Archive for the ‘Gene Therapy Research’ Category

In October 2019, Jordan Janz became the first person in the world to receive an experimental therapy for cystinosis, a rare genetic disease. The treatment was physically grueling. Doctors extracted blood stem cells from Janzs bone marrow and genetically modified them in a lab. Meanwhile, he underwent chemotherapy to clear out the remaining faulty cells in his bone marrow before he got the newly modified ones. The chemo gave Janz sores in his mouth so painful that he couldnt eat. He lost his head full of pale-blond hair.

But Janz, then a 20-year-old from Alberta, Canada, had signed up for this because he knew that cystinosis was slowly killing him. The mutated gene behind this disease was causing toxic crystals of a molecule called cystine to build up everywhere in his body. He threw up constantly as a kid. Visible crystals accumulated in his eyes. And his kidneys were now failing. Cystinosis patients live, on average, to 28.5 years old.

Fortunately, the experimental gene therapy seemed to work; Janz began to feel better. His hair grew back in a stubble, but to his shock, it came in a different color: dark, almost black. In the two and half years since, his hair has settled into a dark blond, which is still markedly different from the almost white blond of before. My girlfriend actually said the other day that she feels like shes dating a different person, Janz told me.

Of all the things the experimental gene therapy was expected to altersuch as the severity of his cystinosis symptomshair color was not one of them. That was very surprising, Stephanie Cherqui, a stem-cell scientist at UC San Diego and the principal investigator of the gene-therapy trial, told me. But as she and her colleagues dug into the literature on the disease, they found that darker hair wasnt a sign of something going awry; instead it might be a very visible sign of the gene therapy working.

Doctors had observed years ago that cystinosis patients tend to be paler than their families. Manythough certainly not allhave blond hair and pale skin. One study in mice found that the gene thats mutated in cystinosis patients normally plays a role in the production of the dark-brown pigment melanin. Janz had always been a bit self-conscious about how pale he was. His whole family is pretty pale, Janz said. But I'm, like, a whole different paleor I was. The hair change, as far as hes concerned, was a nice surprise.

But how did genetically modifying his blood cells change his hair color? While the mutation that causes cystinosis affects virtually every cell in his body, gene therapy did not change the DNA of every cell in his body, only a tiny fraction of them. Scientists chose to genetically tweak blood stem cells because they have a special ability: Some eventually become white blood cells, which travel to all different parts of the body, Jeffrey Medin, who studies gene therapy at the Medical College of Wisconsin, told me. White blood cells normally go into all our different tissues and organs to patrol for pathogens.

Janzs new white blood cells were genetically modified to express the gene that is mutated in cystinosis, called CTNS. Once they traveled to his eyes, skin, and gut, the white blood cells began pumping out the missing protein encoded by the gene. Cells in the area began taking up the protein and clearing away long-accumulated cystine crystals. In Janz, the anti-cystine proteins from his modified blood cells must have reached the hair follicles in his skin. There, they cleared out the excess cystine that was blocking normal melanin production, and his hair got darker. The same phenomenon has played out in other people: So far in the gene-therapy trial, four of the five patientsall of whom are whitehave gotten darker hair. (The fifth patients hair is just starting to grow back post-therapy.) The investigators have since added hair biopsies to the trial in order to track the color changes in a more systematic fashion.

The sudden hair-color changes were surprising to Cherqui and her colleagues, but they are consistent with the role of the cystinosis gene in hair pigments, says Robert Ballotti, a melanin researcher at the French National Institute of Health and Medical Research. But he has also found that pigmentation and cystinosis can interact in unexpected ways. Not all people with cystinosis are pale, and in particular, Black patients tend not to have skin or hair that is any lighter. Maybe there is not a strict correlation between the gravity of the disease and pigmentation, Ballotti says.

Hair color is one way in which patients in the clinical trial are teaching scientists about the full scope of the CTNS gene, which is still not fully understood. Cherqui had helped discover the gene, as a graduate student more than 20 years ago, and her research has hinted at other functions for it in cell growth and survival, too. More and more, we understand that there are many functions of the protein that we didn't know, she said.

Thats why patients on the standard treatment, a drug called cysteamine, still get sicker and die of their disease, Cherqui said. Removing cystine is not enough. It doesnt help that cysteamine has some pretty nasty side effects: It causes stomach pain, nausea, and diarrhea. When Janz was very young, he needed a stomach tube to get the medication around the clock. Cysteamine also has a rotten, fishlike smell. I had a lot of difficult times as a younger kid, says Jacob Seachord, another patient in the trial whose hair went from blond to brown. I smelled really bad from medication, so I didn't make a lot of friends.

Gene therapy actually replaces the missing protein, theoretically filling in all of its functions, known and unknown. All five patients in the gene-therapy trial have gone off their oral cysteamine, and preliminary data show they now have fewer cystine crystals in their eyes, skin, and gut. Their vision has gotten slightly better, too. But improvements in kidney function are more elusive. Seachord had a kidney transplant before the gene therapy and is doing well. Janz had advanced kidney disease before the trial, and he will need a kidney transplant in a few months.

For adults with cystinosis, Cherqui said, it may be too late for gene therapy to help their kidneys. They have already accumulated a lifetime of kidney damage from cystine. Gene therapy cant reverse the damage thats been done, but we can correct it going forward, Medin said. We can stop progression. In diseases like cystinosis, patients may have to get gene therapy at a young age, probably before 10, Cherqui said. If it works, a future kid who has cystinosis might be cured through gene therapypreventing them from needing a lifetime of cysteamine or a kidney transplant. And it just might change their hair color, too.

The rest is here:
An Experimental Gene Therapy Changed His DNAAnd His Hair Color - The Atlantic

Alaunos Therapeutics and the National Cancer Institute Extend Cooperative Research and Development Agreement for Development of Personalized TCR-T Cell Therapies To 2025

NCI will lead the Company's personalized TCR-T cell therapy program using the Company's proprietary non-viral Sleeping Beauty technology

HOUSTON, June 27, 2022 - Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company, today announced that the Company has extended its Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), an institute of the National Institutes of Health, using the Alaunos Sleeping Beauty technology through January 2025.

Under the terms of the CRADA, the NCI will work to generate proof of concept utilizing the Company's proprietary non-viralSleeping Beauty technology for personalized TCR-T cell therapy. In this setting, T-cell receptors (TCRs) that react to the patient's tumor will be identified from the patient and used to generate a TCR-T cell therapy. This approach could potentially apply to a wide range of solid tumor cancer patients. Alaunos believes that the non-viralSleeping Beauty technology could rapidly and cost effectively produce safe and potent TCR-T cell therapies without the complexity of gene editing or viral approaches. Research conducted under the CRADA will be led by Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI's Center for Cancer Research.

"We are privileged to extend the productive collaboration with Dr. Rosenberg, a cell therapy pioneer. Dr. Rosenberg and the NCI are working to develop personalized cancer therapies using our novel TCR-T cell platform," commented Kevin S. Boyle, Sr., Chief Executive Officer of Alaunos. "Our collaboration reinforces our commitment to improving the lives of cancer patients with solid tumors. We look forward to continuing our collaborating with Dr. Rosenberg and his team to generate proof of concept in this personalized TCR-T approach."

Drew Deniger, Ph.D., Vice President, Research & Development at Alaunos added, "Having worked alongside Dr. Rosenberg for many years, I am confident that his team at the NCI will be successful in developing personalized TCR-T therapies using our non-viralSleeping Beauty technology. As the world's experts in Sleeping Beauty, we believe that our non-viral means of adding the TCR to T cells is well suited for a personalized approach, with potential to further increase the addressable population for TCR-T therapies."

About Alaunos Therapeutics

Alaunos is a clinical-stage oncology-focused cell therapy company, focused on developing T-cell receptor (TCR) therapies based on its proprietary, non-viralSleeping Beauty gene transfer technology and its TCR library targeting shared tumor-specific hotspot mutations in key oncogenic genes including KRAS, TP53 and EGFR. The Company has clinical and strategic collaborations with The University of Texas MD Anderson Cancer Center and the National Cancer Institute. For more information, please visit http://www.alaunos.com.

Forward-Looking Statements Disclaimer

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," "believes" or other words or terms of similar meaning. These statements include, but are not limited to, statements regarding the Company's business and strategic plans, the anticipated outcome of preclinical and clinical studies by the Company or its third-party collaborators, the Company's cash runway, and the timing of the Company's research and development programs, including the anticipated dates for filing INDs, enrolling and dosing patients in and the expected timing for announcing preclinical data and results from the Company's clinical trials. Although the management team of Alaunos believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Alaunos, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include, among other things, changes in the Company's operating plans that may impact its cash expenditures; the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Alaunos' product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Alaunos' intellectual property rights; and competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Alaunos, including those risks and uncertainties listed in the most recent periodic report filed by Alaunos with the Securities and Exchange Commission. Alaunos is providing this information as of the date of this press release, and Alaunos does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events, or any other reason.

Investor Relations Contact:

Alex Lobo

Stern Investor Relations

Alex.lobo@sternir.com

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Alaunos Therapeutics Inc. published this content on 27 June 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 27 June 2022 12:14:02 UTC.

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Alaunos Therapeutics : and the National Cancer Institute Extend Cooperative Research and Development Agreement for Development of Personalized TCR-T...

BOSTON--(BUSINESS WIRE)--Carbon Biosciences (Carbon), a Longwood Fund founded biotech company and emerging leader in the development of novel parvovirus-derived gene therapies, today announced a $38 million Series A financing led by Agent Capital. Agent is joined by Longwood Fund, Astellas Venture Management LLC, the Cystic Fibrosis Foundation, Solasta Ventures, University of Tokyo Innovation Platform (UTokyoIPC), and Camford Capital. Carbon is harnessing novel parvovirus vectors that can deliver larger gene therapy payloads with enhanced tissue specificity and with minimal neutralizing immunity. The company plans to use the Series A funding to advance the development of Carbons programs for genetic diseases, initially building on the groundbreaking research of scientific co-founders John F. Engelhardt, Ph.D., Director, Center for Gene Therapy at the University of Iowa, and Robert M. Kotin, Ph.D., Professor of Microbiology and Physiological Systems at the University of Massachusetts Chan Medical School.

Carbons platform has the potential to expand the role of gene therapy in treating some of the worlds most devastating and difficult to treat diseases, said Joel Schneider, Ph.D., President and CEO of Carbon Biosciences. Our vision is to enable a new generation of genetic medicines with differentiated vectors that have the potential to address the immunological, targeting and payload limitations inherent in current viral and non-viral delivery technologies. As the first jointly funded program launched under a collaborative agreement between the Cystic Fibrosis Foundation and Longwood Fund, we are motivated by the potential impact our research may have in significantly improving care for cystic fibrosis patients.

Identifying vectors that can effectively deliver therapeutics to target tissues, such as the lung, has been a major challenge in realizing the full potential of gene therapy, said Geeta Vemuri, Ph.D., Managing Partner and Founder of Agent Capital. We believe that Carbon's proprietary platform can address this challenge by leveraging novel vectors from the broader parvovirus family to deliver optimal payloads to specific tissues. Carbons technology will enable a diversified pipeline with potential applications across the wide range of tissues impacted in many unaddressed diseases.

Carbons novel platform addresses key challenges with AAV and non-viral based therapies. Our lead program is the first gene therapy program demonstrating tissue tropism to the lung with the capacity to deliver the full length CFTR gene and an appropriate promotor, said John F. Engelhardt, Ph.D., the Roy J. Carver Chair in Molecular Medicine, and Director, Center for Gene Therapy at the University of Iowa. Preliminary pre-clinical data as well as studies on human populations suggest wide applicability of our lead clinical candidate and the potential to re-dose patients.

Carbons scientific co-founders and scientific advisory board members are among the most experienced thought leaders in the gene therapy field. In addition to Kotin and Engelhardt, Carbons co-founders are David Steinberg, who served as founding CEO (Longwood Fund); Jianming Qiu, Ph.D., (University of Kansas); Ziying Yan, Ph.D., (University of Iowa); Sebastian Aguirre, Ph.D., (Carbon Biosciences); and Lucy Liu, Ph.D., (Longwood Fund). Carbons Scientific Advisory Board consists of John Engelhardt; Jay Chiorini Ph.D., Senior Investigator at NIH National Institute of Dental and Craniofacial Research; Beverly Davidson, Ph.D., Chief Scientific Strategy Officer at Childrens Hospital of Philadelphia; and Adrian Thrasher, Ph.D., MBBS, FRCP, MRCP, Professor at University College London (UCL), Great Ormond Street Institute of Child Health, Head of Infection and Inflammation and Director, Clinical Gene Therapy GMP Facility at UCL.

In conjunction with the financing, Joel Schneider, Ph.D, joins as President and CEO. Chen Schor, Adicet Bio President and CEO, joins as Board Chair. David Steinberg, Geeta Vemuri, Robert Kotin and Derek Yoon, President and CEO of Solasta Ventures, join the Board of Directors.

About Carbon Biosciences

Carbon Biosciences is expanding the therapeutic potential of gene therapy through its proprietary platform which leverages novel parvoviruses that have been pressure tested by nature to target specific tissues and carry a larger cargo with minimal neutralizing immunity and the potential to re-dose. Founded by Longwood Fund and gene therapy pioneers, John F. Engelhardt, Ph.D., and Robert M. Kotin, Ph.D., Carbon is expanding the gene therapy toolbox for the treatment of the worlds most devastating and difficult to treat diseases. For more information, please visit our website http://www.carbonbio.com and follow us on LinkedIn.

About Longwood Fund

Longwood Fund is a venture capital firm dedicated to creating and investing in novel healthcare companies that develop important treatments to help patients while targeting significant value for investors. The Longwood team has a long history of successfully launching and building important life science companies while providing operational leadership and strategic guidance. Collectively, the Partners at Longwood Fund have co-founded 24 companies with over 20 launched or marketed drugs and therapies, as well as over two dozen clinical stage assets, all focused on helping patients in need. Companies founded by Longwood Fund, or its principals prior to the founding of the Firm, as lead investor and CEO/CBO include Vertex, Acceleron, Momenta, Alnylam, Sirtris, Vor, TScan, Pyxis Oncology, Immunitas, Be Biopharma, ImmuneID, Tome Bio, Photys, and Carbon Biosciences. For more information, visit http://www.longwoodfund.com.

About Agent Capital

Agent Capital is an international life sciences investment firm that supports disruptive healthcare companies focusing on novel, differentiated therapeutics and treatments that address unmet patient needs. Agent Capital aligns with scientists, entrepreneurs, and other investors to develop the next generation of healthcare innovations, leverages their industry expertise and successful track record to source premier deals, accelerate value, and drive successful exits. Their first fund invested in 15 portfolio companies, the majority of which have executed collaborations with major pharmaceutical companies and successfully raised additional capital in the private or public markets. For more information, please visit Agent Capital's website at http://www.agentcapital.com.

Originally posted here:
Carbon Biosciences Launches with $38 Million Series A Financing to Advance Novel Gene Therapy Platform and Pipeline - Business Wire

The DNA sequencing newcomer Ultima Genomics won the crown for the biggest U.S private biotech investment in May 2022. Other top fundraising firms are developing gene and cell therapies in addition to cultured leather and omics-based diagnostics.

The headliner private biotech investment in the US in May 2022 went to the Californian firm Ultima Genomics. The company exited stealth mode with $600 million to fund the development of high-throughput sequencing technology. The aim is to bring down the cost of sequencing the whole genome to just $100 compared to the roughly $1,000 it costs with current technology.

Ultima Genomics is upping the scale of its sequencing platform by blending a range of advances in fluidics, sequencing chemistry and machine learning. The firm is applying its sequencing technology to multiple types of omics approaches, including whole-genome sequencing, single-cell, and methylation sequencing.

The second biggest private biotech investment in the U.S. went to Kriya Therapeutics, which is based in California and North Carolina. The $270 million Series C round will advance Kriyas preclinical-stage gene therapy pipeline for the treatment of a range of ophthalmological and rare genetic conditions.

The biggest private investment raised by an industrial biotechnology firm in North America in May 2022 was an $82 million Series C round by Vestaron. Based in North Carolinas Research Triangle Park, the firm is developing peptides to protect crops from insect pests. Unlike some chemical pesticides in current use, Vestarons products are designed to be non-toxic for humans and other vertebrates that arent the direct target of the pesticide.

North Americas biggest biotech Series A round was closed by the San Francisco company Terremoto Biosciences. The company will use the winnings to fund the development of a type of small molecule that binds to proteins in a different way than traditional drugs do. This allows the company to put in the crosshairs proteins that were previously considered undruggable.

The Series A runner up in May 2022 was VitroLabs in California, which raised $47.4 million to fund the development of cultured leather. Cultured leather involves farming animal cells to produce leather, rather than using the whole animal. Like the field of cultured meat, the intention is to reduce the land, energy and water requirements of manufacturing animal-derived products.

In the seed round division, the champion was San Diego-based Pleno Inc., which took home $15 million. The company is developing a multi-omic platform to accelerate clinical diagnostics and research. By using a new type of signal-processing technology, the firm claims it can obtain much more information from polymerase chain reaction (PCR) tests and next-generation sequencing technology than from current methods.

Another notable seed round went to the New York-based Oviva Therapeutics, Inc. The firm raised $11.5 million to fund research into the aging process in women. This research focuses on slowing aging in the ovaries, which could lead to a longer lifespan in general.

One major biotech investment in the U.S. in May didnt make this list because it was of a new investment firm called Enavate Sciences. Enavate was launched by the healthcare investment firm Patient Square Capital with a $300 million boost and aims to support drug developers with vital cash as they bring novel medicines to the market.

Looking forward to June, were already seeing some huge investments, with the frontrunner so far being a $625 million Series D round by the biomanufacturing heavyweight National Resilience, Inc.

Cover image via Elena Resko

Read the rest here:
The biggest US biotech investments in May 2022 - Labiotech.eu

PRESS RELEASE

INTESA SANPAOLO INCREASES ITS SUPPORT

OF UNIVERSITY RESEARCH UNDER THE SCOPE OF THE NRRP

THE BANK PARTNERS THE UNIVERSITIES OF BOLOGNA, MILAN,

NAPLES AND PADUA TO SPEED UP PROJECTS ON

BIG DATA, SUSTAINABLE MOBILITY, AGRITECH AND GENE

THERAPIES

Milan, 27 June 2022 - Intesa Sanpaolo intends to strengthen its support of university research by taking part in four National Centres for chain research as founding member of the four Foundations linked to important universities, such as the Bologna Technopole together with the Italian National Institute for Nuclear Physics (INFN), the Polytechnic University of Milan, the University of Naples Federico II and the University of Padua, which will develop new technologies for the sustainable, green digital transition, in line with the European Union Research strategic agenda and the fourth Mission of the NRRP, which finances the new centres with 1.6 billion.

Intesa Sanpaolo consolidates its commitment with the aim of speeding up the activity in synergy with the public sector, to promote the innovation and dissemination of technologies, fostering the sharing of routes and projects between universities, research entities, enterprises and start-ups. With this intervention, Intesa Sanpaolo, the only banking group of the founding members, will be able to take part together with Research Centres and Universities, in the development of projects, being an active partner in the development of technological transfer models to local enterprises and helping determine the strategic guidelines of research, as well as contributing towards the pursuit of the NRRP.

More specifically, the four Centres are as follows:

Thanks to the four new Centres, the Bologna Technopole, the Polytechnic University of Milan and the universities of Naples and Padua will act as hubs connecting with numerous other universities throughout Italy, involving an ever greater number of sites, researchers and enterprises with a view to carrying out advanced research on the topics, respectively, of big data, sustainable mobility, agritech and gene therapy.

The new Centres in fact operate through a Hub&Spoke model, in which the hubs, comprising the centres operating in synergy with some of Italy's most important universities and various private companies, will coordinate and manage the activities of the spokes, represented by entities and other local universities aiming to aggregate public and private subjects interested in making a contribution towards research into specific topics across the country.

Intesa Sanpaolo will be operating directly in 16 spokes, making the professionalism and competences present in the various bank structures involved in these research areas available, as well as technological resources useful to pursuing the objectives.

The main goal is to increase the growth potential of the Italian economic system, through a significant increase in investments in research and development and fostering the technological transfer between universities and enterprises. The new national centres will be able to help reduce or eliminate Italy's delays in terms of innovation: according to an analysis carried out by the Intesa Sanpaolo Research Department, despite the progress made in recent years, Italy is still a long way behind other European countries, and Germany in particular, in terms of spending in research and development. In this context, the interaction is facilitated between the academic and production worlds, allowing for an improvement in the number of manufacturing businesses that collaborate with universities (8%), considerably below the German figure (18%).

The Intesa Sanpaolo Group has always supported investments by enterprises in research and development, both through the management of public incentives for R&D&I and through direct financing for research and innovation. Over time, Intesa Sanpaolo has assessed approximately 2,000 R&D projects with direct financing in excess of 2.5 billion for SMEs, large enterprises and start-ups: this is flanked by the offer of technological, industrial and financial consultancy services for participation in European Research and Innovation programmes and Intellectual Property. Intesa Sanpaolo also supports innovative SMEs and start-ups with a market share respectively of 50% and 30%, also through specific initiatives, believing them to be one of the main vehicles of innovation for enterprises.

The new initiative integrates with the Group's global strategy, which, thanks to dedicated structures like the Innovation Centre and the newly-established CENTAI laboratory for advanced research in Artificial Intelligence, is present in the main Italian accelerators and acts as a nationally-important interlocutor in regard to research and innovation.

Carlo Messina, CEO of Intesa Sanpaolo: " Research centres are a great opportunity for both the Italian university research system and the business world and it is essential we support their

development. Our adhesion to this initiative is a concrete response by the Bank, which makes new resources available to speed up a structural transition in our country towards new models, closely correlated with the NRRP. We aim to offer a new boost to combine private initiative and public decision-makers, like universities, and help generate a culture of knowledge and innovation, to the

benefit of the entire social and economic system".

Ferruccio Resta, Chairman of CRUI: "The investments and measures implemented by the NR RP acknowledge a central role played by research and advanced training in developing a country that seeks to, and indeed must, grow innovatively. They provide a clear interpretation of the relationship between university and enterprise, which is increasingly close on the essential topics guaranteeing competitiveness in both the public and private sectors. The extensive, shared participation of Intesa Sanpaolo, leading Italian bank, shows the value of a tool (the National Centres), which if used properly can assure a significant change that will be completed well beyond 2026".

Press information

Intesa Sanpaolo

Media Relations Territorial Bank and Local Media stampa@intesasanpaolo.comhttps://group.intesasanpaolo.com/en/newsroom

About Intesa Sanpaolo

Intesa Sanpaolo is Italy's leading banking group - serving families, businesses and the real economy - with a significant international presence. Intesa Sanpaolo's distinctive business model makes it a European leader in Wealth Management, Protection & Advisory, highly focused on digital and fintech. An efficient and resilient Bank, it benefits from its wholly- owned product factories in asset management and insurance. The Group's strong ESG commitment includes providing 115 billion in impact lending by 2025 to communiti es and for the green transition, and 500 million i n contributions to support people most in need, positioning Intesa Sanpaolo as a world leader in terms of social impact. Intesa Sanpaolo is committed to Net Zero by 2030 for its own emissions and by 2050 for its loan and investment portfolios. An engaged patron of Italian culture, Intesa Sanpaolo has created its own network of museums, the Gallerie d'Italia , to host the bank's artistic heritage and as a venue for prestigious cultural projects.

News: group.intesasanpaolo.com/en/newsroom/news

Twitter: twitter.com/intesasanpaolo

LinkedIn: linkedin.com/company/intesa-sanpaolo

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Intesa Sanpaolo S.p.A. published this content on 27 June 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 27 June 2022 10:43:02 UTC.

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North America Is Expected To Account For Around 40% Of The Global Reprocessed Medical Devices Market Share By The End Of 2028

Increasing focus on healthcare in developing nations with less spending potential will see major adoption of reprocessed medical devices through 2028, says a Fact.MR analyst

Fact.MR A Market Research and Competitive Intelligence Provider: The reprocessed medical devices market stood at a valuation of US$ 2.05 Bn in 2020, and is projected to surge to US$ 5.9 Bn by the end of 2028.

Increasing healthcare expenditure owing to the rising patient pool is expected to bolster demand for reprocessed medical devices as healthcare institutions focus on lowering operational costs. Increasing geriatric population, favorable regulatory policies, and rising focus on healthcare are anticipated to be other trends propelling reprocessed medical devices market potential over the coming years.

Demand for reprocessed medical devices is anticipated to be high in developing economies where healthcare spending potential is less.

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Key reprocessed medical device suppliers are focusing on launching new and innovative products to advance medical device reprocessing procedures and increase revenue potential.

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Winning Strategy

Reprocessed medical device manufacturers are focusing on mergers and acquisitions to expand their product portfolios and increase their sales. Favorable regulatory policies are also expected to promote reprocessed medical device suppliers to advance their sales potential and increase revenue.

Reprocessed medical device companies are investing in the development of better reprocessing facilities to meet increasing demand. Favorable government initiatives to boost the adoption of reusable medical devices are also expected to be capitalized on by key reprocessed medical devices market players over the forecast period.

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Key reprocessed medical device market players are focusing on mergers and acquisitions to increase their business scope across the world and increase sales.

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The study divulges essential insights on the market on the basis of device type (cardiovascular devices, general surgery devices, laparoscopic devices, orthopedic external fixation devices, gastroenterology biopsy forceps) and across six major regions (North America, Latin America, Europe, East Asia, South Asia & Oceania, and the Middle East & Africa).

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Antibody Drug Conjugates Contract Market - Antibody Drug Conjugates(ADC) Contract Market by Condition Type (Myeloma, Lymphoma, Breast Cancer, Other Condition Types), Application Type (Cleavable Linker, Non-cleavable Linker), and By Region - Global Research 2022 to 2032

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Increasing Healthcare Expenditure to Drive Sales of Reprocessed Medical Devices at 14.9% CAGR through 2028: Fact.MR Survey - BioSpace

Wilmington, Delaware, United States: CRISPR is a genome-editing tool. It is used by researchers/health care professionals to alter DNA sequences and modify gene function.

CRISPR is also used to correct genetic defects, treat human diseases, and yield better crop varieties

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CRISPR stands for Clusters of Regularly Interspaced Short Palindromic Repeats. The CRISPR-Cas9 system consists of two key molecules: Cas9 and guide RNA, which introduces a change or mutation into the DNA at the desired position.

Cas9 is an enzyme that acts as a pair of molecular scissors and can cut the two strands of DNA at a specific location in the genome so that bits of DNA can then be added or removed

The guide RNA (also known as gRNA) is a small piece of pre-designed RNA sequence (about 20 bases long) located within a longer RNA scaffold. The scaffold part binds to DNA and the pre-designed sequence guides Cas9 to the right part of the genome.

This makes sure that the Cas9 enzyme cuts at the right point in the genome. High orthogonality, versatility, and efficiency of CRISPR technology make it a preferred genome editing technology.

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Key Drivers, Restraints, and Opportunities of Global CRISPR Technology Market

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In March 2020, researchers at New York genome center developed a new CRISPR screening technology to target RNA, including RNA of novel viruses such as COVID-19. In November 2019, researchers at ETH Zurich, Switzerland, swapped CAS9 enzyme for CAS 12a, which allowed the researchers to edit genes in 25 target sites.

North America to Dominate Global CRISPR Technology Market

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Key Players of Global CRISPR Technology Market

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CRISPR Technology Market: Rise in Demand for Gene Therapeutics to Drive the Global Market - BioSpace

Biotechnology company Flip Gene Therapeutics has signed an agreement with Aptamer Group to combine its inducible gene therapy platform with the use of Optimer binder technology.

Under the agreement Aptamer will develop binders to several small molecule targets for incorporation into the partners gene therapy platform with the goal of developing pharmacologically inducible gene therapeutics controllable with a gene switch.

Gene therapies treat diseases and conditions through the delivery of genetic material; however, major gene therapy platforms do not allow for precise control of gene expression after the therapy is administered.

Optimer binders used as a component of a gene switch could enable responsive gene expression controlled by dosing certain small molecule drugs.

The commercial terms of the agreement include initial upfront payments for Optimer development, with the potential for further licensing payments for the developed Optimer binders, and development and commercial milestone payments upon clinical and commercial success.

Dr Arron Tolley, CEO of Aptamer Group said: "I am delighted to have established this agreement to develop Optimer binders to enable inducible treatments in this exciting field of gene therapy. We will be working closely with our partner Flip Gene Therapeutics on developing and assessing the Optimer binders to function as part of their gene therapy platform. The interest in pursuing Optimer binders as part of their therapeutic modality offers a strong endorsement of the technology."

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Switching into Gene Therapy Expression Labmate Online - Labmate Online

Theclinical laboratory services marketis expected to experience market growth during the forecast period of 2021 to 2028. Data Bridge Market Research analyzes that the market is growing with a CAGR of 6.6% during the forecast period of 2021 to 2028 and is projected to reach USD 1.26,592.85 million by 2028. Growing application of high throughput testing and advancements in clinical diagnostic methods act as a growth driver for the European laboratory services market clinical.

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Clinical Laboratory Services Market Scope and Market Size

The clinical laboratory services market is segmented by specialty, vendor, application, and service type.Growth between segments helps you analyze growth niches and strategies to approach the market and determine your main application areas and the difference between your target markets.

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Country Level Analysis of the Clinical Laboratory Services Market

The clinical laboratory services market is analyzed and market size information is provided by country specialty, vendor, application, and service type as given above.

The countries covered in the Clinical Laboratory Services market report are Germany, France, UK, Italy, Spain, Russia, Turkey, Belgium, Netherlands, Switzerland and the rest of Europe.

Germany is leading the growth of the European market due to the growing demand for early and accurate diagnosis of diseases.

The country section of the report also provides individual market impacting factors and regulatory changes in the national market that impact current and future market trends.Data points such as new sales, replacement sales, country demographics, regulatory acts, and import-export tariffs are some of the major indicators used to forecast the market scenario for each country.In addition, the presence and availability of European brands and the challenges they face due to significant or rare competition from local and national brands, the impact of sales channels are considered while providing a forecast analysis of national data.

Competitive Landscape and Clinical Laboratory Services Market Share Analysis

Clinical Laboratory Services market competitive landscape provides details by competitor.Details included are company overview, company financials, revenue generated, market potential, research and development investment, new market initiatives, production sites and facilities , company strengths and weaknesses, product launch, product testing pipelines, product approvals, patents, product breadth and scope, application dominance, technology lifeline curve .The data points provided above are only related to the companys focus related to the clinical laboratory services market.

Major companies dealing with clinical laboratory services are Mayo Foundation for Medical Education and Research (MFMER), Eurofins Scientific, UNILABS, SYNLAB International GmbH, HU Groups Holdings, Inc., Sonic Healthcare, ACM Europe Laboratories, Amedes Holding GmbH, Abbott, Charles River, Siemens Healthineers AG and Genomic Health, among other national players.DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Numerous contracts, agreements, collaborations and launches are also initiated by the companies across the globe which are also accelerating the clinical laboratory services market.

For instance,

Product launch, business expansion, rewards and recognition, joint ventures and other strategies by the market player enhance the companys footprint in the Clinical Laboratory Services Market which also benefits to the growth of the organizations profits.

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Clinical Laboratory Services Market is projected to rise to a valuation of around USD 1.26,592.85 million by 2028 Designer Women - Designer Women

EDINBURGH, Scotland, May 18, 2022 /PRNewswire/ -- TC Biopharm (Holdings) PLC ("TC Biopharm" or the "Company") (NASDAQ: TCBP) (NASDAQ: TCBPW), a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer and viral indications, announced today announced the formation of a scientific advisory board (SAB) to advance its gamma-delta T cell therapy, OmnImmune, for the treatment of Acute Myeloid Leukemia (AML).

"We are honored to have these remarkable and accomplished cell therapeutics and scientific leaders join TC BioPharm's Scientific Advisory Board," said Bryan Kobel, CEO of TC BioPharm. "These individuals have made significant contributions and pioneered breakthroughs in cell therapy research and therapeutics, and together, they bring a wealth of knowledge and experience for TC BioPharm, as we work to develop our proprietary therapies to treat blood cancers and develop our platform into other oncological areas. We wil continue to expand our SAB to bring other expertise in cell therapy modalities to reflect our ongoing R&D efforts as well. TCBP looks forward to the input of these outstanding individuals as we advance our platform technology in allogeneic gamma deltas and their contribution to our ongoing research and development efforts in a number of project areas."

Members of the TC BioPharm Scientific Advisory include;

Mark Bonyhadi, Ph D., will lead the SAB. He is a senior advisor to Qiming Venture Partners USA and former Vice President of Research at Juno Therapeautics (acquired by Celgene). Dr. Bonyhadi has more than 30 years of experience in biopharmaceutical leadership roles in the US, specifically in the research and development of commercially viable approaches to take cell and gene therapies, as well as regenerative medicines, from the lab to the clinic and for subsequent commercial development. Prior to his role as vice president of Research at Juno Therapeutics Inc (acquired by Celgene Corporation), he was Director of Global Business Development for Cell Therapy at Invitrogen (which merged to become Life Technologies and was subsequently acquired by Thermo-Fisher) and prior to that, Vice President ofResearch at Xycte Therapies and a Senior Scientist at SyStemix, Inc. He was formerly the chair of the Industry Liaison Committee for the American Society for Gene and Cell Therapy (2015-2016). He is also the inventor on 11 patents and an author on 40 publications. He currently is a member of the scientific advisory board for Akron Biotech and also serves as a Non-executive Director at TCBP and as a Non-executive Director at Integra Therapeutics.

Uma Lakshmipathy, Ph D., has two decades of experience in cell biology, stem cells and translational research. She is currently the Director of R&D in Science and Technology and Head of Patheon Translation Services in Pharma Services Group at Thermo Fisher Scientific. Her work is focused on developing end-to-end, standardized processes and analytics for cell therapy and support translational services destined towards cGMP manufacturing. She has a strong foundation in development of clinical-grade reagents and processes, viral and non-viral methods of cell modification and, analytical platforms for comprehensive cell therapy product characterization. As a junior faculty at the Stem Cell Institute, University of Minnesota, she was involved in ex vivo gene repair of disease mutations in adult stem cells. She has a doctoral degree in Molecular Biophysics from the Center for Cellular and Molecular Biology in India, postdoctoral experience in DNA double strand break repair from University of Minnesota Medical School and has authored several scientific publications, books and patents.

Erin Adams, Ph D., is the Joseph Regenstein Professor of Biochemistry and Molecular Biology at the University of Chicago and an expert in molecular immunology. She explores the molecular cues that the human immune system uses to distinguish between healthy and diseased tissue. Her primary focus is on unconventional, tissue resident effector cells in the human immune system including T cells, MR1-restricted and CD1-restricted T cells. Her laboratory research seeks to understand the role of these cells types in the immune response process and what signals regulate their activity in tissue homeostasis and disease. She has received multiple honors, including being named a Searle Scholar, a Kavli Fellow and awarded a Cancer Research Foundation Junior Investigator Award.

About TC BioPharm (Holdings) PLCTC BioPharm is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of gamma-delta T cell therapies for the treatment of cancer and viral infections with human efficacy data in acute myeloid leukemia. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems and can intrinsically differentiate between healthy and diseased tissue. TC BioPharm uses an allogeneic approach in both unmodified and CAR modified gamma delta t-cells to effectively identify, target and eradicate both liquid and solid tumors in cancer.

TC BioPharm is the leader in developing gamma-delta T cell therapies, and the first company to conduct phase II/pivotal clinical studies in oncology. The Company is conducting two investigator-initiated clinical trials for its unmodified gamma-delta T cell product line - Phase 2b/3 pivotal trial for OmnImmune in treatment of acute myeloid leukemia and Phase I trial for ImmuniStim in treatment of Covid patients using the Company's proprietary allogenic CryoTC technology to provide frozen product to clinics worldwide. TC BioPharm also maintains a robust pipeline for future indications in solid tumors and other aggressive viral infections as well as a significant IP/patent portfolio in the use of CARs with gamma delta t-cells and owns our manufacturing facility to maintain cost and product quality controls.

Forward Looking StatementsThis press release may contain statements of a forward-looking nature relating to future events. These forward-looking statements are subject to the inherent uncertainties in predicting future results and conditions. These statements reflect our current beliefs, and a number of important factors could cause actual results to differ materially from those expressed in this press release. We undertake no obligation to revise or update any forward-looking statements, whether as a result of new information, future events or otherwise. The reference to the website of TC BioPharm has been provided as a convenience, and the information contained on such website is not incorporated by reference into this press release.

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TC BioPharm Announces Formation of Scientific Advisory Board with Renowned Cell Therapy Experts - GuruFocus.com

Cell therapy research is the process of developing and testing whole cells that can be delivered to a patient to treat or cure a disease. Bone marrow transplants are one of the earliest cell therapies developed where bone marrow cell from a healthy person are used to repopulate the bone marrow of patients with some cancers. Newer cell therapies are cells that may be harvested from altered to target a disease or may be cells that are produced in a laboratory. The cell therapy is infused or transplanted into the patient as a treatment for a disease.

Genes are responsible for creating the proteins that control cellular processes. In many diseases, genetic instructions to code for the protein are missing or defective. Gene therapy research is the process by which scientists develop and test a DNA or RNA molecule that could be delivered back to the patient to treat the disease. Scientists first identify the impacted gene and the nature of the defect in patient cells. Depending on the nature of the defect, a gene therapy can be developed that is a proper copy of the gene or suppresses the defective copy of the gene from being used by the cell. The gene therapy is then put inside of a vector or a nanoparticle that can target the therapy to the proper cells when it is administered to the patient. Once delivered to the patient, the gene therapy provides genetic instructions for that can be used by the cell to correct or minimize the impact of the defect.

Researchers work with contract research organizations, like Charles River, by using gene and cell therapy services to prove efficacy and safety through various study types prior to submitting to agency for approval for first-in-human clinical trials. During and after approval of human trials, researchers continue to work with contract research organizations for clinical trial support, extended nonclinical safety studies and manufacturing support. Additional steps and studies are taken following clinical trials to ensure safety in the manufacturing stages before market release.

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Cell and Gene Therapy Services | Charles River

Professor Rachel Lennon, Professor of Nephrology at the University of Manchester said: Rare diseases have an enormous impact on an individuals life, and they do not attract the critical mass of researchers required to enable rapid improvements in treatment. This Kidney Research UK-Stoneygate award to establish a UK hub for Alport Research is fabulous news and it will transform our ability to increase awareness, improve understanding of the condition and to accelerate new treatment options for patients. By bringing together expertise in cell and matrix biology, genetics and clinical practice, we aim to improve genetic testing and to progress a range of therapy options to extend kidney survival in patients with Alport syndrome.

Sandra Currie, chief executive of Kidney Research UK said: Rare diseases offer a unique challenge to charities such as ours and the overall scientific community. Often, there are limited resources attributed to sourcing new treatments as well as an overall lack of understanding into the causes, impacts and ways to treat them. Embarking on this new collaborative way of working, we are tackling this issue head on by bringing together a wealth of facilities and expertise. With this new Hub, we have the potential to accelerate the discovery and testing of new treatments and possibly even a cure through gene therapy.

The diagnosis and treatment of Alport syndrome have improved in the last decade. However, more progress is urgently needed. With the additional resources and funding, researchers are optimistic that results and transformative treatments could begin to emerge from the hub within the next five years.

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Collaborative research approach set to accelerate treatment of rare kidney condition - The University of Manchester

This article was originally posted on genetherapylive.

Cell and gene therapies are currently being evaluated as possible avenues of treatment for multiple inherited retinal diseases in the field of ophthalmology. Here we highlight the most promising therapies currently in development.

GT005 is a gene therapy by Gyroscope Therapeutics currently being evaluated in the recruiting phase 1/2 FocuStrial (NCT03846193) for the treatment of dry AMD. The company announced positive interim data in February 2021.

Regenerative Patch Technologies is developing the cell therapy CPCB-RPE1 for the treatment of geographic atrophy (GA) in dry AMD. It is being evaluated in 16 participants in a phase 1/2 trial (NCT02590692), positive data from which were presented in June 2021.

The cell therapy ASP7317 (Astellas Pharma) is being developed for GA in dry AMD and is being evaluated in 18 participants in a phase 1 trial (NCT03178149).

AAVCAGsCD59/HMR1002, a gene therapy developed by Hemera Biosciences for the treatment of wet AMD, is being evaluated in 25 participants enrolled in an ongoing phase 1 clinical trial (NCT03585556).

Regenxbios gene therapy RGX-314 is being evaluated in the recruiting phase 2/3 ATMOSPHERE trial (NCT04704921) for the treatment of wet AMD. Regenxbio presented positive initial data at the Retina Society 54th Annual Scientific Meeting in October 2021.

Adverum Biotechnologies is developing the gene therapy ADVM-022 for wet AMD, which is being evaluated in the phase 1 OPTIC trial (NCT03748784). The company plans to initiate a phase 2 trial after observing positive results in the OPTIC trial presented at the Retina Society meeting. The therapy was also being evaluated in diabetic macular edema, but that development was halted after serious adverse events, including irreversible vision loss, were seen in participants in the phase 2 INFINITY trial (NCT04418427).

Editas Medicines gene editing therapy, EDIT-101, is being evaluated in LCA type 10 in the phase 1/2 BRILLIANCE trial (NCT03872479). Data presented at the XIXth International Symposium on Retinal Degeneration demonstrated efficacy in some patients as well as safety concerns such as retinal tears and hemorrhage.

The gene therapy rAAV2-CBSB-hRPE65 is being developed by University of Pennsylvania and National Eye Institute. The therapy is being evaluated in 15 participants ina phase 1 trial (NCT00481546).

Sepofarsen (QR-110) is an RNA antisense oligonucleotide in development by ProQR Therapeutics. The therapy is being evaluated in the phase 2/3 ILLUMINATE trial (NCT03913143), results from which were published in Nature Medicine in April 2021. The last patient completed their last, 12-month visit in January 2022.

MeiraGTX's gene therapy cevaretigene ritoparvovec/AAV RPE65 met the primary endpoints in the phase 1/2 OPTIRPE65 trial (NCT02781480) in 2019. Additional clinical progress is expected in 2022.

Gensight Biologics gene therapy GS010 has so far demonstrated positive data in 90 participants in the phase 3 REFLECT trial (NCT03293524), including statistically significant improvements in visual acuity.

4D-110 is 4D Molecular Therapeutics gene therapy currently being evaluated in a recruiting phase 1 study (NCT04483440). The therapy has been well-tolerated in the low-dose cohort but serious adverse events (AEs) were observed in the high-dose cohort.

The University of Alberta is studying the gene therapy RAAV2.REP1 in 6 participants in a phase 1/2 (NCT02077361). Positive 5-year results were presented at The Association for Research in Vision and Ophthalmology (ARVO) 2021 meeting.

The University of Oxford is studying a similar RAAV2.REP1 gene therapy in 30 participants in a phase 2 trial (NCT02407678).

STZ eyetrials gene therapy RAAV.hCNGA3 is being evaluated in the recruiting phase 1/2 Colourbridge trial (NCT02610582).

Applied Genetic Technologies is developing AGTC-401 (NCT02599922) and AGTC-402 (NCT02935517) for CNGB3 and CNGA3 achromatopsia, respectively. Both phase 1/2 trials have demonstrated tolerable safety profiles for the gene therapies.

MeiraGTX is also developing gene therapies for both CNGB3 and CNGA3 achromatopsia. AAV CNGB3 (NCT03001310) and AAV CNGA3 (NCT03758404) are both in phase 1/2 studies.

QR-1123 is another antisense oligonucleotide from ProQR Therapeutics being evaluated in the recruiting phase 1/2 AURORA trial (NCT04123626) for RHO-RP.

MeiraGTXs gene therapy AAV2-RPGR has demonstrated positive data in X-linked RP (XLRP) in a phase 1/2 study (NCT03252847). The therapy will be further evaluated in the phase 3 Lumeos trial (NCT04671433).

Positive efficacy data from Applied Genetics phase 1/2 trial (NCT03316560) of the gene therapy AGTC-501/rAAV2tYF-GRK1-RPGR was presentedat the American Academy of Ophthalmology (AAO) 2021 Annual Meeting. The therapy is being further evaluated in the phase 2/3 VISTA trial(NCT04850118) for XLRP.

4Ds gene therapy 4D-125 was well-tolerated for XLRP in a phase 1/2 trial(NCT04517149), according to data presented at the 2021 American Society of Retina Specialists (ASRS) 39th Annual Meeting.

The National Eye Institute is evaluating RS1 AAV in a phase 1/2 trial (NCT02317887) for XLRP.

Coave Therapeutics gene therapy CTx-PDE6b (AAV2/5-hPDE6B)is being evaluated for PDE6b-RP in a phase 1/2 trial (NCT03328130).

GenSights GS030-DP gene therapy in combination with the GS030-MD optogenetic device has shown positive efficacy and safety data in treating end-stage blindness in RP. These data, from a phase 1/2 trial (NCT03326336), were presented at the 2021 AAO meeting.

Novartis is evaluating their gene therapy CPK805 in RLBP1-RP in a first in-human phase 1/2 trial (NCT03374657) that is currently recruiting.

ProQRs ulteversen/QR-421a gene therapy showed efficacy in the phase 1/2 Stellar trial (NCT03780257) for Usher syndrome and RP in March 2021. ProQR plans to soon advance the program into finalstage clinical testing.

The gene therapy RST-001 is being evaluated in a phase 1/2 trial (NCT02556736). Updates have been sparse for the therapy, which was developed by RetroSense Therapeutics, which was acquired by Allergan in 2016, which was then acquired byAbbvie in 2020.

The first 4 patients treated in the phase 1/2 trial (NCT04278131) of BS01 are able to detect light and motion,Bionic Sight announced in March 2021. The trial continues to enroll participants.

Nanoscope Therapeutics vMCO-I/MCO-010is being evaluated in a phase 1/2 trial (NCT04919473). Positive data in participants with autosomal recessive RP were presented at the ASRS 2021 meeting.

STZ eyetrials gene therapy rAAV.hPDE6A is being evaluated for PDE6A-RP in the recruiting phase 1/2 Pigment trial (NCT04611503).

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A Wrap-Up of Ophthalmologic Cell And Gene Therapies Currently in Development - MD Magazine

REDWOOD CITY, Calif. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, today announced an exclusive agreement with the Medical University of South Carolina (MUSC) Foundation for Research Development to license next generation complement-targeted gene therapies for the treatment of geographic atrophy and other ocular diseases.

Geographic atrophy, also known as atrophic age-related macular degeneration (AMD), is an advanced form of AMD, a progressive retinal disease affecting millions of adults worldwide. Patients with geographic atrophy experience irreversible loss of vision with significant impact on quality of life due to the chronic and progressive nature of the disease. There are currently no FDA-approved treatments available for geographic atrophy.

"This partnership highlights the broad potential of Kriyas technology and R&D platforms to drive innovation in diseases with established biology, and furthers our mission of developing transformative gene therapies for diseases with high unmet need," said Theresa Heah, M.D., M.B.A., President and Chief Medical Officer of Kriyas ophthalmology division. "We believe that complement hyperactivity is a clinically validated target implicated in the pathogenesis of retinal degeneration in geographic atrophy, and has the potential to address other ocular diseases. We are excited to develop gene therapies that precisely target this pathway, in collaboration with academic partners who have been at the forefront of characterizing the biology underlying these diseases.

The complement system plays a crucial role in the bodys innate immune system by enhancing its ability to clear pathogens and damaged cells, and regulating inflammatory immune responses through complement control proteins. Dysregulation and hyperactivity of the complement system is associated with the onset and progression of serious inflammatory diseases, including geographic atrophy and other ocular conditions. Through this agreement, Kriya is advancing gene therapies that are designed to durably express engineered molecules that selectively reduce complement hyperactivity at the site of pathology following one-time administration.

Geographic atrophy due to age-related macular degeneration is a devastating disease with a profound impact on patients, as there are no approved treatments for the irreversible loss of central vision that often occur, said Peter K. Kaiser, M.D., Professor of Ophthalmology, Cole Eye Institute, Cleveland Clinic. Recent advances in complement inhibitor therapies have provided important additional evidence that targeting complement holds great promise in treating geographic atrophy. I am excited by the potential of restoring balance to the complement system with a one-time gene therapy that can deliver a meaningful long-term solution and a major advancement in the field of retinal disease.

By targeting the inhibition of complement proteins directly involved in complement activation, our approach has the potential to deliver a selective and profound biological effect, said Dr. Brbel Rohrer, Ph.D., Professor of Ophthalmology at the Medical University of South Carolina, and co-inventor of the technology. After having confirmed the potential of this gene therapy strategy in mouse models of age-related macular degeneration, we are very excited to take the next step towards a clinical application for patients with geographic atrophy by partnering with Kriya, a leader in the development of novel gene therapies.

About Kriya

Kriya is a fully integrated company pioneering novel technologies and therapeutics in gene therapy. The company aims to revolutionize how gene therapies are designed, developed, and manufactured, improving speed to market, and delivering significant reductions in cost. Kriya is organized into four principal business units: Kriya Technologies, Kriya Therapeutics, Kriya R&D, and Kriya Manufacturing. The company is advancing a deep and diversified pipeline of innovative gene therapies in multiple therapeutic area divisions, with current pipeline programs in ophthalmology, oncology, rare disease, and chronic disease. Kriya was founded by pioneers in the biopharmaceutical industry and is backed by leading life sciences and technology investors. The company has core operations in Silicon Valley, California and Research Triangle Park, North Carolina. For more information, please visit http://www.kriyatx.com and follow on LinkedIn.

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Kriya Licenses Next Generation Complement-Targeted Gene Therapies for the Treatment of Geographic Atrophy and Other Ocular Diseases - Business Wire

Gene therapies have yielded promising results for individuals experiencing rare diseases. However, these groundbreaking therapies come with their own unique set of challenges regarding who will be able to access them, how much they will cost, and how the policymaking and scientific processes will conflict as more and more therapies undergo clinical trials.

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Last week, we convened a panel of experts to address these questions and discuss potential solutions in our latest 5 Slides Were Watching conversation, led by State of Reforms DJ Wilson. The panel featured Danny Seiden, president & CEO of the Arizona Chamber of Commerce and Industry, Dr. Jennifer Hodge, U.S. DMD Gene Therapy Lead at Pfizer, Dr. Rafael Fonseca, chief innovation officer at Mayo Clinic, and Dr. Sharon Hesterlee, chief research officer at the Muscular Dystrophy Association.

Hesterlee brought a slide showing the prevalence of rare diseases in Arizona, noting that 5,500 Arizonans were estimated to be living with rare genetic neuromuscular diseases that were potentially treatable with gene therapy. She highlighted that Charcot-Marie-Tooth disease and Myotonic dystrophy were the most prominent, and that both diseases currently have gene therapy treatments in preclinical development.

She emphasized that ethics need to be an important part of the conversation, and that it will be critical to educate patients and families about the treatments irreversible implications as more and more therapies begin to launch.

Its a permanent change to someone. What we see in particular with parents of a child who has a pediatric disease, they are put in a very difficult position because they have to make a decision without always understanding all of the science and all of the implications.

So I think there is a huge requirement for the physician [who does the informed consent] to be very clear, and then the parents have to decide if it doesnt work, my child cannot be redosed, my child may not be eligible for another trial I think thats been a big challenge and something that weve tried to help our community in the neuro-muscular disease space navigate.

Seiden brought a slide displaying the economic benefits that would come with the increased prevalence of gene therapies. He noted that outdated systems of payment would not be applicable to this kind of treatment, and that these therapies would allow for one-time costs as opposed to a lifetime of treatment for patients with rare diseases.

When you deal with rare diseases, you need to look at it on an annualized basis over the cost of a lifetime, because gene therapy has the potential to save money and a lot of heartache for the patients and the families involved with it Arizona is one of a handful of states that allows for value-based purchasing when it comes to Medicaid contracts With the [Arizona Health Care Cost Containment System (AHCCCS)], which is by far the largest provider, theyve recognized that you have to look at patient outcomes. Its not just about that initial upfront cost.

Hodge presented a slide illustrating the unmet needs of individuals with rare diseases and the potential impacts that gene therapies can have on these individuals. She emphasized the urgent need for innovative treatments for these diseases, as 95% of rare diseases worldwide have limited or no approved treatment options, and 80% of those rare diseases have a genetic cause. She said this makes patients with rare disease collectively one of the most underserved communities in medicine today.

She said educating every organization involved in the process of developing these therapies on the stories of real patients affected by these diseases will be critical as gene therapies move through both scientific and legislative processes.

Its really to address the underlying cause of rare diseases at the root, meaning the genetics, not the symptoms It cant be a line item in a bill, it cant be something on a piece of paper that you hear about, it has to be someone telling their story [and] thinking about the patient and what theyre going through.

You can learn so much by just sitting and talking and just hearing their story, and little things that you didnt even know affected them We need to bring that to more of the audience thats involved in making some of these decisions so they can see it as more than just a line on a piece of paper when theyre deciding something.

Fonseca showed a slide explaining some specific uses of gene therapy that could potentially provide individualized, life-saving treatment to people with red blood cell diseases, as well as preventive genetic interventions for diseases like cancer.

When you think about this approach in looking at the rare disorders, it turns out that by extrapolation, a lot of the diseases that we consider common also become more and more individualized, and therefore, theyre more and more unique. More and more, we see approaches that have to be very, very much [a] tailored design for patients

To have someone who is born with [a red-blood cell disorder] return to normal red blood cell function is just enormous. This is a worldwide problem, its a problem thats associated with pain, serious medical problems, a shorter lifespan, and great expenditures for the health system, and so [Im very excited about where were at with this].

Wilson highlighted that while few gene therapies have been officially launched in the market, many are currently in pre-clinical and clinical trials and are expected to provide promising health solutions for the future.

Continued here:
5 Slides We're Discussing: Gene therapy and the promise for rare disease - State of Reform - State of Reform

Cell lines

Human PC cell lines LNCaP and PC-3 obtained from JCRB Cell Bank (Osaka, Japan), and 22Rv1 purchased from The European Collection of Authenticated Cell Cultures were maintained in RPMI-1640 (FUJIFILM Wako Pure Chemical, Osaka, Japan) containing penicillin, streptomycin and 10% fetal bovine serum (Equitech-Bio, Kerrville, TX). The docetaxel-resistant 22Rv1 cell line, 22Rv1DR, was previously described18. All cell culture experiments were performed using cells within less than 20 passages except for PC-3PRF cells stably transfected with Tet-on tetracycline-inducible perforin expression vector and docetaxel-resistant 22Rv1DR cells.

Docetaxel was purchased from Selleckchem (Houston, TX, USA).

The perforin expression vector for Tet-On system (pT-Rex-DEST30-perforin) was purchased from Thermo Fisher Scientific (Waltham, MA, USA). The pcDNA6/TR regulatory vector (Thermo Fisher Scientific) and pT-Rex-DEST30-perforin vector were transfected to PC-3 cells using Lipofectamine 2000 (Thermo Fisher Scientific). Transfected cells were selected under 500g/ml G418 and 10g/ml Blasticidin (Thermo Fisher Scientific). Perforin was induced by 1g/ml of tetracycline. The human PSA promoter-driven perforin expression vector (pDRIVEperforin-psa-hpsa) was purchased from InvivoGen (San Diego, CA, USA).

Whole cell lysates were harvested and lysed in RIPA buffer containing the protease inhibitor cocktail (Sigma-Aldrich St. Louis, MO, USA). Western blot analysis was performed as described previously19. The anti-PSA and anti--actin antibodies were purchased from Cell Signaling Technology (Danvers, MA, USA). The immunoreactive proteins were detected using horseradish peroxidase-conjugated anti-rabbit antibody (Cell Signaling Technology) and ImmunoStar (FUJIFILM Wako Pure Chemical).

Perforin expression in conditioned medium, mouse serum and harvested xenograft tumors was measured by human perforin ELISA kit according to manufacturers instruction (Abcam, Cambridge, UK).

SS-cleavable and pH-activated lipid-like material (ssPalmM), 1,2-Dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE), cholesterol and 1,2-Dimyristoyl-rac-glycero-3-methoxypolyethylene glycol-2000 (DMG-PEG2000) were purchased from NOF Corporation (Tokyo, Japan). Encapsulation of plasmid vector in lipid nano-particles was conducted according to a previous report20. First, plasmid DNA and protamine solutions (0.3mg/mL and 0.144mg/mL) were prepared in 10mM HEPES buffer (pH5.3). Plasmid DNA/protamine core particle was prepared by the drop-wise addition of 1mL of the protamine solution into the 1mL of the DNA solution with vortexing. Liposome was composed of ssPalmM, DOPE, cholesterol and DMG-PEG2000 in a molar ratio of 3:4:3:0.5. Lipids (3.3mol of total lipids) were dissolved in 2mL of ethanol and the lipid solution (2mL) was rapidly diluted with an equal volume of the plasmid DNA/protamine core particle suspension with vortexing. The solution was further diluted with 36mL of 10mM HEPES (pH 5.3) to obtain 5% ethanol(v/v) concentration. The diluted solutions were concentrated to ten times by Amicon 8400 ultrafiltration stirred cell with a Biomax membrane (Merck Millipore, Allen, TX, USA) following further serial ultrafiltration with 100mM HEPES (pH 7.4) and 10mM HEPES (pH 7.4) using Amicon 8050 ultrafiltration stirred cell with a Biomax membrane. Finally, the liposome solution was filtrated by 0.45m of pore size Millex HV (Merck Millipore).

Liposome concentration was measured as total cholesterol concentration in the presence of sodium dodecyl sulfate using a Cholesterol E test Wako (FUJIFILM Wako Pure Chemical) and the total amount of fatty acids was calculated based of the molar ratio of each lipid. DNA concentration in liposomes was determined using Quant-iT Picogreen dsDNA Assay Kit (Thermo Fisher Scientific) in the presence of Triton X-100. Particle size and -potential were measured at 25C using Zetasizer Nano-S90 (Malvern Panalytical, Worcestershire, UK) after 50 times dilution of samples with distilled water.

This study was approved by the Medical Review Board of Gifu University, Graduate School of Medicine (No. 2018219). A written informed consent was obtained from participants and blood was collected from male volunteers without clinically detectable cancer. All methods were performed in accordance with the relevant guidelines and regulations in compliance with the Declaration of Helsinki. Human PBMCs were isolated by Ficoll-Paque density gradient centrifugation according to the manufacturers instructions (Amersham Biosciences, Piscataway, NJ).

Cells were seeded on 96-well plates. Twenty-four h after seeding, agents with or without PBMCs were added. Cell viability was determined using WST-1 assay kit (Roche Diagnostics, Mannheim, Germany). The mean value obtained from PBMCs alone was deducted from the values obtained from co-culture of prostate cancer cells and PBMCs.

All animal experiments were approved by the Gifu University Animal Experiment Approval Committee (No. 2019116) and carried out in accordance with the approved guidelines. This study is compliant with the ARRIVE guidelines. Six-week-old male athymic nude mice (BALB/cSlc-nu/nu) were purchased from Japan SLC, Inc. (Shizuoka, Japan). A suspension of 22Rv1DR cells (1107) cells in PBS was mixed with Matrigel (1:1) in a final volume of 0.2mL. The mixture was subcutaneously injected to generate tumors. Two weeks after the injection, tumor volume was measured and mice were randomly assigned to 2 groups (n=5). Agents were intravenously administrated via tale vein. The tumor volume and body weight were monitored and measured once a week. Four weeks after treatment, mice were sacrificed and the resected tumors were weighed.

Statistical analysis was performed using Graph Pad Prism 7 version 7.03 (Graph Pad Software, CA, USA). Comparison of 2 groups was made using t-test or MannWhitney U test. Comparison among 4 groups was made using one-way ANOVA with Tukeys post hoc for multiple comparisons. Differences were considered significant if p<0.05.

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Gene therapy of prostate cancer using liposomes containing perforin expression vector driven by the promoter of prostate-specific antigen gene |...

SAN DIEGO--(BUSINESS WIRE)--Excellos, Incorporated, a cell therapy Contract Development and Manufacturing Organization (CDMO), announces its official corporate launch and closing of $15M in growth funding from Telegraph Hill Partners (THP).

Built on the foundation of the San Diego Blood Bank (SDBB), Excellos is focused on supplying cGMP cellular products and services, together with process development and manufacturing expertise to scientists and clinicians working with cell and gene therapies. The companys collection network consists of nine SDBB centers in the San Diego area that see an average of over 70,000 diverse donors annually, as well as exclusive access to select, consented material from SDBBs public cord blood bank. Excellos also has access to a nationwide collection network giving it one of the largest cellular material procurement portfolios. Uniquely connecting its broad collection network to state-of-the-art cGMP and R&D facilities in San Diego, Excellos provides a full suite of innovative end-to-end capabilities to facilitate the development and manufacture of autologous and allogeneic cell therapies. Excellos experience includes working with therapeutic companies developing chimeric antibody receptor-engineered T cells (CAR-T), tumor-infiltrating lymphocytes (TILs), and providing isolated immune cells that are integral to the advancement of immunotherapies.

Excellos will create highly characterized, standardized cell products and custom services that are essential for advanced therapeutics, said David Wellis Ph.D., CEO of Excellos. The SDBB had been incubating Excellos for a number of years, but fully capitalizing on the rapidly growing opportunity required significantly more resources than the SDBB was able to provide. We will now be able to focus exclusively on the needs of the expanding cell and gene therapy industry through the development of our data-driven platform to enable the characterization of cellular therapeutics starting at the donor level. The funding from THP will allow a significant facility expansion and overall growth in the capabilities of our organization.

The growth in the cell and gene therapy industry is driving a surge in demand for critical human cells, tissues, and services to support the development and commercialization of new products, said Paul Grossman, Ph.D., J.D., Partner at THP. We look forward to partnering with Excellos and their proven leadership team. Their exclusive access to one of the industrys largest donor bases, coupled with their technology-focused research and development activities, will help to accelerate the advancement of cell and gene therapies for those patients in need. Paul Grossman, Alex Herzick, and Deval Lashkari from THP have joined the Excellos Board concurrent with the financing.

Excellos founding leadership team brings a wealth of commercial and industry experience to bear. Chief Executive Officer David Wellis Ph.D., previously served as CEO of SDBB for nine years where he foresaw the need for products and services to serve the then nascent cell and gene therapy industry. His guidance allowed the organization to meet these needs, a path that ultimately led to the formation of Excellos. David has also held senior leadership roles at both Illumina and GenVault Corporation. Chief Commercial Officer George Eastwood, brings significant experience in the cell and gene therapy space, with a focus on cGMP materials and cell manufacturing. He served as VP, Global Sales and Business Development for HemaCare, and in his over five years there, he saw the company through a period of dramatic expansion that culminated in its acquisition by Charles River in 2020. Chief Scientific Officer Rob Tressler Ph.D., brings vast experience in R&D for advanced cell-based therapies. Most recently, as CSO of SDBB, he led the cell therapy, immunohematology, components manufacturing, and cord blood banking labs. Rob also has extensive drug development experience, both as Head of Preclinical Oncology at Geron Inc. and VP of R&D at Cellerant Therapeutics.

About Excellos: Built on the foundation of the San Diego Blood Bank, Excellos is focused on supplying cGMP cellular products and services, together with process development and manufacturing expertise to scientists and clinicians working with cell and gene therapies. Excellos is dedicated to improving human life by providing critical products and services to life science research and the next generation of cell and gene therapies. Learn more at http://www.excellos.com

About Telegraph Hill Partners: Telegraph Hill Partners, founded in 2001 and based in San Francisco, CA, invests in commercial stage life science, medical technology, and healthcare companies. For more information, please see http://www.telegraphhillpartners.com

Excerpt from:
Excellos Launches to Accelerate Innovation in the Cell and Gene Therapy Industry - Business Wire

MaxHggblom Distinguished Professor and ChairDepartment of Biochemistry and MicrobiologySchool of Environmental and Biological SciencesRutgers-New BrunswickHonored for distinguished contributions to understanding both the fundamental and application components of microbialbiotransformationsof pollutants, especially chlorinated aromaticcompoundsand metalloids.

MaxHggblomis a renowned research scientist and educator with a large body of microbial ecology and environmental biotechnology research that has expanded our understanding of how the biodegradation of environmental pollutants, such as dioxins and PCBs,impact our planet.

His research interests revolve around thebioexploration, cultivation and characterization of novel microbes.His research on bacteria has provided a foundation for applications that address the pollution problems facing impacted industrialized and urbanized environments.

Hggblomslab is also actively studying microorganisms that degrade pharmaceutical and personal care products in aquatic environments.

Over the past decadesthediverse chemicalsin pharmaceutical and personal care productshave emerged as a major group of environmental contaminants in numerous watersheds around the world; therefore, it is important to understand how microbes can degrade them.There is much to explore and learn,Hggblomadded.

Hggblomswork also touches climate change, particularly the roles and responses of microbes in rapidly changing environments, such as the Arctic.In his lab at Rutgers, students have the unique opportunity to exploreareas of research such asthe biodegradation and detoxification of anthropogenic pollutant chemicals, including certainpesticides;respiration of rare metalloids; or life in the frozen tundra soils.

For several years,my lab has worked on studying the microbial ecology of Arctic tundra soils to understand how the changing conditions impact microbial activity and turnover of soil organic matter, and consequently enhanced greenhouse gas flux,Hggblomsaid. This is an important area of research as the threat of microbial contribution to positive feedback of greenhouse gas flux is substantial.

His lab recently received funding from the National Science Foundation to studyhowdiverse microbial communitiesare established insoils.Hggblomwill work with an international research team of scientists from the U.S., China, South Africa and Finland to study soils from the three differentregionsacross Arctic, Tibetan Plateau and Antarctic habitats to expand our understanding of how soil ecosystems respond in critical polar regions.

Emily EversonLayden

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Twelve Rutgers Professors Named Fellows of the American Association for the Advancement of Science - Rutgers Today

WASHINGTON, Jan. 25, 2022 (GLOBE NEWSWIRE) -- The GlobalHemophilia Market size is expected to reachUSD 14.2 Billionby 2028, exhibiting a CAGR of5.3%during the forecast period. Hemophilia is a bleeding condition, which leads to the prolonged bleeding after injury or a surgery due to a delay in the blood clotting, The Global Hemophilia Market is anticipated to grow at a substantial rate in coming years because of increasing cases of genetic abnormalities and prevalence of Hemophilia, states Vantage Market Research, in a report, titledHemophilia Market by Type (Hemophilia A, Hemophilia B, Hemophilia C, Others), by Treatment (On-demand, Prophylaxis), by Therapy (Replacement therapy, ITI therapy, Gene therapy), by Region (North America, Latin America, Europe, Asia Pacific) - Global Industry Assessment (2016 - 2021) & Forecast (2022 - 2028).The market size valued atUSD 12.1 Billionin 2021.

Click Here To Access The Free Sample Report @ https://www.vantagemarketresearch.com/hemophilia-market-1216/request-sample

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(Please note that the sample of this report has been updated to include the COVID-19 impact study prior to delivery.)

Market Overview:

Increasing Establishment of Hemophilia Treatment Centers to Drive the Market

The availability of limited treatment therapy options and growing burden on regulatory bodies towards the treatment is resulted to increase R&D efforts. The public as well as private healthcare bodies are heavily investing in development of specialized clinics that are established to meet the targeted needs of patients. In this regard, there is increasing number of Hemophilia treatment centres that also aims at proving treatment to underprivileged patients.It is recommended by authorities that people who are suffering from Hemophilia should visit a treatment center for optimal care and health education to stay healthy. The establishment of healthcare centres is anticipated to fuel theHemophilia Market growth.Additionally, efforts are been undertaken by regulatory bodies for spreading awareness regarding the disease and providing information about the effective treatment.

The COVID-19 outbreak has affected various industries worldwide. TheHemophilia Marketis no exception. Governments across the world took severe actions like border seals, lockdown, and implementing strict social distancing measures, in order to stop swift spread of COVID-19. These actions led to severe impact on the global economy impairing various industries. The impact of COVID-19 on the market demand is considered while estimating the current and forecast market size and growth trends of the market for all the regions and countries based on the following data-points:

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Benefits of Purchasing Hemophilia Market Reports:

The Report on Hemophilia Market Highlights:

Various Supportive Initiatives to Drive Market Growth in Asia Pacific

Asia Pacificis anticipated to witness fastest CAGR over the forecast period. The awareness campaigns and supportive initiatives taken by the government to commence the early screening of neonates, is about to boost the demand for diagnostic tools related to Hemophilia in the region. Some other factors such as advanced healthcare ecosystem and capacity of people to spend on such expensive medical services are defining the regional business growth. Additionally, easy medical reimbursement schemes are promoting the market growth.

List of Prominent Players in the Hemophilia Market:

Read Full Research Report @ https://www.vantagemarketresearch.com/industry-report/hemophilia-market-1216

Recent Developments:

December, 2021:The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for Hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal which could allow patients in Europe to access the therapy sooner,

December, 2021:Global biotherapeutics leader CSL Behring announced that the Committee for Medicinal Products for Human Use (CHMP), the chief scientific body of the European Medicines Agency (EMA) accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorisation Application (MAA). Etranacogene dezaparvovec (also known as EtranaDez), currently being studied in the Phase 3 HOPE-B clinical trial, is an investigational gene therapy for people living with hemophilia B, a life-threatening bleeding disorder.

December, 2021:Patients with severe Hemophilia can develop inhibitors against factor VIII or IX, preventing factor replacement therapy from working, said Dr. Guy Young (University of Southern California, CA, USA). A quarter of patients develop these inhibitors, leading to a worse prognosis. Novel agents are needed to protect these patients from bleeding events and arthropathy and improve their quality of life. In addition, the current IV therapies need to be administered multiple times per week, resulting in venous access issues and poor adherence.

December, 2020:Pfizer Inc. and Sangamo Therapeutics, Inc., a genomic medicines company, announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe Hemophilia A.

Browse the Report Hemophilia Market by Type (Hemophilia A, Hemophilia B, Hemophilia C, Others), by Treatment (On-demand, Prophylaxis), by Therapy (Replacement therapy, ITI therapy, Gene therapy), by Region (North America, Latin America, Europe, Asia Pacific) - Global Industry Assessment (2016 - 2021) & Forecast (2022 - 2028) @ https://www.vantagemarketresearch.com/blog/hemophilia-225379

This market titled Hemophilia Market will cover exclusive information in terms of Regional Analysis, Forecast, and Quantitative Data Units, Key Market Trends, and various others as mentioned below:

Treatment: - On-demand, Prophylaxis

Therapy: - Replacement therapy, ITI therapy, Gene therapy

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Global Hemophilia Market to Reach $14.2 Billion by 2028 - - GlobeNewswire

The switch design works solely for certain subsets of cancers, specifically non-small-cell lung cancer cells with an EGFR gene mutation, where drugs that target mutated proteins in the cancer cell are already on the market.

We are taking a careful approach to design and testing, Pritchard said. We will look specifically for failures within the switch, and analyze what we find, sort of like when civil engineers analyze a building or bridge failure after the fact. Failures help us understand where our ideas about cancer therapy are incomplete, and what we can do to fix them and increase our knowledge.

After initial tests on cancer cell lines, the researchers will test the dual-switch gene drives on human organoids, provided by the University of Massachusetts Medical School, which are patient-derived cancer cells that more closely mimic real tumors.

Co-investigator Shelly Peyton, Armstrong Professional Development Professor at University of Massachusetts Amherst and expert in tissue engineering, will lead the design of microenvironments to determine how the gene therapy functions under different conditions. Peytons team will study how certain switches or parameters fail, or why they function well in some environments but not others.

The research here is trying to take the challenge of cancer treatment and flip it on its head, said Scott Leighow, fifth-year doctoral student in biomedical engineering, who gathered the preliminary data that were key to securing the grant. If we can do that, we'll have a therapy that can handle resistant forms of cancer a lot better than what's currently in our arsenal.

If the study is successful, the researchers will test their treatments on animal models to show proof-of-concept, Leighow said. Far in the future, the technology has the potential to offer a cellular gene therapy that might assist cancer patients who are not candidates for surgery.

The grant is part of a new consortia created by the NCI to promote collaborative approaches to synthetic biology for cancer applications.

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$2.5M grant awarded to flip the switch on lung cancer drug resistance - Penn State News

Patient 1 with Sandhoff disease realized normalization of Hex A enzyme activity by Month 1, achieving 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 3

Patient 2 with Tay-Sachs disease achieved Hex A enzyme activity 5-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 1

First-ever data supporting bicistronic vector approach in humans, TSHA-101 is designed to deliver both HEXA and HEXB genes in the endogenous ratio

Conference call and live webcast today at8:00 AM Eastern Time

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported positive initial serum -hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, which represent two forms of GM2 gangliosidosis. Todays data are the first ever to support the bicistronic vector approach in humans delivering both HEXA and HEXB genes in the endogenous ratio.

TSHA-101 is the first bicistronic vector in clinical development, representing an important first for the field of gene therapy, noted RA Session II, President, Founder and CEO of Taysha. TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis. We expect to provide continued updates on the program, with additional clinical data anticipated by the end of 2022.

Based on natural history data, patients with asymptomatic GM2 gangliosidosis have Hex A enzyme levels that are at least 5% of normal activity. Key patient findings for Hex A enzyme activity following treatment with TSHA-101 include:

Patient 1 (Sandhoff disease)

Patient 2 (Tay-Sachs disease)

Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development at Taysha added, GM2 gangliosidosis is a progressive and life-limiting disease with no treatment options. Normalization of patient Hex A enzyme activity levels 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history supports TSHA-101s ability to potentially make a meaningful difference in the lives of patients with Sandhoff and Tay-Sachs diseases after a single intrathecal administration. We look forward to submitting a protocol amendment to expand patient enrollment in the ongoing Phase 1/2 trial and providing additional updates later this year.

Preliminary data suggest that TSHA-101 was well-tolerated with no significant drug-related events.

Patient 1 (Sandhoff) demonstrated signs of clinical improvement at Month 3 and was deemed stable to travel home. Upon returning home, Patient 1 (Sandhoff), who was unvaccinated, was exposed to a family member symptomatic for an upper respiratory infection, possibly Covid-19, and was hospitalized with pneumonia before contracting a secondary hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection. On January 14, 2022, the patient succumbed to pneumonia and pleural effusion with a concomitant hospital-acquired MRSA infection. The principal investigator has made the initial assessment that the death was unrelated to study drug. Final determination from the independent data safety monitoring board (DSMB) is anticipated in the near term.

TSHA-101 is an investigational gene therapy that delivers both the HEXA and HEXB genes that comprise the -hexosaminidase A enzyme. The two genes are driven by a single promoter within an AAV9 capsid ensuring that the two sub-units of Hex A are produced in the endogenous ratio within each cell, which is important to ensure efficient production of the enzyme. TSHA-101 is the first and only bicistronic vector currently in clinical development for GM2 gangliosidosis and has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA and Orphan Drug Designation from the European Commission. TSHA-101 is administered intrathecally and is currently being evaluated in a single arm, open-label Phase 1/2 clinical trial for the treatment of infants with GM2 gangliosidosis sponsored by Queens University. Additional clinical safety and efficacy data are expected by the end of 2022.

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

Conference Call and Webcast Information

Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to provide an update on the GM2 gangliosidosis program. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13726741. The live webcast and replay may be accessed by visiting Tayshas website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.

About Taysha Gene Therapies

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

Forward-Looking Statements

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20220127005353/en/

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Taysha Gene Therapies Announces Positive Initial Biomarker Data For TSHA-101 - BioSpace

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that Leber Hereditary Optical Neuropathy (LHON) subjects treated with LUMEVOQ continued to experience significantly improved vision four years after a single injection of the gene therapy. The findings come from RESTORE (CLIN06), the long-term follow-up study to which participants in the RESCUE1 and REVERSE2 Phase III pivotal trials were invited.

When RESTORE subjects enrolled in the study, 2 years after the one-time injection, they had already experienced clinically meaningful improvements relative to the lowest point (the nadir) of their best-corrected visual acuity (BCVA): +18.8 ETDRS letters equivalent* in their LUMEVOQ-treated eyes and +17.3 letters equivalent in their sham-treated eyes. Four years after treatment, the bilateral improvement from nadir was sustained, with LUMEVOQ-treated eyes achieving a mean improvement against nadir of +22.5 letters equivalent and sham-treated eyes demonstrating a mean improvement of +20.5 letters equivalent.

The impact of such results on patients is demonstrated by increases in the self-reported quality of life (QoL) scores at Year 4 vs. baseline. Mean overall QoL increased by a clinically meaningful magnitude relative to baseline, driven by clinically meaningful increases in the sub-scores corresponding to mental health and the ability to carry out activities autonomously (e.g., role difficulties, dependency, near and far activities, general vision).

The 4-year RESTORE long-term extension study provides patients with Leber Hereditary Optic Neuropathy and their families as well as the neuro-ophthalmology community with highly informative data about both the efficacy and safety of intravitreal LUMEVOQ therapy, commented Dr. Robert Sergott, Director, Neuro-Ophthalmology Service, Wills Eye Hospital, and Founding Director and CEO, William H. Annesley EyeBrain Center, Thomas Jefferson University, Philadelphia, PA, USA. Compared to the natural history of LHON, the 4-year data extend and validate the 3-year observations by confirming that objective visual acuity improvement is sustained and is associated with improved functional visual quality of life without any long-term safety concern.

The RESTORE findings underline the therapeutic value of GenSights pioneering one-time treatment for LHON: durable and clinically significant improvement in visual function coupled with impressive safety, noted Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. The body of evidence we have now accumulated is without doubt good news for patients needing an urgent solution for their brutal blinding condition, and consequently we are continuing to work vigorously with the relevant authorities to bring regulatory review process to a successful conclusion.

RESTORE is one of the largest long-term follow-up studies for a rare disease treatment, with 62 subjects accepting the invitation to enroll. All subjects, who were affected by LHON caused by a mutated ND4 mitochondrial gene, were treated with an intravitreal injection of LUMEVOQ in one eye and with sham injection in the other.

Table 1. BCVA Mean Improvement Vs. Nadir* In LUMEVOQ Long-Term Follow-Up (RESTORE)

2 Years Post-Injection

3 Years Post-Injection3

4 Years Post-Injection

LogMAR(Std Error)

LettersEquivalent**

LogMAR(Std Error)

LettersEquivalent**

LogMAR(Std Error)

LettersEquivalent**

LUMEVOQ-treated eyes

-0.375(0.306)

+18.8

-0.410(0.365)

+20.5

-0.453(0.440)

+22.5

Sham-treated eyes

-0.346(0.291)

+17.3

-0.387(0.369)

+19.4

-0.406(0.361)

+20.5

Note: The RESTORE sample consists of the RESCUE and REVERSE participants who accepted to be followed in the long-term follow-up study. Year 4 values were the LogMAR readings nearest to 1461 days post treatment recorded between 1461 +/- 273 days post- treatment. Missing values were imputed using the Last Observation Carried Forward (LOCF) method. *Nadir = worst best-corrected visual acuity recorded from baseline to Year 4. ** Assessments of best-corrected visual acuity (BCVA) were recorded in LogMAR. The change from nadir in LogMAR was converted to letters equivalent improvement by multiplying the LogMAR by -50 (ref. J.T. Holladay, J Refrac Surgery, 1997;13, 388-391).

Responder analyses at Year 4 indicate that improved BCVA was a benefit for a substantial proportion of the study participants. 71.0% of RESTORE subjects achieved Clinically Relevant Recovery (CRR)4 against nadir four years after treatment, and 80.7% of them had on-chart vision (BCVA 1.6 LogMAR) in one or both eyes.

Viewed against the trend in vision typically seen in untreated patients5, the findings represent a significant departure from the natural progression of LHON.

Safety findings at 4 years post-injection were consistent with previous readouts, which concluded that LUMEVOQ is well-tolerated: no serious adverse events were recorded among LUMEVOQ-treated eyes, and no discontinuations occurred due to ocular events. There were no systemic serious adverse events or discontinuations related to study treatment or study procedure.

The review of the European Marketing Authorisation Application for LUMEVOQ is ongoing, with the decision from the CHMP expected in Q4 2022.

References and notes:

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of subjects have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,200-1,500 new subjects who lose their sight every year in the United States and the European Union.

About LUMEVOQ (GS010; lenadogene nolparvovec)

LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About RESCUE, REVERSE, and RESTORE

RESCUE and REVERSE were two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint measured the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, were used for statistical purposes. Both trials were adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between drug-treated and sham-treated eyes, adjusted to baseline.

The secondary endpoints involved the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis was performed, including the proportion of patients who maintained vision (< ETDRS 15L loss), the proportion of patients who gained 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics included automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality-of-life scales, biodissemination and the time course of immune response. Readouts for these endpoints were at 48, 72 and 96 weeks after injection.

The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were invited to participate in a long-term follow-up study, RESTORE, for three additional years.

The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment. The secondary objective is to assess the long-term treatment efficacy of the therapy and the quality of life (QoL) in subjects up to 5 years post-treatment. The first subject was enrolled on January 9, 2018. 61 subjects have enrolled.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767RESTORE: NCT03406104

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GenSight Biologics Reports Clinically Meaningful Vision Improvement is Maintained 4 Years After One-time Treatment with LUMEVOQ Gene Therapy -...

PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today announced that its Investigational New Drug (IND) application for its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN), was cleared by the U.S. Food and Drug Administration (FDA).

SBT101 is the first AAV-based gene therapy in development specifically designed for people living with AMN, an adult-onset degenerative spinal cord disease caused by mutations in the ABCD1 gene. SwanBio plans to initiate a randomized, placebo-controlled Phase 1/2 clinical trial designed to assess the safety and explore the efficacy of SBT101 in patients with AMN in the second half of 2022.

Todays IND clearance is a formative milestone for SwanBio, enabling us to evolve from a preclinical company to a truly integrated research and development organization, underscoring the expertise of our team and potential of our technology platform, said Tom Anderson, chief executive officer and director of SwanBio Therapeutics. SBT101 has the potential to become the first disease-modifying treatment for patients with AMN, a devastating and progressive disease with no approved treatments. We look forward to initiating clinical development of SBT101 later this year, bringing us closer to our ultimate goal of delivering life-changing treatments to patients.

The clinical program for SBT101 builds on SwanBios unique understanding of AMN, including new insights being gathered in an ongoing natural history study. SwanBio is deeply committed to the AMN community and has worked closely with patients, family members, and expert physicians including SwanBio Co-Founder Dr. Florian Eichler to ensure that its clinical programs are designed to meet their needs. SwanBio is supported by long-term investment partners Syncona Ltd. and Mass General Brigham Ventures, which both have proven track records in gene therapy, particularly in AAV-focused therapies.

About SBT101SBT101 is the first AAV-based gene therapy in development designed to compensate for the disease-causing ABCD1 mutation, to increase ABCD1 expression, and reduce very long chain fatty acid (VLCFA) levels specifically for people living with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of AMN disease markers in mouse models and was shown to be well-tolerated in non-human primates at six months post-treatment.

About AdrenomyeloneuropathyAdrenomyeloneuropathy (AMN) is the adult-onset degenerative spinal cord disease that affects people living with adrenoleukodystrophy (ALD), a category of rare, genetic, and metabolic conditions. AMN is characterized by progressive loss of mobility, incontinence, and debilitating pain. It affects adults with mutations in the ABCD1 gene, which encodes a protein essential to the processing and breakdown of very long chain fatty acids (VLCFA). Without a functioning version of this protein there is an accumulation of VLCFA to toxic levels that leads to progressive dysfunction of the central nervous system. Between 8,000-10,000 men in the U.S. and E.U. are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom control.

About SwanBio TherapeuticsSwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com.

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SwanBio Therapeutics Announces FDA Investigational New Drug Clearance for First AAV-Based Gene Therapy for the Treatment of Adrenomyeloneuropathy -...

DUBLIN--(BUSINESS WIRE)--The "Protein Expression Global Market Report 2021: COVID-19 Growth and Change to 2030" report has been added to ResearchAndMarkets.com's offering.

The global protein expression market is expected to grow from $2.01 billion in 2020 to $2.13 billion in 2021 at a compound annual growth rate (CAGR) of 6%. The market is expected to reach $3.03 billion in 2025 at a CAGR of 9.2%.

Major players in the protein expression market are Agilent Technologies, Bio-Rad Laboratories, Thermo Fisher Scientific Inc., New England Biolabs and Promega Corporation.

The protein expression market consists of sales of protein expression vectors, competent cells, reagents, equipment and related services. Protein expression is a process in which proteins are synthesized, modified, regulated and controlled in living organisms according to the host cell. Protein expression included yeast expression, insect expression, and bacterial expression, algal expression and mammalian cell expression.

The protein expression market covered in this report is segmented by protein expression into yeast expression, mammalian expression, algae expression, insect expression, bacterial expression, cell-free expression. It is also segmented by end use into pharmaceutical and biotechnological companies, academic research, contract research organizations; by product into reagents, competent cells, expression vectors, services, instruments and by application into therapeutic, industrial, research.

Government regulations related to protein therapeutics and production of biologics may hinder the protein expression market growth. Government regulations on biologics to undergo rigorous preclinical and clinical trials prior to regulatory approval, and time consuming process for approval of biologics with regards to health and the safety of any individual are restraining the market growth.

Marketing and distribution of biologics including insulin, hormones, therapeutic antibodies, and vaccines depends upon the successful completion of clinical trials, which is a long, expensive, and uncertain process. According to FDA, for an approval of new biologic, Under the regulations (21 CFR 314.81(b)(2)(vii) and 601.70, a clinical trial approval usually takes 10- 12 months where firms are required to submit a report annually on the status of clinical safety, clinical efficiency, clinical pharmacology, and nonclinical toxicology study.

Companies in the industry are increasingly adopting Microfluidics technology to enhance protein expression tests in order to reduce the time, cost, labor, and increase the accuracy and performance. The microfluidics technology effectively analyzes biological samples than the traditional (macroscale) instruments.

Microfluidics technology is used to measure the expression of proteins on cells and optimizes the output to generate results regarding protein expression. Therapeutics-on-a-chip (TOC) uses microfluidic platform and is able to synthesize proteins in a point of care setting to reduce cost associated with storage and transportation of therapeutic proteins.

For instance, companies such as MissionBio, NanoCellect Biomedical, RainDance Technologies and Sphere fluidics have implemented this technology in protein expression test.

Increase in demand for biologics to counter various genetic disorders and chronic diseases is one the major factors driving the research and sales of protein expression market. Biologics is a medicine produced from living organisms or contains components of living organisms such as protein, tissue, genes, allergens, cells, blood components, blood, and vaccines.

The increasing use of biologics (therapeutic protein and others) to cure chronic diseases such as cancer, cardiovascular conditions and genetic disorders, is increasing the demand for protein expression devices and equipment. According to the World Health Organization, chronic disease prevalence is expected to rise by 57% globally, by the year 2020.

Hence the rising demand for biologics is driving the protein expression market. For instance, according to an article published by Chemistry World, analysts expect the biologics market to hold a market share of more than a quarter of the entire pharmaceutical market by 2020. The global biologics market is expected to grow at 9.9% during 2018-2024.

The Protein Expression market in the U.S. is governed by Food and Drug Administration (FDA) that lays down a series of guidelines for the manufacturers and retailers of this industry. Within FDA, Center for Drug Evaluation and research (CDER) regulates biological products under FDA 101 which includes gene therapy products and vaccines. These regulations ensure quality, safety and efficacy of biological therapeutics products, and speed up innovations that make these products safer, and effective.

The US's FDA announced a fast-track initiative to review its drugs and biologics policy to speed the availability of therapies to patients with serious conditions, orphan drugs for rare disease, while preserving the safety and efficacy standards. FDA also removed a rule (Section 610.21 of the FDA code) which specified minimal potency limits for certain antibodies and antigens.

The European Medicines Agency has also introduced policies which include a provision to waive the scientific advice fee, which encourage more academic groups and small companies to propose candidates for biologics.

Key Topics Covered:

1. Executive Summary

2. Protein Expression Market Characteristics

3. Protein Expression Market Trends and Strategies

4. Impact Of COVID-19 On Protein Expression

5. Protein Expression Market Size and Growth

5.1. Global Protein Expression Historic Market, 2015-2020, $ Billion

5.1.1. Drivers Of the Market

5.1.2. Restraints On the Market

5.2. Global Protein Expression Forecast Market, 2020-2025F, 2030F, $ Billion

5.2.1. Drivers Of the Market

5.2.2. Restraints On the Market

6. Protein Expression Market Segmentation

6.1. Global Protein Expression Market, Segmentation by Protein Expression, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.2. Global Protein Expression Market, Segmentation by End Use, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.3. Global Protein Expression Market, Segmentation by Product, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.4. Global Protein Expression Market, Segmentation by Application, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7. Protein Expression Market Regional and Country Analysis

7.1. Global Protein Expression Market, Split by Region, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7.2. Global Protein Expression Market, Split by Country, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/iix75m

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Global Protein Expression Market Research Report 2021 Featuring Major Players - Agilent Technologies, Bio-Rad Laboratories, Thermo Fisher Scientific,...

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, announced a research and development collaboration with Haihe Laboratory of Cell Ecosystem to develop hematopoietic stem cell regenerative therapies and platform technology by combining resources and expertise from both sides.

The Haihe Laboratory of Cell Ecosystem, run by the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, is focused on conducting fundamental research, innovation, and translation in the cell ecosystem.

Under the agreement, both parties will jointly develop hematopoietic stem cell regenerative therapies, including the development of innovative genetically-modified hematopoietic stem cell therapies and the exploration of novel biomarkers to optimize quality control for stem cell production.

With top-notch resources and industry-university-research cooperation, well facilitate the development of cell-based medicine and therapies, said Professor Tao Cheng, Deputy Director of Haihe Laboratory of Cell Ecosystem and President of the Institute of Hematology and Blood Diseases Hospital at the Chinese Academy of Medical Sciences and Peking Union Medical College, a leading hematology researcher who has made a series of discoveries relating to the regulatory and regenerative mechanisms of hematopoietic stem cells. Hematopoietic stem cells (HSCs) have the potential for long-term self-renewal and can differentiate into various types of mature blood cells. These stem cells can be harnessed to provide treatment for a broad range of diseases such as hematological tumors, autoimmune diseases, and hereditary blood disorders. We believe that this collaboration with EdiGene will accelerate the innovation and translation in the field of HSCs, thus enabling healthier patients with new therapies."

Professor Cheng was awarded the second prize of the National Natural Science Award 2020 as the first author of work on basic and translational research that advanced the development of adult hematopoietic stem cells for therapeutic applications.

EdiGene is scaling up clinical translation and development of the first gene-editing hematopoietic stem cell therapy in China following the 2021 approval by the China National Medical Products Administration its IND for its investigational therapy ET-01. Our team has extensive experience in the development and translation of cutting-edge technologies including hematopoietic stem cell and gene editing, said Dong Wei, Ph.D., CEO of EdiGene. "This collaboration with Haihe Laboratory of Cell Ecosystem will further our exploration in the field of hematopoietic stem cells. The partnership with this leading academic institute and our translational know-how enable us to move forward in bringing more innovative treatment options to patients in China and around the world.

In 2021, EdiGene initiated a Phase I multicenter clinical trial of ET-01, its gene-editing hematopoietic stem cell therapy for transfusion-dependent -thalassemia. EdiGene has enrolled the first patient at the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College. Currently, the clinical trial is being conducted in Tianjin and Guangdong-Hong Kong-Macao Greater Bay Area (Greater Bay Area). EdiGene also presented its latest research on new surface markers and migration of hematopoietic stem cells at the 63rd Annual Meeting of the American Society of Hematology (ASH) in 2021.

About Haihe Laboratory of Cell Ecosystem

The Haihe Laboratory of Cell Ecosystem ("the Laboratory"), run by the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, is one of the five registered Haihe Laboratories approved by Tianjin Municipal People's Government. With the goal of promoting population health with cell ecosystem, the Laboratory adheres to developing technological frontier, enhancing peoples health, and promoting research, innovation, and development of cell ecosystem in five key areas: cellular ecosystem, cellular ecology and immunity, cellular ecological imbalance and major diseases, cellular ecological reconstruction and frontier technology of cellular ecological research.

About Institute of Hematology and Blood Diseases Hospital (IH), Chinese Academy of Medical Sciences & Peking Union Medical College (CAMS/PUMC)

Founded in 1957, IH is a tertiary specialty hospital under the National Health Commission of China and is the supporting unit of the National Clinical Research Center of Hematologic Diseases and the State Key Laboratory of Experimental Hematology. It is also the main founding unit of Tianjin Base, the core base of the Chinese medical science and technology innovation system with the goal of becoming "the innovation hub of hematology in China." IH mainly engages in basic research, applied research, clinical diagnosis and treatment of hematological diseases, standard-setting, new technology research, new drug evaluation, and translation in hematology and related fields. IH is leading in the diagnosis and treatment of hematological diseases in China and a global scale and has made original achievements. Since 2010, IH has been awarded first place in the Hospital Specialty Reputation Ranking (Hematology) for 12 consecutive years. It has won first place in the Hematology Specialty Ranking for ten consecutive years since 2010 and ranked the first in hematology by the Scientific and Technological Evaluation Metrics (STEM) for Chinese hospitals for eight consecutive years since 2014.

About EdiGene, Inc

EdiGene is a global, clinical-stage biotechnology company focused on translating gene editing technologies into transformative therapies for patients with serious genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China and Cambridge, Massachusetts, USA. More information can be found at http://www.EdiGene.com.

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EdiGene Enters Strategic R&D Collaboration with Haihe Laboratory of Cell Ecosystem to Develop Hematopoietic Stem Cell Regenerative Therapies and...