Category Archives: Gene therapy

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

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

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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

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.

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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.

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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.

Story continues

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

Disclaimer: The views expressed here are those of the author expressed in their private capacity 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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CRISPR/nCas9 Could Be Used to Treat Mucopolysaccharidosis IV A - AZoNano

PARIS, Sept. 6, 2022 /PRNewswire/ -- Coave Therapeutics ('Coave'), a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases, today announced the appointments of Catherine Mathis, PharmD, as Vice President Regulatory Affairs and Julien Berger, as Head of Legal Affairs.

Ms. Mathis will define and oversee the regulatory strategy and operations supporting the advancement of Coave's programs. Mr. Berger will lead the Company's legal activities and act as Corporate Secretary.

"We are delighted to welcome Catherine and Julien to the team at Coave. The newly created regulatory and legal positions demonstrate the growth of the company and the rapid advances we are making with our pipeline of novel coAAV gene therapies focused on CNS and eye diseases. Catherine brings a depth of experience in regulatory affairs for gene therapy based programs that will be invaluable as we plan and implement our regulatory strategy for our three CNS programs targeting protein degradation. Julien has extensive experience representing and advising companies in various legal activities at all stages of the pharma and biotech life cycle, supporting the implementation of operations while ensuring the protection of the companies' rights and interests. Catherine and Julien are key and timely additions to our leadership team and we look forward to working with them as we strive to transform the treatment of CNS diseases with our first-in class gene therapy products," said Rodolphe Clerval, CEO of Coave Therapeutics.

Catherine Mathis, PharmDCatherine has over 30 years' experience in clinical research and regulatory affairs in the pharmaceutical and biotech industries and has developed robust expertise for gene therapy development from Phase 1 to Phase 3 under US and EU regulations. She conducted many regulatory filings, IND and Scientific Advice meetings with the US Food and Drug Administration (FDA), European Medicines Agency (EMA) and other European national competent authorities.

Catherine spent 20 years at Transgene as Senior Director, Head of Regulatory Affairs setting up and leading the regulatory affairs department and supervising global regulatory activities for the development of Transgene's gene therapy products. Since Transgene, she has held senior leadership and regulatory roles at Voisin Consulting Life Sciences, TxCell (a subsidiary of Sangamo Therapeutics), Elsalys Biotech and most recently Enterome.

Catherine holds a PharmD and a Master's degree in applied and basic toxicology from Paris University. She began her career in clinical research roles at Ipsen and Sanofi Pasteur.

Julien BergerJulien brings to Coave almost 20 years of global, regional and local healthcare legal affairs experience among corporate, commercial, medical, R&D, clinical operations and M&A activities. He joins Coave from Galapagos where he served as Senior Legal Counsel Director providing legal support and advice to its global teams.

Prior to Galapagos, Julien spent over 15 years in the legal team at Genzyme, most recently as Legal Director at Sanofi Genzyme following its acquisition. Julien developed and led the legal department for Genzyme's French entity, supporting the growing business in five therapeutic areas, managing the launch of products in addition to supporting Genzyme's acquisition by Sanofi.

Julien is a Business Law graduate from the universities of Lyon and Strasbourg.

About Coave Therapeutics

Coave Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases.

Coave Therapeutics' next-generation AAV-Ligand Conjugate ('ALIGATER') platform enables targeted delivery and enhanced gene transduction to improve the effectiveness of advanced gene therapies for rare diseases.

The company is advancing a pipeline of novel therapies targeting CNS and eye diseases where targeted gene therapy using chemically modified AAVs (coAAVs) has the potential to be most effective.

Coave Therapeutics, which is headquartered in Paris (France), is backed by leading international life science and strategic investors Seroba Life Sciences, Tha Open Innovation, eureKARE, Fund+, Omnes Capital, V-Bio Ventures, Kurma Partners, Idinvest, GO Capital and Sham Innovation Sant/Turenne.

For more information, please visit http://www.coavetx.comor follow us on LinkedIn

CONTACTS

Coave TherapeuticsRodolphe Clerval, CEO[emailprotected]

MEDiSTRAVA ConsultingSylvie Berrebi, Eleanor Perkin, Mark Swallow PhD[emailprotected]Tel: +44 203 928 6900

SOURCE Coave Therapeutics

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Coave Therapeutics Strengthens Leadership Team with the Appointments of Catherine Mathis as Vice President Regulatory Affairs and Julien Berger as...

As part of WuXi AppTec's ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 - "Delivering on the Promise of New Modalities" - so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs.

We continue our interview series with Yann Chong Tan, Founder & CEO of Nuevocor, a preclinical-stage biotech company specializing in gene therapy for cardiomyopathies. Recently, Nuevocor closed on $24 million Series A Financing to advance their novel gene therapies. The company believes they are well-positioned to treatLMNA (lamin A/C gene) and other genetic cardiomyopathies, amongst other cardiac diseases.

Nuevocor focuses on developing gene therapy-based treatments that have the potential to restore cardiac function in diseased hearts. In your opinion, what are the top challenges in current therapeutic intervention for this disease?

Yann Chong: At Nuevocor, we focus on using gene therapy to treat genetic cardiomyopathies. Cardiomyopathy is estimated to affect more than 1 in 250 people and is a disease where the heart gradually loses its ability to pump blood efficiently. Treatment options are limited and the only cure is a heart transplant. With the advent of precision medicine over the past decades, we now know that a large fraction of cardiomyopathies has genetic causes. As each of these genetic cardiomyopathies has a different mechanistic driver, a challenge is understanding the underlying root causes to design targeted treatments.

How is your gene therapy approach helping to address these challenges? How is it different from existing approaches?

Yann Chong: The majority of genetic cardiomyopathies are inherited in an autosomal dominant manner where one copy of the mutated gene drives the disease even though the patient has a second, normal copy of the gene. This makes a gene replacement approach unworkable. There are also no mutation hotspots for gene editing to be attractive. Our approach is to identify genetic modifiers of cardiomyopathies and deliver them to patients via AAV-mediated gene therapy. To identify such genetic modifiers as therapeutic targets for genetic cardiomyopathies, we utilize our PrOSIA mechanobiology platform to understand the molecular and biomechanical basis of genetic cardiac disease.

To illustrate the power of our approach, our lead programme targets LMNA dilated cardiomyopathy. This is the second most common genetic dilated cardiomyopathy and has the worst prognosis, with 70% of patients having cardiac death, a heart transplant or a major cardiac event by the age of 45. Our treatment reduces biomechanical stress on the nucleus of LMNA mutant heart cells, thereby reducing nuclear damage in LMNA dilated cardiomyopathy. In animal models, we have extended lifespan by more than 4-fold with our approach, which is unprecedented for this rare disease.

What are critical challenges in realizing the full potential of your gene threapy? What are Nuevocor's solutions?

Yann Chong: The benefits of AAV-mediated gene therapy have been demonstrated with drug approvals and have made a significant difference in patients' lives. Recent clinical trials, however, have highlighted that there are still quite some ways to go in ensuring that the therapy is safe for patients especially at high doses. The field is coming together to solve these issues and there has been incredible progress in understanding the cause of the safety challenges and in designing solutions.

We are actively working on addressing these safety challenges by examining how we can lower the AAV dose and ensuring that our therapeutic protein is not immunogenic. We have made significant progress in the past year and we are confident that we will be able to have a solution in the coming year.

To make a prediction, what could be the next big scientific breakthrough in the life science industry?

Yann Chong: It is difficult to predict the next big invention, but what is clear is that techniques regularly used in research to perturb systems and answer biological questions, such as anti-sense oligos, or mRNA and DNA vectors for expressing and regulating genes, have formed the basis for new drugs in recent years. There is no turning back from these new nucleic acid therapeutic modalities, which promise to address the root cause of diseases so as to provide cures for patients. Enabling wider use of these technologies is in the interest of patients and their families.A challenge for these modalities is delivering the therapeutic molecule to the intended cell types, ideally with good specificity and importantly, also in a way that is safe. We expect current delivery methods to mature, and at the same time, a proliferation of different delivery methods, both viral and non-viral. It is likely that each delivery method will have its own advantage in being able to deliver well to different organs and cell types. We anticipate the next breakthrough to be the ability of the field to deliver to different cell types achieved through the coexistence of multiple viral and non-viral delivery technologies.

Thank you for your insights! Any closing remarks to our readers?

Yann Chong: We believe in moving our therapies as quickly as possible into the clinic, and collaborations between different groups with complementary capabilities can accelerate the process. Capabilities do not respect borders and ideally collaborations should be cross-border too. Nuevocor is headquartered in Singapore, a cosmopolitan global city with English as the main communication and is in many ways a bridge between Asia and the West. It should come as no surprise that we already have global collaborations with our strong ties to North America, Europe and Asia. We are always actively seeking more collaborations to move the science forward.

Yann Chong Tan, PhD, brings a decade of experience in biotech and commercialisation leadership roles to Nuevocor. He is an inventor on multiple patents and an entrepreneur who has founded multiple biotech companies. Dr. Tan was previously the Chief Innovation Officer at the Genome Institute of Singapore, where he initiated and led strategic programmes resulting in multiple healthcare and biotech spin-offs, one of which is Nuevocor. Previously, as Chief Technologist at Atreca, Inc., a company he co-founded in 2012 from his academic work, Dr. Tan led and built up Atreca's core foundational antibody discovery technology and discovered the antibody now in clinical trials. Atreca is listed on NASDAQ.

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Delivering on the Promise of New Modalities: An Interview With Yann Chong Tan, Founder & CEO, Nuevocor - Marketscreener.com

One thing you learn when doing research in the Amazon is that the jungle comes to life at night.

On a night trail my team and I came across a two-striped forest-pitviper.

In capturing this rare and venomous snake to take measurements, I never felt scared or in danger. I was just so thrilled to see such a cool animal and get the opportunity to take some amazing photos of it.

I studied biology at university, and the natural world has always been a fascination of mine, with missed school meaning I spent a huge amount of time at home watching nature documentaries.

I first visited the Amazonon a school trip to Ecuador at age 15, staying in an eco-tourist lodge, and havesince wanted to return for a longer and more immersive trip but the haemophilia Id been battling for most of my life didnt make it easy.

Haemophilia means the blood doesnt clot as it should because some blood-clotting proteins are missing: haemophiliacs can bleed for an extremely long time after even a small cut or injury.

The bleeding can happenspontaneouslyand for no obvious reason; andthe most dangerousbleeds are usually the internal ones.

While I didnt suffer any bleeds on that earlier trip, on some previous holidays with friends, I had, meaning Id have to stay in to rest for several days until I could bear weight on the injured area.

My diagnosis of haemophilia Bcame when I was one.My mum raised me as a single mother and did an amazing job at ensuring I understood my condition and how to communicate any pain from as early as possible.

I dont recall any of the early trauma, however I was hyper aware of my condition throughout school. On occasions I would be taken home early for treatment or rushed to hospital after a bleed.

Growing up with haemophilia stopped me from doing certain things. I wasnt allowed to take part in contact sports at school, which can be quite isolating when all your friends are playing for the rugby and football teams.

I experienced a fair amount of internal joint and muscle bleeds growing up. These are extremely painful and debilitating repeated, traumatic bleedinginto my left ankle has left me with irreversible damage to the joint, which still affects me today.

The most painful bleeds I have experienced are into the iliopsoas muscle, a large muscle inthe inner hip, which was excruciatingly painful and debilitating.

I didnt havea bad quality of life living with haemophilia, but there was no ignoring the fact it impacted my life in a fairly significant way.

Idhave bi-yearly check-ups with a doctor at my haemophilia centre.We would discuss my reported bleeds, look at how well my treatment wasworking and take blood tests. Id also have to inject myself with medication intravenously three times a week.

This was not a pleasant experience, firstly for my mum who treated me as a child. Then, around age seven,I started to learn to inject myself. Hitting a vein isnt easy when youreso young but it wasa necessary step towards becoming independent.

Growing up, Id hear repeatedly, over many years, thatgene therapy an area of medicine that corrects a medical issue stemming from genes, and which offer a cure for haemophiliacs through introducing consistent, steady clotting was always just fiveyears away.

So,I resigned myself to the fact thatI probably wouldnt see it realised until I was an old man.

Then, during a regular check-up, my doctor told me about a trial that was currently enrolling for gene therapy. Having followed treatment advances and scientific news in haemophilia for a few years, I couldnt wait to hear more about it.

I had plenty of questions for my doctor, but I also sought out answers from other professionals in the haemophilia community, like nurses and other doctors and leading patient advocates.

Ultimately, I gained enough confidence in the treatment for me to feel comfortable in progressing with the trial.

If nobody puts themselves forward for these trials, youd never knowthetrue potential of the treatment. So I decided to volunteer.

My reasoning was that as Idbeen fairly fortunate in my life with haemophilia I had access to safe, effective treatment I had a chance to give something back to the community.

In all kinds of fields, gene therapy has huge potential. But we need the data and I washappy to contribute to that.

AlthoughI often had to remind myself why I was doing it, I dont regret a thing and am so grateful for being able to take part in the trial

My gene therapy started inFebruary 2020 when I received the one-dose of gene therapy via intravenous infusion. As part of the trial protocol, I started on high dose immunosuppression at the beginning of March. Then Covid struck.

I had a weakened immune system and had to shield in temporary accommodation alone,close to my hospital for nearly sixmonths.

It was a really worrying and uncertain time for me especially because,despite having haemophilia, I would not have otherwisehad to shield during the pandemic. It was only due to being part of the trial and on immunosuppression that this was the case.

The only time I left the apartment was to attend the hospital multiple times a week for blood tests, to handin my biological samples and forgeneral observations. I didntsee anyone elseon the trial, only my doctor and nurse.

I wont sugarcoat how difficult the first year of the trial was for me. I experienced very strong side effects from the immunosuppression. It was the most unwell I have felt in my life.

Every bodily function felt out of kilter; weight gain, trouble sleeping, acne, nausea, migraines, erratic bowel movements, mood swings, tremors and on top of all of this, I had to deal with the mental health impact of isolating.

But althoughI often had to remind myself why I was doing it, I dont regret a thing and am so grateful for being able to take part in the trial.

After nine months I was allowed to leave the shielding accommodation and thankfully, around the year mark, things started to get easier. Hospital visits became less frequent andI stopped immunosuppression.

I couldalsostart experiencing the benefits that gene therapy offered.

Simply being able to go to the gym or go for a run without wondering if my clottingfactor levels were high enough to protect me from bleeding, felt liberating.

Before the therapy, I would require regular injections of clottingfactor to keep my clottinglevels high enough in order to prevent bleeding, but Icould still experience a breakthrough bleed.

Butsince thetherapy, my factor levels are in the mild haemophilia range, which is high enough to prevent most bleeding without the need for regular injections.

This year, I realised Ineeded a break after the intensity of trial and the past twoyears. I wanted to get out there and truly test the efficacy of thegene therapy.

Having saved some money during the pandemic thanks to shielding in accommodation provided by the clinical trial, I found anopportunity to go abroad; and I jumped at the chance.

I went away for around two months, spending seven weeks living at a research station in the Peruvian Amazon, as part of an internship with a conservation organisation, Fauna Forever followed by 10 days travelling in Southeast Peru and hiking the Inca trail.

Shortly after I attended the World Federation of Haemophilia 2022 Conference in Montreal, spending several weeks road-tripping across Canada and hiking around the national parks.

Due to thepractical limitations of storing and transporting enough medication, I would never usuallygo away for more than two weeks.

A huge amount of planning was required to ensure I always had access to medication and local haemophilia centres.

When I visited Peru and Canada, I took fouremergency doses of factor with me just in case.

I didnt use any. I didnt have a single bleed, which was a huge relief.

Taking a trip like this is something that would have been made so much more difficult if I was still reliant on regular clottingfactor replacement. Id have to have taken a lot of treatments with me, worried about keeping themcool enough, and injecting myself regularly.

Having experienced a life without severe haemophilia, it would be difficult to go back to the way things were.

I definitely hope to travel more, especially more spontaneously, as I can now enjoy day to day life without the burden of my condition. Post gene therapy I realise just how much of my time and energy managing my symptoms required.

Im just focusing on the here and now, and enjoying what I have.

Do you have a story youd like to share? Get in touch by emailing jess.austin@metro.co.uk.

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I am a haemophiliac - I spent years terrified of bleeding - Metro.co.uk