Category Archives: Genetic Therapy

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced preclinical data will be presented this week on ARCUS genome editing including two oral presentations and two posters at the American Society of Gene & Cell Therapy (ASGCT), May 16-19, 2022 at the Walter E. Washington Convention Center in Washington, D.C.

The preclinical findings presented this week at the ASGCT conference are very encouraging and further support our novel gene editing approach with ARCUS and our plans to advance three wholly owned product candidates, PBGENE-PH1, PBGENE-HBV and PBGENE-PCSK9, to the clinic over the next three years, said Derek Jantz, Ph.D., Chief Scientific Officer and Co-founder of Precision BioSciences. Translating these preclinical results to the clinic will further validate ARCUS as a premier gene editing platform with the precision and versatility needed to develop novel therapeutics that aim to deliver functional cures for conditions including PH1 and chronic hepatitis B.

Oral Presentations:

Abstract #447: Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo

Data from this preclinical study demonstrate Precisions gene editing approach designed to eliminate hepatitis B virus (HBV). ARCUS efficiently targeted and degraded HBV covalently closed circular (cccDNA) by 85% and reduced expression of Hepatitis B Surface Antigen (HBsAg) by 77% in HBV-infected primary human hepatocytes (PHH). Similar levels of editing were achieved in novel mouse and NHP models following lipid nanoparticle (LNP) delivery of ARCUS mRNA, resulting in a 96% reduction in HBsAg in mice. These data suggest that LNP-delivered ARCUS mRNA is a promising approach and potential functional cure for chronic hepatitis B. Precision will continue developing its PBGENE-HBV product candidate using LNP delivery and expects to submit an IND/CTA in 2024.

Abstract #811, Presidential Symposium and Presentation of Top Abstracts: AAV-Meganuclease-Mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver1

Preclinical data will be presented during the Presidential Symposium and highlight an ARCUS-based gene insertion approach for the treatment of ornithine transcarbamylase (OTC) deficiency. This strategy was informed by previous work in adult NHPs, which showed safe, efficient and stable reductions of PCSK9 following adeno associated virus (AAV) delivery of an ARCUS nuclease. In this study, therapeutically meaningful and stable levels of OTC expression were observed in NHPs. Treatment was well-tolerated in all animals, showing no evidence of transaminase elevations or liver histopathology in any ARCUS-treated animals. Preliminary data suggests that the level of editing is stable over one year. Together, these data demonstrate preclinical feasibility of using an ARCUS-mediated gene insertion approach for the treatment of OTC deficiency.

Poster Presentations:

Abstract #239: Optimization of Hydroxyacid Oxidase 1 (HAO1) Targeting ARCUS Nucleases for the Treatment of Primary Hyperoxaluria Type 1 (PH1)

Preclinical data presented in this poster demonstrate HAO1-targeting and optimization of ARCUS nucleases delivered via AAV in NHPs. A single infusion of AAVs carrying first generation HAO1-targeting nucleases resulted in >95% knockdown of HAO1 protein in NHP liver and increases in serum glycolate up to 80M. ARCUS nuclease optimization through iterative rounds of protein engineering resulted in improvements in both potency and specificity of HAO nucleases. These data demonstrate the ability to optimize ARCUS nucleases to specifically target the HAO1 gene to control the glyoxylate metabolic pathway responsible for PH1. Precision has initiated a NHP study for PBGENE-PH1 delivered by LNP and expects to submit an IND or CTA in 2023.

Abstract # 561: ARCUS Gene Editing to Eliminate MELAS-associated m.3243A>G Mutant Mitochondrial DNA2

Preclinical data presented in this poster demonstrate Precisions gene editing approach to shift mitochondrial DNA (mtDNA) heteroplasmy for the m.3243A>G mtDNA mutation. This mutation resides in mitochondrial tRNA and is responsible for >80% of cases of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS). This study found that mitoARCUS was successfully trafficked to the mitochondria and, once there, specifically cleaved and eliminated m.3243G mutant mtDNA without impacting wild-type (WT) mtDNA. This resulted in preferential replication of WT mtDNA, and, consequently, improvements in mitochondrial function. Precision BioSciences protein engineering and optimization platform allowed for the generation of highly specific nucleases that accurately discriminated between WT and mutant mtDNA despite only a single nucleotide difference. Together, these data showcase the promise of ARCUS as a potential in vivo gene editing approach for the treatment of disease-causing heteroplasmic mtDNA mutations.

About Precision BioSciences, Inc.

Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS genome editing platform. ARCUS is a highly precise and versatile genome editing platform that was designed with therapeutic safety, delivery and control in mind. Using ARCUS, the Companys pipeline consists of multiple ex vivo off-the-shelf CAR T immunotherapy clinical candidates and several in vivo gene editing candidates designed to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit http://www.precisionbiosciences.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our preclinical studies, including targeting hepatitis B cccDNA with ARCUS nucleases in novel animal models, ARCUS-based gene insertion approach for the treatment of OTC deficiency, HAO1-targeting and optimization of ARCUS nucleases, the expected timing of future IND and CTA filings, and expected IND updates and advancement of preclinical programs to IND or CTA. In some cases, you can identify forward-looking statements by terms such as aim, anticipate, approach, believe, contemplate, could, estimate, expect, goal, intend, look, may, mission, plan, potential, predict, project, promising, should, suggest, target, will, would, or the negative thereof and similar words and expressions.

Forward-looking statements are based on managements current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical studies and clinical trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators development of product candidates; our or our collaborators ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators ability to enroll patients; changes in interim top-line and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of COVID-19 pandemic and variants thereof, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption Risk Factors in our Annual Report on Form 10-K for the fiscal year ended December 31 2021, as any such factors may be updated from time to time in our other filings with the SEC, including, but not limited to, our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2022, to be filed with the SEC, which are accessible on the SECs website at http://www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

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1 University of Pennsylvanias Gene Therapy Program oral presentation sponsored by iECURE2 Research conducted in collaboration with the University of Miami Miller School of Medicine

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Precision BioSciences Announces Preclinical Data Showcasing Premier In Vivo Gene Editing Capabilities at American Society of Gene & Cell Therapy...

ReportLinker

The global synthetic biology market is expected to grow from US$ 10,544. 16 million in 2021 to US$ 37,850. 85 million by 2028; it is estimated to grow at a CAGR of 20. 0% from 2021 to 2028. The report highlights trends prevailing in the synthetic biology market and factors driving its growth.

New York, May 19, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Synthetic Biology Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Products, Technology, and Application" - https://www.reportlinker.com/p06279470/?utm_source=GNW The increasing investments in synthetic biology and the rising number of start-ups are driving the market growth. However, the renewed regulations for biotechnology hamper the market growth.

Synthetic biology is the science of designing, altering, and building simple organisms to perform specific therapeutic or industrial utilities. The organisms created are genetically modified organisms (GMOs), which do not require a definition that distinguishes them from genetic modifications.

The rising number of start-ups is expected to support market growth during the forecast period.Biotechnology entrepreneurs easily raise funds and procure equipment and space from governments of the respective countries.

Indie Bio (California, US) and EU (Ireland) are among the first synthetic biology accelerators.The start-ups are emerging in Asia Pacific, as governments in this region are providing funds for the domestic development of synthetic biology.

For instance, the Government of India funded IITM Bioincubator, a department of the Indian Institute of Technology Madras, to start a state-of-the-art research facility for cancer biology and a Bioinformatics Infrastructure Facility.The funds were provided by agencies such as the Council of Scientific and Industrial Research (CSIR), the Department of Biotechnology (DBT), and the Department of Science and Technology (DST).

The Indian Institute of Technology Madras raised US$ 7.86 million (550 million rupees) in the fiscal year 20162017. In China, Chinaccelerator is a financer that provides mentorship programs for helping start-ups. It is also associated with SOSV, a venture capital and investment management firm,, which helps establish start-ups by providing funds under programs such as RebelBio and Indie Bio. The easy availability of funds for ideas is motivating entrepreneurs in the world to establish synthetic biology businesses.

Siolta Therapeutics, a US-based firm founded in 2016, produces therapeutic microbial consortia to prevent and treat inflammatory illnesses. It evaluates clinical data and develops mixed-species therapies for oral supplementation. Unlike existing medications for allergies, the microbiome-based approach operates before the onset of the immune dysfunction cascade. STMC-103H, a flagship therapeutic candidate of Siolta Therapeutics, is being studied for the treatment of allergic asthma as well as other chronic inflammatory illnesses, including atopic dermatitis and allergic rhinitis.

Kinnva Ltd., established in 2017, is a synthetic biology start-up catering to biotech, cleantech, and Agri-tech segments. Using a unique fermentation technology and sophisticated processing methods, Kinnva produces high-value biochemicals for applications in the food, feed, nutraceuticals, and cosmetics industries.

Genecis, a Canadian firm established in 2016, employs synthetic biology techniques to convert the trash into high-value commodities. The company used bacterial culture to treat food waste from landfills into compostable polyhydroxyalkanoate (PHA) bioplastics. These bioplastics are suitable for the food, agriculture, and textiles businesses, among others, willing to replace plastics with more sustainable alternatives. PHA polymers, unlike ordinary plastics, disintegrate within a year.

Thus, start-ups are contributing significantly to the synthetic biology market expansion.

Based on product, the synthetic biology market is segmented into oligonucleotides, chassis organisms, enzymes, and xeno-nucleic acid.The oligonucleotides segment is likely to hold the largest share of the market in 2021.

Moreover, the same segment is anticipated to register the highest CAGR in the market during the forecast period of 2021 to 2028.Based on technology, the synthetic biology market is segmented into, gene synthesis, genome engineering, measurement & modeling, cloning & sequencing, nanotechnology, and others.

In 2021, the gene synthesis segment is likely to hold the largest share of the market.However, the genome engineering segment is expected to register highest CAGR during 2021 to 2028.

The growth of genome engineering segment is owing to the rising applications of genetic engineering and gene therapy. Further, based on application, the synthetic biology market is segmented into medical applications, industrial applications, enviornmental applications, food and agriculture, and others. The medical applications segment is further segmented as, drug discovery & therapeutics and pharmaceuticals. In 2021, the medical applications segment held the largest market share, and it is expected to register the highest CAGR during 20212028.

Various organic and inorganic strategies are adopted by companies operating in the synthetic biology market.The organic strategies mainly include product launches and product approvals.

Inorganic growth strategies witnessed in the market are acquisitions, collaboration, and partnerships.These growth strategies have allowed the synthetic biology market players in expanding their business and enhancing their geographic presence, along with contributing to the overall market growth.

Additionally, growth strategies such as acquisitions and partnerships helped them strengthen their customer base and extend their product portfolios. A few of the significant developments by key players in the synthetic biology market are listed below.

In February 2022, Thermo Fisher Scientific announced the launch of GeneMapper Software, a flexible genotyping software package that provides DNA sizing and quality allele calls for all Thermo Fisher Scientific electrophoresis-based genotyping.

In January 2022, Thermo Fisher Scientific announced the launch of Oncomine BRCA Assay GX. It is a targeted next-generation sequencing (NGS) assay designed to provide comprehensive amplification of all coding regions of the human BRCA1 and BRCA2 genes, enabling accurate and sensitive detection of various mutation classes.

In April 2022, Merck KGaA Acquisition of MAST Platform from Lonza, a Leading Automated Bioreactor Sampling System. The acquisition of the MAST platform is another milestone to accelerate innovation in Mercks Process Solutions business unit.

In February 2022, Merck announced the transaction closing to acquire Exelead, following regulatory clearances and the fulfillment of other customary closing conditions, for approximately USD 780 million in cash.

A few of the key primary and secondary sources referred to while preparing the report on the synthetic biology market are the World Health Organization (WHO), the National Institute of Health (NIH), and the National Center for Biotechnology Information (NCBI).Read the full report: https://www.reportlinker.com/p06279470/?utm_source=GNW

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

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In 2021, the gene synthesis segment is likely to hold the largest share of the market.However, the genome engineering segment is expected to register...

Paddy Doherty leaned back in the narrow hospital bed and watched a bag of clear liquid drain into a vein in his left arm, whisking billions of invisible strands of mRNA into the torrent of his bloodstream.

It was only in February hed learned the pinching pains in his toes and gasping for breath that had begun to haunt his daily dog walks through the hills of Donegal County were caused by a deformed protein piling up in his nerves and heart. Now here he was, a few months later, at a clinic in central London, so that doctors could try to fix the genetic defect hed inherited the same one that had already killed his father, an uncle, and a cousin.

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From burst bubble to medical marvel: How lipid nanoparticles became the future of gene therapy - STAT

Photo: Scott Strazzante/San Francisco Chronicle (AP)

Google announced Monday it will allow ads for stem cell treatments approved by the Food and Drug Administration to appear in search results starting in July. The tech giant previously banned any ads for stem cell therapies, FDA-approved or otherwise.

In an update to its policies page first spotted by Gizmodo, the company said that, starting July 11, it will permit search engine ads for stem cell therapies given the thumbs up from the FDA, a very small list of just 23 companies that treat some blood disorders and cancers, according to the FDAs website.

At the same time, Google is clarifying its policy language on stem cell therapy ads, which would allow a global cell or gene therapy company to advertise if the ads are are exclusively educational or informational in nature, regardless of regulatory approval status. Google did not clarify what would constitute educational or informational, nor did the company respond to a request for comment how it will restrict less-than-reputable products from being advertised with its technology going forward. We will update the story if we hear more.

The search engine said it banned all advertising for stem cell treatments back in 2019, proclaiming at the time it was restricting ads that have no established biomedical or scientific basis. In 2021, the company clarified that it was restricting ads for experimental treatments meant for so-called biohacking or other DIY genetic engineering, as well as any cell or gene therapies like stem cell therapy.

Despite the pledge to ban such ads or Mondays announced change, a simple Google search reveals just how easily bad actors can get around the restrictions. Searching for stem cells for neuropathy reveals several misleading ad results for stem cell treatments that are not FDA approved, though at least one maker claims it is FDA registered and another says its treatment is supported by FDA master files.

Paul Knoepfler, a professor at the University of California Davis School of Medicine who researches stem cells and cancer, has written before about Googles problematic search engine ad policies that allow stem cell companies to easily advertise their products in spite of the tech giants rules. In an email, he told Gizmodo he is concerned How effectively the new rule for strictly educational ads would be maintained, particularly given the context of Google Search now so often highly ranking promotional clinic websites arguably presented as educational material.

Stem cells as an industry have grown rapidly in recent years and are expected to continue doing so, with MarketWatch reporting in February the $2.75 billion industry is expected to more than double to $5.72 billion by 2028.

Stem cell treatments are approved by the FDAs Cellular, Tissue and Gene Therapies Advisory Committee. Though some companies claim in advertising they have FDA approval, being listed on clinicaltrials.gov database or being registered with the FDA isnt full-on approval, according to the agencys guidelines. The fact that companies regularly run around Googles existing policies leaves even more questions on the table. Knoepfler asked whether clinical trial recruitment be allowed, when hes often seen such trials already claiming their treatment already works.

Perhaps good citizens in the regenerative medicine world want the opportunity to run such ads related to clinical trial recruitment, but even exclusively educational ads of that type with good intentions could run into ethical issues, Knoepfler added.

Shoshana Wodinsky contributed reporting.

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Google Reverses Ban on Ads for All Stem Cell Therapies, Will Allow FDA-Approved Ones - Gizmodo

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a biotechnology company with a mission to discover, develop and deliver curative therapies that address the underlying causes of heart disease, announced that it will present preclinical data for its TN-401 and DWORF gene therapy programs, as well as data on its capsid engineering capabilities at the upcoming American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting taking place May 1619, 2022, in Washington, D.C. and virtually.

TN-401 is Tenayas gene therapy candidate being developed for the potential treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations. Mutations of the PKP2 gene are the leading genetic cause of ARVC and can result in severe disease, including significant arrhythmia and sudden cardiac death in adults and children. These mutations are estimated to affect more than 70,000 patients in the U.S. alone. Data being presented at ASGCT will detail the impact of a single dose of PKP2 gene therapy on arrhythmias, disease progression and survival in a Pkp2-deficient mouse model of ARVC. Tenaya will support the establishment of a global natural history study of ARVC caused by PKP2 mutations in 2022 and expects to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration for TN-401 in 2023.

The companys DWORF gene therapy is being developed for the potential treatment of genetic dilated cardiomyopathy (DCM). DWORF is a muscle specific micro-peptide first discovered by Tenaya co-founder Eric Olson, Ph.D. that acts on the SERCA pathway, which is widely considered to be a promising target in heart failure. Data to be shared at ASGCT will review initial tolerability and efficacy of adeno-associated viral (AAV) delivered DWORF in a DCM mouse model. Tenayas DWORF program is currently at candidate selection stage.

Tenaya believes its capsid engineering efforts will ultimately help support successful clinical development of its product candidates by enhancing the efficacy and safety of gene therapies. Data being presented for Tenayas capsid engineering efforts will showcase the work being done to enhance the specificity and expression of genes delivered to cardiomyocytes using novel capsids.

Details of the presentations are as follows:

Tuesday, May 17, 20225:30 p.m. 6 :30 p.m. ETCardiovascular and Pulmonary Diseases session

To view full event programming, please visit the ASGCT 25th Annual Meeting website.

About Tenaya Therapeutics

Tenaya Therapeutics is a biotechnology company committed to a bold mission: to discover, develop and deliver curative therapies that address the underlying drivers of heart disease. Founded by leading cardiovascular scientists from Gladstone Institutes and the University of Texas Southwestern Medical Center, Tenaya is developing therapies for rare genetic disorders as well as for more prevalent heart conditions through three distinct but interrelated product platforms: Gene Therapy, Cellular Regeneration and Precision Medicine. For more information, visit http://www.tenayatherapeutics.com.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as will, potential, expects and believes, and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, Tenayas plans to present preclinical data on its TN-401 and DWORF gene therapy programs at the ASGCT 25th Annual Meeting; the therapeutic potential of TN-401 as a treatment for ARVC caused by PKP2 gene mutations; Tenayas plans to support the establishment of a global natural history study of ARVC caused by PKP2 mutations and expectations regarding the timing of the IND filing for TN-401; the therapeutic potential of Tenayas DWORF gene therapy program as a treatment for DCM; and Tenayas belief that its capsid engineering efforts will enhance the efficacy and safety of gene therapies. The forward-looking statements contained herein are based upon Tenayas current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: the availability of data at the referenced times; risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenayas ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; the timing, progress and results of preclinical studies for TN-401, DWORF and Tenayas other programs; Tenayas ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Tenayas manufacturing and operations, including preclinical studies and planned clinical trials; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Tenayas manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenayas reliance on third parties; Tenayas ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled Risk Factors in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

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Tenaya Therapeutics to Present Preclinical Data on Gene Therapy Programs and Platform Capabilities at the American Society of Gene and Cell Therapy...

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market Research Report by Type (Antigen Gene Therapy, Cancer Gene Therapy, and Cytokine Gene Therapy), Vector Type, Application, Region (Americas, Asia-Pacific, and Europe, Middle East & Africa) - Global Forecast to 2027 - Cumulative Impact of COVID-19" report has been added to ResearchAndMarkets.com's offering.

The Global Gene Therapy Market size was estimated at USD 2,828.62 million in 2021, USD 3,463.59 million in 2022, and is projected to grow at a Compound Annual Growth Rate (CAGR) of 22.62% to reach USD 9,617.32 million by 2027.

In this report, the years 2019 and 2020 are considered historical years, 2021 as the base year, 2022 as the estimated year, and years from 2023 to 2027 are considered the forecast period.

Cumulative Impact of COVID-19:

The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Competitive Strategic Window:

The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects.

It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

Market Share Analysis:

The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others.

Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

The report answers questions such as:

Key Topics Covered:

1. Preface

1.1. Objectives of the Study

1.2. Market Segmentation & Coverage

1.3. Years Considered for the Study

1.4. Currency & Pricing

1.5. Language

1.6. Limitations

1.7. Assumptions

1.8. Stakeholders

2. Research Methodology

2.1. Define: Research Objective

2.2. Determine: Research Design

2.3. Prepare: Research Instrument

2.4. Collect: Data Source

2.5. Analyze: Data Interpretation

2.6. Formulate: Data Verification

2.7. Publish: Research Report

2.8. Repeat: Report Update

3. Executive Summary

4. Market Overview

5. Market Insights

5.1. Market Dynamics

5.1.1. Drivers

5.1.1.1. Significant way of treatment to eradicates the practice of surgery, drugs, or other procedures that lead to side effects on the health of individuals

5.1.1.2. Innovation in gene therapy for rare & cardiovascular diseases treatment coupled with the awareness regarding the ability of gene therapy

5.1.1.3. Application in the treatment of various diseases such as cancer, haemophilia, parkinson's disease, and HIV

5.1.1.4. Government support and ethical acceptance of gene therapy for cancer treatment

5.1.2. Restraints

5.1.2.1. High cost associated with the gene therapies

5.1.3. Opportunities

5.1.3.1. Upsurge in a number of gene therapy treatment centers in developed countries

5.1.3.2. Increasing adoption for gene therapy for the oncological disorder

5.1.3.3. Growing strategic alliances and ongoing research for orphan diseases

5.1.4. Challenges

5.1.4.1. Potential disorders associated with the immune response

5.2. Cumulative Impact of COVID-19

5.3. Cumulative Impact of 2022 Russia Ukraine Conflict

6. Gene Therapy Market, by Type

6.1. Introduction

6.2. Antigen Gene Therapy

6.3. Cancer Gene Therapy

6.4. Cytokine Gene Therapy

6.5. Suicide Gene Therapy

6.6. Tumor Suppressor Gene Therapy

7. Gene Therapy Market, by Vector Type

7.1. Introduction

7.2. Non-viral Vectors

7.3. Viral Vectors

8. Gene Therapy Market, by Application

8.1. Introduction

8.2. Cardiovascular Diseases

8.3. Genetic Diseases

8.4. Infectious Diseases

8.5. Neurological Diseases

8.6. Oncological Disorders

For more information about this report visit https://www.researchandmarkets.com/r/mfwu95

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Gene Therapy Market Research Report 2022: Antigen Gene Therapy, Cancer Gene Therapy, and Cytokine Gene Therapy - Global Forecast to 2027 - Cumulative...