WAKEFIELD, Mass., September 07, 2022--(BUSINESS WIRE)--Myrtelle Inc., ("Myrtelle" or the "Company"), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has classified the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal Product (ATMP), specifically a Gene Therapy Medicinal Product (GTMP). ATMP classification, which is determined by the Committee for Advanced Therapies (CAT), was established to regulate cell and gene therapy and tissue engineered medicinal products, support development of these products, and provide a benchmark for the level of quality compliance for pharmaceutical practices. As a designated GTMP product, rAAV-Olig001-ASPA will follow the Centralized Procedure through the EMA and benefit from a single evaluation and authorization process. Additional benefits established through the ATMP regulation include pathways for Scientific Advice and significant fee reductions for such advice.

rAAV-Olig001 is a novel vector from a class of recombinant AAVs (rAAVs) that selectively target oligodendrocytes the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons and makes up the brains white matter. The Companys lead program is in Phase 1/2 clinical development for Canavan disease (CD) a fatal childhood genetic disorder characterized by the degeneration of the white matter in the brain. The production of myelin is affected in CD due to a mutation in the Aspartoacylase gene (ASPA) leading to deficiency in Aspartoacylase enzyme (ASPA). The oligodendrocyte-targeted gene therapy using the rAAV-Olig001 vector is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartic Acid (NAA), a neurochemical abundant in the brain, and supporting myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.

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In addition to ATMP classification, rAAV-Olig001-ASPA has been granted US Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA which support the Companys mission to provide treatments for patients with CD. "The designation by the EMA of rAAV-Olig001-ASPA as a Gene Therapy Medicinal Product as a potential treatment for patients with Canavan disease provides important benefits in the development of this innovative therapy. The ATMP classification will facilitate discussions with the EMA as part of our strategy to seek product registration in the EU," said Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle.

ABOUT MYRTELLE

Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer for its lead program in Canavan disease. For more information, please visit the Companys website at: http://www.myrtellegtx.com.

ABOUT CANAVAN DISEASE

Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. CD patients are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD and only palliative treatments are available. More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.

Forward-Looking Statements

This press release contains forward-looking statements. Words such as "may," "believe," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding rAAV-Olig001-ASPA as a potential treatment for patients with Canavan disease. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelles program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelles expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220907005109/en/

Contacts

Media:Jordana HolovachHead of Communications and CommunityMyrtelle Inc.781-621-2797 Ext. 102jholovach@myrtellegtx.com

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Myrtelles rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European...

Sangamo Therapeutics is continuing to enroll and treat patients with a rare genetic condition called Fabry disease in an early clinical trial of a gene therapy it is developing, announcing Tuesday updated results from the first 11 study participants.

Data to date has shown Sangamos medicine to be safe, with no serious treatment-related adverse reactions, and suggests the gene therapy is working as intended. As a result, Sangamo has begun the dose expansion phase of the trial, enrolling new patients to receive the fourth and highest dose tested in the initial dose escalation cohort.

Sangamo, which has a pipeline of experimental cell, gene replacement and gene editing therapies, is already planning for a potential Phase 3 study, should results from the current trial continue to prove positive. It, along with Freeline Therapeutics, have the most advanced gene therapies in clinical development for Fabry, which is one of an array of inherited conditions known as lysosomal storage disorders.

In Fabry, mutations in a gene called GLA lead to low levels of an enzyme thats needed to prevent the buildup of a certain toxin in cells, causing a constellation of symptoms that over time can become severe and life-threatening. Sangamos therapy is designed to deliver a functional copy of the GLA gene into the body via a type of modified virus that acts as a courier of sorts.

The updated trial results released Thursday show that, among the five earliest treated patients, enzyme levels rose to several times a normal average. In three, enzyme levels were 10 to 17 times higher. Notably, in one patient with the highest toxin level pre-treatment, the gene therapy led to a 40% decline in toxin levels within 10 weeks after dosing.

Five patients have been able to discontinue standard drugs, which in the case of Fabry is an enzyme replacement therapy that must be taken chronically to control toxin levels.

According to Sangamo, there were no treatment-related side effects rated by investigators as more significant than mild, and no patient experienced elevations in liver enzymes that can sometimes signal broader safety concerns.

In the next part of the trial, Sangamo plans to enroll up to six participants in six different cohorts.

Sangamo and Freeline were previously joined by Avrobio in developing a Fabry gene therapy. But in February Avrobio announced it would stop work on its program after disappointing findings and prioritize research elsewhere. Amicus Therapeutics and UniQure also have Fabry gene therapy programs, although Amicus recently had to pivot after plans to spin out its gene therapy business fell apart.

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Sangamo presses ahead with Fabry disease gene therapy - BioPharma Dive

Hopes run high in Philadelphia that the region the scientific home of two of the first cell and gene therapies approved by the FDA will remain a major player as the cutting-edge treatments assume a bigger role in medicine.

To make that happen, Philadelphias life sciences industry will need not just scientists, management, and money, but also skilled workers to help laboratories run smoothly at an ever-growing number of biotech companies in the region and eventually to manufacture cures and treatments for rare diseases and elusive types of cancer.

To help build that skilled workforce, the Wistar Institute, the University City Districts West Philadelphia Skills Initiative, and partners have launched a new biomedical technician training program.

It will enroll 18 students in a 12-week paid training program at Wistar, potentially followed by an additional 10 weeks of hands-on work at Iovance Biotherapeutics Inc. in the Navy Yard and then a $23-an-hour manufacturing job. Iovance, which now employs 150 people in Philadelphia, is developing cancer treatments using cell therapy.

Iovance did not say how many of the trainees it would hire. Iovance officials will interview them after they complete the Wistar part of the training.

We expect to have a number of opportunities to which program participants can apply, Tracy Winton, Iovances senior vice president for human resources, said in a statement.

Cell and gene therapies are still in the early stages of development, but Philadelphia scientists have long played a central part. Luxturna, a gene therapy cure for a rare form of congenital blindness, and Kymriah, a cell therapy treatment for some forms of leukemia, are based on the work of Philadelphia scientists. Both received FDA approval in 2017.

Cell therapy uses modified cells to carry treatment into the body. Gene therapy involves the replacement of defective genes that cause what are typically rare diseases.

The new training effort, scheduled to start Sept. 22, builds on one started in 2000 at Wistar, a nonprofit biomedical research institute in University City, in partnership with Community College of Philadelphia. The original Wistar program, which provided general preparation for work in biotech and until this year was spread over two summers for each cohort, has graduated 196 students.

Recruitment for the new program, which Wistar designed to specifically prepare individuals for jobs at Iovance, started Aug. 23 and runs through Friday. As of last Friday morning, 263 people had applied, according to the West Philadelphia Skills Initiative (WPSI), which for a decade has been training Philadelphians for specific jobs at individual employers, such as Childrens Hospital of Philadelphia and SEPTA.

WPSI is handling recruitment selection for the Iovance training. The selection process for the 18 open spots includes an assessment of mathematical ability and an interview, said Cait Garozzo, managing director of WPSI.

Some folks, obviously, are very desperate for a job, any job, and were not trying to connect people that just want any job to this opportunity. Were trying to connect people that want a career in this industry to this opportunity, Garozzo said.

This is the first time WPSI and Wistar have worked together. Other supporters are the Chamber of Commerce of Philadelphia and the Philadelphia Industrial Development Corp.

If this is successful, we really think this could be a game changer for this region, said Kristy Shuda McGuire, dean for biomedical studies at Wistar. We think this is something we could repeat. We could have more cohorts each year if there are single employers who are interested in this and have a lab-based position and would be interested in taking a whole cohort.

The total budget for the training program was not disclosed.

Wistars original training program which expanded this year to include Montgomery County Community College and will be open to students at Bucks County Community College and Camden County College next year typically sends graduates into biotech jobs or on to further education, McGuire said.

Among the graduates of the Wistar program that have gone on to build careers in life sciences is Lois Tovinsky, 36, who completed the program in 2013 and is now laboratory operations manager for Chimeron Bio, a biotech start-up in the Curtis Building that is working on RNA therapeutics against cancer.

Tovinsky graduated from college with a degree in political science in 2008, when the economy collapsed and jobs were hard to find. She heard about the Wistar program in a science class at Community College of Philadelphia and saw it as a chance to fulfill her interest in science and leap from her job as a dog walker into a science career.

I came to the program with no practical skills in the lab, and my knowledge of science was really just the few courses I had taken and my own interest and enthusiasm that I had for it, said Tovinsky, who now mentors students in the Wistar program.

Tylier Driscoll, 21, a biology major at Community College of Philadelphia, was one of 15 students in the Wistar training cohort that finished early last month.

I definitely wanted to do something over the summer that wasnt working at Aldi, Driscoll said. Before this, I hadnt had any lab experience and I really wanted to get a feel for what it was like to work in a lab. I was working at a supermarket at the time. This is the perfect opportunity for me to get into my field.

As part of his training, he spent five weeks working at BioAnalysis LLC, a contract research organization in Kensington that performs quality analysis on the viruses used in gene therapy.

Now, Driscoll has a part-time job at BioAnalysis that he starts Tuesday, the same day he goes back to CCP for the fall semester. He plans to finish his associate degree in the spring and then attend either Drexel University or Temple University for his bachelors degree.

Lake Paul, the president and founder of BioAnalysis, which he called a minority-owned biotech, said the Wistar program is an awesome opportunity and one that reminds him of his own experience. Paul said he grew up in the hood in Miami and wouldnt have obtained his doctorate at Purdue University without the Upward Bound programs that helped him pursue education.

It is a wonderful, exciting, and unique opportunity for these students, both underrepresented folks and regular folks. And to give them actual training like this is unparalleled, said Paul.

The Philadelphia Inquirer is one of more than 20 news organizations producing Broke in Philly, a collaborative reporting project on solutions to poverty and the citys push toward economic justice. See all of our reporting at brokeinphilly.org.

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As Philly becomes a hub for life sciences, a new program will train workers for jobs in the field - The Philadelphia Inquirer

Tegan Taylor: There are a few things in life that are just inevitable; death, taxes, the genes you're born with. At least, that has been the case for pretty much every generation up until now. Gene therapies have the potential to change the trajectory of disease, and I've been talking to two people on the frontline of that shift.

Until about three years ago, Robert Lamberth had a disease that was incurable. I mean, it was literally in his genes.

Robert Lamberth: Not as a newborn, but yes, very, very young when I had my first bleed. Three, I think it might have been for me, back in the early '80s it was, a long time ago now.

Tegan Taylor: When he was born, he inherited a certain recessive gene that stopped his body from producing one of the essential factors you need for your blood to clot.

Robert Lamberth: Bleeding internally into my major weight-bearing joints, so ankles and knees. And as I got older, I'd have more odd bleeding into muscles in my legs and parts of my stomach and those sorts of things, so it was a little bit more serious when you have large muscle bleeds. The pressure of the bleeding can affect your organs, so that's quite serious.

Tegan Taylor: Managing haemophilia A is miles easier than it was a couple of decades ago. When he was little, Robert needed intravenous injections of his missing clotting factor, given in a hospital. When he got older, he didn't need to go to hospital anymore. Regular injections of the clotting factor were a feature of his life all the way through into his 30s. But not anymore.

John Rasko: We dream of cures in gene therapy but hesitate to use the word

Tegan Taylor: For decades, John Rasko has been chasing ways to change people's fates.

John Rasko: For the last 20-plus years we've been doing clinical trials using viral vectors to transfer a gene into humans for a therapeutic purpose.

Tegan Taylor: Professor Rasko is a haematologist and pathologist who spent much of his career studying genes, stem cells and basically how to hack processes inside the human body. And he is one of many scientists around the world trying to figure out ways of swapping out disease-causing genes in a way that, in time, could be used for pretty much any genetic disease.

John Rasko: When we reflect on rare diseases, it's often worth remarking and reminding ourselves that rare diseases of course by definition are rare, usually less than one in 5,000 or 10,000 people, but collectively rare diseases are very common when you add them all up because there are many thousands of them, lead to a burden of disease such as the commonality of diabetes or even some forms of cancer. So the problem is that of all the rare diseases, which some people say are more than 4,000 affecting humans, 80% of those rare diseases have a genetic basis. And of those diseases, only 5% have a specific therapy. So this is an incredible unmet need in human health.

Tegan Taylor: And the solution he and his colleagues have come up with might sound a bit familiar. It works in a similar way to the Covid vaccine made by AstraZeneca. It uses a harmless virus to take a genetic message into the body.

John Rasko: And that vector system is used to then ferry that genetic payload intravenously to the liver where it takes up residence, and hopefully after a single injection, corrects that person's genetic abnormality for the rest of their life. It's unimaginable, but a single injection can alter the course of a genetic disease that would otherwise affect a person from birth to death.

Tegan Taylor: Robert was part of the clinical trial Professor Rasko was involved in, testing the gene therapy.

Robert Lamberth: It would be three years ago now in May 2019 when I had that one single dose of the good stuff, and then that clearly worked its magic and now I'm growing my own factor VIII. I've had one breakthrough bleed.

Gene therapy for me, Tegan, has been quite revolutionary, so from a position of having 0.5% of clotting factor in my blood, I'm now growing my own factor VIII in my liver and I'm at about 15% clotting factor, which is an extraordinary growth.

Tegan Taylor: In August, Europe granted conditional approval for a haemophilia A therapy like the one Robert received. It hasn't been approved in Australia yet, although we do use gene therapy for other conditions, like spinal muscular atrophy, and genetic causes of blindness.

John Rasko: We are only at the very start of this genetic revolution. There are thousands of genetic diseases that affect humans, and we've only just started scratching the surface of where we can go with these gene-based therapeutics.

Tegan Taylor: Because Robert got the gene therapy as an adult, he's still living with the damage haemophilia A had already done to his body, but that doesn't mean it hasn't been transformative.

Robert Lamberth: I can just do so much more. I can be out there doing everything that I love at work and at play and going to the gym, without fear of having a micro-bleed the next day and being cross and crotchety and painful and grumpy at work, and then it turning into a more major bleed and then having to go and seek therapy, which means even more down-time. The sooner that we could roll out some gene therapy for younger people would be great.

Tegan Taylor: Robert Lamberth, who received gene therapy for haemophilia A, finishing us off there. And we also heard from Professor John Rasko from Royal Prince Alfred Hospital and the Centenary Institute at the University of Sydney.

Norman Swan: It's interesting how things have advanced there, Tegan. A few years ago, not so long ago, gene therapy could have been quite toxic because of the virus that they were using to carry the gene in, and you've got to hit the target, it can't be wasteful, and sometimes the virus did harm in its own right. So it's taken a long time to get that right, but the potential, as John Rasko says, is huge and it goes from cancer through to these inborn errors that you get such as haemophilia A.

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The gene therapy that could transform the lives of millions - ABC News

James R. Martin/Shutterstock

A cell and gene therapy accelerator formed by Mayo Clinic, Hibiscus BioVentures and Innoforce is officially up and running, the partners announced Wednesday.

Mayflower BioVentures will identify and launch companies around technologies that address unmet patient needs.

Mayo Clinic hasaccumulated a portfolio of next-generation immune system modulators as well as numerous novel therapeutics in cell and gene therapy. Now Mayo is sharing those discoveries and research capabilities in hopes of reaching patients.

We have the first right to look at the technologies that Mayo considered to be high-value opportunities, Chris Jeffers, CEO of Hibiscus BioTechnology, told BioSpace. Thats an internal designation. And we have the first rights to create companies from those.

It gives Hibiscus the opportunity to incubate and accelerate the companies within Mayflower anywhere between one and two years. Eventually our goal is to graduate those companies to be self-sufficient, independent companies that can obtain their own funding once they leave the accelerator, Jeffers added.

Andrew Danielsen, chair of Mayo Clinic Ventures, told BioSpace that each company will be within Mayflower and owned by the investor syndicate proportionally.

Mayflower has been in the works for at least a year, Jeffers and COO Sia Anaganostou shared, adding that they have been working with Mayo on identifying and developing several companies, which they expect to announce in the coming months.

While unable to provide further details, Jeffers said the areas of focus are anticipated to range from various types of stem cell therapies and gene therapies covering a number of conditions. Some of those technologies are ancillary to cellular therapies, while some are for new pathways to try to escape from traditional immuno-oncology, he said.

Mayflower will be run by the Hibiscus management team.

This is a big push from the Mayo Clinic to really increase its commercialization in this space. Were really proud to be associated with such a fantastic institution with unparalleled clinical expertise, Jeffers said, adding that these factors are a real differentiator.

Danielsen spoke of transitioning research from bench to bedside.

We believe this collaboration can bridge the gap between industry and innovative cell and gene therapy research, enabling emerging startups to navigate the challenges of producing meaningful, novel therapeutics that transform health and medicine, he said in a statement.

Mayos Center for Regenerative Biotherapeutics focuses on advancing regenerative technologies from discovery into early phase clinical studies.

Hibiscus is a venture capital firm focused on building patient-focused companies around new technology and helping to develop those discoveries into commercial drugs and therapies. Hibiscus is comprised of Hibiscus Biotechnology, a venture studio that works to build companies from scratch, and Hibiscus Capital Management, a VC firm that invests in promising early-stage biotech companies.

Innoforce is a partnership-focused biopharma company targeting advanced therapy medicinal products (ATMPs) and biologics. It offers contract development and manufacturing services including GMP manufacturing of plasmid DNA, RNA, viral vector and cell products.

Any revenue generated by Mayflower will go toward Mayo Clinics patient care, education and research.

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Mayflower BioVentures to Announce New Cell & Gene Therapy Companies within Months - BioSpace

GRENOBLE, France--(BUSINESS WIRE)--Carroucell, the microcarrier supplier for cell culture in bioreactor, announced today that it has raised a total of 1.5 million. The funding includes the closing of a Series A financing, led by the Novalis Biotech Acceleration fund and with participation of Crdit Agricole des Savoie (CADS), as well as support from Bpifrance. The funding will be used to accelerate corporate growth through industrialization of the companys platform technology and ramping up of mass production processes to GMP standards.

Carroucell has developed a disruptive technological platform that offers unique flat shape microcarriers with a glass xenofree composition for cell culture in bioreactors. Unlike existing technologies, the combination of these novel microcarriers combined with the flexibility of the production process enables a faster, more optimized scale-up of the clinical phases. This more cost-effective process could provide customers with a more accelerated time and pathway to market.

For the first time, microcarrier customization and a more customer-oriented service are available for the development of the new applications into the cell culture and bioproduction market. There are many challenges with biomanufacturing performance. We believe our unique microcarrier technology and ability to address customer specific needs will overcome most challenges and stimulate a revolution in the sector moving forward, said Tarek Fathallah, Founder and President of Carroucell.

Carroucell is creating a new standard in biomanufacturing, which could help to facilitate patient access to many more innovations in cell and gene therapy in the future, said Jan Van den Berghe, co-founder and managing director of Novalis Biotech, who has also been appointed to the board of directors. When customers adopt Carroucell's technology platform, they are able to optimize the yield and the quality of the cell culture, solving the low-performance problem in bioproduction we see today.

The complex environment of cell culture in bioreactors and the increasing number of new applications requires an innovative approach to guarantee the balance of the system. Carroucells microcarrier plays the role of regulator of this system by ensuring its optimization, said Takis Breyiannis, CEO of Carroucell.

About Novalis BiotechNovalis Biotech (Ghent, Belgium) is an early-stage venture capital investor in technologies that revolutionize healthcare. The companys core competence lies in digitalization in the life sciences with a focus on bioinformatics, genomics and diagnostics. Novalis strongly believes in applying innovative enabling technology to advance the prevention, diagnosis, or treatment of a disease. For more information, please visit http://www.noval.is.

About CarroucellCarroucell is disrupting the biomanufacturing sector with its patented, innovative microcarrier and flexible process solution for customers. The microcarriers are based on a major innovation in the field of sol-gel process, which allows the production of bioactive microstructures not achievable by existing technologies. In the bioreactor, cells can cling and multiply in "3D" and allows cultivation of a large quantity of cells in a restricted volume. Carroucell has a partnership with Etablissement Franais du Sang (EFS), which enabled the validation of its microcarriers and facilitated first commercial orders. Carroucell was founded in 2016 by Tarek Fathallah. For more information, visit http://www.carroucell.com.

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Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market - Business Wire

September 7, 2022

Company Name: AnGes Inc.

Presentative: Ei Yamada, President & CEO

AnGes Announces Discontinuation of Development of HGF Gene Therapy Product for Additional Indication of Chronic Arterial Occlusive Disease with Rest Pain in Japan

AnGes, Inc. hereby announces that it has decided to discontinue the development of an HGF gene therapy product it has been working on for the additional indication of chronic arterial occlusive disease with rest pain in Japan. Details are as follows.

1. Background to development of HGF gene therapy product

The HGF gene therapy product is the first gene therapy product to be approved in Japan. It is a core project we have been involved in since our foundation. In March 2019, we obtained marketing approval with conditions and time limit in Japan, claiming improvement of arteriosclerosis obliterans with lower limb ulcer as the efficacy, effect, or performance, and we started selling the product in September 2019. We subsequently completed enrollment of the target number of patients to conduct an approval condition-basedpost-marketing evaluation for this indication. Furthermore, in December 2021, we also completed administration for a Phase 3 Clinical Trial for approval of the additional indication of chronic arterial occlusive disease with rest pain in Japan.

In addition, a Phase 2 Clinical Trial in the US of the HGF gene therapy product for arteriosclerosis obliterans with lower limb ulcer in patients with chronic arterial occlusion has also been progressing largely according to plan.

2. Discontinuation of development for additional indication of rest pain in patients suffering from chronic arterial occlusion in Japan

We completed administration for the Phase 3 Clinical Trial of the HGF gene therapy product for the additional indication of chronic arterial occlusive disease with rest pain and have been organizing and analyzing the data. As a result, we found that the results are such that we failed to meet the primary endpoints for rest pain. Based on these results, AnGes decided to discontinue development for approval of the HGF gene therapy product in Japan for the additional indication of chronic arterial occlusive disease with rest pain.

(Note)This document has been translated from the Japanese original for reference purposes only.

In the event of any discrepancy between this translation and the Japanese original, the original shall prevail.

3. Future development plans for HGF gene therapy product

AnGes will push ahead as planned with preparations to apply to obtain the approval of the HGF gene therapy product in Japan, with improvement of arteriosclerosis obliterans with lower limb ulcer as the stated efficacy, effect, or performance.

We will also continue with the Phase 2 Clinical Trial in the US of the HGF gene therapy product for arteriosclerosis obliterans with lower limb ulcer and push ahead with development aiming to quickly progress through the clinical trial stages.

4. Future outlook

The impact that discontinuation of the development of the HGF gene therapy product for the additional indication of chronic arterial occlusive disease with rest pain in Japan will have on our full-year consolidated financial results and financial position for the current fiscal year will be minimal. AnGes will promptly disclose any future material developments.

AnGes, Inc.

Public Relations & Investor Relations Group

https://www.anges.co.jp/en/

(Note)This document has been translated from the Japanese original for reference purposes only.

In the event of any discrepancy between this translation and the Japanese original, the original shall prevail.

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AnGes : Announces Discontinuation of Development of HGF Gene Therapy Product for Additional Indication of Chronic Arterial Occlusive Disease with Rest...

The Cell and Gene Therapymarket revenue was $$ Million USD in 2016, grew to $$ Million USD in 2022, and will reach $$ Million USD in 2030, with a CAGR of % during 2022-2030.

The global Cell and Gene Therapy market is expected to grow with a CAGR of $$%, during the forecast period 2019-2025, the market growth is supported by various growth factors and major market determinants. The market research report is compiled by Report Ocean by conducting rigorous market study and include the analysis of the market based on segmenting the geography and market segmentation.

The market study was done on the basis of:

Major Players in Cell and Gene Therapy market are:

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Most important types of Cell and Gene Therapy products covered in this report are:

Most widely used downstream fields of Cell and Gene Therapy market covered in this report are:

Top countries data covered in this report:

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Global Cell and Gene Therapy Market Development Strategy Pre and Post COVID-19, by Corporate Strategy Analysis, Landscape, Type, Application, and Leading 20 Countries covers and analyzes the potential of the global Cell and Gene Therapy industry, providing statistical information about market dynamics, growth factors, major challenges, PEST analysis and market entry strategy Analysis, opportunities and forecasts. The biggest highlight of the report is to provide companies in the industry with a strategic analysis of the impact of COVID-19. At the same time, this report analyzed the market of leading 20 countries and introduce the market potential of these countries.

Chapter 1 is the basis of the entire report. In this chapter, we define the market concept and market scope of Cell and Gene Therapy, including product classification, application areas, and the entire report covered area.

Chapter 2 is the core idea of the whole report. In this chapter, we provide a detailed introduction to our research methods and data sources.

Chapter 3 focuses on analyzing the current competitive situation in the Cell and Gene Therapy market and provides basic information, market data, product introductions, etc. of leading companies in the industry. At the same time, Chapter 3 includes the highlighted analysisStrategies for Company to Deal with the Impact of COVID-19.

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Chapter 4 provides breakdown data of different types of products, as well as market forecasts.

Different application fields have different usage and development prospects of products. Therefore, Chapter 5 provides subdivision data of different application fields and market forecasts.

Chapter 6 includes detailed data of major regions of the world, including detailed data of major regions of the world. North America, Asia Pacific, Europe, South America, Middle East and Africa.

Chapters 7-26 focus on the regional market. We have selected the most representative 20 countries from 197 countries in the world and conducted a detailed analysis and overview of the market development of these countries.

Chapter 27 focuses on market qualitative analysis, providing market driving factor analysis, market development constraints, PEST analysis, industry trends under COVID-19, market entry strategy analysis, etc.

Key Points:Define, describe and forecast Cell and Gene Therapy product market by type, application, end user and region.Provide enterprise external environment analysis and PEST analysis.Provide strategies for company to deal with the impact of COVID-19.Provide market dynamic analysis, including market driving factors, market development constraints.Provide market entry strategy analysis for new players or players who are ready to enter the market, including market segment definition, client analysis, distribution model, product messaging and positioning, and price strategy analysis.Keep up with international market trends and provide analysis of the impact of the COVID-19 epidemic on major regions of the world.Analyze the market opportunities of stakeholders and provide market leaders with details of the competitive landscape.

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Cell and Gene Therapy Market 2022 Sales and Growth Rate, Assessment to 2030 | Gilead Sciences, MolMed, Vericel - Taiwan News

Purespring Therapeutics

Purespring Therapeutics strengthens scientific leadership with the appointment of Alice Brown as Chief Scientific Officer

London 07 September 2022 Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, today announces the appointment of Alice Brown as its Chief Scientific Officer, effective immediately.

Alice brings more than a decade of experience working in advanced therapies, spanning both large pharma and early-stage biotech companies. Prior to joining Purespring, Alice held the roles of VP Research and VP Gene Engineering at GammaDelta Therapeutics; she then briefly joined the Cell Therapy Innovation department at Takeda Pharmaceuticals, upon the acquisition of GammaDelta Therapeutics by Takeda at the beginning of 2022.

At GammaDelta Alice built and led the research team and was responsible for developing multiple cell therapy programs from discovery through to completion of pre-clinical development. Before GammaDelta, Alice was Director of Biology at PsiOxus Therapeutics. She has a degree and PhD in Biochemistry from the University of Bristol and carried out her post-doctoral research in Immunology at Imperial College London.

Alice will lead Puresprings research team to oversee the progression of the Companys therapeutic programmes. With a focus on building its pipeline, the Company has rapidly expanded its in-house team through a number of senior appointments. Alice will work closely with Puresprings co-founder Moin Saleem in his new capacity as Chief Scientific Advisor to build Puresprings unparalleled pipeline of AAV gene therapies for chronic kidney diseases.

Richard Francis, Chief Executive Officer of Purespring, said: With an ambition to revolutionise the treatment of kidney diseases, we are moving at speed and a cornerstone of this is putting the right team in place. With decades of work on the podocyte, Professor Moin Saleem is a world leader in gene therapy for the kidney and his work has been critical to establishing Purespring. Combining this with Alices expertise in moving ATMPs through research and into the clinic, we are in the best possible position to move our innovative treatments closer to people suffering from kidney diseases.

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Prof. Moin Saleem, Co-founder and Chief Scientific Advisor of Purespring, said: Over the last year, we have made tremendous progress in our preclinical work at Purespring. Expanding our in-house capabilities is a vital step in shifting our focus towards the clinic and Alice is a critical part of this. With an impressive track record working within similar-stage companies, I believe Alice is well positioned to lead the research team in this next phase of Puresprings development, and I look forward to working with her.

Alice Brown, Chief Scientific Officer of Purespring, commented: Purespring is providing leadership in innovation which has been sorely lacking in the kidney space through its approach to using AAV therapy to target the podocyte. I cant think of a more exciting company to work for at the moment and its a privilege to be working with Professor Moin Saleem, who has done so much to advance AAV gene therapy in the kidney. I am thrilled to be working with such a strong team.

- ENDS -

For further information, contact:

Purespring:

Richard Francis, CEOcontact@purespringtx.com+44 (0)20 3855 6324LinkedIn

Consilium Strategic Communications:

Amber Fennell, Jessica Hodgson, Genevieve Wilsonpurespring@consilium-comms.com

Notes to Editors

About Purespring

Purespring is the first company to treat kidney diseases by directly targeting the podocyte, a specialised kidney cell implicated in many kidney diseases, through AAV gene therapy.

Headed by former Sandoz CEO, Richard Francis, Purespring was founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, where he heads a world leading group researching glomerular diseases. Purespring seeks to advance gene therapies for the treatment of both monogenic and non-monogenic chronic renal diseases that are currently poorly addressed with existing treatments.

The company also has a proprietary in-vivo pipeline engine, FunSel, which is a library of all biological factors that could be candidates for gene therapy, combined with a screening method to evaluate these factors in disease models. FunSel allows Purespring to discover new gene therapy candidates across all indications, unconstrained by genetics, to find the right candidate to make the best therapy.

An initial 45 million commitment to Purespring from Syncona Ltd is enabling Purespring to progress its assets to the clinic. Synconas Chief Investment Officer, Chris Hollowood, serves as Chairman. For more information please visit: purespringtx.com and follow us on LinkedIn.

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Purespring Therapeutics strengthens scientific leadership with the appointment of Alice Brown as Chief Scientific Officer - Yahoo Finance

Mucopolysaccharidosis IV A (MPS IVA) is a metabolic disorder caused by the mutations ofthe GALNSgene, leading to the accumulation of chondroitin 6-sulfate and glycosaminoglycans (GAGs) keratan sulfate in the lysosomal lumen.

Study:Delivery and assessment of a CRISPR/nCas9-based genome editing system on in vitro models of mucopolysaccharidoses IVA assisted by magnetite-based nanoparticles. Image Credit:vchal/Shutterstock.com

Enzyme replacement therapy has shown advantages in treating such disorders in patients. However, this therapy has disadvantages, including impact on the bone lesion and recovery of oxidative profile.

Recently, a gene therapy based on CRISPR/nCas9 system was presented as a promising strategy to treat in vitroMPS IVA model. An article published in the journalScientific Reportsused CRISPR/nCas9 system on several MPS IVA fibroblasts withGALNSmutations to evaluate their potential in gene therapy.

The CRISPR/nCas9 system was combined with magnetite-based nanoparticles entrapped in a liposome-based novel non-viral vector to develop safer vectors for gene therapy. The CRISPR/nCas9 treatment increased the enzyme activity of wild-type levels and reduced the lysosomal mass, glycosaminoglycans (GAGs) accumulation, and oxidative stress based on mitochondria.

Viral vectors composed of magnetite-based nanoparticles entrapped in liposomes showed similar results as lipofectamine, a conventional transfection agent. Overall, the results confirmed CRISPR/nCas9 system as a promising genome editing tool for treating MPS IVA. Besides, the potential use of magnetite-based nanoparticles entrapped in liposomes was illustrated as an ideal delivery system for gene therapy.

MPS IVA is an autosomal recessive lysosomal storage disorder characterized by intracellular accumulation of keratan sulfate and chondroitin-6-sulfate. Under impaired activityofthe GALNSgene, various oxidative stress and inflammation-based pathological signal pathways might occur.

The autosomal recessive MPS IV A affects approximately 1 in 200,000 lives and is characterized by various symptoms, including severe skeletal abnormalities, hearing loss, corneal clouding, heart valve disease, and other impairments.

Enzyme replacement therapy is the most accepted treatment for MPS IV A. Here, recombinant enzyme is injected into patients to replace the missingGALNSenzymatic activity. To improve the delivery of recombinantGALNSto the bones of MPS IV A patients, one proposed variant ofGALNScontains six additional glutamate residues at the N terminus.

Technologies based on CRISPR/Cas have been repurposed for genome modification, mediating a true revolution in synthetic biology and metabolic engineering. With numerous applications in diverse prokaryotic and eukaryotic species, CRISPR/Cas9 approaches continue to facilitate the design and construction of synthetic organisms.

CRISPR/Cas9 was adapted from a naturally occurring genome editing system used by bacteria as an immune defense. CRISPR/Cas9 is a cost-effective and highly specific nuclease-based genome editing mechanism. Besides Cas9 variants, Cas9 nickase (nCas9) shows an increased target-specific efficiency.

Some genome editing-based novel strategies have overcome the challenges in conventional gene therapy. Particularly, CRISPR/nCas9 showed an increased On-targetcut efficiency and decreased the Off-targeteffect.

Recently, gene therapy based on CRISPR/nCas9 system was reported as a potential approach toward MPS IVA in vitro treatment, devoid of the Off-targeteffects and with pathological biomarkers recovery to wild-type levels. Although viral vectors were used due to their natural transduction capacity, the non-viral counterparts could overcome the limitation posed by the viral vectors.

For example, a cell-penetrating vector based on magnetite-silver nanoparticle-based core-shell and pH-responsive magnetite-based nanoparticles were prepared as a carrier for nucleic acids with the ability to form a complex with nucleic acids through a pH-responsive polymer.

The presence of membrane translocating peptide Buforin II on the carrier enabled them to escape the endolysosomal degradation pathway. Furthermore, the fabricated magnetite-based nanoparticles were entrapped in liposomes to prevent extracellular nuclease-induced DNA degradation.

In the present study, the potential of magnetite-based nanoparticles entrapped liposomes were tested as CRISPR/nCas9 carriers by conducting in vitro experiments on MPS IVA fibroblasts with different GALNSgene mutations. The results revealed an increase in the GALNS activity for all MPS IVA fibroblasts, indicating the robustness of the CRISPR/nCas9 system when coupled with magnetite-based nanoparticles.

The effect of magnetite-based nanoparticles coupled CRISPR/nCas9 system was evaluated by using various biomarkers and the results demonstrated a reduction in lysosomal mass, GAGs, and mitochondrial reactive oxygen species (mtROS), confirming the potential of CRISPR/nCas9 system in treating MPS IVA.

To summarize, the CRISPR/nCas9-based genome edition system was used to treat MPS IVA containing different mutations in four in vitro models using a non-viral vector composed of magnetite-based nanoparticles entrapped in liposomes for the delivery of the CRISPR/nCas9 system.

The results revealed an increased GALNS activity for all the MPS IVA fibroblasts, demonstrating a reduced lysosomal mass, mtROS, and GAGs after treatment with CRISPR/nCas9 system using both magnetite-based nanoparticles entrapped in liposomes and conventional liposomes.

The -hexosaminidase activity was also observed to have recovered after transfecting the CRISPR/nCas9 system, confirming the CRISPR/nCas9 system as a potential strategy for MPS IVA treatment when combined with a non-viral vector. Thus, through the present study, the importance of genetic backgrounds during the evaluation of therapeutic strategies was demonstrated for therapy success.

Leal, A.F et al.(2022) Delivery and assessment of a CRISPR/nCas9-based genome editing system on in vitro models of mucopolysaccharidoses IVA assisted by magnetite-based nanoparticles.Scientific Reports12, 15045. https://www.nature.com/articles/s41598-022-19407-x

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CRISPR/nCas9 Could Be Used to Treat Mucopolysaccharidosis IV A - AZoNano