20k for treatment to help teenager walk – Southend Standard

A 13-year-old boy who cannot walk is desperate to raise 20,000 for life-changing stem cell treatment in Panama.

St Thomas More pupil, Marcus McCarthy, from Southchurch, was diagnosed with cerebral palsy a neurological condition that impacts his limbs.

He has already undergone more than ten operations.

The family aims to ask 20,000 people to donate just 1 each.

Marcus mum, Scarlett, 34, said: Recently, I was researching other people with the condition who have had stem cell treatment - which is kind of like science magic.

I came across this clinic in Panama. Marcus and I talked about it, hes 13, he knows what hes going through. He said lets try Mum.

I contacted the clinic and they assessed Marcus based on the information I gave and they said they would be happy to accept him for treatment.

But its not funded, its a private clinic in Panama.

When it comes to your own childs health, money cant be an object.

The Stem Cell Institute in Panama provides therapies using specially screened and processed mesenchymal stem cells sourced from umbilical cord tissue.

Scarlett, added: He is a very strong willed boy, he enjoys his physiotherapy and likes swimming and boxing - hes a very psychical boy.

He just started his GCSEs and his top choice was GCSE P.E. - his P.E. said yeah go for it!

That just shows his sense of character.

Hes like, thats not going to stop me.

But it doesnt mean that that strong-will doesnt come with a lot of frustration.

He wants to get rid of this.

It doesnt make him who he is though. His confidence, strength and his humour is what makes him.

He wants to have a normal functioning life.

Visit, justgiving.com/campaign/NextMountain4Marcus?

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20k for treatment to help teenager walk - Southend Standard

Stem Cell Therapy Market 2020 by Top Key Players, Product Type, End-user Applications, Region and Forecast to 2025 – The Daily Chronicle

This minutely analyzed and systematically articulated business intelligence report based on global Stem Cell Therapy market is poised to enable seamless navigation to all crucial market participants and other report readers and stakeholders interested to carving a new growth route to offset multiple challenges in global Stem Cell Therapy market. Besides dedicating a significant crux of the report in re-imaging and re-assessing conventional marketing strategies, the report is designed to push the boundaries in unravelling new and improved damage control practices to align with growth needs and manufacturer preferences.

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Global Stem Cell Therapy market research report presentation demonstrates and presents an easily understandable market depiction, lending crucial insights on market size, market share as well as latest market developments and notable trends that collectively harness growth in the global Stem Cell Therapy market.

Scope of the Report This aforesaid Stem Cell Therapy market has noted a growth estimate of xx million US dollars in 2020 and is also likely to show favorable growth worth xx million US dollars through the forecast tenancy until 2025, clocking at an robust CAGR of xx% through the forecast period, 2020-25.

The report has included significant details about various facets covering manufacturer activities to offset the challenges prevalent during COVID-19.

The report in the following sections, emphasizes details on various market players, stakeholders, and participants. Details on upstream and downstream developments, production and consumption patterns are also addressed in the report to influence holistic and balanced growth in the global Stem Cell Therapy market.

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Stem Cell Therapy Market Segmentation Type Analysis of Stem Cell Therapy Market:

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

Adipose Tissue-Derived Mesenchymal SCs Bone Marrow-Derived Mesenchymal SCs Embryonic SCs Other Sources

Applications Analysis of Stem Cell Therapy Market:

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

Musculoskeletal Disorders Wounds & Injuries Cardiovascular Diseases Gastrointestinal Diseases Immune System Diseases Other Applications

Regional Analysis: Global Stem Cell Therapy Market Segment Assessment: Global Stem Cell Therapy Market 1. The report in its subsequent sections critically examines the crucial possibilities teeming in the global Stem Cell Therapy market and stresses on the tangibility of segments in enticing a favorable growth journey. 2. Besides segregating the market in accordance with broad categories and segments, the report makes accurate deductions in isolating the segment responsible for steady and balance growth trail. 3. With such decisive information outlined in the report, report readers can well assess and propagate competent growth strategies to ensure healthy investment returns. 4. Further, details on regional as well as country-wise developments are also portrayed in the report with specific references also of country wise developments that tend to push million dollar growth opportunities. 5. The report also further answers key questions about the raw material and manufacturing equipment incorporated by leading players and the manufacturing process incorporation

Vendor Profiling: Global Stem Cell Therapy Market 1. The report in its subsequent sections unfurls crucial inputs pertaining to the chief competitors in the Stem Cell Therapy market. Additional information related to other contributors and notable key players and contributors with reference to local and regional dominance also find requisite mention in the report. 2. Each of the players mentioned in the report have been thorough scrutinized on the basis of their company and product portfolios to make logical deductions related to strategic planning and winning business decisions. 3. All the listed players mentioned in the report has been subject to thorough assessment and evaluation process to unfurl valuable insights on company profiles, their positioning in the competitive graph, portfolio diversifications as well as notable business moves that have proved success and steady revenue generation.

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Stem Cell Therapy Market 2020 by Top Key Players, Product Type, End-user Applications, Region and Forecast to 2025 - The Daily Chronicle

GMP Cell Banking Services Market | Government Bodies Increase Flow of Funding to Discover Treatment Options for Rare Diseases – BioSpace

The global GMP cell banking services market is expected to gain stupendous demand avenues in the forthcoming years. This growth is attributed to increasing demand for GMP Cell banking services from the enterprises engaged in the pharmaceutical and stem cell research industries. Cell banks use a conventional technique named cryopreservation to keep the cells materials preserved. At the same time, cell banks also prevent the natural cell division process; thereby improve the shelf life of products preserved in the cell banks.

One of the important factors owing to which GMP cell banking services are gaining momentum among scientists and research institutes is the cost-effectiveness of these services. In recent years, there is remarkable growth in the use of GMP cell banking services among gamut of research institutes from all across the world. This scenario depicts that the vendors working in the global GMP cell banking services market will witness stupendous demand opportunities in the forthcoming period.

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GMP Cell Banking Services Gain Momentum among Stem Cell Research Institutes Due to Cost-effectiveness and Quality They Offer

In recent years, there is noteworthy increase in the number of people living with various rare diseases. This situation has compelled scientists working in all worldwide locations to focus on the discovery of novel options to treat these health issues. To accelerate these research activities, government bodies of many countries from all over the world are financially supporting these research projects. This factor is positively impacting on the development of the global GMP cell banking services market.

A case in point is funding of research on rare diseases by Food and Drug Administration (FDA), the US. FDA makes use of programs mandated by congress, such as Orphan Products Grants Program, which funds studies of natural history and for conducting clinical trials for rare medical conditions.In addition to that, establishment of several resource centers for stem cell banking is estimated to positively influence the global GMP cell banking services market. For instance, Kuala Lampur based cord blood bank, Stemlife Berhad started resource center for stem banking in Brunei-based private hospital, Jerudong Park Medical Center.

North America is regarded as one of the most promising regions due to the presence of substantial mammalian cell. This type of cells is estimated to generate most of the revenue in the North America market.Asia Pacific is another important lucrative region in the market with mammalian cell type accounting for most of the revenue in the region. A rise in the research and development on rare diseases together with increased funding for the same is estimated to drive the market in Asia Pacific.

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On regional front, players working in the GMP cell banking services market are projected to gain fantastic development opportunities in North America and Asia Pacific. Presence of substantial mammalian cell is said to be one of the key reasons driving the growth of GMP cell banking services market in North America.

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Competitive Analysis:

Charles River Laboratories International, Inc., Paragon Bioservices, Inc., Goodwin Biotechnology Inc., Lonza Group Ltd., WuXi AppTec Group, Eurofins Scientific

About TMR Research

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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GMP Cell Banking Services Market | Government Bodies Increase Flow of Funding to Discover Treatment Options for Rare Diseases - BioSpace

The Canine Stem Cell Therapy Market To Go Strong With 4.2% CAGR Between 2026 – Crypto Daily

New York City, United States The change during the COVID-19 pandemic has overhauled our dependence on pattern setting developments, for instance, expanded reality, computer generated reality, and the Healthcare web of things. The unfulfilled cash related targets are persuading the relationship to grasp robotization and forefront advancements to stay ahead in the market competition. Associations are utilizing this open entryway by recognizing step by step operational needs and showing robotization in it to make an automated structure as far as might be feasible.

Persistence Market Research (PMR) has published a new research report on canine stem cell therapy. The report has been titled, Canine Stem Cell Therapy Market: Global Industry Analysis 2016 and Forecast 20172026.Veterinary research has been used in regenerative and adult stem cell therapy andhas gained significant traction over the last decade.

Canine stem cell therapy products are identified to have gained prominence over the past five years, and according to the aforementioned research report, the market for canine stem cell therapy will expand at a moderate pace over the next few years.

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Company Profiles

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Though all animal stem cells are not approved by FDA, veterinary stem-cell manufacturers and university researchers have been adopting various strategies in order to meet regulatory approvals, and streamline and expedite the review-and-approval process. The vendors in the market are incessantly concentrating on research and development to come up with advanced therapy, in addition to acquiring patents.

In September 2017, VetStem Biopharma, Inc. received European patent granted to the University of Pittsburgh and VetStem received full license of the patent then. This patent will eventually provide the coverage for the ongoing commercial and product development programs of VetStem and might be also available for licensing to other companies who are rather interested in this field.

The other companies operating in the global market for canine stem cell therapy are VETherapy Corporation, Aratana Therapeutics, Inc., Regeneus Ltd, Magellan Stem Cells, Animal Cell Therapies, Inc., and Medrego, among others.

According to the Persistence Market Research report, the globalcanine stem cell therapy marketis expected to witness a CAGR of 4.2% during the forecast period 2017-2026. In 2017, the market was valued at US$ 151.4 Mn and is expected to rise to a valuation of US$ 218.2 Mn by the end of 2026.

Burgeoning Prevalence of Chronic Diseases in Dogs to Benefit Market

Adipose Stem Cells (ASCs) are the most prevalent and in-demand adult stem cells owing to their safety profile, ease of harvest, and use and the ability to distinguish into multiple cell lineages. Most early clinical research is focused on adipose stem cells to treat various chronic diseases such as arthritis, tendonitis, lameness, and atopic dermatitis in dogs.

A large area of focus in veterinary medicine is treatment of osteoarthritis in dogs, which becomes more prevalent with age. Globally, more than 20% dogs are suffering from arthritis, which is a common form of canine joint and musculoskeletal disease. Out of those 20%, merely 5% seem to receive the treatment.

However, elbow dysplasia in canine registered a prevalence rate of 64%, converting it into an alarming disease condition to be treated on priority. Thereby, with the growing chronic disorders in canine, the demand for stem cell therapy is increasing at a significant pace.

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Expensive Nature of Therapy to Obstruct Growth Trajectory

Expensive nature and limited access to canine stem cell therapy has demonstrated to be a chief hindrance forestalling its widespread adoption. The average tier II and tier III veterinary hospitals lack the facilities and expertise to perform stem cell procedures, which necessitates the referral to a specialty vet hospital with expertise veterinarians.

A trained veterinary physician charges high treatment cost associated with stem cell therapy for dogs. Generally, dog owners have pet insurance that typically covers maximum cost associated with steam cell therapy to treat the initial injury but for the succeeding measures in case of retreatment, the costs are not covered under the pet insurance. The stem cell therapy is thus cost-prohibitive for a large number of pet owners, which highlights a major restraint to the market growth. Stem cell therapy is still in its developmental stage and a positive growth outcome for the market cannot be confirmed yet.

About- Persistence Market Research (Healthcare)

Unprecedented access to a world of information has given rise to the empowered, albeit distrustful, consumer. So is the case with healthcare, where the patient has been rendered more informed and conscientious thanks to the extraordinary penetration of the Internet. The discerning patient now demands more affordable, sophisticated, transparent, and personalized healthcare services, creating the need for new models for care.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes

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The Canine Stem Cell Therapy Market To Go Strong With 4.2% CAGR Between 2026 - Crypto Daily

Global Cord Blood Banking Market Is Expected to Reach USD 25.90 billion by 2027 : Fior Markets – GlobeNewswire

September 29, 2020 04:00 ET | Source: Fior Markets

Newark, NJ, Sept. 29, 2020 (GLOBE NEWSWIRE) -- As per the report published by Fior Markets, theglobal cord blood banking market is expected to grow from USD 16.88 billion in 2019 and to reach USD 25.90 billion by 2027, growing at a CAGR of 5.5% during the forecast period 2020-2027.

The global cord blood banking market is witnessing significant growth from recent years. This growth is attributed to the rising awareness about therapeutic applications of cord blood, government initiatives and funding in developed economies, increasing prevalence of life-threatening genetic diseases, and growing clinical trials in regenerative medicines. A growing number of investors and genetic disorders will propel the market for cord blood banking.

The blood that stays in the placenta and umbilical cord post-delivery is known as cord blood. A process of collecting the cord blood and cryogenically freezing and extracting the stem cells and other cells of the immune system for the potential future medical use is known as cord blood banking. It consists of a rich source of stem cells that can treat various genetic diseases. A very small amount of cord blood can be collected from a single umbilical cord. These umbilical cord contain a large number of stem cells and is only the type of stem cells stored for future use. The medical use of stem cells and immune system cells in cord blood is expanding rapidly. The cord blood banking process includes the collection process through storage and newborn stem cells for future medical purposes.

The global cord blood banking market is expected to witness significant growth, owing to the increasing number of facilities, increasing use of cord blood in the treatment of chronic diseases, and rising prevalence of chronic diseases. The factors restraining the market growth are lack of uniformity in regulatory policies, large out-of-pocket expenditure, and low acceptance rate in developing countries. Increasing research concerns about cord blood application for the treatment of genetic and idiopathic disorders will provide market growth opportunities.

DOWNLOAD FREE SAMPLE REPORT AThttps://www.fiormarkets.com/report-detail/418891/request-sample Key players operating in global cord blood banking market are CBR Systems, Singapore Cord Blood Bank, Cryo-Save, ViaCord LLC, Global Cord Blood Corporation, Jeevan Stem Cell Foundation, LifeCell, Cordlife, Americord, and FamiCord. To gain a significant market share in the global cord blood banking market, the key players are now focusing on adopting strategies such as product innovations, mergers & acquisitions, recent developments, joint venture, collaborations, and partnership. FamiCord and Cryo-Save are the major players operating in cord blood banking.

Public cord blood bank segment dominated the market and held the largest market share of 58.2% in the year 2019 On the basis of type of bank, the global cord blood banking market is segmented into public and private. Public cord blood bank segment dominated the market and held the largest market share of 58.2% in the year 2019. This growth is attributed to various funding initiatives, and various government initiatives for encouraging expectant mothers to donate cord blood cells in public banks. The private cord blood banks are expected to witness significant growth, owing to the growing awareness among expectant parents and high demand to safeguard the health of family members.

Storage segment dominated the market and held the largest market share of 51.26% in the year 2019 On the basis of services, the global cord blood banking market is segmented into processing and storage. Storage segment dominated the market and held the largest market share of 51.26% in the year 2019. This growth is attributed to the increasing number of expectant parents utilizing the preservation of newborn cord blood cells. The processing segment is expected to witness significant growth due to various technological advancements in cord blood processing methods.

Blood disorders segment dominated the market and held the largest market share of 26.7% in the year 2019 On the basis of application, the global cord blood banking market is segmented into cancer, blood disorders, immuno-deficiency disorders, metabolic disorders, and bone marrow failure syndrome. Blood disorders segment dominated the market and held the largest market share of 26.7% in the year 2019. This growth is attributed to the increasing application of cord blood cells in the treatment of blood disorders and the increasing prevalence of beta-thalassemia. Immuno-deficiency disorders are expected to witness significant growth, owing to the rapid and reliable recovery of immune function, low viral transmission rate, and low risk of graft versus host diseases.

Research institute segment dominated the market and held the largest share of 41.5% in the year 2019 On the basis of end-user, the global cord blood banking market is segmented into hospitals, research institutes, and specialty clinics. Research Institute segment dominated the market and held the largest share of 41.5% in the year 2019. This growth is attributed to the growing pervasiveness of genetic disorders and growth in the number of clinical trials for exploring new diseases. Hospitals segment is expected to witness significant growth, owing to the advancement in cord blood banking, increasing prevalence of genetic diseases.

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Regional Segment of Cord Blood Banking Market

On the basis of geography, the global cord blood banking market is classified into North America, Europe, Asia-Pacific, Middle East & Africa, and South America. North America held the major share of 25.8% in the year 2019. This growth is attributed to the presence of a large number of genetic disorders, continuous efforts by the researchers to explore new therapeutic applications of cord blood cells. The Asia-Pacific region market is anticipated to witness significant growth, owing to the growth potential in emerging economies like China and India due to the higher birth rate.

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Global Cord Blood Banking Market Is Expected to Reach USD 25.90 billion by 2027 : Fior Markets - GlobeNewswire

Developments In Stem Cell Therapy Market Top Regions, Countries, Sales, Revenue, Leading Companies, Types, Demand, And Forecast Assessment – The Daily…

"The niche and established Stem Cell Therapy Market 2020 Covering Prime Players, Regions, Countries, And Growth Trends Forecast To 2026 is studied in this research report. The Industry dynamics with Stem Cell Therapy future-proofing business plans are analyzed to offer sustainable growth for our clients. The insights-driven data covering the Stem Cell Therapy competition, product demand across different operating regions, countries, and a variety of applications are covered. Actionable insights offered by Reports Check will help achieve you in achieving exemplary growth with feasible investments. To strengthen the position in the global and domestic Stem Cell Therapy Market revenue, size & share analysis, import-export details, production capacity, and utilization ratio is offered.

Stem Cell Therapy Market is expected to reach XX.XX Mn USD by 2026 and it will capture lucrative opportunities with rising demand. The market dynamics, SWOT analysis, Porters Analysis, the risk assessment will create discerning growth and profit-driven strategies. Reports Checks analyst team is fine-tuned to answer all client queries and well as to deliver custom solutions based on their requirements.

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Study Period: 2015-2026

Base Year: 2019

Estimated Year With COVID-19 Impact Analysis:2020

Forecast Period Duration:2020-2026

Historical Period Duration:2015-2018

Important and Top Product Types:

Autologous Allogeneic

By Various Applications:

Musculoskeletal Disorder Wounds & Injuries Cornea Cardiovascular Diseases Others

By Top Companies On Global, Regional And Country Level:

Osiris Therapeutics NuVasive Chiesi Pharmaceuticals JCRPharmaceutical Pharmicell Medi-post Anterogen Molmed Takeda (TiGenix)

By Geographical Stem Cell Therapy Industry Presence:

The current Stem Cell Therapy Market landscape, detailed insights, and market drivers with restraints and trends are studied in this report. The detailed market performance during the forecast period is presented in this report. The Y-o-Y growth and CAGR is presented in this report to support the readers in making appropriate business plans. The quantitative development opportunities and innovations in Stem Cell Therapy Industry are presented.

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All details including Stem Cell Therapy market investors, manufacturers, service providers, distributors, stakeholders are provided. This research study by Reports Check helps the industry experts, researchers, investors, and business aspirants. The macro and microeconomic factors with substantive Stem Cell Therapy insights are analyzed in this study.

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Development trends, competitive analysis, and growth dynamics are key aspects of this report

Past, present, and forecast Stem Cell Therapy industry evaluation with complete performance monitoring of top players,.

Each product type and application regional and country-level bifurcation offer comprehensive and niche Stem Cell Therapy analysis

The product portfolio, Stem Cell Therapy company profiles, SWOT, and Porters Five Forces analysis offers quality inputs

Pre and Post pandemic situations, ways to overcome pandemic, Stem Cell Therapy developments across different macro and micro-segments are analyzed

Market size, product pricing analysis, Stem Cell Therapy cost structures, raw material, and downstream buyers analysis is conducted

In-depth primary and secondary techniques applied by Reports Check provide quality, reliability, thorough analysis, and competitive market intelligence

Forecast regional, country-level dynamics, expected growth rate, revenue generation, upcoming segments, and Stem Cell Therapy vendors, R&D status is specified

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Developments In Stem Cell Therapy Market Top Regions, Countries, Sales, Revenue, Leading Companies, Types, Demand, And Forecast Assessment - The Daily...

Global Stem Cell Therapy for Osteoarthritis Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts Research – The…

The global Stem Cell Therapy for Osteoarthritis market report by Dataintelo provides a detailed analysis of the area marketplace expanding; competitive landscape; global, regional, and country-level market size; market growth analysis; market share; opportunities analysis; product launches; recent developments; sales analysis; segmentation growth; technological innovations; and value chain optimization. The report offers a comprehensive list of key players, their strategies they adopt to sustain in the market. All of this and more information is covered in XXX pages.

The Coronavirus (COVID-19) pandemic has affected every aspect of life worldwide. It has forced various industries to re-evaluate their strategies and adopt new ones to sustain during these trying times. The latest report includes the current COVID-19 impact on the market.

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The Global Stem Cell Therapy for Osteoarthritis Market report is a comprehensive analysis of the current and future analysis, which is based on historic data. This provides the reader with quantified data, enabling them to take well informed business decisions. The report has been written using primary and secondary research. It includes predictive analysis, Porters 5 force analysis, SWOT analysis, and real-time analytics. Several graphs have been provided to support the data and for a clear understanding of various facts and figures.

The report has been divided into product types, application, end-users, and regions. These segments provide accurate calculations and forecasts for sales in terms of volume and value. This analysis can help customers increase their business and to take calculated decisions.

The Global Stem Cell Therapy for Osteoarthritis Market Report has been Segments into:

Global Stem Cell Therapy for Osteoarthritis Market Size & Share, By Product Types,

Monotherapy Combination Therapy

Global Stem Cell Therapy for Osteoarthritis Market Size & Share, By Applications,

Osteoarthritis (unspecified) Knee Osteoarthritis Shoulder Osteoarthritis Hip Osteoarthritis

Global Stem Cell Therapy for Osteoarthritis Market Size & Share, By Regions and Countries/Sub-regions,

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The regional analysis segment is a highly comprehensive part of the report on the global Stem Cell Therapy for Osteoarthritis market. This section offers information on the sales growth in these regions on a country-level Stem Cell Therapy for Osteoarthritis market.

The historical and forecast information provided in the report span between 2018 and 2026. The report provides detailed volume analysis and region-wise market size analysis of the market.

The key players covered in this study

Mesoblast Regeneus U.S. Stem Cell Anterogen Asterias Biotherapeutics

Competitive Landscape of the Stem Cell Therapy for Osteoarthritis Market

The chapter on competitive landscape provides information about key company overview, global presence, sales and revenue generated, market share, prices, and strategies used.

Our analysts engage in extensive primary and secondary research to cull out in-depth and authentic information. Primary research includes gathering information from official government and company websites, journals, and reports. Secondary research includes interviews conducted with VPs, chairmen, directors, and other distinguished sales managers.

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Global Stem Cell Therapy for Osteoarthritis Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts Research - The...

Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I – BioSpace

BOSTON and LONDON, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

We are encouraged by EMAs decision to grant PRIME designation to OTL-203, which was based on an initial clinical assessment of data supporting the potential benefit of our HSC gene therapy for patients with MPS-IH beyond the current standard of care, said Anne Dupraz-Poiseau, PhD., chief regulatory officer of Orchard. In 2021, we look forward to building upon the promising early data in the ongoing proof-of-concept study and plan to initiate a registrational trial to advance a potential new treatment for patients.

The PRIME program is designed to enhance regulatory support in the EU for the development of promising investigational medicines that, based on early clinical data, may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. PRIME aims to provide multiple benefits so that these medicines can reach patients earlier: enhanced interaction and early dialogue with EMA, guidance on the overall development plan and regulatory strategy, and the potential for accelerated assessment of the marketing authorization application. For more information please visit the EMA website at http://www.ema.europa.eu.

Additional interim data was recently presented from the ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203 in the severe Hurler subtype of MPS-I. Eight patients have been treated in the study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Orchard expects to release full proof-of-concept results at one year and initiate a registrational study for OTL-203 in 2021.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated. Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidate referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidate referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidate referred to in this release, will not be successfully developed, approved or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates or that long-term adverse safety findings may be discovered; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended June 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Molly Cameron Manager, Corporate Communications +1 978-339-3378 media@orchard-tx.com

Original post:
Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I - BioSpace

Stem Cell Therapy Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 – The Daily Chronicle

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