking outsourcing market in North America in the near future.

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Cell Banking Outsourcing Market: High Demand for Stem Cell Therapies to Trigger Growth of the Market - BioSpace

AllTheResearchs published a research report on the Canine Stem Cell Therapy market, which represents a study for the period from 2020 to 2026. The research provides a near look at the market scenario and dynamics impacting its growth. This report highlights the crucial developments along with other events happening in the market which are marking on the growth and opening doors for future growth in the coming years. Additionally, the report is built based on the macro- and micro-economic factors and historical data that can influence the growth.

The global Canine Stem Cell Therapy market was valued at US$ 118.5 Mn in 2018 and is expected to reach US$ 240.7 Mn in 2026, growing at a CAGR of 9.3% during the forecast period.

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Key Players:

The global Canine Stem Cell Therapy market has been comprehensively analyzed and the different companies that occupy a large percentage of the market share in the regions mentioned have been listed out in the report. Industry trends that are popular and are causing a resurgence in the market growth are identified. A strategic profile of the companies is also carried out to identify the various subsidiaries that they own in the different regions and who are responsible for daily operations in these regions.

The Key Players Covered in Canine Stem Cell Therapy Market Study are:

How Report will help you to make decisions for business:

Before purchasing the report, lets discuss with the analyst @ https://www.alltheresearch.com/speak-to-analyst/206

Canine Stem Cell Therapy Market Segmentation

Canine Stem Cell Therapy market is split by Type and by Application. For the period 2015-2026, the growth among segments provide accurate calculations and forecasts for sales by Type and by Application in terms of volume and value. This analysis can help you expand your business by targeting qualified niche markets.

Market Segmentation by Type:

Market Segmentation by Applications:

COVID-19 Impact on Canine Stem Cell Therapy Market:

The outbreak of COVID-19 has brought along a global recession, which has impacted several industries. Along with this impact COVID Pandemic has also generated few new business opportunities for Canine Stem Cell Therapy Market. Overall competitive landscape and market dynamics of Canine Stem Cell Therapy has been disrupted due to this pandemic. All these disruptions and impacts has been analysed quantifiably in this report, which is backed by market trends, events and revenue shift analysis. COVID impact analysis also covers strategic adjustments for Tier 1, 2 and 3 players of Canine Stem Cell Therapy Market.

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Research objectives:

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About AllTheResearch:

AllTheResearch was formed with the aim of making market research a significant tool for managing breakthroughs in the industry. As a leading market research provider, the firm empowers its global clients with business-critical research solutions. The outcome of our study of numerous companies that rely on market research and consulting data for their decision-making made us realise, that its not just sheer data-points, but the right analysis that creates a difference.While some clients were unhappy with the inconsistencies and inaccuracies of data, others expressed concerns over the experience in dealing with the research-firm. Also, same-data-for-all-business roles was making research redundant. We identified these gaps and built AllTheResearch to raise the standards of research support.

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Canine Stem Cell Therapy Market: Market Growth Factors, Applications, Regional Analysis, Key Players and Forecasts by 2026 - PRnews Leader

The global stem cell and regenerative medicines market should grow from $21.8 billion in 2019 to reach $55.0 billion by 2024 at a compound annual growth rate (CAGR) of 20.4% for the period of 2019-2024.

Report Scope:

The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries. The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

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The report details market shares of stem cell and regenerative medicines based on products, application, and geography. Based on product the market is segmented into therapeutic products, cell banking, tools and reagents. The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes:

28 data tables An overview of global markets for stem cell and regenerative medicines Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 Details of historic background and description of embryonic and adult stem cells Information on stem cell banking and stem cell research A look at the growing research & development activities in regenerative medicine Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

Summary

The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018. The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024. Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018. The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period. The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study

Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications. New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future. The biopharmaceutical companies are investing significantly in cell-based therapeutics. The government organizations are funding research and development activities related to stem cell research. These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments. The market is impacted through adoption of stem cell therapy. The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.

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More:
Stem Cell and Regenerative Therapy Market to Remain Balanced During the the COVID-19 Period - re:Jerusalem

Global Stem Cell Therapy Market Overview:

The report includes background information along with a detailed overview of the global Stem Cell Therapy market. This data explains the development of the Stem Cell Therapy market as well as technological advances. The report also covers various end-user applications in the Stem Cell Therapy market. The market has been divided into several segments based on specific data that represents the total market share by the forecast year 2025. In addition, the Stem Cell Therapy market data is derived from competitive partners, key players and their revenues over the years. It also includes data on numerous players from all over the world, leading to huge fragmentation of the global Stem Cell Therapy market. The forecast for 2020-2025 highlights the growth of the Stem Cell Therapy market in terms of CAGR.

The impact of the Covid-19 outbreak on the Stem Cell Therapy industry, including potential opportunities and challenges, driving forces and risks, is also explored and evaluated in this study. Based on various scenarios (optimistic, pessimistic, very optimistic, most likely, etc.), we present an estimate of the impact of Covid-19 on a Stem Cell Therapy writer and a forecast for market growth for 2020-2025.

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Global Stem Cell Therapy Market Research analyzes vital geographic regions, provides a detailed assessment, including key market trends in accordance with their growth, future technologies, industry drivers, problems, regulatory policies, profiles of key players.

The research process included the study of various factors affecting the Stem Cell Therapy industry, including government policy, market environment, competitive environment, historical data, key technological advances and current trends shaping the market, current market trends and technological progress in related industries, and market risks, opportunities, market barriers and challenges.

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Competitive Analysis:

Major players place great emphasis on technological innovation to improve efficiency levels. The industrys growth prospects are reflected in ensuring continual improvement in player processes and optimal strategies used by companies to combat the COVID-19 situation. The company profile section of players such as Marriott International, Hilton Worldwide and AccorHotels includes relevant information such as name, subsidiaries, website, headquarters, market position, market growth / bust, historical background or comments on growth, as well as the top 3 closest competitors by market capitalization. / income along with contact information. Each companys revenues, annual growth, gross and operating margins are presented in an easy-to-understand tabular format over the past 5 years and in a separate section on recent events such as mergers and acquisitions, patent approvals, new project launches, etc.

Stem Cell Therapy Market Segmentation

By Types:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCsBone Marrow-Derived Mesenchymal SCsEmbryonic SCsOther Sources

By Applications:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal DisordersWounds & InjuriesCardiovascular DiseasesGastrointestinal DiseasesImmune System DiseasesOther Applications

Research Methodology

The Global Stem Cell Therapy Market Research was conducted by a team of industry experts and professionals with deep market knowledge. Researchers determine the intensity of competition and also study the opportunities for growth in the market using the parameters of the Porters five forces model method. The report also conducts a SWOT analysis of the Stem Cell Therapy market, identifying strengths, weaknesses, opportunities and threats in the market. The report will help companies entering the market get a complete picture of current and future market trends.

Market Dynamics

This report explores various market factors driving the rapid growth and expansion of the Stem Cell Therapy market. The report includes a detailed study of the history of pricing, supply and demand dynamics, price trends, etc., which play a huge role in the market. The report also examines the impact of government initiatives and policies on market stability. The report also analyzes market opportunities and weaknesses, which can help companies develop appropriate strategies.

Some of the major objectives of this report:

1. To provide a detailed analysis of the market structure along with the forecast of the various segments and sub-segments of the global Stem Cell Therapy Market.2. To provide insights about factors affecting market growth. To analyze the Stem Cell Therapy Market based on various factors- price analysis, supply chain analysis, porter five force analyses, etc.3. To provide historical and forecast revenue of the Stem Cell Therapy Market segments and sub-segments with respect to four main geographies and their countries- North America, Europe, Asia, and the Rest of the World.4. Country-level analysis of the market with respect to the current market size and future prospective.5. To provide country-level analysis of the market for segment by application, product type, and sub-segments.6. To provide strategic profiling of key players in the market, comprehensively analyzing their core competencies, and drawing a competitive landscape for the market.7. Track and analyze competitive developments such as joint ventures, strategic alliances, mergers and acquisitions, new product developments, and research and developments in the Stem Cell Therapy Market.

Table of Contents: Stem Cell Therapy Market

Chapter 1. Stem Cell Therapy Market OverviewChapter 2: State of the World Market and Forecasts by RegionChapter 3: State of the World Market and Forecast by TypeChapter 4: Global Market State and Prospects for the Downstream SectorsChapter 5: Analyzing Market DriversChapter 6: The State of Market Competition of Major ProducersChapter 7: Major Producers and Market DataChapter 8: Exploration and Production Market AnalysisChapter 9: Cost and Gross Profit AnalysisChapter 10: Analyzing Marketing StatusChapter 11: Conclusion of the Market ReportChapter 12: Research Methodology and Background Information

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Contact Us :

Ryan JohnsonAccount Manager Global3131 McKinney Ave Ste 600, Dallas,TX75204, U.S.A.Phone No.: USA: +1 972-362 -8199/ +91 9665341414

Original post:
Stem Cell Therapy Market Size 2020 Growth Analysis and Share by Key Players, Trends with Revenue with COVID-19 Impact Forecast to 2026 - PRnews...

Complete study of the global Stem Cell Therapy market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global Stem Cell Therapy industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on Stem Cell Therapy production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

The global Stem Cell Therapy market report reveals pertinent data on segment performance, growth potential in the coming years as well as vendor and manufacturer activities, aligning appropriately with consumption and production developments.

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The pandemic of Coronavirus (COVID-19) has affected every aspect of life worldwide. This has brought along several changes in market conditions. The rapidly changing industry scenario and initial and future assessment of the impact are covered in the report. The Stem Cell Therapy market report puts together a concise analysis of the growth factors influencing the current business scenario across various regions. Significant information pertaining to the industry analysis size, share, application, and statistics are summed in the report in order to present an ensemble prediction.

Some of the key reader queries that have been adequately addressed in the report include, top vendors and their growth triggering business strategies, regional outlook and growth hotspot identification, followed by prominent growth retardants, challenges and threat probability which result in dwarfed growth outlook. Further, to instill new market participation amongst novel enthusiast, this report clearly focuses teeming business opportunities that ensure smooth rise despite odds and growing competition.

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Competitive Landscape

It is important for every market participant to be familiar with the competitive scenario in the global Stem Cell Therapy industry. In order to fulfill the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Stem Cell Therapy Market Segmentation

Type Analysis of Stem Cell Therapy Market:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCsBone Marrow-Derived Mesenchymal SCsEmbryonic SCsOther Sources

Applications Analysis of Stem Cell Therapy Market:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal DisordersWounds & InjuriesCardiovascular DiseasesGastrointestinal DiseasesImmune System DiseasesOther Applications

Key questions answered in the report:

1.What is the growth potential of the Stem Cell Therapy market?2.Which product segment will grab a lions share?3.Which regional market will emerge as a frontrunner in coming years?4.Which application segment will grow at a robust rate?5.What are the growth opportunities that may emerge in Stem Cell Therapy industry in the years to come?6.What are the key challenges that the global Stem Cell Therapy market may face in future?7.Which are the leading companies in the global Stem Cell Therapy market?8.Which are the key trends positively impacting the market growth?9.Which are the growth strategies considered by the players to sustain hold in the global Stem Cell Therapy market?

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About Us :

Contact Us :

Ryan JohnsonAccount Manager Global3131 McKinney Ave Ste 600, Dallas,TX75204, U.S.A.Phone No.: USA: +1 972-362 -8199/ +91 9665341414

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Stem Cell Therapy Market | Growth Strategies Adopted By Top Key Players,Future Trends ,Application Worldwide And Assessment To 2026 - PRnews Leader

Fremont, CA , Oct. 28, 2020 (GLOBE NEWSWIRE) -- APstem Therapeutics, Inc., a private biopharmaceutical company developing breakthrough stem cell therapies, today announced the completion of an INitial Targeted Engagement for Regulatory Advice on CBER ProducTs (INTERACT) meeting with the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT). The meeting, which included CBER OTAT staff together with the APstem team, focused on the development plan for AP-Skin-01, a novel off-the-shelf allogeneic stem cell product derived from adult pluripotent stem cells (APSCs) for the initial treatment of diabetic ulcers.

Previous efforts to treat diabetic ulcers have been hampered by persistent inflammation, ischemia and delayed re-epithelialization, which together have posed an insurmountable challenge for drug developers. APstems proprietary stem cell technologies and unique attributes of APSCs may circumvent these obstacles. Preclinical results generated to date by APstem support the potential of AP-Skin-01 to treat difficult-to-heal diabetic ulcers by accelerating healing. AP-Skin-01 is designed to provide full skin structural reconstruction with less scar formation, promote angiogenesis and reduce inflammation during the repair process.

The INTERACT meeting we conducted with the FDA is an important first step in our development of AP-Skin-01. We considered the meeting to be very successful, with productive feedback from the FDA reviewers and strong alignment of APstems development approach for AP-Skin-01, said Dr. Min Hu, CEO and president of APstem Therapeutics. I would like to thank the reviewers from FDA CBER OTAT for their time and helpful guidance. We look forward to continuing our planned pre-clinical work into 2021.

Dr. Jane Lebkowski, APstems scientific advisor and an expert in the development of cell and gene therapies, added: The field of chronic diabetic wounds remains a critical unmet medical need in the expanding diabetic population worldwide. This new therapy has high potential to accelerate full skin regeneration. Therefore, we are grateful for FDAs positive feedback to accelerate the development of this product that could treat millions of patients.

INTERACT is the first available FDA interaction and is a key step in the pathway towards an Investigational New Drug (IND) application, the first regulatory step before experimental therapies may be tested in human clinical studies.

About APstem Therapeutics, Inc.

APstem Therapeutics is a biopharmaceutical company dedicated to discovering, developing, manufacturing, and commercializing novel stem cell therapies. APstems breakthrough stem cell therapy platform using our unique APSCs can be applied to multiple indications, including skin (diabetic ulcers), liver, lungs, bone/cartilage, heart, neuron and other tissues. For more information, please visit http://www.apstemtx.com.

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APstem Therapeutics Announces Successful FDA INTERACT Meeting Regarding AP-Skin-01, an Off-the-Shelf Allogeneic Stem Cell Product for the Treatment of...