Category Archives: Gene therapy

A biotechnology company launched Wednesday by life sciences venture firm ATP is the latest startup to debut with a new twist on genetic editing.

With $50 million in funding, Boston-based Ascidian Therapeutics claims its RNA exon editing approach could match the durability of gene therapy while avoiding some of the risks that come with editing DNA.

Its platform is designed to correct for mutations in exons the regions of DNA that contain information needed to make proteins. Ascidian aims to do this by replacing mutated exons with functional RNA copies as DNA is being converted into its chemical cousin.

The company will first target a genetic eye condition called Stargardt disease, which is the most common form of inherited macular degeneration and results in vision loss.

According to Ascidian, its technology can fix genetic errors that other editing approaches cant, and can be applied to widely varied genes. Its lead program can replace more than 20 exons at a time, said Romesh Subramanian, Ascidians CEO.

We are changing chapters in a book rather than whiting-out one letter at a time, Subramanian, said in an interview with BioPharma Dive. Subramanian came to Ascidian from Dyne Therapeutics, a biotech he founded and led as CEO. He previously founded RNA specialist Translate Bio, which was bought by Sanofi last year.

Subramanian claims that Ascidians approach, by focusing on RNA, maintains genome integrity and thereby sidesteps concerns around off-target edits. His company also doesnt rely on foreign enzymes to work, potentially easing immunogenicity risks, he added.

Along with Stargardt disease, Ascidian is looking at other eye conditions, neurological disorders and rare diseases. Subramanian declined to disclose how many drug research programs Ascidian plans to roll out.

Ascidians name is derived from a class of ocean-dwelling invertebrate creatures, which are sometimes known as sea squirts. These creatures use RNA trans-splicing to alter the RNA messengers used by their cells, a process that Ascidian plans to leverage to rewrite RNA for treating disease.

Ascidian is not ATPs first foray into genetic medicine. Last year, Ascidian co-founder and ATP venture partner Michael Ehlers, a former Biogen executive,launched a startup called Intergalactic Therapeuticsthat focuses on non-viral gene therapy. ATP has also built a company called Replicate, which is developing another kind of RNA medicine.

We think the RNA space is a big way of manipulating biology and treating disease across the board, and this approach we've taken to Ascidian defines a new class of RNA therapeutics, Ehlers said.

The company expects to spend the rest of 2022 and 2023 on pre-clinical studies for its lead program, along with developing proof of concept for other candidates targeting neurological and neuromuscular diseases.

Gene editing research was catalyzed by the discovery of CRISPR, which has now been extended and adapted to support several different gene editing technologies. But biotech companies are also exploring RNA editing, which in part appeals to scientists because it doesnt change the underlying DNA.

It has drawn in larger drugmakers, too: Roche and Eli Lilly have recently formed partnerships with Shape Therapeutics and ProQR Therapeutics, respectively, to develop treatments for a wide variety of diseases.

More here:
Ascidian starts up with $50M and a twist on RNA editing - BioPharma Dive

Exopharms technology has put the company in a leadership-position in both nave exosomes and engineered exosomes as it progresses to a product-first company.

Its been a challenging yet productive two years for the Exopharm (ASX:EX1) team.

The company has made significant progress over this period and now owns a tool chest of 7 important exosome manufacturing technologies, as well as well identifying two new in-house GM (genetic medicine) product programs.

At the centre of Exopharms is its proprietary manufacturing technologies, which enable the company to develop its own exosome-medicine products and harness their unique drug-delivery capabilities.

Preliminary results from a recently completed study detected no immunogenicity or toxicity of exosomes manufactured by Exopharm, which means they are suitable for clinical pursuit.

The study also helped to validate Exopharms exosomes as a safe drug-delivery chassis meaning they could be used as safe delivery vehicles inside a persons body to deliver drug doses at high frequencies.

Separate earlier clinical trials with the company Plexaris product also demonstrated safety and efficacy in wound healing using Exopharm-produced exosomes.

These results have now put Exopharm in a leadership-position in both nave exosomes and engineered exosomes.

Exosomes are naturally occurring nano-vesicles that are secreted in huge numbers by cells throughout the human body, and are a natural and powerful way that cells communicate and coordinate.

Within the last 7 years, researchers have envisaged using exosomes as delivery vehicles inside a persons body like programmable, targeted and safe nano-sized drones.

Drugs that had delivery problems, like mRNA, DNA and some small molecules, could be packaged into exosomes at a bioprocessing facility to produce modern medicines which were safer, more tolerable, and more specific to the target tissue/destination than their cargo alone.

Over the last few years, Exopharm had worked on developing the technology to manufacture these exosomes, and that led the market to value it as a platform technology company.

But now, the technology has developed significantly and Exopharm believes that market perception is outdated.

Exopharm emphasised that it is now a product-first company after announcing two GM programs under development.

The Exopharm team has assessed hundreds of potential exosome products over the past 15 months using strict assessment criteria and detailed commercial scoping.

From that work now comes two new programs with several potential product variants in each program.

The first lead program is treating Cystic Fibrosis (CF) using exosome-based additive CFTR mRNA gene therapy and nebuliser delivery to lungs.

CF is the most common autosomal recessive disease with >100,000 CF patients worldwide (around 40,000 in US) and the median age of death is 32 years. Lung disorder is the main cause of morbidity and mortality.

There is no cure for cystic fibrosis, but treatment can ease symptoms, reduce complications and improve quality of life.

Current treatment options include antibiotics, anti-inflammatory medications, mucus-thinners, bronchodilators, oral pancreatic enzymes and CFTR modulators.

Exopharm however believes that additive gene therapy is ideal for CF compared to some other genetic diseases.

The Exo-RNA approach, which is gene therapy delivered via exosomes, has several advantages which includes avoiding the use of viral vectors.

The Exo-RNA gene therapy approach also poses no risk of potential insertion into the host genome as the exosomes loaded with CFTR mRNA are formulated to be administered via a nebuliser (inhaler).

This approach is expected to be more efficacious and cost-effective, with no direct competitors identified.

The CF market is predicted to reach US$31 billion by 2027 with 24% compound annual growth rate (CAGR), up from US$5 billion in 2019.

Elastin is a natural molecule in our bodies produced from the ELN gene. Elastin imparts elasticity in various tissues such as skin, lungs, blood vessels and fascia.

With age and exposure to smoke, levels of elastin decrease and medical and aesthetic issues arise from that elastic deficiency.

An additive gene therapy using ELN mRNA would produce additional Elastin in the body and could bring the elastic tissue structure back to normal.

Exopharm has identified several main product opportunities in this program including Chronic obstructive pulmonary disease (COPD) such as pulmonary emphysema and chronic bronchitis.

Destruction of elastin or abnormalities in elastic fibre assembly are major factors in emphysema and COPD, and an estimated 3.1 million Americans have been diagnosed with emphysema and around 11.2 million people in the country have COPD.

Tobacco use is the number one factor in the progression of COPD, along with pollution, infections and genetic factors.

Other potential treatments in this program also include skin conditions and aesthetic dermatology.

Exopharm will also explore hypertension and arterial stiffness, scar prevention, and photoaging in its Elastin program.

Exopharm says these two programs have been selected after considering their potential future commercial value, and as a way to showcase exosomes as a non-viral nanoparticle chassis to overcome drug-delivery problems that GMs face.

With these two lead programs, the company believes its strategy and future-direction is clear and promising.

Exopharm believes there is an emerging opportunity to treat many medical problems with Genetic Medicines to use exosomes as the drug-delivery chassis for potentially hundreds of new products.

Exopharm can also profit from this opportunity by making its exosome technologies available to others on commercial terms.

The company can also generate revenue by developing a small number of its own products in order to build shareholder-value along the way.

This article was developed in collaboration with Exopharm, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.

Get the latest Stockhead news delivered free to your inbox.

"*" indicates required fields

It's free. Unsubscribe whenever you want.

You might be interested in

Go here to read the rest:
Exopharm identifies two new lead programs as exosomes gain prominence in gene therapy - Stockhead

DUBLIN--(BUSINESS WIRE)--The "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application Area, Therapeutic Area, and Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

With the increasing number of cell and gene therapies being developed and launched for a wide range of therapeutic areas, these modalities are on their way to become one of the highest valued markets in the biopharmaceutical domain. In fact, in 2021, cell and gene therapy developers raised capital worth more than USD 20 billion, registering an increase of 19% from the amount raised in 2020 (~USD 17 billion).

It is worth highlighting that, in February 2022, the USFDA approved second CAR-T therapy, CARVYKTIT, developed by Johnson and Johnson, which can be used for the treatment of relapsed or refractory multiple myeloma. Additionally, close to 1,500 clinical trials are being conducted, globally, for the evaluation of cell and gene therapies. Over time, it has been observed that the clinical success of these therapies relies on the design and type of gene delivery vector used (in therapy development and / or administration). At present, several innovator companies are actively engaged in the development / production of viral vectors and / or non-viral vectors for cell and gene therapies.

In this context, it is worth mentioning that, over the past few years, multiple viral vector and non-viral vector based vaccine candidates have been developed against COVID-19 (caused by novel coronavirus, SARS-CoV-2) and oncological disorders; this is indicative of lucrative opportunities for companies that have the required capabilities for viral vector manufacturing and gene therapy manufacturing.

The viral vector manufacturing and non-viral vector manufacturing landscape features a mix of industry players (well-established companies, mid-sized firms and start-ups / small companies), as well as several academic institutes. It is worth highlighting that several companies that have the required capabilities and facilities to manufacture vectors for both in-house requirements and offer contract services (primarily to ensure the optimum use of their resources and open up additional revenue generation opportunities) have emerged in this domain.

Further, in order to produce more effective and affordable vectors, several stakeholders are integrating various novel technologies; these technologies are likely to improve the scalability and quality of the resultant therapy. In addition, this industry has also witnessed a significant increase in the partnership and expansion activities over the past few years, with several companies having been acquired by the larger firms. Given the growing demand for interventions that require genetic modification, the vector and gene therapy manufacturing market is poised to witness substantial growth in the foreseen future.

Scope of the Report

The "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market (5th Edition) by Scale of Operation (Preclinical, Clinical and Commercial), Type of Vector (AAV Vector, Adenoviral Vector, Lentiviral Vector, Retroviral Vector, Plasmid DNA and Others), Application Area (Gene Therapy, Cell Therapy and Vaccine), Therapeutic Area (Oncological Disorders, Rare Disorders, Neurological Disorders, Sensory Disorders, Metabolic Disorders, Musco-skeletal Disorders, Blood Disorders, Immunological Diseases, and Others), and Geographical Regions (North America, Europe, Asia Pacific, MENA, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2022-2035" report features an extensive study of the rapidly growing market of vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies having in-house manufacturing facilities.

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. VIRAL VECTOR AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE

5. PLASMID DNA AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE

6. VECTOR AND GENE THERAPY MANUFACTURERS (NON-INDUSTRY PLAYERS): MARKET LANDSCAPE

7. VECTOR AND GENE THERAPY MANUFACTURING TECHNOLOGIES: MARKET LANDSCAPE

8. COMPANY COMPETITIVENESS ANALYSIS

9. VECTOR AND GENE THERAPY MANUFACTURERS IN NORTH AMERICA

10. VECTOR AND GENE THERAPY MANUFACTURERS IN EUROPE

11. VECTOR AND GENE THERAPY MANUFACTURERS IN ASIA-PACIFIC

12. OTHER KEY PLAYERS

13. RECENT PARTNERSHIPS

14. RECENT EXPANSIONS

15. STRATEGIC PARTNER ANALYSIS

16. EMERGING VECTORS

17. KEY INSIGHTS

18. COST PRICE ANALYSIS

19. CAPACITY ANALYSIS

20. DEMAND ANALYSIS

21. MARKET SIZING AND OPPORTUNITY ANALYSIS

22. PORTER'S FIVE FORCES ANALYSIS

23. KEY DRIVERS AND CHALLENGES

24. SURVEY ANALYSIS

25. CONCLUDING REMARKS

26. EXECUTIVE INSIGHTS

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/wg8tme

Excerpt from:
Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market Report 2022 - ResearchAndMarkets.com - Business Wire

Cystic fibrosis (CF) is the most common, fatal genetic disease in the United States. About 30,000 people in the United States have the disease. CF causes the body to produce thick, sticky mucus that clogs the lungs, leads to infection, and blocks the pancreas, which stops digestive enzymes from reaching the intestine where they are required in order to digest food.

Mutations in a single genethe Cystic Fibrosis Transmembrane Regulator (CFTR) genecause CF. The gene was discovered in 1989. Since then, more than 900 mutations of this single gene have been identified.

In normal cells, the CFTR protein acts as a channel that allows cells to release chloride and other ions. But in people with CF, this protein is defective and the cells do not release the chloride. The result is an improper salt balance in the cells and thick, sticky mucus. Researchers are focusing on ways to cure CF by correcting the defective gene or correcting the defective protein.

Gene therapy offers great promise for life-saving treatment for CF patients since it targets the cause of CF rather than just treating symptoms. Gene therapy for CF had its start in 1990 when scientists successfully corrected faulty CFTR genes by adding normal copies of the gene to laboratory cell cultures.

In 1993, the first experimental gene therapy treatment was given to a patient with CF. Researchers modified a common cold virus to act as a delivery vehicleor vectorcarrying the normal genes to the CFTR cells in the airways of the lung.

Subsequent studies have tested other methods of gene delivery, such as fat capsules, synthetic vectors, nose drops, or drizzling cells down a flexible tube to CFTR cells lining the airways of the lungs. Researchers are now testing aerosol delivery using nebulizers.

But finding the best delivery system for transporting normal CFTR genes is only one problem that scientists must solve to develop an effective treatment for CF. Scientists must also determine the life span of affected lung cells, identify the parent cells that produce CFTR cells, and find out how long treatment should last and how often it needs to be repeated.

The first cystic fibrosis gene therapy experiments involved lung cells because these cells are readily accessible and because lung damage is the most common, life-threatening problem in CF patients. But scientists hope that the technologies being developed for lung cells will be adapted to treat other organs affected by CF.

Another research breakthrough offers a promising approach to treating cystic fibrosis. Researchers at the University of Washingtons Genome Center and at PathoGenesis Corporation have completed a genetic map for thePseudomonas aeruginosabacterium. This bacterium is the most common cause of chronic and fatal lung infections for people with CF. Scientists hope to use their knowledge of this bacteriums genetic sequence to develop innovative drugs for treating infections caused byP. aeruginosa.

As the amount of information about CF grows, scientists have recognized the need to share their research findings. To facilitate this sharing of information, the Cystic Fibrosis Foundation fundsCystic Fibrosis Foundation Therapeutics(CFFT) located at The University of North Carolina Chapel Hill. The center is becoming a repository for data derived from gene expression studies. By pooling information, researchers hope to accelerate the process of finding a cure for CF.

What Do We Know About Gene Therapy and Cystic Fibrosis? was originally published by the National Institutes of Health.

Follow

A part of the U.S. Department of Health and Human Services, NIH is the largest biomedical research agency in the world.

Link:
What Do We Know About Gene Therapy and Cystic Fibrosis? - The Epoch Times

CSafe joins BioLife's global partner network of cold chain solution providers using the evo cold chain management platform

MONROE, Ohio and BOTHELL, Wash., Oct. 11, 2022 /PRNewswire/ -- CSafe, one of the largest active and passive temperature-controlled shipping solutions providers for pharmaceuticals, and BioLife Solutions, Inc. (NASDAQ: BLFS), a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy ("CGT") and broader biopharma markets, announced today a new partnership to provide a combined global service network to support CGT products, with a focus on enhanced reliability, security and quality.

CSafe joins BioLife's global partner network of cold chain solution providers serving the CGT market. BioLife expects to support 10,000-12,000 evo shipments of CGT starting materials and manufactured doses over the next 12 months. The new partnership will provide expanded supply chain options for any CGT product, at any stage of development, from early clinical-phase trials to global commercial scale.

CSafe will bring even more of its highly reliable global reach to the CGT sector through this new alliance. Operating from 50+ service centers worldwide, the company is a proven, trusted partner for biopharmaceutical manufacturers seeking to navigate supply chain complexity, with a track record that includes shipping solutions for six billion doses of a COVID-19 vaccine.

"CSafe is honored to partner with the team at BioLife Solutions, whose range of CGT bioproduction tools and services is peerless in the market. We are excited to merge these exceptional tools and services with our expertise in global, reliable scaled delivery," said CSafe CEO Patrick Schafer. "CGT is hitting its stride and needs global support. We know how important these therapies are to patients everywhere, and it's our mission at CSafe to protect every shipment."

"We're excited to work with a global partner with a strong history of reliability and performance and a deep dedication to innovative therapies," said BioLife Solutions CEO Mike Rice. "We have the best LN2 technology and cGMP storage facilities in the market, in addition to our other world-class CGT solutions, and we are confident our collaboration with CSafe will extend our reach and result in even more reliability and real-time service for CGT partners."

Media Contacts:April Lynch+1 937-245-6479alynch@csafeglobal.comFor BioLife Solutions:

At the CompanyTroy WichtermanChief Financial Officer(425) 402-1400twichterman@biolifesolutions.com

InvestorsLHA Investor RelationsJody Cain(310) 691-7100jcain@lhai.com

About CSafe

CSafe offers the most comprehensive suite of thermal shipping solutions for pharmaceutical cold chain shipping needs around the world. With a "patient-first" focus, deep industry expertise and commitment to innovation, CSafe continues to deliver industry-leading products in both the active and passive segments. CSafe is the only provider with an end-to-end portfolio including active and passive bulk air cargo, parcel, cell and gene and specialty last-mile use cases with the ability to meet the complete range of pharma cold-chain shipping requirements with industry-leading quality and reliability. Any size, any duration and any temperature CSafe is At the Heart of Your Cold Chain.csafeglobal.com

About BioLife Solutions

BioLife Solutions is a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy and broader biopharma markets. Our tools portfolio includes our proprietary CryoStor and HypoThermosol biopreservation media for shipping and storage, the ThawSTAR family of automated, water-free thawing products, evo cold chain management system, high capacity cryogenic storage freezers, Stirling Ultracold mechanical freezers, SciSafe biologic storage services, and Sexton Biotechnologies cell processing tools. For more information, please visit http://www.biolifesolutions.com, http://www.scisafe.com, http://www.stirlingultracold.com, or http://www.sextonbio.com and follow BioLife on Twitter.

Caution Regarding Forward Looking Statements

Statements in this press release that are not historical facts are "forward-looking statements" that may involve material risks and uncertainties. The company wishes to caution readers not to place undue reliance on such forward-looking statements. All such statements speak only as of the date made.

More here:
CSafe and BioLife Solutions, Inc. announce partnership to expand supply chain solutions for the cell and gene therapy market - PR Newswire APAC - PR...

Covina, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Genomics is the study of all of persons gene. Genomics play role in health and disease. Genomics are widely used in cancer care treatment for diagnosing and treating cancer disease. Structural Genomics and Functional Genomics are two types of Genomics.Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics are variety of applications in genomics. Metagenomics has become the important application in genomics. The newer technique genome editing is used in gene therapy. Genome editing help to introduce gene-editing tools which can able to change existing DNA in cell. Genomics are used in drug discovery due to their properties like high-throughput sequencing & characterization of expressed human genes. Genomics has allowed effective preventive measures, change in drug research strategy and development process in drug discovery due to knowledge about human genes and their functions. A complete human genome contains about 3 billion base pairs of DNA. Pharmacogenomics is the study of genes and their functions to develop safe medications which are effective and can be prescribed based on persons genetic makeup. Pharmacogenomics choose the drug and drug doses that are effective for that particular person by using genetic information about that person. Pharmacogenomics helps in improving patient safety, health care costs and drug efficiency. Single nucleotide variant (SNV) panels are used in pharmacogenetics. Genomics helps to reveal the abnormalities in genes which has drived the development and growth of different types of cancer.Study of cancer genome has improved in understanding the biology of cancer which has enabled to discover new methods for diagnosing & treating the disease. The importance of Genomics in cancer care has provided to discover new drug development and effective treatment in diagnosing and treating the disease which has driven positive impact on target market growth.

The reportGlobal Genomics in Cancer Care Market, By Type (Structural Genomics, Functional Genomics), By Application (Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics, and Others), By End-User (Research Institute, Hospitals, Academic Research Institutes, Diagnostic Centers, and Others) andBy Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2032

Key Highlights:

Request Free Sample Copy of the Report:https://www.prophecymarketinsights.com/market_insight/Insight/request-sample/4953

Analyst View:

Increase in cancer disease, rising emergence of clinical relievance in genomic medicine, recent advancement in genomics, newly developed technology like next-generation sequencing has given rise in use ofGenomics in Cancer Care. Wide variety of applications in Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics has fueled the target market growth. Rising awareness in individual who are pertaining to cancer genomics, rapid growth in biotechnology industries, research institutes, diagnostic centers is expected to have positive impact on Genomics in Cancer Care market. Importance of Genomics in cancer care has enabled to provide effective treatment, new drug development, diagnosing and treating disease which has enhanced the target market growth.As a result, market competition is intensifying, and both big international corporations and start-ups are vying to establish position in the market.

Browse 60 market data tables* and 35figures* through 140 slides and in-depth TOC onGlobal Genomics in Cancer Care Market, By Type (Structural Genomics, Functional Genomics), By Application (Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics, and Others), By End-User (Research Institute, Hospitals, Academic Research Institutes, Diagnostic Centers, and Others) andBy Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2032

To know the upcoming trends and insights prevalent in this market, click the link below:

https://www.prophecymarketinsights.com/market_insight/Genomics-in-Cancer-Care-Market-4953

Key Market Insights from the report:

GlobalGenomics in Cancer CareMarketaccounted for US$ 16.1 Bn in 2022 and is estimated to be US$ 72.61 Bn by 2032 and is anticipated to register a CAGR of 16.3%.TheGlobalGenomics in Cancer CareMarketis segmented based on Type, Application, End-User and Region.

Competitive Landscape & their strategies ofGlobalGenomics in Cancer Care Market:

The prominent players operating in theGlobalGenomics in Cancer CareMarketincludes,Pacific Biosciences Inc., Abbott Molecular Oxford Gene Technology, Roche Diagnostics, Bio-Rad Labs, Illumina Inc., Quest Diagnostics, Beckman Coulter Inc., Intrexon Bioinformatics Germany GmbH, Agilent Technologies, PerkinElmer, Danaher Corporation, Cancer Genetics Inc., Thermo Fisher Scientific Inc., and others.

The market provides detailed information regarding the industrial base, productivity, strengths, manufacturers, and recent trends which will help companies enlarge the businesses and promote financial growth. Furthermore, the report exhibits dynamic factors including segments, sub-segments, regional marketplaces, competition, dominant key players, and market forecasts. In addition, the market includes recent collaborations, mergers, acquisitions, and partnerships along with regulatory frameworks across different regions impacting the market trajectory. Recent technological advances and innovations influencing the global market are included in the report.

Scope of the Report:

About Prophecy Market Insights

Prophecy Market Insights is specialized market research, analytics, marketing/business strategy, and solutions that offers strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high-value opportunities in the target business area. We also help our clients to address business challenges and provide the best possible solutions to overcome them and transform their business.

Some Important Points Answered in this Market Report Are Given Below:

Browse for Related Reports:

1.Single-Cell Genome Sequencing Market, By Type (Reagents and Instruments), By Technology (NGS, PCR, qPCR, MDA, and Microarray), By Application (Circulating Cells, Genomic Variation, Cell Differentiation, Subpopulation Characterization, and Others), By End-User (Forensic Labs, Cell banks and IVF centers, Academic & Research Laboratories, Biotechnology & biopharmaceutical companies, and Hospitals and diagnostic laboratories) and By Region (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa) - Trends, Analysis and Forecast till 2029

2.Global Genomics Market By Product and Services (Consumables, Instruments/Systems, and Services), By Technology (Microarray, Purification, PCR, Sequencing, Nucleic Acid Extraction, and Other Technologies (Gene Editing, Gene Expression, Genotyping, and among others)), By Process (Library Preparation, Sequencing, and Data Analysis), By Application (Diagnostics, Precision Medicine, Agriculture, Drug Discovery & Development, Animal Research, and Other applications (Biofuels, Coal Mines, Marine Research, and Among Others)), By End User (Academic &Government Institutes, Research Centers, Hospitals & Clinics, Pharmaceutical & Biotechnology Companies, and Other End Users (Agri-genomics organizations, NGOs, among others)), and By Region (North America, Europe, Asia Pacific, Middle East, and Africa) - Trends, Analysis and Forecast till 2029

3.Global Lab-On-a-Chip Market, By Product Type (Instruments, Reagents and Consumables, Software, and Services), By Application (Genomics and Proteomics, Diagnostics, Drug Discovery, Others), By End-User (Hospitals, Academic and Research Institute, Diagnostic Lab, Homecare settings, and Others) and By Geography (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)- Trends, Analysis and Forecast till 2029

Read the original here:
Genomics in Cancer Care Market is estimated to be US$ 72.61 billion by 2032 with a CAGR of 16.3% during the forecast period 2032 - By PMI -...

Stefan Oelrich, head of Bayers pharmaceuticals department, admitted at the World Health Summit that the COVID-19 vaccine is gene therapy. He smugly stated that the drug companies knew people would reject the vaccine if they knew it was in fact a gene-altering injectable. They lied to us for profits as the vaccine certainly did not prevent anyone from contracting or transmitting the virus.

If we had surveyed two years ago if people were willing to take gene or cell therapy and inject it into your body we would have probably had a 95% refusal rate, Oelrich admits while forgetting many took the gene therapy through force.

Twitter has already flagged retweets of this video as misleading. The conspiracy theorists who were told they had no place in society were right as Big Pharma and governments worldwide used the public as guinea pigs for the largest gene therapy study in history. We still do not know the long-term health implications but have seen a variety of health issues and lingering side effects in the short-term. Revolutions have occurred over much less.

Read the rest here:
Bayer Head Admits COVID-19 Vaccine is Gene Therapy

Summary: A new study reports that a new gene therapy shows promise in treating dry age-related macular degeneration. The gene therapy, ophNdi1, directly targets mitochondrial function that is malfunctioning in AMD.

Source: TCD

Researchers from Trinity College Dublin have developed a new gene therapy approach that shows promise for treating the dry form of age related macular degeneration (AMD)a progressive eye disease that affects up to 10% of adults over 65 years of age and is a leading cause of severe vision impairment and blindness in this age group.

Dry AMD cases represent about 8590% of all AMD cases and there are no treatments available to treat or preventdisease progression, underlining the need for developing treatment options for this debilitating disease. In adults, many diseases of aging have been found to have defects of mitochondrial function, including AMD.

The team, in Trinitys School of Genetics and Microbiology, have developed a new gene therapy (ophNdi1) that is the first of its kind to directly target mitochondrial function in cells that are malfunctioning in AMD.

Mitochondria are known as the powerhouses of the cell because they manage the production of energy but their performance dips greatly in dry AMD and this is linked to a deterioration in sight.

The new gene therapy cleverly uses a virus to access the cells that are suffering and deliver the code needed to give the failing mitochondria a lifeline, enabling them to generate extra energy and continue to function in supporting vision.

The therapy has shown benefit in multiple models of dry AMD, offering hope that it could one day progress to a treatment that could help millions across the globe.

Professor Jane Farrar, senior author, said, Critically, this study provides the first evidence in models that directly modulating bioenergetics in eye cells can provide benefit and improve visual function in dry AMD. In doing so, the study highlights the energy powerhouses of the cell, mitochondria, as key targets for dry AMD.

Dr. Sophia Millington-Ward, first author and research fellow in Trinitys School of Genetics and Microbiology, said, The novel gene therapy targeting cellular energy, or mitochondrial function, that we explored for dry AMD consistently provided benefit in the model systems tested.

Many retinal cells, essential for vision, require particularly high levels of energy compared to most other cells, which makes them particularly vulnerable tomitochondrial dysfunction.

The therapy we are developing directly targets mitochondrial function and increases energy production levels in the retina, which leads to better visual function in disease models of dry AMD.

Although there is further work to be done before this could be made available as a treatment for patients, the results give us hope that we are getting closer to a solution to this challenging, debilitating condition.

Author: Press OfficeSource: TCDContact: Press Office TCDImage: The image is in the public domain

Original Research: Open access.AAVmediated gene therapy improving mitochondrial function provides benefit in agerelated macular degeneration models by Sophia MillingtonWard et al. Clinical and Translational Medicine

Abstract

AAVmediated gene therapy improving mitochondrial function provides benefit in agerelated macular degeneration models

With an estimated 196 million people suffering from age-related macular degeneration (AMD) in 2020 and predicted to increase to 288 million by 2040,dry AMD, representing 70%90% of AMD cases, represents an enormous clinical need with no current therapies.

We have demonstrated that NDI1 and an optimised version of NDI1 (ophNdi1), a mitochondrial complex 1 equivalent fromSaccharomyces cerevisiae, provide functional and histological benefit in two murine models of dry AMD as well as benefit in two cellular models of dry AMD. There are no drugs on the market for dry AMD.

However, there are currently a small number of candidate gene therapies in clinical trial (clinicaltrials.gov).

To our knowledge, this is the first demonstration that a gene therapy directly targeting mitochondrial dysfunction provides functional benefit in in vivo models of dry AMD, making this a novel approach to treating this devastating condition.

Read the original here:
New Gene Therapy Shows Promise for Treating Age Related Macular ...

1. After $100M deal, J&J links gene therapy to improved vision in early-phase trial  FierceBiotech
2. Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting  Johnson & Johnson
3. AAO: Janssen announces late-breaking data from a pair of gene therapy programs  Ophthalmology Times
4. #AAO22: J&J's first look at common eye disease portfolio pads the case for PhII of gene therapy  Endpoints News
5. Late-Breaking Phase 1/2 Data Demonstrates Safety Profile of Investigational Gene Therapy Botaretigene Sparoparvovec (AAV-RPGR) and Sustained Vision Improvement in Patients with X-Linked Retinitis Pigmentosa (XLRP)  GlobeNewswire
6. View Full Coverage on Google News

More:
After $100M deal, J&J links gene therapy to improved vision in early-phase trial - FierceBiotech

Dive Brief:

Roctavians road back to the FDA has been long, as BioMarin has had to gather additional data in support of its therapy. Delays and new safety concerns further hampered the companys resubmission plans.

The treatment is for people with severe hemophilia A, a genetic disease that leaves people with little or no clotting protein needed to prevent excessive bleeding.

While there are many treatments that work by replacing this protein artificially, Roctavian would be the first in the U.S. to address the diseases root cause by replacing the defective gene with a functional copy.

The FDA rejected BioMarins original approval application back in August 2020 and requested more data to prove the treatments benefit for patients over a longer period of time.

BioMarin successfully gathered that follow-up data, showing its gene therapy could restore protein levels to mildlevels and prevent bleeds. The company had expected to file for approval in June but pushed back to September after the FDA asked for further information.

Roctavian gained European approval last month, a few weeks before it was disclosed that a patient treated in clinical trials had developed leukemia. This was the second cancer case reported among patients taking Roctavian. Cases of cancer in clinical trials of gene therapies have been watched closely by U.S. regulators. However, in BioMarins case, genetic testing suggested the case may be naturally occurring, rather than stemming from the therapy. Trial monitors did not call for the study to be halted.

If approved in the U.S., Roctavian is expected to carry a high price tag, likely in the millions of dollars. In Europe, BioMarin is charging around 1.5 million euros, net of discounts. Other gene therapies cleared for the U.S. market have been priced at $2.1 million, $2.8 million and $3.0 million, respectively.

Pfizer and partner Sangamo Therapeutics as well as Spark Therapeutics now owned by Roche are also working on hemophilia gene therapies.

Read more:
BioMarin resubmits its hemophilia gene therapy to the FDA - BioPharma Dive