Archive for the ‘Gene Therapy Research’ Category

Polyplus-transfection presents latest solution portfolio for gene therapy market to Alliance for Regenerative Medicines Meeting on the Mesa

Strasbourg, France, October 12, 2020 Polyplus-transfection(R)SAthe leading biotechnology company that supports the gene and cell therapy market bysupplying innovative transfection solutions, today announces it will present its latest integrated solution to support viral vector manufacturing from process development through to commercialization for the gene therapy sector. The presentation will be delivered at theAlliance for Regenerative Medicine(ARM) annual conference, the 2020 VirtualCell & Gene Meeting on the Mesa. The meeting will be held between Monday October 12 and Friday October 16, 2020.

The Polyplus-transfection presentation will be delivered by Graldine Gurin-Peyrou, Director, Polyplus-transfection and will be available to view on demandonlinethroughout the conference.

Polyplus-transfection specializes in working with viral vector developers and manufacturers to provide innovativetransfection reagentsthat can boost viral vector production. The new Polyplus-transfection complete solution has been designed and developed to improve the critical element in gene therapy production the upstream process development for viral vector manufacturing.

The Polyplus solution involves the development of dedicated transfection reagents depending upon the viral vector type required. This includes first targeting the AAV manufacturing with Polyplus-transfections novel transfection reagentFectoVIR-AAV, The reagent has been specifically developed to improve AAV (adeno-associated virus) production in suspension cell culture system for large-scale manufacturing.

The Polyplus solution also gives the gene therapy market access to the industrysfirst GMP compliant testfor the Polyplus-transfection PEIpro product range to detect residual transfection reagent during the production of ATMPs (advanced therapeutic medicinal products). GMP guidelines specify manufacturers should be aware of both the residual levels of raw materials used in drug products, and the significance of these results. These regulations ensure manufacturers reliably determine residual raw material levels and thus maintain reproducible safety of ATMPs for patient administration. The Polyplus test is able to detect and quantify residual PEIpro throughout the ATMP manufacturing process. As a result, it can be used as a release quality control. The test can be adapted for each ATMP in order to ensure the lowest limits of detection.

Finally, the Polyplus solution is the first globally toenable access to dual sourcing for reagents. This will mitigate the risks to the ATMP industry as the sole provider of the PEIpro-GMP transfection reagent, the most used transfection reagent worldwide and a critical component for the development and manufacture of ATMPs for gene therapies. The dual sourcing approach enables Polyplus to source its PEIpro-GMP product from two distinct subcontracting manufacturing plants. Polyplus is able to ensure sole responsibility and control of the process. ATMP manufacturers are then able to have Polyplus as a single point of contact whilst benefiting from increased production capacity and shortened lead times.

ATMPs and gene therapies are moving through late-stage trials and to commercialization at an exponential rate. This in turn is vastly increasing the demand for GMP reagents. In addition, ATMP therapies are becoming progressively more complex, and this means that Polyplus-transfection has to increase its services to the gene therapy sector, alongside its products, said Graldine Gurin-Peyrou, Director, Polyplus-transfection. Communicating these services to the gene therapy market through the Alliance for Regenerative Medicines Meeting on the Mesa is essential for us as we move forward in our delivery of critical reagents that will ultimately result in therapies reaching patients.

About Polyplus-transfection SA

Polyplus-transfection(R) SA is the leading biotechnology company that supports Gene and Cell therapy, along with other biologics manufacturing and life science research with innovative nucleic acid transfection solutions. Polyplus-transfections strengths are 20 years of experience in manufacturing transfection solutions with tailored scientific and regulatory support to accelerate research and clinical development. Based on the Science Park close to Strasbourg (France), Polyplus-transfection offers an extensive and growing range of transfection reagents available worldwide. For more information, please visit the Polyplus-transfection web site at:www.polyplus-transfection.com.

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Polyplus-transfection presents latest solution portfolio for gene therapy market to Alliance for Regenerative Medicine's Meeting on the Mesa -...

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Viral vector and plasmid DNA Market Size 2020 Market Research, Growth, Manufacturers, Segments and 2027 Forecasts Research - The Think Curiouser

Mercks New VirusExpress Platform Speeds Development of Cell and Gene Therapies

Darmstadt, Germany, October 13, 2020 Merck, a leading science and technology company, has bolstered its viral vector manufacturing capabilities with the launch of its VirusExpress Lentiviral Production Platform. This new platform helps to overcome lentiviral production challenges and can reduce process development time by approximately 40 percent, based on Mercks experience as a contract development and manufacturing organization.

Cell and gene therapies offer the potential for curative treatments and are being developed and commercialized in half the time it has taken traditional therapies, said Angela Myers, head of Gene Editing & Novel Modalities, Life Science, at Merck. We are committed to accelerating manufacturing of cell and gene therapies with the ultimate goal of getting these lifesaving treatments to patients faster. By increasing dose yields and dramatically reducing process development time, this new platform will help us reach this goal.

Using a suspension cell line rather than an adherent-based production, coupled with a chemically defined cell culture media and process with built-in scalability, Mercks VirusExpress Platform meets multiple market needs. In addition to accelerating process development, the suspension culture format allows each batch of virus to be larger yielding more patient doses. Additionally, suspension culture is amenable to true scale-up, while being less labor-intensive. The chemically defined medium eliminates the safety, regulatory and supply chain concerns related to animal- and human-derived materials.

Mercks VirusExpress Platform offers a simplified upstream workflow, making processes easier to manage, adjust and scale. Flexible licensing allows companies to manufacture vectors by using either Mercks contract manufacturing capabilities, a third-party contract development and manufacturing organization, or in-house development.

The Life Science business of Merck is a leading contract development and manufacturing organization combining an integrated portfolio of manufacturing solutions with proven commercialization experience. This new offering underscores Mercks continued investment in cell and gene therapies. In April 2020, the company announced a new 100 million, 140,000-square-foot manufacturing center at its Carlsbad, California, USA, location that will double the existing production capacity and support large-scale commercial manufacturing. Today, the Life Science business of Merck manufactures vectors for two of the first five FDA-approved cell and gene therapies.

The cell and gene therapy market is growing rapidly and continues to show great promise. According to market research leader Arizton, the cell and gene therapy market is expected to reach more than $6.6 billion by 2024[1]. Merck has been involved in this space since clinical trials for gene therapy began in the 1990s.

Operator manufacturing viral vector in a cGMP environment. Mercksnew VirusExpressPlatformincreases dose yields and reduces process development time for cell and gene therapies.

All Merck news releases are distributed by email at the same time they become available on the Merck Website. Please go to http://www.merckgroup.com/subscribe to register online, change your selection or discontinue this service.

About Merck

Merck, a leading science and technology company, operates across healthcare, life science and performance materials. Around 57,000 employees work to make a positive difference to millions of peoples lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices the company is everywhere. In 2019, Merck generated sales of 16.2 billion in 66 countries.

Scientific exploration and responsible entrepreneurship have been key to Mercks technological and scientific advances. This is how Merck has thrived since its founding in 1668. The founding family remains the majority owner of the publicly listed company. Merck holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the business sectors of Merck operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Performance Materials.

[1] http://www.prnewswire.com/news-releases/the-cell-and-gene-therapy-market-to-reach-revenues-of-over-6-6-billion-by-2024---market-research-by-arizton-300957463.html

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Merck's New VirusExpress Platform Speeds Development of Cell and Gene Therapies - PharmiWeb.com

PHILADELPHIA, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced publication of data in a murine model of GM1 gangliosidosis (GM1) demonstrating that a single intracerebroventricular injection of an optimized adeno-associated virus (AAV) into the cerebral spinal fluid (CSF) resulted in significant expression of Beta-galactosidase (-gal) in the brain and peripheral tissues, and demonstrated dose-related reductions in neuronal lysosomal storage lesions, neurological impairment and improvement in survival. These data were published online ahead of print in the November issue of the peer-reviewed scientific journal Human Gene Therapy (HGT).

This study suggests that delivery of an AAV vector optimized to express b-gal directly into the CSF restored b-gal activity in the brain and, if further developed and tested in human clinical trials, may be effective in modifying and preventing the devastating effects of the genetic disease GM1, said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania (Penn) and chief scientific advisor of Passage Bio. The AAV vector used in the study is the same as Passage Bios PBGM01 gene therapy, which is designed to deliver a functional human GLB1 gene into the brain and optimized to express -gal. These preclinical study data support the further development of PBGM01 as a potential therapy for patients suffering from GM1.

GM1 is a rare and often life-threatening monogenic lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid -gal. Reduced -gal activity results in the accumulation of toxic levels of GM1 in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the infantile form, which is characterized by onset in the first six months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately 60 percent of the incidence of 0.5 to 1 in 100,000 live births. Currently, there are no approved disease-modifying therapies available.

Results of the PBGM01 preclinical study were reported in the paper titled, A single injection of an optimized AAV vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis, by Christian Hinderer, M.D., Ph.D., and colleagues, including gene transfer pioneer Dr. Wilson, from the Gene therapy Program, Department of Medicine, University of Pennsylvania Perlman School of Medicine. The study in part was previously presented at the 22nd annual Meeting of the American Society for Cell and Gene Therapy (ASCGT) in 2019.

This research evaluated the impact of single intracerebroventricular administration of the human -gal containing AAV vector on -galactosidase enzyme activity in the murine brain and peripheral tissues, lysosomal storage lesions, neurological function (including neurological exams and gait analysis) and survival in mice lacking the -galactosidase gene. The mice received the single administration at age one month and were evaluated over 300 days. -gal activity was increased significantly in the cerebral spinal fluid and serum of the vector-treated mice compared to vehicle control-treated mice. Significant improvements in gait assessments as measured by stride length and hind paw print length and significant preservation of neurological function as measured by neurological exam scores were observed throughout the study period in the human -gal vector-treated mice. There were significant decreases in lysosomal storage lesions of vector-treated animals and by day 300 all animals that received the two highest doses were still alive, whereas none of the vehicle control-treated animals had survived.

Were excited about being able to soon advance PBGM01 into the clinic, and the potential promise it holds for patients with GM1, the majority of whom are infants and for whom there are no approved disease modifying treatments, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. Our plan is to administer PBGM01 through intra-cisterna magna delivery into the brain, which we believe may offer several benefits in terms of safety, efficiency and distribution compared to other approaches.

Passage Bio expects to initiate dosing of PBGM01 in a Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

This research was supported by a research, collaboration and license agreement with Passage Bio. HGT is the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies. Click here to read the full-text article on the HGT website.

About PBGM01PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual -gal enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a next-generation AAVhu68 capsid administered through intra-cisterna magna (ICM) to deliver a functional GLB1 gene encoding -gal to the brain and peripheral tissues. By reducing the accumulation of GM1 gangliosides, PBGM01 has the potential to halt or prevent neuronal toxicity, thereby restoring developmental potential. In preclinical models, PBGM01 has demonstrated broad brain distribution and wide uptake of the -gal enzyme in both the central nervous system (CNS) and critical peripheral organs, suggesting potential treatment for both the CNS and peripheral manifestations of GM1. The Company has received Orphan Drug and Rare Pediatric Disease designation for PBGM01 for patients with GM1 and expects to initiate dosing of its Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

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

University of Pennsylvania (Penn)Financial DisclosureDr. Wilson is a Penn faculty member and also a scientific collaborator, consultant and co-founder of Passage Bio. As such, he holds an equity stake in the company, receives sponsored research funding from Passage Bio, and as an inventor of certain Penn intellectual property that is licensed to Passage Bio, he may receive additional financial benefits under the license in the future. He is an inventor of intellectual property covering the technology described in paper published in HGT that is licensed from Penn to Passage Bio, and he may receive financial benefits under this license in the future. Penn also holds equity and licensing interests in Passage Bio.

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

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.comzofia.mita@sternir.com

Media:Gwen FisherPassage Bio215.407.1548gfisher@passagebio.com

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Passage Bio Announces Publication of Preclinical Data That Show Single Injection of Optimized AAV Vector into Cerebral Spinal Fluid - 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.

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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.

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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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Tag: Gene Therapy - The Think Curiouser

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Parameters involved in the Personalized Gene Therapy Treatment market includes:

Segmentation Overview:

Personalized Gene Therapy Treatment Market Key Players:

Amgen, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., SynerGene Therapeutics, Inc., Cold Genesys, Inc., Bellicum Pharmaceuticals, Inc., Takara Bio, Inc.,Ziopharm Oncology, Inc., , Sevion Therapeutics, Inc., OncoSec Medical, Inc., and Burzynski Clinic.

The report provides an in-depth geographical analysis of the Personalized Gene Therapy Treatment market, covering important regions, viz, North America, Europe, Asia Pacific, Middle East & Africa, and Latin America. It also covers key countries (regions), viz, U.S., Canada, France, Germany, U.K., Italy, Russia, India, China, Japan, South Korea, Australia, Taiwan, Thailand, Indonesia, Malaysia, Vietnam, Philippines, Mexico, Brazil, GCC, Israel, South Africa, etc.

The competitive analysis section of the report includes prominent players of the Personalized Gene Therapy Treatment market that are broadly studied on the basis of several key factors.

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Personalized Gene Therapy Treatment Market: Dynamics, Segment, Application and Supply Demand Analysis 2020-2030 - The Think Curiouser

For an outstanding business growth, companies must take up market research report service which is vital in todays market place. An influential Gene Therapy Market report also offers top to bottom examination of the market for estimating income, return on investment (ROI) and developing business strategies. This market research report helps out the business in every sphere of trade to take the unmatched decisions, to tackle the toughest business questions and diminish the risk of failure. The industry report highlights general market conditions, estimates market share and possible sales volume of industry. The facts and figures described in this Gene Therapy Market document aids industry in taking sound decisions and planning advertising and sales strategy more successfully.

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Market Analysis: Global Gene Therapy Market

Global gene therapy market is rising gradually with a healthy CAGR of 36.1% in the forecast period of 2019-2026. Increasing incidence of cancer and rare life threatening diseases and strong clinical pipeline drugs for gene therapy are major drivers for market growth.

Key Market Players:

Few of the major competitors currently working in the globalgene therapy marketarePfizer Inc., Thermo Fisher Scientific Inc., F. Hoffmann-La Roche Ltd, Spark Therapeutics, Inc., bluebird bio, Inc., ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc., Sarepta Therapeutics, Novartis AG, MeiraGTx Limited, Rocket Pharmaceuticals, Lonza, Biogen, Gilead Sciences, Inc., REGENXBIO Inc., uniQure N.V., Solid Biosciences Inc., Audentes Therapeutics among others.

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Market Definition: Global Gene Therapy Market

Gene therapy is a technique of insertion of genes into cells and tissues for treatment of any disease. In this technique the defective gene is replaced with a functional gene. It is the strategy of manipulation of expression of specific genes responsible for the disease. This therapy is a promising treatment option for a number of diseases. The application of gene therapy is wide and it is mostly used for treatment of cancer, cystic fibrosis, heart disease, diabetes, AIDS among others.

Gene Therapy Market Drivers

Gene Therapy Market Restraints

Segmentation:Global Gene Therapy Market

Gene Therapy Market : By Type

Gene Therapy Market : By Gene Type

Gene Therapy Market : By Viral Vector

Gene Therapy Market : By Non-Viral Vector

Gene Therapy Market : By Application

Gene Therapy Market : By End Users

Gene Therapy Market : By Distribution Channels

Gene Therapy Market : ByGeography

Key Developments in the Market:

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Gene Therapy Market : Share, Size, Regional Trend, Future Growth, Forecast || Leading Players ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics,...

Pune, Maharashtra, India, October 14 2020 (Wiredrelease) MarketResearch.Biz :The Global Gene Therapy Market Outlook to 2029, Capacity, Generation, Investment Trends, laws, and Company Profiles. The business analysis specialists that provide comprehensive data and understanding of the Gene Therapy market within the globe.

The report provides an in-depth analysis of the world Gene Therapy market with forecasts up to 2029. The report analyzes the market state of affairs and provides future outlook with forecasts up to 2029. The report highlights capability and generation trends from 2017 to 2029 in Gene Therapy market. in-depth coverage of the market with specific policies regarding Gene Therapy is provided within the report. The analysis in addition provides company snapshots of a variety of the most market participants.

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Further, the report acknowledges that in these growing and promptly enhancing the market surroundings, the foremost recent advertising and promoting details are vital to conclude the performance within the forecast amount and create the essential selections for profitableness and growth of the Gene Therapy market. additionally, the report contains An array of things that impact the expansion of the world Gene Therapy market within the forecast amount. Further, this specific analysis additionally concludes the impact on the individual segments of the market.

Prominent players of Gene Therapy including:

Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

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Global Gene Therapy Market Segmentation:

By Vector: Viral vector Retroviruses Lentiviruses Adenoviruses Adeno Associated Virus Herpes Simplex Virus Poxvirus Vaccinia Virus Non-viral vector Naked/Plasmid Vectors Gene Gun Electroporation Lipofection By Gene Therapy: Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Other By Application: Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

The Gene Therapy market report consists of associate analysis of the market size for price in Million USD and volume in elements. The analysis report estimate and validate the market size of Gene Therapy market, completely different all different dependent sub-markets inside the general Gene Therapy trade by using top-down and bottom-up approaches. The Secondary analysis has been accustomed to decide the key players in Gene Therapy market and market shares, rate, and Gene Therapy market future trends are discovered through primary and secondary analysis. The target of this Gene Therapy report is to produce a full study of Gene Therapy market by analyzing all completely different regions.

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The market research report precisely provides to the client:

To achieve a penetrating study of the Gene Therapy Market associate degreed have an exhaustive perception of the market and its economic condition analysis.

Appraise the manufacturing procedure, tidy affairs, and solutions.

Market policies that area unit being acquired by top-most specific organizations

Get associate degree exhaustive delineation of the Gene Therapy Market business.

Comprehend the combative circumstances, important competitors, and Gene Therapy Market leading brands

The main objectives of the market research report are:

To appear at international Gene Therapy Market position, succeeding predict, growth scope, prime market, and prime players.

To gift the Gene Therapy Market advancement among the u. s., Europe, and China.

To strategically profile the Gene Therapy Market key players and fully analyze their growth policies and techniques.

to stipulate, justify, and forecast the Gene Therapy Market by product sort, application, and key regions.

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The report is readily available and can be dispatched immediately after payment confirmation.

Global Gene Therapy Market Report mainly covers ten significant points:

1. An outlook of the Gene Therapy industry.

2. Business Competitive Landscape.

3. Global Gene Therapy Market share from 2020 to 2029.

4. Supply Chain Analysis.

5. Top Players Company Profiles.

6. Analysis of the product types of Gene Therapy.

7. Analysis of the Applications/End-Users of Gene Therapy.

8. Consumption and Export, Import Value by Major Countries.

9. Global Gene Therapy Market Forecast to 2029.

10. Critical success factors and Conclusions.

Table Of Content:-

Chapter 1:Gene Therapy Market Overview.

Chapter 2:Gene Therapy Market Segment Upstream and Downstream and Cost Analysis

Chapter 3:Gene Therapy industry by Type( Market Size & Forecast, Major Company of Product Type)

Chapter 4:Gene Therapy industry by Top Key Players(Sales Revenue, Gross Margin, Price, Main Products, etc)

Chapter 5 and 6:Gene Therapy Industry Competition and Market Demand(Demand Situation, Demand Forecast, Regional Demand Comparison)

Chapter 7:Global Gene Therapy Market report additionally depicts Region Operation (Demand & Forecast by Countries, Regional Output etc).

Chapter 8:Global Gene Therapy Market Price Trends, Manufacturers Gross Margin Analysis, Factors of Price Change.

Chapter 9:This report additionally depicts deals channel, merchants, brokers, wholesalers and market Research Findings and Conclusion, addendum and information source.

Click to View Figures, TOC Mentioned in the Gene Therapy Market Report at : https://marketresearch.biz/report/gene-therapy-market/#toc

At the ending, the Gene Therapy Market report decisions investment embody investigation and development tendency investigation. the first opportunities of this quickest growing international Gene Therapy Market business sections that area unit coated throughout this report. The Gene Therapy Market product specification and services and product value structure with production divided into the most effective regions, technology, and applications.

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Global Gene Therapy Market Investment Feasibility, Evolutionary Production and Regional Analysis | N - PharmiWeb.com

The research report onGlobal Hemophilia Gene Therapy Market2020 deeply studied remarkable features of the industry. The study provides market size, Hemophilia Gene Therapy ongoing trends, drivers, risks, opportunities, as well as major Hemophilia Gene Therapy market segments. It is based on historical information and presents Hemophilia Gene Therapy market requirements. Also, includes different Hemophilia Gene Therapy business approaches preferred by the decision-makers. That enhanced Hemophilia Gene Therapy growth and makes a phenomenal stand in the industry. The Hemophilia Gene Therapy market will raise with a prominent CAGR between 2020 to 2026.

The Hemophilia Gene Therapy Market has witnessed continuous growth in the past few years and is predicted to rise even further during the estimated period. In adding to the inclusive assessment of the market, the report presents upcoming trends, up-to-date Growth Factors, attentive opinions, facts, historical data, and statistically supported and industry validates market data.

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Hemophilia Gene Therapy Market Major Industry Players 2020:

Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline Therapeutics

Firstly, it figures out the main Hemophilia Gene Therapy industry structure, guidelines, deals, agreements, Hemophilia Gene Therapy regulations, and policies. Then covers the prediction of Hemophilia Gene Therapy market share, dynamics, and dominant players. Next, it lineup new Hemophilia Gene Therapy assumption to updates business values. in addition, it examines the Hemophilia Gene Therapy market position, current, and future projects, growth rate, and exploitation. It also scrutinizes world Hemophilia Gene Therapy market chain analysis, cost of raw material. Further, it reveals Hemophilia Gene Therapy downstream/upstream analysis and import-export landscape.

Hemophilia Gene Therapy Market Type Analysis:

Hemophilia AHemophilia B

Hemophilia Gene Therapy Market Applications Analysis:

Hemophilia A Gene TherapyHemophilia B Gene Therapy

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The analysis covers basic information about the Hemophilia Gene Therapy product like industry scope, segmentation, an overview of the market. Likewise, it provides supply-demand data, Hemophilia Gene Therapy investment feasibleness, and elements that limiting the growth of a Hemophilia Gene Therapy industry. Particularly, it serves Hemophilia Gene Therapy product demand, annual revenue, and growth prospects of the industry. The foreseen Hemophilia Gene Therapy market regions along with the present ones assist leading vendors, decision-makers, and viewers/readers to plan effectively Hemophilia Gene Therapy business strategies respectively.

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Global Hemophilia Gene Therapy Industry Research Report Benefits:

* Product executives, industry administrator, Hemophilia Gene Therapy chief regulative officers of the industries.

* Researchers, Hemophilia Gene Therapy examiners, research executives, and laboratory expertise.

* Universities, professors, students, interns, and distinct other academic organizations involved in the Hemophilia Gene Therapy market.

* Writer, reporters, journalists, editors, and webmasters want to know regarding Contract Lifecycle Management System.

* Private/governmental organizations, project managers involved in the Hemophilia Gene Therapy industry.

* Present or future Hemophilia Gene Therapy market players.

TABLE OF CONTENT:

Chapter 1: Hemophilia Gene Therapy Market Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3: Hemophilia Gene Therapy Market Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Hemophilia Gene Therapy Market Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Hemophilia Gene Therapy Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Hemophilia Gene Therapy Market Analysis, Distributors/Traders

Chapter 11: Hemophilia Gene Therapy Market Effect Factors Analysis

Chapter 12: Global Hemophilia Gene Therapy Market Forecast to 2027

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Global Hemophilia Gene Therapy Market New Trends, Future Growth, Outlook, Industry Overview, Application and Forecast 2020-2027 - PRnews Leader

Hemophilia Gene Therapy Market Forecast 2020-2026

The Global Hemophilia Gene Therapy Market research report provides and in-depth analysis on industry- and economy-wide database for business management that could potentially offer development and profitability for players in this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. It offers critical information pertaining to the current and future growth of the market. It focuses on technologies, volume, and materials in, and in-depth analysis of the market. The study has a section dedicated for profiling key companies in the market along with the market shares they hold.

The report consists of trends that are anticipated to impact the growth of the Hemophilia Gene Therapy Market during the forecast period between 2020 and 2026. Evaluation of these trends is included in the report, along with their product innovations.

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The Report Covers the Following Companies:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therap

By Types:Hemophilia AHemophilia BHemophilia Gene Therap

By Applications:Hemophilia A Gene TherapyHemophilia B Gene Therapy

Furthermore, the report includes growth rate of the global market, consumption tables, facts, figures, and statistics of key segments.

By Regions:

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Years Considered to Estimate the Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year: 2020-2026

Important Facts about Hemophilia Gene Therapy Market Report:

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About Industrygrowthinsights:Industrygrowthinsights has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

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Global Hemophilia Gene Therapy Market 2026 Size, Key Companies, Trends, Growth And Regional Forecasts Research - PRnews Leader

Oct. 14, 2020 11:00 UTC

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.

Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 cases in the U.S. and European Union (EU). It is an X-linked disease that primarily occurs in females, but it can be seen very rarely in males. It is usually recognized in children between six to 18 months of age as they begin to miss developmental milestones or lose abilities they had developed. Individuals with Rett syndrome also show symptoms that include loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments, breathing issues and sleep disturbances.

Patients with Rett syndrome are currently managed with symptomatic treatments as there are no therapies approved to treat the underlying cause of disease, said Berge Minassian, M.D., Chief Medical Advisor of Taysha and Chief of Pediatric Neurology at the University of Texas Southwestern Medical Center (UT Southwestern). Dr. Minassian is credited with describing the CNS isoform of the MECP2 gene which is responsible for neuronal and synaptic function throughout the brain. Gene therapy offers a potentially curative option for patients suffering with Rett syndrome.

Rett syndrome is caused by mutations in the MECP2 gene. TSHA-102 is designed to deliver a healthy version of the MECP2 gene as well as the miRNA-Responsive Auto-Regulatory Element, miRARE, platform technology to control the level of MECP2 expression. TSHA-102 represents an important step forward in the field of gene therapy, where we are leveraging a novel regulatory platform called miRARE to prevent the overexpression of MECP2, said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. In collaboration with Sarah Sinnett, Ph.D. to develop miRARE, our goal was to design a regulated construct that allowed us to control MECP2 expression to potentially avoid adverse events that are typically seen with unregulated gene therapies.

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as "rare" affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S.

Obtaining these designations is a validation of decades-long work to identify and optimize a potential gene therapy treatment for this devastating disease, said RA Session II, President, CEO and Founder of Taysha. We are also excited to advance our miRARE platform whereby regulated expression of a transgene is possible on a cellular basis. The miRARE platform has broad applicability across a wide range of monogenic CNS disorders where there is a need to control transgene expression.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

About miRARE

For disorders that require replacement of dose-sensitive genes, we have combined high-throughput microRNA, or miRNA, profiling and genome mining to create miRNA-Responsive Auto-Regulatory Element, or miRARE, our novel miRNA target panel. This approach is designed to enable our product candidates to maintain safe transgene expression levels in the brain. This built-in regulation system is fully endogenous, and does not require any additional exogenous drug application. Instead, the miRARE system utilizes endogenous transgene-responsive miRNA to downregulate transgene expression in the event that overexpression occurs. miRARE may be applicable to a range of diseases where overexpression of a therapeutic transgene is a concern.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-102, to positively impact quality of life and alter the course of disease in the patients we seek to treat, the benefits of, and our ability to develop product candidates using, miRARE, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our prospectus dated September 23, 2020, as filed with the Securities and Exchange Commission (SEC) on September 24, 2020, pursuant to Rule 424(b) under the Securities Act of 1933, as amended, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201014005319/en/

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Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome - BioSpace

The Cell and Gene Therapy market research report added by Market Study Report, LLC, is an in-depth analysis of the latest trends persuading the business outlook. The report also offers a concise summary of statistics, market valuation, and profit forecast, along with elucidating paradigms of the evolving competitive environment and business strategies enforced by the behemoths of this industry.

Executive Summary:

The recent report on Cell and Gene Therapy market offers an in-depth analysis of this industry vertical and talks about the various growth drivers, opportunities, challenges, and other prospects influencing the remuneration. According to the report, the Cell and Gene Therapy market is predicted to expand with a CAGR of XX% during the study duration.

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Crucial insights pertaining to geographical landscape and competitive scenario as well as factors impacting the several market segmentations are encompassed in the document. The report also analyzes the impact of COVID-19 pandemic on the market outlook in the approaching years.

Market Rundown:

Regional outlook:

Product landscape summary:

.

Application scope overview:

.

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Competitive landscape Review:

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Report Objectives:

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Cell and Gene Therapy Market Overview with Detailed Analysis, Competitive landscape, Forecast to 2025 - Eurowire

LEXINGTON, Mass. and AMSTERDAM, Oct. 13, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that two additional patient procedures have been completed in the Phase I/II clinical trial of AMT-130 for the treatment of Huntingtons disease. The ongoing patient enrollment follows a meeting last month of the trials independent Data Safety Monitoring Board (DSMB) to review 90-day follow-up data from the first two patients. The DSMB observed no significant safety concerns to prevent further dosing. The Phase I/II study is a double-blinded, randomized and controlled clinical trial being conducted in the United States. A total of four patients have been enrolled in the study thus far, including two patients treated with AMT-130 and two patients who received imitation surgery.

We are very pleased with the progress being made to advance this first-in-human AAV gene therapy for Huntingtons disease, said Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. This is an important achievement that puts us on our original clinical development timeline, making up for the modest delay in the study earlier this year due to COVID-19. In accordance with the study protocol, patient enrollment is expected to continue after a DSMB meeting to review 90-day follow-up data on these two new patients and 6-month data on the first two patients. We expect that this DSMB review will take place early next year and that patient enrollment in the 10-patient first dose cohort will be completed by mid-2021.

The Phase I/II clinical trial of AMT-130 for the treatment of Huntingtons disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntingtons disease randomized to treatment with AMT-130 or an imitation (sham) surgery across two dose cohorts. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for 5 years after administration of AMT-130. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). Additional details are available on http://www.clinicaltrials.gov (NCT04120493).

AMT-130 is uniQures first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE platform.

About Huntingtons Disease

Huntingtons disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntingtons disease, there are no currently approved therapies to delay the onset or to slow the diseases progression.

About uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether patient enrollment will continue after a DSMB meeting to review follow-up data, whether the DSMB review will take place early next year, and whether patient enrollment in the first dose cohort will be completed by mid-2021.Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures periodic securities filings, including its Annual Report on Form 10-K filed March 2, 2020 and Quarterly Report on Form 10-Q filed on July 30, 2020. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:

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uniQure Announces Enrollment of Next Two Patients in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease - GlobeNewswire

DUBLIN, Oct. 13, 2020 /PRNewswire/ -- The "Viral Vector Purification / Virus Purification Products Market (kit, prepacked column, resin, cassette, filter plate, capsule, reagent and others), 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The Viral Vector Purification / Virus Purification Products Market 2020-2030' report features an extensive study of various products available for viral vector purification, in addition to the current market landscape and future potential of product developers.

Overview

Since the approval and launch of cell and gene therapy products, such as Zolgensma (2019), ZYNTEGLO (2019), Luxturna (2017), KYMRIAH (2017) and YESCARTA (2017), there has been a significant increase in demand for viral vectors. Experts believe that the number of such therapies is likely to double over the next couple of years. It is also worth mentioning that this particular field of medical research received close to USD 10 billion in funding in 2019.

Currently, over 1,000 cell and gene therapy-related clinical trials are being conducted, worldwide. Genetic manipulation is a basic requirement of cell and gene therapy development, and, as a result, gene delivery vectors are considered to be of utmost importance in this domain. So far, viral vectors (such as those based on adeno-associated viruses (AAV), adenoviruses, lentivirus, retroviruses and other viruses) have been shown to be the most versatile gene delivery tools available, having demonstrated high transfection efficiencies in both preclinical and clinical settings.

Further, taking into account the therapeutic efficacy and low side effects profiles of cell and gene therapies, the demand for such interventions is anticipated to grow at a rapid pace, resulting in a proportional increase in need for appropriate vector systems, as well. However, viral vector development and manufacturing is a complex and cost intensive process. One of the primary concerns associated with viral vector production is related to yield; in fact, a singular batch run is estimated to incur losses of up to 70% during the purification step alone.

A number of techniques are presently used for viral vector purification. Over the years, size-based viral purification strategies, such as density-gradient ultracentrifugation, ultrafiltration, precipitation and size-exclusion chromatography (SEC), have become part of the accepted industry standard. However, recently, stakeholders have begun relying more on affinity chromatography-based purification regimens, given its robustness and high selectivity. Presently, several companies claim to offer a diverse range of virus purification solutions, including, filter plates, prepacked chromatography columns and resins, and consolidated kits, for viral vector (virus) purification. As indicated earlier, downstream processing of viral vector products is challenging.

Scope of the Report

An overview of the current market landscape of companies providing products for purification of viruses / viral vectors, using different techniques, such as chromatography, centrifugation and filtration. It features information on the type of product (kit, prepacked column, resin, cassette, filter plate, capsule and reagent), type of purification technique (chromatography, centrifugation and filtration), scale of operation (lab-scale, clinical and commercial), type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others) and details on other physical and operational parameters of the product (such as matrix, pore size, volume of bed, flow rate, operating pressure, working temperature, pH, filtration area and process time).

In addition, the chapter includes information on the purification product developers, including details on the year of establishment, company size and location of headquarters. Elaborate profiles of key players, including an overview of the company, product portfolio (viral vector purification products), recent developments and an informed future outlook.

An analysis evaluating the potential strategic partners (comprising of viral vector-based therapy developers and viral vector manufacturers) for viral vector purification product developers, based on several parameters, such as type of viral vector, developer strength, operational strength, therapeutic area, strength of clinical pipeline and strength of preclinical pipeline.

A clinical trial analysis of completed, ongoing and planned studies of various viral vector-based cell therapies, gene therapies and vaccines (approved / under development). It features detailed analyses of clinical studies of different viral-vector based therapies on the basis of their registration year, phase of development, trial status, type of therapy, therapeutic area, type of sponsor/collaborator, geographical location, number of patients enrolled and key players.

An informed estimate of the annual clinical and commercial demand (in terms of number of patients) for viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. Further, the demand has been segregated on the basis of type of viral vector, type of therapy, therapeutic are and geographical location. A case study on tangential flow filtration (TFF), highlighting the role, advantages and disadvantages of the technique for purification of viral vectors; the chapter features details of products used for TFF, including product type, scale of operation, membrane material, flow rate and filtration area.

A case study featuring the viral vector manufacturers providing commercial scale production, highlighting details on their year of establishment, company size, type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), purpose of production (in-house and contract-basis), and location of headquarters and manufacturing facilities.

One of the key objectives of the report was to estimate the existing market size and identify potential growth opportunities for viral vector purification product developers, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies related to viral vector-based therapies, anticipated growth in target patient population, existing price variations across different purification techniques, and the success of cell and gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2020-2030.

Companies Mentioned

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Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Cell Therapy market.

Trusted Business Insights presents an updated and Latest Study on Cell Therapy Market. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Cell Therapy market during the forecast period.It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

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Industry Insights, Market Size, CAGR, High-Level Analysis: Cell Therapy Market

The global cell therapy market size was valued at USD 5.8 billion in 2019 and is projected to witness a CAGR of 5.4% during the forecast period. The development of precision medicine and advancements in Advanced Therapies Medicinal Products (ATMPS) in context to their efficiency and manufacturing are expected to be the major drivers for the market. In addition, automation in adult stem cell and cord blood processing and storage are the key technological advancements that have supported the growth of the market for cell therapy.

The investment in technological advancements for decentralizing manufacturing of this therapy is anticipated to significantly benefit the market. Miltenyi Biotec is one of the companies that has contributed to the decentralization in manufacturing through its CliniMACS Prodigy device. The device is an all-in-one automated manufacturing system that exhibits the capability of manufacturing various cell types.

An increase in financing and investments in the space to support the launch of new companies is expected to boost the organic revenue growth in the market for cell therapy. For instance, in July 2019, Bayer invested USD 215 million for the launch of Century Therapeutics, a U.S.-based biotechnology startup that aimed at developing therapies for solid tumors and blood cancer. Funding was further increased to USD 250 billion by a USD 35 million contribution from Versant Ventures and Fujifilm Cellular Dynamics.

The biomanufacturing companies are working in collaboration with customers and other stakeholders to enhance the clinical development and commercial manufacturing of these therapies. Biomanufacturers and OEMs such as GE healthcare are providing end-to-end flexible technology solutions to accelerate the rapid launch of therapies in the market for cell therapy.

The expanding stem cells arena has also triggered the entry of new players in the market for cell therapy. Celularity, Century Therapeutics, Rubius Therapeutics, ViaCyte, Fate Therapeutics, ReNeuron, Magenta Therapeutics, Frequency Therapeutics, Promethera Biosciences, and Cellular Dynamics are some startups that have begun their business in this arena lately.

Use-type Insights

The clinical-use segment is expected to grow lucratively during the forecast period owing to the expanding pipeline for therapies. The number of cancer cellular therapies in the pipeline rose from 753 in 2018 to 1,011 in 2019, as per Cancer Research Institute (CRI). The major application of stem cell treatment is hematopoietic stem cell transplantation for the treatment of the immune system and blood disorders for cancer patients.

In Europe, blood stem cells are used for the treatment of more than 26,000 patients each year. These factors have driven the revenue for malignancies and autoimmune disorders segment. Currently, most of the stem cells used are derived from bone marrow, blood, and umbilical cord resulting in the larger revenue share in this segment.

On the other hand, cell lines, such as Induced Pluripotent Stem Cells (iPSC) and human Embryonic Stem Cells (hESC) are recognized to possess high growth potential. As a result, a several research entities and companies are making significant investments in R&D pertaining to iPSC- and hESC-derived products.

Therapy Type Insights of Cell Therapy Market

An inclination of physicians towards therapeutic use of autologous and allogeneic cord blood coupled with rising awareness about the use of cord cells and tissues across various therapeutic areas is driving revenue generation. Currently, the allogeneic therapies segment accounted for the largest share in 2019 in the cell therapy market. The presence of a substantial number of approved products for clinical use has led to the large revenue share of this segment.

Furthermore, the practice of autologous tissue transplantation is restricted by the limited availability of healthy tissue in the patient. Moreover, this type of tissue transplantation is not recommended for young patients wherein tissues are in the growth and development phase. Allogeneic tissue transplantation has effectively addressed the above-mentioned challenges associated with the use of autologous transplantation.

However, autologous therapies are growing at the fastest growth rate owing to various advantages over allogeneic therapies, which are expected to boost adoption in this segment. Various advantages include easy availability, no need for HLA-matched donor identification, lower risk of life-threatening complications, a rare occurrence of graft failure, and low mortality rate.

Regional Insights of Cell Therapy Market

The presence of leading universities such as the Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, and Yale Stem Cell Center that support research activities in U.S. is one of the key factor driving the market for cell therapy in North America. Moreover, strong regulatory and financing support from the federal bodies for expansion of this arena in U.S. as well as Canada is driving the market.In Asia Pacific, the market is anticipated to emerge as a lucrative source of revenue owing to the availability of therapies at lower prices coupled with growing awareness among the healthcare entities and patients pertaining the potential of these therapies in chronic disease management. Japan is leading the Asian market for cell therapy, which can be attributed to its fast growth as a hub for research on regenerative medicine.

Moreover, the Japan government has recognized regenerative medicine and cell therapy as a key contributor to the countrys economic growth. This has positively influenced the attention of global players towards the Asian market, thereby driving marketing operations in the region.

Market Share Insights of Cell Therapy Market

Some key companies operating in this market for cell therapy are Fibrocell Science, Inc.; JCR Pharmaceuticals Co. Ltd.; Kolon TissueGene, Inc.; PHARMICELL Co., Ltd.; Osiris Therapeutics, Inc.; MEDIPOST; Cells for Cells; NuVasive, Inc.; Stemedica Cell Technologies, Inc.; Vericel Corporation; and ANTEROGEN.CO.,LTD. These companies are collaborating with the blood centers and plasma collection centers in order to obtain cells for use in therapeutics development.

Several companies have marked their presence in the market by acquiring small and emerging therapy developers. For instance, in August 2019, Bayer acquired BlueRock Therapeutics to establish its position in the market for cell therapy. BlueRock Therapeutics is a U.S. company that relies on a proprietary induced pluripotent stem cell (iPSC) platform for cell therapy development.

Several companies are making an entry in the space through the Contract Development and Manufacturing Organization (CDMO) business model. For example, in April 2019, Hitachi Chemical Co. Ltd. acquired apceth Biopharma GmbH to expand its global footprint in the CDMO market for cell and gene therapy manufacturing.

In September 2020, Takeda Pharmaceutical Company Limited announced the expansion of its cell therapy manufacturing capabilities with the opening of a new 24,000 square-foot R&D cell therapy manufacturing facility at its R&D headquarters in Boston, Massachusetts. The facility provides end-to-end research and development capabilities and will accelerate Takedas efforts to develop next-generation cell therapies, initially focused on oncology with the potential to expand into other therapeutic areas.

The R&D cell therapy manufacturing facility will produce cell therapies for clinical evaluation from discovery through pivotal Phase 2b trials. The current Good Manufacturing Practices (cGMP) facility is designed to meet all U.S., E.U., and Japanese regulatory requirements for cell therapy manufacturing to support Takeda clinical trials around the world.

The proximity and structure of Takedas cell therapy teams allow them to quickly apply what they learn across a diverse portfolio of next-generation cell therapies including CAR NKs, armored CAR-Ts, and gamma delta T cells. Insights gained in manufacturing and clinical development can be quickly shared across global research, manufacturing, and quality teams, a critical ability in their effort to deliver potentially transformative treatments to patients as fast as possible.

Takeda and MD Anderson are developing a potential best-in-class allogeneic cell therapy product (TAK-007), a Phase 1/2 CD19-targeted chimeric antigen receptor-directed natural killer (CAR-NK) cell therapy with the potential for off-the-shelf use being studied in patients with relapsed or refractory non-Hodgkins lymphoma (NHL) and chronic lymphocytic leukemia (CLL). Two additional Phase 1 studies of Takeda cell therapy programs were also recently initiated: 19(T2)28z1xx CAR T cells (TAK-940), a next-generation CAR-T signaling domain developed in partnership with Memorial Sloan Kettering Cancer Center (MSK) to treat relapsed/refractory B-cell cancers, and a cytokine and chemokine armored CAR-T (TAK-102) developed in partnership with Noile-Immune Biotech to treat GPC3-expressing previously treated solid tumors.

Takedas Cell Therapy Translational Engine (CTTE) connects clinical translational science, product design, development, and manufacturing through each phase of research, development, and commercialization. It provides bioengineering, chemistry, manufacturing and control (CMC), data management, analytical and clinical and translational capabilities in a single footprint to overcome many of the manufacturing challenges experienced in cell therapy development.

Segmentations, Sub Segmentations, CAGR, & High-Level Analysis overview of Cell Therapy Market Research ReportThis report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2019 to 2030. For the purpose of this study, this market research report has segmented the global cell therapy market on the basis of use-type, therapy-type, and region:

Use-Type Outlook (Revenue, USD Million, 2019 2030)

Clinical-use

By Therapeutic Area

By Cell Type

Non-stem Cell Therapies

Therapy Type Outlook (Revenue, USD Million, 2019 2030)

Looking for more? Check out our repository for all available reports on Cell Therapy in related sectors.

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Cell Therapy Market Size, Share, Market Research and Industry Forecast Report, 2020-2027 (Includes Business Impact of COVID-19) - Eurowire

In simple words, apoptosis assays refers to programmed cell deaths, which are able to regulate genetically cell ablation over the period of normal development. It is utilized for the purpose of elimination of unhealthy, unnecessary, and old cells sans any release of harmful materials into the adjoining areas. The morphological attributes of apoptotic cells comprise production of membrane-bound apoptotic bodies, cytoplasm contraction, and chromatin compaction. The growing importance the assay in biotechnology sector is expected to foster development of the global apoptosis assay market in the years to come.

The rising incidences of chronic and infectious diseases across the globe play an important growth factor for the global apoptosis assay market. In addition to that, a rise in the number of cell-based research projects together with increased allocation of funding for cancer research projects is likely to pave way for the development of the global apoptosis assay market over the tenure of analysis, from 2019 to 2029.

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Product, assay type, technique, end use, and region are the five key parameters based on which the global Apoptosis Assay market has been divided. The objective of such segmentation is to offer a clearer, 360-degree view of the market.

Global Apoptosis Assay Market: Notable Developments

The global apoptosis assay market has witnessed significant developments in the recent years. One of such developments pertaining to the market is mentioned below:

Some of the key players in the global apoptosis assay market comprise the below-mentioned:

Global Apoptosis Assay Market: Key Trends

The following drivers, restraints, and opportunities characterize the global apoptosis assay market over the assessment period, from 2019 to 2029.

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There have been on-going developments pertaining to the apoptosis-modulating drugs, which is likely to widen the scope of growth of the global apoptosis assay market over the forecast timeframe, from 2019 to 2029. Extensive use of molecular targeting and apoptosis assays for the purpose of treatment of various chronic diseases and cancer is anticipated to fuel growth of the market in the years to come.

In addition, increased prevalence of autoimmune diseases, such as multiple sclerosis, systemic lupus erythematosus are likely to work in favor of the market over the period of analysis, from 2019 to 2029. According to the findings of American Autoimmune Related Disease Association (AARDA), nearly 50 million Americans were suffering from one or more autoimmune disease in 2018. Presence of such huge base of patients in America only indicates towards vast pool of such patients worldwide, which is likely to augur well for the global apoptosis assay market in the near future.

Global Apoptosis Assay Market: Geographical Analysis

North America region is expected to account for most of the revenue contribution of the global apoptosis assay market and the region is expected to dominate the market throughout the period of analysis, from 2019 to 2029. The US is estimated to be one of the major contributors of the global apoptosis assay market.

The global apoptosis assay market is segmented as:

Product

Technique

Assay Type

End Use

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Apoptosis Assay Market - Rising Incidences of Chronic and Infectious Diseases Drive the Market Growth - BioSpace

Oct. 13, 2020 12:01 UTC

Organizations to work together to integrate Be The Match BioTherapies existing cell therapy infrastructure to manage the supply chain for potential commercialization of omidubicel

BOSTON & MINNEAPOLIS--(BUSINESS WIRE)-- Gamida Cell Ltd.. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and Be The Match BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies, today announced an expansion of their existing strategic collaboration for omidubicel, Gamida Cells advanced cell therapy in Phase 3 clinical development as a potentially life-saving treatment option for patients in need of an allogeneic hematopoietic stem cell (bone marrow) transplant. The broadened agreement represents an important step in both organizations patient access efforts and in Gamida Cells preparation for potential approval by the U.S. Food and Drug Administration (FDA).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201013005030/en/

The original partnership agreement between the organizations focused on the omidubicel development program and leveraged a wide range of Be The Match BioTherapies capabilities and services. This included providing cellular source material from the Be The Match Registry, which offers the most ethnically diverse listing of potential donors and umbilical cord blood units in the world, with 22 million potential donors and more than 300,000 umbilical cord blood units, as well as cell therapy supply chain and logistics management services. In building upon the existing collaboration, Gamida Cell will work through Be The Match BioTherapies for the ordering and supply of cord blood units, which serve as the starting material for omidubicel. The expanded agreement is designed to provide a smooth process throughout the omidubicel therapy supply chain.

Gamida Cells work to bring a new stem cell graft source to patients aligns with our core mission to help organizations deliver cellular therapies that can save more lives and improve the quality of life for patients, said Amy Ronneberg, chief executive officer of the National Marrow Donor Program/Be The Match and Be The Match BioTherapies. Were delighted to expand upon our collaboration to more fully leverage our infrastructure and technology to support the advancement of Gamida Cells efforts to seamlessly bring omidubicel to patients in clinical and commercial settings. Gamida Cells efforts to make stem cell transplant more accessible to patients could be particularly impactful for patients who do not have a matched donor of suitable age.

Be The Match BioTherapies is a respected leader in cell therapy and has an extensive history of assuring broad transplant access through the delivery of source material, globally, for patients in need of a transplant, stated Michele Korfin, chief operating and commercial officer of Gamida Cell. Deepening our collaboration represents an important step for Gamida Cell as the company increases its focus on potentially bringing omidubicel to patients in the commercial setting after reporting that omidubicel met its primary endpoint and all three secondary endpoints in our randomized, multi-center Phase 3 study. We look forward to our continued collaboration with Be The Match BioTherapies to ensure that we have an efficient and reliable cell therapy supply chain that can provide a positive experience for transplant teams and their patients.

In May, Gamida Cell reported that its Phase 3 study of omidubicel met its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a stem cell transplant. Additionally, in October, Gamida Cell reported that all three secondary endpoints for the study related to platelet engraftment, infections and hospitalizations demonstrated statistical significance. Gamida Cell expects to begin submitting the biologics license application for omidubicel to the FDA on a rolling basis in the fourth quarter of 2020.

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including difficulty in finding a matched donor. Omidubicel is designed to potentially serve as a universal alternative to existing donor sources for bone marrow transplant.

About Omidubicel Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In clinical studies (NCT01816230 and NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About Gamida Cell Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

About Be The Match BioTherapies Be The Match BioTherapies is the only cell and gene therapy solutions provider with customizable services to support the end-to-end cell therapy supply chain. Backed by the industry-leading experience of the National Marrow Donor Program (NMDP)/Be The Match, and a research partnership with the CIBMTR (Center for International Blood and Marrow Transplant Research), the organization designs solutions that advance the development of cell and gene therapies across the globe.

Be The Match BioTherapies is dedicated to accelerating patient access to life-saving cell and gene therapies by providing high-quality cellular source material from the Be The Match Registry, the worlds largest and most diverse registry of more than 22 million potential blood stem cell donors and more than 300,000 umbilical cord blood units. Through established relationships with apheresis, marrow collection and transplant centers worldwide, the organization develops, onboards, trains and manages expansive collection networks to advance cell therapies. Be The Match BioTherapies uses a proven integrated model of both cell therapy supply chain and logistics managers, complimented by regulatory compliance experts to successfully transport and deliver life-saving therapies across the globe. Through the CIBMTR, Be The Match BioTherapies extends services beyond the cell therapy supply chain to include long-term follow-up tracking for the first two FDA-approved CAR-T therapies.

For more information, visit http://www.BeTheMatchBioTherapies.com or follow Be The Match BioTherapies on LinkedIn or Twitter at @BTMBioTherapies.

Gamida Cell Forward Looking Statements This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the effect on any cell therapy supply chain or Gamida Cells anticipated timing regulatory filing submissions for omidubicel, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the ongoing global COVID-19 pandemic and manufacturing, clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

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Gamida Cell and Be The Match BioTherapies Expand Strategic Collaboration - BioSpace

Find out which biopharma companies are raking in the cash this week, as companies from around the globe provide updates on their financing rounds and IPOs.

Evotec

The UAE dropped a load of cash to become a 5.6% stakeholder into Germanys Evotec with $236 million worth of shares purchased by their sovereign wealth fund, Mubadala Investment Company. Not one to be left out, existing investor Novo increased its stake to 11% by throwing in another $59 million, bringing Evotecs haul to about $295 million. Evotec is a drug discovery alliance and development partnership company out of Hamburg, Germany. With revenues nearly quadrupling over the last five years and a goal of 10% yearly growth in the future, its no surprise these investors want in big. Evotec plans to use the cash to reach its goals by growing, particularly in the U.S. and Europe as they ramp up global ops.

SQZ Biotech

Allied with Roche to develop new cancer cell therapies, SQZ has turned its focus to the NYSE. With a preliminary goal of $75 million for its IPO, theyve applied for listing as SQZ. While traditional cell therapies require a depletion of the immune cells to improve efficacy, SQZ claims to avoid those limitations with a proprietary technology that, as its name touts, squeezes cells through a microfluidic chip to open the cell membrane and allow the therapeutic load inside. They also tout a huge leg up in manufacturing 24-hour turnaround in comparison with a month or more for current therapies. Plus, without the pre-conditioning to weaken immune systems, SQZs technology aims to shorten hospital stays. The IPO earnings are intended to finance their groundbreaking cancer and infectious disease research.

Galecto Biotech

Just two weeks after completing a $64 million Series D round, Galecto Biotech rounds the corner and goes after the public market, hoping to raise $100 million in its IPO. If successful, the Copenhagen-based company will have totaled over $250 million in financing in just the last two years. Galectos focus is on a wide range of fibrotic disease, with its lead project, GB0139 for idiopathic pulmonary fibrosis, currently in a Phase IIb trial. The Series D and the new funding from this IPO will go toward getting the program through to approval and commercialization.

Codiak BioSciences

After filing to go public for a second time after withdrawing in 2019, Codiak finally hit the market with an $83 million IPO, falling short of its $100 million originally sought when filed in September. The bulk of the funds will be used to advance its lead program ExoSTING through a phase study in advanced or metastatic, recurrent solid tumors, support discover and preclinical R&Dand expand its engEx technology that supports its programs. Another $10 million will go into its second program, exoIL-12, through a Phase I trial in patients with cutaneous T-cell lymphoma.

EdiGene

A Series B of $67 million takes EdiGenes track record up to $100 million raised in the last two years. The Beijing-based biotech is currently leading the gene-editing wave in China with four platforms steadily advancing. The company's top candidate is a treatment for hereditary blood disease, with the next in line being a CAR-T treatment for cancer. CEO Dong Wei hopes their T cell therapeutics can help make a higher quality, lower cost option for patients and their families.

Cedilla Therapeutics

Small molecule-focused Cedilla wraps up a $57.6 million Series B round to drug the undruggable. The funding will go into preclinical work on its first two oncology candidates, which are being kept hush hush for now. They also have about five or six oncology programs running that are years away from the clinic. In addition to the Series B, Cedilla is bringing Casdin CIO and founder Eli Casdin and Boxer senior VP Dominik Naczynski onto its board of directors.

RayzeBio

Debuting with $45 million in Series A money, biotech newbie RayzeBio is ready to defeat cancer with radiopharmaceuticals. With a vision to be the first radiopharma platform in the market, RayzeBio has seven active programs and would like to see one development candidate by the second half of 2021. Radiopharmaceuticals have intrigued the biotech sphere lately, but securing a reliable supply of therapeutic radioisotopes has been a hang up. But recently the industry has devised alternate ways to generate Actinium-225, which is the radioisotope RayzeBio is working with. This new development spurred the drive to launch RayzeBio with the intent to penetrate specific tumor targets. The fledgling biotech is now rolling up its sleeves to get to work with the goal of being first.

Priothera Limited

To get more clinical data on its highly-promising therapy for high risk AML patients, Priothera closed on a $35 million USD Series A. The company's drug mocravimod should enhance the curative potential of allogeneic hematopoietic stem cell transplantation for treating AML. Allogeneic stem cell transplant is currently the only potentially curative approach for AML patients, but has a high mortality rate. This therapy appears promising for improving survival outcomes. Priothera acquired mocravimod from KYORIN Pharmaceutical.

Ori Biotech

Ori Biotech wants to speed up the innovation of cell and gene therapies via its manufacturing platform, and this weeks $30 million Series A is certainly a step in reaching that goal. Typically, a drug discovery pipeline can take an average of a decade to get from lab to patient. Oris platform closes, automates and standardizes manufacturing for cell and gene therapy developers so the company can move its treatments from pre-clinical to scale commercially. This novel automation will reduce cost of goods and the footprint. In addition to taking its platform to the market, Ori is also expecting to double its 8-head employee count in four months, and double that again by next year.

Kanaph Therapeutics

Kanaph beefs up its initial $8 million start in 2019 with a $21 million Series B in South Korea. This round of funding will go toward expediting the clinical development of Kanaphs pipelines, chiefly its TMEkine molecules platform for immuno-oncology and bi-specific Fc fusions for the treatment of retinal disease. Preclinical studies are anticipated to be completed at the end of this year or beginning of next year, and are ready for the next steps.

Rappta Therapeutics

Novo Seeds plants its stake in emerging biotech Rappta Therapeutics in a $10.5 million Series A round. Rapptas primary focus is developing first-in-class anti-cancer drugs that work by activating protein phosphatase 2A (PP2A). The PP2A enzyme is a key tumor suppressor which has historically been tricky to target with drugs. Rappta has derived a unique understanding of the protein along with propriety tools to allow therapeutic reactivation of PP2A, which offers the potential of multiple therapies with this as the platform for a new class of anti-cancer drugs. Jeroen Bakker, Principal at Novo Seeds, will join Rapptas board. Novartis Venture Fund, Advent Life Sciences and one family office also participated in the round.

Lixte Biotechnology

Previously listed on the OTCQB, Lixte is ready to take it to Nasdaq with a $9 million offering of 1.5 million shares at a price range of $5.75 to $6.75. The NY-based biotech has developed two active series LB-100 and LB-200. The current focus is on the LB-100, which targets several types of cancer and has potential for vascular and metabolic diseases. A Phase I trial has already been completed and demonstrated antitumor activity in humans. LB-100 is now in Phase Ib/II.

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Biopharma Money on the Move: October 7-14 - BioSpace

If I had to place money on a neurotech that will win the Nobel Prize, its optogenetics.

The technology uses light of different frequencies to control the brain. Its a brilliant mind-meld of basic neurobiology and engineering that hijacks the mechanism behind how neurons naturally activateor are silencedin the brain.

Thanks to optogenetics, in just ten years weve been able to artificially incept memories in mice, decipher brain signals that lead to pain, untangle the neural code for addiction, reverse depression, restore rudimentary sight in blinded mice, and overwrite terrible memories with happy ones. Optogenetics is akin to a universal programming language for the brain.

But its got two serious downfalls: it requires gene therapy, and it needs brain surgery to implant optical fibers into the brain.

This week, the original mind behind optogenetics is back with an update that cuts the cord. Dr. Karl Deisseroths team at Stanford University, in collaboration with the University of Minnesota, unveiled an upgraded version of optogenetics that controls behavior without the need for surgery. Rather, the system shines light through the skulls of mice, and it penetrates deep into the brain. With light pulses, the team was able to change how likely a mouse was to have seizures, or reprogram its brain so it preferred social company.

To be clear: were far off from scientists controlling your brain with flashlights. The key to optogenetics is genetic engineeringwithout it, neurons (including yours) dont naturally respond to light.

However, looking ahead, the study is a sure-footed step towards transforming a powerful research technology into a clinical therapy that could potentially help people with neurological problems, such as depression or epilepsy. We are still far from that visionbut the study suggests its science fiction potentially within reach.

To understand optogenetics, we need to dig a little deeper into how brains work.

Essentially, neurons operate on electricity with an additional dash of chemistry. A brain cell is like a living storage container with doorscalled ion channelsthat separate its internal environment from the outside. When a neuron receives input and that input is sufficiently strong, the cells open their doors. This process generates an electrical current, which then gallops down a neurons output brancha biological highway of sorts. At the terminal, the electrical data transforms into dozens of chemical ships, which float across a gap between neurons to deliver the message to its neighbors. This is how neurons in a network communicate, and how that network in turn produces memories, emotions, and behaviors.

Optogenetics hijacks this process.

Using viruses, scientists can add a gene for opsins, a special family of proteins from algae, into living neurons. Opsins are specialized doors that open under certain frequencies of light pulses, something mammalian brain cells cant do. Adding opsins into mouse neurons (or ours) essentially gives them the superpower to respond to light. In classic optogenetics, scientists implant optical fibers near opsin-dotted neurons to deliver the light stimulation. Computer-programmed light pulses can then target these newly light-sensitive neurons in a particular region of the brain and control their activity like puppets on a string.

It gets cooler. Using genetic engineering, scientists can also fine-tune which populations of neurons get that extra powerfor example, only those that encode a recent memory, or those involved in depression or epilepsy. This makes it possible to play with those neural circuits using light, while the rest of the brain hums along.

This selectivity is partially why optogenetics is so powerful. But its not all ponies and rainbows. As you can imagine, mice dont particularly enjoy being tethered by optical fibers sprouting from their brains. Humans dont either, hence the hiccup in adopting the tool for clinical use. Since its introduction, a main goal for next-generation optogenetics has been to cut the cord.

In the new study, the Deisseroth team started with a main goal: lets ditch the need for surgical implants altogether. Immediately, this presents a tough problem. It means that bioengineered neurons, inside a brain, need to have a sensitive and powerful enough opsin door that responds to lighteven when light pulses are diffused by the skull and brain tissue. Its like a game of telephone where one person yells a message from ten blocks away, through multiple walls and city noise, yet you still have to be able to decipher it and pass it on.

Luckily, the team already had a candidate, one so good its a ChRmine (bad joke cringe). Developed last year, ChRmine stands out in its shockingly fast reaction times to light and its ability to generate a large electrical current in neuronsabout a 100-fold improvement over any of its predecessors. Because its so sensitive, it means that even a spark of light, at its preferred wavelength, can cause it to open its doors and in turn control neural activity. Whats more, ChRmine rapidly shuts down after it opens, meaning that it doesnt overstimulate neurons but rather follows their natural activation trajectory.

As a first test, the team used viruses to add ChRmine to an area deep inside the brainthe ventral tegmental area (VTA), which is critical to how we process reward and addiction, and is also implicated in depression. As of now, the only way to reach the area in a clinical setting is with an implanted electrode. With ChRmine, however, the team found that a light source, placed right outside the mices scalp, was able to reliably spark neural activity in the region.

Randomly activating neurons with light, while impressive, may not be all that useful. The next test is whether its possible to control a mouses behavior using light from outside the brain. Here, the team added ChRmine to dopamine neurons in a mouse, which in this case provides a feeling of pleasure. Compared to their peers, the light-enhanced mice were far more eager to press a lever to deliver light to their scalpsmeaning that the light is stimulating the neurons enough for the mice to feel pleasure and work for it.

As a more complicated test, the team then used light to control a population of brain cells, called serotonergic cells, in the base of the brain, called the brainstem. These cells are known to influence social behaviorthat is, how much an individual enjoys social interaction. It gets slightly disturbing: mice with ChRmine-enhanced cells, specifically in the brainstem, preferred spending time in their test chambers social zone versus their siblings who didnt have ChRmine. In other words, without any open-brain surgery and just a few light beams, the team was able to change a socially ambivalent mouse into a friendship-craving social butterfly.

If youre thinking creepy, youre not alone. The study suggests that with an injection of a virus carrying the ChRmine geneeither through the eye socket or through veinsits potentially possible to control something as integral to a personality as sociability with nothing but light.

To stress my point: this is only possible in mice for now. Our brains are far larger, which means light scattering through the skull and penetrating sufficiently deep becomes far more complicated. And again, our brain cells dont normally respond to light. Youd have to volunteer for what amounts to gene therapywhich comes with its own slew of problemsbefore this could potentially work. So keep those tin-foil hats off; scientists cant yet change an introvert (like me) into an extrovert with lasers.

But for unraveling the inner workings of the brain, its an amazing leap into the future. So far, efforts at cutting the optical cord for optogenetics have come with the knee-capped ability to go deep into the brain, limiting control to only surface brain regions such as the cortex. Other methods overheat sensitive brain tissue and culminate in damage. Yet others act as 1990s DOS systems, with significant delay between a command (activate!) and the neurons response.

This brain-control OS, though not yet perfect, resolves those problems. Unlike Neuralink and other neural implants, the study suggests its possible to control the brain without surgery or implants. All you need is light.

Image Credit: othebo from Pixabay

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Scientists Found a New Way to Control the Brain With LightNo Surgery Required - Singularity Hub

NEW YORK, Oct. 12, 2020 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced Stacy Lindborg, Ph.D., Executive Vice President and Head of Global Clinical Research, will deliver a presentation at the 2020 Cell & Gene Meeting on the Mesa, being held virtually October 12-16, 2020.

Dr. Lindborg's presentation will be in the form of an on-demand webinar that will be available beginning today. Those who wish to listen to the presentation are required to register here. At the conclusion of the 2020 Cell & Gene Meeting on the Mesa, a copy of the presentation will also be available in the "Investors and Media" section of the BrainStorm website under Events and Presentations.

About the 2020 Cell & Gene Meeting on the Mesa

The conference will feature 80+ on-demand company presentations by leading public and private companies, highlighting their technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, and tissue engineering. Registrants will have access to 15+ expert-led panels and workshops including a mix of both live and on-demand sessions. The conference will be delivered in a virtual format over the course of five days October 12-16. There is also a premier partnering system, partneringONE, allowing registrants to plan 11 meetings with other attendees. For a list of presenting companies, refer to https://www.meetingonthemesa.com/company-presentations/.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com.

Contacts Investor Relations:Corey Davis, Ph.D.LifeSci Advisors, LLCPhone: +1 646-465-1138cdavis@lifesciadvisors.com

Media:Paul TyahlaSmithSolvePhone: + 1.973.713.3768Paul.tyahla@smithsolve.com

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SOURCE Brainstorm Cell Therapeutics Inc

Company Codes: NASDAQ-SMALL:BCLI

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BrainStorm to Present at the 2020 Cell & Gene Meeting on the Mesa - BioSpace

The recent surge in interest in genetically-modified therapies has resulted in a steep rise in demand for different vectors for fundamental and pharmacological research, opening up opportunities for companies with expertise in targeted gene delivery

Roots Analysis has announced the addition of Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA and Other Vectors) report to its list of offerings.

Currently, biopharmaceutical developers are plagued by high development costs, complex production protocols and the need for specialized equipment, in order to ensure the safety and quality of complex biological interventions, such as cell and gene therapies. Consequently, a number of innovator companies have demonstrated a preference for outsourcing vector manufacturing, a key aspect of advanced, genetically-modified product development, to contract service providers.

To order this 430+ page report, which features 140+ figures and 180+ tables, please visit this link

Key Market Insights

The market is fragmented, with over 180 industry players and non-industry playersOver 50% of industry players are large or mid-sized firms. In recent years, the growing demand for vectors has spurred the establishment of several start-ups, as well. Further, more than 80 non-industry players, including universities, research institutes and hospitals, are also currently involved in producing viral vectors or plasmid DNA for use in genetically modified therapies

The demand for vectors for research / clinical use is presently more than that for commercial applicationsApproximately 80% of industry stakeholders presently claim to manufacture vectors at the laboratory and / or clinical scale. However, some firms (around 40, as per our research) have developed / are developing commercial scale capacity for the production of viral vectors or plasmid DNA.

The US and EU have emerged as major vector manufacturing hubsHigh volume of active clinical studies, requiring vectors, being conducted in these regions makes the US and EU the major vector manufacturing hubs. Approximately 50% of the vector manufacturing facilities are located in North America. This is followed by the EU, where approximately 45% of the worlds vector manufacturing facilities are located.

The current installed vector manufacturing capacity in the world is estimated to be over 60,000 LThe major share (70%) of the global vector manufacturing capacity belongs to companies that are manufacturing vectors at both clinical and commercial scales. Across the major global regions, 50% of the total installed vector manufacturing capacity is in the US. This can be attributed to the large number of small-sized and mid-sized companies that are presently situated in this region.

Around 140 partnerships were inked during the last three yearsThe maximum number of deals (37) were reported in 2016, followed by 27 partnerships established in 2018. Around 30% of the deals were related to the manufacturing of vectors across different scales of operation; this was followed by technology licensing agreements (23%).

90% of the market share is captured by viral vectors intended for use in oncological disordersDriven by the rapidly evolving pipeline of genetically modified therapies, including T-cell therapies and vector-based vaccines, and the increasing adoption of advanced production technologies, the vector manufacturing market is projected to grow at an accelerated pace. Specifically, revenues generated from the sales of lentiviral vectors currently represent the largest share of the market, followed by retroviral vectors.

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Key Questions Answered

Close to USD 2 billion (by 2030) financial opportunity within the vector manufacturing market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom there is an evident need for industry stakeholders to modify operational models and expand manufacturing capabilities in order to ensure uninterrupted growth within the market. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles of several companies (including those listed below); each profile features an overview of the company, financial information (if available), vector manufacturing technology, manufacturing facilities, vector manufacturing experience and an informed future outlook of the company.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/viral-vectors-non-viral-vectors-and-gene-therapy-manufacturing-market-3rd-edition-2019-2030-focus-on-aav-adenoviral-lentiviral-retroviral-plasmid-dna-and-other-vectors/274.html or email [emailprotected]

You may also be interested in the following titles:

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

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Viral and Non-Viral Vector Manufacturing Market is anticipated to grow at an annualized rate of over 20%, claims Roots Analysis - The Think Curiouser

The University of Manchester, part of the prestigious Russell Group of universities, has announced today a groundbreaking gene therapy partnership to ease the lifelong suffering of people with Hunter syndrome.

The University has agreed to a worldwide license and collaborative research funding agreement with AVROBIO, Inc., a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, based in Cambridge, Massachusetts, USA.

The significant partnership agreement is for the clinical development of an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, a rare and deadly lysosomal disorder that primarily affects young boys.

Hunter syndrome, which affects an estimated one in 100,000 males worldwide, causes devastating complications throughout the body and brain, including severe cardiac and respiratory dysfunction, skeletal malformations and hearing impairment. Children with severe cases of Hunter syndrome typically show early symptoms in their toddler years and begin to regress developmentally around age six, losing basic motor skills and cognitive function.

The current standard of care is weekly enzyme replacement therapy (ERT), which can delay some complications but does not halt overall progression of the disease and has not been demonstrated to address cognitive issues. Even with ERT, people with Hunter syndrome face life-limiting symptoms and a significantly reduced life span.

The University of Manchester will sponsor the investigator-led Phase 1-2 clinical trial for Hunter syndrome which is expected to begin in 2021. The Hunter syndrome program was developed by Brian Bigger, a professor of cell and gene therapy at The University of Manchester. Professor Bigger has published preclinical data demonstrating that the introduction of the transgene with an optimised, proprietary tag has the ability to correct peripheral disease and normalise brain pathology.

Primary investigators for the clinical trial will be; Professor Robert Wynn, Consultant Paediatric Hematologist at the Royal Manchester Childrens Hospital and Dr. Simon Jones, Consultant Paediatric Physician for inherited metabolic diseases at the Willink Unit, Saint Marys Hospital and the Manchester Centre for Genomic Medicine.

We feel an enormous urgency to bring forward a treatment that may halt this deadly disease in its tracks, before symptoms emerge and before children lose their physical and cognitive skills, said Professor Bigger. We are delighted to be working with AVROBIO on this program. Both of our teams have deep experience running international clinical trials in other lysosomal disorders. AVROBIO also has a leading gene therapy platform, plato, which is designed to optimise the consistency, predictability and efficacy of its gene therapies and to enable efficient scaling for worldwide commercialization. By working together, we believe we can greatly accelerate development of this important program.

The investigational gene therapy, which will be called AVR-RD-05, involves ex vivo transduction of the patients own hematopoietic stem cells with a therapeutic transgene designed to express functional enzyme the patient needs to maintain cellular health, coupled to a proprietary protein tag that is designed to improve stability of the enzyme in the bloodstream and facilitate uptake by tissues from head to toe. When reinfused into the patient, the gene-modified stem cells are expected to engraft in the bone marrow and produce generations of daughter cells, each carrying the transgene. Those daughter cells are then expected to differentiate into macrophages, microglia and other components of the immune system and circulate throughout the body and central nervous system, potentially enabling widespread distribution of functional enzyme.

Geoff MacKay, AVROBIOs president and CEO said: The lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function. If we treat children early, before their symptoms arise, we hope to prevent the tragic complications that rob these young children of their futures.

We believe our deep experience with investigational gene therapies for lysosomal disorders will enable us to efficiently move the program through clinical development in collaboration with Professor Brian Bigger, who has done tremendous work to develop and optimize this investigational gene therapy. Were proud to add this program to our leading lysosomal disorder pipeline and excited about its potential to change the lives of patients and families living with Hunter syndrome.

The University of Manchesters technology transfer office, The University of Manchester Innovation Factory and AVROBIO have negotiated the exclusive, worldwide license to the technology. Under the terms of the license, AVROBIO will pay The University of Manchester an upfront cash payment and additional payments based on the achievement of development and regulatory milestones. The company will pay The University a mid-single digit percentage royalty on annual net sales of licensed products. Additionally, under the collaborative research funding agreement, AVROBIO will cover budgeted clinical trial costs.

Andrew Wilkinson, CEO of the Universitys technology transfer company, The University of Manchester Innovation Factory said: We are delighted that AVROBIO will be working with teams from The University of Manchester and The University of Manchester Foundation Trust to develop a therapy for this debilitating genetic disease. AVROBIOs strategic focus on bringing new personalised gene therapies to the world along with their technical and commercial expertise in this area make them an excellent partner for the investigational Hunter syndrome gene therapy programme.

About Hunter syndrome

Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), is a lysosomal disorder caused by a mutation in the IDS gene that leads to a deficiency of the lysosomal enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules called glycosaminoglycans (GAGs, also known as mucopolysaccharides). Without functional IDS, toxic levels of GAGs build up throughout the body and central nervous system, causing a wide range of symptoms including cognitive decline and cardiac and respiratory dysfunction. The current standard of care is weekly enzyme replacement therapy, which may delay some symptoms but does not halt the overall progression of disease and does not cross the blood-brain barrier, an intricate web of protective tissue that selectively prevents macromolecules from entering the brain. Even with treatment, people with Hunter syndrome face life-limiting symptoms and a significantly reduced life span. The disorder affects an estimated 1 in 100,000 males worldwide; about two-thirds of cases have an early, severe progressive form.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated using the vector into patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the viral vector.

About The University of Manchester

The University of Manchester is a member of the prestigious Russell Group and one of the UKs largest single-site universities.

We have over 40,000 students, 12,000 staff and, with almost 480,000 former students from more than 190 countries, are home to the largest alumni community of any campus-based university in the UK. No fewer than 25 Nobel laureates have either worked or studied here.

We are thetop UK University for graduate employabilityaccording to The Times and Sunday Times Good University Guide; ranked 27th in the world in the QS World University Rankings (2020) and 6th in the UK. Were also listed as 8th in Reuters Top 100: Europe's most innovative universities (2019).

Visit http://www.manchester.ac.uk for further information or https://www.manchester.ac.uk/discover/vision/ for our latest strategic vision.

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University of Manchester announces partnership with AVROBIO for Hunter syndrome gene therapy - PharmiWeb.com

David Z Wang and colleagues look at the latest advances in brain research and how they might affect treatment of brain disorders

The world has come a long way in solving the mystery of the brain, understanding its fundamental role in human consciousness and discovering methods to treat its disorders. In The Sacred Disease in ~430 BC, Hippocrates wrote that the brain served to house the ventricles, whose main purpose was to be a container and transit point for the breath or air (pneuma) from outside the bodythe force that brought to life our joys, pleasures, laughter, and grief. Thus, the brain was a reservoir for an animated substance that produced the human experience of consciousness and personality rather than the source of that activity itself.1 Our knowledge of the brain and its functional complexity remained at the level of three ventricles where our soul lies (Nemesius, da Vinci) for hundreds of years until modern neuroscience began to uncover the fine network of neuronal circuits that made up the solid substance of the brain.

With the advent of modern neuroimaging, the complex structure of the brain has been brilliantly revealed, and this has helped greatly in the treatment of many brain related disorders. Other articles in this series have provided updates on a wide range of topics, including neurodegenerative diseases, mental disorders, cerebrovascular diseases, epilepsy, monogenic neurological diseases, and in vivo brain function testing.23456 With help from gross anatomy to electronic microscopy, tissue staining to profiling, cell physiology, and synaptic chemistry, neuroscientists have elucidated the mechanisms and pathophysiology of many common brain diseases. For example, trinucleotide repeat expansion is now known to be responsible for many genetically inherited degenerative diseases such as Huntingtons disease, and amyloid precursor gene or presenilin gene mutations can cause Alzheimers disease.

On the other hand, despite centuries of discovery on mechanisms of brain disease, treatment options remain limited. Most treatments still provide only alleviation of symptoms, though recent breakthroughs in gene therapy such as onasemnogene abeparvovec-xioi to treat children with spinal muscular atrophy 7 and reperfusion therapy for acute ischaemic stroke hold the promise to truly revolutionise treatment for neurological disease. While options are available to modify disease expression with medicationssuch as in the treatment of Parkinsons disease, multiple sclerosis, and epilepsywe are far from curing them.

Entering the 21st century, perhaps we now have better ways to understand the mechanism of those brain disorders that are still a mystery and find the precise treatment. The key will likely be interdisciplinary research. Many ongoing brain health research programmes have already been multidimensional, combining neurobiology, physics, engineering, big data science, and artificial intelligence.

In the future, it is likely that humans will be able to live longer, and do so with augmented capabilities supported by machine-human interactions. One exciting advance is new ways of observing in vivo brain-wide activities at the cellular level. A real time, ultra-large scale, high resolution (RUSH) macroscope has recently been developed that can provide video-rate gigapixel imaging of biological dynamics at centimetre scale and micrometre resolution, with a data throughput of up to 5.1gigapixels a second.8 RUSH has enabled in vivo functional imaging of neural networks across the whole mouse brain at single dendrite resolution and brain-wide tracking of leucocytes during pathological processes, and the technology opens up a new horizon for large scale brain imaging to study various brain diseases at a systematic level.8

Another example is the better understanding of the precise number of brain cells needed to complete a particular task. By constructing an explicit model of face selective cells that could decode an arbitrary realistic face from face cell responses and predict the firing of cells in response to an arbitrary realistic face, Chao and colleagues identified that macaques require only 200 cells to remember a face.9 These findings have far reaching significance. For the first time, a specialised task of the brain can be attributed to a specific number and type of brain cells in a specific circuit. This may allow scientists to build artificial models of explicit brain functions and experiment with mechanisms of injury and repair at a cellular or molecular level. Such mapping may aid our understanding of brain function and recovery and guide the rebuilding of brain circuits or resection of dysfunctional brain cells rather than whole tissues. It may also help us pinpoint the cells and circuits that are responsible for addictive behaviours, from smoking to substance use disorders to gambling.

The common belief is that when a brain has been removed, brain death is imminent. However, such belief has recently been shattered. Sestan and colleagues collected brains of 6-8 month old pigs four hours after death and bathed them in specialised perfusate solutions. They found that brain cells and synapses of certain areas of brain began to recover and show signs of cellular activities.10 Their finding suggests that there may be a late window of treatment after onset of brain anoxia when brain tissue can recover, analogous to the benefit of late window thrombectomy. This discovery has taught us that brain cells can survive and recover after loss of circulation, and that favourable conditions may preserve a reservoir of resilient brain cells that are slow progressors to ischaemic necrosis.

Evidence is also emerging on how brain cells can adapt. A recent report of functional neuronal connectivity in adults without apparent loss of function after brain hemispherectomy sheds new light on brain plasticity. The study provides the first comprehensive analysis of whole brain functional connectivity across the full repertoire of resting state networks after hemispherectomy and shows preservation of resting state networks but an increase in internetwork connectivity with other functional brain networks. When hemispheric resection occurred in patients younger than 11, the retained hemisphere was able to protect the jeopardised functions by enhancing cellular interaction and synaptic activity.11

Artificial intelligence (AI) has been widely applied in clinical diagnosis and patient monitoring. Recent studies have attempted to classify or detect Alzheimers disease and other cognitive impairment,1213 acute neurological events,1415161718 focus of epilepsy, autism spectrum disorder, and attention deficit/hyperactivity disorder by using deep learning based algorithms. The data in these AI models include not only medical images but also clinical scores, in vitro diagnostic test results, and other functional and structure information.19202122232425 These studies showed high sensitivity and specificity from their test set, and work is ongoing on how to incorporate the routine use of these AI systems into a clinical setting.

The lack of a large dataset from multiple centres, the limited coverage of a disease spectrum, and unclear risk of using AI are major limitations of these blackbox systems. In contrast, Wang and colleagues have recently proposed a vascular aware unsupervised learning technique, VasNet,26 which provides the end users with explainable images, including both vascular structures and multidimensional features such as anatomical, physiological, biochemical, and cellular details. The enriched outputs could augment human decision making on treating vascular diseases and contribute to the emergence of the next generation of healthcare engineering.

The US Food and Drug Administration has already approved several automatic quantitative measurement software systems for disease classification (eg, NeuroQuant, Quantib, RAPID). Brain morphometry analysis software can automatically examine segments of brain tissue and detect minute changes. This technology can help early detection of degenerative brain diseases by comparing the results from individuals with a large dataset and images of healthy people. To take racial differences in the brain into account, some Asian companies have developed software based on datasets acquired from Asian populations (http://quant-health.com). Use of a deep learning based segmentation algorithm could improve the accuracy and test-retest stability in segmenting and measuring the volume of brain structure, abnormal lesions, perfusion deficit area, and other characteristics. The resulting quantified values could be used to assign a clinical score automatically, avoiding the variation arising from subjective measurement and interobserver inconsistency.

AI algorithms can also objectively analyse the data collected from a depth camera or wearable devices, assess behaviour, and evaluate facial expressions.272829 The quantified values produced would not be affected by the physicians experiences, and errors can be avoided since the spatial-temporal resolution of the hardware is much smaller than visual evaluation by humans. Such early detection may allow treatment of a disease before a person shows clinical signs of brain dysfunction. Quantified measurements can be used as biomarkers to monitor the progress of the disease and help evaluate the efficacy of precision therapy.

One of the potential ways of curing a brain disorder is to correct its diseased protein structure. Many neurological diseases are caused by misfolded proteins, including Huntingtons, Parkinsons, and Alzheimers disease. AlphaFold, a Google company, has successfully predicted a protein structure by using large genomic data. The 3D models of proteins that AlphaFold generates are far more accurate than any that have come beforemaking significant progress on one of the core challenges in biology. The ability to predict a proteins shape from its DNA sequence is useful to scientists because it is fundamental to understanding its role within the body, as well as diagnosing and treating diseases believed to be caused by protein misfolding.30

We have entered into an exciting new era of brain science research and discovery. With the advent of AI, advanced imaging, genomics, psychosocial analytics, and protein engineering we may be closer than ever to new precision medicine approaches to treat many brain disorders.

In the past decade, neuroscience and brain research have entered into a new era

It is now possible to understand brain physiology and pathophysiology better through direct and in vivo observation of live brain

In the coming years, artificial intelligence will likely be part of brain science and assist or replace certain brain function

Genetic or protein alterations may provide a cure for many brain disorders in the near future

Contributors and sources: DZW drafted the first manuscript. LHS, TYQ, and QHD critically reviewed and revised the manuscript. DZW is an expert in stroke clinical research. LHS is an expert in neuroscience research and stroke care quality improvement. TYQ is an expert in big data and artificial intelligence. QHD is an expert in brain research and artificial intelligence.

Competing interests: We have read and understood BMJ policy on declaration of interests and declare that we have no competing interest.

Provenance and peer review: Commissioned; externally peer reviewed.

This article is part of a series launched at the Chinese Stroke Association annual conferenceon 10 October 2020,Beijing, China.Open access fees were funded by the National Science and Technology Major Project. The BMJ peer reviewed, edited, and made the decision to publish these articles.

Gupta A, Ayhan M, Maida A. Natural image bases to represent neuroimaging data. Proceedings of 30th international conference on machine learning. Vol 28. Atlanta, GA. 2013:987-94.

Evans R, Jumper J, Kirkpatrick J, et al. De novo structure prediction with deep-learning based scoring. In: Thirteenth critical assessment of techniques for protein structure prediction. Abstracts, 1-4 December 2018.

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Decoding the brain through researchthe future of brain health - The BMJ

Cancer Gene Therapy Market report offers in-depth analysis of the industry size, share, major segments, and different geographic regions, forecast for the next five years, key market players, and premium industry trends. It also focuses on the key drivers, restraints, opportunities and industry challenges.

Growing prevalence of cancer with rising mortality rates will augment cancer gene therapy industry forecast in the coming years. Cancer therapies incorporated with genetically modified genes ai in blocking the growth of the tumors.

Efficient PCR and isothermal amplification technologies for detecting mutations and CRISPR gene editing tools are some technical developments. These advancements have led to innovations and ensured availability of advanced cancer gene therapies driving the industry trends.

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Estimates have stated that the global cancer gene therapy market value is likely to cross an annual valuation of USD 2.5 billion by 2025.

Technological developments are majorly fueling cancer gene therapy growth. Genetically modified genes which block tumor growth have been incorporated into recent cancer therapies. Efficient isothermal amplification techniques and PCR technology are transforming the way gene mutations are detected. Recent launch of CRISPR gene editing tools is claimed to help enhance the process of gene therapy development. These innovations and advancements in technology are anticipated to propel cancer gene therapy industry size.

The in-vivo segment is estimated to witness about 22% growth over the forecast period owing to its multiple offered benefits. In-vivo gene therapy consists of direct delivery of therapeutic genes into the target cell, a process which has shown effective results in cancer treatment. Viral vectors are delivered using in-vivo gene therapy which help in stopping the activity of tumor inducing genes and has exhibited positive results in clinical trials.

Biopharmaceutical firms held approximately 48% of the industry revenue share in 2018. Major firms such as Roche and Novartis are working on cancer gene therapies that have high adoption rate of both non-viral and viral vectors. These firms are also carrying out clinical trials that are favoring the demand for such vectors, consequently driving market growth.

Broadening awareness regarding the availability of advanced cancer therapies have stimulated the market for cancer gene therapy in China. Government initiatives and funds have encouraged researchers to carrying out extensive R&D activities linked to cancer gene therapy.

Meanwhile, on a global landscape, companies like Vigene Biosciences, Cobra, Uniqure, Sirion Biotech, Bluebird Bio, Caribou, Ziopharm, Finvector, Cellectis and Sarepta Therapeutics are leading the cancer gene therapy market. These firms are focusing on enhancing their market position through business strategies such as product launches, mergers and acquisitions, among others.

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Partial Chapter of the Table of Content

Chapter 4. Cancer Gene Therapy Market, By Type

4.1. Key segment trends

4.2. Ex-vivo

4.2.1. Market size, by region, 2014 2025 (USD Million)

4.3. In-vivo

4.3.1. Market size, by region, 2014 2025 (USD Million)

Chapter 5. Cancer Gene Therapy Market, By Product

5.1. Key segment trends

5.2. Viral vectors

5.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.2. Adenoviruses

5.2.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.3. Lentiviruses

5.2.3.1. Market size, by region, 2014 2025 (USD Million)

5.2.4. Retrovirus

5.2.4.1. Market size, by region, 2014 2025 (USD Million)

5.2.5. Adeno associated virus

5.2.5.1. Market size, by region, 2014 2025 (USD Million)

5.2.6. Herpes simplex virus

5.2.6.1. Market size, by region, 2014 2025 (USD Million)

5.2.7. Vaccinia virus

5.2.7.1. Market size, by region, 2014 2025 (USD Million)

5.2.8. Others

5.2.8.1. Market size, by region, 2014 2025 (USD Million)

5.3. Non-viral vectors

5.3.1. Market size, by region, 2014 2025 (USD Million)

5.4. Others

5.4.1. Market size, by region, 2014 2025 (USD Million)

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Cancer Gene Therapy Market 2020 by industry trends, statistics, key companies growth and regional forecast - News by Decresearch

Strategic Acquisitions Lead to Innovations in Freezers and Bioproduction of Gene Therapies

Companies in the cryopreservation equipment market are focusing on strategic acquisitions to expand their product portfolio. For instance, BioLife - a manufacturer of cryopreservation freeze media, announced its acquisition of Custom Biogenic Solutions - a producer of cryopreservation equipment for the biotech industry, to expand its portfolio of liquid nitrogen laboratory freezers and other cryogenic equipment.

Strategic acquisitions have led to rise in investments in improving the technology of freezers. As such, freezers segment is expected to account for the highest revenue of the cryopreservation equipment market. The segment is projected to reach a value of ~US$ 3.5 Bn by the end of 2027. Companies are increasing efficacy of cloud-based monitoring systems that help in evaluating biologic sample storage conditions.

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Developments in cryogen techniques have led to innovations in liquid nitrogen laboratory freezers. As such, liquid nitrogen cryogen segment dominates the cryopreservation equipment market and is projected to reach a value of ~US$ 5.1 Bn by 2027. Companies are tapping into opportunities for the development of tools for bioproduction of cell and gene therapies.

Automated Systems in Sample-prep Support Cryo-em Analysis for Protein and Drug Discovery

The cryopreservation equipment market is witnessing innovations in advanced sample preparation systems. For instance, TTP Labtech - a manufacturer of products within sample management, announced the launch of its next-gen automated system for sample-prep. Companies are making efforts to innovate in cryogenic electron microscopy (cryo-EM) in advanced sample preparation systems. As such, sample preparation systems are projected for exponential growth in the cryopreservation equipment market.

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Innovations in sample-prep systems are paving the way for cryo-EM analysis, which is instrumental in protein research and drug discovery. High-quality foil grids in systems for cryo-EM analysis are increasingly replacing manual processes in the healthcare industry. Thus, stakeholders in biotechnology and research laboratories are benefitting from these advanced systems to assess structure of biomolecules and support protein research. Advanced sample-prep systems are pervasively replacing conventional methods of NMR (Nuclear Magnetic Resonance) and X ray crystallography. These systems offer areas for researchers to solve complex protein structures, which was not possible with conventional methods. Improved 3D imaging and high speed blot-free plunging are key attributes that are attracting research companies in the cryopreservation equipment market landscape.

Stem Cell Storage and New Cell Manufacturing Plants Create Income Sources for Companies

Emergence of new cell manufacturing plants is complementing the growth of the cryopreservation equipment market. For instance, Cellex Incorporated - a biotechnology company, announced the launch of its cell manufacturing plant in Cologne, Germany to produce advanced therapy medicinal products for cryopreservation and cell purification, among others.

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Companies in the cryopreservation equipment market are expanding their services in long-term cryo-storage of advanced therapy medicinal products. Thus, manufacturing innovative cell therapy products for cancer is beneficial for creating new income opportunities for manufacturers of cryopreservation equipment. However, growth of the stem cells industry is another driver of the cryopreservation equipment market growth. Growing awareness about stem cell storage at birth is gaining importance in the cryopreservation equipment market landscape.

Cutting-edge Sensor Technology Aids pH and Co2 Measurement in Benchtop Incubators

The cryopreservation equipment market is largely consolidated with three dominant players accounting for a combined share of ~66% of the cryopreservation equipment market. However, difficulty to establish the right culture environment and incubator conditions for laboratories and IVF clinics are some of the challenges faced by emerging players. Hence, manufacturers in the cryopreservation equipment market are increasing production capabilities to develop equipment that provide insights on pH measurement. For instance, Planer a supplier of controlled temperature products, innovated in PetriSenseST, a sensor that provides monitoring of pH and CO2 in benchtop incubators.

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Incubators type segment dominates the cryopreservation equipment market and is projected to reach a value of ~US$ 2.9 Bn by 2027. Hence, equipment companies are increasing technical expertise in sensor technology to support incubator applications in laboratories and IVF clinics. Portability and flexibility of petri dish-sized sensors is gaining application in laboratory equipment. Thus, manufacturers in the cryopreservation equipment market are expected to increase their scope for incremental opportunities by developing advanced sensor equipment to cater to the needs of end users in labs and clinics.

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Cryopreservation Equipment Market: Increase in Demand for Regenerative Medicines to Accelerate Market Growth - BioSpace