Archive for the ‘Gene Therapy Research’ Category

In this interview, Arnaud Delobel, the R&D and Innovation Director at Quality Assistance talks to AZoM about the emerging field of gene therapy with adeno-associated viruses (AAV).

Our organization, Quality Assistance, is a contract research organization. Our work provides scientific, regulatory, and technological expertise to our clients, who develop innovative human medicinal products. Our work is strictly compliant with US and EU regulations.

Regarding analytical protocol and methods, we offer customized solutions. When it comes to the acquisition and interpretation of data, Quality Assistance provides a wide array of cutting-edge equipment, which can then be included in the regulatory dossier. Our work is performed in a single site based in Belgium.

With 40 years of experience, Quality Assistance is a stable, reliable analytical partner working hard for our customers.

The Emerging Field of Gene Therapy with Adeno-Associated Viruses - Wyatt and Quality AssistancePlay

To support the development of innovative products, Quality Assistance offers our customers the benefit of our analytical expertise to determine drug safety, quality, and efficacy. This is where Quality Assistance's customized solutions are essential, including the development and validation of specific methods for each product and the design of analytical protocols.

Quality Assistance also offers characterization services, stability studies, battery testing, PK biomarker, and immunogenicity studies.

With our strong product-dedicated expertise, Quality Assistance has worked for 40 years on producing and managing new chemical entities, along with working on peptides and oligonucleotides. Over the last two decades, the company has worked on recombinant proteins such as monoclonal antibodies and antibody-drug conjugates.

Our services for emerging markets, such as mRNA-based therapies, cell and gene therapies, and viral vectors, have been a focus for development over the last few years.

There is definitely an increasing demand for analytical methods for characterizing AAV therapies. Over the last few years, most projects we handled that were in the research phase switched to the clinical phase. With this switch, robust analytical methods, offering results that can be included in regulatory dossiers, became essential.

To keep up with this demand our organization invested inWyatt SEC-MALS system, which is a powerful tool for characterizing AAVs. We can now provide our customers with reliable, validated methods, and results that can be included in regulatory files.

Image Credit: ShutterShock/Kateryna Kon

We use Wyatts DAWN multi-angle light scattering detector with size-exclusion chromatography (SEC-MALS). We also use Wyatts Eclipse asymmetric-flow field-flow fractionation (AF4) instrument along with a multi-detector system for the separation and characterization of AAVs (MD-AF4). Our multi-detector system includes a Wyatt Optilab refractive index (RI) detector, a UV detector, and a DAWN multi-angle light scattering (MALS) equipped with online dynamic light scattering (DLS) detection module for in-depth analysis.

Our organization has over a decade of experience with Wyatt systems. We have employed them to characterize monoclonal antibodies, antibody-drug conjugates, other recombinant proteins, and vaccines.

It was clear to us, from the growing demand for AAV analytics and the capabilities offered by the SEC-MALS system from Wyatt, that we needed to present this service to our customers.

Image Credit: ShutterShock/Kateryna Kon

Light scattering techniques can be used to replace a range of different analytical techniques. Along with concentration detectors, light scatteringcan be considered a muti-attribute method that allows the analysis of size, molecular masses of the protein and the nucleic acid component, concentration, empty/full ratio and aggregate content with one system.

Additionally, it doesnt require primer or antibody reagents and it offers better precision compared to PCR and ddPCR and increased robustness to AUC. A final benefit to highlight is the simplicity of implementing the method in a GxP environment.

Thanks to the 21 CFR Part 11 compliance offered by Wyatts products, we can now employ AF4-MALS in a GxP-compliant environment.

To include data in a regulatory dossier for a characterization study, it is always essential to have complete data integrity. Such compliance with AF4 is not only an advantage for us but is also a significant benefit for our customers.

For AAV analytics in a GxP-compliant environment, Quality Assistance can offer SEC-MALS and AF4-MALS to our customers. This new flexibility in analytical methodologies offers notable advantages, including an improved time to market, thanks to the ability to analyze several critical quality attributes with a single method.

At present, the AAV market is growing. Though it is clear that there will be a need for robust and reliable analytical techniques, it is difficult to say where such developments will stop and what the future will look like. Many developments can currently be seen in analytical techniques, both in the physical/chemical part and the biological methods.

Image Credit: ShutterShock/Kateryna Kon

Further developments will invariably take place. It is difficult to say precisely which changes will be applied in a compliant environment. Still, it is likely that the analytical market for AAVs will continue to expand.

Wyattsystems are not only for AAV-based products but also for recombinant proteins such as monoclonal antibodies, antibody-drug conjugates, and vaccines such as polysaccharide vaccines. Another use is the development of a range of applications for mRNA-based therapies.

Image Credit: Wyatt Technology

Wyattsystems are versatile and flexible and can rapidly analyze and produce results from different samples in a range of formulations. Finally, all our work is performed in a compliant environment, which is vital for our customers.

The Emerging Field of Gene Therapy with Adeno-Associated Viruses - Wyatt and Quality AssistancePlay

Arnaud Delobel is the R&D and Innovation Director at Quality Assistance. He graduated from ECPM Strasbourg and specialized in mass spectrometry of proteins and peptides during his Ph.D. at the CNRS in Gif-sur-Yvette (France). After a post-doc in Lige (Belgium) focused on proteomics, he joined Quality Assistance in 2006 and held several positions in the company, eventually becoming R&D and Innovation Director in 2016.

As the head of the R&D Department, he supervises internal R&D projects, aiming at implementing new analytical technologies that Quality Assistance will ultimately provide to their customers. He also oversees a team of Product Experts responsible for identifying market needs and interpreting and translating this intelligence into developing new R&D services.

This information has been sourced, reviewed and adapted from materials provided by Wyatt Technology.

For more information on this source, please visit Wyatt Technology.

Disclaimer: The views expressed here are those of the interviewee and do not necessarily represent the views of AZoM.com Limited (T/A) AZoNetwork, the owner and operator of this website. This disclaimer forms part of the Terms and Conditions of use of this website.

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The Emerging Field of Gene Therapy with Adeno-Associated Viruses - AZoM

As activity in this space grows, so do the hurdles in moving these products forward.

Cell and gene therapy (CGT)its risks and promisesare succinctly summarized in this description of clinical trial number NCT01129544, a Phase I/II study in children born with X-linked severe combined immunodeficiency (SCID-X1), an inherited, rare, and life-threatening disease. The eight-person trial, which began in May 2010, continues today. The following paragraph has been edited.1

Gene transfer is still research for two reasons. One, not enough children have been studied to tell if the procedure is consistently successful. [And] we are still learning about its side effects and doing gene transfer safely. In previous trials, five children developed gene transfer-related leukemia; four are in remission; one died.

If the above information has stifled the research communitys scientific curiosity about CGT, it is not evident. Evidence from numerous sourcesClinicalTrials.gov, the Alliance for Regenerative Medicine (ARM), FDAare chock-a-block with studies, trials, and figures showing these therapies popularity. In the second quarter of 2022, 3,633 such treatments were in development, up from 1,745 in May 2021. The vast majority are in the preclinical stage.2,3

Some sources are revealing more.

Most indicate that academics now have a remarkable presence in the CGT development space, including sponsorship. Last year, for the first time, ARM included sponsorship figures in its twice-annual industry report.4 Academic- and government-sponsored trials far exceeded industry for sponsored trials in CGT. Stephen Majors, senior director for public affairs, ARM, says the alliance knew of academias presence for the past few years, but only was able to get data this year from its partner, Global Data.

Less reliable, but still noteworthy, are data from ClinicalTrials.gov: for active Phase I trials, industry has 89; others, which covers academia and government, have 50. Industry enrollment for Phase I is 172; others, 116.Phase III is one for others, eight for industry.

A little disruption in pharmas corner of the world? It seems that way. While basic bench to preclinical to clinical trial has long been the traditional route to FDA approvaland no one interviewed for this article suggested a reroutewhat it does imply is that pharma members have some competition from the spin-offs and academic biotechs that historically they have absorbed.

There are suspected trends that we are watching, says Majors.As to whether academias presence in this spot can be called a trend depends on ones definition of what a trend is. The Centers for Disease Control and Prevention (CDC) considers changes over a number years to determine a trend; financial investment firms typically evaluate over a two-year period.Considering that CGT companies raised $23.1 billion in 2021, 16% more than 2020,3 the answer to the above question could be, maybe.

The CGT space is still immature, according to Mike Rea, founder of Protodigm, a self-described exploratory research organization that partners with biopharma clients on alternative development and commercial solutions. Physicians need time to be comfortable with these therapies, notes Rea, so they may not be used on a regular basis.

For example, physicians have to understand how to deliver the gene, agrees cardiologist Arthur M. Feldman, MD, PhD, whose lab worked on a heart failure-related mutation in BAG3 for decades.

Last month, the company he founded, Renovacor, agreed to be acquired by Rocket Pharmaceuticals.5 We are asking physicians to do something they never did before and to understand a very different set of information, including risk/benefit discussions that they didnt learn about in medical school, he says. Feldman is a Laura H. Carnell Professor of Medicine, Division of Cardiology, and a member of the Center for Neurovirology and Gene Editing at the Lewis Katz School of Medicine at Temple University.

Chris Learn, Parexels vice president of cell and gene therapy, is unequivocal regarding academias increased presence in the drug development space focused around these treatments. He cites MD Anderson and Moffitt Cancer Center as two institutions that are sponsoring their own trials. The lines are really blurring here, he tells Applied Clinical Trials. It is indisputable.

The following is a look at how academia is showing up in various reports.

In its 2022 report4, ARM separated sponsorship, type of therapygene, cell-based, and tissue engineeringand trial phase. What these data show are industry far exceeding academic and government sponsored trials for gene therapy, while for cell therapy alone, the reverse is true: 656 cell therapy trials for academic and government, and 424 for industry. For gene therapy, there are 84 for the academics and government, and 222 for industry. In a later report, ARM found non-industry trials dropped.

Pharma Intelligences Pharma R&D Annual Review does not break down trials by their sponsors. It does, however, break down whats in the pipeline in various categories, including by the number of therapies per company, and by disease type.6 In numbers captured prior to March 2020, the analysis reported 1,849 companies with asingle drug in its pipeline, up from 1,633 in 2019, comprising more than half of all drug companies. As for types of therapies, gene therapy was in third place, the same spot it occupied in 2019. (Cancer-related therapies occupy the top spots.) Overall, biotech therapies in the pipeline increased by 13.2% in 2020 over 20196,135 vs. 5,422. Cellular therapy, the field in which academia is dominating, rose to 14th place, up from 33.

In 1982, Feldman was a resident in the cardiac care unit at the Johns Hopkins Hospital in Baltimore when he took care of a 22-year-old woman, a native Pennsylvanian, who was dying of heart failure. Sadly, we didnt have drugs with which to treat her, he recalls. Feldmans involvement with the case and the womans family led to his career as a cardiologist, he says. Twenty years later in Philadelphia, he was asked to see a heart-failure patient in consult, who turned out to be the aunt of the younger woman. It would take almost another 10 years until the technology became available to identify the genomic anomaly in this family. Here, a genetic variant that is produced by one of two alleles causes the protein product to be unstable. The result: the cell removes it, so the person with the variant has just half the amount of required protein.

BAG3 is an interesting protein that is found in the heart, the skeletal muscles, and the nervous system, including the brain. Its function is to help remove degraded and misfolded proteins, stop apoptosis or programmed cell death, and maintain the structure of the skeletal muscles. A missing allele isnt the only genetic cause for heart failure, Feldman said. Other patients, while having the correct amount of DNA, have a point mutationa single amino acidin half of the produced DNA. That single letter is the wrong amino acid in the specific site in the protein.

Around this time, Kamel Khalili, PhD, Laura H. Carnell Professor, and chair of the department of microbiology, immunology, and inflammation; director of the Center for Neurovirology and Gene Editing; and director of the Comprehensive NeuroAIDS Center, Lewis Katz School of Medicine, Temple University, had created a method by which he could excise the HIV virus from patients using the new technique of CRISPR-Cas9.

Khalili believes that BAG3 may be involved in the pathogenesis of HIV-1 in brain diseases and protein quality control caused by viral infection as well as several other disorders, including Alzheimers disease and dementia. BAG3 changes the homeostasis of the cell, he says. The only solution is to fix the cell. Khalili has used CRISPR technology to excise the viral genome in both small and large investigational animals and has recently started a Phase I trial to test the safety of the new gene-editing treatment. Khalili, too, started a company, but Temple holds the license. In the case of Renovacor, it was granted the license by Temple.

As a scientist, when you are doing something in biomed research, [the] goal is to translate bench work to the clinic for [the] wellness of people. We are doing long hours and long days because we want to help. We are trying to see if discovery can help people, says Khalili. I know my limit, I stop at business aspects. My interest is to discover research which can help populations.

Was Feldman happy with his business experience? As a company gets bigger, others join the team who fulfill other roles, like acquiring funding or developing the actual product, he says. Releasing the control reins are difficult. But if it speeds up the timeline to get an approved product into the clinic, then its all worth it, he adds.

Researchers such as Feldman and Khalili, says Kaspar Mossman, PhD, director of communications and marketing at QB3, a University of California biotech accelerator, are normally not deeply interested in business. He notes the new flagship space in UC Berkeley called Bakar Lab. So far, it has 25 companies, one-third from university labs. They collaborate, they share equipment, [at times] they merge, Mossman tells Applied Clinical Trials.

And, he adds, Academics tend to be very smart individuals. The more time they spend in business, they learn stuff and become serial founders, says Mossman. They are honest about not wanting to be a CEO.

In terms of business, the academics employers are also pretty smart. The huge bugaboo with CGT commercialization is the manufacturing processthe need for an apheresis unit, ultra-cold storage, and regulated cell processing facilities.

Some institutions are building their own manufacturing facilities to more easily meet the increasingly complicated standards pertaining to regenerative medicine production. Harvard, MD Anderson, Moffitt, the University of Pennsylvania, and the University Hospital of Liege in Belgium8 all have or are planning to build their own facilities.

As for how academias presence impacts the traditional pharma space, those interviewed cited pros and cons. More research is better, more companies vying for venture capital funding is not. But more trials mean more competition among similar therapies, which, says Majors, is a good thing.

We need experimentation, adds Rea. If left to pharma, he says, the research wouldnt happen. Smaller biotechs are taking the risk. Over the last 10 years, Rea believes pharma has been slow in the risk-taking department. Once upon a time, pharma didnt have many competitors. Now, with many numerous smaller companies with viable assets, willing to accept a smaller net profit, the competition is creating some angst. Pharma cant project everyones movement, says Rea. The gene/cell therapy landscape [for products] is huge.

Likely adding to the angst: Those smaller biotechs are getting financial help. Between April 4, 2021, and June 24, 2021, of 23 start-up financing deals, 19 involved academics.2

Learns viewpoint is different. He says there are too many players out there, and while large pharma may be averse to risk, I really do believe what we are witnessing are simply market forces that have played into this. There is so much cash coming in, he continues, that people can be blinded by the pitfalls. The CGT area, he adds, is bloated and he says the industry needs an overall strategy.

Learn doesnt think that academias presence in the CGT space is a flash in the proverbial pan. The enthusiasm to find cures is real, and some research institutions have the endowments to see the trials through. I think it is just the beginning, says Learn. Academia will put their futures in front of them. Why put all your sweat equity into it and not have any fiduciary benefit of the approved product?

In Pharma Intelligences 2020 Pharma R&D Review, its author questioned the wisdom of so many drugs, overall, in the pipeline4,001 added in 2018 and 4,730 added in 2019, for a total of 17,737 drug candidates. [A]re the industrys eyes getting too big for its belly? Unless it can continue to provide [approved therapies] then a certain degree of control in the pipeline might be advisable, the report stated.6

And now to costs. While no one doubts these cures change lives, the question of access persists. FDAs approval of Bluebird Bios second therapy this year, branded as Skysona, for early but active cerebral adrenoleukodystrophy, is expected to cost $3 million. Learn doubts that payers are jumping up and down to get Skysona on their formularies.

Its still a fairly dicey business proposition for companies to invest in this field, Steven Pearson, MD, president of the Institute for Clinical and Economic Review (ICER), said recently.8Theres still a risk that next-generation therapies will not flourish even in developed countries health systems, he added.

One positive development in the US, however, occurred late last month when Congress reauthorized the Prescription Drug User Fee Act (PDUFA) for the next five years, 2023-2027. The action maintained FDAs authority to collect fees from manufacturers and keep and recruit agency staff to review the increased number of CGT applications. Majors says most of FDAs review of CGT products involves scalability and consistent reproducibility in the manufacturing process, which, of course, means traveling.

According to a Senate press release9, FDA is seeking to hire at least 320 new staff members. In a statement, Pharmaceutical Research and Manufacturers of America (PhRMA) said a modern regulatory framework supported by PDUFA helps ensure patients have timely access to lifesaving medicines.

PDUFA reauthorization aside, there is little argument that the field of CGT, from research and drug discovery through commercialization, is advancing rapidly. In turn, so are the unique operational and manufacturing challenges that these therapies present. This reality may thin the currently crowded playing field in CGT going forward, with those sponsors and partners best prepared to deliver on the numerous touchpoints required separating from the pack.

Christine Bahls, Freelance Writer for Medical, Clinical Trials, and Pharma Information

Read the original here:
The Risk-Reward Proposition for CGT Clinical Trials - Applied Clinical Trials Online

The Rising in the Cell and Gene Therapy Manufacturing Market is also depicted in this research report. The research is a great blend of qualitative and quantitative data, covering significant market developments, industry and competitive issues, gap analysis, and new opportunities and trends in the Cell and Gene Therapy Manufacturing Market. The reports goal is to give cutting-edge market knowledge and assist decision-makers in making smart investment decisions by presenting an overview of the Cell and Gene Therapy Manufacturing Market. Additionally, the research identifies and analyses upcoming trends, as well as important drivers, challenges, and opportunities. Historical data available in the report elaborates on the development of the Cell and Gene Therapy Manufacturing on national, regional, and international levels.

In this report, a comprehensive study of the competitive scenario among the leading vendors has been depicted in the subsequent sections, along with detailed insight into the sales captured by the participant and the share each vendor accounted for in the overall Market share. The Cell and Gene Therapy Manufacturing research report is isolated into different segments to work on the cognizance of the market elements. The study considers the present scenario of the market and its market dynamics for the period 2022-2030. This report is a top to bottom and intense investigation of the current details of the worldwide market covering various aspects related to insights and development of the business.

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Key Companies Covered

In this section of the report, the researchers have done a comprehensive analysis of the prominent players operating and the strategies they are focusing on to combat the intense competition. Company profiles and market share analyses of the prominent players are also provided in this section. Additionally, the specialists have done an all-encompassing analysis of each player. They have also provided reliable revenue, market share, and rank data of the companies for the period 2022-2030. With the assistance of this report, key players, stakeholders, and other participants will be able to stay abreast of the recent and upcoming developments in the business, further enabling them to make efficient choices. Mentioned below are the prime players taken into account in this research report:

3P Biopharmaceuticals, ABL, Inc., AGC Biologics, Advent BioServices Ltd., Akron Biotech, Aldevron, Anemocyte S.r.l

Cell and Gene Therapy Manufacturing Market Segments

This report has explored the key segments: by Type and by Application. The lucrativeness and growth potential have been looked into by the industry experts in this report. This report also provides revenue forecast data by type and by application segments based on value for the period 2022-2030

Market Segments by Type:

Pharmaceutical and Biotechnology , Academic and Research Institutes, Other

Market Segments by Application:

Clinical Manufacturing, Commercial Manufacturing

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Market research fills the gap between goods or services and their target market or client. Market research insights assist a manufacturer or service provider in developing a product or service plan that satisfies the specific needs of the target market.

Primary Market Research:

Primary market research is a type of study that involves acquiring information from target industries, either through a third party or directly from customers.

Quantitative Market Research:

Quantitative market research is a method of gathering data from the target market and consumers that can be quantified.

Qualitative Market Research:

Qualitative market research is a means of gathering qualitative data from target markets via the use of instruments and procedures such as focus groups and interviews.

Secondary Market Research:

Secondary market research is an investigative strategy in which a firm relies on publicly available data to learn more about its customers and target markets.

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Each of the regional market segments is analyzed and studied in the sense of the major regional spectrum of market reach in the market report. The study also provides a comprehensive overview of key insights, such as import, export, development, demand, and consumption

North America (United States, Canada, and Mexico)Europe (Germany, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, Korea, India, and Southeast Asia) South America (Brazil, Argentina, Colombia, etc.)The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

The study of the market includes

Explanation of consumer preferences, trends, opportunities, and factors impacting storage technologies, as well as an analysis of current industry trends. Market share study by end-user, service, application, and region Investments in R&D and demonstration projects are covered, as well as a full overview of the market. Assessment of regulatory trends and incentives, regional economic development rate, implementation timeline, and environmental constraints

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Cell and Gene Therapy Manufacturing Market Trends, Opportunities, and Breakthrough Point During (2022-2030)- 3P Biopharmaceuticals, ABL, Inc., AGC...

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:

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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

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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:

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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

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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 -...

DUBLIN--(BUSINESS WIRE)--The "Biopharmaceutical Contract Manufacturing Organization (BCMO) Market, 2022" report has been added to ResearchAndMarkets.com's offering.

This report, Biopharmaceutical Contract Manufacturing Organization (BCMO) Market, 2022, describes and discusses the global contract manufacturing markets, specifically focusing on the production of biotech (large molecule) products including extensive coverage of the biotechnology segment of the global BCMO industry.

Sales estimates are provided by segment and region, expressed in current dollars. Estimates are provided for the historic 2016 to 2021 period and forecasts are provided through 2026.

Further, this report examines third party manufacturing of potential and commercialized prescription drug products throughout the world. Potential drug products are those in clinical (Phase I - Phase III) testing prior to regulatory approval and require relatively small amounts of product for these evaluations; commercialized drugs are those which have received regulatory approval and have been introduced to mass markets. In some cases, drugs have received regulatory approval in selected regions (such as the EU, but not in others such as the US).

The report contains the following market information:

Key Topics Covered:

Chapter 1: Executive Summary

Chapter 2: Overview

Chapter 3: The Contract Manufacturing Industry

Chapter 4: Total Global BCMO Market

Chapter 5: The BCMO Market by Region

Chapter 6: Pharmaceutical & Biotech Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/664983

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World Biopharmaceutical Contract Manufacturing Organization (BCMO) Market Research Report 2022: Focus on Growth Factors - Biotechnology, Biosimilars,...

Renewed National Institute for Health and Care Research Birmingham Biomedical Research Centre continue funds for developments around inflammatory diseases

Published 14 October 2022

Local people with cancer and heart disease are amongst those set to benefit from a major injection of research funding which will develop new diagnostic tools and treatments for those with cancer, liver and heart disease, and many more illnesses.

The NIHR Birmingham Biomedical Research Centre has today been granted more than 30 million of funding from the National Institute for Health and Care Research, a major funder of global health research and training, to support world-leading research into inflammation and the myriad of diseases and health issues that it can cause.

The centre unites leading NHS providers led by the University Hospitals Birmingham NHS Foundation Trust and academic institutions led by the University of Birmingham. The partnership sees eight organisations working closely with charities and businesses to support research into inflammation which causes or worsens many common long-term illnesses including arthritis, liver disease and cancer.

The announcement sees the NIHR Birmingham BRC increase its funding almost 3-fold and will enable researchers to focus on eight areas of illness including heart disease, womens health, and common complications from inflammation. It will also enable researchers to consider new tests and biomarkers for disease, health technologies including stem cells and gene therapy, patient experiences and data science.

The funding will allow us to make a step-change in our work tackling different forms of cancer, trialling new drugs for liver disease, and dealing with antimicrobial resistance.Professor Philip Newsome

Professor Phil Newsome, Director of Research and Knowledge Transfer at the University of Birminghams College of Medical and Dental Sciences and Director of the NIHR Birmingham Biomedical Research Centre said:

Inflammation plays a central role in many health conditions, with millions of people in the UK alone experiencing inflammatory diseases such as arthritis and bronchitis.

The significant increase in funding for the NIHR Birmingham Biomedical Research Centre will enable us to provide an outstanding environment for world-leading clinical research. The funding will allow us to make a step-change in our work tackling different forms of cancer, trialling new drugs for liver disease, and dealing with antimicrobial resistance.

Patients will benefit from the increased funding for the NIHR Birmingham BRC through collaborative research that has seen nearly 1,000 clinical trials and informed UK clinical guidelines.

Researchers will look at eight themes to continue to understand and help patients manage inflammation-based diseases including cancer, arthritis, and liver disease. The investment of the NIHR funding in biomedical research will enable clinicians, researchers, patients and supporters to find new treatments such as the development of new immunotherapies, which are types of cancer treatments to support the body to fight cancer.

When Joy needed a liver transplant, the team at University Hospitals Birmingham involved in her care made her aware about an opportunity to take part in a trial through the BRC to better understand sarcopaenia.

In many chronic inflammatory diseases, including liver disease, loss of muscle mass and strength (sarcopaenia) occurs. Importantly sarcopaenia contributes to poor patient quality of life, reduced ability to cope with challenges such as surgery or infection and higher risk of death. The NIHR Birmingham BRC has been investigating inflammatory sarcopaenia to find the best treatments and support for patients who experience inflammation-related muscle loss.

Joy said: It was interesting to be made aware of muscle loss and to be encouraged to do tests [to assess sarcopaenia]. Following the two-day assessment, I was encouraged to increase protein in my diet because of muscle loss and the trial reinforced how important that was. I think the trial made me feel like I was doing something positive at a time when some things in my life were dampening down.

After the transplant, one of the first people I saw in hospital was a member of the research team. She had heard I had had the op and made a point of dropping by. We had a lovely chat. As a beneficiary of liver transplant and the improving outcomes for liver transplant patients, I am heartily in favour of research in this as well as other medical fields.

Tim Jones, Chief Innovation Officer, at University Hospitals Birmingham NHS Foundation Trust said:

We are delighted with the award to the NIHR Birmingham BRC which builds on our successful track record of joint working in Inflammatory disease, the award will significantly support the acceleration of new discoveries for the benefit of our patients.

Researchers based at the NIHR Birmingham BRC will also support a major improvement in how data and digital healthcare can improve patient outcomes.

The Data, Diagnostics and Decision Tools theme will see the development of new infrastructure and innovation to tackle major challenges in the use and interpretation of data in biomedical research. Bringing together expertise across a range of disciplines including clinical trials, health informatics, and artificial intelligence, increased funding will see improvements in the way data is held and used to uphold the highest levels of research integrity.

The expanded funding will also enable the NIHR Birmingham BRC to invest in research excellence and create additional capacity, to collaboratively focus on key wider areas of clinical practice including the development of new tests and biomarkers, and next generation therapies such as stem cell and gene therapy.

Mark Maybury joined the Birmingham Rheumatology Research Group in 2018, supported by the NIHR-funded Birmingham Biomedical Research Centre. Coming from a clinical role as a physiotherapist and musculoskeletal sonographer, Mark has been working with academics to support research trials for rheumatoid arthritis drugs and has been involved in pioneering work in the use of ultrasound-guided synovial joint biopsies.

Mark said: One of the many reasons that attracted me to move into this research post at this institution was the opportunity to learn and during my time at the Birmingham BRC I have been given the opportunity for personal study and involvement in research. But as well as learning myself, Ive also had the opportunity to pass on my knowledge - training research fellows in diagnostic ultrasound and to develop new instrumentation for ultrasound guided synovial biopsy. The results of the studies I have worked on will help influence the treatment of hundreds of thousands of patients, not only in the UK but worldwide, which is more patients than I ever could ever help as a clinician. I find that a very sobering thought."

The investment from NIHR is hugely important for researchers working across the BRC partner institutions, to continue to tackle some of the critical health themes that affect our region.Professor David Adams

Professor David Adams, Pro-vice Chancellor and Head of the College of Medical and Dental Sciences at the University of Birmingham, and director of the previous NIHR Birmingham BRC said:

The investment from NIHR is hugely important for researchers working across the BRC partner institutions, to continue to tackle some of the critical health themes that affect our region.

"The funding will allow us to bring together teams that can use an in depth understanding of disease processes to deliver new therapies and diagnostic tests for a range of chronic inflammatory diseases for which we currently have few effective treatments.

The NIHR Birmingham BRC is among 20 centres across England that have been awarded a combined 790 million by the National Institute for Health and Care Research, to translate scientific discoveries into new treatments, diagnostic tests and medical technologies for patients.

NIHR Biomedical Research Centres are partnerships between healthcare professionals and academics in the countrys leading NHS trusts and universities. The centres, part of NIHRs research infrastructure, receive substantial levels of sustained funding to attract the best scientists and create an environment where experimental medicine can thrive.

The Birmingham Biomedical Research Centre is made up of the following partners:

Professor Lucy Chappell, Chief Executive of the NIHR, said:

Research by NIHR Biomedical Research Centres has led to a number of ground-breaking new treatments, such as new gene therapies for haemophilia and motor neurone disease, the world-first treatment for CreutzfeldtJakob disease, a nose-drop vaccine for whooping cough, and the first UK-wide study into the long-term impact of COVID-19.

This latest round of funding recognises the strength of expertise underpinning health and care research across the country and gives our nations best researchers more opportunities to develop innovative new treatments for patients.

For media enquiries for the University of Birmingham, please contact Tim Mayo, Press Office, University of Birmingham, tel: +44 (0)7920 405040: email: t.mayo@bham.ac.uk

The mission of the National Institute for Health and Care Research (NIHR) is to improve the health and wealth of the nation through research. We do this by:

NIHR is funded by the Department of Health and Social Care. Its work in low and middle-income countries is principally funded through UK Aid from the UK government.

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New 30M research injection to improve treatment of inflammatory diseases - University of Birmingham

Naoto T. Ueno

Naoto T. Ueno has been selected to be the next director of the University of Hawaii Cancer Center. Uenos appointment is pending the UH Board of Regents approval at the boards October 20 meeting. His appointment would be effective December 12, 2022.

Ueno is currently the executive director of the Inflammatory Breast Cancer Research Program at the University of Texas MD Anderson Cancer Center, where he is also a tenured professor of medicine.

He has held various faculty and administrative positions there since 1996. Ueno has 15 years of leadership experience, and nearly 30 years of experience in research and education, particularly in stem cell transplant, gene therapy, targeted therapy and immunotherapy.

His research focus is inflammatory breast cancer (IBC)the most lethal and aggressive form of breast cancer with a high metastasis (spread) rate. Working with his team members, he created a comprehensive research program and clinic devoted to IBC, which, under his leadership, emerged as the worlds largest and most renowned for rare breast cancer.

Ueno is also widely regarded for his preclinical development and research efforts that translated into clinical trials. These valuable outputs will be of great benefit to the cancer centers clinical trials and community building efforts.

Dr. Ueno possesses the leadership skills, background and experience to lead the UH Cancer Center for years to come, I am confident he will continue to advance the UH Cancer Center and their mission to reduce the burden of cancer in communities throughout Hawaii, said UH Mnoa Provost Michael Bruno.

Ueno said his mission as director is to ensure that every patient with cancer lives to the fullest extent. He wishes to prevent cancer in Hawaii and the Pacific through patient-centered cancer strategies, high-quality cancer care, research, prevention and education. In his previous roles, he helped build a team culture reflecting diversity and psychological safety.

I am honored and excited for this tremendous opportunity to work with a new team and to build collaborative partnerships inside and outside of the UH Cancer Center, said Ueno. Being raised in both Asia and the U.S., I have a long history of interest in Hawaiis diverse culture and demographics. I truly believe the UH Cancer Center has a unique opportunity to build a world-class clinical and translational program that serves the community to reduce the suffering of cancer patients.

As a cancer survivor, Ueno brings more depth in his role as director, rendering the ability to empathize with cancer patients. He has also published two books about empowering patients in cancer care.

Dr. Uenos leadership experience, coupled with his ability to share common ground with cancer patients, make him uniquely qualified to improve cancer patient care in Hawaii and the Pacific, said Bruno.

Ueno earned his MD from Wakayama Medical College in Japan. He went on to earn his PhD in cancer biology from The University of Texas Graduate School of Biomedical Sciences. He received postgraduate training through internships and fellowships with the United States Naval Hospital in Yokosuka, Kanagawa Japan, Montefiore University Hospital (internal medicine), the University of Pittsburgh Medical Center (internal medicine) and The University of Texas MD Anderson Cancer Center (medical oncology and stem cell transplantation).

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Naoto T. Ueno announced as new UH Cancer Center director | University of Hawaii System News - University of Hawaii

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.

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

ReportLinker

The Global Viral Vectors Market size was estimated at USD 1,291. 23 million in 2021 and expected to reach USD 1,464. 47 million in 2022, and is projected to grow at a CAGR 13. 67% to reach USD 2,785.

New York, Oct. 14, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Viral Vectors Market Research Report by Type, Disease, Application, End User, Region - Global Forecast to 2027 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p06342341/?utm_source=GNW 63 million by 2027.

Market Statistics:The report provides market sizing and forecast across 7 major currencies - USD, EUR, JPY, GBP, AUD, CAD, and CHF. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2020 are considered as historical years, 2021 as the base year, 2022 as the estimated year, and years from 2023 to 2027 are considered as the forecast period.

Market Segmentation & Coverage:This research report categorizes the Viral Vectors to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Type, the market was studied across Adeno-associated Viral Vectors, Adenoviral Vectors, and Retroviral Vectors.

Based on Disease, the market was studied across Cancers, Genetic Disorders, and Infectious Diseases.

Based on Application, the market was studied across Gene Therapy and Vaccinology.

Based on End User, the market was studied across Pharmaceutical & Biopharmaceutical Companies and Research Institutes.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam. The Europe, Middle East & Africa is further studied across Denmark, Egypt, Finland, France, Germany, Israel, Italy, Netherlands, Nigeria, Norway, Poland, Qatar, Russia, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, and United Kingdom.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, and the long-term effects are projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlying COVID-19 issues and potential paths forward. The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Cumulative Impact of 2022 Russia Ukraine Conflict:We continuously monitor and update reports on political and economic uncertainty due to the Russian invasion of Ukraine. Negative impacts are significantly foreseen globally, especially across Eastern Europe, European Union, Eastern & Central Asia, and the United States. This contention has severely affected lives and livelihoods and represents far-reaching disruptions in trade dynamics. The potential effects of ongoing war and uncertainty in Eastern Europe are expected to have an adverse impact on the world economy, with especially long-term harsh effects on Russia.This report uncovers the impact of demand & supply, pricing variants, strategic uptake of vendors, and recommendations for Viral Vectors market considering the current update on the conflict and its global response.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Viral Vectors Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis:The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario:The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitors strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles:The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Viral Vectors Market, including ABL Inc., Batavia Biosciences B.V., BioNTech IMFS GmbH, Biovian Oy, Cell and Gene Therapy Catapult, Cevec Pharmaceuticals GmbH, Creative Biogene, FinVector Vision Therapies, Fujifilm Diosynth Biotechnologies, GeneOne Life Science, Inc., Genezen Laboratories, Lonza Group AG, Merck KGaA, Miltenyi Biotec GmbH, Novasep Inc., Sirion-Biotech GmbH, Spark Therapeutics Inc., Thermo Fisher Scientific Inc., and Wuxi AppTec Co., Ltd..

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:1. What is the market size and forecast of the Global Viral Vectors Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Viral Vectors Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Viral Vectors Market?4. What is the competitive strategic window for opportunities in the Global Viral Vectors Market?5. What are the technology trends and regulatory frameworks in the Global Viral Vectors Market?6. What is the market share of the leading vendors in the Global Viral Vectors Market?7. What modes and strategic moves are considered suitable for entering the Global Viral Vectors Market?Read the full report: https://www.reportlinker.com/p06342341/?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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Viral Vectors Market Research Report by Type, Disease, Application, End User, Region - Global Forecast to 2027 - Cumulative Impact of COVID-19 - Yahoo...

Andrew P. Byrnes, Ph.D.

Office of Tissues and Advanced TherapiesDivision of Cellular and Gene TherapiesGene Transfer and Immunogenicity Branch

Andrew.Byrnes@fda.hhs.gov

Chief, Gene Transfer and Immunogenicity Branch

BS, MS, Yale University, Department of Molecular Biophysics and Biochemistry

PhD, Oxford University, Department of Human Anatomy

Postdoctoral Fellow, Johns Hopkins University, School of Hygiene and Public Health

Gene therapy holds great promise for treating cancer, inherited disorders, and other diseases. Gene therapy uses carriers called 'vectors' to deliver genes to tissues where they are needed. Researchers are currently investigating the safety and effectiveness of a variety of different gene therapy vectors in hundreds of clinical trials in the US.

We are studying one type of commonly-used gene therapy vector that is made from a disabled cold virus -- the adenovirus vector. While adenovirus vectors are very efficient at delivering genes, adenovirus vectors are not always easy to target to the correct tissue. In addition, adenovirus vectors can cause toxic effects that limit the amount of vector that doctors can give to patients. We are particularly interested in how to safely deliver large amounts of adenovirus vectors intravenously, with the goal of specifically targeting tumors and other tissues.

New adenovirus gene therapy vectors are tested in animals before human clinical trials begin, and it is important for both researchers and the FDA to know how well these animal studies can predict safety. Thus, another of our major goals is to develop animal models that reliably predict the safety and effectiveness of adenovirus vectors in humans.

Our studies will help us to understand the mechanisms for adenovirus vector targeting and toxicity, and the relevance of animal models to human outcomes. This new knowledge will enable researchers to design safer and more effective gene therapy vectors.

Adenovirus (Ad) vectors have shown considerable promise in animal models and are currently being used in numerous clinical trials, especially for the therapy of cancer. We are interested in improving the safety and efficacy of Ad vectors, especially when administered through the vascular system. Certain properties of Ad vectors make them hazardous to administer intravenously in large doses, and our laboratory is trying to understand and fix this problem.

One of our major areas of interest is the innate immune response to Ad vectors. These rapid responses can cause serious toxicity and may severely limit the doses of Ad vectors that are safe to use. In addition, we are also studying how cells in the liver such as Kupffer cells and hepatocytes recognize Ad, since the liver is the major site at which Ad vectors are cleared from the circulation. A better understanding of these mechanisms will help us to develop strategies to improve vector efficacy and reduce toxicity. We will also gain a better understanding of the advantages and disadvantages of using different animal species to predict the behavior of Ad vectors in humans, which is particularly relevant to the regulatory work of the FDA.

Recent work from our lab and others has shown that Ad vectors are heavily influenced by plasma proteins that rapidly opsonize the vectors after intravenous injection. We found that natural IgM antibodies bind to Ad vectors, activate complement, and reduce liver transduction. Intriguingly, the Ad hexon protein specifically binds to coagulation factor X (FX), and we found that recruitment of FX by Ad vectors protects them against neutralization by complement. These findings show that Ad vectors recruit a number of plasma proteins that interact in complex ways with each other and with cells, and that these host proteins ultimately help to determine whether the vector successfully reaches its target.

In the long run, a better fundamental understanding of Ad vector biology will facilitate the design of safer Ad vectors that are easier to target. Better animal models will be important for testing novel vectors for safety and efficacy.

02/22/2022

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Safety and Effectiveness of Gene Therapy | FDA

The cutting-edge Cellaca PLX system, designed by the companys Nexcelom unit, combines best-in-class image cytometer hardware, software, validated consumables and trackable data reporting all in one system without requiring complex calibration procedures or intense training requirements. To further streamline the customer experience, optimized reagent kits with validated antibodies from PerkinElmers BioLegend business are also part of the proprietary solution.

The new offering provides researchers expanded cell sample CQA analysis options beyond flow cytometry and staining methods, which historically have required a variety of different instruments and analytical methods. By combining these capabilities, researchers can now detect multiple markers simultaneously (multiplexing) and perform immunophenotyping and viability assays in seconds with an easy-to-use, modern user interface.

"Pharmaceutical companies have invested heavily in cell and gene therapy, but they struggle to assess the complex cell samples required to meet immense scientific demands and regulatory rigor across their research and manufacturing processes," said Alan Fletcher, senior vice president, Life Sciences, PerkinElmer. "While the Cellaca PLX Image Cytometer platform is therapeutic area agnostic, it is expected to be especially beneficial for researchers working in CAR-T cell therapy who want to streamline their phenotyping of immune cells for downstream processes."

PerkinElmers Nexcelom unit is a leading provider of automated cell counting technology and image cytometry products for cell analysis, including the original and widely adopted Cellaca MX high-throughput automated cell counter. Learn more about the new platform and other image cytometry instruments and reagents at BioProcessing International East from September 27-30 in Boston where PerkinElmer is showcasing the latest innovations across its extensive Life Science and cell and gene therapy portfolio in booths 625 and 631. Product demonstrations can be scheduled here.

About PerkinElmer

PerkinElmer is a leading, global provider of end-to-end solutions that help scientists, researchers and clinicians better diagnose disease, discover new and more personalized drugs, monitor the safety and quality of our food, and drive environmental and applied analysis excellence. With an 85-year legacy of advancing science and a mission of innovating for a healthier world, our dedicated team of more than 16,000 collaborates closely with commercial, government, academic and healthcare customers to deliver reagents, assays, instruments, automation, informatics and strategic services that accelerate workflows, deliver actionable insights and support improved decision making.

We are also deeply committed to good corporate citizenship through our dynamic ESG and sustainability programs. The Company reported revenues of approximately $5.0 billion in 2021, serves customers in 190 countries, and is a component of the S&P 500 index. Additional information is available at http://www.perkinelmer.com. Follow PerkinElmer on LinkedIn, Twitter, Facebook, Instagram, and YouTube.

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PerkinElmer Unveils Industry-first Cell Analysis Solution to Streamline ...

Biopharma focuses on streamlining biomanufacturing and supply chain issues to drive uptake of cell and gene therapies.

Cell and gene therapies (CGTs) offer significant advances in patient care by helping to treat or potentially cure a range of conditions that have been untouched by small molecule and biologic agents. Over the past two decades, more than 20 CGTs have been approved by FDA in the United States and many of these one-time treatments cost between US$375,00 and US$2 million a shot (1). Given the high financial outlay and patient expectations of these life-saving therapies, it is essential that manufacturers provide integrated services across the whole of the supply chain to ensure efficient biomanufacturing processes and seamless logistics to reduce barriers to uptake.

The following looks at the who, what, when, and why of biomanufacturing and logistics in CGTs in the bio/pharmaceutical industry in more detail.

According to market research, the global gene therapy market will reach US$9.0 billion by 2027 due to favorable reimbursement policies and guidelines, product approvals and fast-track designations, growing demand for chimeric antigen receptor (CAR) T cell-based gene therapies, and improvements in RNA, DNA, and oncolytic viral vectors (1).

In 2020, CGT manufacturers attracted approximately US$2.3 billion in investment funding (1). Key players in the CGT market include Amgen, Bristol-Myers Squibb Company, Dendreon, Gilead Sciences, Novartis, Organogenesis, Roche (Spark Therapeutics), Smith Nephew, and Vericel. In recent years, growth in the CGT market has fueled some high-profile mergers and acquisitions including bluebird bio/BioMarin, Celgene/Juno Therapeutics, Gilead Sciences/Kite, Novartis/AveXis and the CDMO CELLforCURE, Roche/Spark Therapeutics, and Smith & Nephew/Osiris Therapeutics.

Many bio/pharma companies are re-considering their commercialization strategies and have re-invested in R&D to standardize vector productions and purification, implement forward engineering techniques in cell therapies, and improve cryopreservation of cellular samples as well as exploring the development of off-the-shelf allogeneic cell solutions (2).

The successful development of CGTs has highlighted major bottlenecks in the manufacturing facilities, and at times, a shortage of raw materials (3). Pharma companies are now taking a close look at their internal capabilities and either investing in their own manufacturing facilities or outsourcing to contract development and manufacturing organizations (CDMOs) or contract manufacturing organizations (CMOs) to expand their manufacturing abilities (4). Recently, several CDMOsSamsung Biologics, Fujifilm Diosynth, Boehringer Ingelheim, and Lonzahave all expanded their biomanufacturing facilities to meet demand (5).

A major challenge for CGT manufacturers is the seamless delivery of advanced therapies. There is no room for error. If manufacturers cannot deliver the CGT therapy to the patient with ease, the efficacy of the product becomes obsolete. Many of these therapies are not off-the-shelf solutions and therefore require timely delivery and must be maintained at precise temperatures to remain viable. Thus, manufacturers must not only conform to regulations, but they must also put in place logistical processes and contingency plans to optimize tracking, packaging, cold storage, and transportation through the products journey. Time is of the essence, and several manufacturers have failed to meet patient demands, which have significant impacts on the applicability of these agents.

Several CAR T-cell therapies have now been approved; however, research indicates that a fifth of cancer patients who are eligible for CAR-T therapies pass away while waiting for a manufacturing slot (6). Initially, the manufacture of many of these autologous products took around a month, but certain agents can now be produced in fewer than two weeks (7). Companies are exploring new ways to reduce vein-to-vein time (collection and reinfusion) through the development of more advanced gene-transfer tools with CARs (such as transposon, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) among others, and the use of centralized organization with standardized apheresis centers (5). Others are exploring the use of the of allogeneic stem cells including Regen Biopharma, Escape Therapeutics, Lonza, Pluristem Therapeutics, and ViaCord (7).

Several gene therapies have also been approved, mainly in the treatment of rare disease (8). Many companies are evaluating novel gene therapy vectors to increase levels of gene expression/protein productions, reduce immunogenicity and improve durability including Astellas Gene Therapies, Bayer, ArrowHead Pharmaceuticals, Bayer, Bluebird Bio, Intellia Therapeutics, Kystal Biotech, MeiraGTx, Regenxbio, Roche, Rocket Pharmaceuticals, Sangamo Therapeutics, Vertex Pharmaceuticals, Verve Therapeutics, and Voyager Therapeutics (8).

While many biopharma companies have established their own in-house CGT good manufacturing practice (GMP) operation capabilities, others are looking to decentralize manufacturing and improve distribution by relying on external contracts with CDMOs and CMOs such as CELLforCURE, CCRM, Cell Therapies Pty Ltd (CTPL), Cellular Therapeutics Ltd (CTL), Eufets GmbH, Gravitas Biomanufacturing, Hitachi Chemical Advances Therapeutic Solutions, Lonza, MasTHerCell, MEDINET Co., Takara Bio, and XuXi PharmaTech (6, 9, 10).

The top 50 gene therapy start-up companies have attracted more than $11.6 billion in funds in recent years, with the top 10 companies generating US$5.3 billion in series A to D funding rounds (10). US-based Sana Biotechnology leads the field garnering US$700 million to develop scalable manufacturing for genetically engineered cells and its pipeline program, which include CAR-T cell-based therapies in oncology and CNS (Central Nervous System) disorders (11). In second place, Editas Medicine attracted $656.6 million to develop CRISPR nuclease gene editing technologies to develop gene therapies for rare disorders (12).

Overall, CGTs have attracted the pharma industrys attention as they provide an alternative route to target diseases that are poorly served by pharmaceutical and/or medical interventions, such as rare and orphan diseases. Private investors continue to pour money into this sector because a single shot has the potential to bring long-lasting clinical benefits to patients (13). In addition, regulators have approved several products and put in place fast track designation to speed up patient access to these life-saving medicines. Furthermore, healthcare providers have established reimbursement policies and manufacturers have negotiated value- and outcome-based contracts to reduce barriers to access to these premium priced products

On the downside, the manufacture of CGTs is labor intensive and expensive with manufacturing accounting for approximately 25% of operating expenses, plus there is still significant variation in the amount of product produced. On the medical side, many patients may not be suitable candidates for CGTs or not produce durable response due to pre-exposure to the viral vector, poor gene expression, and/or the development of immunogenicity due to pre-exposure to viral vectors. Those that can receive these therapies may suffer infusion site reactions, and unique adverse events such as cytokine release syndrome and neurological problems both of which can be fatal if not treated promptly (14).

Despite the considerable advances that have been made in the CGT field to date, there is still much work needed to enhance the durability of responses, increase biomanufacturing efficiencies and consistency and to implement a seamless supply chain that can ensure these agents are accessible, cost-effective, and a sustainable option to those in need.

Cleo Bern Hartley is a pharma consultant, former pharma analyst, and research scientist.

BioPharm InternationalVol. 35, No. 10October 2022Pages: 4951

When referring to this article, please cite it as C.B. Hartley, "Growth in Cell and Gene Therapy Market," BioPharm International 35 (10) 4951 (2022).

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Growth in Cell and Gene Therapy Market - BioPharm International

Allogene Therapeutics has begun the first pivotal test of an off-the-shelf cell therapy for cancer.

The biotechnology company, which has been at the forefront of a push in recent years to develop such treatments, known as allogeneic therapies and derived from donor cells, announced the start of two trials on Thursday. One will test a blood cancer drug known as ALLO-501A, while the other will evaluate a regimen Allogene uses to prepare patients for treatment. Assuming positive results, Allogene expects the studies will support approval applications for both of them.

The studies represent milestones for allogeneic cell therapies, which are meant to be more convenient alternatives to the personalized CAR-T treatments that have come to market for a handful of blood cancers. Allogene, spun out of Pfizers cell therapy work in 2018, is the largest and most advanced among the companies advancing them. Its run by former executives of Kite Pharma, which successfully developed the cell therapies now sold by Gilead Sciences.

Allogene raised more than a half a billion dollars in private financing and an initial public offering to fund its work. The company has had a bumpy ride since then, however. Its cell therapies, including ALLO-501A, have shown promise, but also face lingering questions about their durability and effectiveness compared to CAR-T treatments. The field has also gotten more competitive, with an emerging group of companies advancing therapies using different types of cells and CAR-T moving into earlier lines of care.

Additionally, Allogenes research was delayed by the unexpected finding of a chromosomal abnormality in a treated patient. The companys treatment was exonerated, but the FDA froze Allogenes programs for months during the investigation. Longtime partner Servier cut ties with the company last month as well.

The company now has the chance to prove how its technology stacks up. ALLO-501A is being tested in a Phase 2 study in patients with relapsed or refractory large B cell lymphoma, a setting for which multiple CAR-T treatments are already available.

In a research note, RBC Capital Markets analyst Luca Issi noted that its trial, ALPHA2, mimics the design of the studies underlying approvals of those treatments for lymphoma. Its a single-arm study of about 100 patients whove previously received at least two prior treatments, but not a CAR-T therapy. The study will be judged by ALLO-501As ability to induce a response. Allogene didnt disclose a bar for success, but Issi, after speaking with management, said executives believe efficacy needs to be in the range of approved CAR-T therapies.

Notably, the company is testing a single dose of the treatment, not a repeat-dosing regimen Allogene has been experimenting with to strengthen the effects of ALLO-501A. Issi indicated the decision was made for ease and convenience.

Allogenes other study, EXPAND, is a registrational trial for ALLO-647, an antibody drug the company is using to prepare patients for treatment. That study will enroll about 70 patients and have a control arm that doesnt receive Allogenes drug. Updates are expected by the end of the year, Allogene said.

Allogene shares climbed 12% in pre-market trading Friday, though at about $11, shares still trade well below their highs of about $54 apiece in 2020.

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Allogene starts first pivotal trials of an 'off-the-shelf' cell therapy for cancer - BioPharma Dive

Click to Access Audio Press Release

CHICAGO, Oct. 1, 2022 - The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the primary results from the Phase 1/2 study evaluating the investigational gene therapy botaretigene sparoparvovec (formerly AAV-RPGR) in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene. Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile, and efficacy assessments in this proof-of-concept study demonstrated encouraging improvements in retinal sensitivity, visual function and functional vision.1 These findings and additional updates, including data from a Phase 1 trial of investigational gene therapy JNJ-81201887 (JNJ-1887) for patients with geographic atrophy (GA), a late-stage and severe form of age-related macular degeneration (AMD), were presented in late-breaking oral presentations today at the Retina Subspecialty Day program of the American Academy of Ophthalmology (AAO) 2022 Annual Meeting (Abstracts #30071754 and #30071749).

XLRP is a rare condition estimated to impact one in 40,000 people globally.2,3 People with XLRP have progressive vision loss, starting in childhood with night blindness.4 Over time, they lose their peripheral vision leading to legal blindness by middle age.4 Botaretigene sparoparvovec is being investigated in collaboration with MeiraGTx Holdings plc to treat patients with XLRP caused by disease-causing variants in the eye-specific form of the RPGR (RPGR ORF15) gene. Through a one-time administration, botaretigene sparoparvovec is designed to deliver functional copies of the RPGR gene to counteract the loss of retinal cells with the goal of preserving and potentially restoring vision for those living with XLRP. Currently, there are no approved treatments for XLRP.4

"Individuals living with XLRP often begin to experience symptoms in childhood, and as retinal degeneration progresses toward blindness, they can start to feel a sense of hopelessness as there are no treatments to turn to," said Michel Michaelides, B.Sc., M.B., B.S., M.D. (Res), FRCOphth, FACS, Consultant Ophthalmologist, Moorfields Eye Hospital, Professor of Ophthalmology, University College London and lead investigator. "These results from the MGT009 study are promising, as they represent the potential for botaretigene sparoparvovec to preserve vision and ultimately restore hope for these patients."

The primary endpoint of the MGT009 study (NCT03252847) was safety, with secondary endpoints measuring changes in assessments of three domains of visionretinal sensitivity, visual function and functional visionat specified time points post-treatment.1 In the study's dose escalation and expansion phases, significant sustained or increased functional improvement in each visual domain was observed in participants treated with botaretigene sparoparvovec compared to the randomized untreated control arm of the study at six months post-treatment.1

Analyses of the pooled low and intermediate dose cohorts demonstrated improvement in retinal sensitivity in the treated eyes compared to untreated eyes in the randomized concurrent control arm as measured by both full-field static perimetry and microperimetry.1 An improvement in mean retinal sensitivity as measured by static perimetry in the central 10-degree area of the retina was observed at six months in the treated eyes compared to untreated eyes in the randomized concurrent control arm [in the full analysis of pooled low and intermediate doses across adults: 1.96 decibel (dB); (95% CI: 0.59, 3.34); and in the sensitivity analysis when applying the Phase 3 criteria: 2.42 (0.91, 3.93)].1

As part of the study, patients performed a functional vision assessment using a visual mobility maze to assess their ability to navigate through simulated real-life obstacles across a broad range of controlled light. At week 26, improvement in walk time was observed between the treated eyes in the low and intermediate dose cohorts and the untreated eyes in the randomized concurrent control arm at low illumination levels (full analysis nominal p-value < 0.05 at lux 1 and lux 16; in the sensitivity analysis when applying the Phase 3 criteria nominal p-value < 0.01 at lux 1, lux 4 and lux 16).1

The safety profile of botaretigene sparoparvovec observed in MGT009 was consistent with previous reports.1 Botaretigene sparoparvovec demonstrated an adverse event (AE) profile that was anticipated and manageable.1 Most AEs were related to the surgical delivery procedure, were transient and resolved without intervention.1 There were no dose-limiting events.1 A total of three serious adverse events (SAEs) were observed in the overall Phase 1/2 MGT009 clinical study; two SAEs, which were previously reported, were observed in the dose-escalation phase of the study (n=10; one retinal detachment and one panuveitis in the low dose cohort), and a single additional SAE of increased intraocular pressure was observed in the dose escalation phase and resolved with treatment.1

"Without an approved treatment option available, people with XLRP are faced with the inevitable fate of going blind in the prime of life," said James List, M.D., Ph.D., Global Therapeutic Area Head, Cardiovascular, Metabolism, Retina & Pulmonary Hypertension, Janssen Research & Development, LLC. "We're in a race to save sight for these patients and are encouraged by the strength of the data that we've shared so far. We look forward to advancing the clinical development of botaretigene sparoparvovec as part of our mission to preserve and potentially restore vision for these patients."

Further sensitivity analysis was conducted on study participants by applying the Phase 3 LUMEOS (NCT04671433) study eligibility criteria that corroborated the endpoints selected for the Phase 3 study.1 Currently, the LUMEOS study of botaretigene sparoparvovec for the treatment of patients with XLRP with disease-causing variants in the RPGR gene is actively dosing patients.

Phase 1 Data Evaluating JNJ-1887 in Geographic AtrophyJanssen also presented late-breaking data from a Phase 1, open-label, multicenter, dose-escalation, safety and tolerability study (NCT03144999) of a single intravitreal injection of JNJ-1887 in patients with advanced non-exudative (dry) age-related macular degeneration (AMD) with GA. GA is an irreversible condition that affects more than five million individuals worldwide.5 It has a devastating impact on GA patients' health-related quality of life, including their ability to read, drive and perform other day-to-day activities.5 In this study, patients (n=17) were sequentially enrolled at a low, intermediate and high dose without steroid prophylaxis, and all three doses of JNJ-1887 met the primary endpoint of safety over the two-year follow-up period.6 In addition, the supportive efficacy measures, including evaluation of GA lesion growth rates, showed a continual decline in lesion growth over six-month increments.6 These results are the first shared from the Company's common eye disease portfolio and indicate further evaluation of this investigational gene therapy is warranted.6

About the Phase 1/2 MGT009 Trial and Botaretigene SparoparvovecThe Phase 1/2 MGT009 trial (NCT03252847) was an open-label, multicenter dose escalation study that enrolled patients aged five years and older with X-linked retinitis pigmentosa (XLRP) caused by disease causing variants in the retinitis pigmentosa GTPase regulator (RPGR) gene at multiple sites in the United States and the United Kingdom. The primary endpoint was safety and tolerability; secondary endpoints assessed retinal sensitivity, visual function and functional vision.

The clinical study was composed of three parts: dose-escalation, pediatric dose-confirmation and an expansion phase. In the dose escalation phase, adult patients were treated at three escalating doses of botaretigene sparoparvovec; a low (2x1011 vg/mL), an intermediate (4x1011 vg/mL), and a high (8x1011 vg/mL) dose. In the expansion phase, 42 adult male patients were randomized to either immediate treatment with one of two low or intermediate doses or an untreated concurrent control arm with deferred treatment. At six months, the untreated control arm was randomized to receive either the low or intermediate treatment doses. Botaretigene sparoparvovec was administered through subretinal delivery in only one eye. The adult patients received treatment at three doses. The pediatric cohort (n=3) was only treated with an intermediate dose of botaretigene sparoparvovec.

Botaretigene sparoparvovec has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME), Advanced Therapy Medicinal Product (ATMP) and Orphan designations by the European Medicines Agency (EMA).

About the Janssen and MeiraGTx Strategic CollaborationIn January 2019, Janssen Research & Development, LLC entered into a worldwide collaboration and license agreementwith MeiraGTx Holdings plc, a clinical-stage gene therapy company, to develop, manufacture and commercialize its clinical-stage inherited retinal disease portfolio. Botaretigene sparoparvovec is being developed as part of a collaboration and license agreement. In addition to forming a research collaboration to explore new targets for other inherited retinal diseases, Janssen is working with MeiraGTx to build core capabilities in viral vector design, optimization and manufacturing.

About the Phase 1 JNJ-1887 Trial and JNJ-1887JNJ-81201887 (JNJ-1887), formerly referred to as AAVCAGsCD59, is an investigational gene therapy for the treatment of people with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). JNJ-1887 is designed to increase the expression of a soluble form of CD59 (sCD59) intended to protect retinal cells to slow and prevent disease progression. JNJ-1887 was evaluated in a Phase 1 clinical trial (NCT03144999), an open-label, single-center dose escalation study to determine the safety of JNJ-1887 in adults 50 or older with advanced dry AMD with GA. The patients were treated at three escalating doses of JNJ-1887 without steroid prophylaxis through a single intravitreal injection in one eye.

This Phase 1 study met its primary endpoint of safety in all doses of JNJ-1887 (n=17), with supportive efficacy measures including evaluation of GA lesion growth rates, which showed a continual decline in lesion growth over six-month increments.

JNJ-1887 has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) and Advanced Therapy Medicinal Product (ATMP) designation by the European Medicines Agency (EMA).

About the Janssen Pharmaceutical Companies of Johnson & JohnsonAt Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism, & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.

Learn more at http://www.janssen.com. Follow us at@JanssenGlobal. Janssen Research & Development, LLC is part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

Dr. Michaelides is a scientific founder of, and consultant to, and has a financial relationship with MeiraGTx.

Cautions Concerning Forward-Looking StatementsThis press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding botaretigene sparoparvovec and JNJ-81201887. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, any of the other Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year endedJanuary 2, 2022, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online atwww.sec.gov,www.jnj.comor on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

References1Michaelides, M et al. Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP). Abstract #30071754. Presented at the 2022 American Academy of Ophthalmology Annual Meeting.

2Boughman JA, Conneally PM, Nance WE. Population genetic studies of retinitis pigmentosa. Am J Hum Genet. 1980;32(2):223235.

3Fishman GA. Retinitis pigmentosa. Genetic percentages. Arch Ophthalmol. 1978;96(5):822826. doi:10.1001/archopht.1978.03910050428005.

4 Wang DY, Chan WM, Tam PO, et al. Gene mutations in retinitis pigmentosa and their clinical implications. Clin Chim Acta. 2005;351(1-2):5-16.

5 Cohen, MN et al. Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy (GA) Due to Age-related Macular Degeneration (AMD). Abstract #30071749. Presented at the 2022 American Academy of Ophthalmology Annual Meeting.

6 Singh RP, Patel SS, Neilsen JS, et al. Patient-, caregiver- and eye care professional-reported burden of geographic atrophy secondary to age-related macular degeneration. Am J Ophthalmic Clin Trials. 2019;2(1):1-6.

Investor Contact:Raychel KruperOffice +1 732-524-6164rkruper@its.jnj.com

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Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting - Johnson &...

Essential that patients have the information they need to make informed choices in the future

Australians with inherited retinal disease (IRD) have a strong interest in undergoing gene therapy to prevent and treat blindness but theres a critical need for education programmes to help them make informed choices about future treatments, new research shows.

IRD is the umbrella term for broad group of genetic eye conditions, including retinitis pigmentosa and Stargardts disease, that cause progressive vison loss and blindness. They are the most common cause of blindness in working-age Australians, affecting more than 13,000 people nationally.

The study, led by the Centre for Eye Research Australia and University of Melbourne,reveals the results of the first national survey asking Australians living with IRD and their carers about their knowledge and views on gene therapy.

It provides new insight into patients knowledge of emerging gene therapies, the methods used, their willingness undergo future treatments and their views on the potential costs and logistics.

The findings demonstrated the need for continuing, targeted education about the outcomes and risks of gene therapy, and the difference between clinical research and approved treatments.

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Education vital to help patients explore gene therapies for blindness: Australian study - BSA bureau

Acquisition of select PACT assets enables AmplifyBio to expand beyond safety, efficacy, and toxicology services to enable "living medicine" developers to shorten timelines and mitigate risk when moving to the clinic

WEST JEFFERSON, Ohio and SOUTH SAN FRANCISCO, Calif., Oct. 3, 2022 /PRNewswire/ --AmplifyBio, a contract research organization (CRO) focused on accelerating innovation across pharmaceutical modalities; today announced the acquisition of select assets fromPACT Pharma, Inc., a privately held biopharmaceutical company developing neoantigen-specific T cell receptor cell therapies. The deal will provide AmplifyBio with advanced characterization platforms, bioinformatics capabilities, and 40 drug development experts to enhance their cell and gene therapy service offerings. AmplifyBio will also acquire the South San Francisco advanced laboratory space.

With the acquisition of these assets, AmplifyBio aims to provide an early, consistent characterization of a treatment's purity, potency, and viability throughout the life cycle of therapeutic development. Unlike small molecules, there is no single, consistent process for cell and gene therapy companies to research, develop, and test their therapeutics. The gap that exists in characterization between the discovery phase and preclinical testing leads to material changes in a therapeutic during development, which can in turn create manufacturing inconsistencies and safety concerns during scale-up.

"Many biologics developers have adopted the phrase 'the process is the product' to describe how their therapeutic is differentiated based on a unique development process," said AmplifyBio Chief Executive Officer (CEO) and President J. Kelly Ganjei. "Rather than create our own, individual technique, AmplifyBio aims to replace that saying with a new one: 'the product is the product. Our acquisition of these assets from PACT Pharma means that cell and gene therapies can now be differentiated based on safety and efficacy profiles and specific product characteristics, not development processes."

"This deal allows PACT to retain its core intellectual property and continue our mission of developing novel, neoantigen-targeted T-Cell Therapies," added Scott Garland, PACT Pharma's CEO. "At the same time, we're working with AmplifyBio to leverage our platforms to offer a unique combination of optimization, characterization, safety and efficacy services to a wider range of clients seeking to better understand the immunology of their adoptive cell therapies."

AmplifyBio was spun out in 2021 from Battelle, a not-for-profit organization that advances science and technology to have the greatest impact on our society and economy. Following today's acquisition of the South San Francisco facility, AmplifyBio plans to add a third site in New Albany, Ohio that consists of 350,000 square feet of multi-function lab spaces. There, AmplifyBio will build on its advanced therapy services by adding capabilities for complex genotypic and phenotypic characterization analysis for late-stage development. The company expects to add additional development platforms and partnerships to become a commercial accelerator delivering safe, effective, reproducible advanced therapies to patients.

About AmplifyBioAmplifyBio is a leading preclinical CRO focused on toxicology, safety, and pharmacology testing to advance therapeutics for the betterment of human health. Spun out of Battelle in May of 2021, AmplifyBio's mission is to continue to provide exceptional CRO study services in an agile environment better suited to commercial goals and expand analytic capabilities to serve the dynamic needs of advanced therapy development. Clients of AmplifyBio enjoy the peace of mind that comes from decades of experience in GLP and non-GLP study design and execution, combined with rapid investment in technology, expertise, and infrastructure that together provide the critical components of a reliable, agile partnership.

Media Contact AmplifyBio[emailprotected]For Inquiries to PACT[emailprotected]

SOURCE AmplifyBio

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AMPLIFYBIO ACQUIRES PACT PHARMA ASSETS TO ENHANCE CELL AND GENE THERAPY CHARACTERIZATION CAPABILITIES - PR Newswire

Demand for viral-vector-based gene therapies has risen to unprecedented levels, thanks to their potential to help treat previously incurable diseases. The two vectors most in the spotlight? Lentiviral (LV) vectors and adeno-associated viral (AAV) vectors due to the increased research and positive clinical results they are seeing across a wide range of applications, including cancer, heart disease, and hematologic and genetic disorders. The more drug developers look to expand this range of therapeutic areas, the greater the demand for commercial-scale development. So its important to understand not only how these two vectors can be applied to drug development, but also the capabilities required for scale-up that allows us to bring these innovative therapies to patients.

LV vectors are derived from the single-stranded RNA retrovirus HIV-1, and have been used extensively because of their ability to infect non-dividing cells, efficiently integrate into the host genome, carry large transgene loads, and allow for long-term transgene expression. They are predominantly used as delivery vehicles for introducing genetic modifications into cell therapies, such as CAR-T, and HSC gene therapies. Importantly, recent regulatory approvals and clinical successes with LV vectors are spurring even more interest among drug developers.

Lets look at the benefits of LV vectors in more detail:

However, LV vectors also present two major risks to safety.

The first is a risk of accidental exposure because HIV can self-replicate during manufacturing thanks to the lentiviruss high mutation and recombination rate.Though research shows that the risk is low, it remains a major safety concern for lab engineers and workers during development. Before using a lentiviral vector system, a risk assessment must be completed and documented. Typically, lentiviral vectors may be safely handled using either BSL-2 or BSL-2 enhanced controls, depending upon the risk assessment.

The second risk is the potential for oncogenes to occur in cells through insertional mutagenesis. For this reason, lentiviral vectors are predominantly used for cell therapy applications with genetic modification of cells ex-vivo. Only limited use is seen for direct in vivo therapies.

Unlike their LV cousins, AAV vectors are single-stranded DNA parvoviruses that can replicate only in the presence of helper viruses, such as the adenovirus, herpes virus, human papillomavirus, and vaccinia virus. Following several landmark approvals, AAV vectors are currently being used for in vitro, ex vivo, and in vivo research. AAV therapies predominantly target rare genetic disorders for which the patient population tends to be highly limited. As the market is so small, drug developers feel immense pressure to be first to market to commercialize their therapies.

The biological elements of AAV vectors make them a very attractive candidate for gene therapy for several reasons:

As with LV vectors, AAV vectors come with several drawbacks that affect their applications and efficiency.

Firstly, AAV vectors are limited by their restricted capacity for insertion of transgene DNA; because of their relatively small transgene size, they are unable to deliver genes larger than 4.8 kilobytes. Secondly, the generation of neutralizing antibodies against AAV in non-human primates (NHP) and humans may attenuate the curative effects of AAV-mediated gene therapies and limit the size of patient populations suitable for these therapies. Thirdly, there are several different serotypes and capsids for AAVs, all of which have different production and purification requirements and vary greatly with respect to function and efficacy. Fourthly, AAV drug products have varying degrees of empty and partially filled capsids, and these have implications for safety and efficacy. Generally, the highest possible percentage of AAV particles with the full transgene DNA is desired, and this varies significantly depending on the production method, AAV serotype, and the transgene itself. The latter two factors introduce significant manufacturing challenges for AAV therapies.

Overall, the industrys collective ability to successfully scale up LVV and AAV vectors faces two challenges:

i) Manufacturing each viral vector currently requires different processes, so companies cannot apply a one-size-fits-all approach to their upstream and downstream processes. Therefore, manufacturing requires immense scientific and market expertise to make the informed decisions necessary for developing a robust plan.

ii) Given the industrys limited experience with commercial-scale viral vector supply, companies need to work closely with regulatory agencies. This can be especially challenging during the transition from preclinical to commercial, where complexities arise that can cause potential delays resulting in increased costs.

As demand continues to rise, pharma companies must understand how to navigate these challenges to continue delivering their life-saving medications.

Head of Commercial Development for Viral Vector, Cell and Gene Technologies (CGT) at Lonza

She works closely with the innovation, operations, engineering, strategic marketing, and business teams to enable prioritization, strategic development and commercialization of viral vector production services for CGT. Suparnas background is in Neuroscience, and she earned her PhD in Neuropharmacology from the University of Toronto. She has over 15 years of broad pharmaceutical and CDMO experience driving innovation, drug discovery, product and service development for CNS, oncology, and cell and gene therapy.

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The Pros and Cons of Lentiviral and Adeno-Associated Viral Vectors - The Medicine Maker

Gene and cell-based treatment is promising solutions for the control and cure of some chronic and life-threatening diseases such as sickle-cell disease (SCD), haemophilia, blood cancers, and HIV. Most of the current gene therapy clinical trials on SCD and HIV are conducted in North America.The treatment is either by using someone elses cells or those of the patient. Gene therapy, also called genetic engineering, involves getting ones cells (a patient), improving them either by enhancing them to fight disease or as a replacement for the diseased cells and using them to treat the disease.

Unlike in agriculture where a lot of the genetic engineering is on seed, Dr Cissy Kityo, the executive director at Joint Clinical Research Centre (JCRC) says in medicine, the human seed (ova or sperm) or the embryo is not touched.Its not about engineering custom humans as this has no current ethical basis. Therefore, it presents a new treatment paradigm, Dr Kityo says.

Gene therapy is administered once in a lifetime. Therefore, for someone with HIV, that eliminates the burden of taking ARTs. It also has the potential to save the overall healthcare cost and increase the individuals productivity.Research is ongoing to ensure this treatment is effective, safe, and free from off-target effects and any contamination.

The processDr Francis Ssali, the deputy executive director in charge of clinical care and research at JCRC, says genetic modification involves a series of processes, the first of which is to collect specialised white blood cells called T-cells and blood-forming stem cells from the patients blood.These cells are then taken to a clean medical laboratory where they are counted, checked for viability, and purified. Thereafter, the gene to correct the disease is inserted into these cells and this is done by either using special enzymes called CRISPR or by the use of self-inactivating partial viruses called Lentiviral vectors. The lentiviral vector delivers the required gene into the cells without resulting in viral infection in the patients cells, he says.

The process of introducing the corrective gene into the patients cell is called transduction and it can take between four to seven days to perform in the laboratory. Once the cells have received this gene modification, they are checked for quality and safety before they are ready for reinfusion back into the patient.In some instances, the patient is given medical treatment to enable them to receive the gene therapy cells, he adds.However, Dr Ssali says the current approaches to gene-therapy cell manufacturing are labour intensive and take a relatively long time to prepare, and require a large clean laboratory space.

Thankfully, there are newer laboratory instruments that can automate this genetic engineering work in a single closed instrument, with efficiency, he says.Uganda has 1.4 million people living with HIV and 400,000 people living with sickle cells yet adherence to medicine is inconsistent for some.Some HIV-resistant viral variants have emerged which threaten the efficacy of the treatment programme. As such, genetic engineering will be a blessing.Globally, the first-generation cure trials for HIV were done, second-generation trials are coming up and there is hope that soon a short-term cure will be got.

Ugandan perspectiveIn Uganda, Dr Ssali says the hope is that by 2030, Uganda will have controlled HIV/Aids greatly and also contributed to finding a functional cure.Dr Kityo says JCRC hopes to start HIV gene therapy trials in Uganda in 2024.The other focus is technology transfer where these gene therapy products are produced where they are needed, more efficiently, and more cost-effectively. That is why there will be more compact systems rather than the large labs, she adds.

In Africa, Uganda ranks fifth among countries with sickle cell disease and whereas bone-marrow transplants can cure SCD, only 10 percent of the eligible patients can get a matched donor. Nonetheless, with gene therapy, this will not be an issue since the patients own cells are used.Thankfully, the current gene therapy treatment technologies for HIV are the same used in sickle cell cure research. That is why preparing to address HIV also works to tackle the sickle cell disease, Dr Kityo says.

The joint Clinical Research Centre is working towards building the research teams and creating the necessary infrastructure for this novel research and clinical care. Arthur Makara, the coordinator of Uganda Biotechnology and Biosafety Consortium, calls for several partnerships because even when JCRC creates these technologies, they need help to mass produce them for a bigger population. Gene therapy only works on an individual, not on the sperm or ovary. Therefore, Dr Kityo says even after treatment, a sickle cell patient will still have sickle cell gene but normal cells in their marrow and live a normal life.

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Gene therapy brings hope to people with sickle cell, HIV - Monitor

(Precision Vaccinations)

Encouraging news from researchers at Fred Hutchinson Cancer Center in Washingtonrecently showed that an experimental gene therapy for herpes simplex virus infectionsmaysuppressthe amount of transmissible virus.

Drs.Keith JeromeandMartine Aubert, the virologists leading the research effort, reported the innovative treatment dramatically reduced or even eliminated viral shedding in treated mice compared to controls.

During multiple experiments, the researchers found substantial reductions in oral and genital viral shedding in the treated mice, with many of those treated showing no detectable virus shed.

This is an important milestone since the U.S. FDA has not approved any herpes prevention vaccine as of October 4, 2022.

"If you ask people living with Herpes simplex virus (HSV)what they care about, what they care most about is whether they have to worry about giving this virus to someone else, and shedding is how that happens," Dr. Jerome said in a media release issued on September 26, 2022.

Previously,Jerome and Aubert reportedthat the experimental drug could eliminate more than 90% of the latent herpesvirus in nerve clusters near the faces of the mice injected with the enzyme-carrying adeno-associated viruses (AAVs).

In this new study, they describe how they have tested the therapy for the first time to treat infections in a cluster of nerves called dorsal root ganglia near the genital tract of mice.

They found the experimental therapy reduced the latent herpes virusby about97%.

These results reinforce the curative potential of gene editing for latent orofacial and genital HSV disease, and provide a framework for additional safety studies before human trials can begin, wrote these researchers in a non-peer-reviewed study published on September 26, 2022.

HSV establishes latency in ganglionic neurons of the peripheral nervous system, from which it can reactivate, causing recurrent disease and possible transmission to a new host.

Current anti-HSV therapy does not eliminate latent HSV and thus is only suppressive rather than curative.

These researchers developed a potentially curative approach to latent HSV infection and pathogenesis based on gene editing using HSV-specific meganucleases delivered by AAVvectors.

This non-human study's results demonstrated that a dual meganuclease therapy, composed of two anti-HSV-1 meganucleases delivered by a triple AAV serotype combination (AAV9, AAV-Dj/8, AAV-Rh10), can eliminate up to 97% of latent HSV DNA from ganglia in both ocular and vaginal mouse models of latent HSV infection.

Using a novel pharmacological approach to reactivate latent HSV-1 in mice with the bromodomain inhibitor JQ-1, theydemonstrated that this reduction in ganglionic viral load leads to a significant reduction of viral shedding from treatedvs. control mice, with many treated mice showing no detectable virus shedding.

In general, the novel therapy was found well tolerated, although dose-ranging studies showed hepatotoxicity at high AAV doses, consistent with previous observations in animals and humans.

Also, in agreement with previous literature, theyobserved subtle histological evidence of neuronal injury in some experimental mice, although none of the mice demonstrated observable neurological signs or deficits.

The Hutch scientists noted that this study'spositivenews is tempered by recentconcerns within the gene therapy fieldabout the potential for therapies using AAVs to cause liver and nerve damage.

Jerome and Aubert are still hopeful that they can get FDA approval to test the therapy in humans in an early-stage clinical trialbefore the end of 2023.

The work was funded by the U.S. NIH, the Caladan Foundation, and more than 1,600 individual donors. Cellectis developed the meganucleases used in this research.

Disclosure note:Fred Hutch and certain of its scientists may benefit financially from this work in the future.

PrecisionVaccinations publishes fact-checked, research-based vaccine news manually curated for mobile readers.

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Herpes Gene Therapy Further Refined in Mice - Precision Vaccinations

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Various factors are responsible for the market's growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy market. This report is a consolidation of primary and secondary research, which provides market size, share, dynamics, and forecast for various segments and sub-segments considering the macro and micro environmental factors. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market.

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Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market will reach at $ 543.23 mn by 2032 demand and future scope with Russia-Ukraine Crisis...

Bloomsbury Genetic Therapies launches with Seed financing of 5 million to develop potentially curative gene therapy treatments for rare neurological and metabolic diseases

London, UK, 7 October 2022 Bloomsbury Genetic Therapies Limited (Bloomsbury), a biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases, based on clinically proven gene therapy technologies, today announced its launch with 5 million Seed financing led by UCL Technology Fund.

Bloomsburys innovative approach is designed to optimise therapeutic efficacy and safety, enable high manufacturability, accelerate development timelines and maximise regulatory success to create a pipeline of highly differentiated first- or best-in-class programs. The Companys lead programs are liver and CNS targeted gene therapies:

Liver targeted therapies: BGT-OTCD: Ornithine Transcarbamylase Deficiency (OTCD)

CNS targeted therapies: BGT-DTDS: Dopamine Transporter Deficiency Syndrome (DTDS)

BGT-NPC: Niemann-Pick Disease Type C (NPC)

BGT-INAD: Infantile Neuroaxonal Dystrophy (INAD)

These programs are underpinned by the unique medical insights derived from the internationally recognised clinics run by the companys academic founders, a deep understanding of disease biology and world-class translational science resulting from the close collaboration between Bloomsburys academic founders. Additionally, the viral vectors in the Companys programs have been designed with well-characterised adeno-associated virus (AAV) capsids that have demonstrated success in clinical and/or commercial settings. They have also been combined with innovative cassettes and routes of administration designed for optimal therapeutic effect.

Bloomsbury is developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases, said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. Our approach has been designed with a single goal in mind - to deliver the promise of gene therapy to rare disease patients by leveraging our industrys existing state-of-the art technology and we have already attracted a highly talented team of founders and experienced scientific and business professionals to the Company to that effect as we progress our first liver targeted gene therapy program, BGT-OTCD for the treatment of Ornithine Transcarbamylase Deficiency, into the clinic.

Our commitment to Bloomsbury is a great example of our strategy to seek to build truly innovative companies anchored by exceptional science and experienced teams, added Leigh Brody, Investment Manager of UCL Technology Fund. We are excited by the potential to develop best-in-class products in the gene therapy space.

Bloomsbury was founded as a spinout from UCL, and was supported by UCL Business, who helped foster the progression of these translational projects, supporting the team whilst managing the intellectual property, and making this deal possible. Bloomsbury is underpinned by world-leading gene therapy and rare disease expertise from the Companys academic founders:

After years of working relentlessly to identify and develop potential treatments for OTCD, NPC, DTDS and INAD, we are excited to have partnered with the team at Bloomsbury and are confident that these programs are in safe hands to be progressed through the development stage and towards regulatory approvals, with an ultimate goal of providing curative therapies to patients, added Professors Gissen, Kurian, Rahim and Waddington.

Senior leadership

Bloomsbury is led by Co-Founder & Chief Executive Officer, Adrien Lemoine, who brings 20 years of experience in pharma and biotech, including senior commercial, strategy and operation roles at AstraZeneca and GSK and joined the Company from Orchard Therapeutics where he served as Chief Business Officer. In this role he played a leading part in the companys fundraising efforts (Series B, Series C, NASDAQ IPO and follow-on financing) which raised over $750M and built the companys pipeline as well as setting-up Orchards discovery research efforts, alongside managing the program leadership & management function.

Alongside him on the senior leadership team are:

Board of Directors

Bloomsbury has also assembled an impressive trio of industry leaders as Independent Directors to assist management in driving the business forward; namely:

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Bloomsbury Genetic Therapies launches with Seed financing of 5 million to develop potentially curative gene therapy treatments for rare neurological -...

The prespecified exploratory analyses of the secondary survival endpoint demonstrated a >90% reduction in risk of death alone or in risk of death/permanently assisted ventilation at 24 weeks, when adjusted for baseline imbalances in risk (p=0.028 to p=0.075, unadjusted for multiple comparisons) with the CNM-Au8 30 mg dose. These survival results were statistically consistent for the 30 mg dose between the regimen only and full analysis sets, which included shared placebo from other regimens participating in the Healey ALS Platform trial (Regimens A, B, and D). This survival signal is consistent with results previously reported by Clene in the Phase 2 RESCUE-ALS trial with CNM-Au8.

The full analyses, including data on biomarkers of neurodegeneration and exploratory efficacy results, are expected later in 2022. The open-label extension will continue to follow participants and provide data updates in the future. Clene is in discussions with the Healey & AMG ALS Center to offer a broader EAP of CNM-Au8 30 mg for eligible participants of closed regimens and others.

Based on these topline findings, Clene has selected the CNM-Au8 30 mg dose for continued development in ALS. The CNM-Au8 60 mg dose did not demonstrate a survival benefit. CNM-Au8 was well-tolerated, and there were no drug-related serious adverse events or significant safety findings reported.

"There remains a high unmet medical need for treatments for people living with ALS. The potential survival benefit with CNM-Au8 at 30 mg is encouraging. Additional pre-specified exploratory analyses of both the RCT and open-label extension part of the study will be shared once available," said Merit Cudkowicz, M.D., MSc, principal investigator and sponsor of the Healey ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chief of the Department of Neurology at Massachusetts General Hospital, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. "We are thankful to the many people who participated in this study. We will learn from these results and continue to use these data to inform future advances in ALS trial design," she concluded.

Robert Glanzman, M.D., FAAN, Clene's Chief Medical Officer, said, "We are very pleased to see a survival benefit in a broad population of people who had already been living with ALS for up to three years. Importantly, this is the second Phase 2 study demonstrating a survival benefit following CNM-Au8 treatment. CNM-Au8's mechanism of enabling energy metabolism and efficiency may not be reflected in the slope of ALSFRS-R change after only 24 weeks of treatment. These Healey ALS Platform Trial results support advancement of the CNM-Au8 30 mg dose. We look forward to discussions with U.S. regulatory authorities at an End of Phase 2 meeting for our CNM-Au8 development program in ALS."

Rob Etherington, Clene's President and CEO, added, "The survival results from this trial together with the consistent benefit seen in the open-label extension of the Phase 2 RESCUE-ALS trial, based on up to 31.5 months of long-term follow-up, support the rationale for treating neuronal and glial energetic failure with CNM-Au8. We have now completed multiple Phase 2 studies in ALS and MS, building a body of evidence demonstrating that CNM-Au8 supports cellular energy production, improving myelination and neuronal viability. We believe supporting brain energetic capacity translates to patient benefit, including survival. We will work closely with regulatory health authorities, ALS experts, and patient representatives to determine the proper path for FDA and EMA approval. Clene remains committed to advancing CNM-Au8 clinical programs to the ultimate goal of FDA approval. To support this effort, Clene is pursuing paths, including strategic partnerships, and is in dialogue with various potential partners."

Michael Hotchkin, Clene's Chief Development Officer, concluded, "We thank the ALS community for its support of the Healey ALS Platform trial. Furthermore, we thank the site investigators for their research excellence and dedication to patients, and we thank Dr. Cudkowicz and the team at the Healey & AMG ALS center for their leadership and for the development of the platform trial. Most importantly, we thank people living with ALS who participated in the study and their families for their effort and willingness to engage in clinical research."

Conference Call and Webcast Information

Clene will host a conference call and webcast at 8:30 am EDT to discuss the Healey ALS Platform trial topline results for CNM-Au8. Members of Clene's executive team will lead the discussion.

Time and Date: 8:30 a.m. EDT on Oct. 3, 2022 Investors: 1 (888) 660-6179 (toll-free) or 1 (929) 203-1946 (toll) Conference ID: 5318408 Press *1 to ask or withdraw a question, or *0 for operator assistance .

To access the live webcast, please register online at this link . Participants are requested to register at a minimum 15 minutes before the start of the call. A replay of the call will be available two hours after the call and archived on the same web page for six months. A live audio webcast of the call will be available on the Investors section of the Company's website Events page. An archived webcast will be available on the Company's website approximately two hours after the event.

About the Healey ALS Platform Trial The Healey ALS Platform Trial is a perpetual multi-center, randomized, double-blind, placebo-controlled program designed to evaluate the efficacy and safety of multiple investigational products utilizing a shared placebo group in people living with amyotrophic lateral sclerosis (ALS). In the CNM-Au8 regimen, 161 participants were randomized to 30 mg CNM-Au8, 60 mg CNM-Au8, or placebo as adjunct to standard of care for a 24-week treatment period. Active drug was offered to all participants who were eligible and elected to continue into the open-label extension. The primary outcome of the trial was the change in disease severity over time as measured by ALSFRS-R through 24 weeks accounting for mortality (analyzed using a Bayesian shared parameter model). Prespecified secondary efficacy endpoints included the Combined Assessment of Function and Survival joint rank test (CAFS), change in respiratory function as measured by slow vital capacity (SVC), and overall survival. For more information, please see ClinicalTrials.gov Identifier: NCT04297683 .

About CNM-Au8 CNM-Au8 is Clene's lead asset in mid- and late-stage clinical development for the treatment of multiple sclerosis and amyotrophic lateral sclerosis. An oral suspension of gold nanocrystals, CNM-Au8 was developed to protect neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8 is a federally registered trademark of Clene Nanomedicine, Inc.

About Clene Clene is a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative disease by targeting energetic failure, an underlying cause of many neurological diseases. The company is based in Salt Lake City, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit http://www.clene.com or follow us on Twitter , LinkedIn and Facebook.

Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended, which are intended to be covered by the "safe harbor" provisions created by those laws. Clene's forward-looking statements include, but are not limited to, statements regarding our or our management team's expectations, hopes, beliefs, intentions or strategies regarding our future operations. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words "anticipate," "believe," "contemplate," "continue," "estimate," "expect," "intends," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "will," "would," and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements represent our views as of the date of this press release and involve a number of judgments, risks and uncertainties. We anticipate that subsequent events and developments will cause our views to change. We undertake no obligation to update forward-looking statements to reflect events or circumstances after the date they were made, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Accordingly, forward-looking statements should not be relied upon as representing our views as of any subsequent date. As a result of a number of known and unknown risks and uncertainties, our actual results or performance may be materially different from those expressed or implied by these forward-looking statements. Some factors that could cause actual results to differ include our ability to demonstrate the efficacy and safety of our drug candidates; the clinical results for our drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; our ability to achieve commercial success for our drug candidates, if approved; uncertainty regarding whether potential strategic partnerships will result in any agreements or transactions, or, if completed, any agreements or transactions will be successful or on attractive terms; our limited operating history and our ability to obtain additional funding for operations and to complete the development and commercialization of our drug candidates; and other risks and uncertainties set forth in "Risk Factors" in our most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q. In addition, statements that "we believe" and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this press release, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned not to rely unduly upon these statements. All information in this press release is as of the date of this press release. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this press release.

Source: Clene Inc.

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Ginkgo Bioworks Acquires Circularis to Strengthen Capabilities in Cell and Gene Therapy - Investing News Network

DetailsCategory: DNA RNA and CellsPublished on Friday, 07 October 2022 11:34Hits: 395

Enrollment continues in clinical trial of MB-106 under Mustangs IND; next data disclosure anticipated 4Q 2022

Ongoing clinical trial of MB-106 at Fred Hutch continues to demonstrate high efficacy, durable responses, and favorable safety profile across wide range of hematologic malignancies

WORCESTER, MA, USA I October 06, 2022 I Mustang Bio, Inc. (Mustang) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the first patient has been treated in its multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106, Mustangs first-in-class CD20-targeted, autologous CAR T cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas (B-NHL) and chronic lymphocytic leukemia (CLL). The patient did not experience cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center (Fred Hutch). The multicenter trial under Mustangs Investigational New Drug Application (IND) builds upon the initial, ongoing Phase 1/2 clinical trial taking place at Fred Hutch in a single-center study under Fred Hutchs IND.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang said, The first clinical trial under Mustangs IND is an important milestone in the ongoing development and evaluation of MB-106. Data presented at several prestigious medical meetings earlier this year from the initial, ongoing Phase 1/2 clinical trial at Fred Hutch show that MB-106 continues to demonstrate high efficacy and a favorable safety profile across patients with a wide range of hematologic malignancies. We look forward to providing updates on our multicenter MB-106 clinical trial as it progresses and anticipate reporting efficacy data in the fourth quarter of this year.

Interim data from 28 patients treated in the initial, ongoing Phase 1/2 investigator-sponsored clinical trial at Fred Hutch continue to support MB-106 as a viable CAR T cell therapy for B-NHLs and CLL. As of September 9, 2022, the interim data show:

We are excited to broaden the evaluation of MB-106 with this multicenter clinical trial under Mustangs IND. To date, the data from the initial, ongoing clinical trial at Fred Hutch continue to demonstrate a high rate of complete and durable responses, said Mazyar Shadman, M.D., M.P.H., Study Chair, Associate Professor and physician at Fred Hutch and University of Washington. In addition, MB-106 has shown potential to treat patients in an outpatient setting and provide another immunotherapy option for patients treated previously with CD19-directed CAR T cell therapy.

About Mustangs Multicenter MB-106 Phase 1/2 clinical trialThe six-center Phase 1/2 clinical trial is a three-arm study targeting CLL and B-NHL including FL, diffuse large B-cell lymphoma and mantle cell lymphoma. Included in the eligibility criteria are patients who have relapsed after treatment with CD19 CAR-T cell therapy. Additionally, the FL arm will evaluate other indolent histologies including Waldenstrom macroglobulinemia, a rare type of B-NHL for which the U.S. Food and Drug Administration recently granted MB-106 Orphan Drug Designation. Since the Mustang-sponsored multicenter clinical trial is using the same lentiviral vector as the Fred Hutch-sponsored single-center trial, the FDA has allowed dose escalation to begin at a higher dose than what was originally conducted at Fred Hutch.

An estimated 287 patients are anticipated to be enrolled in the trial. All patients must have evidence of CD20 expression in both phases of the clinical trial. In Phase 1, escalating MB-106 dose levels will be tested independently in each arm using a 3+3 design. Patients will be enrolled in one of three arms, based on their primary diagnosis.

A total of up to 18 patients are anticipated to be treated in each Phase 1 arm, including six patients at the maximum tolerated dose, prior to proceeding to the Phase 2 portion of the study for each respective arm, where a total of up to 71 patients will participate in each independent arm. Safety of each dose level will be reviewed for each arm until the maximum tolerated dose has been reached and the recommended Phase 2 dose (RP2D) has been established for each arm. An assessment of the safety and tolerability of the dose will be made by the Safety Review Committee based on the data from the 28-day dose-limiting toxicity observation period.

In Phase 2, specific arms of relapsed or refractory CD20-positive B-cell NHL or CLL patients will be treated with MB-106 at the respective RP2D for each arm. Each arm will initially include up to 20 patients. Based on the results of the interim analysis, up to an additional 51 patients may be added to each of the arms.

Additional information about the trial can be found on clinicaltrials.gov using the identifier NCT05360238.

About MB-106 (CD20-targeted autologous CAR T Cell Therapy)CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustangs research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and was exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody. MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients cells to create the MB-106 drug product produced at Mustang Bios Worcester, MA, cell processing facility for administration in the multicenter Phase 1/2 clinical trial under Mustang Bios IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on these trials can be found athttp://www.clinicaltrials.govusing the identifierNCT05360238for the Mustang multicenter trial andNCT03277729for the ongoing trial at Fred Hutch.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as lentiviral gene therapies for severe combined immunodeficiency. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit http://www.mustangbio.com.

SOURE: Mustang Bio

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Mustang Bio Announces First Patient Treated in Its Multicenter Phase 1/2 Clinical Trial of MB-106, a First-in-Class CD20-targeted, Autologous CAR T...

For medical geneticist Dr. Xiao-Ru Yang, BSc 13, MD16, few things are more rewarding than helping a patient understand what caused their mysterious, unexplained illness answers those patients often seek for many years.

Thanks to a new University of Calgary fellowship generously supported by Albertas only neurogeneticist, Dr. Oksana Suchowersky, MD 78, and her partner, the board chair of the Alberta Cancer Foundation, Dr. Chris Eagle, BSc 73, MD 77, Yang is poised to help sleuth out new diseases and improve health while adding Canadian capacity in the rapidly advancing field of neurogenetics.

There are so many diagnostic odyssey families, who get tests and investigations done for many years, but still have no diagnosis. To be able to tell these families, I think we actually have the answer now and see their reaction, is really incredible, she says.

Every case is different. It's really like solving a puzzle and you're motivated to continue to work and study these things until you do.

Yang is the first recipient of Canadas first clinical fellowship in neurogenetics and medical genetics, the 12-month Dr. Oksana Suchowersky and Dr. Chris Eagle Clinical Fellowships, offered alternating years at UCalgary and the University of Alberta.

The fellowship provides up to $100,000 to support training for exceptional clinical fellows who have completed residency in neurology or medical genetics. It allows trainees to conduct clinical or translational research or advanced subspecialty training in the area of neurogenetics (the study of the role of genetics in the development and function of the nervous system) or genetics (the diagnosis and management of genetic disorders).

Cumming School of Medicine alumni and UCalgary philanthropists, Oksana Suchowersky and Chris Eagle.

Courtesy Oksana Suchowersky and Chris Eagle

Over the past number of years, genetics has mushroomed its becoming an important point in how we diagnose and even treat patients. We're starting to use gene therapy in a lot of different specialties, says Suchowersky.

And yet in medical school, students receive only about seven hours of general training in genetics, and funding for clinical fellowships is very limited. That started the discussions between Chris and myself about the importance in developing expertise in genetics.

The donors have worn many hats in Albertas health care and education communities. Suchowersky is an adjunct professor and former department head of medical genetics at UCalgary. She is a professor in the departments of Medicine (Neurology), Medical Genetics and Paediatrics at the University of Alberta and director of the Neurogenetics and Huntington disease program.

Eagle is a past president and CEO of Alberta Health Services and past CEO of Calgary Health Trust. He is also a professor emeritus, former department head of anesthesiology and assistant dean of medical education at UCalgarys Cumming School of Medicine (CSM). He says:

We've watched generations of talented young physicians leave Alberta to get training elsewhere sometimes they return, sometimes they don't. This fellowship is an opportunity to keep or bring people here. Its also about making the care thats given in Alberta the best in the world.

Yang started her training this summer under the supervision of Dr. Francois Bernier, MD, Dr. Billie Au, MD, PhD and Dr. Mike Innes, MD, at the Alberta Childrens Hospital (ACH) all international leaders in their field. Shell learn how to analyze genomic data and how to ultimately apply emerging genetic sequencing technologies in the clinic.

The funding of this fellowship is transformational, supporting advanced training of the some of the best physicians in one of most rapidly advancing fields of medicine. Canada desperately needs academic and physician leaders to maximize the impact of genomic medicine and neurogenetics and UCalgary have consistently been at the forefront of these fields, says Bernier, professor and head of Medical Genetics.

Dr. Yang will be among the new generation of leaders in genomics thanks to this fellowship.

Rapid advances in genomics the study of genes and their functions are allowing researchers and physicians to customize health care and treat individuals according to their genetic makeup. This precision medicine approach is giving physicians more tools to understand what their patients need and to provide highly personalized, precise care.

There are more than 4,800 known, rare syndromes, affecting between one and two million Canadians. More accessible gene sequencing has allowed UCalgary geneticists to diagnose many previously undiagnosed, complex chronic diseases, which has a positive impact on families as well as the health-care system.

The cost to sequence the full genome in one single person used to be $100 million, but now costs about $1,000.

Yang moved from Halifax to Calgary with her family as a teenager and completed her five-year residency in medical genetics at the CSM earlier this year. During the COVID-19 pandemic, she became a mom to a now two-year-old boy. She previously had the opportunity to spend time during her residency in clinic with Suchowersky, who teaches at both UCalgary and UAlberta, and Yang looks forward to learning from the neurogeneticist again as part of the fellowship.

"I'm so grateful for this investment that Dr. Suchowersky and Dr. Eagle are making into training the next generation of physicians in the fields of genetics and neurology. I get to train with some really fantastic physicians and scientists and it wouldn't happen without their generosity, Yang says.

I hope through the course of my career that I can pay it forward by furthering the education of other trainees, like they're doing for me right now.

See the article here:
Clinical fellowship offers opportunity to diagnose previously undiagnosable diseases through genetic 'detective' work - University of Calgary

LONDON, Oct. 04, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that the first patient has been dosed in the second dose cohort of the Phase 1/2 MARVEL-1 clinical trial of FLT190 in Fabry disease, a debilitating inherited disorder that leads to progressive organ damage and can result in early death as a result of a harmful build-up of fat in cells due to an enzyme deficiency.

We believe FLT190 has the potential to be a life-changing therapy for people with Fabry disease by providing durable enzyme activity above the normal range with a one-time treatment, and we are excited to have initiated this cohort in MARVEL-1, said Pamela Foulds, MD, Chief Medical Officer at Freeline. We have multiple trial sites now open across five countries with more expected to open by year-end. We look forward to reporting updated safety and efficacy data in the first half of next year.

Freeline recently reached alignment with the U.S. Food and Drug Administration to dose patients in MARVEL-1 in the United States. Efficacy and safety data from the first dose cohort of MARVEL-1 (in which patients received 7.5e11 vg/kg of FLT190) supported progression to the second, mid-dose cohort (1.5e12 vg/kg).

MARVEL-1 is an international, multicenter, adaptive dose-escalation and dose-expansion Phase1/2 clinical trial in adult men ( 18 years) with classic Fabry disease. The trial is evaluating the safety and efficacy of one-time treatment with FLT190 in up to four dose cohorts. Efficacy measures include-GalA activity levels and clearance of globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3) as measured in plasma and urine. In the dose-escalation part, patient dosing is staggered to allow for adequate monitoring of both safety and efficacy. Patients will be monitored for nine months after dosing and will be eligible to participate in a long-term follow-up study for at least five years (MARVEL-2).

About Fabry Disease

Fabry disease is caused by a mutation in the GLA gene, which encodes for the enzyme -galactosidase A (-Gal A) that is required to metabolize a certain type of fat called globotriaosylceramide 3 (Gb3). As a result of deficient -Gal A production or function, Gb3 accumulates in the lysosome of various cell types and causes damage, commonly leading to a variety of painful and debilitating renal, cardiac, skin, neurological, and gastrointestinal conditions. A Gb3 metabolite, called lyso-Gb3, is considered a good biomarker for Fabry disease and may contribute to disease pathogenesis. Fabry disease can lead to premature death with renal failure and cardiac disease being the most common causes of death. Fabry disease affects approximately 16,000 people in the United States, European Union and Japan.

About FLT190 for Fabry Disease

FLT190 is a gene therapy candidate designed to generate durable plasma -Gal A levels above the normal range to enable sufficient enzyme activity in relevant tissues with a one-time treatment. FLT190 uses Freelines proprietary AAVS3 capsid to introduce a functional GLA gene into liver cells to produce functional -Gal A. AAVS3 has been rationally designed to enable effective liver cell transduction and strong and durable enzyme production at low doses and with a good safety profile. From the liver, -Gal A can circulate in the blood and be taken up by various tissues in the body, where it is transported to the cells and lysosomes and can break down Gb3.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The company is dedicated to improving patient lives through innovative, one-time treatments that may provide functional cures for inherited, systemic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector and capsid (AAVS3), along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into the patients bloodstream. The company has clinical programs in hemophilia B, Fabry disease, and Gaucher disease Type 1. Freeline is headquartered in the UK and has operations in Germany and the U.S.

Forward-Looking Statements

This press release contains statements that constitute forward-looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the opinions, expectations, beliefs, plans, objectives, assumptions or projections of Freeline Therapeutics Holdings plc (the Company) regarding future events or future results, in contrast with statements that reflect historical facts. All statements, other than historical facts, including statements regarding FLT190s potential to be a life-changing therapy for people with Fabry disease by providing durable enzyme activity above the normal range with a one-time treatment and that the Company expects to open more sites in its MARVEL-1 clinical trial by year end and plans to report updated safety and efficacy data from such trial in the first half of next year, are forward-looking statements. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project, expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward-looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the uncertainties inherent in research and development of the Companys product candidates, including statements regarding the timing of initiation, enrollment, continuation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the Companys ability to design and implement successful clinical trials for its product candidates; whether the Companys cash resources will be sufficient to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Companys expected timeline; the potential for a pandemic, epidemic or outbreak of infectious diseases in the United States, United Kingdom or European Union, including the COVID-19 pandemic, to disrupt and delay the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; business interruptions resulting from geopolitical actions, including global hostilities, war and terrorism, global pandemics or natural disasters, including earthquakes, typhoons, floods and fires; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing history, which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. The Company cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated, or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties, and other matters can be found in the Companys Annual Report on Form 20-F for the fiscal year ended December 31, 2021, and in subsequent reports on Form 6-K, in each case including in the sections thereof captioned Cautionary Statement Regarding Forward-Looking Statements and Item 3.D. Risk factors. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with the U.S. Securities and Exchange Commission (the SEC). You may review these documents by visiting EDGAR on the SEC website at http://www.sec.gov.

Media Contact:

Arne Naeveke, PhDVice President, Head of Corporate Communicationsarne.naeveke@freeline.life+1 617 312 2521

IR Contact:

Naomi Aokinaomi.aoki@freeline.lifeSenior Vice President, Head of Investor Relations & Communications+ 1 617 283 4298

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Freeline Initiates Dosing of Second Cohort in MARVEL-1 Trial of FLT190 Gene Therapy Candidate for People with Fabry Disease - GlobeNewswire