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DUBLIN--(BUSINESS WIRE)--Sep 21, 2020--

The "Protein Therapeutics Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

The global protein therapeutics market grew at a CAGR of around 7% during 2014-2019. Looking forward, the publisher expects the global protein therapeutics market to continue its moderate growth during the next five years.

Protein therapeutics refers to artificially synthesized protein-based medicines. They are fast-acting, potent medicines that deliver small protein molecules to the body in a specific amount. They usually consist of recombinant forms of naturally occurring proteins, such as monoclonal antibodies, insulin, fusion proteins, erythropoietin, interferon, human growth hormones (HGH) and follicle-stimulating hormones. They aid in treating chronic medical ailments, such as cancer, diabetes, neurodegenerative disorders and immunological, hematological, hormonal and genetic disorders. Various combination therapy drugs are also used with protein therapeutics that can be inhaled, injected or orally administered.

The increasing prevalence of chronic medical ailments is one of the key factors driving the growth of the market. In line with this, the rising awareness among the masses regarding the benefits of protein therapeutics, such as minimal risks of side effects and high efficiency, are contributing to the market growth. Monoclonal antibodies are being widely researched and used for the treatment of various viral and bacterial diseases and pharmaceutical companies are using protein therapeutics for drug discovery and development. The sudden outbreak of the coronavirus disease (COVID-19) is further providing growth opportunities to market players. For instance, Molecular Partners AG, a Switzerland-based clinical stage biotechnology company, is developing a new class of protein therapeutics, called DARPin, to inhibit the proliferation of the virus.

Additionally, the development of novel recombinant proteins, peptides, antibody-based drugs and plasma proteins is acting as other-growth inducing factors. These protein therapeutics are extensively used in replacement therapies to treat genetic and autoimmune disorders, such as dysfibrinogenemia, afibrinogenemia, and hypofibrinogenemia. Other factors, including extensive research and development (R&D) activities in the field of protein engineering, along with significant improvements in the healthcare infrastructure, are expected to drive the market further.

Key Questions Answered in This Report:

Key Topics Covered:

1 Preface

2 Scope and Methodology

2.1 Objectives of the Study

2.2 Stakeholders

2.3 Data Sources

2.3.1 Primary Sources

2.3.2 Secondary Sources

2.4 Market Estimation

2.4.1 Bottom-Up Approach

2.4.2 Top-Down Approach

2.5 Forecasting Methodology

3 Executive Summary

4 Introduction

4.1 Overview

4.2 Key Industry Trends

5 Global Protein Therapeutics Market

5.1 Market Overview

5.2 Market Performance

5.3 Impact of COVID-19

5.4 Market Forecast

6 Market Breakup by Component

6.1 Monoclonal Antibodies (mAbs)

6.1.1 Market Trends

6.1.2 Market Forecast

6.2 Human Insulin

6.2.1 Market Trends

6.2.2 Market Forecast

6.3 Erythropoietin

6.3.1 Market Trends

6.3.2 Market Forecast

6.4 Clotting Factors

6.4.1 Market Trends

6.4.2 Market Forecast

6.5 Fusion Protein

6.5.1 Market Trends

6.5.2 Market Forecast

6.6 Others

6.6.1 Market Trends

6.6.2 Market Forecast

7 Market Breakup by Therapy Area

7.1 Metabolic Disorders

7.1.1 Market Trends

7.1.2 Market Forecast

7.2 Immunological Disorders

7.2.1 Market Trends

7.2.2 Market Forecast

7.3 Hematological Disorders

7.3.1 Market Trends

7.3.2 Market Forecast

7.4 Cancer

7.4.1 Market Trends

7.4.2 Market Forecast

7.5 Hormonal Disorders

7.5.1 Market Trends

7.5.2 Market Forecast

7.6 Genetic Disorders

7.6.1 Market Trends

7.6.2 Market Forecast

7.7 Others

7.7.1 Market Trends

7.7.2 Market Forecast

8 Market Breakup by Function

8.1 Enzymatic and Regulatory Activity

8.1.1 Market Trends

8.1.2 Market Forecast

8.2 Special Targeting Activity

8.2.1 Market Trends

8.2.2 Market Forecast

8.3 Vaccines

8.3.1 Market Trends

8.3.2 Market Forecast

8.4 Protein Diagnostics

8.4.1 Market Trends

8.4.2 Market Forecast

9 Market Breakup by Region

9.1 North America

9.2 Asia Pacific

9.3 Europe

9.4 Latin America

9.5 Middle East and Africa

10 SWOT Analysis

11 Value Chain Analysis

12 Porters Five Forces Analysis

13 Price Analysis

14 Competitive Landscape

14.1 Market Structure

14.2 Key Players

14.3 Profiles of Key Players

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/pi68kz

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Global Protein Therapeutics Market (2020 to 2025) - by Component, Therapy Area, Function & Region - ResearchAndMarkets.com - The Baytown Sun

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that statistical analysis of pooled data from LUMEVOQ trials and natural history studies found a statistically significant and clinically meaningful difference between the visual outcomes in LUMEVOQ-treated patients and untreated patients.

Treated eyes showed progressive and sustained improvement from Month 12 to Month 52, in contrast to the absence of recovery over the same period for untreated eyes. At Month 18, the difference became statistically significant (p=0.01). By Month 48, the difference between the mean visual acuity in treated patients and that in untreated patients was both statistically significant (p<0.01) and clinically meaningful (-0.33 LogMAR, or +16.5 ETDRS letters equivalent, in favor of treated eyes).

Note: All patients had a confirmed G11778A mutation in the ND4 mitochondrial gene and were at least 15 years old. The diagram shows the Locally Estimated Scatterplot Smoothing (LOESS) curves for visual acuity in LUMEVOQ-treated patients and untreated patients. The shaded areas represent the 95% confidence interval for the mean BCVA. Treated eyes refer to all eyes (LUMEVOQ and sham) from the RESCUE, REVERSE and CLIN06 trials (N=76 patients / 152 eyes). Untreated eyes refer to patient-level data from the REALITY study and a matched data set from two prospective and eight retrospective natural history studies1 (N=208 patients / 408 eyes). LOESS curves were estimated using a non-parametric, local regression model that treated each eye as independent of the other. LOESS curves are shown from Month 12 to depict post-treatment progression among treated patients (93% of LUMEVOQ patients had already been treated within 12 months from onset). *Statistically significant difference between mean visual acuity of treated and untreated eyes at M18, M24, M36 and M48, as illustrated by the non-overlapping confidence intervals.

The analysis compared data from the completed Phase III trials RESCUE and REVERSE studies and interim results from the long-term follow-up CLIN06 study to a matched sample created from the REALITY registry study and 10 other natural history studies1. The natural history studies were identified from an extensive review of the scientific literature and selected based on specific inclusion criteria for their patient-level data. In all, the visual outcomes in 76 treated patients could be compared to the visual outcomes of 208 untreated patients.

The extra granularity of this analysis down to individual patient data confirms that the natural history of visual outcomes in ND4-LHON patients is poor and provides the best comparison we have for assessing the therapeutic efficacy of gene therapy, commented Dr. Nancy J. Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology at the Emory University School of Medicine in Atlanta, GA, USA, and a global authority on LHON who recently completed a meta-analysis of the natural history of ND4-LHON.2

Separate analyses of patients enrolled in RESCUE and REVERSE demonstrated similarly favorable results compared to untreated patients. Full findings from the indirect comparison were included in the European Marketing Authorisation Application (MAA) for LUMEVOQ and are being prepared for publication in a peer-reviewed journal.

This indirect comparison represents a significant contribution to our understanding of LUMEVOQs therapeutic effect," said Dr. Jos-Alain Sahel, MD, Director of the Institut de la Vision (Sorbonne-Universit/Inserm/CNRS), Paris, France; Chairman of the Department of Ophthalmology at Centre Hospitalier National dOphtalmologie des XV-XX, Paris, France; Professor and Chairman of the Department of Ophthalmology at University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center), USA; and Co-Founder of GenSight Biologics. The use of a large external control group, including analyses of patient-level data, provides more evidence for a significant treatment-related visual improvement.

The findings are a gratifying outcome of our push to overcome a key challenge for assessing LUMEVOQs efficacy, namely the inability of sham eyes to act as a control group, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. We are excited to take this evidence that LUMEVOQ modifies the disease outcome forward into our conversations with national and regional authorities.

The LUMEVOQ MAA was filed in September, and the decision is expected in H2 2021. The Company is also working towards submitting LUMEVOQs Biologics License Application (BLA) to the FDA in H2 2021.

GenSight will host a conference call today, September 21, 2020, at 10am CEST in French, and at 2.00pm CEST (8.00am EST) in English, to discuss these results.

Webcast & Conference call in French (10am CEST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0)1 7037 7166United Kingdom: +44 (0)20 3003 2666Password: GenSight FR

Webcast link: https://bit.ly/3mAS0Vy

Webcast & Conference call in English (2.00pm CEST / 8.00am EST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0) 1 7037 7166United Kingdom: +44 (0) 20 3003 2666Password: GenSight ENG

Webcast link: https://bit.ly/35RCfnl

A replay of the calls and webcasts will be available by using the above links.

Rationale for the Indirect Comparison

In both the REVERSE and RESCUE studies, as well as in the long-term follow-up study CLIN06, unilaterally injected patients experienced an unexpected visual improvement in their contralateral eye, which mirrored the sustained and clinically relevant gain in eyes treated with LUMEVOQ. This bilateral improvement eliminated the control group that was to consist of sham eyes in the original trial design. An indirect comparison methodology, based on formal statistical methods applied to an external control group, was needed to assess the magnitude of LUMEVOQ efficacy.

Methodology Highlights

The sample of LUMEVOQ-treated patients included all data from the two pivotal studies REVERSE and RESCUE and Year 3 data from the ongoing extension study CLIN06. This approach yielded a treated patient pool consisting of 76 patients (152 eyes), with LUMEVOQ-injected eyes and sham-treated eyes considered equivalent, based on the contralateral effect demonstrated in the studies.

The external control group included data from the REALITY Natural History registry and patient-level data from 10 published articles1 on ND4-LHON, which were identified from a systematic review of the literature. ND4-LHON studies were included in the indirect comparison only if they had individual patient data that would allow indirect comparison with LUMEVOQ-treated patients: confirmed ND4 genotype, at least 15 years of age, at least one BCVA measurement with associated time of vision loss. The final external control group included 208 patients (408 eyes).

The visual acuity curves of treated and untreated patients were defined using a Locally Estimated Scatterplot Smoothing (LOESS), non-parametric, local regression model. They were aligned in terms of time from vision loss for the statistical tests performed at M12, M18, M24, M36 and M48 from vision loss and at the last available observation. The starting time point for the statistical tests (12 months post-vision loss) was defined to enable assessment of LUMEVOQ efficacy, as 93% of LUMEVOQ patients were treated by that time.

1The 10 studies that passed the inclusion criteria were: Hotta 1995, Lam 2014, Nakamura 1993, Newman 1991, Qu 2007, Qu 2009, Romero 2014, Sadun 2004, Yang 2016, and Zhou 2010.

2Newman NJ, Carelli V, Taiel M and Yu-Wai Man P. Visual outcomes in Leber hereditary optic neuropathy patients with the m.11778G>A (MTDN4) mitochondrial DNA mutation. J Neuro-Ophthalmol. In Press. 2020.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About LUMEVOQ (GS010)

LUMEVOQ (GS010) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint measures the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis was evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767

About CLIN06 (RESCUE and REVERSE Long-term Follow-up)

CLIN06 is the long-term follow-up study of ND4 LHON subjects treated with LUMEVOQ (GS010) gene therapy in the RESCUE or REVERSE Phase III Clinical Trials. The total study period for an individual subject is 3 years, i.e., 5 years post-gene therapy administration. No study treatment is administered during CLIN06.

The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment. The secondary objective is to assess the long-term treatment efficacy of the therapy and the quality of life (QoL) in subjects up to 5 years post-treatment. The first subject was enrolled on January 9, 2018. 61 subjects have enrolled.

ClinicalTrials.gov Identifiers:CLIN06: NCT03406104

About REALITY

REALITY is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on subjects medical charts and the administration of surveys on Health-Related Quality of Life (HRQoL) and direct and indirect costs associated with the disease.

The study aimed to recruit at least 50 subjects (both adult and pediatric) chiefly in the following countries: Spain, Italy, France, United Kingdom and the United States.

The primary objectives for the REALITY study were: to describe the evolution of visual functional and structural changes and other associated symptoms in patients with LHON; understand the impact of LHON-related vision loss on the HRQoL; and understand the economic burden for patients and their families arising from direct and indirect costs associated with the disease. The secondary objective is to describe the relationship between genetic, lifestyle and/or environmental factors and the expression of the LHON phenotype.

The first subject was enrolled on 3 January 2018. Enrollment was completed in early Q2 2020.

ClinicalTrials.gov Identifiers:REALITY LHON Registry: NCT03295071

The rest is here:
GenSight Biologics Reports New Analysis Demonstrating Statistically Significant and Clinically Meaningful Difference Between Visual Outcomes in...

Viral Vector and Vaccine Market report is an excellent report that makes it possible to the Viral Vector and Vaccine industry can be highly benefited with this market research report which brings market and competitive landscape clearly into the focus and help make better decisions. Market segmentation has also been performed in detail based on various parameters that include applications, verticals, deployment model, end user, and geography. Expert solutions combined with potential capabilities prepare this winning Viral Vector and Vaccine Market document to be outperforming for the Viral Vector and Vaccine

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Viral vector & vaccine market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to growing with the CAGR of 14.50% in the above mentioned forecast period. Rising prevalence of contagious and chronic diseases such astuberculosis, HIV, and cancer has given a boost to market for finding effective solution for these problems whereas upcoming new disease like with outbreak of COVID-19 has raised demand for minimal invasive technique for treatment for these rising medical problems.

The Leading Market Players Covered in this Report are :

Regional Analysis Includes:

Asia-Pacific-China, Japan, Korea, India, and Southeast Asia

Europe-Germany, France, UK, Russia, and Italy etc.

North America-The United States, Mexico, and Canada

South America-Brazil, Argentina, Columbia, etc.

The Middle East and Africa-Saudi Arabia, UAE, Egypt, Nigeria, and South Africa

Deep analysis about market status, enterprise competition pattern, advantages and disadvantages of enterprise products, industry development trends, regional industrial layout characteristics and macroeconomic policies, industrial policy has also be included.

Viral vector & vaccine market is segmented on the basis of type, workflow, application and end use. 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.

Viral vector & vaccine market is segmented on the basis of type, workflow, application and end use. 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.

From raw materials to downstream buyers of this industry will be analyzed scientifically, the feature of product circulation and sales channel will be presented as well. In a word, this report will help you to establish a panorama of industrial development and characteristics of the Viral Vector and Vaccine Market.

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Recent Developments

o Market Overview and growth analysiso Import and Export Overviewo Volume Analysiso Current Market Trends and Future Outlooko Market Opportunistic and Attractive Investment Segment

Key Questions Answered by Viral Vector and Vaccine Market Report

1. What was the Viral Vector and Vaccine Market size in 2018 and 2019?; what are the estimated growth trends and market forecast?2. What will be the CAGR of Viral Vector and Vaccine Market during the forecast period (2020 2027)?3. Which segments (product type/applications/end-user) were most attractive for investments in 2018?How these segments are expected to grow during the forecast period (2020 2027).4. Which manufacturer/vendor/players in the Viral Vector and Vaccine Market was the market leader in 2018?5. Overview on the existing product portfolio, products in the pipeline, and strategic initiatives taken by key vendors in the market.

There are 13 Chapters to thoroughly display the Viral Vector and Vaccine Market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

Chapter 1: Viral Vector and Vaccine Overview, Product Overview, Market Segmentation, Market Overview of Regions, Market Dynamics, Limitations, Opportunities and Industry News and Policies.

Chapter 2: Viral Vector and Vaccine Industry Chain Analysis, Upstream Raw Material Suppliers, Major Players, Production Process Analysis, Cost Analysis, Market Channels and Major Downstream Buyers.

Chapter 3: Value Analysis, Production, Growth Rate and Price Analysis by Type of Viral Vector and Vaccine.

Chapter 4: Downstream Characteristics, Consumption and Market Share by Application of Viral Vector and Vaccine.

Chapter 5: Production Volume, Price, Gross Margin, and Revenue ($) of Viral Vector and Vaccine by Regions (2020 2027).

Chapter 6: Viral Vector and Vaccine Production, Consumption, Export and Import by Regions (2020 2027).

Chapter 7: Viral Vector and Vaccine Market Status and SWOT Analysis by Regions.

Chapter 8: Competitive Landscape, Product Introduction, Company Profiles, Market Distribution Status by Players of Viral Vector and Vaccine.

Chapter 9: Viral Vector and Vaccine Market Analysis and Forecast by Type and Application (2020 2027).

Chapter 10: Market Analysis and Forecast by Regions (2020 2027).

Chapter 11:Industry Characteristics, Key Factors, New Entrants SWOT Analysis, Investment Feasibility Analysis.

Chapter 12:Market Conclusion of the Whole Report.

Chapter 13:Appendix Such as Methodology and Data Resources of This Research.

Find More Competitor in TOC with Profile Overview Share Growth Analysis @https://www.databridgemarketresearch.com/toc/?dbmr=global-viral-vector-and-vaccine-market

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GLOBAL CONGRESS ON ELDERLY CARE, GERONTOLOGY AND GERIATRICS

The world is ageing rapidly. People aged 60 and older make up 12.3 per cent of the global population, and by 2050, that number will rise to almost 22 per cent. With this surge many issues are underlying related to blood pressure, diabetes, heart failure issues, arthritis, cancer malignancy, joint pains, tuberculosis and majorly the lack of awareness regarding the changing behavioural patterns in elderly people at home leading to abuse of them by their kin.

The goal of this Conference is uniting all well-known gerontologists, geriatrics scholars, policy decision-makers, professional activists, related companies, and researchers to discuss and share knowledge in the emerging field of Aging and Gerontology and its related areas of research and how we can move towards more Healthy Living, Healthy Aging to Promote an Ageless Era and hence widening professional contact and create new opportunities, including establishing new collaborations to all the representatives.Registration Link: https://bit.ly/34ueelz

This content has been distributed via CDN Newswire press release distribution service. For press release enquires please mail us at contact@cdnnewswire.com.

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Viral Vector and Vaccine Market To 2027 Reporting And Evaluation Of Recent Industry Developments | - PharmiWeb.com

AMRYT PHARMA PLC

( Amryt ” or the Company ”)

Exercise of Warrants & Issue of Ordinary Shares and Total Voting Rights

DUBLIN, Ireland, and Boston MA, September 21 2020, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, announces that an institutional investor has exercised subscription rights relating to 4,229,753 zero cost warrants (the Warrants”).

These Warrants were issued in September 2019 as part of the Company’s acquisition of Aegerion. Certain institutional investors elected to receive Warrants to subscribe for new ordinary shares of 0.06 each (Ordinary Shares”), in place of the same number of Ordinary Shares, as consideration for the Company’s acquisition of Aegerion and their equity investments in the Company in September 2019. Each warrant entitles the holder to subscribe for one Ordinary Share for no additional consideration.

In order to satisfy the exercise of the Warrants, the Company will issue 4,229,753 ordinary shares of 0.06 each (the New Ordinary Shares”) to the institutional investor. It is expected that admission to trading of the New Ordinary Shares on AIM will become effective, and that dealings in the New Ordinary Shares will commence at 8.00 a.m. BST on 22 September 2020.

On admission of the New Ordinary Shares, the issued share capital of the Company will comprise 167,593,296 Ordinary Shares. The Company holds 4,864,656 Ordinary Shares in treasury. Therefore, the total number of voting rights in the Company is 162,728,640. This figure may be used by shareholders as the denominator for the calculation by which they will determine if they are required to notify their interest in, or a change to their interest in, the Company under the FCA’s Disclosure Guidance and Transparency Rules. The Company will also have in issue 8,966,520 zero cost warrants.

Enquiries :

Rory Nealon, CFO/COO

LifeSci Advisors, LLC

Consilium Strategic Communications

About Amryt

Amryt is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases. Amryt comprises a strong and growing portfolio of commercial and development assets.

Amryt’s commercial business comprises two orphan disease products.

Juxtapid®/ Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolaemia ("HoFH") in the US, Canada, Columbia, Argentina and Japan (under the trade name Juxtapid®) and in the EU (under the trade name Lojuxta®). HoFH is a rare genetic disorder which impairs the body's ability to remove low density lipoprotein ("LDL") cholesterol ("bad" cholesterol) from the blood, typically leading to abnormally high blood LDL cholesterol levels in the body from before birth - often ten times more than people without HoFH - and subsequent aggressive and premature cardiovascular disease.

Myalept® / Myalepta® (metreleptin) is approved in the US (under the trade name Myalept®) as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL) and in the EU (under the trade name Myalepta®) for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and above and familial or acquired partial lipodystrophy (PL) in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. Metreleptin is also approved for lipodystrophy in Japan. Generalised and partial lipodystrophy are rare disorders characterised by loss or lack of adipose tissue resulting in the deficiency of the hormone leptin, produced by fat cells and are associated with severe metabolic abnormalities including severe insulin resistance, diabetes, hypertriglyceridemia and fatty liver disease.

Amryt's lead development candidate, FILSUVEZ® is a potential treatment for the cutaneous manifestations of EB, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. In September 2020, Amryt reported positive top line results from its pivotal global phase 3 trial of FILSUVEZ® in EB. The primary endpoint of the trial was met (p-value = 0.013). FILSUVEZ® has been granted Rare Pediatric Disease Designation and has also received a Fast Track Designation from the U.S. Food and Drug Administration. The global market opportunity for EB is estimated by the Company to be in excess of $1.0 billion.

In March 2018, Amryt in-licenced a preclinical gene-therapy platform technology, AP103, which offers a potential treatment for patients with Dystrophic Epidermolysis Bullosa, a subset of EB, and is also potentially relevant to other genetic disorders.

For more information on Amryt, including products, please visit http://www.amrytpharma.com .

This announcement contains inside information for the purposes of article 7 of the Market Abuse Regulation (EU) 596/2014.

The person making this notification on behalf of Amryt is Rory Nealon, CFO/COO and Company Secretary.

Financial Advisors Shore Capital (Edward Mansfield, Daniel Bush, John More) are NOMAD and Joint Broker to Amryt in the UK. Stifel (Ben Maddison) are Joint Broker to the company in the UK. Davy (John Frain, Daragh O’Reilly) act as Joint Broker to the company.

Forward-Looking Statements Statements in this announcement with respect to Amryt's business, strategies, timing for completion of and announcing results from the EASE trial, the potential impact of closing enrollment in the EASE trial, as well as other statements that are not historical facts are forward-looking statements involving risks and uncertainties which could cause the actual results to differ materially from such statements. Statements containing the words "expect", "anticipate", "intends", "plan", "estimate", "aim", "forecast", "project" and similar expressions (or their negative) identify certain of these forward-looking statements. The forward-looking statements in this announcement are based on numerous assumptions and Amryt's present and future business strategies and the environment in which Amryt expects to operate in the future. Forward-looking statements involve inherent known and unknown risks, uncertainties and contingencies because they relate to events and depend on circumstances that may or may not occur in the future and may cause the actual results, performance or achievements to be materially different from those expressed or implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and consummate investments. Many of these risks and uncertainties relate to factors that are beyond each of Amryt's ability to control or estimate precisely, such as future market conditions, the course of the COVID-19 pandemic, currency fluctuations, the behaviour of other market participants, the outcome of clinical trials, the actions of regulators and other factors such as Amryt's ability to obtain financing, changes in the political, social and regulatory framework in which Amryt operates or in economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representation or warranty, express or implied, is made regarding future performance. No person is under any obligation to update or keep current the information contained in this announcement or to provide the recipient of it with access to any additional relevant information that may arise in connection with it. Such forward-looking statements reflect the Company’s current beliefs and assumptions and are based on information currently available to management.

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Adding abemaciclib to hormonal therapy reduces the risk of cancer recurrence by 25% in patients with high-risk early hormone receptor positive (HR+) human epidermal growth factor receptor 2 negative (HER2-) breast cancer, according to results from a study atESMO 2020. (1)

This is the first time in more than 20 years that we have seen an advance in the adjuvant treatment of this form of breast cancer, said lead author Prof Stephen Johnston, from the Royal Marsden Hospital NHS Foundation Trust, London, UK. He explained that hormone receptor positive breast cancer is the commonest form of breast cancer, affecting 70% of patients, with most being diagnosed with early disease.

Many of these patients can be cured with currently available treatments: surgery, radiotherapy, chemotherapy and hormone treatment. But about 20% have high-risk disease and will develop a recurrence either locally in the breast or elsewhere in the body over the first ten years of treatment, he explained.

These patients with high-risk early breast cancer show a degree of resistance to hormone therapy, relapsing early despite everything we currently give them, said Johnston. CDK4/6 inhibitors, such as abemaciclib, have transformed the way we treat metastatic breast cancer over the last few years, overcoming primary endocrine resistance and improving survival. So it was an obvious step to see whether adding abemaciclib to hormone treatment in patients with high-risk early breast cancer could reduce the risk of their cancer returning.

The international phase 3 monarchE study included 5637 patients with HR+ HER2- early breast cancer with clinical and/or pathological risk factors putting them at high risk for relapse. After completing their primary treatment they were randomised on an open-label basis to abemaciclib (150mg twice daily for two years) plus endocrine therapy or endocrine therapy alone.

We found a 25% reduction in recurrence of cancer with the first two years when abemaciclib was added to hormone therapy compared to hormone therapy alone, reported Johnston. During this time 11.3% of patients in the control group had a relapse of their cancer compared to 7.8% of those in the abemaciclib group, an absolute difference of 3.5% which translates to a 25.3% reduction in risk. Most of the reductions occured in sites of distant metastases, especially to liver and bone.

This is the first study to show that adding a CDK4/6 inhibitor to endocrine therapy significantly improves invasive disease free survival in the adjuvant setting, said Giuseppe Curigliano, Associate Professor of Medical Oncology at the University of Milan, Italy, and Chair of the ESMO Guidelines Committee.

This is a very important trial and the findings will change practice. Once approved for high risk HR+ HER2- early breast cancer the new standard of care for these patients will be to add two years of abemaciclib to endocrine therapy, he suggested.

Curigliano suggested it would have been interesting to have included genetic signature into the assessment of patients at high risk, in addition to number of positive lymph nodes, tumour size, histologic grade and Ki-67 (a marker of proliferation). Johnston said that tissue and plasma samples had been collected from all of the study participants for translational research that will include looking at genomic signatures and response to abemaciclib.

The safety data are important, particularly the number of patients treated with abemaciclib who had to discontinue or required dose reductions due to side-effects, said Curigliano. A total of 463 (16.6%) of patients discontinued abemaciclib due to adverse events, most commonly diarrhea; 306 of these continued on endocrine therapy. The protocol allowed dose reduction from 150 to 100mg twice daily if required. He noted: Adherence to treatment will be an important issue to be considered in the real life population of patients when this treatment is approved and used in clinical practice.

Curigliano added, For the future it will be important to understand if we can potentially spare chemotherapy in this group of patients treated with a CDK4/6 inhibitor. This would need to be investigated in a randomised clinical trial.

Reference:Johnston SRD, Harbeck N, Hegg R, et al.Abemaciclib combined with endocrine therapy for the adjuvant treatment of HR+, HER2-, node-positive, high risk, early breast cancer (monarchE). 2020; doi:10.1200/JCO.20.02514

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Adding Abemaciclib to Hormonal Therapy Reduces Risk of Cancer Recurrence in Patients With High-Risk Early HR+ HER2- Early Breast Cancer - Technology...