Global Gene Therapy Industry – GlobeNewswire

November 19, 2020 05:27 ET | Source: ReportLinker

New York, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Industry" - https://www.reportlinker.com/p05817594/?utm_source=GNW 6% in the year 2020 and thereafter recover and grow to reach US$3.3 billion by the year 2027, trailing a post COVID-19 CAGR of 19.5% over the analysis period 2020 through 2027. Governments worldwide are focusing all healthcare resources on fighting the global pandemic. Billions of dollars have poured into researching COVID-19 drugs, therapies and vaccines. Over US$8 billion globally excluding the U.S. has been pledged only for vaccine development. The U.S. has independently pumped billions of dollars into COVID-19 research and response. The massive reallocation of funds and reprioritization of efforts has left a glaring gap in other sectors of healthcare. Gene therapy which holds promise for treating cancer, cystic fibrosis, heart disease, diabetes, hemophilia & AIDS, is slumping due to lack of research funds & reduced footfall of patients seeking treatment. Given the complex and fragile manufacturing and delivery system along with funding models of the industry, COVID-19 has emerged as a black swan event. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites. There are concerns regarding administration of cell and gene therapies. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centres. Treatments intended to be delivered into ICUs are being impacted by bed reservations made for patients with COVID-19 infection.

R&D and preclinical activities are also affected by supply shortages as a result of strong demand for consumables like reagents and PPE from COVID-19 laboratories. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrolments for protecting participants from the risk of infection. These issues are delaying activation of new sites, prompting players to postpone new clinical trials. However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. While companies targeting paediatric diseases suspended trials, others dealing with oncology maintained the pace. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. These issues are attributed to confluence of various factors like travel ban, withdrawal of several services from healthcare sites and the risk of virus transmission. In addition, these disruptions are anticipated to threaten existence of certain cell and gene therapy companies, particularly small-scale biotech players that are in pre-commercial phase and rely on external funding. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.

In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Gene therapies hold promise to improve the condition of patients where traditional cancer treatments such as radiation and chemotherapy are not effective. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. Oncology will remain the key area of focus for gene therapy applications. Cancer therapies represent the leading category, as is gauged through robust rise in the number of molecules being tested across numerous clinical trials. Novartis which recently bagged the U.S. FDA approval for Kymriah, a gene therapy designed for the treatment of hematological cancer, is seeking to gain commercial approval in established and emerging countries. Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals. More developmental focus will also be shed on monogenic rare diseases which have clearer genomic targets and the unmet need in smaller patient populations. Majority gene therapies so far have come to market through accelerated review pathways of regulatory authorities. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed. In the coming years, there will be significant improvement in the number of approvals for new gene therapies. The growth is anticipated to emerge from different modalities including RNAi, ASOs and CRISPR gene editing based therapeutics which offer long term opportunities for growth. These technologies are generating much excitement for investors.

Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=GNW

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE I-1

II. EXECUTIVE SUMMARY II-1

1. MARKET OVERVIEW II-1 A Prelude to Gene Therapy II-1 Classification of Gene Therapies II-1 Impact of Covid-19 and a Looming Global Recession II-2 COVID-19 Causes Gene Therapy Market to Buckle & Collapse II-2 COVID-19 Impact on Different Aspects of Gene Therapy II-2 Manufacturing & Delivery II-2 Research & Clinical Development II-3 Commercial Operations & Access II-3 Managing Derailed Operations II-4 Focus on Clinical Development Programs II-4 Targeting Manufacturing & Delivery Strategies II-4 Securing Supplies II-4 Remote Working II-4 Gene Therapy Set to Witness Rapid Growth Post COVID-19 II-5 By Vector Type II-5 VIRAL VECTORS ACCOUNT FOR A MAJOR SHARE OF THE MARKET II-5 Adeno-Associated Virus Vectors II-6 Lentivirus II-6 NON-VIRAL VECTORS TO WITNESS FASTER GROWTH II-7 US and Europe Dominate the Gene Therapy Market II-8 Oncology Represents the Largest Indication for Gene Therapy II-9 Market Outlook II-9 WORLD BRANDS II-10

2. FOCUS ON SELECT PLAYERS II-16 Recent Market Activity II-18 Select Innovations II-24

3. MARKET TRENDS & DRIVERS II-25 Availability of Novel Therapies Drive Market Growth II-25 Select Approved Gene Therapy Products II-26 Adeno-associated Virus Vectors - A Leading Platform for Gene Therapy II-27 Lentiviral Vectors Witness Increased Interest II-27 Rising Cancer Incidence Worldwide Spurs Demand for Gene Therapy II-28 Exhibit 1: Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 II-28 Exhibit 2: Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 II-29 Exhibit 3: Number of New Cancer Cases and Deaths (in Million) by Region for 2018 II-30 Compelling Level of Technology & Innovation to Ignite Gene Therapy II-30 Promising Gene Therapy Innovations for Treatment of Inherited Retinal Diseases II-31 Gene Therapy Pivots M&A Activity in Dynamic Domain of Genomic Medicine II-31 M&As Rampant in Gene Therapy Space II-31 Gene Therapy Deals: 2018 and 2019 II-32 Emphasis on Formulating Robust Regulatory Framework II-33 Strong Gene Therapy Pipeline II-33 Gene Therapy: Phase III Clinical Trials II-33 OHSU Implements First-Ever LCA10 Gene Therapy Clinical Trial with CRISPR II-35 Growing Funding for Gene Therapy Research II-35 Market Issues & Challenges II-35

4. GLOBAL MARKET PERSPECTIVE II-37 Table 1: World Current & Future Analysis for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-37

Table 2: World Historic Review for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-38

Table 3: World 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2017, 2020 & 2027 II-39

Table 4: World Current & Future Analysis for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-40

Table 5: World Historic Review for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-41

Table 6: World 10-Year Perspective for Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-42

Table 7: World Current & Future Analysis for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-43

Table 8: World Historic Review for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-44

Table 9: World 10-Year Perspective for Non-Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-45

Table 10: World Current & Future Analysis for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-46

Table 11: World Historic Review for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-47

Table 12: World 10-Year Perspective for Oncological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-48

Table 13: World Current & Future Analysis for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-49

Table 14: World Historic Review for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-50

Table 15: World 10-Year Perspective for Rare Diseases by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-51

Table 16: World Current & Future Analysis for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-52

Table 17: World Historic Review for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-53

Table 18: World 10-Year Perspective for Neurological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-54

Table 19: World Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-55

Table 20: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-56

Table 21: World 10-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-57

III. MARKET ANALYSIS III-1

GEOGRAPHIC MARKET ANALYSIS III-1

UNITED STATES III-1 Table 22: USA Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-1

Table 23: USA Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-2

Table 24: USA 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-3

Table 25: USA Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-4

Table 26: USA Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-5

Table 27: USA 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-6

CANADA III-7 Table 28: Canada Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-7

Table 29: Canada Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-8

Table 30: Canada 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-9

Table 31: Canada Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-10

Table 32: Canada Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-11

Table 33: Canada 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-12

JAPAN III-13 Table 34: Japan Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-13

Table 35: Japan Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-14

Table 36: Japan 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-15

Table 37: Japan Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-16

Table 38: Japan Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-17

Table 39: Japan 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-18

CHINA III-19 Table 40: China Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-19

Table 41: China Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-20

Table 42: China 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-21

Table 43: China Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-22

Table 44: China Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-23

Table 45: China 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-24

EUROPE III-25 Table 46: Europe Current & Future Analysis for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 III-25

Table 47: Europe Historic Review for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-26

Table 48: Europe 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2017, 2020 & 2027 III-27

Table 49: Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-28

Table 50: Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-29

Table 51: Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-30

Table 52: Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-31

Table 53: Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-32

Table 54: Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-33

FRANCE III-34 Table 55: France Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-34

Table 56: France Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-35

Table 57: France 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-36

Table 58: France Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-37

Table 59: France Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-38

Table 60: France 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-39

GERMANY III-40 Table 61: Germany Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-40

Table 62: Germany Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-41

Table 63: Germany 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-42

Table 64: Germany Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-43

Table 65: Germany Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-44

Table 66: Germany 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-45

ITALY III-46 Table 67: Italy Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-46

Table 68: Italy Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-47

Table 69: Italy 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-48

Table 70: Italy Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-49

Table 71: Italy Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-50

Table 72: Italy 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-51

UNITED KINGDOM III-52 Table 73: UK Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-52

Table 74: UK Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-53

Table 75: UK 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-54

Table 76: UK Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-55

Table 77: UK Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-56

Table 78: UK 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-57

REST OF EUROPE III-58 Table 79: Rest of Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-58

Table 80: Rest of Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-59

Table 81: Rest of Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-60

Table 82: Rest of Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-61

Table 83: Rest of Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-62

Table 84: Rest of Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-63

ASIA-PACIFIC III-64 Table 85: Asia-Pacific Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-64

Table 86: Asia-Pacific Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-65

Table 87: Asia-Pacific 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-66

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Global Gene Therapy Industry - GlobeNewswire

Children’s Hospital delivers life-saving gene replacement therapy to infant – Loma Linda University Health

Loma Linda University Childrens Hospital recently gave its first administration of a new gene replacement therapy to an infant who screened positive for spinal muscular atrophy (SMA), the top genetic killer of children under the age of two, potentially providing a symptom-free life for the patient.

Childrens Hospital physicians said the new drug therapy, Zolgensma, is a revolution in neurological pediatric care.

This therapy represents a new opportunity for us to intervene and change childrens long-term prospects, and sometimes if we can treat the disease in time, we can restore them to full health, said David Michelson, MD, chief of the division of child neurology and director of the pediatric muscular dystrophy association clinics at Childrens Hospital. This therapy is a chance to wipe a patients health slate clean.

The patient was diagnosed with SMA through the state of Californias newborn screening program. Even though the patient seemed perfectly healthy during the first weeks of life, the healthcare team was working against the clock to treat the patient before the first potential onset of symptoms. Studies have shown that infants who receive the therapy as soon after diagnosis as possible have better outcomes than those who receive delayed treatment.

SMA is a rare genetic disease that causes a persons muscles to weaken and become smaller over time. In an infant, this affects development, including crawling, walking, sitting and head control. Severe types of SMA affect the muscles used for swallowing and breathing and, untreated, lead to ventilator dependence and a high risk of mortality.

According to Michelson, the patients neurologist, there are four types of SMA. These types differ in both their severity and at what age they will manifest in a persons body. SMA type I, the most common of the severe forms, can present in infants as early as three months and is often fatal by the age of two.

The new therapy involves one of only three approved drugs to treat SMA in children under two and is the only single-dose treatment option for SMA patients. Hospitals across the country are only beginning to be able to provide this therapy to qualifying patients since the therapy received FDA approval in May 2019.

Michelson said he and his team dont yet know exactly how the treatment will affect the patient.

We may not know how much we helped since we were able to treat him before he developed symptoms, Michelson said. Best case scenario is he will never develop any symptoms of the disease.

Read more:
Children's Hospital delivers life-saving gene replacement therapy to infant - Loma Linda University Health

Global Precision Cancer Therapies Market 2020: Analysis, Industry Growth, Current Trends and Forecast till 2025 – The Daily Philadelphian

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Global Precision Cancer Therapies Market 2020: Analysis, Industry Growth, Current Trends and Forecast till 2025 - The Daily Philadelphian

Cutting-edge Treatment Offers Hope to Infant With Spinal Muscular Atrophy – Adventist Review

November 17, 2020

By Sheann Brandon, Loma Linda University Health

Loma Linda University Childrens Hospital in Loma Linda, California, United States, recently gave its first administration of a new gene replacement therapy to an infant who screened positive for spinal muscular atrophy(SMA). SMA is the top genetic killer of children under the age of two, and the treatment potentially provides a symptom-free life for the patient.

Childrens Hospital physicians said the new drug therapy Zolgensma is a revolution in neurological pediatric care.

This therapy represents a new opportunity for us to intervene and change childrens long-term prospects, and sometimes if we can treat the disease in time, we can restore them to full health, David Michelson, chief of the division of child neurologyand director of the pediatric muscular dystrophy association clinics at Childrens Hospital, said. This therapy is a chance to wipe a patients health slate clean.

The patient was diagnosed with SMA through the state of Californias newborn screening program. Even though the patient seemed perfectly healthy during the first weeks of life, the health-care team worked against the clock to treat the patient before the first onset of symptoms. Studies have shown that infants who receive the therapy as soon after diagnosis as possible have better outcomes than those who receive delayed treatment.

SMA is a rare genetic disease that causes a persons muscles to weaken and become smaller over time. In an infant, this affects development, including crawling, walking, sitting, and head control. Severe types of SMA affect the muscles used for swallowing and breathing and, untreated, lead to ventilator dependence and a high risk of mortality.

According to Michelson, the patients neurologist, there are four types of SMA. These types differ in their severity and at what age they will manifest in a persons body. SMA type I, the most common of the severe forms, can present in infants as early as three months and is often fatal by the age of two.

The new therapy involves one of only three approved drugs to treat SMA in children under two and is the only single-dose treatment option for SMA patients. Hospitals across the country are only beginning to provide this therapy to qualifying patients since the treatment received U.S. Food and Drug Administration (FDA) approval in May 2019.

Michelson said he and his team dont yet know exactly how the treatment will affect the patient.

We may not know how much we helped, since we were able to treat him before he developed symptoms, Michelson said. Best case scenario is he will never develop any symptoms of the disease.

The original version of this story was posted on the Loma Linda University Health news site.

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Cutting-edge Treatment Offers Hope to Infant With Spinal Muscular Atrophy - Adventist Review

Molecular Testing With Universal Method Comparable to Traditional Method in Hereditary Solid Tumors – Targeted Oncology

In multiple solid malignancies, including breast, ovarian, colorectal, and pancreatic cancers, there is a subset of patients with a hereditary predisposition for these diseases, but the current testing criteria do not mention this subset. In an effort to provide more information of genetic testing in this population, a study of Memorial Sloan Kettering Cancer Center (MSKCC) patients was conducted to test the traditional guideline-based method of testing versus universal testing of a broad cancer patient population over a 5-year period.

A total of 7235 patients were included in the analysis and tested for 76 to 88 cancers. Through this study, investigators uncovered pathogenic and likely pathogenic (P/LP) variants in 7.5% (95% CI 6.6%- 8.4%) of 3,341 patients with breast cancer, 17.4% (95% CI, 14%- 21.6%) of 384 those with ovarian cancer, 13.5% (95% CI, 9.8%- 18%) of 252 patients with colorectal, and 8.8% (95% CI, 5.1%-14.8%) of 136 patients with pancreatic cancer.

Overall, the study found that testing with universal method was comparable to the guideline-based method, implying that universal testing can expand genetic testing to patient populations who are in need but are currently underserved.

In an interview with Targeted Oncology during the 2020 Association for Molecular Pathology (AMP) Annual Meeting, Ozge Ceyhan-Birsoy, PhD, assistant directorof the Laboratoryfor Molecular Medicine, MSKCC, discussed genetic testing methods for patients with hereditary predisposition and the molecular research underway at MSKCC to improve testing in this patient population.

TARGTED ONCOLOGY: In recent years, what advances have we see in cancer genetics?

Ceyhan-Birsoy: There have been significant advances in the range of genetic testing options for cancer patients in recent years. More patients are now able to receive molecular testing on their tumors to identify optimal targeted therapies for their cancer and germline genetic testing to uncover hereditary cancer predisposition. A paired analysis of tumor and normal DNA is increasingly being adapted, which improves the interpretation of both somatic and germline mutations. Additionally, incorporation of RNA analysis has expanded the scope of mutations that can be detected and characterized. Finally, the use of cell-free DNA now allows us to profile a patients tumor using only their blood.

TARGETED ONCOLOGY: How can hereditary predisposition inform oncologist for care/treatment decisions?

Ceyhan-Birsoy: Identifying hereditary mutations that predispose patients to cancer has important implications for their treatment and management. There are established targeted therapies available now for certain germline defects. For instance, germline mutations in certain homologous recombination and mismatch repair genes can predict response to PARP inhibitor and immune-checkpoint inhibitor therapies, respectively. Some therapies may pose high risk for patients with particular gene mutations, such as radiation therapy risks for patients with germline TP53 mutations. In addition, identifying hereditary cancer predisposition is critical to allow timely surveillance and prophylactic interventions for future cancers that the patient may be at higher risk of developing. As germline mutations are heritable, this information provides the opportunity for early surveillance in the patients family members, as well.

TARGETED ONCOLOGY: Can you explain how this MSKCC study came about?

Ceyhan-Birsoy: Genetic testing for hereditary cancer predisposition is traditionally performed in a guideline-dependent and targeted manner. In current practice, only patients who meet established criteria from national and professional organizations receive genetic testing and typically get tested for a small number of genes selected based on their tumor type, age of onset, and family histories. MSKCC has been 1 of the first institutes to pilot a universal testing approach for cancer patients, providing comprehensive germline testing of all known cancer predisposition genes without pre-selection of patients based on traditional genetic testing criteria. We have been performing both targeted and universal testing for our patients since 2015.

In this study, we aimed to understand how the yields (positive rates) of these 2 testing approaches compare to each other in greater than 4000 patients who had traditional and more than 9,000 patients who had universal testing at MSKCC in the past 5 years. We also assessed whether universal testing identified additional findings that would have been missed in a targeted testing approach for any given patient.

TARGETED ONCOLOGY: What are the key results of this analysis?

Ceyhan-Birsoy: We saw that universal germline testing without preselection of patients based on current guidelines yielded comparable rates of positive results to traditional guideline-dependent testing approach, particularly in patients with breast, ovarian, and pancreatic cancers. In addition, universal testing uncovered mutations that predispose to other cancers in about 9% of patients in genes that are not routinely tested for their diagnosis. Approximately half of those conferred high to moderate risk to cancer and about 40% of them implicated early surveillance or prophylactic surgery recommendations to prevent other cancers.

TARGETED ONCOLOGY: What is a key takeaway from your AMP 2020 presentation and explain the implications of these findings?

Ceyhan-Birsoy: Our results suggest that the preselection of patients for genetic testing based on the current guidelines may not significantly increase the likelihood of identifying a germline mutation in certain patient populations. A universal and comprehensive testing approach further provides the benefit of identifying hereditary risk for other cancers, allowing early surveillance and prophylactic interventions.

TARGETED ONCOLOGY: How can this information be applied in oncology clinics?

Ceyhan-Birsoy: Our study underlies the advantages of universal and comprehensive testing for cancer patients. However, there are many challenges that may limit the application of this approach for all cancer patients, including the cost of testing, resources needed to provide pre-test and post-test genetic counseling to patients, and the potential to discover more variants of uncertain significance that may lead to higher number of inconclusive results. Future efforts should be dedicated to providing wider groups of cancer patients access to genetic testing, which can aid in their clinical care and in the care of their family members.

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Molecular Testing With Universal Method Comparable to Traditional Method in Hereditary Solid Tumors - Targeted Oncology

Alpha Thalassemia Market Research Provides an In-Depth Analysis on the Future Growth Prospects and Industry Trends Adopted By the Competitors |…

Global Alpha Thalassemia Market 2025 Report Provides Porters Five Forces Analysis Illustrates the Potency of Buyers & Suppliers Operating in the Industry & the Quantitative Analysis of The Global Market from 2019 to 2025 is Provided to Determine the Market Potential.

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Chapter Two: Global Alpha Thalassemia Market Competition by Manufacturers

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Chapter Four: Global Alpha Thalassemia Consumption by Regions

Chapter Five: Global Alpha Thalassemia Production, Revenue, Price Trend by Type

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Chapter Eight: Alpha Thalassemia Manufacturing Cost Analysis

Chapter Nine: Marketing Channel, Distributors and Customers

Chapter Ten: Market Dynamics

Chapter Eleven: Global Alpha Thalassemia Market Forecast

Chapter Twelve: Research Findings and Conclusion

Chapter Thirteen: Methodology and Data Source 13.1 Methodology/Research Approach 13.1.1 Research Programs/Design 13.1.2 Market Size Estimation 13.1.3 Market Breakdown and Data Triangulation 13.2 Data Source 13.2.1 Secondary Sources 13.2.2 Primary Sources 13.3 Author List 13.4 Disclaimer

About HongChun Research: HongChun Research main aim is to assist our clients in order to give a detailed perspective on the current market trends and build long-lasting connections with our clientele. Our studies are designed to provide solid quantitative facts combined with strategic industrial insights that are acquired from proprietary sources and an in-house model.

Contact Details: Jennifer Gray Manager Global Sales + 852 8170 0792 [emailprotected]

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Alpha Thalassemia Market Research Provides an In-Depth Analysis on the Future Growth Prospects and Industry Trends Adopted By the Competitors |...

Intrathecal Pumps Market 2020 Industry Size and Share Evolution to 2026 by Growth Insight, Trends – The Daily Philadelphian

The Global Intrathecal Pumps Market to gain from rapid technological advancements taking place in the market. Recently Fortune Business Insights has published a report, titled Intrathecal Pumps Market Size, Share and Global Trend By Medication (Baclofen, Hydromorphone Morphine, Clonidine, Midazolam, Bupivacaine, Ziconotide), By Application (Pain Management, Spasticity Management), By End Users (Hospitals & Clinics, Ambulatory Surgery Centers, Others), and Geography Forecast till 2026

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Intrathecal Pumps Market 2020 Industry Size and Share Evolution to 2026 by Growth Insight, Trends - The Daily Philadelphian

$30 billion to fight COVID-19 over next five years – Sydney Morning Herald

NSW will commit $29.6 billion to a COVID-19 health and economic package over the next five years following unprecedented emergency health spending to weather the pandemic.

Treasury forecasters are working on an assumption that a vaccine will be available by mid-next year, with an estimated 20 per cent of people vaccinated by October.

NSW will spend a total of $1.6 billion on COVID-19 programs in 2020-2021 including $500 million to support COVID clinics, quarantine programs, extra pathology testing and contact tracing.Credit:Getty/Lisa Maree Williams

Social distancing restrictions are likely to be maintained until a vaccine is widely available and most of the population immunised.

Up to $1.6 billion of the COVID-19 response budget will be used for 2020-2021 health programs, including $500 million to support clinics, quarantine programs, extra pathology testing and contact tracing.

More than $458 million will be used to fast track elective surgeries delayed by the pandemic and $20 million will accelerate COVID-specific research and clinical trials.

Some $385 million will be used to purchase additional personal protective equipment.

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Treasurer Dominic Perrottet described the mental health toll as one of the pandemics most insidious hidden costs.

More than $66 million will fund additional mental health clinicians and peer workers, expanding the Police, Ambulance and Clinical Early Response model, enhancing therapeutic activities in inpatient units, increasing capacity and responsiveness of the Mental Health line and expanding virtual mental health services

Overall health spending accounted for one third of the total NSW Budget, Health Minister Brad Hazzard said.

It included $66 million over three years beginning in 2021-22 to fund additional specialist mental health clinicians. Almost $56 million over four years will go to end of life and palliative care, including 5000 additional non-clinical End of Life Support, specialist allied health professionals, education and training, and bereavement and psychosocial support services.

Almost $50 million in 2020-2021 will fund access to lifesaving cell and gene therapies, including CAR T-cell therapy for children and young adults with acute lymphoblastic leukaemia, adults with diffuse large B-cell lymphoma, as well as gene therapy services for blinding eye disease and children with spinal muscular atrophy, and monoclonal antibody therapy for children with neuroblastoma.

A $17 million-pilot program will provide in-home care for pregnant women suffering from hyperemesis gravidarum (severe morning sickness), and Tresillian and Karitane will receive more than $10 million over four years to extend virtual residential parenting services.

There is $10 million over four years to plan and undertake a NSW stroke ambulance pilot project geared towards rapid diagnosis and treatment of strokes.

The governments hospital building boom over the past several years continues. Treasurer Dominic Perrottet said the 29 upgraded hospitals and health facilities announced before the 2019 election would be under way before March 2023.

The Australian Medical Association NSW said it was concerned by the state government's continued emphasis on funding health infrastructure over current funding for staffing and resourcing.

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"Hospitals and healthcare staff are struggling to meet patient demand," AMA NSW President Dr Danielle McMullen said.

The recent concerns raised about resourcing and services at Blacktown Hospital highlight the importance of funding all hospitals appropriately," she said.

Dr McMullen was also concerned funding to fast track elective surgeries wont move the needle enough to improve patient access.

We can see that access is deteriorating and wait times are getting worse," she said. "Patients are waiting more than a year for elective surgery in some cases and we know these figures only tell half the story.

Get our Coronavirus Update newsletter for the day's crucial developments and the numbers you need to know. Sign up toThe Sydney Morning Herald'snewsletterhereandThe Age'shere.

Kate Aubusson is Health Editor of The Sydney Morning Herald.

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$30 billion to fight COVID-19 over next five years - Sydney Morning Herald