Archive for the ‘Gene Therapy Research’ Category

Bench-to-bedside

Armed with this knowledge, further experiments showed that giving G-CSF, which is already used clinically as an immune system booster in cancer patients to mice with non-mutant IDH1 also increased their survival. And giving it in combination with the immune-stimulating gene therapy had an even bigger impact.

The team also confirmed that patients who have gliomas with mutated IDH1 also have higher levels of G-CSF circulating in their blood a clue that the findings will be applicable beyond the mouse models.

The next step, says Lowenstein, will be to work on moving these findings into a clinical trial, building on the current, ongoing trial using the immunotherapy/gene therapy combination.

Our study shows two main things: Patients with the IDH1 mutation may benefit from immunotherapy due to the G-CSF their tumors are producing, he said. And patients without the mutation may benefit from combining treatment with G-CSF and immunotherapy.

Additional authors include Brandon L. McClellan, Ruthvik P. Avvari, Rohit Thalla, Stephen Carney, Margaret S. Hartlage, Santiago Haase, Maria Ventosa, Ayman Taher, Neha Kamran, Li Zhang, Syed Mohammed Faisal, Felipe J. Nez, Mara Beln Garcia-Fabiani, Wajd N. Al-Holou, Daniel Orringer, Jason Heth, Parag G. Patil, Karen Eddy, Sofia D. Merajver, Peter J. Ulintz, Joshua Welch, Chao Gao, Jialin Liu and Gabriel Nez all of U-M; Shawn Hervey-Jumper of University of California, San Francisco; and Dolores Hambardzumyan of the Tisch Cancer Institute, Mount Sinai School of Medicine, New York.

Funding for the work was provided by National Institutes of Health and National Institute of Neurological Disorders & Stroke (R37-NS094804, R01-NS105556, R21- NS107894, R01- NS076991, R01-NS082311, R01-NS096756; the U-M Department of Neurosurgery; the Pediatric Brain Tumor Foundation, Leahs Happy Hearts Foundation, Ians Friends Foundation, Chad Tough Foundation, Pediatric Brain Tumor Foundation, and Smiles for Sophie Forever Foundation, National Cancer Institute (T32-CA009676), American Brain Tumor Association Basic Research Fellowship and a Rogel Cancer Center Scholar Award.

Paper cited: G-CSF secreted by mutant IDH1 glioma stem cells abolishes myeloid cells immunosuppression and enhances the efficacy of immunotherapy, Science Advances. DOI: 10.1126/sciadv.abh3243

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Glioma subtype may hold the secret to the success of immunotherapies - Michigan Medicine

SAN DIEGO--(BUSINESS WIRE)--DTx Pharma, Inc. (DTx), a privately-held biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease, announced that Dr. Arthur Suckow, Co-Founder and CEO, will present at the Ophthalmology Innovation Summits Retina Innovation Showcase on October 7, 2021, during the Annual Scientific Meeting of the American Society of Retina Specialists.

Dr. Suckow will make a presentation during the Spotlight on Cell and Gene Therapy session and discuss DTx Pharmas most advanced program for retinitis pigmentosa (RP). RP is a rare disorder that affects roughly 1 in 4,000 people, both in the United States and worldwide. People with the disease tend to initially develop night blindness, followed by total blindness, as a result of the death of their photoreceptor cells. No therapeutic options exist for this condition that can be caused by more than 300 mutations of 100 different genes.

DTx Pharmas proprietary FALCON (Fatty Acid Ligand Conjugated Oligonucleotide) delivery platform is designed to improve the efficacy of RNA therapies by using fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body. Using this novel technology platform, DTx Pharma can improve the cellular uptake of targeted RNA therapies and apply therapies to multiple cell types in the eye, with the goal of developing a gene agnostic therapy for RP.

There are no effective therapeutics for this debilitating disease. We look forward to presenting our preclinical data this week and to working with the experts in retinitis pigmentosa to evaluate a game-changing therapeutic in patients over the next several years, says Dr. Suckow.

About DTx Pharma

DTx Pharma, Inc. is a privately held biotechnology company based in San Diego, CA creating novel RNA-based therapeutics to treat the genetic drivers of disease. The companys proprietary delivery technology platform utilizes fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body. In preclinical studies, DTx has demonstrated cellular uptake and broad activity of oligonucleotides in the retina, muscle, heart, neurons, T cells, and other specialized cell types. To advance the FALCON platform toward and into clinical development, DTx has raised more than $100M in combined investment from several of the worlds leading healthcare investors including RA Capital Management and Access Biotechnology, pharmaceutical companies such as Eli Lilly and Company, the National Institute of Health (NIH), and research foundations such as the CMT Research Foundation (CMTRF). To learn more about DTx Pharma, please visit http://www.dtxpharma.com and follow DTx on LinkedIn and Twitter @DTxPharma.

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DTx Pharma to Present at the OIS Retina Innovation Showcase - Business Wire

DUBLIN, September 30, 2021--(BUSINESS WIRE)--The "Viral Vectors And Plasmid DNA Manufacturing Market Size By Product Type, By Application, By End Product, By Geographic Scope And Forecast" report has been added to ResearchAndMarkets.com's offering.

The Global Viral Vectors and Plasmid DNA Manufacturing Market was valued at USD 583.71 Million in 2020 and is projected to reach USD 1,866.90 Million by 2028, growing at a CAGR of 15.40% from 2021 to 2028.

A growing number of patients opting for gene therapy is a major factor propelling the growth of the Viral Vectors and Plasmid DNA Manufacturing market. Gene therapy is a leading field in medical science, which promises new treatment development for patients suffering from various disease. Genetically modified therapies have emerged as a promising treatment approach for various diseases (primarily ones that currently have no cure), including inherited disorders and certain viral infections. Demand for plasmid DNA is rising steeply because of a boom in gene therapy development.

This report provides an all-inclusive environment of the analysis for the Viral Vectors And Plasmid DNA Manufacturing Market. The market estimates provided in the report are the result of in-depth secondary research, primary interviews and in-house expert reviews. These market estimates have been considered by studying the impact of various social, political and economic factors along with the current market dynamics affecting the Viral Vectors And Plasmid DNA Manufacturing Market growth.

Along with the market overview, which comprises of the market dynamics the chapter includes a Porter's Five Forces analysis which explains the five forces: namely buyers bargaining power, suppliers bargaining power, threat of new entrants, threat of substitutes, and degree of competition in the Viral Vectors And Plasmid DNA Manufacturing Market. It explains the various participants, such as system integrators, intermediaries and end-users within the ecosystem of the market. The report also focuses on the competitive landscape of the Viral Vectors And Plasmid DNA Manufacturing Market.

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The report will provide a valuable insight with an emphasis on the global market including some of the major players such as Merck KGaA, Lonza, FUJIFILM Diosynth Biotechnologies U.S.A., Inc., Cobra Biologics Ltd., Brammer Bio, Waisman Biomanufacturing, Genezen, YPOSKESI, Advanced BioScience, Laboratories, Inc. (ABL, Inc.), Novasep Holding S.A.S, ATVIO Biotech Ltd, and Others.

Key Topics Covered:

1 Introduction

2 Research Methodology

3 Executive Summary

3.1 Market Overview

3.2 Global Viral Vectors and Plasmid DNA Manufacturing Market Regional Insights

3.3 Global Viral Vectors and Plasmid DNA Manufacturing Market Geographical Analysis

3.4 Global Viral Vectors and Plasmid DNA Manufacturing Market, by Product Type

3.5 Global Viral Vectors and Plasmid DNA Manufacturing Market, by Application

3.6 Global Viral Vectors and Plasmid DNA Manufacturing Market, by End Product

3.7 Future Market Opportunities

3.8 Global Market Split

4 Market Outlook

4.1 Global Viral Vectors and Plasmid DNA Manufacturing Market Outlook

4.2 Market Drivers

4.2.1 Increasing Number of Patients Opting for Gene Therapy

4.2.2 Rising Prevalence of HIV/Aids and Growing R&D Funding from Several Organizations

4.3 Restraints

4.3.1 Manufacturing Challenges Pertaining to Large Scale Production of Vectors

4.4 Opportunities

4.4.1 Growing Healthcare Infrastructure and Government Support

4.4.2 Growing Number of Gene Therapy Candidates, Coupled With Their Rapid Progression Through Various Phases of Clinical Development

4.5 The Impact of Covid-19

4.6 Porters Five Force Model

4.7 Product Life Line

5 Market, by Product Type

5.1 Overview

5.2 Viral Vector

5.3 Plasma DNA

5.4 Non-Viral DNA Vectors

6 Market, by Application

6.1 Overview

6.2 Cancer

6.3 Inherited Disorder

6.4 Infectious Diseases

6.5 Others

7 Market, by End Product

7.1 Overview

7.2 DNA Vaccines

7.3 Gene Therapy

7.4 Immunotherapy

7.5 Others

8 Market, by Geography

8.1 Overview

8.2 North America

8.3 Europe

8.4 Asia-Pacific

8.5 Row

9 Competitive Landscape

10 Company Profiles

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

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210930005708/en/

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Viral Vectors and Plasmid DNA Manufacturing Market 2021; Growing Number of Gene Therapy Candidates, Coupled with their Rapid Progression through...

LAWRENCEVILLE, N.J., Oct. 05, 2021 (GLOBE NEWSWIRE) -- Celsion Corporation (NASDAQ: CLSN), a clinical-stage development company focused on DNA-based immunotherapy and next-generation vaccines, announces the strengthening of its management team with a new hire and a promotion in its vaccine development program, and the hiring of a veteran clinical trial project manager for its Phase II GEN-1 immunotherapy study in advanced ovarian cancer. These changes all are effective immediately and are as follows:

Carlo Iavarone, Ph.D. joins as Senior Director, Non-Clinical Research

Subeena Sood, Ph.D. promoted to Senior Manager, Biology and Preclinical Studies

Beth J. Llewellyn joins as Director of Clinical Operations

Dr. Iavarone will serve as project leader for the PLACCINE vaccine initiative. He will be based in Huntsville, Ala. and brings to Celsion more than 15 years of experience investigating and leading the development of vaccines, including molecular target identification and characterization of RNA vaccines. Most recently, from 2019 until 2021 he was a science advisor for both Guidepoint and Clora, providing input for a viral target and RNA vaccine delivery system. Dr. Iavarone joined GlaxoSmithKline in 2015 as a senior scientist studying small molecules and RNA vaccines in animal and human cell lines. From 2007 until 2015 he held positions of increasing responsibility at Novartis, including as a principal scientist for a melanoma vaccine project.

Dr. Iavarone has authored more than 15 papers on oncology and vaccine research that were published in peer-reviewed journals. He holds a Ph.D. in Molecular Pathology and Physiopathology from Federico Il University in Naples, Italy, and did his post-doctoral work at Novartis in Siena, Italy.

Dr. Sood is responsible for assay development and in vivo experiments for the PLACCINE DNA vaccine and gene therapy program, and also is based in Huntsville. She has experience with several pharmaceutical companies in experiment design, pharmacological and biochemical assays, manufacturing process design and development, and optimization and implementation of Quality by Design. Dr. Sood joined Celsion as manager of animal research in 2019, where she has designed and conducted all preclinical research. Prior to Celsion, since 2017 she was a Formulation Scientist II at Novocol Healthcare. From 2016 to 2017 Dr. Sood was a Research Associate II at Nektar Therapeutics, and from 2015 to 2016 she was a Quality Control Chemist I at Par Pharmaceuticals. She also worked in regenerative medicine as a Research Fellow at Medstar Heart Institute, Washington Hospital Center in Washington, D.C. from 2010 to 2013.

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Dr. Sood has authored more than 25 articles published in peer-reviewed journals, mainly in the area of cardiology and oxidative stress. She received her Ph.D. in Pharmacology from the All India Institute of Medical Sciences in New Delhi, and was a post-doctoral associate at the Baylor College of Medicine.

Ms. Llewellyn is responsible for the management of the ongoing Phase II OVATION 2 Study with GEN-1 in advanced ovarian cancer and will be based at our corporate office in Lawrenceville. Previously she was the President of 2L Pharma, a clinical operations consulting firm she founded in 2014. Her work included preparing protocols for Investigational New Drug submissions to the U.S. Food and Drug Administration, clinical trial site qualification and compliance and functioning as a liaison between clinical trial sites, contract research organizations and study sponsors. From 2011 to 2014 she was a Clinical Operations Management Consultant for Alba Therapeutics with oversight for all clinical activities related to a Phase IIb protocol investigating the use of a novel pharmaceutical agent for celiac disease. From 2010 to 2011 she was a Clinical Research Associate for Nabi Biopharmaceuticals, where she was responsible for providing in-house and field study monitoring, operational guidance and general assistance for multiple protocols investigating the use of a novel vaccine.

Ms. Llewellyn has been involved in over 30 clinical trials in a variety of therapeutic areas including oncology and infectious disease. She received a B.A. in psychology from Ohio University. Her graduate training in experimental psychology included studies in research design and statistical analysis.

As we continue to advance the development of our PLACCINE DNA-mediated vaccine platform and or Phase II study of GEN-1, we are delighted to deepen our bench strength with these talented and experienced professionals, said Michael H. Tardugno, Chairman, President and Chief Executive Officer of Celsion. Dr. Iavarone brings impressive clinical development experience particularly in RNA vaccines, which is so important Celsions work to develop a SARS-CoV-2 vaccine utilizing a DNA plasmid that encodes for multiple viral antigens.

Dr. Sood has been instrumental in the successful development of assays used to evaluate biological activity of our first generation of vaccines. She has proven herself to be a capable scientific leader, whose expertise will be relied upon as we complete our preclinical work to establish proof of concept using for the PLACCINE platform Covid-19 as a benchmark vaccine.

Lastly, as we advance our Phase 2 study with GEN-1 in advanced ovarian cancer, we welcome Ms. Llewellyn to Celsion. She is charged with ensuring that trial enrollment proceeds as planned, and we are confident she will capably address any protocol issues that might arise, Mr. Tardugno continued. Overall, we believe that with this strengthened team we are better able to realize the promise of Celsions technologies for the benefit of patients and our stockholders.

About Celsion Corporation

Celsion is a fully integrated, clinical stage biotechnology company focused on advancing a portfolio of innovative cancer treatments, including immunotherapies, DNA-based therapies and directed chemotherapies through clinical trials and eventual commercialization. The companys product pipeline includes GEN-1, a DNA-based immunotherapy for the localized treatment of ovarian cancer. ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin, is under investigator-sponsored development for several cancer indications. Celsion also has two feasibility stage platform technologies for the development of novel nucleic acid-based immunotherapies and other anti-cancer DNA or RNA therapies. Both are novel synthetic, non-viral vectors with demonstrated capability in nucleic acid cellular transfection. For more information on Celsion, visit http://www.celsion.com.

Forward-looking Statements

Forward-looking statements in this news release are made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon current beliefs, expectation, and assumptions and include statements regarding the platform having the potential to provide broad protection against coronavirus disease 2019 (COVID-19), and possible future mutations of SARS-CoV-2 or other coronaviruses. These statements are subject to a number of risks and uncertainties, many of which are difficult to predict, including the ability of the Companys platform to provide broad protection against COVID-19, and possible future mutations of SARS-CoV-2 or other coronaviruses, the issuance of a patent to the Company for use of its technology platform for treating or preventing infection with the SARS-CoV-2 virus that causes COVID-19, unforeseen changes in the course of research and development activities and in clinical trials; the uncertainties of and difficulties in analyzing interim clinical data, particularly in small subgroups that are not statistically significant; FDA and regulatory uncertainties and risks; the significant expense, time and risk of failure of conducting clinical trials; the need for Celsion to evaluate its future development plans; possible acquisitions or licenses of other technologies, assets or businesses; possible actions by customers, suppliers, competitors or regulatory authorities; and other risks detailed from time to time in the Celsion's periodic filings with the Securities and Exchange Commission. Celsion assumes no obligation to update or supplement forward-looking statements that become untrue because of subsequent events, new information or otherwise.

Celsion Investor ContactJeffrey W. ChurchExecutive Vice President and CFO609-482-2455jchurch@celsion.com

LHA Investor RelationsKim Sutton Golodetz212-838-3777kgolodetz@lhai.com

# # #

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Celsion Corporation Adds Key Resources to its Vaccine Development Initiative and Clinical Trial Capabilities - Yahoo Finance

An Ottawa biotech startup has raised millions of dollars to scale up its technology that aims to supercharge production of a key component in emerging gene therapies for cancer and other diseases.

Virica Biotech announced the series-A round, which was led by New York-based Dynamk Capital, on Monday. The company did not reveal the exact value of the deal, which is expected to close later this year with additional contributions from follow-on investors, but co-founder and CEO Jean-Simon Diallo said it was a multimillion-dollar investment.

Founded in 2018, Virica Biotech makes specialized compounds that help boost production of viral vectors, which deliver material into infected cells thats designed to fix defective genes responsible for diseases such as cancer and hereditary blindness.

Diallo used a cooking analogy to describe the process, likening viral vectors to kernels of popcorn that need the catalyst his firm provides to rapidly reproduce.

If you dont apply some heat, youre going to get maybe a few popped kernels, but thats it, he said. Adding our (product) basically allows you to fully pop the popcorn. Were kind of supercharging the microwave, if you will.

Diallo began working on the technology alongside pioneering Ottawa Hospital cancer researcher Dr. John Bell a decade ago as a way to help cancer-fighting viruses bypass the human immune systems defences.

When financing proved hard to come by due to the lengthy approvals process for such therapies, Diallo found another use for the compound that didnt require the same stringent testing: an additive to boost the effectiveness of traditional vaccines that contain virus particles.

Investors soon took note, and the company raised nearly a million dollars in seed funding by early 2020, just as the pandemic triggered a worldwide R&D effort to develop a new vaccine against COVID-19.

While his compound doesnt work on mRNA vaccines that have widely used in the fight against the coronavirus, Diallo says the pandemic gave his firm a proverbial shot in the arm nonetheless.

It forced us to get set up very quickly, he explained. There was certainly a lot of interest in the technology. COVID kind of got us moving. We realized that the opportunity for improving the manufacturing yield of gene therapies in particular was quite enormous.

Diallo says Virica now has contracts with more than 20 manufacturers of gene therapies that hope his products will make their treatments cheaper and more effective.

The firms revenues have more than doubled in the past 18 months, and Diallo expects Viricas headcount to grow from 16 to at least 30 by next year as demand for its technology ramps up. In addition, he says he still sees a big upside in the vaccine market, which now accounts for about 15 per cent of the firms sales.

Historically, the vaccine industry has always been very slow to move, he said. Hopefully, (the COVID) crisis will change things a little bit for the better because everybody had to kind of roll up their sleeves and innovate.

Virica was initially based out of Bayview Yards as part of Invest Ottawas pre-accelerator program. But the growing startup struggled to find suitable lab space in the region until the new Ottawa of Ottawa Health Innovation Hub helped it secure a 3,000-square-foot facility at the University of Ottawa Heart Institute that officially opened this week.

Diallo, a biochemistry professor at uOttawa and a senior scientist at the Ottawa Hospital Research Institute, said he considered moving the company to Montreal and Toronto. He called on the citys business and political leaders to beef up support for biotech ventures like his that are in the early stages of commercializing their products.

We were really glad to be able to stay in Ottawa, Diallo said. There is a biotech sector in Ottawa, it is growing and we need to work together to foster it. Otherwise, people like me are going to leave. Its that simple.

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Ottawa biotech startup Virica gets multimillion-dollar funding injection for gene therapy-boosting technology - Ottawa Business Journal

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been granted orphan drug designation from the European Commission for TSHA-101, an AAV9-based bicistronic gene replacement therapy in development for GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease.

GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A. The prognosis is devastating, with infantile forms often leading to death within the first four years of life and juvenile onset patients rarely surviving beyond mid-teens, said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. Residual Hex A activity correlates with the severity of GM2, and based on our understanding of this correlation, small increases in Hex A activity are likely to lead to significant improvements in clinical outcomes and quality of life. Based on dose-dependent improvements in survival in preclinical models, we are highly encouraged that our novel bicistronic gene therapy approach with TSHA-101 has the potential to be a life changing therapy for patients suffering from this rapidly progressive disorder with no current treatment options.

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder that is part of a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A (Hex A) enzyme. These genetic defects result in progressive dysfunction of the central nervous system. Residual Hex A enzyme activity determines the severity of the disease. The infantile form of the disease has an onset of symptoms usually before six months of age with residual Hex A enzyme activity of less than 0.1%. Juvenile onset occurs between 1.5 and five years of age with residual Hex A enzyme activity of approximately 0.5%. Early adult onset of the disease has residual Hex A enzyme activity of between 2% to 4%. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.

TSHA-101 is an investigational gene therapy that delivers the HEXA and HEXB genes that make up the -hexosaminidase A enzyme. The two genes are driven by a single promoter within the AAV9 bicistronic vector ensuring that the 2 sub-units of Hex A are produced in a one-to-one ratio within each cell, which is important to ensure efficient production of the transgene. TSHA-101 is the first and only bicistronic vector currently in clinical development and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA). TSHA-101 is administered intrathecally and is currently being evaluated in a single arm, open-label Phase 1/2 clinical trial for the treatment of infants with GM2 gangliosidosis sponsored by Queens University. Preliminary clinical safety and biomarker data are expected by year-end 2021.

The European Commission grants orphan drug designation for medicines being developed for the diagnosis, prevention or treatment of treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-101, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-101s eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, both of which are available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis -...

DUBLIN--(BUSINESS WIRE)--The "Nucleic Acid Therapeutics CDMO Market - A Global and Regional Analysis: Focus on Product, Technology, and End User - Analysis and Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

The global nucleic acid therapeutics CDMO market was valued at $1,546.4 million in FY2020 and is estimated to reach $4,463.7 million by 2030.

The completion of human genome sequencing and the elucidation of the molecular pathways that are critical in the disease molecule interaction have offered an unprecedented opportunity and growth for the development of nucleic acid-based therapeutics. However, to keep with the manufacturing and development of such therapies, the pharmaceutical companies have established partnerships with the contract development and manufacturing company (CDMO) which are the viable alternatives to the in-house development of the drugs. Moreover, the success of the respective business model has also led these CDMOs to become an integral part of such pharmaceutical companies' value chain.

The increasing willingness to outsource drug development to the CDMOs, and the rising need for pharmaceuticals have resulted in the expansion of the global market for nucleic acid therapeutics CDMO.

In the past decade, there has been a vast increase in the amount of gene sequence information that has the potential to revolutionize the way diseases are categorized and treated. Traditional diagnoses, largely anatomical or descriptive in nature, are likely to be superseded by the molecular characterization of the disease. The fact that certain genes drive key disease processes will also enable the rational design of gene-specific therapeutics. Antisense oligonucleotides represent a technology that can play multiple roles in this process. Further, at present, there are 16 nucleic acid therapies approved by the FDA and EMA and many more in the pipeline implying the reliance and acceptance over the usage of such therapies in the market.

Market Growth Drivers

Market Challenges

Market Opportunities

Key Questions Answered in this Report:

Key Topics Covered:

1 Markets

1.1 Industry Outlook

1.2 Product Definition

1.3 Global Nucleic Acid Therapeutics CDMO Market Footprint, ($ Million), 2020-2030

1.4 Current Nucleic Acid Therapeutic CDMOs Landscape

1.5 Significant Usage of Nucleic Acid in Therapeutics

1.6 Types of Nucleic Acid Synthesized for Therapeutics

1.7 Market Dynamics

1.7.1 Market Drivers

1.7.1.1 Accelerating Shift of the Pharmaceutical Market Toward Innovative Biologic and Cell and Gene Therapy Products

1.7.1.2 Reduction in Overall Manufacturing Cost at CDMOs

1.7.1.3 Rising Approvals of Nucleic Acid Therapeutics

1.7.2 Restraints

1.7.2.1 Lack of Expertise in Nucleic Acid Manufacturing

1.7.2.2 Supply Chain and Logistical Challenges

1.7.2.3 Difficult Therapeutic Classification Due to Wide Variety of Nucleic Acids

1.7.3 Opportunities

1.7.3.1 Increasing Outsourcing Trend Among Pharmaceutical Companies

1.7.3.2 Accelerating Research and Development Along with Technology

1.7.3.3 Growth in Developing Countries

2 Competitive Landscape

3 Chemical Synthesis Method

4 Technology

5 End-User

6 Products

7 Regions

8 Markets - Competitive Benchmarking & Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/1x5210

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Nucleic Acid Therapeutics CDMO Market to Reach $4463.7 Million by 2030 - ResearchAndMarkets.com - Business Wire

Shape Therapeutics CEO Francois Vigneault. (Shape Photo)

Shape Therapeutics has signed a deal potentially exceeding $3 billion with pharma giant Roche to support the development of gene therapies for Alzheimers and Parkinsons disease, the Seattle company announced Wednesday.

Shapes RNA editing technologies can change the sequence of RNA, which encodes the bodys protein building blocks. The company will deploy this technology with Roche in preclinical studies against targets relevant for these neurological conditions as well as certain rare diseases.

The collaboration may also leverage the biotech companys technologies for gene delivery. This system is designed to deliver RNA editing technology or other payloads directly to specific areas of the body, such as the nervous system or muscle.

Shape aims to unlock the next breakthrough in RNA technologies in the gene therapy space across a wide range of therapeutic areas, said Francois Vigneault, co-founder and CEO, in a statement.

Shapes RNA editing technology could potentially be used to change the amount of a key regulatory protein in the body or treat genetic diseases. The companys gene delivery technology is based on AAV vectors, a platform currently used to treat several rare conditions.

Under the agreement, Shape is eligible to receive an initial payment as well payments for hitting development, regulatory and sales milestones potentially exceeding $3 billion in aggregate value. Any products from the collaboration will be developed and commercialized by Roche.

The collaboration comes on the heels of a recent $112 million Series B investmentin June for the biotech, founded in 2018 with RNA editing technology spun out of the lab of co-founderPrashant Mali, a bioengineer at the University of California, San Diego.

Vigneault was former VP of research at Juno Therapeutics, a Seattle cell therapy company acquired by Celgene in 2018 for $9 billion. Other Juno veterans include Shapes head of platform technologies Adrian Briggs and vice president and head of research David Huss.

The new collaboration may reflect growing drug company interest in neurosciences, which is on the upswing after a lull several years ago. The recent FDA approval of Biogens Alzheimers drug Aduhelm may also be propelling interest in the field.

This new collaboration is also perfectly aligned with our broader efforts across the Roche Group to unlock the full potential of gene therapy, said James Sabry, Head of Roche Pharma Partnering, in a statement.

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Seattle biotech firm Shape Therapeutics inks gene therapy deal with Roche worth up to $3B - GeekWire

Parkinsons disease is the second most common neurodegenerative disorder in the United States, affecting at least 500,000 people. Its a progressive disease, but can be managed with treatment. For now, the condition is diagnosed by symptoms rather than specific lab tests.

By the time a formal diagnosis is made, the National Institute of Neurological Disorders and Stroke (NINDS) notes that the disease has usually progressed to a point where people have difficulty controlling bodily movements.

Thats why its important to know whether its possible to prevent this disease.

Currently, there is no therapy or treatment that can slow the progression of Parkinsons or effectively relieve advanced symptoms, according to the NINDS.

By the time classic motor symptoms of Parkinsons disease show up, a significant loss of brain cells and function have already occurred. Scientists are investigating ways to detect early signs of the disease, to potentially stop or slow the progression.

Researchers aim to learn more about biomarkers of the early stages of the disease. Finding reliable biological identifiers might help doctors diagnose and treat Parkinsons earlier. Identifying these signs would give them more time to try therapeutic treatments before the disease has progressed.

For example, research indicates that it may be useful to study the activity of a neuronal protein in the brain known as -synuclein, or alpha-synyclein.

A 2019 study noted that measurements of -synuclein have shown encouraging preliminary results with regard to potential early diagnosis. Another 2019 study also examined how -synuclein accumulates in the brains of people suspected of having Parkinsons disease.This information could be used to develop therapies, perhaps antibody therapy, to prevent that accumulation from happening.

Scientists are also working to learn more about environmental factors and genetic factors that might contribute to the risk of developing Parkinsons. One recent genetic research breakthrough is the development of a DNA chip called NeuroX, which could potentially determine a persons risk, but more research is needed.

Parkinsons disease is the result of complicated combination of interconnected events, as one 2016 study described it. Since aging is the most common risk factor, future treatments may need to take degeneration of certain neurons into account.

While its not yet known if there are surefire ways to prevent Parkinsons disease, there are a few things experts recommend.

For example, you might try incorporating physical activity into your routine and eating a healthy and balanced diet for a variety of health reasons. So far, research into nutritional supplements is lacking. However, if you have specific dietary needs, talk to your doctor to see if supplementation is appropriate.

Could CBD oil help? Its possible, but we dont know for sure yet. Some research, including a 2018 review of studies, suggest that cannabidiol (CBD) might help prevent Parkinsons disease. However, the studies are mostly animal studies and there is not yet a body of research involving humans and CBD.

Some experts suggest that you may be able to delay some of the effects of Parkinsons disease through regular physical activity. Ideally this would include a combination of exercise that includes:

The NINDS has funded a number of studies to learn more about the impact of exercise, including whether exercise might help people delay the need for medication.

There are a variety of options for treating and managing Parkinsons symptoms, most of which involve medications that address the brains low levels of dopamine. Dopamine is a chemical in your brain that affects movement, and Parkinsons causes your brain to lose neurons that produce this chemical.

Medications that address this include levodopa, or levodopa combined with carbidopa. Or your doctor might prescribe a dopamine agonist, which mimics the action of dopamine in your brain. Other drugs used to treat Parkinsons include:

Another possible treatment option is deep brain stimulation (DBS). DBS was approved by the U.S. Food and Drug Administration in 1997. Many people have found that this treatment, which involves sending electrical impulses into the brain via tiny electrodes, helps control tremors once treatment with levodopa is no longer effective.

A small 2018 study found that DBS seemed to slow the progression of tremors in people with Parkinsons disease. It also found that DBS could be used effectively in people with an earlier disease stage than previously thought.

Scientists hope that more treatments may become available in the future, as they learn more about which drug may or may not be effective at slowing or halting the progression of the disease.

For example, a randomized, double-blind trial of 62 patients found that people with Parkinsons who took a drug usually used to treat diabetes seemed to stop the progression of the Parkinsons symptoms. They received weekly injections of exenatide for 48 weeks.

It was a relatively small study, and longer-term trials are needed, according to the researchers. A larger study involving more patients is currently ongoing.

If you are already living with Parkinsons disease, here are some tips to manage it:

There are medications that can help treat the symptoms of Parkinsons disease, and scientists are currently conducting research that could result in new treatment and therapies.

For example, you might one day have the option to take a medication used to treat prostate gland enlargement if youre at risk for developing Parkinsons disease.

The results of a study published in early 2021 suggest that certain medications often used to treat enlarged prostates are associated with a decreased risk of developing Parkinsons disease. Specifically, the researchers compared terazosin, doxazosin, and alfuzosin, which enhance energy metabolism, to tamsulosin, which is also used to treat benign enlarged prostates. They found that the latter did not seem to have the same effect.

The findings built on their previous research, which suggested that the use of terazosin, doxazosin, and alfuzosin was associated with slower progression and fewer complications in people with Parkinsons disease.

Researchers are also looking into the potential of stem cells to create new neurons to produce dopamine. They are also researching a protein called glial cell-derived neurotrophic factor, or GDNF, to potentially slow the progression of Parkinsons.

Ongoing research into a gene called LRRK2 or LARK2 and how it may interact with other genes related to Parkinsons disease is also promising, as it may shed light on how the disease progresses and how it might be halted.

For now, the symptoms of Parkinsons disease can be managed with medication and potentially deep brain stimulation. But research is underway to look for earlier methods of detection, as well as better treatments. Eventually, we might even have a way to prevent it from developing in the first place.

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How to Prevent Parkinson's Disease: Tips, Medications, and Research - Healthline

We sat down for an interview with Professor Botond Roska, MD PhD, one of the worlds leading experts in the study of vision and the retina and last years recipient of the Krber Prize, to discuss the ground-breaking therapy he and his team have developed and already successfully used to return some vision to a handful of blind patients.

Can you tell us about the exact nature of your research for which you received the Krber Award?

It is sometimes difficult to know exactly what you get an award for, but I think I received it for two distinct but related scientific pursuits: firstly, my research on understanding vision, and secondly, for developing a treatment called optogenetic vision restoration therapy, through which a completely blind person can be sensitized to light, allowing them to see again.

FactProfessor Botond Roska, MD PhD, is a biomedical researcher and one of the foremost experts on vision, the retina, and treating diseases that cause blindness. He is a founding director of the Institute for Molecular and Clinical Ophthalmology Basel (IOB) in Switzerland and a Professor at the University of Basel. In 2019, he was awarded the Order of Saint Stephen, the highest national honor bestowed by Hungary, as well as the Louis-Jeantet Prize for Medicine. In 2020, he won the Krber European Science Prize for his research on a gene therapy that could be utilized to restore some vision to the fully blind, which he successfully implemented with his team for the first time this May.

What exactly does this procedure you developed entail?

It is a so-called two-component therapy. We inject a gene therapy vector into the eye, which is a small, virus-like particle with a DNA that encodes a light-sensitive protein. We target this protein at some elements of the blind retina. The vector also has a goggle that records the world, and then projects a picture onto the retina in a very specific way, in a particular color.

How effective is it now, and how effective can it potentially become?

The therapy restores a certain amount of vision, but not full sight. Based on the few patients we have had so far, the first of whom we recently published a paper about, the therapy allows patients to recognize objects, but it does not enable them to read, for instance.

However, there is a lot of room to improve. This is only the very first step, the very first optogenetic therapy.

What kinds of blindness or visual impairment can it help or cure?

It is useful in cases when someone is fully blind as a result of photoreceptor dysfunction, but their retina is still connected to their brain via an intact optic nerve.

How profound an effect do you think being able to heal peoples vision at a large scale might have on society?

Blindness is one of the conditions that people name as the worst affliction to have. In fact, in a recent survey conducted in the U.S., participants named it as the worst condition they could suffer from out of a whole host of common health problems, ranking it above cancer, Alzheimers, and other truly devastating diseases.

Our whole life is spent looking at phones and computers. Particularly during the pandemic, we could have almost no social interactions or opportunities to work without these, so that blind people were essentially cut off from the world apart from what little interaction they could have with it through hearing.

Therefore, we hope that such therapies, once they become widely available since they are in the clinical trial phase at the moment will improve many peoples lives tremendously.

Where do you think your research might lead you in the future?

There are three main directions. The first one is simple; as we discussed, we need to keep researching and improving optogenetic vision therapies, expanding them to target other cell types in the retina.

The second one is to do with the fact that if the optic nerve is missing, we cannot provide any therapies at the moment. This is something we are working on at my institute together with researcher Dniel Hillier, who is leading these efforts. We aim to find ways of restoring vision when there is no optic nerve.

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Hungarian-born researcher Dr. Botond Roska has been awarded the 2019 Louis-Jeantet Prize for Medicine for the discovery of basic principles of visual information processing and the development of therapeutic strategies, such as gene therapy, to restore vision in retinal disorders. Within the last few months, the neurobiologist (who celebrates his 50th birthday this year) received []Continue reading

The third is that most visual impairment is partial blindness, in which the method we developed cannot be used, so we are interested in looking into very large diseases affecting a lot more people. We want to try to slow down the degeneration or restore more vision to those with partial sight loss.

Why did you choose to dedicate your career to understanding human vision at the hardware level?

It was a chain of random events. I did not plan to be a vision researcher. I just wanted to understand things. When I finished my medical school, I was quite sure I did not want to treat patients for a living, I was more interested in understanding the human body. I decided to go into research, and I met someone who was researching the retina. I started my research in this field, and came up against an increasing number of questions that I found fascinating.

I went from topic to topic, physiology, virology, the molecular biology of the eye, then towards the end of my graduate studies, I read a paper that said it was possible to make cells light-sensitive using molecules form other organisms. At that time, I understood the retina quite well, and thought that I could combine my knowledge with these findings to try to design a therapy. My lab and myself are both interested in gaining scientific insight as well as designing therapies, and that is the path I plan to continue on for the rest of my career.

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The findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible, Botond Roska said.Continue reading

What is the worst and best part of the publicity youve received as a result of your success?

The best part is certainly that blind people can become informed that we are working on a therapy, which can provide them with some hope. It is also important for my institute, which can more easily recruit brilliant scientific minds who will come up with even better therapies down the line.

On the other hand, while there is nothing really bad about publicity per se, it can sometimes be time-consuming. Our paper about our first patient came out in May, and it has been downloaded around 90,000 times, with just about every country in the world reporting on our findings. And of course, all of the press outlets in all these countries bombarded our inboxes with requests. We were basically paralyzed for almost a month.

As someone who is called upon with increasing frequency to explain your cutting edge research, how do you approach the issue of science communication?

Communication is very important. It is part of our lives. In Switzerland specifically, it is taken very seriously, and it is part of our jobs as researchers to explain things to the public. We also have professionals to help us navigate the world of public relations.

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President Jnos der decorated neurobiologist Botond Roska with the Hungarian Saint Stephen Order on Tuesday, Hungarys national holiday. Hungarian-born Researcher Awarded for Helping People Regain Their Sight Each year on the holiday of the founder of our state we celebrate those Hungarians whose outstanding talent and achievements serve the well-being and prosperity of many others, []Continue reading

The difficulty today is finding the balance between our work and its communication to the world. Because our research reaches a tremendous number of people extremely quickly thanks to the global nature of social media and other platforms, we get a lot of requests for interviews and articles. The difficulty for us is balancing our responsibility to inform the public of our progress with actually making some. Sometimes, it feels very much like we are in the eye of the storm.

Many people consider you a likely future Nobel laureate. Do you believe you might receive the award?

I dont think about any of that. It does not occupy any of my time or of my imagination. At this moment, there are still great challenges ahead of us if we want to make the therapies as good as they can be. That is what I concentrate on. We also need to focus on innovating and coming up with new tools to help realize our scientific vision. This is also a costly and time-consuming endeavor.

What would you tell aspiring scientists, how can they best succeed in their scientific endeavors?

I think that the key is to come to science with an incredible desire to solve interesting problems. I never wanted to be successful in the public eye, just accomplished at solving scientific questions. For some reason, some scientists are more present in the media, and some of them win prizes, while others win prizes and choose not to have a public presence. Often, the most brilliant and prolific scientists do not even win prizes.

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Returning Vision to The Fully Blind - Interview with Dr. Botond Roska - Hungary Today

Ryder Comer was born with SMA, the best treatment option was an infusion that cost $2.1 million. Now, his mom is working to add SMA to Idaho's newborn screening.

MIDDLETON, Idaho When Jake and Haley Comer of Middleton welcomed their new baby boy Ryder into the world, they were completely overjoyed. But, they quickly realized something might not be right.

"The whole pregnancy was completely healthy, there were no signs of anything." said Haley Comer, Ryder's proud mom. "As soon as he was born though, he was diagnosed with low muscle tone. No parent wants to think that something is wrong with their perfect little baby."

The Comers went to their pediatrician to get some answers. The doctor agreed that newborn Ryder was "floppy" and had low muscle tone. He recommended genetic testing to see if there was a reason for that. While waiting for the results in the following weeks, the unthinkable happened. Ryder stopped breathing.

"The day after Ryder turned a month old, I found him unresponsive at home, I had him in a little baby carrier. So, I called 911 and started CPR and luckily was able to revive him. We were rushed to the ER, and that initiated a month-long stay in the hospital," said Haley Comer with tears in her eyes.

While they were at St. Luke's, the genetic testing results came back. The news was not what the Comers were hoping for.

"We got our test results back and Ryder was diagnosed with SMA, Spinal Muscular Atrophy Type 1," said Haley. "It was hard, there were a lot of tears, but at the same time we want to be brave. You have to put on a brave face for your baby."

SMA is a progressive, rare genetic disease that impacts the muscles. Babies with SMA Type 1 face many physical challenges, including muscle weakness and trouble breathing, coughing, and swallowing.

Doctors at St. Luke's in Boise told the Comers about a promising new treatment option. A gene therapy infusion that could save baby Ryder's life.

"They told us that they would be transferring him to Salt Lake City to Primary Children's Hospital, because no hospitals in Idaho performed this infusion," said Haley. "A couple days later, we met with a neurologist in Salt Lake and learned about our treatment options. This gene therapy option seemed like the best one. It's a once in his lifetime, one-hour long, gene therapy infusion. He is missing a very important gene that produces survival motor neuron protein. That helps our body move our muscles. It basically injects this gene into his body, and then his body learns to keep producing this survival neuron protein to keep his muscles alive."

#7sHero Tonight at 10 p.m. on KTVB youll meet this precious baby boy, Ryder Comer of Middleton. His parents Jake &...

It sounded like the best option for Ryder, but the Comers were stunned when they found out just how much the infusion treatment would cost.

"It's called Zolgensma, and it costs $2.1 million for one single infusion," said Haley.

Zolgensma is FDA-approved for patients with all forms and types of SMA who are under 2 years of age. Research shows babies dosed with Zolgensma as soon after diagnosis as possible have had better results than those who waited to begin treatment. It's given through an IV infusion that takes about an hour, and it's a one-time treatment.

The Comers weren't even sure their insurance would cover it, but they moved forward with trying to get the pricey infusion procedure preauthorized.

"It was terrifying when they told us," said Jake Comer, Ryder's dad. "I was like I hope you take monthly payments for the rest of my life."

Incredibly, the approval from their insurance company, United Healthcare, went smoothly and quickly.

"We paid our deductible, and our out-of-pocket, and they took care of the rest," said Jake in disbelief. "That definitely took the stress of our shoulders during such a traumatic time, so we are very grateful."

The Comers say it was all a whirlwind. Ryder received the infusion within 24 hours.

"We feel pretty fortunate because a lot of families had to fight for weeks and months to get their insurance to cover this," said Haley. "For Ryder, his disease had progressed so much by the time he got it, he was at the point where every day mattered."

After the $2.1 million infusion, Ryder started making strides within days. The changes they've seen over the past several months have been so wonderful for the Comers.

"His breathing got stronger, his voice got stronger, his cry got louder, he had a really weak cry at first," said Haley. "Now he can lift his arms above his head and grab toys! He can't quite hold his head up yet, but we are working on it. He is in feeding and physical therapy, and we are doing everything we can to get him stronger."

So far, Ryder is the only baby in Idaho with SMA to receive this gene therapy infusion. Haley has a new passion. She is now dedicated to helping other SMA families like theirs.

"What we are really working toward right now is to have SMA added to Idaho's newborn screening. Idaho is one of the last remaining states that does not include SMA on its newborn screening test. It's just a simple blood test. Before the approval of his gene therapy two years ago, the life expectancy for babies diagnosed with SMA Type 1 was not much past two years old. That is just devastating for families. So having SMA added to Idaho's newborn screening will allow these babies to have access to treatment so much sooner. The sooner you get a diagnosis and have treatment, the better outcomes you will have in life."

Haley has been in contact with the Idaho Department of Health and Welfare, and with the organization CURE SMA, which has been working to add this disease to every state's newborn screening. She's in the early stages, but this mom's goal is to make that happen here in Idaho, too.

"We just think that every baby deserves to be treated as soon as they possibly can," said Haley.

August is SMA Awareness Month. Baby Ryder and his parents are paving the way for other Idaho babies born with SMA.

"He's expected to get stronger and hopefully meet those milestones of walking and talking and crawling and all those things, but we just don't know, we are working hard in therapy to give him the best possible chance. One day a time."

For more information on SMA, click here. If you'd like to help the Comer family with their medical expenses, there is a GO FUND ME set up to do so.

See all of the heartwarming segments in ourYouTube playlisthere:

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Ryder's Story: Middleton baby with rare genetic condition gets a $2.1 million infusion to give him a fighting chance - KTVB.com

NEW YORK An international team of researchers has used transcriptomic data from ulcerative colitispatients to develop a predicted polygenic transcriptional risk score, or PPTRS,that can identify UC-affected individuals at fivefold elevated risk of progressing to surgical resection of the large bowel.

In a paper published on Thursday in the American Journal of Human Genetics, the Georgia Institute of Technology-led team noted that 5 percent to 10 percent of people with UC require bowel resection, or colectomy, within five years of diagnosis, but that polygenic risk scores based on genome-wide association studies generally don't provide meaningful prediction of progression to surgery. However, studies of Crohn's disease have shown that gene expression profiling of GWAS-significant genes provides some stratification of risk of progression to complicated disease through transcriptional risk scoring, or TRS.

In their paper, the researchers demonstrated that a measured TRS based on bulk rectal gene expression in a cohort of UC patients had a positive predictive value approaching 50 percent for colectomy. Single-cell profiling demonstrated that the disease-associated genes were active in multiple diverse cell types from both the epithelial and immune compartments, and expression quantitative trait locusanalysis identified genes with differential effects at baseline and the one-year follow-up, the researchers said. But for the most part, they found that differential expression associated with colectomy risk was independent of local genetic regulation.

Overall, their data suggested that prediction of gene expression from relatively small transcriptome datasets can be used in conjunction with transcriptome-wide association studies for stratification of risk of disease complications.

The researchers began by performing differential expression analysis between baseline rectal RNA-seq biopsies of individuals in the PROTECT multicenter pediatric inception cohort study of response to standardized colitis therapy. Analyses were done on 21 affected individuals who progressed to colectomy and 310 who did not. They identified downregulation of 783 transcripts in the individuals who underwent colectomy and upregulation of 1,405 transcripts overall.

They also obtained rectal biopsy RNA-seq data for 92 affected individuals at week 52 and observed a marked shift in gene expression at follow-up, prompting them to ask whether local regulation of the gene expression might contribute to this effect. They found that there were 72 SNPs that were significantly regulating 308 genes at both time points.

Further examination of the expression of colectomy-associated genes in a single-cell RNA-seq dataset obtained from rectal biopsies provided strong evidence that both epithelial and immune cells contributed to the risk of disease progression, the researchers said.

The researchers then performed a TWAS to capture the effects of all polymorphisms within 1 Mb of each transcript expressed in the PROTECT rectal biopsies and then used the weights to predict gene expression in a validation cohort from the UK Biobank. They tested for differential predicted gene expression in 70 percent of the validation samples and discovered about 800 genes either upregulated or downregulated in UC-affected individuals relative to non-IBD control individuals. They then derived a PPTRS for UC based on the effect sizes of the minor alleles and applied it to the remaining 30 percent of the validation samples, as well as to the PROTECT genotypes, and found that the PPTRS efficiently discriminated UC-affected individuals from non-IBD control individuals.

Significantly, it also discriminated the individuals who underwent colectomy versus those who didn't in both the UK Biobank and PROTECT.

"More extensive single-cell profiling, combined with cell-type-specific genetic analysis of gene expression, is likely to lead to the development of even better transcriptional risk signatures," the authors concluded. "It is also likely that such focused and personalized analysis may highlight specific pathological mechanisms active in particular affected individuals."

They did note, however, that these results were limited by the relatively small sample size of colectomies in the PROTECT study, and that validation of cross-ancestry assessments and the evaluation of the consistency of gene expression prediction across populations should be a high priority.

In an email, corresponding author and GIT researcher Greg Gibson noted that while the study's multiple layers of replication show that transcriptional profiling of the rectum greatly enhances risk stratification for risk of colectomy, this was not a clinical trial, so the approach is not yet approved for evaluation of patients.

"We hope that it will progress to implementation in the near future," he added."The prediction from genotypes alone is less likely to have clinical utility since the precision is still quite low, so that aspect is more research oriented."

He further noted that the approach he and his colleagues used could also be applied to a wide range of diseases, and that they are pursuing that research.

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Ulcerative Colitis Study Analyzes Gene Expression to Measure Risk of Progression to Surgery - GenomeWeb

Brain development that occurs after birth is also important. Rebecca Saxe at MIT is working to understand the brain structures and activities responsible for social cognition, which allows us to consider the mental states of other people.

Saxe has discovered a particular brain region that is key; by studying how activity in this region and others changes over the course of childhood, she may be able to understand how social abilities develop. She has also found that these brain activity patterns are altered in people with autism spectrum disorders.

Even though researchers are starting to understand some of the processes that govern development and have identified things that can derail it, were far from being able to intervene when such problems occur. But as we gain insights, we could someday test therapies or other ways to address these developmental issues.

Computational neuroscientists use mathematical models to better understand how networks of brain cells help us interpret what we see and hear, integrate new information, create and store memories, and make decisions.

Understanding how the activity of neurons governs cognition and behavior could lead to ways to improve memory or understand disease processes.

Terry Sejnowski, a computational neurobiologist at the Salk Institute, has built a computer model of the prefrontal cortex and analyzed its performance on a task in which a person (or machine) has to sort cards according to a rule thats always changing. While humans are great at adapting, machines generally struggle. But Sejnowskis computer, which imitates information flow patterns observed in the brain, performed well on this task. This research could help machines think more like humans and adapt more quickly to new conditions.

Aude Oliva, the MIT director of the MIT-IBM Watson AI Lab, uses computational tools to model and predict how brains perceive and remember visual information. Her research shows that different images result in certain patterns of activity both in the monkey cortex and in neural network models, and that these patterns predict how memorable a certain image will be.

Research like Sejnowskis may inspire smarter machines, but it could also help us understand disorders in which the function of the prefrontal cortex is altered, including schizophrenia, dementia, and the effects of head trauma.

Researchers are trying to determine the genetic and environmental risk factors for neurodegenerative diseases, as well as the diseases underlying mechanisms.

NHUNG LE

Improving prevention, early detection, and treatment for diseases like Alzheimers, Parkinsons, Huntingtons, chronic traumatic encephalopathy, and ALS would benefit millions of people around the world.

Yakeel Quiroz, at Massachusetts General Hospital, studies changes in brain structure and function that occur before the onset of Alzheimers symptoms. Shes looking for biomarkers that could be used for early detection of the disease and trying to pinpoint potential targets for therapeutics. One potential biomarker of early-onset Alzheimers that shes founda protein called NfLis elevated in the blood more than two decades before symptoms appear. Quiroz has also identified a woman with a protective genetic mutation that kept her from developing cognitive impairments and brain degeneration even though her brain showed high levels of amyloid, a protein implicated in Alzheimers development. Studying the effects of this beneficial mutation could lead to new therapies.

Researchers at the Early Detection of Neurodegenerative Diseases initiative in the United Kingdom are analyzing whether digital data collected by smartphones or wearables could give early warnings of disease before symptoms develop. One of the initiatives projectsa partnership with Boston Universitywill collect data using apps, activity tracking, and sleep tracking in people with and without dementia to identify possible digital signatures of disease.

As we learn more about the underlying causes of neurodegenerative diseases, researchers are trying to translate this knowledge into effective treatments. Advanced clinical trials targeting newly understood mechanisms of disease are currently under way for many neurodegenerative disorders, including Alzheimers, Parkinsons, and ALS.

Connectomics researchers map and analyze neuronal connections, creating a wiring diagram for the brain.

Understanding these connections will shed light on how the brain functions; many projects are exploring how macro-scale connections are altered during development, aging, or disease.

Mapping these connections isnt easythere may be as many as 100 trillion connections in the human brain, and theyre all tiny. Researchers need to find the best ways to label specific neurons and track the connections they make to other neurons in remote parts of the brain, refine the technology to collect these images, and figure out how to analyze the mountains of data that this process produces.

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Eight ways scientists are unwrapping the mysteries of the human brain - MIT Technology Review

State College, Pennsylvania., August 30, 2021 / PRNewswire / -NeuExcell Therapeutics (www.neuexcell.com), A gene therapy company focused on neurodegenerative diseases has announced a Series Pre-A funding round of over $ 10 million. The round was led by Co-Win Ventures and was attended by other institutional investors Yuan Bio, Oriza Seed, Tsingyuan and Inno Angel.

We are honored to join this very reputable group of investors, he said. Peter Tombros, Chairman of the Board of Directors of NeuExcell Therapeutics. Investor experience and support will enable us to leverage our unique neuroregenerative gene therapy platform across multiple neurodegenerative indications. This funding strength validates our strategy and biotechnology. Further examine our science in the industry.

Professor Gong Chen, co-founder and chief scientific advisor of the company, said: There is an urgent need for breakthrough therapies like us.

I think this is a great opportunity to invest in experienced leadership, he said. Xin Huang, Managing Partner of Co-Win Ventures. NeuExcells unique technology has the potential to act as a platform for treating many neurodegenerative diseases, providing hope for breakthrough new therapies for patients who do not have the right choices today.

With the end of this successful pre-A round, we welcome him. Xin Huang Jonathan Sun attended the board meeting.

About NeuExcellTherapeutics

NeuExcell is a privately held early stage genetic engineering company headquartered in Pennsylvania, USA When Shanghai, China.. Its mission is to improve the lives of patients suffering from neurodegenerative diseases and damage to the central nervous system. Based on Professor Gong Chens scientific research, we have developed a potentially destructive nerve repair technique through the conversion of astrocytes to neurons. In vivo By introducing neural transcription factors through adeno-associated virus (AAV) -based gene therapy. NeuExcells pipeline covers major neurodegenerative diseases such as stroke, Huntingtons disease, amyotrophic lateral sclerosis (ALS), Alzheimers disease, Parkinsons disease, traumatic brain injury, spinal cord injury, and glioma. increase.

About Co-Wof Venture

Founded in 2009, Co-Win Ventures is an early stage investor in healthcare and TMT with a focus on equality, transparency, sharing and innovation. Co-Wins business network China When USA..Total AUM is about US $ 1 billion, Co-Win aims to be a reliable partner for great entrepreneurs to build breakthrough technologies and businesses. Co-Win Ventures has helped more than 140 portfolio companies, including leading leaders in their respective sub-sectors, including Cytek, Connect, Thrive (acquired by Nasdaq-listed company EXAS), Taimei Technology, Genecast, Sinovation and Augta. ..

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NeuExcell Therapeutics Raises Over $ 10 Million Series Before Round To Keep The Company Growing | Around The Web-Pennsylvania

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NeuExcell Therapeutics Raises Over $ 10 Million Series Before Round To Keep The Company Growing | Around The Web-Pennsylvania - Pennsylvania News...

SAN FRANCISCO--(BUSINESS WIRE)--AllStripes (Company), a healthcare technology company dedicated to unlocking treatments for people affected by rare diseases, today announced the Company completed a $50 million Series B financing round.

The financing was led by Lux Capital, a current investor, joined by JAZZ Venture Partners, Spark Capital, Medidata Solutions (a Dassault Systmes company), McKesson Ventures, and Maveron, along with angel investors including Arif Nathoo, CEO of Komodo Health, and Leila Zegna, Director of the Kabuki Syndrome Foundation.

The funding will support launching 100 new rare disease research programs while expanding global operational footprint, technology and data automation enhancements to improve research insights, further developing the platform capabilities to enhance the user experience and strengthen the life sciences offerings, and continuing to invest in growing the companys team to support creating the playbook for rare disease research.

Beginning research on a rare condition can feel like being dropped into a new world without a map and we are on a mission to change that with data, said Nancy Yu, CEO and Co-founder, AllStripes. Were proud that so many of our current investors have recognized the progress weve made and are continuing to support our vision to transform rare disease research. This investment will allow us to better support the rare disease community, where each persons experience is essential to understanding disease progression - ultimately leading to new treatments for rare disease patients around the globe.

AllStripes has a successful track record of bringing together patient organizations, families, experts and life science partners to advance research, said Adam Goulburn, Partner, Lux Capital and AllStripes board member. If we are going to improve rare disease treatments, these groups must work together. We believe in AllStripes vision to transform rare disease research and empower patients. If the past year showed us anything, it is that patients want a voice when it comes to their health, and their engagement with research is invaluable.

There are more than 7,000 rare diseases and only 5 percent have treatments. By working with AllStripes, we hope to improve the number of treatments available by accelerating research for rare diseases, said James M. Wilson, M.D., Ph.D., Rose H. Weiss Professor and Director, Orphan Disease Center; Director, Gene Therapy Program at the University of Pennsylvania; Professor in Departments of Medicine and Pediatrics, Perelman School of Medicine.

AllStripes is currently partnering with more than 30 patient advocacy organizations across its 40 conditions, supporting more than 3,000 users to date. As a public benefit corporation (PBC), AllStripes is advocating for the importance of real-world evidence in development of treatments and is continuing to build tools that make research more inclusive for the global rare disease community. The Company is committed to transparent data collection and sharing data across the life sciences continuum to advance clinical research.

AllStripes collaborates with various biopharmaceutical companies and other entities on real-world evidence studies, including: HemoShear Therapeutics, Inc., Orphan Disease Center at the University of Pennsylvania, Taysha Gene Therapies, Inc., UCB Biopharma SRL, and Novartis Pharma AG. These joint research programs aim to enhance clinical understanding of rare disease treatment research. AllStripes and the University of Pennsylvania's Orphan Disease Center are focused on clinical understanding of Lesch-Nyhan disease and Crigler Najjar syndrome type 1, with the goal of facilitating therapeutic discovery for both conditions. One of AllStripes most recent partnerships with HemoShear Therapeutics aims to gather real-world evidence on patients' medical experience with serious metabolic diseases through the Journey to Understand MMA and PA (JUMP) study.

To learn more about AllStripes, its partners and its commitment to the rare disease community, please visit allstripes.com.

About AllStripesAllStripes is a healthcare technology company dedicated to unlocking new treatments for people with rare diseases. AllStripes has developed a technology platform that generates regulatory-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, JAZZ Venture Partners, Spark Capital, Medidata Solutions, McKesson Ventures, Maveron, and a number of angel investors. For more information, visit http://www.allstripes.com.

About Lux CapitalLux Capital invests in emerging science and technology ventures at the outermost edges of what is possible. We partner with iconoclastic inventors challenging the status quo and the laws of nature to bring their futuristic ideas to life. Over the past two decades, Lux has expanded from its New York City roots to Silicon Valley, and built a $4 billion AUM firm of more than 30 full-time professionals, with the versatility to invest at any stage.

JAZZ Venture PartnersJAZZ Venture Partners invests in companies that extend the boundaries of human performance improving how we live, learn, work, play, and experience the world. JAZZ seeks breakthroughs at the frontiers of technology and science, such as advances in artificial intelligence, neurobiology, augmented reality, and closed-loop human-computer systems. JAZZ portfolio companies are unlocking human potential in health, mind-body wellness, accelerated learning and training, sports, entertainment, and the enterprise. More information about JAZZ can be found at http://www.jazzvp.com.

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AllStripes Announces $50 Million Series B Financing to Advance Global Rare Disease Research - Business Wire

VANCOUVER, British Columbia & BASEL, Switzerland--(BUSINESS WIRE)--Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, today announced that it has entered into a global collaboration and license agreement with Takeda Pharmaceutical Company Limited (Takeda) for the development and commercialization of novel nonviral gene therapies to treat up to two undisclosed rare liver diseases. This is the second collaboration between Genevant and Takeda, following an earlier 2021 agreement to develop nucleic acid therapeutics directed to specified targets in hepatic stellate cells to treat liver fibrosis.

LNP provides a compelling approach to deliver on the promise of gene therapy, and our leadership position in the LNP space is well established. We have enjoyed working with our Takeda colleagues to develop hepatic stellate cell-directed treatments to treat liver fibrosis and are delighted to expand the relationship further with this second collaboration, said Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation.

Building on our existing foundation with Genevant in liver fibrosis, were excited to expand our work together to develop life-altering, nonviral gene therapies for specified rare liver diseases, said Bernard Allan, head of liver disease research at Takeda. Genevants expertise in the development of LNPs for clinical applications, coupled with Takedas drug development capabilities and history in gastroenterology, gives us a great opportunity to develop new treatment options for patients with liver disorders.

Under the terms of the agreement, Genevant is eligible to receive up to $303 million in upfront and potential milestone payments, plus royalties on future product sales. Takeda has exclusive rights to utilize Genevants LNP technology in the development and commercialization of specified nonviral gene therapies for up to two undisclosed rare liver diseases.

About Genevant Sciences

Genevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, and decades of experience and expertise in nucleic acid drug delivery and development. The Companys scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Companys LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. For more information, please visit http://www.genevant.com.

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Genevant Sciences Announces Global Collaboration and License Agreement with Takeda to Develop Novel Nonviral Gene Therapies for Up to Two Rare Liver...

Using cells from brain tumour patients as a 3D printing material, researchers have made a model of their tumour to test the efficacy of potential treatments before using them for real inside the body. "A chunk" of the tumour from a glioblastoma patient is used to print a model matching their MRI scans. The patient's blood is then pumped through the printed tumour, made with a compound that mimics the brain, followed by a drug or therapeutic treatment. While previous research has used such "bioprinting" to simulate cancer environments, the Tel Aviv University scientists involved in this research say they are the first to print a "viable" tumour.

Medulloblastoma, whilst being a common paediatric malignant central nervous system tumour, represent a small proportion of brain tumours in adults. This research reports on theunique subgroup-specific cytogenetic features of adult medulloblastoma, which are distinct from those in younger patients, and correlate with survival disparities. The findings suggest that clinical trials that incorporate new strategies tailored to high-risk adult medulloblastoma patients are urgently needed. These findings complement theimportant progress being made on medulloblastoma by researchers at our Research Centre at QMUL.

Chronobiology is a promising area for research in our area as circadian clock genes are linked to tumour biology and outcomes in multiple cancers, including glioma. In this research, the relationship between circadian clock genes, IDH mutational status, and prognosis in glioma patients was examined to try and gain further understanding into theassociation between circadian clock gene expression, the glioma microenvironment and patient survival.

Researchers in Brazil have discovered a set of biomarkers that can be used to predict which patients diagnosed with glioblastoma may have tumours that are more resistant to radiation therapy. This discovery could help doctors choose the treatment that should enable patients to live longer. Glioblastoma usually has to be surgically removed, followed by chemotherapy or radiation therapy, or both, to eliminate what could not be surgically removed and ensure the tumour does not return. However, patient survival tends to be low owing to the tumours resistance to treatment. According to most estimates, 80% of patients submitted to radiation therapy suffer a relapse a few months later. These researchers claim that their study provides important information for use in screening patients prior to deciding whether radiation therapy should be used. Its a large signature. We identified 31 genes that may indicate resistance or sensitivity to radiation when altered. So, its possible to verify whether there are tumour cells that are highly resistant to radiation, potentially assisting physicians in deciding on the optimal treatment.

Some facts and figures from the American Cancer Society in a new report illustrating thatalthough incidence rates of adult brain tumours are decreasing 5-year survival rates remain low.

There are over 120 different types of brain tumours and although distinguishing between them can be difficult it is nevertheless essential for effective treatment. This year, the Gertrud Reemtsma Foundation isawarding the International Prize for Translational Neuroscience to two researchers who have made a significant contribution to improving the diagnosis of brain tumours. Hai Yan from Duke University School of Medicine was instrumental in identifying two tumour proteins that are typical for certain sub-groups of gliomas. Andreas von Deimling and his group from the University of Heidelberg have developed antibodies that bind to these altered proteins. The tumours can now be classified at the molecular level. A patients cancer can thus be classified more precisely and the therapy adapted accordingly.

There wont be a research update next week but it will return on 10th September.

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3D printed tumours, adult medulloblastoma and the circadian clock - Brain Tumour Research

The latest published report namely Cancer Gene Therapy Market Growth 2021-2028 added by DBMR offers an insightful take on the historical data of the market and predictions for 2021 to 2028 time-period. The report provides an understanding of the Cancer Gene Therapy industry competitors, the sales channel, growth potential, potentially disruptive trends, industry product innovations and the value/volume of size, market segments, and market share of the best actors/products. Current market trends and dynamics are assessed which helps in mapping the track of the global market. A chapter-wise format has been used to ease the readability and complexity of the data. Each chapter is further categorized into its respective segments containing well-structured data.

According to DBMR, the GlobalCancer Gene Therapy Marketis expected to see a growth rate of 32.54%. The high success rate of cancer gene therapy along with clinical trial and the preclinical trial is gaining popularity among the patient which is leading towards the market.

Download Sample (350 Pages PDF) Report: To Know the Impact of COVID-19 on this Industry@https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-cancer-gene-therapy-market

Market Overview of Global Cancer Gene Therapy

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period. High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

The topic of market segmentation covers research and analysis based on application, vertical, deployment model, end user, and geography. In addition, competitive analysis assists to get ideas about the strategies of key players in the market via an international Cancer Gene Therapy report. Some of these strategies can be named as; new product launches, expansions, agreements, partnerships, joint ventures, acquisitions, and others that help to broaden their footprints in the Cancer Gene Therapy industry. The market share of key competitors on global level is studied where main regions such as Europe, North America, Asia Pacific and South America are covered in the reliable Cancer Gene Therapy market report.

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The titled segments and sub-section of the market are illuminated below

By Therapy (Oncolytic Virotherapy, Gene Induced Immunotherapy, Gene Transfer)

By End User (Hospitals, Oncology Institutes, Biotechnological Companies, Clinical Research Laboratories)

Top Key Manufactures or Players (this may not be a complete list and extra companies can be added upon request):

Adaptimmune

GlaxoSmithKline plc

bluebird bio, Inc

Merck & Co., Inc

CELGENE CORPORATION

Anchiano Therapeutics

Achieve Life Sciences

..

Complete Report is Available (Including Full TOC, List of Tables & Figures, Graphs, and Chart)@https://www.databridgemarketresearch.com/toc/?dbmr=global-cancer-gene-therapy-market

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Based on therapy, the cancer gene therapy market is segmented into oncolytic virotherapy, gene induced immunotherapy and gene transfer. The oncolytic virotherapy is further sub-segmented into adenovirus, lentivirus, retro virus, adeno associated virus, herpes simplex virus, alpha virus, vaccinia virus, simian virus and others. The gene induced immunotherapy is further sub-segmented into delivery of cytokines gene and delivery of tumor antigen gene. The gene transfer is further sub-segmented into naked/plasmid vectors, electroporation, sonoporation, magnetofection and gene gun.

Cancer gene therapy market has also been segmented based on the end user into hospitals, oncology institutes, biotechnological companies and clinical research laboratories.

According to the Regional Segmentation the Cancer Gene Therapy Market provides the Information covers following regions:

North America

South America

Asia & Pacific

Europe

MEA (Middle East and Africa)

The key countries in each region are taken into consideration as well, such as United States, Canada, Mexico, Brazil, Argentina, Colombia, Chile, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Belgium, Austria, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, China, Japan, Taiwan, South Korea, Singapore, India, Australia and New Zealand etc.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

For More Information or Query or Customization Before Buying, Visit@https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-cancer-gene-therapy-market

Scope of Report:

The Market report lists the most important competitors and provides the insights strategic industry Analysis of the key factors influencing the market. This report will help you to establish a landscape of industrial development and characteristics of the Cancer Gene Therapy market. The Global Cancer Gene Therapy market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, price, cost, revenue and gross margins.

Important Features that are under offering & key highlights of the report:

Market Data Segmentation with production, consumption, revenue (million USD), and Price Analysis

Detailed overview of Cancer Gene Therapy market

Changing market dynamics of the industry and Impact of Influencing Factors

In-depth market segmentation by Type, Application and other major segments etc.

To analyse and forecast the Global Cancer Gene Therapy market, in terms of value and volume.

Which segment has the potential to gain the highest market share?

To help decision maker from new offer perspective and benchmark existing marketing strategy.

Correlate cost structure historical data with key business segments.

Analyse marketing contribution and customer acquisition by up-selling and cross selling.

Identifying Influencing factors keeping Global Cancer Gene Therapy Market Intense, factored with periodic analysis of CR4 & CR8 concentration ratio & HHI Index.

Order a Copy of Cancer Gene Therapy Market Report@https://www.databridgemarketresearch.com/checkout/buy/singleuser/global-cancer-gene-therapy-market

With tables and figures helping analyze worldwide Global Cancer Gene Therapy market, this research provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

Major Highlights of TOC:

Chapter One: Market Overview

1.1. Introduction

1.2. Scope/Objective of the Study

Chapter Two: Executive Summary

2.1. Introduction

Chapter Three: Market Dynamics

3.1. Introduction

3.2. Market Drivers, Trends, Challenges, Opportunities

Chapter Four: Market Factor Analysis

4.1. Porters Five Forces

4.2. Supply/Value Chain

4.3. PESTEL analysis

4.4. Market Entropy

4.5. Impact Analysis Post COVID-19

Continued !!!

Chapter Nine: Methodology and Data Source

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

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Cancer Gene Therapy Market 2021 Global Industry Size, Share, Growth, COVID-19 Impact Analysis, and Drivers Analysis Research Report 2027|...

The latest Cell and Gene Therapy Market report serves as a valuable resource for data concerning the industrys trajectory over the forecast duration. It critically examines the key growth stimulants, bottlenecks, and rewarding prospects that are influencing the industry dynamics. Moreover, the literature consists of detailed segmentation data, as well as in-depth profiles of key companies in this domain.

Experts analysts state that Cell and Gene Therapy market size is anticipated to amass notable gains, accumulating a valuation of XX USD over the projected timeframe 2021-2026. In addition, projections for the various sub-markets are also validated in the report. Proceeding further, the research literature is well equipped with different methodologies to assist the stakeholders in seamlessly navigating any form of business crisis.

Key highlights from the Cell and Gene Therapy market report:

Request Sample Copy of this Report @ https://www.nwdiamondnotes.com/request-sample/3811

Cell and Gene Therapy market segments covered in the report:

Product gamut: Rare Diseases , Oncology , Hematology , Cardiovascular , Ophthalmology , Neurology and Other Therapeutic Classes

Application spectrum: Pharmaceutical and Biotechnology Companies , Research and Academic Institutions , Contract Research Organizations (CROs) , Hospital and Others

Competitive dashboard:

Regional bifurcation:

Key Questions Addressed by the Report

What Are the Key Opportunities in Global Cell and Gene Therapy Market?

What Will Be the Growth Rate from 2021 to 2026?

What Are the Factors That Will Impact/Drive the Market?

Which Segment/Region Will Have Highest Growth?

What is the Role of Key Players in the Value Chain?

What is the Competitive Landscape in the Industry?

Request Customization on This Report @ https://www.nwdiamondnotes.com/request-for-customization/3811

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Cell and Gene Therapy Market Forecasts to 2026: Global Industry Growth, Share, Size, Trends and Segmentation Report - Northwest Diamond Notes

INTRODUCTION Given their ability to treat a wide range of clinical conditions, advanced therapy medicinal products, including cell and gene therapies, have revolutionized the delivery of healthcare.

New York, Aug. 24, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell Therapy Packaging Products and Services Market by Type of Therapy, Package Engineering Design, Scale of Operation and Geography : Industry Trends and Global Forecasts, 2021-2030" - https://www.reportlinker.com/p06130493/?utm_source=GNW Presently, more than 30 cell-based therapy products have received marketing approvals across various geographies, while more than 1,000 therapeutic candidates are under clinical evaluation. Considering the lucrative investment opportunity within this domain, several investors are actively supporting ongoing / future product development programs in this upcoming field of pharmacological interventions. In fact, there has been a 240% increase in investment activity, in terms of capital amount invested, between 2019 and 2020. However, the ultimate success of cell therapies is dependent on the safe and timely delivery of viable doses of therapeutic cells to the right patient. In this context, it is worth highlighting that drug product manufacturing, packaging and logistics operations involving cell therapies are both complex and challenging. Moreover, in order to ensure the stability of such products across the supply chain, specific temperature conditions (cryogenic, ambient or refrigerated) are required, in addition to specialized packaging material. Considering that most cell therapies are personalized, it is imperative that errors in labeling are avoided at all costs.

Given the need for costly and specialized equipment and the complexities associated with handling cell-based therapy products, innovators in the healthcare industry are likely to involve contract service providers for their drug product production, packaging and transportation requirements. Over time, regulators across the world, including the US Food and Drug Administration (FDA) and the International Air Transport Association (IATA), have established guidelines for the packaging, storage and transport of cell therapies. Since CMOs / CDMOs with the necessary capabilities to handle cell therapies are also likely to have established processes that comply with the current acceptable standards across different global regions, it is better for therapy developers to strategically partner with the aforementioned players instead of focusing on developing in-house capabilities. Presently, close to 60 companies claim to be offering cell therapy packaging services and associated products. In fact, it is estimated that close to 30% of drug substance and drug product related operations of advanced therapy medicinal products are already outsourced to capable CMOs / CDMOs. Further, over the last few years, several service providers have also forged strategic alliances among themselves, in order to further expand existing capabilities and augment their respective service offerings. Given the rising trend of outsourcing in the healthcare industry, and the ongoing efforts of service providers to further improve their portfolios, we believe that the cell therapy packaging products and services market is likely to evolve at a steady pace in the next few years.

SCOPE OF THE REPORT The Cell Therapy Packaging Products and Services Market by Type of Therapy (T-cell Therapies, Dendritic Cell Vaccines, Stem Cell Therapies, NK Cell Therapies and Other ATMPs), Package Engineering Design (Primary and Secondary Packaging), Scale of Operation (Clinical and Commercial) and Geography (North America, Europe, Asia Pacific and Rest of the World) , 2021-2030 report features an elaborate study of the current scenario and future opportunity within the cell therapy packaging products and services market. In addition, the report features an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain. Amongst other elements, the report includes: An overview of the current market landscape of cell therapy packaging services providers, including a detailed analysis based on their year of establishment, company size, location of headquarters, package engineering design (primary and secondary), type of packaging (active and passive), packaging material (dry ice and liquid nitrogen), type of passive system used, temperature ranges supported, type of cells handled and additional services offered. A detailed assessment of the current market landscape of cell therapy packaging products, providing information on type of packaging container (bags, vials, shipping container and others), container fabrication material (plastic and others), storage temperature conditions, type of cells packed and usability of containers. In addition, the chapter highlights analysis of cell therapy packaging product provider(s), based on various parameters such as year of establishment, company size and location of headquarters and key players. An in-depth competitiveness analysis of cell therapy packaging service providers based in different geographies, by taking into consideration the service strength of a company (based on its experience), packaging portfolio (package engineering design, type of packaging, type of packaging material and temperature ranges supported) service portfolio (type of additional services offered) and company size (small, mid-sized and large companies). An analysis of the various partnerships established between cell therapy packaging providers and cell therapy developers, during the period, 2016-2021. It includes a brief description of various types of partnership models (namely service alliance, technology / platform integration agreement, merger and acquisition) adopted by stakeholders engaged in this domain. It is worth mentioning that the data captured during our research was analyzed based on several parameters, such as year of partnership, type of partnership, purpose of partnership, type of service covered and geographical location of players involved in a partnership. A list of more than 250 cell therapy developers anticipated to partner with cell therapy packaging providers, which have been shortlisted on the basis of developer strength (based on company size), type of therapy and pipeline maturity (based on stage of development of drug candidate). A case study highlighting the companies that claim to have the required expertise and capabilities for development and manufacturing of cell therapies, along with the information on their year of establishment, company size, location of headquarters and location of manufacturing facility. In addition, the chapter presents analysis on type of cells manufactured, source of cells, scale of operation and manufacturing capabilities / services of the aforementioned companies. Detailed profiles of the key players offering cell therapy packaging services and products. Each profile includes a brief overview of the company, details on cell therapy service portfolio, information on cell therapy packaging product portfolio, recent developments and an informed future outlook.

One of the key objectives of the report was to estimate the existing market size and future growth potential of the cell therapy packaging providers market. We have provided informed estimates on the likely evolution of the market in the short to mid-term and long term, for the period 2021-2030. Our year-wise projections of the current and future opportunity have further been segmented across [A] type of therapy (T-cell therapies, dendritic cell vaccines, stem cell therapies, NK cell therapies and other advanced therapy medicinal products), [B] package engineering design (primary and secondary packaging), [C] scale of operation (clinical and commercial) and [D] geography (North America, Europe, Asia Pacific and Rest of the World)

All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified.

KEY QUESTIONS ANSWERED Who are the leading players offering cell therapy packaging services and products? What are the key considerations and challenges associated with cell therapy packaging? Which partnership models are commonly adopted by stakeholders engaged in this industry? Which players are likely to partner with cell therapy packaging service and product providers? What factors are likely to influence the evolution of the cell therapy packaging service providers market? What are the likely future trends in cell therapy packaging services market? How is the current and future opportunity likely to be distributed across key market segments?

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary research. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been validated from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views

While the focus has been on forecasting the market till 2030, the report also provides our independent views on various non-commercial trends emerging in this industry. The opinions are solely based on our knowledge, research and understanding of the relevant market trends gathered from various secondary sources of information.

CHAPTER OUTLINES Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the cell-based therapy manufacturing market and its likely evolution in the short to mid-term, and long term.

Chapter 3 provides a general introduction to the pharmaceutical filling and packaging, including information on types of pharmaceutical packaging. The chapter lays emphasis on cell therapy packaging, primary and secondary packaging of cell therapies. In addition, it discusses the key considerations and challenges associated with cell therapy packaging.

Chapter 4 features an overview of the current market landscape of cell therapy packaging services providers, including a detailed analysis on the year of establishment, company size and location of headquarters. In addition, the chapter highlights the analysis on the package engineering design (primary and secondary), type of packaging (active and passive), packaging material (dry ice and liquid nitrogen) and type of passive system used. Further, the chapter also presents analysis on temperature ranges supported, type of cells handled and additional services offered.

Chapter 5 fpresents a detailed assessment of the current market landscape of cell therapy packaging products, providing information on type of packaging container (bags, vials, shipping container and others), container fabrication material (plastic and others), storage temperature conditions, type of cells packed and usability of containers. In addition, the chapter highlights analysis of cell therapy packaging product provider(s), based on various parameters such as year of establishment, company size and location of headquarters and key players.

Chapter 6 includes profiles of the key players offering cell therapy packaging services and products. Each profile includes a brief overview of the company, details on cell therapy service portfolio, information on cell therapy packaging product portfolio, recent developments and an informed future outlook.

Chapter 7 presents in-depth competitiveness analysis of cell therapy packaging service providers based in different geographies, by taking into consideration the service strength of a company (based on its experience), packaging portfolio (package engineering design, type of packaging, type of packaging material and temperature ranges supported) service portfolio (type of additional services offered) and company size (small, mid-sized and large companies).

Chapter 8 provides an analysis of the various partnerships established between cell therapy packaging providers and cell therapy developers, during the period, 2016-2021. It includes a brief description of various types of partnership models (namely service alliance, technology / platform integration agreement, merger and acquisition) adopted by stakeholders engaged in this domain. It is worth mentioning that the data captured during our research was analyzed based on several parameters, such as year of partnership, type of partnership, purpose of partnership, type of service covered and geographical location of players involved in a partnership.

Chapter 9 presents a list of more than 250 cell therapy developers anticipated to partner with cell therapy packaging providers, which have been shortlisted on the basis of developer strength (based on company size), type of therapy and pipeline maturity (based on stage of development of drug candidate).

Chapter 10 features a case study highlighting the companies that claim to have the required expertise and capabilities for development and manufacturing of cell therapies, along with the information on their year of establishment, company size, location of headquarters and location of manufacturing facility. In addition, the chapter presents analysis on type of cells manufactured, source of cells, scale of operation and manufacturing capabilities / services of the aforementioned companies.

Chapter 11 presents a detailed market forecast analysis, providing insights on the likely growth of continuous manufacturing equipment providers market for the period 2021-2030. In addition, the chapter presents the likely distribution of the projected future opportunity based on type of therapy (T-cell therapies, dendritic cell vaccines, stem cell therapies, NK cell therapies and other advanced therapy medicinal products), package engineering design (primary and secondary packaging), scale of operation (clinical and commercial) and geography (North America, Europe, Asia Pacific and Rest of the World).

Chapter 12 is a summary of the overall report, which presents the insights on the contemporary market trends and the likely evolution of cell therapy products and services market.

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix that provides the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06130493/?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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Cell Therapy Packaging Products and Services Market by Type of Therapy, Package Engineering Design, Scale of Operation and Geography : Industry Trends...

News Release

Tuesday, May 11, 2021

An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health. The researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatments for their condition, known as severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. The findings were published today inthe New England Journal of Medicine.

ADA-SCID, which is estimated to occur in approximately 1 in 200,000 to 1,000,000 newborns worldwide, is caused by mutations in theADAgene that impair the activity of the adenosine deaminase enzyme needed for healthy immune system function. This impairment leaves children with the condition highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first two years of life.

These findings suggest that this experimental gene therapy could serve as a potential treatment option for infants and older children with ADA-SCID, said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). Importantly, gene therapy is a one-time procedure that offers patients the hope of developing a completely functional immune system and the chance to live a full, healthy life.

People with ADA-SCID can be treated with enzyme replacement therapy, but this treatment does not fully reconstitute immune function and must be taken for life, usually once or twice weekly. Transplants of blood-forming stem cells, ideally from a genetically matched sibling donor, can provide a more lasting solution. However, most people lack such a donor. Additionally, stem cell transplants carry risks such asgraft-versus-host disease and side effects from chemotherapy medications given to help the donor stem cells establish themselves in the patients bone marrow.

The new research evaluated an experimental lentiviral gene therapy designed to be safer and more effective than previously tested gene-therapy strategies for ADA-SCID. This gene therapy involves inserting a normal copy of theADAgene into the patients own blood-forming stem cells. First, stem cells are collected from the patients bone marrow or peripheral blood. Next, a harmless virus is used as a vector, or carrier, to deliver the normalADAgene to these cells in the laboratory. The genetically corrected stem cells then are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the cells establish themselves in the bone marrow and begin producing new immune cells.

The experimental gene therapy, developed by researchers from the University of California, Los Angeles (UCLA) and Great Ormond Street Hospital (GOSH) in London, uses a modified lentivirus to deliver the ADA gene to cells. Previous gene-therapy approaches for ADA-SCID have relied on a different type of virus called a gamma retrovirus. Some people who have received gamma retroviral gene therapies have later developed leukemia, which scientists suspect is due to the vector causing activation of genes that control cell growth.The lentiviral vector is designed to avoid this outcome and to enhance the effectiveness of gene delivery into cells.

The results come from three separate Phase 1/2 clinical trials, two conducted in the United States and one in the United Kingdom. The U.S. trials, led by principal investigator Donald Kohn, M.D., of UCLA, enrolled 30 participants with ADA-SCID ranging in age from 4 months to 4 years at UCLA Mattel Childrens Hospital and the NIH Clinical Center in Bethesda, Maryland. The U.K. study, conducted at GOSH and led by principal investigator Claire Booth, M.B.B.S., Ph.D., enrolled 20 participants ranging in age from 4 months to 16 years. Most participants acquired and retained robust immune function following gene therapy 96.7% after two years in the U.S. studies and 95% after three years in the U.K. study and were able to stop enzyme replacement therapy and other medications. Of the two participants for whom gene therapy did not restore lasting immune function, one restarted enzyme replacement therapy and later received a successful stem cell transplant from a donor, and the other restarted enzyme replacement therapy. The lentiviral gene therapy appeared safe overall, although all participants experienced some side effects. Most of these were mild or moderate and attributable to the chemotherapy that the participants received.

Researchers observed similar outcomes in all three trials, although there were some differences between the studies. Stem cells were collected from bone marrow in the U.S. trials and from peripheral blood in the U.K. trial. In one of the U.S. trials, 10 children were treated with genetically corrected stem cells that had been frozen and later thawed. The two other trials used fresh stem cell preparations. In the future, the freezing procedure known as cryopreservation may allow stem cells to be more easily transported and processed at a manufacturing facility far from the patients home and shipped back to a local hospital, reducing the need for patients to travel long distances to specialized medical centers to receive gene therapy. A trial of the cryopreserved treatment is now underway at the Zayed Centre for Research into Rare Diseases in Children in London, in partnership with GOSH.

For more information about the trials described in the New England Journal of Medicine paper, visit ClinicalTrials.gov under identifiers NCT01852071, NCT02999984 and NCT01380990. The investigational lentiviral gene therapy, which is licensed to Orchard Therapeutics, has not been approved for use by any regulatory authority.

The research was funded in part by three NIH Institutes: NIAID; the National Heart, Lung and Blood Institute; and the National Human Genome Research Institute. Additional funding was provided by the California Institute for Regenerative Medicine, the Medical Research Council, the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children National Health Service Foundation Trust and University College London, and Orchard Therapeutics.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

DB Kohn, C Boothet al. Autologousex vivolentiviral gene therapy for adenosine deaminase deficiency.New England Journal of MedicineDOI: 10.1056/NEJMoa2027675 (2021).

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Read more:
Gene therapy restores immune function in children with rare immunodeficiency - National Institutes of Health

Jesse Owens

Making waves in the fight against disease at the University of Hawaii is the Institute for Biogenesis Researchs (IBR) youngest full-time faculty member, 36-year-old Jesse Owens. Owens, who also earned his PhD in cell and molecular biology from UH Mnoa, was awarded $2.3 million from the National Institutes of Health (NIH) over the next five years to develop a new, safer and more efficient approach to gene therapy. He leads a team of collaborators from UH Mnoas John A. Burns School of Medicine (JABSOM), MIT and UC Davis.

The $2.3 million grant is significant because it was a first-time submission and received a score in the 1st percentile, higher than 99% of grants submitted from institutions across all 50 states in the U.S.

Directed evolution of a sequence-specific targeting technology for therapeutic gene delivery to the human genome, Owens grant addresses drawbacks to current genome editing technologies that randomly insert a therapeutic gene, which can disrupt important host genes and potentially cause cancer. Methods like CRISPR (clustered regularly interspaced short palindromic repeats), a family of DNA sequences in genomes of organisms, are inefficient in non-dividing cells and delivering large DNA cargos. However, the technology that Owens is developing can direct large pieces of DNA to a safe location in the genome that can be used in all body tissues, both dividing and non-dividing, which will allow us to cure more types of diseases.

This is an R01, which is the coveted personal grant that people get to do researchits bringing absolutely state-of-the-art technology that hes inventing to do new gene therapy in Hawaii and this is why were very proud of him, said IBR Director Steven Ward.

I think the schools here in Hawaii are underestimated Owens

Not only is he faculty here, he got his PhD here, he was trained here, he was raised on the Big Island, added Ward. Hes the product of that and it just shows you that Hawaii can do some of the worlds greatest biomedical research.

In addition to the NIH funding, Owens recently signed a sponsored-research agreement with a private company specializing in non-viral gene therapies called SalioGen Therapeutics. The goal of this collaboration is to advance the tools he is developing in the lab into clinical-stage gene therapy candidates.

Born on Hawaii Island, Owens shares his Hilo High School alma mater with renowned Nobel Prize winner Jennifer Doudna, who developed CRISPR gene therapy. He hopes to inspire other children from Hawaii to pursue a career in science.

I think the schools here in Hawaii are underestimated really, but you can succeed coming out of Hawaii, for sure, Owens said.

Read more on the JABSOM website.

This research is an example of UH Mnoas goal of Excellence in Research: Advancing the Research and Creative Work Enterprise (PDF), one of four goals identified in the 201525 Strategic Plan (PDF), updated in December 2020.

Continue reading here:
$2.3M boost puts Hawaii gene therapy research on the map | University of Hawaii System News - UH System Current News

Dive Brief:

Biogen's big move into gene therapy came just over two years ago with the $800 million acquisition of Nightstar Therapeutics, a developer of eye medicines.

Since then, the company has inked an agreement with Sangamo Therapeutics to explore gene editing as a way to treat neurological diseases, pushed further into gene therapies for the eye through a deal with Germany's ViGeneron, and just recently announced plans to spend $200 million on a gene therapy manufacturing facility in North Carolina.

These investments have come both as Biogen's peers pour money into gene therapy, and as the company faces investor pressure to diversify its business.

Gene therapy research has made a resurgence, in large part, because the technology and how it interacts with the body are now better understood. The Food and Drug Administration has approved a handful of cell and gene therapies over the last few years, and expects to be reviewing and approving between 10 and 20 annually by 2025.

Yet, there are still major challenges in this cutting-edge field, with one of the biggest being delivery. To be effective, gene therapies must achieve the complicated task of carrying their payloads to the appropriate cells without getting destroyed by the body's defense mechanisms. That task can be especially difficult for therapies going after highly protected areas, such as the brain.

Companies that specialize in gene therapy delivery have therefore been in high demand. Over the last year, for example, a startup called Dyno Therapeutics has caught the attention of heavyweight gene therapy developers like Novartis, Roche and Sarepta Therapeutics.

Dyno's capsid technology is meant to create more targeted gene therapies that are less likely to trigger an immune response. Just last week, the company said it had raised an additional $100 million in fresh funding from tech investor Andreessen Horowitz and several other venture firms.

Also recently, the gene editing startup Beam Therapeutics agreed to buy Guide Therapeutics, which makes tools to deliver genetic medicines into cells, in a deal valued at $120 million.

Capsigen now adds to this flurry of activity with its Biogen collaboration. Per deal terms, Biogen will hold an exclusive license to Capsigen's technology for an undisclosed number of CNS and neuromuscular disease targets.

"One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles," Al Sandrock, Biogen's head of research and development, said in a May 11 statement. "We are investing for the long-term by building platform capabilities and advanced manufacturing technologies with the goal of accelerating our efforts in gene therapy."

Read the original here:
Biogen looks to build better gene therapies through latest deal - BioPharma Dive

Gene therapies such as Novartis spinal muscular atrophy treatment Zolgensma deliver a surrogate copy of a gene to replace a dysfunctional one in the body. The CRISPR gene editing tool could offer an alternative strategyone that could allow for a mutated gene to be fixed on site. But scientists arent sure whether such a technique, known as gene drive, would work at scale.

Researchers at the University of California, San Diego (UCSD) say they may have found a way to make CRISPR efficient in gene therapy. They have developed a technology called CopyCatcher, which candetect and quantify events in which a genetic element is copied precisely from one chromosome to another during CRISPR-based gene editing.

In fruit flies, CopyCatcher revealed unexpectedly high rates of gene conversion, according to results published in Nature Communications. With help of the new tool and DNA screening, the team also identified the c-MYC gene as an inhibitor of genetic copying in human embryonic cells. The researchers said the findings lay the groundwork for developing CRISPR-based gene therapy for humans.

The promise of CRISPR-based gene drives is that one chromosome bearing the drive can cutthe other chromosome, which uses the drive as a template torepair the damage. UCSD used CopyCatcher to measure the efficiency of such a system in living animals.

CopyCatcher carries a highly sensitive detectorgene that produces fluorescent proteins only if the target genetic element copies itself onto a sister chromosome, allowing investigators to detect and quantify gene conversion.

In fruit flies, the frequency of gene conversion was unexpectedly high, at 30% to 50% of cells in the targeted tissues of the eye and thorax, the team reported.

RELATED: MIT and UCSF researchers create CRISPR 'on-off switch' that controls gene expression without changing DNA

But the rates of chromosome copying dropped sharply to just 4% to 8% of cells in human cells. That may be because mammalian chromosomes dont typically engage in chromosome pairing but rely instead on a different mechanism to fix DNA cuts. The researchers showed they could use certain techniques to improve copying, suggesting that human cells might be induced to perform efficient gene conversion.

By using a genetic screen, the team identified several factors affecting how DNA selects a repair pathway. The gene c-MYC emerged as a prime inhibitor of templated gene conversion. Cutting the expression of c-MYC by half increased the production of fluorescence marker expression by 2.5-fold in human embryonic cells compared with control cells, the team showed.

If high-efficiency gene editing could be achieved in human cells, CRISPR-based gene therapies could be developed to treat a variety of genetic disorders including blood diseases, hearing loss, spinal muscular atrophy, congenital heart defects and others, the researchers suggested.

These studies provide a clear proof of principle for a new type of gene therapy in which one copy of a mutated gene could be repaired from a partially intact second copy of the gene, Ethan Bier, Ph.D., the studys senior author, said in a statement. The need for such a design occurs in genetic situations with patients with inherited genetic disorders, if their parents were carriers for two different mutations in the same gene.

For future studies, the researchers plan to use CopyCatcher to identify additional factors that can be manipulated to push the choice of DNA repair toward the chromosome-pairing mechanism. That could improve the efficiency of CRISPR-based editing, they believe.

Read more:
How a genetic 'CopyCatcher' could open the door to CRISPR-based precision gene therapy - FierceBiotech

Cell and gene therapies rapid penetration in clinical trials globally is testimony to the incredible potential these in understanding, treating, and curing diseases. The cell and gene therapy clinical trials market is rapidly evolving, touching numerous frontiers in personalized medicine, especially for chronic diseases. A number of gene therapies approved by the U.S. FDA reinforces the potential. Pharmaceutical companies in clinical trials that test cell and gene therapies have bloomed strikingly, most notably in oncology, eye diseases, and rare hereditary diseases. A partial list of the top diseases that attract massive attention of contract research organizations in cell and gene therapy market are type 1 diabetes, Parkinsons disease, spinal cord injuries, amyotrophic lateral sclerosis, the Alzheimers disease, and osteoarthritis.

The number of cell and gene therapies is seeing marked increase year over year. According to an estimate, there were more than a thousand cell and gene therapy clinical trials by 2019. To complement the trend, investments by pharma companies are also rising by large bounds in those years.

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The study on the cell and gene therapy clinical trials offers deep expounding of current and emerging business strategies, elements of competition, facets of markets attractiveness, and insights into regional growth dynamics across the globe.

Cell And Gene Therapy Clinical Trials Market: Key Trends

Clinical trials pertaining to advanced therapy medicinal product (ATMP) are making consistent increase in some developed nations. A predominant percentage of these in recent years have been viral vector mediated gene therapies. Thus far, some remarkable strides have been witnessed in this direction, enriching the investment scope in the cell and gene therapy market. The effect has been notices in all phases, from Phase I to Phase IV.

A prevalent trend over the past few years is the focus on oncology. Oncology--notably including haematological malignancies and solid tumourshave been at the center of ATMP clinical trials. Metabolic disease trials have also seen a significant increase, cementing revenue growth in the cell and gene therapy market. Advances made in gene therapy trials continue to pave way to new vistas for oncology research, both in vivo and in vitro.

Cell And Gene Therapy Clinical Trials Market: Competitive Dynamics and Key Developments

The profound potential of cell and gene therapies (CGT) notwithstanding, their successful clinical translation is, no doubt, rests on panoply of problems. These also determine the key restraints for the evolution of the cell and gene therapy market. The high degree of personalization that CGT entails, factors affecting their efficacy and safety are difficult to ascertain, if not impossible. For one, obtaining cells from donors is replete with some unique challenges, such as invasiveness of the process to patients. So are the lack of availability of cutting-edge biomarkers and targets anchored on which gene therapies will show their potential. The whole process of delivering CGT in clinical trials is itself associated with some tall challenges for contract research organizations.

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Having put these perspectives, the prospects have limitless potential waiting to be extracted, and researchers are not disheartened by the aforementioned challenges. In oncology alone, a number of new approaches have added liveliness to CGT clinical trials. Biotech companies are testing new waters in allogeneic therapies. T-cell receptor (TCR) are increasingly penetrating safety and feasibility trials, adding momentum to the cell and gene therapy market.

Some of the industry players likely to invade the space of these limitless possibilities are;

Cell And Gene Therapy Clinical Trials Market: Regional Assessment

North America has been at the cynosure of attention for CGT trials. European nations have also been showing substantial potential for generating revenues in the global market. In coming years, Asia Pacific is expected to show high growth potential

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Cell And Gene Therapy Clinical Trials Market in 2021 | Expansive Coverage on the Latest Developments in the Market - BioSpace