SAN FRANCISCO and KYOTO, Japan, April 18, 2024 /PRNewswire/ -- Shinobi Therapeutics, a biotechnology company developing a new class of immune evasive iPS cell therapies, today announced a partnership with Panasonic Holdings Corp and Kyoto University's Center for iPS Cell Research and Application (CiRA). Through this strategic collaboration, the organizations aim to engineer a novel manufacturing platform to produce iPS-T cell therapies more efficiently and at lower cost than is possible with currently available technology.

"To make promising iPS-T cell therapies accessible to the broader population, Panasonic is committed to developing a manufacturing platform that will produce safe cells for therapies at the lowest possible cost," said Yuki Kusumi, Representative Director and President of Panasonic Holdings Corporation. "Reducing the production time and cost of cell therapies must be done in a manner that does not compromise safety or efficacy, and we are thrilled to see the Japanese biotech and engineering communities coming together to make that happen."

Cell therapies have shown remarkable promise in treating blood cancers and other intractable diseases, but manufacturing costs render these therapies inaccessible to many patients around the world. Shinobi's iPS-T cell technology, built upon a decade's worth of iPSC research pioneered at CiRA by Shinobi co-founder Shin Kaneko using iPSCs originally created by Nobel laureate Shinya Yamanaka, will be used to support the creation of a closed-system manufacturing device created by Panasonic, opening up an entirely new paradigm for cell therapy production.

"Advancements in iPS cell production and Shinobi's genetic modification of iPSCs for immune evasion have made regenerative T cell therapy increasingly feasible," said Shin Kaneko, Co-Founder at Shinobi. "The automated cultivation device developed in this joint research will significantly accelerate this, contributing to the realization of a world where state-of-the-art regenerative killer T cell therapy can be provided for every patient."

The newly announced partnership will leverage Panasonic's manufacturing expertise to develop a new method of producing iPS-T cell therapies in a closed-system process. The first phase of the partnership will be completed in April 2025, when the companies expect to release the initial prototype.

"While cell therapies have the potential to transform patient care across a wide range of intractable diseases, we have a long road ahead to overcome the challenges in manufacturability and accessibility," said Dan Kemp, CEO at Shinobi. "We are fortunate to be working with the most renowned partners across the academic and industry landscape as we endeavor to put cell therapies within reach for all patients who need them."

About Shinobi TherapeuticsShinobi Therapeutics is a biotechnology company developing a new class of off-the-shelf immune evasive iPSC-derived cell therapies. Based on the research of scientific co-founders Shin Kaneko, M.D., Ph.D., at Kyoto University and Tobias Deuse, M.D., at University of California, San Francisco, Shinobi has created a new allogeneic CD8a iPS-T cell platform that demonstrates comprehensive immune evasion from all arms of the immune system. For more information, please visit http://www.shinobitx.com.

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Visiongain has published a new report entitled Cell Therapy Technologies Market Report 2024-2034: Forecasts by Product (Sera, Media, Reagent, Cell Engineering Product, Cell Culture Vessels, Equipment, Systems and Software, Others), by Cell Type (T-Cells, Stem Cells, Other Cells), by Process (Cell Processing, Cell Preservation, Distribution, and Handling, Process Monitoring and Quality Control), by End-users (Biopharmaceutical & Biotechnology Companies, CROs, Research Institutes and Cell Banks) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies AND COVID-19 Impact and Recovery Pattern Analysis.

The cell therapy technologies market is estimated at US$7,041.3 million in 2024 and is projected to grow at a CAGR of 10.7% during the forecast period 2024-2034.

The rise in chronic diseases like cancer, cardiovascular issues, and autoimmune disorders has created a pressing need for effective treatments. Supportive regulatory frameworks have encouraged the development & commercialization of cell therapies. Additionally, increased awareness and acceptance of these therapies among healthcare professionals and patients are driving demand further. Advancements in cell therapies offer lucrative opportunities for market players. Companies are focusing on enhancing the efficacy & safety of these therapies to provide better disease management outcomes for patients.

Download Exclusive Sample of Report https://www.visiongain.com/report/cell-therapy-technologies-market-2024/#download_sampe_div

How has COVID-19 had a Significant Impact on the Cell Therapy Technologies Market?

The COVID-19 pandemic has affected the market for cell therapy technologies market significantly. The pandemic initially caused significant disruptions to the manufacturing and supply chains of numerous industries, including the biotechnology sector. As a result, there were delays in cell therapy clinical trials, regulatory approvals, and commercialization initiatives. Furthermore, the shift in healthcare resources towards the management of the pandemic led to a reduction in funding and attention for medical research unrelated to COVID-19, such as the development of cell therapies.

However, the pandemic also made clear how crucial cutting-edge medical innovations like cell therapies are to solving the world's health crises. Consequently, there has been a surge in interest and funding for the study and advancement of cell therapy as a means of treating not only COVID-19 but also other chronic illnesses and infectious diseases. Additionally, the pandemic's adoption of telemedicine and remote monitoring has sped up the acceptance of decentralised clinical trials, which could advance cell therapy technologies by lowering trial costs and increasing patient access. The COVID-19 pandemic has, in the long run, created opportunities for innovation, collaboration, and growth, even though it initially presented challenges to the cell therapy technology market. The cell therapy sector is positioned to have a significant impact on how healthcare and illness management are provided in the future, even as the globe struggles to cope with the pandemic's aftermath.

How will this Report Benefit you?

Visiongains 305-page report provides 109 tables and 173 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the cell therapy technologies market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Cell Therapy Technologies. Get financial analysis of the overall market and different segments including product, cell type, process, end-users and capture higher market share. We believe that there are strong opportunities in this fast-growing cell therapy technologies market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

Rise in Prevalence of Chronic & Degenerative Diseases

The healthcare sector faces numerous challenges from chronic illnesses like cancer, heart disease, neurological ailments, and autoimmune disorders. The management or cure of many disorders is frequently only partially successful with conventional therapeutic options.

With the ability to replace, regenerate, or repair damaged tissues or organs, cell therapy presents a viable substitute. Much emphasis has been paid to cell treatments' capacity to treat diseases at their root and encourage long-term healing.

Notable advancements in cell treatment technologies have been made over time to address degenerative and chronic illnesses. For example, developments in stem cell research have made it possible to identify and isolate several types of stem cells, each with a unique therapeutic potential. In order to create novel cell-based therapeutics, researchers are looking into the utilisation of hematopoietic stem cells, induced pluripotent stem cells, and mesenchymal stem cells.

Rigorous Efforts by Companies Towards Development of Proprietary & Supportive Technologies Anticipated to Boost Industry Growth

In regenerative medicine, cell therapy, which employs living cells to treat or cure diseases, has emerged as a promising area of study. Nevertheless, the efficacy of cell therapies is contingent upon the accessibility of cutting-edge technologies that facilitate the production, characterization, and transportation of cells.

Significant investments are being made by companies in the cell therapy industry in research and development of proprietary technologies that improve the safety, effectiveness, and scalability of cell therapies. The technologies in question comprise an extensive array of domains, such as tools for cell characterization, cell isolation and expansion techniques, and cryopreservation methods.

The advancement of cell culture systems is a primary area of emphasis. Organisations are currently engaged in the development and refinement of culture media, growth factors, and bioreactors that establish an optimal milieu for cellular proliferation while preserving the viability and functionality of the cells. The primary objectives of these proprietary culture systems are to increase cell yields, decrease production expenses, and facilitate the scalable production of cell therapies.

Considerable interest is being devoted to supportive technologies that affect cell isolation and purification. Innovative methods are being developed by businesses to isolate particular cell populations from complex mixtures, thereby ensuring the quality and purity of cells used in therapies. These technologies reduce the possibility of contamination or undesired cell populations while facilitating the efficient isolation of therapeutic cell types.

Cryopreservation technologies are indispensable for the transportation and long-term storage of cells. Organisations are presently preoccupied with the advancement of cryopreservation techniques that preserve the genetic stability, viability, and functionality of cells throughout the freezing and thawing processes.

These developments guarantee the presence of viable cells during therapy administration, notwithstanding the logistical obstacles that may arise from cell storage and transportation.

The development of proprietary and supportive technologies will therefore likely contribute to the expansion of the global market for cell therapy technologies.

Get Detailed ToC https://www.visiongain.com/report/cell-therapy-technologies-market-2024/

Where are the Market Opportunities?

Emerging nations present a substantial potential for the progression and integration of cell therapy technologies. These countries are currently experiencing notable advancements in their healthcare systems, as significant financial resources are being allocated to accommodate the growth of their populations. Concurrent with this growth, developing nations are confronted with an increasing prevalence of chronic and non-communicable ailments as a result of urbanisation, alterations in lifestyles, and the ageing of their populations. Cell therapy technologies are of particular relevance in these regions due to the innovative solutions they offer to address these urgent medical needs.

Moreover, in comparison to developed countries, the execution of clinical trials in emerging economies frequently demonstrates greater cost-effectiveness, predominantly attributable to reduced labour and operational expenditures. The financial benefits associated with this incentive motivate pharmaceutical companies and research institutions to investigate and advance cell therapies in these areas. Furthermore, numerous developing nations provide favourable regulatory structures and incentives in order to promote the progress and acceptance of cutting-edge medical technologies, such as cell therapies. The convergence of these elements renders developing nations an optimal setting for the proliferation and integration of cell therapy technologies, holding the potential to yield substantial advantages for healthcare providers and patients.

Competitive Landscape

The major players operating in the cell therapy technologies market are Thermo Fisher Scientific Inc., Novartis AG, Gilead Sciences, Inc., Merck KGaA, Danaher Corporation, Bristol-Myers Squibb Company, Sartorius AG, FUJIFILM Diosynth Biotechnologies, Lonza, GE Healthcare, Terumo BCT, Avantor, Inc., Bio-Techne Corporation, and Corning Incorporated among others. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.

Recent Developments

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Visiongain is one of the fastest-growing and most innovative independent market intelligence providers around, the company publishes hundreds of market research reports which it adds to its extensive portfolio each year. These reports offer in-depth analysis across 18 industries worldwide. The reports, which cover 10-year forecasts, are hundreds of pages long, with in-depth market analysis and valuable competitive intelligence data. Visiongain works across a range of vertical markets with a lot of synergies. These markets include automotive, aviation, chemicals, cyber, defence, energy, food & drink, materials, packaging, pharmaceutical and utilities sectors. Our customised and syndicatedmarket research reportsoffer a bespoke piece of market intelligence customised to your very own business needs.

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Cell Therapy Technologies market is projected to grow at a CAGR of 10.7% by 2034: Visiongain - GlobeNewswire

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NINA MOINI: The Minneapolis Saint Paul International Film Festival is currently under way. And tonight, a documentary will premiere called "Sequencing Hope." The film is directed by Lindsey Seavert and Maribeth Romslo. It follows an Alabama family who came to Minnesota to get their young daughter life-saving gene therapy for a rare disease. Let's listen to a clip from the trailer.

CELIA GRACE HAMLETT: Can you hold my hand?

[CHUCKLES]

They give me the medicine.

SUBJECT 1: There is lots of other gene therapy research on the horizon.

SUBJECT 2: That's got far-reaching consequences to move medicine forward.

SUBJECT 3: In my heart, I feel that the good Lord has something in store for Celia Grace.

SUBJECT 4: We just pray, Lord, for a miracle. We pray for a healing for Celia Grace.

SUBJECT 5: You know, when it comes to your kids, you're going to do whatever it takes to protect them.

SUBJECT 6: If it saves one child, then I feel like we have accomplished something.

NINA MOINI: Celia Grace Hamlett was four-years-old when she came to M Health Fairview Masonic Children's Hospital in 2021 and became the first person in the US to undergo the experimental gene therapy. Her family's in town for the film's premiere tonight and Celia Grace's dad, Gary, joins us now along with their doctor, Doctor Paul Orchard. Thank you both for being here.

GARY HAMLETT: Thank you.

PAUL ORCHARD: Thank you, Nina.

NINA MOINI: Yeah, and Gary, let me start with you if I might. Tell us about your daughter, Celia Grace. She's seven-years-old now and I understand she was diagnosed with this rare and often fatal genetic disorder, MLD, when she was diagnosed at three-years-old. What were her options at that point?

GARY HAMLETT: Well, at that point had one or had two options. One was bone marrow transplant or the other was gene therapy that was only being done in Milan, Italy.

NINA MOINI: Wow. Doctor Orchard, can you tell us all what MLD is?

PAUL ORCHARD: Certainly. Appreciate the opportunity to speak with you today. So metachromatic leukodystrophy is a rare inherited disorder. It's what we call a lysosomal disease. The lysosome is an organelle within cells that help break down materials that the cell is attempting to get rid of.

And there's a number of enzymes that are present in the lysosome that help accomplish that. Arylsulfatase A. is one of those. And in this circumstance, if you are unlucky enough to receive a mutation within the arylsulfatase gene from both mom and from dad, then you're affected with the disease.

But both parents who have one normal copy of the gene are absolutely fine. There's nothing to suggest that they have any sort of problem, but again, if you receive an abnormal copy from both parents then you see the disease. And in this situation, it's primarily a neurologic disorder. It occurs in kids as young as one or so in terms of manifestations of the disease, but it's progressive and lethal if there's no therapy.

NINA MOINI: Wow, that's just so much to take in, Gary. And you know, you mentioned having to maybe think about treatment over in Milan. How did you hear about the treatment for MLD that was right here in Minnesota?

GARY HAMLETT: Our doctor neurologist in Alabama, Doctor Matt, is the one that contacted us and said, what would y'all think if I told y'all y'all's daughter was going to make history books? At that point we said, what do you mean? And she said, well, your daughter may be the first child in the United States to receive gene therapy for MLD and it will be done at the Masonic Children's Hospital in Minneapolis under the care of Doctor Paul Orchard.

NINA MOINI: And then what did you think? I mean, were you going to have to pay for that?

GARY HAMLETT: Yes. At that point we didn't really care what it cost us being able to save our daughter's life. So our community started doing fundraisers to try to raise money to pay for this.

NINA MOINI: Wow, yeah. Doctor Orchard, can you explain how the gene therapy works and is it accessible to most people or is it just too costly?

PAUL ORCHARD: Well, the gene therapy clinical trials occurred in Europe, as Gary was alluding to, and the data was sufficiently positive that it was approved as therapy in the EU, essentially. So it's been licensed therapy there for several years, but none of the clinical trials have been done here in the US.

And because of the promise of this new therapy, we were gearing up to being able to offer this regardless, but there was the opportunity in this situation from Celia Grace's diagnosis to be able to intervene. So it's just become licensed therapy in the last month or so, as March 18th.

But prior to that and for Celia Grace, we had to petition the FDA to allow us to use it because it's still considered experimental therapy, and get all the approvals from all the various regulatory groups to be able to do that. So it took some time, but it opened the doors. And now we've treated a total of five patients with compassionate use therapy.

NINA MOINI: All right. Is it still pretty pricey, though? I understand it's among some of the priciest treatments.

PAUL ORCHARD: Yes, it is very expensive. So for the compassionate use treatment as an experimental therapy, the company actually donated the cell product, but it's millions of dollars now as licensed therapy.

NINA MOINI: Yeah. So still working to make it more accessible. Gary, you said something that really struck me in the trailer for the film. You said that you take care of people for a living. I understand you work in law enforcement, but you couldn't fix this for your daughter. And it seems like this film is really an exploration of your family's journey. Tell me how did that feel to feel sort of helpless in the moment, but then to see her go through this journey and be, I mean, cured?

GARY HAMLETT: We just felt very helpless, not knowing the outcome of it, how sick Grace was. Just thinking that we were going to lose our daughter. Possibly by the age of five-years-old.

NINA MOINI: Yeah.

GARY HAMLETT: And seeing her now as a normal seven-year-old, running, playing, is going to graduate in kindergarten, and it's just an amazing feeling.

NINA MOINI: Yeah, I'm sure. And so how is she doing? Tell us a little bit just about how she's getting around fine, and she's feeling well.

GARY HAMLETT: Oh, she is rambunctious, non-stop playing, running, doing her schoolwork. She is just like a typical seven-year-old little girl.

NINA MOINI: Yeah, and I understand some more patients are going to be undergoing that same treatment as Celia Grace, which is great news, Doctor Orchard.

PAUL ORCHARD: Yes, I hope it's going to be widely available. As you mentioned, the cost is going to be significant and attempting to determine how we're going to do this. The vast majority of these patients that we treat are obviously not from Minnesota.

And so being able to get insurance that's going to work across state lines and going to be sufficient for this is going to be a challenge. But that's one of the things that we're currently working on.

NINA MOINI: OK, and Gary, I'll leave the last question for you here. What do you hope people will take away from watching your family's story in this documentary?

GARY HAMLETT: The struggles of not knowing the outcome of your child. The struggles of possibly knowing that you will only have a few years with your child. And then knowing that there are people out there willing to help and willing to do anything possible to save your daughter or your son. I just can never repay everybody that along this journey for what they have done for my child.

NINA MOINI: Yeah, and it really sounds like it's some of the best parts of humanity and also some of the hardest struggles that anyone will go through. Thank you so much for sharing that journey and for being here, Gary. And to you as well, Doctor Orchard, thank you, and congratulations on this film reaching an audience today.

PAUL ORCHARD: Thank you.

GARY HAMLETT: Thank you so much.

NINA MOINI: Gary Hamlett is the father of Celia Grace Hamlett and Doctor Orchard is a pediatric blood and marrow transplant physician at M. Health Fairview. Both are featured in the documentary, "Sequencing Hope," which is premiering tonight at 7:00 PM. We'll have that information on our website, mprnews.org.

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Liam Shore is a third-year researcher at the University of Liverpool, in the Department of Philosophy. His research interests fall within the domain of ethics, notably on the ethics of digital and biotechnologies.

The Making of a Philosopher

Im a philosopher, but I havent always been one, so how does someone become a philosopher? And more fundamentally, why would anyone want to become one?

As a rare vocation, youd be forgiven for supposing that philosophers are an extinct species who once roamed the Athenian plazas during early antiquity, gesticulating poignantly and wearing togas. Well, happily they do exist today, sans the togas, largely unnoticed, behind the scenes on ethics boards, or engaging in fundamental first-principles critiques of.well.everything.

A question arises: if philosophers critique everything, how do they develop knowledge to criticise specialist areas? This becomes particularly poignant in an applied ethics context. My own personal journey, from Technologist to Philosopher, shows that one practically needs to be educated in two disciplines to become a bona fide philosopher.

When deciding what subjects to study, and what career to pursue, I was torn between multiple strong interests. In third place, Technology; in second place, Medicine; and in first place, Philosophy. In my case, I took the reverse path toward becoming a philosopher. Namely, I studied technology, worked in the biological sciences industry, and returned to academia with domain-specific expertise to enter into the philosophy sub-field of ethics. The beauty of philosophy for me, and the reason why I personally had the desire to pursue becoming a philosopher, is that philosophy, being able to critique everything, can powerfully converge disparate interests. It is this quality that made philosophy my first love, and so my PhD journey began, delving into the ethics of radical life extension.

Understanding Rejuvenation Biotechnologies

Recently, breakthroughs in rejuvenation biotechnologies, particularly those of the Strategies for Engineered Negligible Senescence (SENS) variant, have garnered little attention, and yet constitute steps towards a paradigm-altering event. SENS therapies, like maintaining classic cars to prolong their lifespan, seeks to do the same for our bodies as we age. SENS suggests that ageing is caused by the accumulation of cellular and molecular damage throughout the body over time, and advocates posit that by repairing or reversing this damage, it is possible to rejuvenate tissues and organs, thereby extending a persons healthy lifespan. Ultimately, by seeking to tackle age-related diseases at their root, via interventions such as stem cell therapies, the aim is to bring age-related diseases fully under comprehensive medical control. Overall, the eventual aim of SENS is to combine a panel of these therapies to combat all preceding causes of age-related diseases, and consequently, tackle ageing itself!

Although this sounds futuristic, there are therapies in various stages of development, with the furthest along being in clinical trials. Advocates claim that these therapies could, in due course, function well enough to rejuvenate a persons body to a youthful state. In effect, this is a process that, amongst other things, removes damage and replaces cells, enabling the body to regain a healthy condition. The outcome of extending good health is that it prolongs life, as it postpones the onset of age-related diseases until higher chronological ages. Accordingly, if someone repeatedly receives these therapies throughout life, this could constitute a potentially radical life-extending situation, as periods of poor health may be postponed repeatedly, allowing one to maintain optimal physiological functioning for longer, thereby delaying death itself!

A Case for Philosophical Inquiry The SENS approach to rejuvenation biotechnologies represents a bold vision for extending healthy lifespans and combating age-related diseases. However, realising this vision requires careful consideration of the ethical implications of extending human lifespan, making the SENS approach a question for philosophical research.

The most common ethical concerns for life-extending technologies are Health Equity i.e. fairness in health opportunities for all; Longevity/Population Dynamics i.e. understanding how long people live & how populations change; Environmental Impacts i.e. the effects of human activities on nature and Informed Consent/Autonomy i.e. respecting peoples right to make their own decisions.

Nevertheless, although important, these concerns dont engage with how this technology impacts what we find meaningful at the profoundest level as human beings. However, my research incorporates all the aforementioned ethical concerns and delves deeper into the realms of identity, purpose, and meaning in life, primarily through an existentialist lens.

Existentialism, as a philosophical theory, concerns itself with questions of: the nature of individual existence, authenticity of self, human freedom, and the search for purpose/meaning in life. It is via this prism that Im currently defining a taxonomy of values supported by radical life extension advocates, with this taxonomy categorising virtues like fairness, compassion, and autonomy, providing a structured framework for ethical analysis. In addition, Im exploring how a SENS-induced radically extended life may impact what we value. And next, I plan to explore whether the consequences of SENS therapies could result in mental ageing, in essence a feeling of listlessness, a sense of ennui, or a notion of world-weariness.

Overall, I hope that my research will deliver original insights to help us work towards a future where radically extended healthspans are possible, while fully prioritising and ensuring human well-being.

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Genetically engineered B cells could produce super-antibodies to HIV

Researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune systems B cells to spur them to produce broadly neutralizing antibodies against HIV. In theory, such an approach could control or eliminate the infection without need for ongoing antiretroviral therapy. Shown is the engineered adenovirus designed to deliver HIV superantibody genes into B cells.

HIV infections can be controlled with medication, but such therapy must continue throughout patients lives because no strategy exists to eliminate the virus from the body or control the infection without ongoing treatment.

With the aim of developing such a strategy, researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune systems B cells to spur them to produce broadly neutralizing antibodies against HIV. In theory, such an approach could control or eliminate the infection without need for ongoing antiretroviral therapy.

Permanent ways to control or eliminate HIV infection remain elusive, and their development is a major goal of the field, said David T. Curiel, MD, PhD, the Distinguished Professor of Radiation Oncology. The idea of modifying B cells which naturally produce antibodies to ensure that they manufacture specific antibodies that are broadly effective at targeting HIV is an exciting strategy. We have brought together a great team with expertise in HIV, gene therapy, and animal models of infection to work toward this goal.

Curiels co-principal investigators are Michael R. Farzan, PhD, of Harvard Medical School and Boston Childrens Hospital, and Mauricio de Aguiar Martins, PhD, of the University of Florida.

Over the decades since HIV appeared, researchers have learned that about 1% of people with the virus are able to produce what might be considered superantibodies against the virus. Such individuals known as elite neutralizers can produce antibodies against multiple strains of HIV.

Some people naturally have antibodies that can bind and destroy or deactivate very diverse strains of HIV, and we now have the ability to build those types of antibodies in the lab, said Paul Boucher, a doctoral student in Curiels lab. But just giving other patients these superantibodies is not an ideal solution, because these proteins would stay in the body only temporarily. Instead, our approach is to genetically modify the cells responsible for making antibodies the immune systems B cells so they can always produce superantibodies against HIV whenever they may need to.

Such engineered B cells could create, in theory, a state of permanent vaccination against the virus. Even if such a gene therapy doesnt fully clear HIV from the body, the strategy could allow the amount of virus in the body to be controlled, keeping it at a minimal level and creating a functional cure, according to the researchers.

The strategy involves modifying a different type of virus, called adenovirus. When used in gene therapy, such viruses are genetically disabled so they cant cause disease. The researchers then could engineer the adenovirus to carry the gene responsible for manufacturing broadly neutralizing antibodies to HIV. In the same viral vector, they also could include genes responsible for manufacturing the CRISPR/Cas9 gene editing proteins. In this way, the gene therapy delivery vehicle would carry into the body both the antibody gene that will be edited into the B cell genome and the genes to build the molecular tools to carry out that editing.

Using a three-part targeting strategy, the researchers would design the adenovirus to deliver its genetic payload only to B cells, avoiding other cell types. They have developed ways to modify the virus so that it is targeted directly to a protein that is expressed on the surface of B cells and no other cell types. The researchers can further restrict the targeting by using genetic methods to ensure that the CRISPR/Cas9 proteins can only be manufactured when their genes are delivered into B cells. Finally, they have developed strategies to modify the adenovirus in a way that stops its natural tendency to accumulate in the liver.

This strategy to modify B cells is distinct from another adenoviral gene therapy approach to HIV treatment that is currently in clinical trials led by principal investigator Rachel M. Presti, MD, PhD, a professor of medicine in the Division of Infectious Diseases at Washington University School of Medicine. HIV is difficult to eliminate from the body because the virus integrates its genome into the DNA of the infected individuals T cells. The strategy currently in clinical trials is focused on using precise targeting of the CRISPR/Cas9 gene editing proteins to excise the virus from the genomes of all of a patients infected T cells. This strategy is being tested in a first-in-human, phase 1 clinical trial to determine its safety and preliminary efficacy at various doses.

Curiel said engineered B cells are ripe for developing new therapies to treat a wide variety of diseases. In November, a genetically engineered B cell therapy was administered to a patient for the first time at the University of Minnesota Medical Center. In that case, the therapy was designed to treat mucopolysaccharidosis type 1, a life-threatening condition in which the body lacks an enzyme necessary to break down large sugar molecules inside cells.

Gene therapy with engineered B cells is an exciting new area of research, Curiel said. We look forward to combining our expertise in adenovirus gene therapy, HIV infection and preclinical models of disease to realize our plan for developing an HIV therapy that we hope can permanently control the infection.

This work is supported by the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH), grant number 1R01-AI174270-01A1. This content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

About Washington University School of Medicine

WashU Medicine is a global leader in academic medicine, including biomedical research, patient care and educational programs with 2,900 faculty. Its National Institutes of Health (NIH) research funding portfolio is the second largest among U.S. medical schools and has grown 56% in the last seven years. Together with institutional investment, WashU Medicine commits well over $1 billion annually to basic and clinical research innovation and training. Its faculty practice is consistently within the top five in the country, with more than 1,900 faculty physicians practicing at 130 locations and who are also the medical staffs of Barnes-Jewish and St. Louis Childrens hospitals of BJC HealthCare. WashU Medicine has a storied history in MD/PhD training, recently dedicated $100 million to scholarships and curriculum renewal for its medical students, and is home to top-notch training programs in every medical subspecialty as well as physical therapy, occupational therapy, and audiology and communications sciences.

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$6.2 million to help develop gene therapy for HIV Washington University School of Medicine in St. Louis - Washington University School of Medicine in...

Recommendation and review posted by Bethany Smith

A type of gene therapy called CAR-T that has extended survival for thousands of patients with leukemia and other blood cancers is being adapted at UC San Francisco to treat people with glioblastoma, the most common and deadly adult brain tumor.

This new more powerful version of CAR-T employs a novel technology developed at UCSF called synthetic notch (synNotch) that both protects healthy tissue from damage and enables the treatment to work more effectively.

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Approximately 12,000 Americans are diagnosed each year, with an average survival of just 15 months.

UCSF opened enrollment this week for a clinical trial that is using the technology for the first time in people. A second trial, also at UCSF, is slated for 2025.

Approximately 12,000 Americans are diagnosed each year with glioblastoma. Patients survive on average for just 15 months after their diagnosis, and new treatments are urgently needed.

This project is a prime example of bench-to-bed translation within UCSF, representing the strengths in basic and clinical science, said Hideho Okada, MD, PhD, a physician-scientist and director of the UCSF Brain Tumor Immunotherapy Center. We have a truly home-grown project here.

Okada has received up to $11 million for the first trial from the California Institute for Regenerative Medicine (CIRM), which funds stem cell and gene therapy research for incurable diseases and disorders through all stages of clinical trial development.

Initial funding for the second trial is provided by the National Cancer Institute Specialized Programs of Research Excellence (NCI SPORE).

We hope that the treatment will prolong lives for patients with glioblastoma, said Okada, who is a professor of neurosurgery at UCSF and a member of the Weill Institute for Neurosciences. However, the primary goal of the current phase 1 study is to ensure safety and characterize any toxicities.

When tested in mice, Okada said the therapy provided a robust and long-lasting result that was more remarkable than anything he had encountered during 30 years of brain tumor research.

The CIRM-funded trial will be led by principal investigator Jennifer Clarke, MD, MPH. It is open to patients with newly diagnosed glioblastoma, who have completed standard-of-care treatment. Tumors must have a mutation found in approximately 20% of glioblastomas, and that can be identified by the UCSF500 cancer gene panel test.

The second study will be open to glioblastoma patients whether or not they have the mutation.

CAR-T refers to chimeric antigen receptor T-cells, which are cancer-killing immune cells that have been extracted from the patient and genetically modified to recognize and destroy antigens that appear on the surface of cancer cells. These supercharged CAR-T cells are then infused back into the body to attack tumor cells.

For many patients with leukemia and other blood cancers, CAR-T has demonstrated long-term remission, but the approach hasnt worked against brain tumors. Glioblastoma cells are more diverse than blood cancer cells, and they can evade CAR-T. Many of the antigens made by the tumors are also found in healthy tissue, leaving them open to attack.

To overcome these obstacles, Okada drew from the synNotch system developed by Wendell Lim, PhD, director of the UCSF Cell Design Institute and professor in the UCSF Department of Cellular and Molecular Pharmacology.

The technology allowed scientists to program CAR-T cells to target specific antigens on tumor cells, without touching those found in healthy tissue. They also do not succumb to T-cell exhaustion, a common problem with CAR-T therapies, because they are more metabolically stable and use less energy to fight cancer longer.

Weve created a system that is flexible and thorough and addresses the major concerns weve had about using CAR-T cells against solid tumors, Lim said. These cells act like computers: integrating multiple units of information and making complex decisions.

About the California Institute for Regenerative Medicine (CIRM): AtCIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of todays most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio,CIRMis one of the worlds largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

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Gene Therapy Is Halting Cancer. Can It Work Against Brain Tumors? - UC San Francisco

Recommendation and review posted by Bethany Smith

Gene therapy research is booming. Since the U.S. Food and Drug Administration (FDA) issued its first approval for a gene therapyin 2017, oncology researchers have been breaking barriers in gene therapy trials, followed by an explosion in mRNA research during the COVID pandemic. Today, this trailblazing science is providing new ways to approach rare diseases and new hope when other investigational interventions have failed. In fact, themajorityof approved gene therapies are for rare diseases 14 are currently in Phase III trials for 10 rare diseases and 45 gene therapies are in early stages of development to treat 30 rare diseases. We see great potential for gene therapies, said Leslie Johnston, senior vice president of biotech delivery for Parexel. As more products are approved, it will gain traction and more companies will look to expand their therapies into other therapeutic indications. This progress presents tremendous potential to change more patients lives across many different diseases. This could be gene therapys moment. But to fully seize it, the industry must clear some complex hurdles. Gene therapies pose several unique challenges for clinical research, including ethical and safety considerations, regulatory hurdles, precarious logistics, and potentially staggering costs. These challenges may already be having ramifications: New U.S. patients treated with gene therapies approved or in development areexpected to fallby one-third from 2025 to 2034. The key to clearing these hurdles? Cooperation between sponsors, sites, regulators, patients, and other stakeholders is essential to expediting the advancement of life-saving gene therapies. Regulators should address risks without limiting innovation Gene therapy trials are strictly regulated and rightly so, due to the novel nature of the intervention and the potential long-term consequences. Gene therapy interventions also carry inherent safety risks, including the potential for unintended genetic changes or adverse immune reactions. Ensuring patient safety requires rigorous monitoring and adherence to strict protocols. However, obtaining regulatory approval under these conditions is time consuming and resource intensive. To avoid hampering scientific progress, regulators should aim to ensure that requirements are appropriately rigorous without being unmanageably onerous. Thankfully, the FDA is paying close attention to gene therapy and has demonstrated a desire to work with drug developers toward the success and approval of these treatments. Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA, has expressed his hope for an exponential, if not logarithmic, increase in gene therapy approvals. There is a lot of excitement that this could potentially make a big difference for the treatment of human disease, said Dr. Marks in hisremarksto the National Press Forum last November. The FDA is going beyond mere rhapsodizing and taking action to accelerate gene therapy. Last year, the agencylaunched a pilot programcalled Support for Clinical Trials Advancing Rare Disease Therapeutics, or START. This program is designed to accelerate the development and approval process for treatments targeting rare diseases by providing regulatory guidance, assistance, and incentives to sponsors conducting clinical trials in this field. The program represents an important step forward in fostering innovation and collaboration between regulatory bodies and sponsors. In addition, the FDA is working toharmonize global requirementsfor the review of gene therapies. Encouraging and facilitating international cooperation and harmonization of regulatory standards including mutual recognition agreements and shared regulatory pathways for multinational clinical trials can help streamline gene therapy development globally and help bring innovations to patients faster. Even with this progress, regulators should continue to help accelerate gene therapy research by streamlining regulatory pathways specifically tailored to gene therapies. This means providing clear guidance on requirements for preclinical and clinical development, fostering collaboration between stakeholders to share knowledge and best practices, and offering expedited review processes for gene therapy products aimed at treating serious or life-threatening diseases. With a staggering2,500 cell and gene therapyinvestigational new drug applications (INDs) on file, the FDA approved justfivecell and gene therapies in 2023. Dr. Marks hassuggestedthat accelerated approval, which has successfully advanced cancer and HIV/AIDS treatments, may be the most appropriate path for this new category of treatments. But, regulators also need to commit to proactively partner with developers to understand the patient population and the risks and benefits of each new therapy. Likewise, researchers, industry stakeholders, and patient advocacy groups should engage with regulators to help them understand the unique challenges and opportunities in the field of gene therapy. This can help regulators adapt regulatory frameworks to ensure patient safety while expediting the development and approval of promising treatments. Sites and sponsors must be prepared Of course, sites and sponsors also have a crucial part to play in advancing this promising field of medicine. Clinical trial sites should enhance their capacity to conduct gene therapy trials safely and effectively and sponsors should do their part to assist sites in these efforts. By working closely with clinicians and regulators, sponsors can ensure that the trial development process aligns with clinical needs and regulatory standards. Sponsors should have a thorough understanding of FDA requirements pertaining to design, preclinical testing, and long-term follow-up. Better alignment from the outset will lead to more efficient trial designs, faster regulatory approvals, and ultimately quicker patient access to therapies. For example, sponsors working with mRNA and other genetically engineered therapies in North America not only have to go through institutional review board (IRB) review, they also have to navigate additional requirements from the U.S. National Institutes of Health (NIH) Office of Science PolicyGuidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules(NIH Guidelines). These requirements usually involve an additional biosafety risk assessment review from an institutional biosafety committee (IBC) in addition to IRB review. NIH Guidelines apply for any research involving recombinant or synthetic nucleic acids (e.g. genetically engineered materials) that receives NIH support or takes place at sites that have received NIH support for such research. Even when there is zero NIH support, IBC review is considered a best practice. IBC review and inspection helps sites ensure they are fully prepared by identifying areas for improved biosafety protections and calling out gaps in current standard operating procedures (SOPs). Proactive coordination and integration of these separate review processes can speed trial timelines and help sponsors consistently address any potential concerns or issues. Sites can also be better prepared by pre-registering an IBC. The NIH takes six to eight weeks or more to approve a new registration, in addition to IBC review time so by registering an IBC before they even have a trial, sites can save a month or more in startup time over a site that waited to register. Clinical trial sites looking to host gene therapy studies must be prepared in other ways, as well, both in terms of knowledge and infrastructure. Gene therapy studies require specialized infrastructure for manufacturing, storing, and administering genetic material to adhere to strict biosafety guidelines. Something as simple as having an upholstered chair in the infusion room which would pose an unacceptable contamination risk if genetic materials were to spill would require the site to rethink their current processes. Rigorous training is also key due to the added risk of spreading genetic material to caregivers and others in close contact with patients. Research staff must be specially trained to handle, deliver, and dispose of this material safely. Of course, these measures can seem intimidating for sites that are already cost-constrained. Large academic medical centers with more resources and experience are more likely to be well-positioned for these studies. For instance, they may already have conducted bench, animal, and/or agricultural research with genetic engineering or have the funding to make any needed adjustments such as purchasing special equipment. But to maximize the potential number of sites where this research can be conducted and therefore reach more potential participants sponsors might consider providing help in the form of financial assistance, training curricula, SOP guidance, and more to smaller sites seeking to conduct gene therapy research. Logistical complexities depending on the investigational medicine and therapeutic area are among the most complicated challenges in gene therapy trials, added Johnston. From collecting the specimen from the patient, modifying it, storing it, transporting it, and then returning it back to the patient all comes with tremendously unique logistical challenges and requires equally unique equipment, technology, and expertise. And it can be cost-prohibitive. Patients must be fully on board Of course, the most essential stakeholder in any clinical trial is the patient. In gene therapy research, which can be particularly demanding, patients must have a complete understanding of and commitment to their involvement. Understanding the potential risks and benefits can help patients make informed decisions and navigate the study process. First, it's crucial for patients to adhere strictly to the protocol provided by the clinical trial team, including following medication schedules, maintaining specific hygiene practices, and attending all study visits. They should strive to maintain optimal health to enhance the body's response to gene therapy. And to avoid delays, patients should maintain open and honest communication with the clinical trial team, reporting any changes in symptoms, side effects, or general health as soon as they occur. Trial participants also need to be in it for the long haul. Because gene therapy interventions aim to produce lasting effects, even cures, they typically require long-term patient follow-up to assess efficacy and safety. But they may also need to have incredible patience. Johnston explained, There are many complexities that can impact study progress. For example, unpredictable logistical challenges like a weather event or vehicle accident could delay a temperature-sensitive delivery to a site, or data review outcomes could require an indeterminate pause period. Patience and agility are must-haves, but it is difficult for patients potentially depending on this new therapy to save or change their lives. Lastly, the industry cannot forget the patient. Involving patients and patient advocacy groups in the regulatory process can help ensure that the development of gene therapies is aligned with patient needs and priorities, as well as shed light on risk-benefit perspectives from a patients viewpoint. The more these perspectives are considered from the beginning, the greater the chance of a trials success. Rita Naman, co-founder of the Mighty Milo Foundation, emphasizes the need for a more collaborative and patient-centered approach to gene therapy development. "For ultra-rare diseases likeSPAX5, gene therapy offers a glimmer of hope where traditional treatments do not. But logistical hurdles make these therapies expensive and inaccessible, explained Naman. Closer collaboration with patients, industry, and regulators could streamline these processes, drive costs down, and speed trials. Patients like my son, and their caregivers, plus advocacy groups should be invited into the earliest discussions to prevent false starts or missed milestones in gene therapy development especially as the patients priorities dont always line up with the sponsors. In the fight for gene therapy breakthroughs, cooperation is key. The road to operationalizing gene therapy clinical trials is laced with land mines and potholes. To capture the full potential of novel gene therapy research, a new level of collaboration between sponsors, CROs, sites, oversight committees, regulatory bodies, and patients is paramount. Patients want access to novel gene treatments, and they want it fast. Sponsors want to deliver but fight logistical and financial obstacles. Regulators want to ensure safety first, especially considering such new, promising science, concluded Johnston. These three goals may seem conflicting at times, so we need to strike a balance of safety and speed, so patients dont miss their only potential treatment opportunity. A seasoned industry veteran with more than 25 years of experience, James Riddle is senior vice president of global review operations at Advarra. Riddles expertise includes large program management and growth, operational processes, development and implementation of technology solutions, and management of large Human Research Protection Programs (HRPP), Biosafety programs (IBC) and Institutional Animal Care and Use programs (IACUC). Riddle has directed numerous clients in achieving Part 11 compliance and meeting computer system validation requirements.

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Gene Therapy is Having its Moment: Can the Clinical Research Ecosystem Seize It? - Contract Pharma

Recommendation and review posted by Bethany Smith

One of their biggest successes uses gene therapy to treat a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency.

Children with AADC deficient are missing the enzyme that produces dopamine and serotonin in the central nervous system. This affects pathways in the brain responsible for motor function and emotions.

As a result, these children cant coordinate the movements of their head, face and neck. They often dont reach normal childhood milestones, such as sitting up or walking by themselves.

Along with her mother, Arcelia Ramirez, they traveled 800 miles from their home near Omaha, Neb., so that Delilah could have this life-changing gene therapy surgery at Ohio State Wexner Medical Center.

But now, Delilah has changed so much for the better. On her 9th birthday, she blew out a candle on her cupcake on purpose. This was the first time she had ever blown out a birthday candle.

She's like a different kid. Her sleeping is a lot better. She can walk now, she can self-feed, said Arcelia Ramirez. When she started using a fork, that was a reason to celebrate. When she started using a straw, that was a reason to celebrate. Walking was a big, big milestone for her that we just celebrated.

So we are bringing in a correctly spelled sequence of the gene, said Bankiewicz, who is also chief scientific officer at the Ohio State Gene Therapy Institute.

This helps ensure we put the genetic material in exactly the right place, so the brain will start making dopamine and serotonin again, said Elder, who also is a professor of neurological surgery. This

therapy is designed to approach both parts of the brain that control movements and emotions.

This breakthrough in treating patients with AADC was decades in the making.

It requires a use of the technology and devices that we had to develop and establish over the years to do these surgeries very precisely, very carefully and then do it safely, Bankiewicz said. The issue of, Is it going to work? It's no longer being questioned. It works.

In addition to expanding this method to central nervous system diseases such as Alzheimers, Parkinsons, Multiple System Atrophy and Huntingtons disease, Elder and Bankiewicz are also trying to edit genetic mutations in other neurological disorders, including brain tumors.

We are not treating a gene that causes Parkinson's or Alzheimer's, Bankiewicz said. We're using this technology to deliver a therapeutic that we believe will, in a positive way, affect the progression of the disease.

# # #

Media Contact: Eileen Scahill, Wexner Medical Center Media Relations, Eileen.Scahill@osumc.edu

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The future of gene therapy has arrived, and it's changing lives - Wexner Medical Center - The Ohio State University

Recommendation and review posted by Bethany Smith

The global gene therapy market size was valued at USD 8.75 billion in 2023 and is poised to grow from USD 10.47 billion in 2024 to USD 52.40 billion by 2033, growing at a CAGR of 19.6% in the forecast period (2024-2033).

Gene therapy is a technique that uses a gene to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patients cells with a healthy version of that gene. Both inherited genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia) have been treated with gene therapy.

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The development of the market is owing to an increase in the number of gene therapy-based discoveries, increasing investment in this sector, and rising approval of gene therapy products. According to the WHO, 10 to 20 new cell and gene therapies are expected to be approved each year by 2025.

Continuous developments in recombinant DNA technology are anticipated to enhance the efficiency of gene therapy in the coming years. Hence, ongoing progresses in recombinant DNA technology are anticipated to expand the number of ongoing clinical trials for gene therapy. Primarily, these advancements are taking place in the context of various gene-editing tools and expression systems to augment the R&D for products. The advent of CRISPR/Cas9 nuclease, ZFN, and TALEN allows easy & precise genome editing. As a result, in recent times, the gene-editing space has witnessed a substantial number of research activities, which, in turn, is expected to influence the growth of the gene therapy market.

The growth of the gene therapy market is expected to be majorly benefitted from the increasing prevalence of cancer. The ongoing increase in cancer patients and related death per year emphasizes the essential for the development of robust treatment solutions. In 2020, there were around 18.1 million new cases of cancer worldwide. 9.3 million of these cases involved men, while 8.8 million involved women. Continuing developments in tumor genetic studies have delivered substantial information about cancer-related molecular signatures, which in turn, is expected to support ongoing clinical trials for cancer therapeutics.

With rising demand for robust disease treatment therapies, companies have focused their efforts to accelerate R&D for effective genetic therapies that target the cause of disease at a genomic level. . Furthermore, the U.S. FDA provides constant support for innovations in this sector via a number of policies with regard to product manufacturing. In January 2020, the agency released six final guidelines on the manufacturing and clinical development of safe and efficient products.

Furthermore, facility expansion for cell and gene therapies is one of the major factors driving the gene therapy market growth. Several in-house facilities and CDMOs for gene therapy manufacturing have begun investing to enhance their production capacity, which, in turn, is anticipated to create lucrative opportunities for market players. For instance, in April 2022, the FDA approved commercial licensure approval to Novartis for its Durham, N.C. site. This approval permits the 170,000 square-foot facility to make, test, and issue commercial Zolgensma, as well as manufacture therapy products for current & upcoming clinical trials.

Cell and Gene Therapy Market :https://www.biospace.com/article/releases/u-s-cell-and-gene-therapy-clinical-trial-services-industry-is-rising-rapidly/

Gene Therapy Market Report Highlights

U.S. Gene Therapy Market Size in U.S. 2024 to 2033

The U.S. gene therapy market size was estimated at USD 3.19 billion in 2023 and is projected to surpass around USD 18.50 billion by 2033 at a CAGR of 19.22 % from 2024 to 2033.

North America dominated the market in 2023 with the largest revenue share of 65.12% in 2023. This region is expected to become the largest routine manufacturer of gene therapy in terms of the number of approvals and revenue generated during the forecast period. Increasing investments in R&D from large and small companies in the development of ideal therapy drugs are anticipated to further boost the market.

Furthermore, the increasing number of investments by the governments and the growing prevalence of targeted diseases are the factors fueling the market. According to the Spinal Muscular Atrophy Foundation, in 2020, around 10,000 to 25,000 children and adults in the U.S. were affected by spinal muscular atrophy, making it a fairly common disease among rare diseases.

Europe is estimated to be the fastest-growing regional segment from 2024 to 2030. This is attributed to its large population with unmet medical needs and increasing demand for novel technologies in the treatment of rare but increasingly prevalent diseases. Asia Pacific market for commercial application of genetic therapies is anticipated to witness significant growth in the forecast period, which can be attributed to the easy availability of resources, local presence of major companies, and increased investment, by the governments.

UK Gene Therapy Market

The UK gene therapy market is anticipated to witness accelerated growth over the forecast period, due to increased investments by various big companies and governments, including the NHS & research laboratories. For instance, in March 2022, the UK government invested USD 326.45 million to accelerate healthcare research and manufacturing. Under this investment, additional $80 million of the fund will help companies at the forefront of invention with their commercial-scale manufacturing investments in areas like gene and cell therapies, as well as improved diagnostic technologies, among others. Various mergers & partnerships between manufacturers, universities, and other government bodies are expected to boost the market over the forecast period.

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What is gene therapy used for?

Most gene therapies are still in the clinical trial phase. Clinical trials play an important role in finding treatments that are safe and effective. Clinical trials are investigating gene therapy for the treatment ofcancer,macular degenerationand other eye diseases, certaingenetic conditionsandHIV/AIDS.

The U.S. Food and Drug Administration (FDA) has approved two gene therapies for use in the U.S.:

Is gene therapy safe?

The first gene therapy trial was run more than thirty years ago. The earliest studies showed that gene therapy could have very serious health risks, such as toxicity, inflammation, and cancer. Since then, researchers have studied the mechanisms and developed improved techniques that are less likely to cause dangerous immune reactions or cancer. Because gene therapy techniques are relatively new, some risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research, clinical trials, and approved treatments are as safe as possible.

Comprehensive federal laws, regulations, and guidelines help protect people who participate in research studies (called clinical trials). The U.S. Food and Drug Administration (FDA) regulates all gene therapy products in the United States and oversees research in this area. Researchers who wish to test an approach in a clinical trial must first obtain permission from the FDA. The FDA has the authority to reject or suspend clinical trials that are suspected of being unsafe for participants.

The National Institutes of Health (NIH) also plays an important role in ensuring the safety of gene therapy research. NIH provides guidelines for investigators and institutions (such as universities and hospitals) to follow when conducting clinical trials with gene therapy. These guidelines state that clinical trials at institutions receiving NIH funding for this type of research must be registered with the NIH Office of Biotechnology Activities. The protocol, or plan, for each clinical trial is then reviewed by the NIH Recombinant DNA Advisory Committee (RAC) to determine whether it raises medical, ethical, or safety issues that warrant further discussion at a RAC public meeting.

An Institutional Review Board (IRB) and an Institutional Biosafety Committee (IBC) must approve each gene therapy clinical trial before it can be carried out. An IRB is a committee of scientific and medical advisors and consumers that reviews all research within an institution. An IBC is a group that reviews and approves an institution's potentially hazardous research studies. Multiple levels of evaluation and oversight ensure that safety concerns are a top priority in the planning and carrying out of gene therapy research.

The clinical trial process occurs in three phases. Phase I studies determine if a treatment is safe for people and identify its side effects. Phase II studies determine if the treatment is effective, meaning whether it works. Phase III studies compare the new treatment to the current treatments available. Doctors want to know whether the new treatment works better or has fewer side effects than the standard treatment. The FDA reviews the results of the clinical trial. If it determines that the benefits of the new treatment outweigh the side effects, it approves the therapy, and doctors can use it to treat a disorder.

What are CAR T cell therapy, RNA therapy, and other genetic therapies?

Several treatments have been developed that involve genetic material but are typically not considered gene therapy. Some of these methods alter DNA for a slightly different use than gene therapy. Others do not alter genes themselves, but they change whether or how a genes instructions are carried out to make proteins.

Cell-based gene therapy

CAR T cell therapy (or chimeric antigen receptor T cell therapy) is an example of cell-based gene therapy. This type of treatment combines the technologies of gene therapy and cell therapy. Cell therapy introduces cells to the body that have a particular function to help treat a disease. In cell-based gene therapy, the cells have been genetically altered to give them the special function. CAR T cell therapy introduces a gene to a persons T cells, which are a type of immune cell. This gene provides instructions for making a protein, called the chimeric antigen receptor (CAR), that attaches to cancer cells. The modified immune cells can specifically attack cancer cells.

RNA therapy

Several techniques, called RNA therapies, use pieces of RNA, which is a type of genetic material similar to DNA, to help treat a disorder. In many of these techniques, the pieces of RNA interact with a molecule calledmessenger RNA(or mRNA for short). In cells, mRNA uses the information in genes to create a blueprint for making proteins. By interacting with mRNA, these therapies influence how much protein is produced from a gene, which can compensate for the effects of a genetic alteration. Examples of these RNA therapies include antisense oligonucleotide (ASO), small interfering RNA (siRNA), and microRNA (miRNA) therapies. An RNA therapy called RNA aptamer therapy introduces small pieces of RNA that attach directly to proteins to alter their function.

Epigenetic therapy

Another gene-related therapy, called epigenetic therapy, affectsepigenetic changesin cells. Epigenetic changes are specific modifications (often called tags) attached to DNA that control whether genes are turned on or off. Abnormal patterns of epigenetic modifications alter gene activity and, subsequently, protein production. Epigenetic therapies are used to correct epigenetic errors that underlie genetic disorders.

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Vector Insights

The AAV segment shows a significant revenue contribution of 22.9% in 2023. Several biopharma companies are offering their viral vector platform for the development of AAV-based gene therapy product. For instance, in September 2016, Lonza signed an exclusive agreement with Massachusetts Eye and Ear to support its novel Anc-AAV gene therapy platform for development and commercialization of next-generation gene therapies based on their AAV platform. Similarly, RegenxBio had made an agreement with companies AveXis & Biogen in March 2014 and May 2016, respectively, which would allow both companies to use RegenxBios AAV vector platform for development of gene therapy molecules. Furthermore, in May 2021, Biogen Inc. and Capsigen Inc. entered into a strategic research partnership to engineer novel AAV capsids that have the possibility to deliver transformative gene therapies, which can address the fundamental genetic causes of numerous neuromuscular and CNS disorders. In July 2021, the U.S. Department of Commerces National Institute of Standards and Technology (NIST), National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and United States Pharmacopeia (USP) announced a collaboration to evaluate analytical methods and develop standards for AAV. As part of this partnership, NIST and USP will be conducting an interlaboratory study in which several laboratories will measure these serious quality attributes, and their results will be linked and examined. This collaboration will support the development of new promising gene therapies that will significantly advance peoples lives.

Indication Insights

The spinal muscular atrophy (SMA) segment dominated the market in 2023. Although SMA is a rare disorder, it is one of the most common fatal inherited diseases of infancy. The development of Zolgensma (AVXS-101), has proven its effectiveness in treating SMA and altering the phenotype of the illness. The FDA approved Novartis' Zolgensma approval in May 2019, which is aimed at treating the underlying cause of SMA. As of now, Zolgensma is the only gene treatment in this field to have been approved. The approval of this gene therapy is evidence of the growing use of therapies to treat serious hereditary illnesses like SMA.

The Beta-Thalassemia Major/SCD segment is anticipated to register the fastest CAGR over the forecast period. Gene therapy for SCD and -thalassemia is based on transplantation of gene-modified hematopoietic stem cells. Clinical and preclinical studies have shown the efficacy and safety of this therapeutic modality. However, several other factors, such as suboptimal gene expression levels & gene transfer efficiency, limited stem-cell dose and quality, and toxicity of myeloablative regimens are still hampering its efficacy. Despite these challenges, in June 2019, bluebird Bios Zynteglo (formerly LentiGlobin) received conditional approval in Europe for the treatment of -thalassemia and is expected to receive U.S. FDA approval in August 2022. Moreover, the product has already received Orphan Drug status by the U.S. FDA for treatment of patients with sickle cell disease (SCD). Furthermore, in April 2021, Vertex Pharmaceuticals and CRISPR Therapeutics amended partnership for the development, production, and commercialization of CTX001 in sickle beta thalassemia and cell disease. These achievements in this segment are anticipated to significantly boost the adoption of the product in this segment.

Route of Administration Insights

The intravenous segment dominated the global gene therapy market in 2023. Large number of approved products along with strong pipeline for IV candidates is the major reason for the segment dominance. The segment is also expected to emerge as the most lucrative over the forecast period.

Recent Developments

Some of the prominent players in the Gene therapy market include:

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the global gene therapy market.

Indication

Vector Type

Route of Administration

By Region

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Gene Therapy Market Size Poised to Surge USD 52.40 Billion by 2033 - BioSpace

Recommendation and review posted by Bethany Smith

The global cancer gene therapy market size was accounted for USD 2.95 billion in 2023 and it is increasing around USD 18.11 billion by 2033 with a CAGR of 19.9% from 2024 to 2033, according to a new report by Nova One Advisor.

Cancer Gene Therapy Market Overview

Cancer is a group of diseases that involve abnormal cell growth which can spread to respective parts of the body. Cancer can spread throughout the human body.Gene therapyis a kind of treatment in which the genes that are not normal or are missing in the patients cells are replaced with normal genes. Cancer gene therapy is a technique for treating cancers where the therapeutic DNA is introduced in the gene of the individual suffering from cancer.

Due to a high success rate in preclinical as well asclinical trials, cancer gene therapy is gaining high popularity all over the world. There are numerous techniques utilized in cancer gene therapy. In one of the gene therapy techniques, either the mutated gene is replaced with a healthy gene, or the gene is inactivated if its function is abnormal. In a newly developed technique, new genes can be introduced in the body of the patient to help fight against cancer cells.

Further, the ongoing extensive research and development (R&D) strategies implemented bybiopharmaceuticalfirms for producing novel therapeutic drugs are driving the market growth notably.

The market players can aim towards expansions, collaborations, joint ventures, acquisitions, and partnerships to advance capabilities in gene therapy. This would help in yielding effective therapeutic drugs for treating different kinds of cancers. In April 2022, GSK plc announced the acquisition of Sierra Oncology for 1.6 billion ($1.9 billion). This acquisition would help GSK plc in enhancing its capabilities with respect to targeted therapies for treating rare forms of cancer.

Biotechnologyfirms are evaluating novel gene therapy vectors for increasing levels of protein production/gene expression, reducing immunogenicity, and improving durability.

The top cancers in terms of the count of new cases in 2020 all over the world were Lung Cancer (2,206,771 cases),Breast Cancer(2,261,419 cases),Prostate Cancer(1,414,259 cases), Colorectal Cancer (1,931,590 cases), Stomach Cancer (1,089,103 cases), and Liver cancer (905,677 cases). In 2018, there were around 134,632 new cancer cases and 89,042 cancer-related fatalities. Breast and liver cancers were among the most common tumors in terms of incidence and mortality. The high prevalence of breast cancer cases enhances the scope for CRISPR/Cas9-based gene editing for breast cancer therapy and VISA-claudin4-BikDD gene therapy.

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Key Takeaways:

Cancer Gene Therapy Market Size in U.S. 2024 to 2033

The U.S. cancer gene therapy market size was valued at USD 1.25 billion in 2023 and is anticipated to reach around USD 7.94 billion by 2033, growing at a CAGR of 20.31% from 2024 to 2033.

North America accounted for the largest share of over 61.15% in 2023. This is attributed to the conducive environment facilitated by the government and the National Cancer Institute that supports research and development activities to enhance cancer therapeutics. Further, the presence of key market players in the region, their research efforts in devising gene therapy for cancer treatment, and collaborative efforts among market players to enhance research are boosting the market growth in the region. For instance, in August 2022, Merck & Co., Inc., collaborated with Orna Therapeutics Inc., for discovery, development, and commercialization of multiple programs, inclusive of utilization of mRNA for cancer gene therapy.

Europe is estimated to be the fastest-growing region over the forecast period due to increase in research funding for novel therapeutics by government bodies and increasing demand for novel therapeutics that could help combat the growing incidence of cancer cases across the region. Moreover, The European Unions Horizon Europe Mission on Cancer was launched in September 2023 so as to offer funds to a broad spectrum of activities that are intended to lower Europes cancer burden by accelerating research and innovation in cancer therapeutics. The mission is anticipated to help over 3 million cancer survivors by the year 2033.

The cancer gene therapy market in the Asia Pacific (APAC) region is segmented into India, China, Japan, South Korea, and the rest of the Asia Pacific (APAC) region. China dominated the Asia Pacific region followed by Japan and India in 2023.

The Latin America, Middle East, and African (LAMEA) cancer gene therapy market is segmented into North Africa, South Africa, Saudi Arabia, Brazil, Argentina, and the Rest of LAMEA. The Middle East and the Latin America region are anticipated to have notable growth in the cancer gene therapy market during the forecast period. Brazil held the largest share in the LAMEA region in 2023. Due to low literacy, uncertainty, and civil war in African countries, the cancer gene therapy market in Africa is expected to grow at a comparatively slow rate.

What are the importance of Cancer Gene Therapy?

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Types of gene therapy for cancer

Gene therapy aims tocontrolthe altered genesor genetic mutationsof a cancertoprevent the cancers growth.This approach to using our own cells and genes to treat cancer is called somatic gene therapy.Thistype of gene therapydoes not impact germ-line cells in the reproductive system, meaning none of the genetic changescan bepassedon to otherfamily members.

There are four types of somatic gene therapy: gene editing; gene replacement; gene addition; and gene inhibition.

Gene editing is correcting the cells gene to fix the imbalance by snipping out the faulty part of the gene and changing the cancers DNA. This type of gene therapy may correct the alteration rather than trying to remove it. Gene replacement is just that: replacing the faulty or nonworking gene with a healthy copy of it. This type of gene therapy is another form of trying to fix the genetic change rather than trying to remove it.

Gene addition is adding novel genetic code to a different cell usually an immune system fighter cell to help it combat the protein linked to the damaged gene. CAR T-cell therapy is an example of gene addition. This form of gene therapy isnt adding a copy of an already-existing gene but rather an entirely new gene usually with the intent of killing the cancer cell via the immune system. Doctors may also add a new gene directly to the cancer cell that causes the cancer cell to commit apoptosis (kill itself).

Gene inhibition simply shuts down the faulty gene. This can either kill the cell or prevent it from acting in a cancerous manner, such as growing and replicating exponentially.

Steps of gene therapy

Gene therapy is a new and potentially curative approach to treating cancer, but researchers still have so much to learn. While the steps below may seem straightforward, each part of the process requires years of study to develop the technologies.

Researchers must first identify the gene and protein linked to the cancer. The next steps are:

Steps of CAR T-cell therapy

CAR T-cell therapy has a slightly different process than more direct forms of gene therapy. CAR T cells are lab-generated fighter cells with specific, anti-cancer genetic code. Adding this genetic code is the gene therapy component of CAR T-cell therapy. CAR stands for chimeric antigen receptor, which is the new genetic code added to the T cells.

There are six CAR T-cell therapy agents approved by the U.S. Food and Drug Administration for different blood cancers. These approvals validate CAR T cells as an effective form of cancer gene therapy to improve patient life expectancy.

Doctors first draw blood from a patient and separate the T cells, which are white blood cells leading the immune systems defense against viruses, diseases and more unwanted intruders. T cells aim to protect the body from cancer, but theyre often ineffective at doing so.

The process of drawing blood from patients and separating the T cells is called apheresis.

After removing T cells from the body, the steps of CAR T-cell therapy are:

A similar process occurs for CAR NK-cell therapy. Scientists create chimeric antigen receptors to strengthen natural killer (NK) cells, another white blood cell of the immune system.

How long does CAR T-cell therapy take?

There are six CAR T-cell therapies approved for types of three blood cancers: myeloma, leukemia and lymphoma. CAR T-cell therapy infusions can take place in an inpatient or outpatient care setting, but the patient must be closely monitored at all times.

CAR T-cell therapy can lead to side effects, most notable cytokine release syndrome.

The entire CAR T-cell process lasts approximately one month, not including the recovery time after treatment:

For the first seven days after receiving the CAR T-cell infusion, patients must remain under medical supervision. For weeks 2-4 of the post-infusion timeline, patients must remain within a short drive of their medical facility to respond to any issues.

The total recovery period from CAR T-cell therapy is usually 2-3 months following infusion, according to the Dana-Farber Cancer Institute.

There are several studies for CAR T-cell therapies for cancer. Participating in a clinical trial helps advance cell and gene therapy research and can advance much-needed therapies to more patients in need.

Therapy Insights

Gene induced immunotherapy dominated the market with a revenue share of over 41.9% in 2023. The dominance of the segment can be attributed to research studies aiming to lower the proliferation of various types of cancer by strengthening the immune system. Many gene therapies for cancers are designed on the basis of immunotherapy elements. For instance, PROVENGE (by Dendreon Corporation) is an autologous cellular immunotherapy designed to stimulate a subjects immune system against prostate cancer.

Oncolytic virotherapy is expected to grow at the fastest rate over the forecast period owing to the favorable outcomes and the level of efficacy offered by oncolytic virotherapy. Oncolytic viruses can combat cancer cells without disturbing the healthy cells in vicinity by stimulating natural killer cells. Moreover, there are lucrative research grants for the research on oncolytic virotherapy. For instance, in July 2022, the researchers at the Center for Nuclear Receptors and Cell Signaling at the University of Houston received a USD 1.8 million grant from the National Institutes of Health to work on oncolytic virotherapy.

End-use Insights

Biopharmaceutical companies led the market with a revenue share of over 50.0% in 2023. This is attributed to the increasing global prevalence of different types of cancers owing to various hereditary, environmental, and lifestyle risk factors. Moreover, the market is driven by increasing adoption of elemental gene therapy options by biopharmaceutical giants to design cancer therapeutic regimes. Many novel therapeutic drugs are under different phases of trials and firms are striving to market them in different regions across the globe. For instance, in January 2020, bluebird bio, Inc. launches its drug, ZYNTEGLO in Germany to be used as a one-time gene therapy solution for patients aged 12 years and above.

The biopharmaceutical companies segment is projected to grow at the fastest rate over the forecast period. The increasing global prevalence of malignant tumors is a key factor driving the market. Moreover, an increased interest in oncology therapeutics research and development is resulting in a rise in the number of FDA approvals of gene therapy drugs. For instance, there are 6 FDA-approved cancer gene therapy drugs with Tecratus, Abcema, and Kymriah being the recent approvals.

Recent Developments:

Some of the prominent players in the Cancer gene therapy market include:

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2023 to 2033. For this study, Nova one advisor, Inc. has segmented the global cancer gene therapy market.

Therapy

End-use

By Region

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Excerpt from:
Cancer Gene Therapy Industry is Rising Rapidly Up to USD 18.11 Bn by 2033 - BioSpace

Recommendation and review posted by Bethany Smith

-First efficacy signals demonstrated for a gene therapy under development for Oculopharyngeal Muscular Dystrophy (OPMD) which affects ~15,000 patients worldwide-

- BB-301 facilitated improvements across multiple measures of swallowing function in the first Phase 1b/2a clinical study subject as compared to pretreatment assessments conducted during the observational natural history portion of the study-

-Virtual R&D Day being held today at 9:00 am EDT, details below-

HAYWARD, Calif., April 18, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (Benitec or Company), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary Silence and Replace DNA-directed RNA interference (ddRNAi) platform, today announces positive interim clinical data from the 90-day timepoint following the administration of BB-301 to the studys first subject (Subject 1) treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).

To date, no clinical studies have systematically demonstrated a clinical improvement in OPMD patients across both objective and subjective measures of swallowing. We are, therefore, pleased to report positive interim clinical data from multiple radiographic measures as well as subject-reported outcome measures from the first subject treated with BB-301, said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. We are highly encouraged by these early clinical trial results and for the hope that they may offer to patients and caregivers, and we look forward to reporting additional results and continuing to treat patients as they enter the dosing portion of the study from the Natural History observational lead-in period.

BB-301 Interim Clinical Study Results:

During the OPMD Natural History Study, which represents the pre-dose observational period for each subject, Subject 1 experienced progressive worsening of dysphagia as demonstrated by the results of the videofluoroscopic swallowing studies (VFSS), the cold water timed drinking test, and the key subject-reported outcome measure (the Sydney Swallow Questionnaire). Videofluoroscopic swallowing studies represent the gold standard analytical method for the quantitative assessment of dysphagia (swallowing difficulty) in the clinical setting.

At the 90-day timepoint following the administration of BB-301, Subject 1 demonstrated improvements in key videofluoroscopic assessments which correlated with the observation of similar improvement in the key subject-reported outcome measure as compared to the average values for the respective assessments completed during the pre-dose observational period (as summarized in Figure 1). Notably, the results of many assessments completed at the 90-day timepoint demonstrated improvements over the initial measurements assessed at the subjects first visit for the natural history observational study which occurred more than 12 months prior to the 90-day assessment.

The most significant VFSS improvements at Day 90 were observed for swallowing tasks centered on the evaluation of pharyngeal constrictor muscle function and swallowing efficiency in the context of the consumption of thin liquids, solid foods and thick, non-solid foods (e.g., yogurt or pudding) (Figure 1). The VFSS improvements correlated with an improvement in the key subject-reported outcome measure the Sydney Swallow Questionnaire, indicating an improvement in swallowing function as reported by Subject 1 (Figure 1).

Figure 1: Improvement in All Outcomes at 90-Days Post-BB-301 Injection*

*Company data on file

Regarding the BB-301 safety profile observed to date, no Serious Adverse Events have been observed for the two subjects that have received BB-301. Transient Grade 2 Gastroesophageal Reflux Disease or GERD (i.e., acid reflux or heartburn) was observed for the two subjects that received BB-301. For both subjects, the GERD resolved following the completion of a short course of common prescription medications approved for the treatment of GERD.

OPMD is a rare progressive muscle-wasting disease caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, for which there is currently no effective drug therapy. The disease is characterized by swallowing difficulties (dysphagia), limb weakness and eyelid drooping (ptosis). Dysphagia worsens over time and can lead to chronic choking, regurgitation, aspiration pneumonia, and in severe cases, death. Available clinical and surgical interventions are limited in scope and effectiveness and do not address the underlying progressive muscle weakness.

Virtual R&D Event Information: This live virtual R&D Event, featuring two OPMD key opinion leaders, will be held at 9:00 AM EDT today, April 18th, 2024 and can be accessed here. The event replay will be placed on the News & Events tab on the Investor page of the Benitec website.

About BB-301

BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1. The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein.

About Benitec Biopharma, Inc.

Benitec Biopharma Inc. (Benitec or the Company) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary Silence and Replace DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitecs website at http://www.benitec.com.

Forward Looking StatementsExcept for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitecs plans to develop and potentially commercialize its product candidates, the timing of completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitecs product candidates, the intellectual property position, and other forward-looking statements.

These forward-looking statements are based on the Companys current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Companys ability to protect and enforce its patents and other intellectual property rights; the Companys dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Companys products and the products of the Companys collaboration partners; the acceptance of the Companys products and the products of the Companys collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Companys ability to satisfy its capital needs through increasing its revenue and obtaining additional financing, given market conditions and other factors, including our capital structure; our ability to continue as a going concern; the length of time over which the Company expects its cash and cash equivalents to be sufficient to execute on its business plan; the impact of the COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus and similar events, which may adversely impact the Companys business and pre-clinical and clinical trials; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Companys reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements.

Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 ikoffler@lifesciadvisors.com

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/a47d2f41-3feb-49a7-a58f-62e5b0dd4332

Originally posted here:
Benitec Biopharma Reports Positive Interim Clinical Trial Data for First OPMD Subject Treated with BB-301 in Phase 1b ... - GlobeNewswire

Recommendation and review posted by Bethany Smith

Scientists say they have sufficient genetic material to recover the northern white rhino. But that does not mean it will be an easy process.

Just two northern white rhinos remain on the planet. Both of them are female, named Najin and her daughter Fatu. The last male, Sudan, died in 2018.

Researchers at the San Diego Zoo Wildlife Alliance in California examined skin cells extracted from 12 distinct northern white rhinos preserved in their Frozen Zoo. Using a computer model, they simulated the outcome of utilizing the genetic material from these rhinos to produce sperm and egg cells. What next? The cells, transformed into embryos, could be carried by a closely related subspecies, the southern white rhino (Ceratotherium simum simum).

Read more from the original source:
Frozen skin cells could save the Northern White Rhino, say researchers - Interesting Engineering

Recommendation and review posted by Bethany Smith

Generative A.I. technologies can write poetry and computer programs or create images of teddy bears and videos of cartoon characters that look like something from a Hollywood movie.

Now, new A.I. technology is generating blueprints for microscopic biological mechanisms that can edit your DNA, pointing to a future when scientists can battle illness and diseases with even greater precision and speed than they can today.

Described in a research paper published on Monday by a Berkeley, Calif., startup called Profluent, the technology is based on the same methods that drive ChatGPT, the online chatbot that launched the A.I. boom after its release in 2022. The company is expected to present the paper next month at the annual meeting of the American Society of Gene and Cell Therapy.

Much as ChatGPT learns to generate language by analyzing Wikipedia articles, books and chat logs, Profluents technology creates new gene editors after analyzing enormous amounts of biological data, including microscopic mechanisms that scientists already use to edit human DNA.

These gene editors are based on Nobel Prize-winning methods involving biological mechanisms called CRISPR. Technology based on CRISPR is already changing how scientists study and fight illness and disease, providing a way of altering genes that cause hereditary conditions, such as sickle cell anemia and blindness.

Originally posted here:
Generative A.I. Arrives in the Gene Editing World of CRISPR - The New York Times

Recommendation and review posted by Bethany Smith

The ambitious idea of using CRISPR to cure genetic diseases before birth is one step closer to reality. Scientists reported on Monday that they used a form of the technology known as base editing to alter the DNA of laboratory monkeys in the womb, substantially reducing the levels of a toxic protein that causes a fatal liver disease before the animals had even been born.

The research, by a team at the University of Pennsylvania and the Childrens Hospital of Philadelphia (CHOP), will be presented next month at the annual meeting of the American Society of Gene and Cell Therapy, potentially paving the way for human trials.

But arguably the bigger deal, said study co-leader William Peranteau, is that CRISPR base-editing machinery, packaged in lipid nanoparticles, made it into a number of organs beyond the liver, including the heart, kidney, diaphragm, and skeletal muscles. We were surprised to see that we were able to achieve moderate editing in some of these organs, which traditionally have been more difficult to access.

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In a scientific first, researchers use CRISPR base editing to treat liver disease in fetal monkeys - STAT

Recommendation and review posted by Bethany Smith

This Springer Nature service is currently unavailable. This could be due to abnormal network traffic or problems with our systems. We are working to resolve the issue.

Error details: 503 Backend is unhealthy Server: cache-lga13620-LGA

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Ultrasensitive single-step CRISPR detection of monkeypox virus in minutes with a vest-pocket diagnostic device - Nature.com

Recommendation and review posted by Bethany Smith

ZUG, Switzerland and BOSTON, April 22, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced an oral presentation highlighting the Company's lipid nanoparticle (LNP) approach for ocular editing will be presented at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place May 7 11, 2024, in Baltimore, MD and virtually.

The abstract describes our proprietary capabilities to deliver to and edit genes in the eye, opening a potential new focus area. Multiple LNPs as well as modified gRNAs and mRNAs were screened to achieve maximal editing in vivo. These optimized components have been applied to target myocilin (MYOC). Mutations of MYOC in trabecular meshwork cells have been linked to severe glaucomatous conditions. In human primary trabecular meshwork cells, up to 95% MYOC editing and 85% protein knockdown were seen. This novel approach aims to facilitate glaucoma treatment using transient expression of editing machinery targeting MYOC.

Title: Development of an In Vivo Non-Viral Ocular Editing Platform and Application to Potential Treatments for Glaucoma Session Type: In-Person Oral Presentation Session Title: Ophthalmic and Auditory: Delivery Innovations Abstract Number:87 Location: Room 318 323 Session Date and Time: Wednesday, May 8, 2024, 1:30 p.m. 3:15 p.m. ET

The accepted abstract is available online on the ASGCT website. The data are embargoed until 6:00 a.m. ET on the presentation day, Wednesday May 8, 2024. A copy of the presentation will be available at http://www.crisprtx.com once the presentation concludes.

About CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics has transformed from a research-stage company advancing programs in the field of gene editing, to a company that recently celebrated the historic approval of the first-ever CRISPR-based therapy and has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY (exagamglogene autotemcel) was approved in some countries to treat eligible patients with either of those conditions. The Nobel Prize-winning CRISPR science has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer and Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. To learn more, visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) its ongoing and/or planned preclinical studies, clinical trials and pipeline products and programs, including, without limitation, the status of such studies and trials, potential expansion into new indications and expectations regarding data generally (including expected timing of data releases) as well as the data in the above-described abstract and any associated poster and the data that is being presented as described above; (ii) the safety, efficacy and clinical progress of its various clinical and preclinical programs including the program described in the oral presentation and poster; (iii) the data that will be generated by ongoing and planned preclinical studies and/or clinical trials, and the ability to use that data for the design and initiation of further preclinical studies and/or clinical trials; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. AlthoughCRISPR Therapeuticsbelieves that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the efficacy and safety results from ongoing pre-clinical studies and/or clinical trials will not continue or be repeated in ongoing or planned pre-clinical studies and/or clinical trials or may not support regulatory submissions;pre-clinical study and/or clinical trial results may not be favorable or support further development; one or more of its product candidate programs will not proceed as planned for technical, scientific or commercial reasons; future competitive or other market factors may adversely affect the commercial potential for its product candidates; uncertainties inherent in the initiation and completion of preclinical studies for its product candidates and whether results from such studies will be predictive of future results of future studies or clinical trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for its technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K and in any other subsequent filings made byCRISPR Therapeuticswith theU.S. Securities and Exchange Commission, which are available on theSEC'swebsite atwww.sec.gov.CRISPR Therapeuticsdisclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact: Susan Kim +1-617-307-7503 susan.kim@crisprtx.com

Media Contact: Rachel Eides +1-617-315-4493 rachel.eides@crisprtx.com

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CRISPR Therapeutics to Present Oral Presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 ... - GlobeNewswire

Recommendation and review posted by Bethany Smith

CRISPR technologys initial applications were primarily focused on targeted genome editing, but its use has expanded into cell line engineering, which holds promises of groundbreaking advancements, insists Promega'sPhilip Hargreaves

CRISPR technology has come a long way since its discovery as a natural defence mechanism in bacteria. Scientists have harnessed its potential to precisely edit genes, paving the way for groundbreaking advancements in genetic engineering. Initially, CRISPR was primarily used for editing specific genes within organisms, but its scope has expanded rapidly to include the manipulation of entire cell lines. This ability to edit cell lines using CRISPR technology has unlocked a myriad of possibilities.

For many years researchers and drug developers have used immortalised cell lines (derived from humans but engineered to have stable properties over many divisions) to aid their work. These cell lines typically have the gene and/or protein target of interest expressed within them. CRISPR methodology has allowed quicker and more optimal manipulation of these cell lines.

As of now, CRISPR cell line technology is being used extensively for medical and agricultural applications. In medicine, researchers are employing CRISPR to engineer cell lines for therapeutic purposes, intending to treat diseases at the genetic level. For instance, CRISPR has been used to edit the genes of immune cells, enabling them to better target and eliminate cancer cells.

The Future Landscape

Looking ahead, the future of CRISPR cell line technology holds even more transformative possibilities. One of the key areas of exploration is regenerative medicine, where researchers aim to harness CRISPR to engineer cells for tissue repair and organ regeneration. The ability to precisely edit and manipulate cell lines could open new avenues for treating degenerative diseases and injuries, potentially revolutionising the field of transplantation.

The ability to precisely edit and manipulate cell lines could open new avenues for treating degenerative diseases and injuries, potentially revolutionising the field of transplantation

CRISPR technology is further poised to play a crucial role in the development of novel therapies for genetic disorders. The ability to edit problematic genes could pave the way for more effective treatments and even cures for conditions that were once considered incurable, such as Alzheimers.

Despite the remarkable progress in CRISPR technology, challenges and roadblocks still need to be addressed. Off-target effects, unintended mutations, and the potential for unpredictable consequences remain significant hurdles in developing and applying CRISPR-based therapies. Researchers are actively working to enhance the precision and safety of CRISPR technology to mitigate these risks.

CRISPR-engineered cell lines

One way to use CRISPR engineering to generate cell lines, which Promega is successfully offering, is to incorporate HiBiT technology. This 11 amino acid peptide can be fused to a target protein and serves as a luminescent tag. HiBiT can be integrated by knocking in the tag to the endogenous locus of the target using CRISPR gene editing, to help create a truer picture of protein behaviour and regulation in their natural cellular environment. HiBiT has a dynamic range spanning nine logs, which allows for the detection of extremely small quantities of tagged proteins.

In terms of applications, HiBiT's versatility is unmatched, as quantitative assays can be performed in both endpoint and live-cell formats, without the need for target-specific antibodies. From measuring target protein abundance to studying targeted protein degradation, protein secretion and receptor recycling, HiBiT opens up a myriad of possibilities in biomedical research. Its role in drug discovery is particularly noteworthy, enabling more precise and efficient screening of drug effects on cellular proteins.

The accessibility and affordability of CRISPR technology also need to be addressed to ensure that its benefits are not confined to a privileged few. Wide-scale adoption and integration of CRISPR cell line technology into various sectors will require collaborative efforts from researchers, policymakers, and the private sector. To aid in this endeavour, Promega now offers a comprehensive selection of pre-built CRISPR-edited cell line pools and clones, including HiBiT fusions.

Wide-scale adoption and integration of CRISPR cell line technology into various sectors will require collaborative efforts from researchers, policymakers, and the private sector

This development opens doors for researchers and developers as it reduces the costs involved in developing cell lines from scratch. Not only will this save budgets, but it also saves time. This means drug development times can be reduced by as much as 12 months, leading to vital medications being available much sooner.

The future of CRISPR cell line technology is undoubtedly exciting and promises transformative advancements in medicine, agriculture, and beyond. As scientists continue to unravel the mysteries of genetic engineering, there is a delicate balance between innovation and ethical stewardship to consider. But with efforts to maximise the technologys potential to make it more accessible and affordable, the future is bright.

Philip Hargreaves Ph.D, is director of strategic marketing & business development at Promega

Pic: Brano

More here:
The evolution of CRISPR cell line technology - Lab News

Recommendation and review posted by Bethany Smith

SNIPR Biome

SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical trials in haematological cancer patients

Phase 1b/2a trial will evaluate SNIPR001 for the prevention of E.coli infections in patients undergoing hematopoietic stem cell transplantation

Copenhagen, April 22 2024: SNIPR Biome ApS (SNIPR), the company pioneering the development of precision medicines using CRISPR technology for microbial gene therapy, announces today that it has received $5.48 million from Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) to co-fund a Phase 1b/2a clinical trial in hematological cancer patients.

The trial will evaluate SNIPR001, the first CRISPR-armed phage therapeutic that specifically targets E. coli in the gut, for the prevention of E. coli bloodstream infections in hematological cancer patients who are undergoing hematopoietic stem-cell transplantation (HSCT) and are colonized with Fluoroquinolone Resistant (FQR) E. coli. Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.

Despite the significant advances in hematologic cancer therapy over the past decade, infectious complications, and antimicrobial resistance (AMR) continue to pose significant threats to patients and clinical outcomes1. Currently, there are no approved therapies for the prevention of bloodstream infections (BSIs) in hematological cancer patients. SNIPR Biome is developing SNIPR001 to address this urgent unmet need to combat infections in hematological cancer patients.

Preclinical data published in Nature Biotechnology described SNIPR001s ability to selectively target and remove antibiotic-resistant E. coli strains in the gut, potentially offering a safe treatment which preserves the rest of the gut microbiome. This was supported by interim Phase 1 data published in 2023, which showed that oral dosing of SNIPR001 over seven days across three dosing levels in 24 healthy individuals was well tolerated. Furthermore, SNIPR001 could be recovered in faeces from treated individuals in a dose-dependent manner, and treatment with SNIPR001 numerically lowered gut E. coli levels.

Anticipated to begin later this year, the randomized, double-blinded Phase 1b/2a trial will investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of orally administrated SNIPR001 in 24 patients. It will be conducted at up to 10 sites across Europe and the United States.

CARB-X, a global non-profit partnership dedicated to supporting early-stage antibacterial research and development to address the rising threat of drug-resistant bacteria, has been a long-term collaborator with SNIPR in this field. The funding announced today enables SNIPR to move SNIPR001 into Phase 1b/2a clinical trials and will serve as a cornerstone for a further significant fundraise to enable the Company to continue development of its pipeline of CRISPR-based AMR and gut-directed gene therapies.

Story continues

Dr Christian Grndahl, Co-founder and CEO of SNIPR Biome, commented: Antibiotic resistance is one of healthcares biggest problems today, affecting treatment efficacy and survival among patients who are often already very sick. We are using our knowledge of gene editing and synthetic biology to create highly specific, designer bacteria and phage to disrupt, edit or add genes, and deliver these precision medicines in a carefully targeted way. We are pleased to be continuing our partnership with CARB-X who share our commitment to developing therapies for vulnerable patients.

Erin Duffy PhD, Chief of Research & Development, CARB-X, said: Having underscored safety for SNIPR001 in healthy subjects, SNIPR Biome is now focusing on demonstrating proof-of-mechanism for this novel product, with our support.We are keen to establish a link between gut decolonization and prevention of infection as a novel approach to antimicrobial resistance, and SNIPR001 offers the possibility of doing so.

CARB-X funding for this research is supported by the Biomedical Advanced Research and Development Authority under agreement number: 75A50122C00028, and by awards from Wellcome (WT224842), and Germanys Federal Ministry of Education and Research (BMBF). The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders.

Ends

About SNIPR001 SNIPR001, a CRISPR-armed phage therapeutic that specifically targets E. coli in the gut, is designed to prevent infections from spreading into the bloodstream and represents a promising advancement against antibiotic-resistant pathogens. The pre-clinical studies of SNIPR001 published in Nature Biotechnology2 demonstrated the products activity against multi-drug resistant strains of E. coli and its specificity towards E. coli with no off-target effects toward any of the tested non-E. coli strains. SNIPR successfully completed a Phase 1 trial in the US, also funded by CARB-X, demonstrating safety of SNIPR001 and target engagement with E. coli in the gut of healthy subjects without disturbing the overall gut microbiome (NCT05277350), supporting its potential as a safe and effective preventative therapy for bloodstream infections in hematological cancer patients. SNIPR001 has been granted a Fast-Track designation for the indication Prophylaxis of bloodstream E. coli infections in patients with hematological malignancy at risk of neutropenia from the US Food and Drug Administration (FDA). SNIPR001 is also being developed to directly treat active E. coli infections.

About SNIPR BIOME SNIPR Biome is a Danish clinical-stage biotech company pioneering the development of precision medicines using CRISPR technology for microbial gene therapy. We are pioneering a novel use of CRISPR/Cas technology to better treat and prevent human diseases through precision killing of bacteria or gene modification. SNIPR Biome was the first company to orally dose humans with a CRISPR therapeutic and the first company to have been granted US and European patents for the use of CRISPR for targeting microbiomes. SNIPR technology is used in collaborations with Novo Nordisk A/S, CARB-X, SPRIN-D, and MD Anderson Cancer Center. For more information, visit http://www.sniprbiome.com and follow us on LinkedIn and X.

About CARB-X

CARB-X (Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator) is a global non-profit partnership dedicated to supporting early-stage antibacterial research and development to address the rising threat of drug-resistant bacteria. CARB-X supports innovative therapeutics, preventatives and rapid diagnostics. CARB-X is led by Boston University and funded by a consortium of governments and foundations. CARB-X funds only projects that target drug-resistant bacteria highlighted on the CDCs Antibiotic Resistant Threats list, or the Priority Bacterial Pathogens list published by the WHO, with a priority on those pathogens deemed Serious or Urgent on the CDC list or Critical or High on the WHO list. https://carb-x.org/ | X (formerly Twitter) @CARB_X

Contact ICR Consilium Tracy Cheung, Chris Welsh, Davide Salvi SNIPR@consilium-comms.com

SNIPR Biome Dr Christian Grndahl, Co-founder and CEO contact@sniprbiome.com http://www.sniprbiome.com

1 So M. Determining the Optimal Use of Antibiotics in Hematopoietic Stem Cell Transplant Recipients. JAMA Netw Open. 2023 Jun 1;6(6):e2317101 2 Gencay, Y.E., Jasinskyt, D., Robert, C. et al. Engineered phage with antibacterial CRISPRCas selectively reduce E. coli burden in mice. Nat Biotechnol (2023). https://doi.org/10.1038/s41587-023-01759-y

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SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical ... - Yahoo Finance

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Crispr Therapeutics AG (NASDAQ:CRSP)is 2.8% lower at $56.35 this afternoon, continuing a pullback from a Feb. 22, more than two-year high of $91.10. Over the last month, CRSP has erased 20.8% and now sports a 9.6% year-to-date deficit.

For those looking to buy in on the dip, however, the recent pullback puts Crispr Therapeutics stock within one standard deviation of its 320-day moving average, a trendline with historically bullish implications. According to Schaeffer's Senior Quantitative Analyst Rocky White, the equity saw two similar signals in the past three years, after which it was higher one month later each time, averaging an impressive 13.3% gain. A move of similar magnitude would put the shares at roughly $63.85.

Crispr Therapeutics stock's 14-day relative strength index (RSI) of 18.2 is deep in "oversold" territory, which is typically indicative of a short-term bounce. Plus, short interest represents 17.6% of the stock's available float, and would take eight days to cover at CRSP's average pace of trading.

Plus, itsSchaeffer's Volatility Scorecard(SVS) stands at a high 86 out of 100, indicating the stock exceededoptiontraders' volatility expectations in the past 12 month -- a boon for premium buyers.

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Researchers have engineered a Cas enzyme to enhance activity against RNA viruses, resulting in a system that completely blocked the replication of various SARS-CoV-2 strains.

The research, details of which were published in Cell Discovery, was prompted by the need to overcome a barrier to the use of the Cas13d enzyme as an antiviral against human RNA viruses. Studies have shown that CRISPR/Cas13 systems are programmable tools for manipulating RNAs and Cas13d is the most active subtype of the enzyme in mammalian cells, making it the most promising antiviral candidate.

However, the activity of Cas13d is largely restricted to the nucleus. Most RNA viruses only replicate in the cytosol, where Cas13d is barely active in mammalian cells and as such are protected from the antiviral effects enabled by the enzyme, wrote the study's authors, led by Christoph Gruber of the Technical University of Munich (Cell Discov, April 12, 2024, Vol. 10 [1], pp. 1-4).

The problem led Gruber and other scientists from Helmholtz Munich and the Technical University of Munich to investigate why Cas13d is largely restricted to the nucleus and explore ways to bring the enzyme into contact with replicating RNA viruses in mammalian cells.

The research revealed that the enzyme has little activity in the cytosol because the RNA that guides the CRISPR-Cas complex to specific target sequences is found in the nucleus. That finding led the scientists to explore ways to move CRISPR RNAs (crRNAs) from the nucleus to the cytosol.

Through screening and optimization of various designs of shuttling proteins, the researchers developed a system that transfers nuclear crRNAs into the cytosol. The enzyme -- nucleocytoplasmic shuttling Cas13d or Cas13d-NCS for short -- moves nuclear crRNAs to the cytosol, where the protein/crRNA complex binds and degrades complementary target RNAs.

The researchers hypothesized that Cas13d-NCS more adeptly degrades viral cytosolic RNAs than standard Cas13d and tested that idea using a self-replicating RNA from the Venezuelan equine encephalitis RNA virus. The tests showed the engineered approach "targets solely cytosolic RNA with greater efficiency compared to the current Cas13d system," the authors wrote.

To assess antiviral efficacy, the scientists targeted SARS-CoV-2, the RNA virus that causes COVID-19. The assessment showed Cas13d-NCS can completely block the replication of various SARS-CoV-2 strains, they wrote.

"Targeting conserved but weakly expressed viral-coding sequences resulted in relatively weak inhibition, whereas targeting the ubiquitous 3'UTR with a single crRNA resulted in complete inhibition of viral replication," Gruber et al concluded.

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Engineered Cas clears barrier to antiviral CRISPR therapies - LabPulse

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A formidable collaboration between three University of California (UC) schools and leading global life sciences and diagnostics innovator the Danaher Corporation heralds a new era in the fight against rare and deadly genetic diseases, such as sickle cell disease which predominantly impacts Black and Hispanic populations in the U.S. through the innovative use of CRISPR technology.

Spearheaded by the Innovative Genomics Institute (IGI), this joint effort brings together genetics researchers and clinician experts from UC San Francisco, UC Los Angeles, UC Berkeley, and other research institutions, to expedite the development of curative therapies for diseases that have previously lacked effective treatments.

The Danaher-IGI Beacon for CRISPR Cures center will leverage genome editing technology to research a wide range of genetic disorders. The center, which will be led out of the IGI headquarters at UC Berkeley, combines expertise in genetics research, clinical practice, and industry resources to accelerate the development and deployment of CRISPR-based treatments. The goal is to establish new standards for safety and efficacy while streamlining the path from preclinical research to clinical trials.

The unique nature of CRISPR makes it ideal for developing and deploying a platform capability for CRISPR cures on demand, said Fyodor Urnov, PhD, IGIs Director of Technology and Translation, in a press release. Danaher and the IGI are in a unique position to potentially create a first-of-its-kind CRISPR cures cookbook that could be used by any team wishing to take on other diseases.

The centers initial focus will be on hemophagocytic lymphohistiocytosis (HLH) and Artemis-deficient severe combined immunodeficiency (ART-SCID), two conditions characterized by defects in a patients immune system. Traditional treatments for these disorders, such as bone marrow transplants, often fall short due to complications.

By targeting specific gene mutations associated with these diseases, researchers hope to develop therapies that address their underlying causes, improve outcomes, and enhance quality of life for those affected.

Using CRISPR, the IGI has already made incredible advancements in treating sickle cell disease through clinical trials at the Comprehensive Sickle Cell Center at UC San Francisco Benioff Childrens Hospital in Oakland which was established to address racial biases in health care. In 2021, the center received $17 million in funding to advance the use of CRISPR in sickle cell research.

This therapy has the potential to transform sickle cell disease care, said Mark Walters, MD, a pediatric professor at UC San Francisco and principal investigator of the clinical trials. If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.

Since then, researchers have been testing the possibility of replacing the gene that causes sickle cell with a healthy one manufactured using a patients own stem cells. Early tests have been positive, indicating a potential cure for the disease.

With CRISPR, we can speed up the development of improved therapies that can reach all the patients who need them, said Jennifer Puck, MD, a faculty member at the Jeffrey Modell Diagnostic Center for Primary Immunodeficiencies and Institute for Human Genetics, both at UC San Francisco. All patients deserve a sense of urgency. including those with rare diseases, many of whom are children.

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Sixty years ago this month, on April 22, 1964, the New York Worlds Fair opened in Flushing MeadowsCorona Park in Queens, New York. The Fair had the theme of Peace Through Understanding, and was dedicated to Mans Achievement on a Shrinking Globe in an Expanding Universe, as symbolized by the 140-foot-tall stainless-steel globe, known as the Unisphere, that towered over a massive reflecting pool.

The 1964 Worlds Fair wasnt the first one held in Flushing Meadows; the Unisphere was built on the same ground once occupied by the similarly spherical Perisphere, which was constructed for the 1939 Worlds Fair. To kick off the 1964 Fair, President Lyndon B. Johnson delivered opening remarks that evoked the 39 edition, which imagined the 1960s of the future:

But Johnsons reflections on human progress werent all positive. No one prophesied that half the world would be devastated by war, or that millions of helpless would be slaughtered, the President noted, just months after he approved the controversial National Security Action Memorandum 288 escalating the U.S.s involvement in the Vietnam War. No one foresaw power that was capable of destroying man, or a cold war which could bring conflict to every continent.

Overhead view of the 1964 Worlds Fair, showing the Unisphere and surrounding pavilions in Flushing Meadows-Corona Park in Queens, New York.

Johnsons presence at the 1964 Worlds Fair underscored the somber reality facing America at the time. After all, an assassins bullet killed the intended speaker, his predecessor, John F. Kennedy, thrusting hima representative of Washington D.C.s old guardinto the role intended for JFK, a symbol of the New Frontier.

So, who could Fair attendees turn to for hopeful visions of Peace Through Understanding, and Mans Achievement on a Shrinking Globe? Only one man: Walt Disney.

Disney, who had been a weekly presence in American homes for the last decade through his television show, Walt Disneys Disneylandlater titled Walt Disney Presentscreated four attractions for the 64 Worlds Fair. These exhibits were a hit, drawing 135,000 visitors per day during the first season alone, according to The Walt Disney Family Museum.

Walt Disney at the 1964 New York Worlds Fair

Visitors line up to enter Its A Small World at the Worlds Fair in 1965.

Disneys attractions included Great Moments with Mr. Lincoln at the Illinois Pavilion, an animatronic replica of another President who represented hope, and who was also assassinated; Fords Magic Skyway for the Ford Motor Company, where guests rode in Ford vehicles past animatronic dinosaurs; and the now-iconic its a small world, which Disneys Imagineers built in collaboration with Pepsi-Cola as a tribute to UNICEF.

But it was the Carousel of Progress, located in General Electrics Progressland pavilion, that most epitomized Walt Disneys vision of the future. The rotating animatronic show guided audiences through the history of human innovation and provided a comforting escape with its optimistic theme song by the Sherman Brothers, promising, Theres a great, big, beautiful tomorrow/Shining at the end of every day.

In the aftermath of a national tragedy, Walt Disney became a beacon of optimism for fairgoers, and for Americans. His presence fostered a belief in a great big beautiful tomorrow.

Perhaps its that enduring optimism that fuels the persistent conspiracy that Disney, who died in 1966, might still be among us, secretly preserved in hopes of one day being revived.

In 1964, besides contributing to the Worlds Fair, Disney also released his most acclaimed live-action film yet: Mary Poppins. Just a few years earlier, during a screening of To Kill a Mockingbird, Disney had reportedly lamented, Thats the kind of film Id like to make, but I cant. Now, his tender musical adaptation of P.L. Travers novel had achieved the same milestone as Universals adaptation of Harper Lees seminal work: a nomination for Best Picture at the Academy Awards.

Poster for Mary Poppins, touting the film as "Walt Disney's Greatest Achievement."

With his status finally solidified in the entertainment industry, Disney turned his sights to the futurenot of his film studio, but of humanity. Intent on using his renowned imagination to forge a brighter tomorrow, Disney envisioned a utopia designed to last forever. In 1964, the future looked bright for Walt Disney.

In less than three years, he would be dead.

On December 15, 1966, Walt Disney died as a result of complications from lung cancer. As Biography notes, a private funeral was held the next day, and on December 17, his body was cremated and interred at Forest Lawn Memorial Park in Glendale, California.

Some conspiracy theorists, however, believe that Disneys remains arent actually at Forest Lawn. Theyll tell you, according to Biography, that Disneys body is instead suspended in a frozen state and buried deep beneath the Pirates of the Caribbean ride at Disneyland in Anaheim, California, awaiting the day when medical technology would be advanced enough to reanimate the animator.

The rumored cryonic freezing of Walt Disney has no clear origin point that Biography could confirm. But its first documented mention is in a 1969 Ici Paris article, reportedly as a prank concocted by disgruntled animators who once worked for Disney seeking to have a laugh at their late taskmasker employers expense. Their motive was seemingly revenge for Disneys strict oversight, an aftermath of a labor uprising in the late 1930s that is chronicled in depth in The Disney Revolt: The Great Labor War of Animations Golden Age.

Initially, whispers suggested Disneys entire body was preserved in a secret facility, but soon the tale focused on the animators head alone, frozen beneath iconic Disneyland attractions like Pirates of the Caribbean, the Matterhorn, the Partners statue at the center of the park, and even the Magic Kingdom castle. Over time, it seems every corner of Disneyland has been rumored to shelter its founders frozen head.

The notion of Walt Disneys icy remains hiding within park attractions might stem from the actualsecrets of Disneyland. There is indeed a hidden space at the top of the Matterhorn, but its home to a basketball court for bored Disney staff, not a frozen former CEO. And while early versions of Pirates of the Caribbean did feature real skeletons from UCLAs medical school, according to SFGate, none belonged to Disney himself.

The grim idea that only Walt Disneys head was placed in cryostasis might have caught on due to its eerie, sci-fi feel, with modern cryonics offering both full-body and head-only preservation options. That singular detail adds a creepy wrinkle to the conspiracy, evoking images more akin to the 1962 horror flick The Brain That Wouldnt Die than the reality of a Hollywood moguls legacy.

Nevertheless, the tale of Walt Disneys frozen head persisted, specifically resurfacing in two biographies released years after his death: Leonard Moselys 1986 Disneys World and Marc Eliots 1993 Walt Disney: Hollywoods Dark Prince, which further embedded the legend into popular culture.

Eliots controversial biography, which was criticized by Disneys family and historians alike for its speculative content, included unfounded allegations against Disney, including claims that he was an FBI informant (which evidence suggests he was not), and that he refused to have flags at half mast at Disneyland when JFK died (which photographic evidence disproves). Nevertheless, Hollywoods Dark Prince fed into a desire to find the dark side of a man often propped up as the symbol of Americana, pushing both the cryonics rumor and the assertions of Disneys rampant antisemitism (also notably debunked) into the mainstream.

Disneys family has firmly denied the rumor that he was cryogenically frozen, and as Biography points out, it has been further discredited by those pointing to the existence of signed legal documents that indicate Disney was in fact cremated and that his remains are interred in a marked plot (for which his estate paid $40,000) at Forest Lawn, the exact location of which is a matter of public record. Plus, the first instance of a person being cryopreserved after his death, James Bedford, didnt occur until nearly a month after Disneys cremation, debunking the timeline of the rumor that Disney was frozen.

Walt Disneys grave site at Forest Lawn Memorial Park in Glendale, California.

Diane Disney, Walts daughter, wrote in a 1972 biography about her famous father that she doubted her father had even heard of cryonics.

Nonetheless, even skeptics who reject the frozen head story might concede that Walt Disney, a famously forward-thinking futurist, could have been aware of cryonics. The concept gained attention in 1964, the same year Disney shifted his focus from film to envisioning his utopian future.

If you were browsing the New Releases shelf at a bookstore in 1964, you might stumble upon an intriguing non-fiction book in between copies of Ernest Hemingways A Moveable Feast and the Warren Commissions The Warren Report: Robert Ettingers The Prospect of Immortality.

Most of us now living have a chance for personal, physical immortality, Ettinger claims in the very first sentence. All you need to do, Ettinger says, is join one established fact with one reasonable assumption.

The fact: At very low temperatures it is possible, right now, to preserve dead people with essentially no deterioration, indefinitely.

The assumption: If civilization endures, medical science should eventually be able to repair almost any damage to the human body, including freezing damage and senile debility or other cause of death.

Six decades later, while we havent mastered the art of repairing all human body damage, our cryopreservation methods have advanced significantly, particularly with the introduction of vitrification by Greg Fahy and William F. Rall in the 1980s. And recent scientific advancements suggest that what we currently understand as death might be more reversible than previously thought.

Some excerpts from Ettingers book resonate with the futuristic optimism of Walt Disneys Tomorrowland. For example, Ettinger writes,If civilization endures ... if the Golden Age materializes, the future will reveal a wonderful world indeed, a vista to excite the mind and thrill the heart. However, even if Disney had encountered Ettingers work, his imagination had already been sparked by another piece of literature before he died.

In May 1960, Horizon magazine published Out of a Fair, a City, an article in which architect Victor Gruen envisioned transforming the 1964 Worlds Fair site into a domed city to test solutions for societal challenges. According to Imagineer Marty Sklars 1999 book, Remembering Walt, Gruens philosophy (further elaborated in Gruens 1964 work, The Heart of our Cities: The Urban Crisis, Diagnosis and Cure,) was a significant influence on Disney during his final yearsso much that he began an ambitious plan to build a community in Florida that would never cease to be a living blueprint of the future.

Disney, utilizing land his corporation discreetly bought in Florida, set out to build an Experimental Prototype Community of Tomorrow adjacent to his planned East Coast theme park. The community aimed to eliminate traffic jams, offer abundant green spaces, and showcase efficient public transportation with the use of a monorail system.

EPCOT world showcase at Walt Disney World Resort, 1982

After Walt Disneys death, the Florida land earmarked for his future city was transformed into EPCOT, the second theme park at Walt Disney World Resort. EPCOT features a Future World section with educational attractions and a World Showcase with international pavilions, operating as a perpetual Worlds Fair.

In his last years, Walt Disney was introspective, focusing not on the prospect of immortality, but on a different sentiment. While the 1964 song Great Big Beautiful Tomorrow planted the seed in millions of young minds, it was another tune by the Sherman Brothers from the same year that resonated deeply with Disney as he reflected on his life. Richard Sherman recalls:

There is little left of the 1964 New York Worlds Fair in Flushing MeadowsCorona Park. The spot where the Carousel of Progress once played in GEs Progressland is now an athletic field. The Vatican pavilion has been replaced by a stone bench. The Unisphere, however, still remains, towering over a park whose occupants have little memory, or even awareness, of the Great Big Beautiful Tomorrow promised at the park 60 years ago.

The Unisphere in Flushing Meadows-Corona Park in March 2024.

For the Baby Boomers who experienced Disneys contributions to the 1964 Worlds Fair, the event offered hope and a last optimistic vision of the future from Uncle Walt. And while today, these visitors can encounter preserved pieces of the Fair at the Queens Museum and experience its a small world at Disney parks worldwide, they cant turn that athletic field back into Progressland. And they cant bring back the man who made it possible.

Walt Disney and Mickey Mouse at the Rose Parade in 1966.

Walt Disneys legacy extends far beyond his films and theme parks. He symbolizes something greater than a sprawling entertainment empire. As biographer Neal Gabler put it in the final pages of Walt Disney: The Triumph of the American Imagination, he demonstrated how one could assert ones will on the world at the very time when everything seemed to be growing beyond control and beyond comprehension.

For conspiracy theorists who want to believe Walt Disney is a frozen head, waiting for revival, perhaps its because they want to believe he asserted his will over the one thing no one has been able to do before. That maybe, if the Golden Age materializes, Walt Disney could even come back to life. And with him would come, once again, the promise of a Great Big Beautiful Tomorrow. A promise he made in Queens 60 years ago.

Michael Natale is the news editor for Best Products, covering a wide range of topics like gifting, lifestyle, pop culture, and more. He has covered pop culture and commerce professionally for over a decade. His past journalistic writing can be found on sites such as Yahoo! and Comic Book Resources, his podcast appearances can be found wherever you get your podcasts, and his fiction cant be found anywhere, because its not particularly good.

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Freezing your body after death with the hope of coming back to life one day sounds like something out of a science fiction movie.

Southern Cryonics, the first body-freezing facility in the Southern Hemisphere, tries to turn this idea into reality.

Were sort of in a race against time, says Southern Cryonics director, Peter Tsolakides, to Neos Kosmos.

Cryonics, coming from the Greek word kros for icy cold, involves the preservation of legally declared dead bodies at extremely low temperatures for potential future revival.

The facility in Holbrook, New South Wales, uses this practice, with the expectation that one day, advancements in medical technology and science will restore patients to health and in the young body.

But the timeframe of this future remains uncertain.

Tsolakides says once you preserve a body, you can keep it (stored) for thousands of years, but the chance of coming back depends on when you freeze it.

A matter of life after death

He says, currently 50 people, are willing to take the risk for a chance at life after death, and the number is growing.

This group consists of 35 investors each contributing $50,000 to $70,000, and 15 subscribers or customers who have paid $150,000 through life insurance.

Tsolakides says there are no guarantees, despite how great the dream of being brought back from the dead might be for some people.

Most of them know something about it (cryonics), but they also look at it and say, look, theres no guarantees, but theres a chance.

And that chance versus being buried in the ground or cremated is a much higher chance coming back.

He estimates the chance of a well-preserved body being revived in 200 years to be around 20%.

He says although its hard to predict what the world will be like in be like in 1,000 years, bodies might be revived when technological advancements have found the key to immortality.

In the real world, nobody will be dying, and most diseases will be cured. So, we will know how to prevent death in a sense, and the next step is to bring back those who have already died, but in a good condition.

Tsolakides says that while they dont know how to bring a person back to life, current developments give you inklings, of what the future is going to be like.

He says progress has to start somewhere, and right now billions are invested in medical research aimed at disease cures.

This includes groups working on brain revival, organ regeneration, cloning, and advancements in artificial intelligence and nanotechnology.

How does cryonics work?

When a person is declared legally dead at the hospital, a cooling process begins.

Chemicals are used to stabilise the body, lowering it to about ice temperature.

Once taken to the funeral home, the body is further cooled and infused with an antifreeze substance until it reaches about -80C.

Next, it goes to the cryonics facility, gradually cooled to -180C and preserved below that temperature, in a large vacuum flask container filled with liquid nitrogen.

Southern Cryonics Greek-Australian director says, theres a brief window of a few hours after legal death, where no deterioration occurs to the body.

Once preserved in liquid nitrogen, it can be stored for thousands of years due to almost no chemical or biological activity at that temperature.

Its a race against time to keep the temperature going down, he says.

But is it possible to freeze a human brain to revive it later?

If you catch them (bodies) under our optimal time, very little damage is occurring to the brain, but that doesnt mean that 200 years from now, that damage cant be repaired, Tsolakides says.

The facility can currently hold up to 40 patients, with each container fitting 4 bodies, but can expand to hold up to six or seven hundred patients if necessary.

The birth and evolution of Southern Cryonics

Tsolakides got interested in cryonics from a young age.

When he came back to Australia around 2012 after working overseas, he saw there were only cryonics facilities in the US and Russia.

He connected with like-minded people and talked about building one in Australia.

We started getting what we call founding members, says Tsolakides, each contributing $50,000 to kickstart the project, eventually totalling 35 members of a non-profit organisation.

We started the facility and that was how it sort of developed.

He says, they chose Holbrook, a small town with about 1,500 people, for a few reasons.

Land there wasnt expensive, and it was halfway between Melbourne and Sydney, making it accessible to over half of Australias population.

Holbrooks nearby Albury airport is crucial for quick patient transportation, and the support from the local council made the decision easier.

Another advantage is its proximity to liquid nitrogen suppliers along the Hume Highway, crucial for the facility.

Holbrooks low history of natural disasters made it a safe choice after a thorough analysis of several years.

The legalities

Tsolakides says Southern Cryonics got all the official approvals from the NSW Department of Health and the local council, to operate as a cemetery but uses a recognised funeral home for mortuary work.

The government groups that we work with helped us a lot. It wasnt like we got resistance or anything like that.

A good idea but not for everyone

Tsolakides was born in Israel to Greek parents.

His mother was from the Greek island of Syros, and his father from Athens.

They briefly lived in Greece before moving to Australia in 1955 when Tsolakides was five years old.

He has a degree in Chemistry and later pursued one in Business Administration.

Throughout his career, he worked primarily in marketing for an oil company.

He grew up and lived in Melbourne for many years before moving to Sydney, a place he now calls home.

His passion in cryonics sparked at about18 after reading Robert Ettingers book, The Prospect of Immortality.

At that age, he didnt worry much about death.

He assumed this will be everywhere, by the time he got old, but soon realised that very few people worldwide were interested in it.

He says that while some are intrigued by cryonics, most view it as a good idea but not for themselves.

Even the US organisation have about five to six thousand members only, with 400 or 500 people suspended, and theyve been going for 50 years.

But that didnt stop him for pursuing his curiosity around cryonics.

Keeping an eye on scientific developments

Tsolakides is determined to improve their techniques and increase success chances, despite challenges or doubts about cryonics.

He says Southern Cryonics along with overseas organisations is monitoring the best way to store a body, leaving the revival work to other scientists.

Its (suspending the body) physically possible to do it now, he says, but of course, you can always improve the processes.

While cryonics remains a controversial field and the chances of revival seem low now, it is yet to be seen whether future technology will ever be able to bring the dead back to life.

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Is it possible to come back from the dead? Australia's first body-freezing facility explores the boundaries of mortality - Neos Kosmos

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Araujo AB, ODonnell AB, Brambilla DJ, Simpson WB, Longcope C, Matsumoto AM, et al. Prevalence and incidence of androgen deficiency in middle-aged and older men: estimates from the Massachusetts Male Aging Study. J Clin Endocrinol Metab. 2004;89:59206. https://doi.org/10.1210/jc.2003-031719

Article CAS PubMed Google Scholar

Mulligan T, Frick MF, Zuraw QC, Stemhagen A, McWhirter C. Prevalence of hypogonadism in males aged at least 45 years: the HIM study. Int J Clin Pract. 2006;60:7629. https://doi.org/10.1111/j.1742-1241.2006.00992.x

Article CAS PubMed Google Scholar

Thirumavalavan N, Scovell JM, Link RE, Lamb DJ, Lipshultz LI. Does solid organ transplantation affect male reproduction? Eur Urol Focus. 2018;4:30710. https://doi.org/10.1016/j.euf.2018.08.012

Article PubMed PubMed Central Google Scholar

Carrero JJ, Qureshi AR, Parini P, Arver S, Lindholm B, Brny P, et al. Low serum testosterone increases mortality risk among male dialysis patients. J Am Soc Nephrol. 2009;20:61320. https://doi.org/10.1681/ASN.2008060664

Article CAS PubMed PubMed Central Google Scholar

Wachterman MW, OHare AM, Rahman O-K, Lorenz KA, Marcantonio ER, Alicante GK, et al. One-year mortality after dialysis initiation among older adults. JAMA Intern Med. 2019;179:98790. https://doi.org/10.1001/jamainternmed.2019.0125

Article PubMed PubMed Central Google Scholar

Kazemeini SM, Mogharabian N, Asadpour A, Naderi G, Kasaeian A, Mousavi A. The effect of renal transplant on hypogonadism and erectile dysfunction due to end-stage renal disease. Saudi J Kidney Dis Transplant. 2021;32:9238. https://doi.org/10.4103/1319-2442.338303

Article Google Scholar

Akbari F, Alavi M, Esteghamati A, Mehrsai A, Djaladat H, Zohrevand R, et al. Effect of renal transplantation on sperm quality and sex hormone levels. BJU Int. 2003;92:2813. https://doi.org/10.1046/j.1464-410x.2003.04323.x

Article CAS PubMed Google Scholar

Reinhardt W, Kbber H, Dolff S, Benson S, Fhrer D, Tan S. Rapid recovery of hypogonadism in male patients with end stage renal disease after renal transplantation. Endocrine. 2018;60:15966. https://doi.org/10.1007/s12020-018-1543-2

Article CAS PubMed Google Scholar

Saha M-T, Saha HHT, Niskanen LK, Salmela KT, Pasternack AI. Time course of serum prolactin and sex hormones following successful renal transplantation. Nephron. 2002;92:7357. https://doi.org/10.1159/000064079

Article CAS PubMed Google Scholar

Shoskes DA, Kerr H, Askar M, Goldfarb DA, Schold J. Low testosterone at time of transplantation is independently associated with poor patient and graft survival in male renal transplant recipients. J Urol. 2014;192:116871. https://doi.org/10.1016/j.juro.2014.03.102

Article CAS PubMed Google Scholar

UNOS Data and Transplant Statistics | Organ Donation Data. UNOS. https://unos.org/data/. Accessed 3 February 2024.

Lincoff AM, Bhasin S, Flevaris P, Mitchell LM, Basaria S, Boden WE, et al. Cardiovascular safety of testosterone-replacement therapy. N Engl J Med. 2023;389:10717. https://doi.org/10.1056/NEJMoa2215025

Article CAS PubMed Google Scholar

Antonucci M, Palermo G, Recupero SM, Bientinesi R, Presicce F, Foschi N, et al. Male sexual dysfunction in patients with chronic end-stage renal insufficiency and in renal transplant recipients. Arch Ital Urol Androl. 2016;87:299305. https://doi.org/10.4081/aiua.2015.4.299

Article PubMed Google Scholar

Miner MM, Khera M, Bhattacharya RK, Blick G, Kushner H. Baseline data from the TRiUS registry: symptoms and comorbidities of testosterone deficiency. Postgrad Med. 2011;123:1727. https://doi.org/10.3810/pgm.2011.05.2280

Article PubMed Google Scholar

Cha J, Han D. Health-related quality of life based on comorbidities among patients with end-stage renal disease. Osong Public Health Res Perspect. 2020;11:194200. https://doi.org/10.24171/j.phrp.2020.11.4.08

Article PubMed PubMed Central Google Scholar

Albaaj F, Sivalingham M, Haynes P, McKinnon G, Foley RN, Waldek S, et al. Prevalence of hypogonadism in male patients with renal failure. Postgrad Med J. 2006;82:6936. https://doi.org/10.1136/pgmj.2006.045963

Article CAS PubMed PubMed Central Google Scholar

Balczar-Hernndez L, Mendoza-Zubieta V, Gonzlez-Virla B, Gonzlez-Garca B, Osorio-Olvera M, Pealoza-Juarez JU, et al. Hypothalamic-pituitary-gonadal axis disturbance and its association with insulin resistance in kidney transplant recipients. J Bras Nefrol. 2023;45:7783. https://doi.org/10.1590/2175-8239-JBN-2021-0250en

Article PubMed Google Scholar

Stenvinkel P. Inflammation in end-stage renal disease: the hidden enemy. Nephrology. 2006;11:3641. https://doi.org/10.1111/j.1440-1797.2006.00541.x

Article PubMed Google Scholar

Wibullaksanakul S, Handelsman DJ. Regulation of hypothalamic gonadotropin-releasing hormone secretion in experimental uremia: in vitro studies. Neuroendocrinology. 1991;54:3538. https://doi.org/10.1159/000125913

Article CAS PubMed Google Scholar

Dong QH, Handelsman DJ. Regulation of pulsatile luteinizing hormone secretion in experimental uremia. Endocrinology. 1991;128:121822. https://doi.org/10.1210/endo-128-3-1218

Article CAS PubMed Google Scholar

Lim VS, Henriquez C, Sievertsen G, Frohman LA. Ovarian function in chronic renal failure: evidence suggesting hypothalamic anovulation. Ann Intern Med. 1980;93:217. https://doi.org/10.7326/0003-4819-93-1-21

Article CAS PubMed Google Scholar

Prem AR, Punekar SV, Kalpana M, Kelkar AR, Acharya VN. Male reproductive function in uraemia: efficacy of haemodialysis and renal transplantation. Br J Urol. 1996;78:6358. https://doi.org/10.1046/j.1464-410x.1996.14624.x

Article CAS PubMed Google Scholar

Levitan D, Moser SA, Goldstein DA, Kletzky OA, Lobo RA, Massry SG. Disturbances in the hypothalamic-pituitary-gonadal axis in male patients with acute renal failure. Am J Nephrol. 1984;4:99106. https://doi.org/10.1159/000166785

Article CAS PubMed Google Scholar

Distiller LA, Morley JE, Sagel J, Pokroy M, Rabkin R. Pituitary-gonadal function in chronic renal failure: the effect of luteinizing hormone-releasing hormone and the influence of dialysis. Metabolism. 1975;24:71120. https://doi.org/10.1016/0026-0495(75)90039-6

Article CAS PubMed Google Scholar

Biasioli S, Mazzali A, Foroni R, DAndrea G, Feriani M, Chiaramonte S, et al. Chronobiological variations of prolactin (PRL) in chronic renal failure (CRF). Clin Nephrol. 1988;30:8692.

CAS PubMed Google Scholar

Carrero JJ, Kyriazis J, Sonmez A, Tzanakis I, Qureshi AR, Stenvinkel P, et al. Prolactin levels, endothelial dysfunction, and the risk of cardiovascular events and mortality in patients with CKD. Clin J Am Soc Nephrol. 2012;7:20715. https://doi.org/10.2215/CJN.06840711

Article CAS PubMed PubMed Central Google Scholar

Adachi N, Lei B, Deshpande G, Seyfried FJ, Shimizu I, Nagaro T, et al. Uraemia suppresses central dopaminergic metabolism and impairs motor activity in rats. Intensive Care Med. 2001;27:165560. https://doi.org/10.1007/s001340101067

Article CAS PubMed Google Scholar

Sievertsen GD, Lim VS, Nakawatase C, Frohman LA. Metabolic clearance and secretion rates of human prolactin in normal subjects and in patients with chronic renal failure. J Clin Endocrinol Metab. 1980;50:84652. https://doi.org/10.1210/jcem-50-5-846

Article CAS PubMed Google Scholar

Carrero JJ, Qureshi AR, Nakashima A, Arver S, Parini P, Lindholm B, et al. Prevalence and clinical implications of testosterone deficiency in men with end-stage renal disease. Nephrol Dial Transplant. 2011;26:18490. https://doi.org/10.1093/ndt/gfq397

Article CAS PubMed Google Scholar

Foresta C, Caretta N, Lana A, De Toni L, Biagioli A, Ferlin A, et al. Reduced number of circulating endothelial progenitor cells in hypogonadal men. J Clin Endocrinol Metab. 2006;91:4599602. https://doi.org/10.1210/jc.2006-0763

Article CAS PubMed Google Scholar

Nilsson E, Stenvinkel P, Liu S, Stedman MR, Chertow GM, Floege J. Serum testosterone concentrations and outcomes in hemodialysis patients enrolled in the EVOLVE trial. Nephrol Dial Transplant. 2023;38:151927. https://doi.org/10.1093/ndt/gfac278

Article CAS PubMed Google Scholar

Rymarz A, Matyjek A, Gomka M, Niemczyk S. Lean tissue index and body cell mass can be predictors of low free testosterone levels in men on hemodialysis. J Ren Nutr. 2019;29:52935. https://doi.org/10.1053/j.jrn.2019.03.078

Article CAS PubMed Google Scholar

Cobo G, Gallar P, Di Gioia C, Garca Lacalle C, Camacho R, Rodriguez I, et al. Hypogonadism associated with muscle atrophy, physical inactivity and ESA hyporesponsiveness in men undergoing haemodialysis. Nefrologia. 2017;37:5460. https://doi.org/10.1016/j.nefro.2016.04.009

Article PubMed Google Scholar

Park MG, Koo HS, Lee B. Characteristics of testosterone deficiency syndrome in men with chronic kidney disease and male renal transplant recipients: a cross-sectional study. Transplant Proc. 2013;45:29704. https://doi.org/10.1016/j.transproceed.2013.08.087

Article CAS PubMed Google Scholar

Skiba R, Rymarz A, Matyjek A, Dymus J, Woniak-Kosek A, Syryo T, et al. Testosterone replacement therapy in chronic kidney disease patients. Nutrients. 2022;14:3444. https://doi.org/10.3390/nu14163444

Article CAS PubMed PubMed Central Google Scholar

Mulhall JP, Trost LW, Brannigan RE, Kurtz EG, Redmon JB, Chiles KA, et al. Evaluation and management of testosterone deficiency: AUA guideline. J Urol. 2018;200:42332. https://doi.org/10.1016/j.juro.2018.03.115

Article PubMed Google Scholar

Brock G, Heiselman D, Maggi M, Kim SW, Rodrguez Vallejo JM, Behre HM, et al. Effect of testosterone solution 2% on testosterone concentration, sex drive and energy in hypogonadal men: results of a placebo controlled study. J Urol. 2016;195:699705. https://doi.org/10.1016/j.juro.2015.10.083

Article CAS PubMed Google Scholar

Maggi M, Heiselman D, Knorr J, Iyengar S, Paduch DA, Donatucci CF. Impact of testosterone solution 2% on ejaculatory dysfunction in hypogonadal men. J Sex Med. 2016;13:12206. https://doi.org/10.1016/j.jsxm.2016.05.012

Article PubMed Google Scholar

Roy CN, Snyder PJ, Stephens-Shields AJ, Artz AS, Bhasin S, Cohen HJ, et al. Association of testosterone levels with anemia in older men: a controlled clinical trial. JAMA Intern Med. 2017;177:48090. https://doi.org/10.1001/jamainternmed.2016.9540

Article PubMed PubMed Central Google Scholar

Svartberg J, Agledahl I, Figenschau Y, Sildnes T, Waterloo K, Jorde R. Testosterone treatment in elderly men with subnormal testosterone levels improves body composition and BMD in the hip. Int J Impot Res. 2008;20:37887. https://doi.org/10.1038/ijir.2008.19

Article CAS PubMed Google Scholar

Cherrier MM, Anderson K, Shofer J, Millard S, Matsumoto AM. Testosterone treatment of men with mild cognitive impairment and low testosterone levels. Am J Alzheimers Dis Other Demen. 2015;30:42130. https://doi.org/10.1177/1533317514556874

Article CAS PubMed Google Scholar

Vaughan C, Goldstein FC, Tenover JL. Exogenous testosterone alone or with finasteride does not improve measurements of cognition in healthy older men with low serum testosterone. J Androl. 2007;28:87582. https://doi.org/10.2164/jandrol.107.002931

Article CAS PubMed Google Scholar

Grossmann M, Hoermann R, Wittert G, Yeap BB. Effects of testosterone treatment on glucose metabolism and symptoms in men with type 2 diabetes and the metabolic syndrome: a systematic review and meta-analysis of randomized controlled clinical trials. Clin Endocrinol. 2015;83:34451. https://doi.org/10.1111/cen.12664

Article CAS Google Scholar

Barton CH, Mirahmadi MK, Vaziri ND. Effects of long-term testosterone administration on pituitary-testicular axis in end-stage renal failure. Nephron. 1982;31:614. https://doi.org/10.1159/000182618

Article CAS PubMed Google Scholar

van Coevorden A, Stolear JC, Dhaene M, van Herweghem JL, Mockel J. Effect of chronic oral testosterone undecanoate administration on the pituitary-testicular axes of hemodialyzed male patients. Clin Nephrol. 1986;26:4854.

PubMed Google Scholar

Pechersky AV, Mazurov VI, Semiglazov VF, Karpischenko AI, Mikhailichenko VV, Udintsev AV. Androgen administration in middle-aged and ageing men: effects of oral testosterone undecanoate on dihydrotestosterone, oestradiol and prostate volume. Int J Androl. 2002;25:11925. https://doi.org/10.1046/j.1365-2605.2002.00335.x

Article CAS PubMed Google Scholar

Pizzol D, Xiao T, Yang L, Demurtas J, McDermott D, Garolla A, et al. Prevalence of erectile dysfunction in patients with chronic kidney disease: a systematic review and meta-analysis. Int J Impot Res. 2021;33:50815. https://doi.org/10.1038/s41443-020-0295-8

Article PubMed Google Scholar

Lawrence IG, Price DE, Howlett TA, Harris KP, Feehally J, Walls J. Correcting impotence in the male dialysis patient: experience with testosterone replacement and vacuum tumescence therapy. Am J Kidney Dis. 1998;31:3139. https://doi.org/10.1053/ajkd.1998.v31.pm9469503

Article CAS PubMed Google Scholar

Chatterjee R, Wood S, McGarrigle HH, Lees WR, Ralph DJ, Neild GH. A novel therapy with testosterone and sildenafil for erectile dysfunction in patients on renal dialysis or after renal transplantation. J Fam Plann Reprod Health Care. 2004;30:8890. https://doi.org/10.1783/147118904322995438

Article PubMed Google Scholar

Cunningham G, Belkoff L, Brock G, Efros M, Gittelman M, Carrara D, et al. Efficacy and safety of a new topical testosterone replacement gel therapy for the treatment of male hypogonadism. Endocr Pract. 2017;23:55765. https://doi.org/10.4158/EP161665.OR

Article PubMed Google Scholar

Gronski MA, Grober ED, Gottesman IS, Ormsby RW, Bryson N. Efficacy of nasal testosterone gel (Natesto) stratified by baseline endogenous testosterone levels. J Endocr Soc. 2019;3:165262. https://doi.org/10.1210/js.2019-00183

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Testosterone deficiency in men with end stage renal disease and kidney transplantation: a narrative review ... - Nature.com

Recommendation and review posted by Bethany Smith

The following is a summary of Effect of Testosterone Replacement Therapy on Sexual Function and Hypogonadal Symptoms in Men with Hypogonadism, published in the February 2024 issue of Endocrinology by Pencina, et al.

For a Testosterone Replacement Therapy for Assessment of long-term Vascular Events and efficacy ResponSE in hypogonadal men (TRAVERSE) study, researchers sought to assess the efficacy of testosterone replacement therapy (TRT) in improving sexual function and hypogonadal symptoms in men with hypogonadism, with a focus on whether these effects are sustained beyond 12 months. The Sexual Function Study, nested within the TRAVERSE trial, specifically evaluated the impact of TRT on sexual activity, hypogonadal symptoms, libido, and erectile function among men with low libido.

Among 5,204 men aged 45-80 years with two testosterone concentrations <300 nag/dL, hypogonadal symptoms, and cardiovascular disease (CVD) or increased CVD risk enrolled in the TRAVERSE trial, 1,161 participants with low libido were included in the Sexual Function Study. Of these, 587 were randomized to receive 1.62% testosterone gel and 574 to placebo gel for their participation. The primary outcome was the change from baseline in sexual activity score, with secondary outcomes including hypogonadal symptoms, erectile function, and sexual desire.

TRT was associated with a significantly greater improvement in sexual activity compared to placebo, with a sustained treatment effect observed at 24 months. Additionally, TRT improved hypogonadal symptoms and sexual desire, although it did not significantly affect erectile function compared to placebo.

In middle-aged and older men with hypogonadism and low libido, TRT for 2 years effectively improved sexual activity, hypogonadal symptoms, and sexual desire. However, it did not demonstrate significant improvements in erectile function compared to placebo.

Reference: academic.oup.com/jcem/article-abstract/109/2/569/7244351

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Enhancing Quality of Life: Testosterone Replacement Therapy for Hypogonadal Men - Physician's Weekly

Recommendation and review posted by Bethany Smith