Archive for the ‘Gene Therapy Research’ Category

Latest published market study on Global Stem Cell and Progenitor Cell-based Therapeutics Market with + data Tables, Pie Chart, high level qualitative chapters & Graphs is available now to provide complete assessment of the Market highlighting evolving trends, Measures taken up by players, current-to-future scenario analysis and growth factors validated with Viewpoints extracted via Industry experts and Consultants.Stem Cell and Progenitor Cell-based Therapeutics Market presents point by point serious investigation including the market Share, Size, Future extension. This investigation classifies the worldwide Stem Cell and Progenitor Cell-based Therapeutics breakdown information by makers, area, type and applications, likewise examines the market drivers, openings and difficulties. The study breaks market by revenue and volume (wherever applicable) and price history to estimates size and trend analysis and identifying gaps and opportunities. Some are the players that are in coverage of the study are VitroBioPharma, ViaCyte, Inc., Vericel., U.S. Stem Cell, Inc., Stemedica Cell Technologies, Inc, etc.

Data Bridge Market Research analyses the Stem Cell and Progenitor Cell-based Therapeutics Market to grow at a CAGR of 9.4% in the above-mentioned forecast period. Increasing focus on stem research is anticipated to create new opportunity for the stem cell & progenitor cell based therapeutics market.

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Overview:

Increasing investment in the R&D and advancement in the stem cell based therapies is expected to enhance the market growth. Some of the other factors such as increasing public-private investments for advancement of stem cell therapies, development of infrastructure of stem cell banking & processing, and rising cases of Parkinson disease will affect the growth of this market.

Some of the factors such as improper guidelines associated with the product development and lack of skilled & trained professional for cell culture technology are hampering the growth of this market.

The Stem Cell and Progenitor Cell-based Therapeutics Market 2020 Document clearly explains what market definition, classifications, applications, engagements and market trends are for the Stem Cell and Progenitor Cell-based Therapeutics industry. The Global Stem Cell and Progenitor Cell-based Therapeutics Market Share analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins. For each manufacturer covered, this report analyzes their Stem Cell and Progenitor Cell-based Therapeutics manufacturing sites, capacity, production, ex-factory price, and revenue and market share in global market. This report studies the industry abilities for each geographical region based on the customer purchasing patterns, macroeconomic parameters, development rate, and market demand and supply states. What is more, it determines the impact of buyers, substitutes, new entrants, competitors, and suppliers on the market.

According to this report Global Stem Cell and Progenitor Cell-based Therapeutics Market will rise from Covid-19 crisis at moderate growth rate during 2020 to 2027. Stem Cell and Progenitor Cell-based Therapeutics Market includes comprehensive information derived from depth study on Stem Cell and Progenitor Cell-based Therapeutics Industry historical and forecast market data. Global Stem Cell and Progenitor Cell-based Therapeutics Market Size To Expand moderately as the new developments in Stem Cell and Progenitor Cell-based Therapeutics and Impact of COVID19 over the forecast period 2020 to 2027.

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Stem Cell and Progenitor Cell-based Therapeutics Market report provides depth analysis of the market impact and new opportunities created by the COVID19/CORONA Virus pandemic. Report covers Stem Cell and Progenitor Cell-based Therapeutics Market report is helpful for strategists, marketers and senior management, And Key Players in Stem Cell and Progenitor Cell-based Therapeutics Industry.

The Global Stem Cell and Progenitor Cell-based Therapeutics segments and Market Data Break Down are illuminated below:

By Disease Type (Parkinson Disease, Huntington Disease)

By Applications (Tissue Engineering, Stem Cells, Gene Therapy, Drug Discovery, Nanotechnology)

By End- Users (Pharmaceutical and Biotechnology Industries, Hospitals, Research Institutes, Biotechnology Companies, Others)

The report can help to understand the market and strategize for business expansion accordingly. In the strategy analysis, it gives insights from marketing channel and market positioning to potential growth strategies, providing in-depth analysis for new entrants or exists competitors in the Stem Cell and Progenitor Cell-based Therapeutics industry. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins. For each manufacturer covered, this report analyzes their Stem Cell and Progenitor Cell-based Therapeutics manufacturing sites, capacity, production, ex-factory price, revenue and market share in global market.

List of TOP KEY PLAYERS in Stem Cell and Progenitor Cell-based Therapeutics Market Report are

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Stem Cell and Progenitor Cell-based Therapeutics Industry 2020 Market Research Report provides exclusive vital statistics, data, information, trends and competitive landscape details in this niche sector. This reports provides current status for Stem Cell and Progenitor Cell-based Therapeutics market forecast till 2027. Likely, the report also focuses on global major manufacturers of Stem Cell and Progenitor Cell-based Therapeutics market providing information such as company profiles, product picture and specification, capacity, production, price, cost, revenue and contact information. Upstream raw materials and equipment and downstream demand analysis is also carried out.

Stem Cell and Progenitor Cell-based Therapeutics Report covers the manufacturers data, including: shipment, price, revenue, gross profit, interview record, business distribution etc., these data help the consumer know about the competitors better. This report also covers all the regions and countries of the world, which shows a regional development status, including market size, volume and value, as well as price data.

Global Stem Cell & Progenitor Cell-Based Therapeutics Market Scope and Market Size

Stem cell & progenitor cell-based therapeutics market is segmented of the basis of disease type, application and end- users. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

On the basis of disease type, the stem cell & progenitor cell-based therapeutics market is segmented into Parkinson disease and Huntington disease.

The application segment of the stem cell & progenitor cell-based therapeutics market is divided into tissue engineering, stem cells, gene therapy, drug discovery, and nanotechnology.

On the basis of end- users, the stem cell & progenitor cell-based therapeutics market is segmented into pharmaceutical and biotechnology industries, hospitals, research institutes, biotechnology companies and others.

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The report offers in-depth assessment of the growth and other aspects of the Stem Cell and Progenitor Cell-based Therapeutics market in important countries (regions), including:

What the Stem Cell and Progenitor Cell-based Therapeutics Market Report Contains:

Organization profiles of the main rivals alongside their strategic activities and market shares.

Assurance and examination of the macro and microeconomic variables that influence the Global Market, as per the regional analysis.

Market Overview for the Global Stem Cell and Progenitor Cell-based Therapeutics Market and the identification of the market elements, including development drivers, limitations, difficulties, and potential opportunities for the market.

Assurance of various elements in charge of changing the market scene, rising future chances and assurance of driving players, which can influence the market on a territorial scale.

Market analysis for the Global Stem Cell and Progenitor Cell-based Therapeutics Market, with an aggressive scene and geographic examination on a worldwide and territorial scale.

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With tables and figures helping analyze worldwide Global Stem Cell and Progenitor Cell-based Therapeutics Market Forecast provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

The content of the study subjects includes a total of 15 CHAPTERS:

Chapter 1, to describe Stem Cell and Progenitor Cell-based Therapeutics product scope, market overview, market opportunities, market driving force and market risks.

Chapter 2, to profile the top manufacturers of Stem Cell and Progenitor Cell-based Therapeutics, with price, sales, revenue and global market share of Stem Cell and Progenitor Cell-based Therapeutics in 2018 and 2019.

Chapter 3, the Stem Cell and Progenitor Cell-based Therapeutics competitive situation, sales, revenue and global market share of top manufacturers are analyzed emphatically by landscape contrast.

Chapter 4, the Stem Cell and Progenitor Cell-based Therapeutics breakdown data are shown at the regional level, to show the sales, revenue and growth by regions, from 2010 to 2020.

Chapter 5, 6, 7, 8 and 9, to break the sales data at the country level, with sales, revenue and market share for key countries in the world, from 2010 to 2020.

Chapter 10 and 11, to segment the sales by type and application, with sales market share and growth rate by type, application, from 2010 to 2020.

Chapter 12, Stem Cell and Progenitor Cell-based Therapeutics market forecast, by regions, type and application, with sales and revenue, from 2020 to 2027.

Chapter 13, 14 and 15, to describe Stem Cell and Progenitor Cell-based Therapeutics sales channel, distributors, customers, research findings and conclusion, appendix and data source.

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Stem Cell and Progenitor Cell-based Therapeutics Market Market Size, Share, Industry Report by Growt - PharmiWeb.com

Santhera will hold a conference call today at 13:00 CEST, 12:00 BST, 07:00 EDT. Details are at the end of this statement.

Pratteln, Switzerland, October6, 2020 Santhera Pharmaceuticals (SIX: SANN) announces the discontinuation of its Phase 3 SIDEROS study with Puldysa (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are in respiratory decline and receive concomitant glucocorticoid treatment. Data from an interim analysis conducted by the independent Data and Safety Monitoring Board (DSMB) concluded that the study was unlikely to meet its primary endpoint. As a consequence, Santhera will discontinue the study, withdraw the European marketing authorization application and end the global development program for Puldysa. The Company intends to initiate a restructuring plan for the business with a focus on retaining key functions for bringing DMD drug candidate vamorolone to patients and execute on its other pipeline programs.

Based on the now completed interim analysis which tested for efficacy, the DSMB has recommended the SIDEROS study be discontinued due to futility. The interim analysis was based on the primary endpoint of the study, the change of forced vital capacity % predicted (FVC%p) from baseline to 18 months of treatment. The outcome revealed that the probability of reaching the primary endpoint at the end of the study is too small to merit the continuation of the study. There were no safety concerns noted by the DSMB.

Santhera will stop the SIDEROS trial (including extension) and participants who are enrolled in the study will discontinue study medication and complete the studys follow-up evaluations. Furthermore, following up on the recommendation from the DSMB, Santhera will discuss the impact of ending the SIDEROS study on ongoing expanded access programs with the corresponding regulatory bodies.

"We would like to thank the patients and the families, as well as investigators and medical professionals, who participated in the SIDEROS study. Without their contributions we would not be able to advance DMD research, said Dario Eklund, Chief Executive Officer of Santhera. While this is obviously not the outcome we expected, all our efforts in DMD will now be focused on progressing the promising drug candidate vamorolone which we recently licensed from ReveraGen to its next inflection point, the readout of 6-month topline data from the pivotal VISION-DMD study planned for the second quarter of 2021.

In connection with this decision, Santhera intends to start a restructuring process, aligning its operations to focus on progressing vamorolone for DMD, lonodelestat for cystic fibrosis and other lung diseases and its discovery-stage gene therapy approach for congenital muscular dystrophy.

Conference CallSanthera will host a conference call today at 13:00 CEST / 12:00 BST / 07:00 EDT. Dario Eklund, CEO of Santhera, will discuss this update. Participants are invited to call one of the following numbers 10-15 minutes before the conference call starts (no dial-in code is required):Europe: +41 58 310 50 00UK: +44 207 107 06 13USA: +1 631 570 56 13

About Santhera Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. Santhera has an exclusive license for all indications worldwide to vamorolone, a first-in-class anti-inflammatory drug candidate with novel mode of action, currently investigated in a pivotal study in patients with DMD as an alternative to standard corticosteroids. The clinical stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases, as well as omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit http://www.santhera.com.

Puldysa and Raxone are trademarks of Santhera Pharmaceuticals.

For further information please contact: public-relations@santhera.com orEva Kalias, Head External CommunicationsPhone: +41 79 875 27 80eva.kalias@santhera.com

Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

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Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone - GlobeNewswire

PITTSBURGH--(BUSINESS WIRE)--Krystal Biotech, Inc. (Nasdaq:KRYS) today announced the presentation of positive preclinical data supporting the ongoing development of KB301, an innovative, investigational therapy designed to reverse the decline of collagen in aging skin. The data will be presented at the American Society for Dermatologic Surgery (ASDS) 2020 Virtual Meeting to be held October 9-11, 2020.

The preclinical program evaluated KB301s ability to transduce clinically relevant skin cells, to express and secrete mature human COL3 in vitro, as well as to confirm proper tissue localization of the transgene without toxicity or systemic vector distribution in vivo.

Key findings from the poster presentation titled In Vitro and In Vivo Pharmacology of KB301, an HSV-1-Based Gene Therapy, for the Treatment of Superficial Skin Depressions include:

The above data presentation will be available on the Scientific Publications page of the Companys website at http://ir.krystalbio.com/select-scientific-publications.

About collagen type III and KB301

The skin is composed of collagen-rich connective tissue composed primarily of types I and III collagen fibrils. Age-related changes in skin are largely due to aberrant collagen homeostasis, caused both by intrinsic (e.g., passage of time, genetics) and extrinsic (e.g., chronic light exposure, pollution) factors, which leads to progressive loss of dermal collagen. KB301 is an investigational therapy designed to restore collagen homeostasis locally via directed expression of full-length human type III collagen gene (COL3A1), thereby reconstructing an optimal physiologic environment in the skin to treat wrinkles and other superficial skin defects. KB301 is manufactured in-house at Krystals fully functional GMP ANCORIS facility, located near corporate headquarters in Pittsburgh. If ultimately successful, KB301 and any additional aesthetic programs will be developed through our wholly-owned subsidiary Jeune, Inc.

About the PEARL-1 Trial

The Phase 1 trial will evaluate the safety, tolerability, and initial efficacy of KB301 injections. Approximately 22 patients will be enrolled across 3 cohorts. The initial, open-label cohort will evaluate the safety and tolerability of 2 different dose levels of KB301 in healthy buttock skin. Following dose selection enrollment will begin in Cohorts 2 and 3, which are double-blind, placebo-controlled, intra-subject evaluations of the safety and efficacy of KB301 in either shallow-to-moderately deep facial wrinkles or moderate-to-severe atrophic acne scars. More information about the PEARL-1 study is available at https://clinicaltrials.gov/ct2/show/NCT04540900.

About Krystal Biotech

Krystal Biotech, Inc. (NASDAQ:KRYS) is a gene therapy company dedicated to developing transformative medicines to treat diseases caused by protein or gene dysfunction. For more information, please visit http://www.krystalbio.com.

About Jeune, Inc.

Jeune, Inc. is the companys wholly-owned subsidiary, which was incorporated in 2019 for the purpose of undertaking preclinical studies for aesthetic skin conditions.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., including but not limited to statements about the development of Krystals product candidates, such as plans for the design, conduct and timelines of ongoing clinical trials of beremagene geperpavec (B-VEC), KB105, KB104, KB301 and KB407; the clinical utility of B-VEC, KB105, KB104, KB301 and KB407, and Krystals plans for filing of regulatory approvals and efforts to bring B-VEC, KB105, KB104, KB301 and KB407 to market; the market opportunity for and the potential market acceptance of B-VEC, KB105, KB104, KB301 and KB407; plans to pursue research and development of other product candidates; the sufficiency of Krystals existing cash resources; the unanticipated impact of COVID-19 on Krystals business operations, pre-clinical activities and clinical trials; and other statements containing the words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, likely, will, would, could, should, continue, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including B-VEC, KB105, KB104, KB301 and KB407, the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption Risk Factors in Krystals annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystals views as of any date subsequent to the date of this release.

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Krystal Biotech to Present Pre-clinical Data Highlighting KB301 for Aesthetic Indications at the ASDS 2020 Virtual Meeting - Business Wire

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 07 October 2020 13:48Hits: 333

NEW YORK, NY & BRISBANE, CA, USA I October 7, 2020 I Pfizer Inc. (NYSE: PFE) and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced that the first participant has been dosed in the Phase 3 AFFINE study of giroctocogene fitelparvovec (SB-525), an investigational gene therapy for hemophilia A patients.

AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement therapy collected in the Phase 3 lead-in study period. Participants will be analyzed throughout the 5-year study period following the single infusion to further assess the durability and efficacy.

The initiation of the pivotal Phase 3 dosing study of giroctocogene fitelparvovec is a significant achievement for Pfizer as we continue our longstanding commitment to improving care for the hemophilia community, said Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development. Enrollment in the lead-in study is progressing well and recruitment is on track for Phase 3. Given the Phase 1/2 study findings to date, we believe that giroctocogene fitelparvovec has the potential to sustain factor levels and reduce annual bleed rates, suggesting this one-time gene therapy could potentially transform the standard of care for eligible patients worldwide.

Data from the Phase 3 lead-in study will provide a baseline for patients evaluated in the Phase 3 study. Updated Phase 1/2 data announced at a Pfizer investor event on September 15, 2020 demonstrated that giroctocogene fitelparvovec was generally well tolerated. Each of the five patients in the high dose cohort sustained FVIII activity levels without bleeds or the need for prophylactic factor through up to 85 weeks. Factor VIII activity levels were sustained at a clinically meaningful level, with a geometric mean of ~71% when measured between the weeks of 9 and 52.

We are encouraged that findings from the Phase 1/2 Alta study met two critically important measures for the hemophilia A patient community, showing clinically meaningful factor levels and reduced bleeds, said Bettina M. Cockroft, M.D., M.B.A, Chief Medical Officer of Sangamo. The progress of this program, the most advanced of our gene therapy product candidates, into Phase 3 is an important milestone for Sangamo, as it represents our first asset in a registrational trial.

Per the terms of the collaboration agreement, Sangamo has now earned a $30 million milestone payment. The giroctocogene fitelparvovec collaboration was established in May 2017. Under the terms, Pfizer is now operationally and financially responsible for research, development, manufacturing and commercialization activities for giroctocogene fitelparvovec following the transfer of the Investigational New Drug (IND) from Sangamo to Pfizer in December 2019. Sangamo is eligible to receive total potential milestone payments of up to $300 million for the development and commercialization of giroctocogene fitelparvovec, and up to $175 million for additional hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will, additionally, receive tiered royalties starting in the low teens and up to 20% of annual net sales of giroctocogene fitelparvovec.

About the AFFINE study

The Phase 3 AFFINE (efficAcy and saFety Factor vIii geNe thErapy in hemophilia A patients; NCT04370054)study is an open-label, multicenter, single arm study to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) male participants with moderately severe to severe hemophilia A. Eligible study participants will have completed at least six months of routine FVIII prophylaxis therapy during the lead-in Phase 3 study (NCT03587116) in order to collect pretreatment data for efficacy and selected safety parameters.

The primary endpoint is impact on ABR through 12 months following treatment with giroctocogene fitelparvovec, as compared to ABR on prior FVIII prophylaxis replacement therapy. The secondary endpoint is FVIII activity level after the onset of steady state and through 12 months following infusion of giroctocogene fitelparvovec.

About giroctocogene fitelparvovec

Giroctocogene fitelparvovec (SB-525 or PF-07055480) comprises a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. The giroctocogene fitelparvovec expression cassette was designed for optimal liver-specific expression of FVIII protein and supports production of high yields of the vector. The giroctocogene fitelparvovec transcriptional cassette incorporates multi-factorial modifications to the liver-specific promoter module, FVIII transgene, synthetic polyadenylation signal and vector backbone sequence.

The U.S. Food and Drug Administration has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to giroctocogene fitelparvovec, which also received Orphan Medicinal Product designation from the European Medicines Agency. Giroctocogene fitelparvovec is being developed as part of a global collaboration between Sangamo and Pfizer.

About Hemophilia A

Hemophilia is a genetic hematological rare disease that results in a deficiency of a protein that is required for normal blood clottingclotting factor VIII in hemophilia A. The severity of hemophilia that a person has is determined by the amount of factor in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems.

Hemophilia A occurs in approximately one in every 5,000-10,000 male births worldwide. For people who live with hemophilia A, there is an increased risk of spontaneous bleeding as well as bleeding following injuries or surgery. It is a lifelong disease that requires constant monitoring and therapy.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

SOURCE: Sangamo Biotherapeutics

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Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment | DNA RNA and Cells | News Channels -...

It will also offer GMP manufacturing services in that Billingham location.

In terms of the driver behind the companys decision to establish such services at its UK site,Andy Ross, spokesperson for Fujifilm Europe, told BioPharma-Reporter:In the gene therapy viral vector CDMO market, Europe is the second largest after North America. Therefore, in order to expand the gene therapy drug CDMO business, Fujifilm will make an initial investment in the UK base this time and [later] expand the gene therapy drug CDMO business to Europe.

The FDB viral vector process development laboratories in the UK are due to be online from spring 2021; the company outlined how the site will be outfitted with equipment to support upstream - suspension and adherent - processes, downstream processing and analytical development.

The GMP manufacturing capabilities of that CDMO are expected to be available at the UK base from autumn 2021; they will support bulk drug substance production of investigational drugs used in early-stage clinical trials.

The company previously announced it was constructing a research center at the UK site in March this year.

Fujifilm has positioned its CDMObusiness for the gene therapy drug of viral vector process development and manufacturing as a priority field, said Ross. It is an area where the companyhas a strong track record, he added.

The company first established a viral vector and vaccine development and manufacturing offer in 2014 at its College Station, Texas site, in the US.

Flagging up Fujifilms USP in relation to viral vector and GMP manufacturing, Ross said the company is able to consistently provide flexible service from process development, formulation development, bulk drug substance production, fill finish and final packaging.We have state of the art facilities and equipment, world class staff, and proprietary expression technologies to quickly develop and provide the optimum production process.

Fujifilm, in another effort to grow this CDMO business, is making a capital investment in FDBs Texas operations. Earlier this year,it announced that this site would be expanded with US$35m (31m) earmarked for the upgrade.

Moreover, as part of the US governmentsOperation Warp Speed, the Biomedical Advanced Research and Development Authority (BARDA), back in August, enlisted the support of FDB for the production of COVID-19 vaccine candidates.

The agreement will see Fujifilm Diosynths Texas site contracted to provide manufacturing capacity through the end of 2021.

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Fujifilm expands its gene therapy drug CDMO business - BioPharma-Reporter.com

Cleveland Clinic released its list of the top 10 medical innovations it believes will change health care next year, including gene therapy for blood disorders, a new class of medications for cystic fibrosis and increased access to telemedicine, according to a news release.

The annual list, which is selected by a panel of Clinic researchers and clinicians, was announced in conjunction with the system's 18th annual Medical Innovation Summit, which was held virtually on Tuesday, Oct. 6.

The committee of experts that selected the breakthrough technologies was led by Dr. Will Morris, executive medical director for Cleveland Clinic Innovations (the system's development and commercialization arm), and Dr. Akhil Saklecha, managing director of Cleveland Clinic Ventures, which was formed three years ago to invest in emerging health care companies that can deliver financial returns to the Clinic.

The Clinic also announced that it will present a new award at next year's summit to a team, organization or individual who has made a significant contribution to health care delivery with a focus on one or more defined areas, according to the release, which notes that details will be released early next year.

Below are the Clinic's top 10 medical innovations for 2021, in order of importance, according to the release:

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Cleveland Clinic unveils top 10 medical innovations for 2021 - Crain's Cleveland Business

Application of ImmTORplus IGANs IgA protease to targetimmunoglobulin A (IgA) deposition, the critical event leading to IgA Nephropathy (IgAN)

Selectas IgA nephropathy program builds upon SEL-212, which has demonstrated ImmTOReffective at tolerizing a highly immunogenic therapeutic enzyme

Investigational New Drug (IND) application expected in Q4 2021

WATERTOWN, Mass., and BOSTON, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc.(NASDAQ: SELB) and IGAN Biosciences, Inc. today announced that they have entered into a Research License and Option agreement to study Selectas ImmTORimmune tolerance platform in combination with IGANs immunoglobulin A (IgA) protease for the treatment of IgA Nephropathy (IgAN). In advance of exercising its option to enter into an exclusive license agreement, Selecta will conduct preclinical research to evaluate the utility of the combination of ImmTOR and IGANs IgA protease.

Previous studies in animal models conducted at independent laboratories established the ability of IGANs IgA protease to remove injurious IgA from kidneys and improve markers of renal dysfunction. These results suggest that it is an excellent candidate to decrease the rate of disease progression and possibly even reverse the disease. The barrier to IgA protease commercialization is the bacterial origin of the protease, which makes it immunogenic. Selectas proprietary immune tolerance platform, ImmTOR, has shown in clinical studies the ability to mitigate the formation of anti-drug antibodies (ADAs) to immunogenic enzymes. This has been demonstrated with its phase 3 lead product candidate, SEL-212, which is a combination of ImmTOR and pegadricase, an immunogenic proprietary pegylated uricase for the treatment of chronic refractory gout. Selecta and IGAN intend to combine IGANs IgA protease with Selectas ImmTOR platform to develop a novel combination product to treat IgA nephropathy, a disease with significant unmet medical need.

We are excited to build on our learnings from SEL-212 in combining an immunogenic enzyme with our ImmTOR platform to develop a product candidate for IgA nephropathy, said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta Biosciences. IGANs IgA protease has shown tremendous promise in preclinical studies and combining it with ImmTOR to prevent ADAs could lead to a transformational product for patients with this debilitating disease.

IGAN is pleased to enter into this agreement with Selecta Biosciences to use their ImmTOR technology with IgA proteases as biological therapy for IgA nephropathy, said Andrew G. Plaut, M.D., Co-Founder and Chief Medical Officer of IGAN Biosciences Inc. In combination, these products offer the prospect of realizing the potential value of IgA proteases to remove kidney IgA deposits, thus reducing progression of renal injury to renal failure.

IgA Nephropathy is characterized by deposition of galactose-deficient IgA1 immunoglobulin in the glomerular mesangium and is a leading contributor to development of chronic kidney disease and renal failure. Genetic or environmental causes that form this abnormal IgA1 and its accumulation in the kidney can result in the development of IgAN. Hypertension, proteinuria and decreased eGFR at the time of diagnosis are associated with poor prognosis. It can result in incremental loss of renal function and results in end stage renal disease (ESRD) in approximately 30 - 40% of patients. There are no approved therapies for the treatment of IgAN.

Under the terms of the research license and option agreement, Selecta will make an initial payment to IGAN. If Selecta exercises its option to enter into an exclusive license agreement, IGAN will receive an additional payment and will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales.

AboutSelecta Biosciences, Inc.Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the bodys natural self-tolerance in autoimmune diseases. The companys first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit http://www.selectabio.com.

About IGAN BiosciencesIGAN Biosciencesis a private biotechnology company advancing IgA protease treatment for IgA deposition diseases. Its founders discovered and characterized IgA proteases during more than forty years of research conducted at Tufts University School of Medicine and Tufts Medical Center, Boston, MA.

Selecta Forward-Looking Statements

This press release, contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Selectas plan to participate in upcoming investor conferences. All such forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Such risks and uncertainties include but are not limited to the companys plans to exercise its option to enter into a licensing agreement, preclinical research and development activities related to IGANs IgA Protease, the ability of the IgA Protease to remove injurious IgA from kidneys and improve markers of renal dysfunction, the companys plans to develop a product candidate to treat IgA Nephropathy or other autoimmune indications, the material impact, if any, of the COVID-19 outbreak on the Companys operations, including supply chain and clinical trials, other COVID-19 related risks and those set forth in the Risk Factors section of Selectas Quarterly Report on Form 10-Q filed with theSecurities and Exchange Commission, orSEC, for the quarter endedMarch 31, 2020, and in other filings thatSelectamakes with theSEC. In addition, any forward-looking statements included in this press release represent Selectas views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date.Selectaspecifically disclaims any obligation to update any forward-looking statements included in this press release.

For more information please contact:

Selecta:

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

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

IGAN Biosciences:Donald CollinsIronwood Investment Management+1-617-757-7620dcollins@ironwoodfunds.com

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Selecta Biosciences and IGAN Biosciences Enter into Research License and Option Agreement to Study Selecta's ImmTOR Immune Tolerance Platform in...

The Global Oncolytic Virus Immunotherapy Market Will Be Driven By Increasing Number Of Clinical Trials Along With New Product Launches Over Next 5 Years Says Kuick Research

NEW DELHI, Oct. 8, 2020 /PRNewswire/ -- "Global Oncolytic Virus Immunotherapy Market, Dosage, Price & Clinical Trials Outlook 2026" Report Highlights:

Download Report:

https://www.kuickresearch.com/report-oncolytic-virus-immunotherapy-market-size-sales-clinical-trials-cancer-oncology-melanoma-report-talimogene-laherparepvec-imlygic--oncorine

or contact: [emailprotected]

Increase in prevalence and mortality rate associated with cancer have always remained a challenge to the modern medicine with respect to the development of more reliable therapies. In the past few years, one of the greatest breakthrough novel cancer therapy that has been developed by the researchers is the oncolytic virus immunotherapy. The overall procedure of the therapy is based on the ability of the oncolytic virus to infect and further lyse the tumor cells by boosting the immune system of the patient. Some of the virus strains that have been tested and proved to be adding to the various applications of the therapy are canine distemper virus, adenovirus, vaccinia virus and reovirus in the preclinical and clinical studies.

The viral toxicity and the in-vitro manipulation of the virus is estimated to be providing successful clinical outcomes, leading to the enhancement and the expansion of the market linked with the therapy. All the clinical trials associated with the therapy are underway but all are estimated to look forward by bringing numerous opportunities for the cancer patients. The market concerned with the growth of the oncolytic virus therapy is focused on increasing the research and development sector, which is apparently considered as an important asset for the therapy in order to change the stringent cancer treatment paradigm available in the industry for a long period of time. The current clinical platform is estimated to be developing a competitive landscape for the other cancer markets as the therapy of interest is getting fragmented at a high speed for different cancer types.

The blockbuster model held by the therapy is also believed to be driving the large shareholder value of the market in the present as well as in the upcoming years. The increase in the perception of highly expensive drugs available under cell and gene therapy are expected to be one of the most regulated drivers for the global expansion of the oncolytic therapy. As a result of strong clinical background and healthy research and development sector, oncolytic virus therapy is becoming more prominent in the cancer landscape. With the primary goal of revolutionizing the cancer therapeutics market, it is estimated that the therapy in a short period of time will be successful in re-defining the unstructured cancer paradigm for the patients with advanced cancers.

Being not only restricted to clinical benefits, the therapy is moving forward to provide better post-treatment lives to the cancer patients. The therapy in the clinical studies have been able to put forward the value of the patients in terms of clinical outcomes as well as patient-related outcome measures, thus causing a wider acceptance to the therapy in the cancer therapeutics market. Some of the primary aim associated with the current clinical pipeline of the therapy involves limited cost expansion and increased success rate. Prominent factors like these are estimated to enhance access of the patient population to effective oncolytic virus therapy.

The future view of the oncolytic virus therapy is estimated to be enabling such innovative mechanisms that would be responsible for providing tremendous amount of opportunities to the cancer patients. Different gene-editing technologies, advancement in genomics and many others will map the therapy among the best-cancer regimens ever discovered. The leverage provided to the therapy in terms of opportunities depicts the possibility of the therapy to get recognized as a real cure to the cancer patients and eventually a dominating market. With the advent of the therapy for the cancer platform, there has been a significant transformation in the current cancer therapeutics market. In financial services, the market is estimated to perform with accuracy and speed in the future years.

As per " Global Oncolytic Virus Immunotherapy Market, Dosage, Price & Clinical Trials Outlook 2026" report findings, it is estimated that the total inflation of the market with respect to clinical platform, wider acceptance in a short period of time will drive the market in the upcoming years. Gradual increase in the interest of the researchers towards oncolytic virus therapy will develop the market which will eventually bring complete revolution in terms of applications to the cancer patients. The future market insight for the therapy is analyzed to provide unlimited number of trends and opportunities, leading to aggregation of all the cancer related bio-pharmaceutical companies and research centers towards the further development and commercialization of the therapy.

Contact:

Neeraj Chawla[emailprotected]+91-9810410366

Logo: https://mma.prnewswire.com/media/1245952/Kuick_Research_Logo.jpg

SOURCE Kuick Research

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Global Oncolytic Virus Immunotherapy Market To Reach US$ 750 Million By 2026 With 500% Absolute Growth - PR Newswire India

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy BMN 307 (BioMarin Pharmaceutical) for the treatment of patients with phenylketonuria (PKU).

BMN 307 is an adeno-associated virus type 5 (AAV5)-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells.

The Company recently initiated an open-label phase 1/2 study (PHEARLESS) to evaluate the safety, efficacy and tolerability of a single intravenous administration of BMN 307 in adults with PKU with PAH deficiency. The study will assess whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.

Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy, said Hank Fuchs, MD, President, Worldwide Research and Development at BioMarin. We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit biomarin.com.

BioMarin, pioneer in phenylketonuria (PKU) and gene therapy, receives FDA Fast Track designation for PKU investigational gene therapy, BMN 307. https://www.prnewswire.com/news-releases/biomarin-pioneer-in-phenylketonuria-pku-and-gene-therapy-receives-fda-fast-track-designation-for-pku-investigational-gene-therapy-bmn-307-301144678.html. Accessed October 5, 2020.

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Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference

DetailsCategory: More NewsPublished on Thursday, 08 October 2020 10:17Hits: 238

Establishment of Social Cooperation Program "Protein Degradation Drug Discovery"

TOKYO, Japan I October 8, 2020 I The University of Tokyo and Eisai Co., Ltd. announced today a collaboration aiming for the development and drug discovery of targeted protein degradation technology has been created, with the establishment of a social cooperation program, "Protein Degradation Drug Discovery". The research time span will last five years from October 1, 2020 to September 30, 2025.

The social cooperation program is established and operated based on funds of private organizations dedicated to conducting research in collaboration with the University of Tokyo regarding shared issues of high common concern.

The "Protein Degradation Drug Discovery" course is to be established within the Graduate School of Pharmaceutical Sciences, the University of Tokyo. Dr. Mikihiko Naito, Former Director of the Division of Molecular Target and Gene Therapy Products, National Institute of Health Sciences, has been inaugurated as a project professor for this program and will lead research with protein degradation technology including SNIPER. This research will combine the world's most advanced ubiquitin-proteasome research as conducted in the graduate school with drug discovery knowledge fostered by Eisai, for the development of new protein degradation technology towards proteins targeted by drugs and the promotion of drug discovery research based on this technology. In addition, through this research, the course will educate and train the next generation of leaders in this research field.

Targeted protein degradation is a series of technologies in which precisely designed compounds force target proteins into proximity with E3 ubiquitin ligase and apply the ubiquitin-proteasome system to induce degradation of the target proteins. The technology provides a means of creating medicines for not only conventional targets such as specific enzymes and receptors, but also disease-related proteins for which drug discovery up to this point has been difficult. Through the development of this technology and drug discovery, the University of Tokyo and Eisai aim to provide new treatment options to patients for which treatment options were previously limited.

About SNIPER

SNIPER (Specific and Nongenetic IAP-dependent Protein Eraser) is a compound which utilizes the ubiquitin-proteasome system to degrade target proteins. This compound is a "hybrid compound", and consists of a moiety that binds to the target protein and a moiety that binds to E3 ubiquitin ligase (IAPs) with an appropriate linker. Designing this compound requires advanced medicinal chemistry and cutting-edge structural biology. When the SNIPER compound brings the target protein and E3 ubiquitin ligase (IAPs) into proximity (Step 1 in the chart below), ubiquitin as a protein degradation tag transfers from the E2 ubiquitin conjugating enzyme to the target protein (2) for recognition of the protein by the proteasome and subsequent degradation (3).

Conventional small-molecule inhibitors bind to the active moiety of target enzymes, and express pharmacological activity by inhibiting the activity thereof. On the other hand, because SNIPER exhibits pharmacological properties by target protein degradation as described above, it is not only expected to exhibit different pharmacological activity from small-molecule inhibitors; it is also predicted to target proteins that do not have enzymatic activity. Similar technologies include PROTAC (PROteolysis TArgeting Chimeras) and Degronimid.

About the Ubiquitin-Proteasome System

The ubiquitin-proteasome system is one of the naturally occurring mechanisms for controlling the degradation of unneeded proteins and is in control of critical movements relating to the preservation of life including cell cycle, transcription regulation, and signal transmission. When ubiquitin as a protein degradation tag connects to unneeded proteins through agents such as the E3 ubiquitin ligase, it marks the protein for recognition by the proteasome for subsequent degradation. In recent years, the relation between the ubiquitin-proteasome system and major human diseases including cancer and neurodegeneration is becoming evident.

About Eisai

Eisai Co., Ltd. is a leading global research and development-based pharmaceutical company headquartered in Japan. We define our corporate mission as "giving first thought to patients and their families and to increasing the benefits health care provides," which we call our human health care (hhc) philosophy. With approximately 10,000 employees working across our global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to realize our hhc philosophy by delivering innovative products to address unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology. As a global pharmaceutical company, our mission extends to patients around the world through our investment and participation in partnership-based initiatives to improve access to medicines in developing and emerging countries.

For more information about Eisai Co., Ltd., please visit https://www.eisai.com

SOURCE: Eisai

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The University of Tokyo and Eisai Announce Research Collaboration for the Development and Drug Discovery of Targeted Protein Degradation Technology |...

Facts & Factors (FnF),a leading market research company recently published a research report onResearch Report on Cell and Gene Therapy Consumables Market: By Product Types, Segments, Size, Share, Applications, Analysis and Forecast, 2020-2026to its research database. This[190+] PDF pages Cell and Gene Therapy Consumables Marketreport analyzes the comprehensive overview of the market comprising an executive summary that covers core trends evolving in the market.

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Cell and Gene Therapy Consumables Market Excessive Growth Opportunities and Trends Estimated in Report Forecast Forecast, 2020-2026 - re:Jerusalem

New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Regenerative Medicine Market by Product, Application, Geography - Global Forecast to 2025" - https://www.reportlinker.com/p04700208/?utm_source=GNW However, the high cost of cell and gene therapies and ethical concerns related to the use of embryonic stem cells in research and development are expected to restrain the growth of this market during the forecast period.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market, by product, during the forecast periodBased on products, the regenerative medicine market is segmented into tissue-engineered products, cell therapies, gene therapies, and progenitor and stem cell therapies.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market in 2019.

The increasing adoption of tissue-engineered products for the treatment of chronic wounds and musculoskeletal disorders and the rising funding for the R&D of regenerative medicine products and therapies are the major factors driving the growth of this segment.

Oncology segment accounted for highest CAGRBased on applications, the regenerative medicine market is segmented into musculoskeletal disorders, wound care, oncology, ocular disorders, dental, and other applications.In 2019, the oncology segment accounted for the highest growth rate.

This can be attributed to the rising prevalence of orthopedic diseases, growing geriatric population, increasing number of stem cell research projects, growing number of clinical researches/trials, and the rich pipeline of stem cell products for the treatment of musculoskeletal disorders.

Europe: The fastest-growing region regenerative medicine marketThe global regenerative medicine market is segmented into North America, Europe, the Asia Pacific, and Rest of the World.The North America region is projected to grow at the highest CAGR during the forecast period in 2019.

The growth in the North American regenerative medicine market can be attributed to rising stem cell banking, tissue engineering, and drug discovery in the region; expansion of the healthcare sector; and the high adoption of stem cell therapy and cell immunotherapies for the treatment of cancer and chronic diseases.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 20%, Tier 2 - 45%, and Tier 3 - 35% By Designation: C-level - 30%, D-level - 20%, and Others - 50% By Region: North America - 36%, Europe - 25%, Asia Pacific - 27%, and Rest of the World 12%

Lits of companies Profiled in the Report: 3M (US) Allergan plc (Ireland) Amgen, Inc. (US) Aspect Biosystems (Canada) bluebird bio (US) Kite Pharma (US) Integra LifeSciences Holdings Corporation (US) MEDIPOST Co., Ltd. (South Korea) Medtronic plc (Ireland) Anterogen Co., Ltd. (South Korea) MiMedx Group (US) Misonix (US) Novartis AG (Switzerland) Organogenesis Inc. (US) Orthocell Limited (Australia) Corestem, Inc. (South Korea) Spark Therapeutics (US) APAC Biotech (India) Shenzhen Sibiono GeneTech Co., Ltd. (China) Smith & Nephew plc (UK) Stryker Corporation (US) Takeda Pharmaceutical Company Limited (Japan) Tego Science (South Korea) Vericel Corporation (US) Zimmer Biomet (US)

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The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall regenerative medicine market and its subsegments.It will also help stakeholders better understand the competitive landscape and gain more insights to position their business better and make suitable go-to-market strategies.

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The global regenerative medicine market is projected to reach USD 17.9 billion by 2025 from USD 8.5 billion in 2020, at a CAGR of 15.9% -...

The label expansion includes the addition of longer-term efficacy data demonstrating sustained phenylalanine (Phe) lowering out to three years and over six years of data further supporting a well-characterized safety profile. The labeling was updated to allow individualized maintenance dosage to achieve blood Phe control (blood Phe concentrations less than or equal to 600 mol/L), taking into account patient tolerability to Palynziq and dietary protein intake.

Palynziq is indicated to reduce blood Phe concentrations in adults with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 mol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe.

Palynziqis BioMarin's second approved treatment for this serious condition. BioMarin also recently announced that the Company had dosed the first participant in the global Phearless Phase 1/2 study of BMN 307, an investigational gene therapy for people with PKU, and potentially its third approved treatment. BMN 307 has Fast Track Designation in the US, as well as Orphan Drug Designation in the U.S. and E.U.

This labeling long term data update is based on efficacy data from an open-label extension study out to three years demonstrating that two-thirds (66%) of the 86 participants treated for at least two years, had a blood Phe level 360 mol/L consistent with the Phe target in American College of Medical Genetics and Genomics (ACMG) recommended guidelines, and 50% of participants had blood Phe levels 120 mol/L at two years.Of the 77 participants treated, 58 (75%), 51 (66%), and 37 (48%) had a blood Phe concentration less than or equal to 600, 360, and 120 mol /L, respectively, at three years of treatment. Additional safety data with over six years of follow up remains consistent with the previous safety profile of Palynziq, irrespective of dose.

PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. PKU affects approximately 1 in 12,500 live births in the United States each year. PKU is marked by an inability to break down Phe, an amino acid that is found in all forms of protein. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological impairment, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, infants are screened at birth to ensure that they are diagnosed early and treated to avoid intellectual disability and other complications. Individuals living with PKU require life-long management, including adherence to a challenging and severely restrictive daily diet of medical foods and formula that avoids the ingestion of Phe that is present in most foods.

"BioMarin is pleased that the FDA has recognized the importance of including an additional dosing option to individuals with PKU. Consistent with the label in Europe, Palynziq is now available in the U.S. at doses of up to 60 mg," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer of BioMarin. "BioMarin remains committed to the PKU community and continues to build on our 15 plus years of research and development, which enabled us to deliver the first two PKU drug therapies. We continue our commitment to further understand the safety and efficacy of our treatments for PKU. We are grateful to the study participants, investigators, study staff and BioMarin employees who were essential to enable this label expansion."

"Adding a maximum dose of 60 mg allows more patients to optimize and achieve treatment goals to keep blood Phe levels within the range set in the medical guidelines or within normal range," said Cary Harding, M.D., professor at Oregon Health & Science University and Steering Committee Chair for the Palynziq program. "More than six years of long-term safety data further supports a well-characterized safety profile similar to the initial data and is important information to physicians considering a chronic therapy."

"It is essential to the PKU community to have treatment options that can control Phe levels and provide long term data on safety and efficacy as it becomes available. The additional information added to the Palynziq label, particularly the long-term safety follow-up demonstrates BioMarin's ongoing commitment to people with PKU," said Christine Brown, MS, executive director of the National PKU Alliance. "BioMarin's 15-year focus on innovative medical research to advance the standard of care in PKU with an existing treatment and for new treatments is transforming the way that the community thinks about this rare genetic disease."

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and an investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About ACMG Guidelines

Practice guidelines issued by ACMG support the need for lifelong management of Phe levels in patients with PKU. The diagnosis and management guidelines were published online in Genetics In Medicine's Advance Online Publication (AOP) service.

The guidelines state that treatment of PKU should be initiated as early as possible and must be continued throughout adulthood and "lifelong," with a goal of maintaining blood levels of Phe for all patients between 120 to 360 micromol/L. Patients treated from the early weeks of life with initial good metabolic control, but who lose that control in later childhood or adult life, may experience both reversible and irreversible neurocognitive and neuropsychiatric consequences.

According to the guidelines "the primary goal of therapy is to lower blood Phe, and any interventions, including medications, or combination of therapies that help to achieve that goal in an individual, without other negative consequences, should be considered appropriate therapy."

Evidence for the guidelines are drawn from two previous independent review processes from the National Institutes of Health (2001) and the Agency for Health Research and Quality (2012). The guidelines can be accessed here.

About Palynziq

Palynziqsubstitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with the PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe. Palynziqis administered using a dosing regimen designed to facilitate tolerability; Palynziq's safety profile consists primarily of immune-mediated responses, including anaphylaxis, for which robust risk management measures effective in clinical trials are in place.

The dosing and administration of Palynziq follows an induction, titration, and maintenance paradigm. Treatment is individualized to the lowest effective and tolerated dosage. Prescribers may consider increasing from 20 mg to 40 mg in patients who have been on 20 mg once daily continuously for at least 24 weeks and who have not achieved blood Phe control. Prescribers may increase dosage to a maximum of 60 mg once daily in patients who have been on 40 mg once daily continuously for at least 16 weeks and who have not achieved blood Phe control. Prescribers are instructed to discontinue treatment in patients who have not responded after 16 weeks of continuous treatment with the maximum dosage of 60 mg once daily. Periodic blood Phe monitoring is recommended, and patients should be counseled on how to adjust their dietary intake, as needed, based on blood Phe concentrations.

To reach a BioMarin RareConnections case manager, please call, toll-free, 1-866-906-6100 or e-mail[emailprotected]. For more information about Palynziq, please visitwww.palynziq.com. For additional information regarding this product, please contact BioMarin Medical Information at [emailprotected].

INDICATION

PALYNZIQ(pegvaliase-pqpz) is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels greater than 600 micromol/L on existing management.

BOXED WARNING: RISK OF ANAPHYLAXIS

WARNINGS AND PRECAUTIONS

Anaphylaxis

Other Hypersensitivity Reactions

ADVERSE REACTIONS

Blood Phenylalanine Monitoring and Diet

DRUG INTERACTIONS

Effect of PALYNZIQ on Other PEGylated Products

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

Pediatric Use

Geriatric Use

You are encouraged to report suspected adverse reactions to BioMarin at1-866-906-6100, or to FDA at1-800-FDA-1088orwww.fda.gov/medwatch.

Please see accompanying full Prescribing Information, including Boxed Warning.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

For additional information, please visit http://www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: expectations regarding the potential impact of Palynziq in the PKU community; the updated Palynziq label allowing individualized maintenance dosage to achieve blood Phe control (blood Phe concentrations less than or equal to 600 micromol/L), taking into account patient tolerability to Palynziq and dietary protein intake; new data supporting Phe lowering and a well-characterized safety profile; the Company's Phase 1/2 Phearless study of BMN 307, including BMN 307 potentially becoming BioMarin's third approved treatment for PKU; and BioMarin's development programs for PKU and BioMarin's ongoing commitment to patients with PKU generally. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: actions by regulatory agencies, results and timing of current and planned clinical trials of BioMarin's products, the risks related to the commercialization of Palynziq, our ability to manufacture sufficient quantities of Palynziq; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in the Company's Securities and Exchange Commission (SEC) filings including the Annual Report on Form10-Q for the quarter ended June 30, 2020, and future filings and reports by the Company. Stockholders are urged not to place undue reliance on forward-looking statements contained in this press release, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation, to update or alter any forward-looking statement, whether as a result of new information, future events, changes in its expectations or otherwise.

BioMarin and Palynziq are registered trademarks of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

https://www.biomarin.com/

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BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for...

New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Soft Tissue Repair Market 2020-2024" - https://www.reportlinker.com/p04796687/?utm_source=GNW Our reports on soft tissue repair market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the rising incidence of accidental injuries, rise in strategic partnerships for distribution of products and reimbursement for soft tissue repair products. In addition, rising incidence of accidental injuries is anticipated to boost the growth of the market as well. The soft tissue repair market analysis includes product segment and geographical landscapes.

The soft tissue repair market is segmented as below: By Product Soft tissue fixation devices and accessories Cell therapy Tissue scaffold

By Geographical Landscapes North America Europe Asia ROW

This study identifies the growing demand for gene therapy as one of the prime reasons driving the soft tissue repair market growth during the next few years. Also, increasing strategic alliances and the development of novel products will lead to sizable demand in the market.

The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our soft tissue repair market covers the following areas: Soft tissue repair market sizing Soft tissue repair market forecast Soft tissue repair market industry analysis

Read the full report: https://www.reportlinker.com/p04796687/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Soft Tissue Repair Market is expected to grow by USD 10.44 bn during 2020-2024, progressing at a CAGR of 11% during the forecast period -...

WORCESTER, Mass., Oct. 08, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that initial Phase 1 data on MB-105, a PSCA-targeted CAR T administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC), will be presented at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat, which is being held October 20 23, 2020.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are pleased that City of Hope will be presenting initial data from the Phase 1 trial of MB-105 at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat. MB-105 is our CAR T cell therapy that is being studied for the potential treatment of prostate cancer. We look forward to continuing to progress this PSCA-targeted CAR T program.

Details of the presentation are as follows:

Title: Clinical Development of PSCA-targeted CAR T cell therapy for mCRPCSession Date and Time:Friday, October 23, 2020, 11:50 a.m. PTPresenter: Tanya Dorff, M.D., Associate Professor, Department of Medical Oncology & Experimental Therapeutics,Head, Genitourinary Cancer Program, City of Hope, Duarte, CA

For more information, please visit the 27th Annual Prostate Cancer Foundation Scientific Retreat website at https://www.pcf.org/scientific-retreat/27th-annual/.

About MB-105 (PSCA CAR T technology)MB-105 was developed in the laboratory of Saul Priceman, Ph.D., assistant professor in City of Hopes Department of Hematology & Hematopoietic Cell Transplantation and a scientist in the T Cell Therapeutics Research Laboratory led by Stephen Forman, M.D., leader of City of Hopes Hematologic Malignancies and Stem Cell Transplantation Institute and the laboratorys director.

The Phase 1 clinical trial of MB-105, one of the first chimeric antigen receptor T cell (CAR T) trials for prostate cancer in the nation, can enroll up to 33 patients. Its primary endpoints are to define the side effects and the best dose of the prostate stem cell antigen (PSCA) CAR T cells in treating patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC). Secondary endpoints include assessing the expansion and persistence of PSCA CAR T cells, the clinical response based on Prostate Cancer Working Group 3 (PCWG3) criteria, the survival outcomes and serum cytokine profiles in peripheral blood pre- and post-therapy, as well as describing the PSCA expression level on tumor cells prior to CAR T cell infusion and the relationship it may have with disease response and toxicities. For more information on this Phase 1 trial, please visit http://www.clinicaltrials.gov using identifier NCT03873805.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Mustang Bio Announces MB-105 Data Selected for Presentation at the Virtual 27th Annual Prostate Cancer Foundation Scientific Retreat - GlobeNewswire

- Kuick Research Report Analyzes Clinical & Commercial Opportunity In Global Bispecific Antibodies Market

NEW DELHI, Oct. 7, 2020 /PRNewswire/ --

"Global Bispecific Antibody Market Opportunity, Drug Sales & Clinical Trials Insight 2026" Report Highlights:

Download Report:

https://www.kuickresearch.com/report-global-usa-europe-japan-bispecific-antibody-antibodies-therapeutics-therapy-market-sales-size--trends-clinical-trials-emicizumab-hemlibra-blincyto--blinatumomab-sales

Despite having several hundred clinical success stories of different immunotherapies against solid and advanced cancers, the overall response rate of the patients has reached approximately 50% even when the patients fit completely under all the criteria of treatment. To pave with the clinical failures, researchers have developed and evaluated bispecific therapeutic antibodies, that are combination of two binding specificities leading to improve selectivity as well as efficacy of antibody-based treatment for cancer. The arrival of bispecific antibody for cancer treatment has estimated to be of great potential for wide range of applications in cancer treatment. The current biotherapeutic market for cancer treatment and prevention is estimated to be dominated by antibody drugs, where bispecific antibody represents a novel ally for the next-generation of cancer therapy.

In the past few years, the remarkable progress that has been made with respect to understanding the important hallmarks of the cancer progression and main epitopes involved in it have led to paradigmatic development in the bispecific antibody capable of offering novel promises to the patients who haven't respond to any medical therapy for cancer. The practice of bispecific antibody is not new i.e. this version of cancer therapeutics is a modified version of monoclonal antibody. By observing the increasing incidence of cancer, the management associated with the bispecific antibody has started to develop several strategies important for clinical management of the cancer in the present and in the future.

As per the analysis conducted, it is estimated that the bispecific antibody therapeutics market is growing at a double-digit rate, thus boosting the overall cancer therapeutics market to extreme, especially when compared with other immunotherapies. Further, trends and opportunities coupled within the market i.e. rise in the number of cancer cases, government favorable policies and many others are tending to drive the growth of the market. In our viewpoint, novel alliances developed by major key players of the market and local manufacturers are estimated to be playing important role for delivering tremendous opportunities for the researchers, patients, stakeholders and the payers.

The analysis of the Global bispecific antibody drug market represents that the market is completely focused on providing a better structure to the overall therapeutics market and offer the pharmaceutical industry a broad perspective of an efficient therapy for every cancer type. The research report provides analysis about the assess growth potential of the market by verifying various driving forces, in combination withall the important trends, opportunities and challenges. In addition to it, the research report also provides a long-term outlook for the industry and various other important measures that have helped the market to attract thousands of investors and local drug manufacturers. Lastly, the report also profiles various pharmaceutical companies of the dominating markets that have been boosting the R&D sector of the market.

Table of Contents

1. Introduction to Bispecific Monoclonal Antibody

1.1 Overview of Monoclonal Antibody

1.2 Overview of Bispecific Monoclonal Antibody

1.3 Advantage of Bispecific Antibodies upon Monospecific Monoclonal Antibodies

2. The Engineering & Development of Bispecific Antibodies

2.1 Bispecific Antibodies Similar to IgG

2.1.1 Quadroma (Hybrid Hydromas) Approach

2.1.2 "Knobs Into Holes" Approach

2.1.3 CrossMab Approach

2.1.4 Dual-Variable-Domain Immunoglobulin Approach

2.2 Small Bispecific Antibodies

2.2.1 Bispecific Diabodies

2.2.2 Bispecific T-Cell Engager Antibodies (BiTEs)

3. Mechanism Of Action Of Bispecific Antibody

3.1 Trifunctional Antibody: Catumaxomab (Removab)

3.2 Blinatumomab

4. Applications of Bispecific Antibodies in Diagnostic & Treatment

4.1 BsMAb for Diagnosis of Bacterial & Viral Infectious Diseases

4.2 BsMAb for Cancer Diagnostic

4.3 BsAbs Blocking Signaling Pathways

4.4 BsAbs Targeting Tumor Angiogenesis

4.5 Specific Delivery of Effector Compounds to Targets

4.6 Bispecific Antibodies & Gene Therapy

5. Global Bispecific Antibody Market Outlook

5.1 Current Market Scenario

5.2 Availability of Bispecific Antibodies

5.2.1 Catumaxomab (Removab)

5.2.2 Blinatumomab

5.2.3 Emicizumab

5.2.4 Duligotumab

5.2.5 SAR 156597

6. Approved Bispecific Antibody Dosage & Price Analysis

6.1 Blincyto

6.2 Hemlibra

7. Approved Bispecific Antibody Sales Analysis

7.1 Blincyto

7.2 Hemlibra

8. Global Bispecific Antibodies Clinical Pipeline Overview

8.1 By Phase

8.2 By Country/Region

8.3 By Company

8.4 By Indication

8.5 Orphan Designated Bispecific Antibodies

9. Global Bispecific Antibodies Clinical Pipeline By Company, Indication & Phase

9.1 Research

9.2 Preclinical

9.3 Phase-I

9.4 Phase-I/II

9.5 Phase-II

9.6 Phase-II/III

9.7 Phase-III

10. Marketed Bispecific Antibodies Clinical Insight

10.1 Blinatumomab - Amgen

10.2 Emicizumab - Chugai Pharmaceutical

11. Global Bispecific Antibody Market Dynamics

11.1 Favorable Market Parameters

11.2 Commercialization Challenges

12. Global Bispecific Antibody Market Future Prospects

13. Competitive Landscape

13.1 Ablynx

13.2 Adimab

13.3 Affimed Therapeutics

13.4 Amgen

13.5 AstraZeneca (MedImmune)

13.6 Chugai Pharmaceutical

13.7 Eli Lilly

13.8 EMD Serono

13.9 Emergent BioSolutions

13.10 Genentech

13.11 Genmab

13.12 Immunomedics

13.13 Jounce Therapeutics

13.14 MacroGenics

13.15 Merus

13.16 Neovii Biotech

13.17 NovImmune SA

13.18 OncoMed Pharmaceuticals

13.19 Pieris

13.20 Regeneron Pharmaceuticals

13.21 Roche

13.22 Sanofi

Contact:Neeraj Chawla[emailprotected]+91-9810410366

Logo: https://mma.prnewswire.com/media/1245952/Kuick_Research_Logo.jpg

SOURCE Kuick Research

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Global Bispecific Antibodies Therapy Blincyto Hemlibra Therapeutics Market Size To Witness 118% Growth To Reach US$ 10 Billion By 2026 - PR Newswire...

Newswise PHILADELPHIA Picture a future where embedded medical devices not only treat and teach patients, but learn from them. Imagine chronic pain being managed without the negative side effects of opioids. Penn Medicine researchers are working to make those scenarios a reality. These initiatives are among innovative new National Institutes of Health (NIH) funded efforts that are mapping the future of medicine.

The NIH has selected two researchers from the Perelman School of Medicine at the University of Pennsylvania to receive its prestigious Directors Awards, part of the NIH Common Funds High-Risk, High-Reward Research Program honoring exceptionally creative scientists. Brian Litt, MD, a professor of Neurology, Neurosurgery, and Bioengineering, was honored with a Pioneer Award for $5.6 million, supporting novel neurodevice research. Gregory Corder, PhD, an assistant professor of Psychiatry and Neuroscience, was selected as a New Innovator Award winner, receiving $2.4 million for research investigating the mechanisms of chronic pain.

This NIH initiative, designed to fuel research endeavors that are more opened-ended and have a potentially broader effect on scientific understanding compared to more traditional research, awards these to scientists to support research over a five-year period. The 2020 Penn recipients are among 85 awardees nationally:

Pioneer Award Ghost in the Machine: Melding Brain, Computer and Behavior

The Pioneer Award challenges investigators to pursue new research directions and develop groundbreaking, high-impact approaches to a broad area of biomedical or behavioral science. This award supports Litts work to develop a new generation of autonomous neurodevicesimplanted machines that can question, record, and combine learning algorithms based on neurological signals and feedback to act and alter human behavior on the fly.

In epilepsy, for example, these devices would predict and prevent seizures; in Parkinsons patients, implants will measure and communicate with patients to improve mobility, reduce tremor and enhance responsiveness. Other implants might improve hearing or psychiatric symptoms by querying patient perceptions, feelings, and altering stimulation patterns algorithmically to improve them. The loop is closed in real time, so the host can change their behavior based upon device feedback to improve their health.

Imagine this: A 30 year-old veteran walks into a bar. None of the patrons are aware of the anti-seizure device in his brain, or the traumatic brain injury that requires it. After he drinks a beer, his phone vibrates with a text from the implantable device asking what hes doing and sharing his probability of a seizure has increased. And with a quick explanation from the veteran, the device stimulates and suggests avoiding a second beverage, Litt explains. Were working towards this future, with the help of the Pioneer Award.

This is a paradigm shift from todays simple devices, which rely on physicians to give device feedback to patients and change simple parameters by hand during occasional office visits. Litts goal is to build a foundation for responsive implants that can collaborate with hosts, linking human experience and perception to machine algorithms, actions, and therapy, predicting and preventing events before they start. Not only will the patient teach the device, but the device will teach the patient.

Grant ID: DP1 NS122038-01.

New Innovator Award Harnessing Cortical Neuromodulation to Disrupt Pain Perception

The New Innovator Award supports unusually innovative research from early career investigators who have not yet received a research project grant or equivalent NIH grant. The award will support Corders efforts to research the mechanisms of chronic paina major health crisis in the United States, affecting millions, and a driver of the opioid epidemic.

Corders goal is to identify which parts of the brain are important for pain perception and which circuits impact pain relief from opioids. He hopes to decode how this neural activity evolves during chronic pain. Once the brain circuits and pathways that contribute to the suffering and perception of pain are identified, they can be targeted for potential therapeutics which could be more effective at reducing pain and without the addictive elements of prescription opioids.

Corder envisions these next generation pain therapeutics leveraging viral-delivered cell-specific gene therapies to disrupt the pain-processing circuits in the cortex. This would will allow patients to sense pain but without the unpleasant aversion.

We currently have a limited understanding of the neural pathways in the brain that contribute to pain, which has been a significant barrier for treating pain efficiently, without negative side effects. But, if we can identify and understand these circuits, we can then try to rewrite the neural code of pain, Corder said. Picture being able to specifically target the one desired brain region or circuit that processes pain in order to provide pain relief. This research will help us lay the groundwork for new classes of therapeutics, which could have a profound and broad impact for treating patients with chronic pain, while reducing the burden of the national opioid crisis.

Grant ID: DP2 GM140923-01.

###

Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.6 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $494 million awarded in the 2019 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 43,900 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2019, Penn Medicine provided more than $583 million to benefit our community.

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Penn Medicine Researchers Receive Prestigious National Institutes of Health Directors Awards - Newswise

LONDON, Oct. 06, 2020 (GLOBE NEWSWIRE) -- (Oncology Drugs Companies Included: F. Hoffmann-La Roche AG, Novartis AG, Bristol-Myers Squibb Company, Merck & Co., Inc., Johnson & Johnson)

The global oncology/cancer drugs market size reached a value of nearly $167.9 billion in 2019, having increased at a compound annual growth rate (CAGR) of 9.8% since 2015. The oncology market size 2020 is expected to decline from $167.9 billion in 2019 to $149.9 billion in 2020 at a rate of -11%. The decline is mainly due to lockdown and social distancing norms imposed by various countries and economic slowdown across countries owing to the COVID-19 outbreak and the measures to contain it. The market is then expected to grow slightly from $201.1 billion in 2021 to $284.5 billion in 2023 at a CAGR of 9.7%. North America has the largest cancer drug market share, accounting for 41.2% of the total market.

The Business Research Companys report titled Oncology Drugs Market - Opportunities And Strategies - Global Forecast To 2030 covers major oncology drugs companies, oncology drugs market share by company, oncology drugs manufacturers, oncology drugs infrastructure market size, and oncology drugs market forecasts. The report also covers the global oncology drugs market and its segments. The cancer market by type is segmented into drugs for lung cancer, pancreatic cancer, breast cancer, prostate cancer, ovarian cancer, colorectal cancer, gastric cancer, kidney cancer, brain tumor, thyroid cancer, skin cancer, bladder cancer, cervical cancer, blood cancer and others, by drug class type into targeted therapy, immunotherapy (biologic therapy), chemotherapy and hormonal therapy, by distribution channel into hospital pharmacies, retail pharmacies/drug stores and others, by route of administration into oral, parental and others, by drug classification into branded drugs and generic drugs.

Request For A Sample Of The Global Oncology Drugs Market Report:

https://www.thebusinessresearchcompany.com/sample.aspx?id=3440&type=smp

Companies in the oncology drugs market are increasing their product innovation through strategic collaborations. To sustain in the increasingly competitive market, organizations are developing innovative products as well as sharing skills and expertise with other such enterprises. While oncology drug companies have long collaborated with each other as well as with academic and research institutions in this market by way of partnerships, in or out licensing deals, this trend has been increasing over the recent years.

New technologies are being implemented in the cancer drug market, such as artificial intelligence in the research and development process, as well as 3D printing devices to mimic the human body for trials and testing of the drugs developed. Companies are also investing in technologies to develop next generation biologics such as antibody drug conjugates, bispecifics, fusion proteins, cell and gene therapy that will be more effective as they are expected to have better potency against the target disease as well as have the ability to treat more than one aspect of the disease. Over the last few years, there has been a significant rise in accelerated approval of cell and gene therapies for cancer treatment by regulatory bodies across the globe. CRISPR technology can be used to discover the non-coding cancer genome. CRISPR-Cas9 genome editing reduces the processing associated with the generation of cell line and animal models of cancer and complex generations. Thus, it generates a better cancer model for target validation and drug evaluation. CRISPRCas9 is accelerating the different stages of oncology drug discovery including target identification, validation and deconvolution, drug synthesis, assessment of drug sensitivity and resistance.

With innovation through technology and rising focus on development of new drugs, the oncology drugs market trends ought to bring advances in cancer therapies in the forecasted period.

Oncology Drugs Market - Opportunities And Strategies - Global Forecast To 2030 is one of a series of new reports from The Business Research Company that provide oncology drugs market overviews, oncology drugs market analyze and forecast oncology drugs market size and growth for the whole market, oncology drugs market segments and oncology drugs market geographies, oncology drugs market trends, oncology drugs market drivers, oncology drugs market restraints, oncology drugs market leading competitors revenues, profiles and market shares in over 1,000 industry reports, covering over 2,500 market segments and 60 geographies.

The report also gives in-depth analysis of the impact of COVID-19 on the market. The reports draw on 150,000 datasets, extensive secondary research, and exclusive insights from interviews with industry leaders. A highly experienced and expert team of analysts and modelers provides market analysis and forecasts. The reports identify top countries and segments for opportunities and strategies based on market trends and leading competitors approaches.

Here Is A List Of Similar Reports By The Business Research Company:

Clinical Oncology Next Generation Sequencing Global Market Report 2020-30: COVID 19 Growth and Change

Interventional Oncology Devices Global Market Report 2020-30: COVID 19 Growth and Change

Cancer Diagnostics Global Market Report 2020-30: COVID 19 Growth And Change

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Oncology Market Size 2020 Particularly Prone To Disruption During Pandemic In The Global Oncology Market - GlobeNewswire

DELHI, India, Oct. 07, 2020 (GLOBE NEWSWIRE) -- "Global Bispecific Antibody Market Opportunity, Drug Sales & Clinical Trials Insight 2026" Report Highlights:

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https://www.kuickresearch.com/report-global-usa-europe-japan-bispecific-antibody-antibodies-therapeutics-therapy-market-sales-size--trends-clinical-trials-emicizumab-hemlibra-blincyto--blinatumomab-sales

Despite having several hundred clinical success stories of different immunotherapies against solid and advanced cancers, the overall response rate of the patients has reached approximately 50% even when the patients who fit completely under all the criteria of treatment. To pave with the clinical failures, researchers have developed and evaluated bispecific therapeutic antibodies, that are combination of two binding specificities leading to improve selectivity as well as efficacy of antibody-based treatment for cancer. The arrival of bispecific antibody for cancer treatment has estimated to be of great potential for wide range of applications in cancer treatment. The current biotherapeutic market for cancer treatment and prevention is estimated to be dominated by antibody drugs, where bispecific antibody represents a novel ally for the next-generation of cancer therapy.

In the past few years, the remarkable progress that has been made with respect to understanding the important hallmarks of the cancer progression and main epitopes involved in it have led to paradigmatic development in the bispecific antibody capable of offering novel promises to the patients who havent respond to any medical therapy for cancer. The practice of bispecific antibody is not new i.e. this version of cancer therapeutics is a modified version of monoclonal antibody. By observing the increasing incidence of cancer, the management associated with the bispecific antibody has started to develop several strategies important for clinical management of the cancer in the present and in the future.

As per the analysis conducted, it is estimated that the bispecific antibody therapeutics market is growing at a double-digit rate, thus boosting the overall cancer therapeutics market to extreme, especially when compared with other immunotherapies. Further, trends and opportunities coupled within the market i.e. rise in the number of cancer cases, government favorable policies and many others are tending to drive the growth of the market. In our viewpoint, novel alliances developed by major key players of the market and local manufacturers are estimated to be playing important role for delivering tremendous opportunities for the researchers, patients, stakeholders and the payers.

The analysis of the Global bispecific antibody drug market represents that the market is completely focused on providing a better structure to the overall therapeutics market and offer the pharmaceutical industry a broad perspective of an efficient therapy for every cancer type. The research report provides analysis about the assess growth potential of the market by verifying various driving forces, in combination with the all the important trends, opportunities and challenges. In addition to it, the research report also provides a long-term outlook for the industry and various other important measures that have helped the market to attract thousands of investors and local drug manufacturers. Lastly, the report also profiles various pharmaceutical companies of the dominating markets that have been boosting the R&D sector of the market.

Neeraj Chawla neeraj@kuickresearch.com +91-9810410366

Table of Contents

1. Introduction to Bispecific Monoclonal Antibody

1.1 Overview of Monoclonal Antibody 1.2 Overview of Bispecific Monoclonal Antibody 1.3 Advantage of Bispecific Antibodies upon Monospecific Monoclonal Antibodies

2. The Engineering & Development of Bispecific Antibodies

2.1 Bispecific Antibodies Similar to IgG

2.1.1 Quadroma (Hybrid Hydromas) Approach 2.1.2 Knobs Into Holes Approach 2.1.3 CrossMab Approach 2.1.4 Dual-Variable-Domain Immunoglobulin Approach

2.2 Small Bispecific Antibodies

2.2.1 Bispecific Diabodies 2.2.2 Bispecific T-Cell Engager Antibodies (BiTEs)

3. Mechanism Of Action Of Bispecific Antibody

3.1 Trifunctional Antibody: Catumaxomab (Removab) 3.2 Blinatumomab

4. Applications of Bispecific Antibodies in Diagnostic & Treatment

4.1 BsMAb for Diagnosis of Bacterial & Viral Infectious Diseases 4.2 BsMAb for Cancer Diagnostic 4.3 BsAbs Blocking Signaling Pathways 4.4 BsAbs Targeting Tumor Angiogenesis 4.5 Specific Delivery of Effector Compounds to Targets 4.6 Bispecific Antibodies & Gene Therapy

5. Global Bispecific Antibody Market Outlook

5.1 Current Market Scenario

5.2 Availability of Bispecific Antibodies 5.2.1 Catumaxomab (Removab) 5.2.2 Blinatumomab 5.2.3 Emicizumab 5.2.4 Duligotumab 5.2.5 SAR 156597

6. Approved Bispecific Antibody Dosage & Price Analysis

6.1 Blincyto 6.2 Hemlibra

7. Approved Bispecific Antibody Sales Analysis

7.1 Blincyto 7.2 Hemlibra

8. Global Bispecific Antibodies Clinical Pipeline Overview

8.1 By Phase 8.2 By Country/Region 8.3 By Company 8.4 By Indication 8.5 Orphan Designated Bispecific Antibodies

9. Global Bispecific Antibodies Clinical Pipeline By Company, Indication & Phase

9.1 Research 9.2 Preclinical 9.3 Phase-I 9.4 Phase-I/II 9.5 Phase-II 9.6 Phase-II/III 9.7 Phase-III

10. Marketed Bispecific Antibodies Clinical Insight

10.1 Blinatumomab - Amgen 10.2 Emicizumab - Chugai Pharmaceutical

11. Global Bispecific Antibody Market Dynamics

11.1 Favorable Market Parameters 11.2 Commercialization Challenges

12. Global Bispecific Antibody Market Future Prospects

13. Competitive Landscape

13.1 Ablynx 13.2 Adimab 13.3 Affimed Therapeutics 13.4 Amgen 13.5 AstraZeneca (MedImmune) 13.6 Chugai Pharmaceutical 13.7 Eli Lilly 13.8 EMD Serono 13.9 Emergent BioSolutions 13.10 Genentech 13.11 Genmab 13.12 Immunomedics 13.13 Jounce Therapeutics 13.14 MacroGenics 13.15 Merus 13.16 Neovii Biotech 13.17 NovImmune SA 13.18 OncoMed Pharmaceuticals 13.19 Pieris 13.20 Regeneron Pharmaceuticals 13.21 Roche 13.22 Sanofi

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Global Bispecific Antibodies Therapy Blincyto Hemlibra Antibody Therapeutics Market Size To Witness 118% Growth To Reach US$ 10 Billion By 2026 -...

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

From Concept to Market: Overcoming the Challenges of Manufacturing and Clinical Trials

Learn how CRO/CDMOs successfully address operational and regulatory challenges for pharmaceutical and biotechnology clients; and how this can make the difference between study success or failure.

Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

The rest is here:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

With ongoing advances in science and technology, the cell and gene therapy pipeline has grown especially robust over the past few years. At present, ClinicalTrials.gov shows more than 4,500 active gene therapy trials globally. In the United States, McKinsey experts expect to see 10 to 20 cell and gene therapy approvals per year over the next five years.

This rise in supply has created a heightened demand for contract development and manufacturing organizations (CDMOs) with biotech expertise. CDMOs typically supply materials and handle production and manufacturing, allowing life sciences companies to focus on innovation and marketing.

The bottleneck stems from a shortage of CDMOs with gene therapy expertise and resources. Considering the critical need for safe, effective gene therapies and the rapid pace of development, it's important for pharma and biopharma to find a CDMO with both gene therapy capabilities and availability to take on new partners nownot 18 months from now.

"Full-service CDMOs that can assist with both development and manufacturing are in highest demand,"said Richard Welch, PhD, vice president, development services for Emergent BioSolutions, a global CDMO and specialty life sciences company headquartered in Gaithersburg, Maryland. "As pharma and biopharma companies move from early phase to late phase, CDMOs need experience with process characterization and process validation as well as commercial production and supply chain."

"The supply chain is much more complex,"added Tarek Abdel-Gawad, senior director of commercial strategy for Emergent BioSolutions. "You aren't just growing cells. You're ensuring viruses, helper viruses, and plasmid DNA work together to produce the molecule of choice. Few companies have the capabilities, equipment, and GMP expertise."

Much of the gene therapy development as of late has stemmed from smaller biotech companies or research universities according to a McKinsey report. Large pharmaceutical companies may partner with these organizations on rare disease or oncology treatments two therapeutic areas where much of the research lies.

Many small to midsize companies have the idea and investor support, but do not have the employees, infrastructure or manufacturing space. "A CDMO is a good partner in those cases,"said Mukesh Mayani, PhD, principal scientist, gene therapy at Sanofi. "You can test your hypotheses and work with a CDMO that has the platform, the people, and the preclinical models. This arrangement speeds up the timeline and allows these innovative companies to focus on other modalities and molecules."

Pharma and biopharma companies of all sizes can learn from this "single-source"approach. Partnering with a CDMO earlier in the processfrom preclinical development through packagingfrees up resources to focus on innovation and communication.

"It is neither simple nor cheap to develop and manufacture gene therapies,"said Dr. Welch. "A CDMO has the built-in skill set to grow viruses at the densities necessary to meet early-phase studies while hitting safety margins. With the clinical trial failure rate as high as it is, working with a CDMO that has experience in different technologies and products makes for a more efficient, cost-effective process."

Although there is a high demand now for CDMOs with gene therapy expertise, the market is quickly growing. According to Grand View Research, the CDMO market is expected to grow from $115.6 billion in 2020 to $157.7 billion in 2025, outpacing the pharmaceutical industry as a whole. New cell and gene therapy CDMOs are emerging and established CDMOs are expanding capabilities.

Before you start your CDMO search, consider the following two factors:

When vetting CDMOs for your gene therapy studies, consider the strengths and weaknesses of your company as well as your potential CDMO partner. A few points to consider include:

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment. Choose a partner that can assist from the earliest phases of product development all the way to commercialization.

Capra, Emily, et al. "Gene therapy coming of age: Opportunities and challenges to getting ahead."McKinsey, October 2, 2019

Link:
Gene therapy solution: The value of a CDMO as your end-to-end partner - BioPharma Dive

SAN RAFAEL, Calif., Oct. 2, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation toBMN 307, an investigational gene therapy for the treatment of individuals with PKU.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation.

"Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

Last week, BioMarin announced that it had dosed the first participant in the global Phearless Phase 1/2 study with BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.BioMarin is conducting this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

BioMarin's clinical program is composed of two key studies. Phearless, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, Phenom, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company's BMN 307 program being eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions, BioMarin's gene therapy manufacturing capabilities and the anticipation that the current manufacturing capabilities will meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future and the impact of using material manufactured at commercial scale in a clinical trial on reducing risk and speeding up overall development timelines. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

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https://www.biomarin.com/

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 -...

Cell and Gene Therapy Market research report is the new statistical data source added by A2Z Market Research.

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JW CreaGene, Vericel, Tego Sciences, CHIESI Farmaceutici, Spark Therapeutics, GC Pharma, MolMed, AnGes, Takeda Pharmaceutical Company, APAC Biotech, Gilead Sciences, Corestem, AVITA Medical, Novartis AG, JCR Pharmaceuticals, Dendreon, CO.DON, Medipost, Osiris Therapeutics, Amgen, Biosolution, CollPlant, Japan Tissue Engineering, Organogenesis, Orchard Therapeutics, Stempeutics Research

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The cost analysis of the Global Cell and Gene Therapy Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

Global Cell and Gene Therapy Market Segmentation:

Market Segmentation by Type:

Cell TherapyGene Therapy

Market Segmentation by Application:

HospitalsWound Care CentersCancer Care CentersAmbulatory Surgical CentersOthers

Regions Covered in the Global Cell and Gene Therapy Market Report 2020:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The report provides insights on the following pointers:

Table of Contents

Global Cell and Gene Therapy Market Research Report 2020 2026

Chapter 1 Cell and Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

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

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

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Cell and Gene Therapy Market Forecast

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Comprehensive Report on Cell and Gene Therapy Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | JW CreaGene, Vericel, Tego...

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in boys with DMD.

Fast Track is a process designed to facilitate the development, and expedite the review, of new drugs that are intended to treat or prevent serious conditions that have the potential to address an unmet medical need. This designation was granted based on data from the Phase 1b study that indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. DMD is a devasting condition and patients, and their parents, are waiting desperately for treatment options. We are working to advance our planned Phase 3 program as quickly as possible.

DMD is a devastating and life-threatening X-linked disease that is caused by mutations in the gene encoding dystrophin, which is needed for proper muscle membrane stability and function. Patients present with muscle degeneration that progressively worsens with age to the extent that they require wheelchair assistance when they are in their early teens, and unfortunately, usually succumb to their disease by the time they are in their late twenties. It is estimated that there are ~10-12,000 individuals affected with DMD in the US.

About PF-06939926

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue. Pfizer initiated the Phase 1b multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 in 2018. The goal of the study is to assess the safety and tolerability of this investigational gene therapy. Other objectives of the clinical study include measurement of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function.

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

DISCLOSURE NOTICE: The information contained in this release is as of October 1, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about PF-06939926, an investigational gene therapy to potentially treat Duchenne muscular dystrophy, including its potential benefits and a planned Phase 3 study for PF-06939926, that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risks associated with initial and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when regulatory authorities will approve the commencement of our planned Phase 3 study; whether and when drug applications may be filed in any jurisdictions for any potential indication for PF-06939926; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether PF-06939926 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of PF-06939926; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

Read more:
Pfizer Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Investigational Gene Therapy - Business Wire

Latest Research Report: Gene Therapy industry

Gene Therapy Market report is to provide accurate and strategic analysis of the Profile Projectors industry. The report closely examines each segment and its sub-segment futures before looking at the 360-degree view of the market mentioned above. Market forecasts will provide deep insight into industry parameters by accessing growth, consumption, upcoming market trends and various price fluctuations.

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

Gene Therapy Market competition by top manufacturers as follow:SangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVicalAdvantagene

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Global Gene Therapy Market research reports growth rates and market value based on market dynamics, growth factors. Complete knowledge is based on the latest innovations in the industry, opportunities and trends. In addition to SWOT analysis by key suppliers, the report contains a comprehensive market analysis and major players landscape.The Type Coverage in the Market are: Ex vivoIn vivo

Market Segment by Applications, covers:Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

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Gene Therapy Market Incredible Possibilities, Growth Analysis and Forecast To 2025 - The Daily Chronicle