HKUs innovative research novelties excel at – EurekAlert

image:Artificial Intelligence MGF Network for Anomalies Detection, developed by Dr Wilton Fok's team, wins two special grand prizes Invention & Innovation CAI Award (China Delegation) and Prize of the Delegation of Malaysia, and a gold medal view more

Credit: The University of Hong Kong

The University of Hong Kong (HKU) triumphed at the 48th International Exhibition of Inventions of Geneva, winning a total of 19 awards, including two special grand prizesInvention & Innovation CAI Award (China Delegation), andPrize of the Delegation of Malaysia.The results were announced yesterday (April 28).

Research teams from Faculty of Architecture, Faculty of Engineering, Faculty of Science,LKS Faculty of Medicine, and two HKU Inno Laboratories, established under the Hong Kong Government's InnoHK programme,garnered two special grand prizes, oneGold Medal with the Congratulations of the Jury, sixGold Medals, sixSilver Medals and fourBronze Medals with their innovations in the event.

TheArtificial Intelligence MGF Network for Anomalies Detection,developed byDr Wilton Fok's team from the Department of Electrical and Electronic Engineering, was winner of the two grand prizes and a gold medal. The system usesAI to analyse in real time human posture and movements in a video to identify anomaly scenarios such as abuse, drowning, and criminal behaviour.

TheProgrammable Cell Niche Engineering Platform,developed byProfessor Barbara Chan's team from the Department of Mechanical Engineering, wonaGold Medal with the Congratulations of the Jury. The system ensures proper cellular functions and results that can enable predictable clinical studies by mimicingthe biological environment for cells.

HKU has showcased this year a total of 17 research inventions that provide solutions to pressing human problems, spanning diverse areas such as biomedicine, vaccine development, artificial intelligence, and aging. Notably, the Advanced Biomedical Instrumentation Centre and the Centre for Virology, Vaccinology and Therapeutics, established by HKU in 2021 under the Hong Kong Government's InnoHK program, have demonstrated exceptional ability in successfully translating their biomedical research achievements into practical applications within a relatively short timeframe. Each have two research inventions selected, including an aptasensor for sepsis diagnosis, a capillary microfluidic platform for point-of-care testing, an interferon-integrated coronavirus vaccine, and NanoComplex vaccine technology.

HKU takes immense pride in the recognition our researchers have received for their innovative research efforts. We are also dedicated to facilitating the transfer of our research findings into tangible applications in the market. Through the powerful synergies generated from our collaborations with global research and industry partners, we strive to facilitate the emergence of pioneering innovations and their transformation into products that improve the well-being of humanity, said Professor Max Shen, Vice-President and Pro-Vice-Chancellor (Research).

Renowned as one of the most distinguished innovation exhibitions in the world, the International Exhibition of Inventions of Geneva (IEIG) is an extraordinary annual global event devoted exclusively to inventions and innovations.

Since first participating in the event in 2018, HKU has received a total of 60 awards including 4 prestigious Gold Medals with Congratulations of the Jury and 21 other gold medals.

HKUs award-winning inventions in 2023:

INVENTION & INNOVATION CAI AWARD (China Delegation) & PRIZE OF THE DELEGATION OF MALAYSIA & GOLD MEDAL

Artificial Intelligence MGF Network for Anomalies DetectionThe invention can be deployed in children's centres, swimming pools, public transport and exhibition centres to detect abnormal behaviours and situations for public safety and security. Using AI to detect human skeletons in a video and analyse their posture and movements, it is able to capture the context information in a long video and address specific portions for detecting multiple anomaly scenarios in real time such as abuse, drowning, terrorist attacks, traffic accidents, fighting and criminal behaviour, more accurately and efficiently.

The invention was developed by the research team of Dr Wilton Fok from the Department of Electrical and Electronic Engineering, The University of Hong Kong.

GOLD MEDALS WITH CONGRATULATIONS OF THE JURY

Programmable Cell Niche Engineering PlatformOne of the reasons many drugs that work in the lab do not work as expected in the clinical trial stage is that scientists grow cells on flat and rigid culture dishes for drug research, which are not biomimetic -- leading to cell responses in lab testing that do not represent what would happen in native tissues of human bodies. The Programmable Cell Niche Engineering Platform mimics the biological environment for cells, which in turn ensures proper cellular functions and results that can enable predictable clinical studies. The multifactorial biochips invented identify the ideal niche for different cell types, equipping scientists with a comprehensive library of individual niche factors to develop optimal culture environments for different types of cells of specialised applications.

The platform was developed by Professor Barbara Chan and her team from the Department of Mechanical Engineering, Biomedical Engineering Programme, The University of Hong Kong.

GOLD MEDALS

1. Respiratory OrganoidsBiomedical research mainly relies on cell lines and animal models for experimentation, which has limited physiological relevance to human biology and pathology. Under this project, a complete respiratory organoid culture system has been the first of its kind to efficiently reconstruct and reproducibly expand the entire human respiratory epithelium in culture plates. All the differentiated respiratory organoids, including nasal, airway and alveolar organoids, adequately mimic the multicellular composition of native respiratory epithelium and phenocopy the functionality. Superior to most existing in vitro models, these organoids represent universal tools with biological relevance for diverse biomedical and pharmaceutical applications, including disease modeling, development of therapeutics, toxicological evaluation and personalised medicine.

The project was originated by Dr Jie Zhou, Dr Man Chun Chiu, Dr Cun Li and Professor Kwok Yung Yuen from the Department of Microbiology, The University of Hong Kong.

2. Bismuth-based Cocktail Therapy for CoronavirusThere is an urgent need for safe and effective therapeutic options for COVID-19, with the SARS-CoV-2 variants associated with enhanced transmissibility, reduced vaccine-induced protection and emerging drug resistance. Under this project, the orally administrated bismuth drug colloidal bismuth subcitrate together with N-acetyl cysteine has been found to be a broad-spectrum anti-coronavirus cocktail therapy against multiple SARS-CoV-2 variants and different coronaviruses. Mixing two US Food and Drug Administration (FDA) approved over counter drugs, 1 molar equivalent of colloidal bismuth subcitrate and 3 molar equivalents of a mucoactive drug N-acetyl cysteine, is a safe and highly potent therapy through oral administration and 10 times more selective in killing the virus than the first FDA approved drug. This therapy is now in phase II/III clinical trial in Hong Kong, with a phase III trial underway in China.

The invention was developed by Professor Sun Hongzhe, Professor Yuen Kwok-yung, Dr Yuan Shuofeng, Dr Wang RunMing, Dr Li Hongyan, Dr Jasper Chan Fuk-Woo, Dr Cheng Tianfan, Mrs Wang SuYu, and Mr Chan Chun Lung of The University of Hong Kong.

3. PERfECT Wearables for Digital HealthEndorsed by over 100 leading scientists worldwide on social media, "HKU PERfECT" is the first wearable platform combining the distinctive features of being highly sensitive, energy efficient, lightweight and ultracompact, enabling decentralised medical technologies for remote and personalised healthcare to cope with an aging population, quarantine and the shortage of medical instrumentation. Compared to current wearable devices that can only measure vital signals (e.g. heart rate, breathing rate, blood pressure, etc.), PERfECT is able to detect molecular indicators in body fluids, which are more informative for fatal/chronic disease screening and monitoring.

The invention was developed by the WISE Research Group from The University of Hong Kong and SESIC LIMITED.

4. Method of Developing a Peptide-based Vaccine Conjugated with 1V209This invention describes the development of a method to conjugate 1V209, a toll-like receptor 7 (TLR7) agonist, to antigens to induce antigen-specific responses. Past synthetic peptide vaccines must be used together with vaccine adjuvants to stimulate T cells against specific antigens to treat cancer, but a simple mixture of antigens and adjuvants can hardly induce enough specific immune responses. To address this issue, we designed bioinspired nanoparticles mimicking the structure of the virus to enhance the antigen-specific immune response. Through safer and greener methods, it used the TLR7 agonist 1V209 to generate peptide-drug conjugates and form a self-adjuvating vaccine to deliver antigens and adjuvants at the same time, inducing a stronger T cell response with improved stability and successful suppression of tumour growth.

The invention was developed by Professor Jiandong Huang and Dr Yefan Hu from the School of Biomedical Sciences, The University of Hong Kong.

5. DipChip Capillary Microfluidic Platform for Point-of-care DiagnosticsDipChip is an automated capillary microfluidic-based point-of-care (POC) microsystem allowing rapid and portable detection of various high-impact and mortality diseases. It is designed and fabricated using state-of-the-art molecular biology and microfluidic technologies, utilising capillary pressure and surface-activating treatments that allow laboratory-level analytics to be conducted in an accessible manner. This microsystem paves the way for a versatile array of clinical and academic applications that require multiple conjugations and washings. End users of DipChip include clinics, hospitals, homes and assisted living healthcare facilities, democratising access to adequate clinical care, and saving precious lives of patients in need.

The platform was developed by Professor Anderson Shum, Dr. Hassan Sammer Ul and Mr. Nicky Lee from Department of Mechanical Engineering and Advanced Biomedical Instrumentation Centre.

SILVER MEDALS

1. Smart Address Plates for Pedestrian Indoor Navigation and Location-Based Services and ManagementTraditionally, pedestrian navigation uses Location Positioning System (LPS) with trilateration to find the users position, which have very high positioning error for outdoor GPS and indoor positioning. This new solution adopts a cost-effective innovative Location Confirmation System (LCS) to accurately locate and guide the user to the destination by using Smart Address Plates (SAP) that transmit stored geographic coordinates with innovative 3D Smart Address (SA) codes to the users even without WiFi or telephone signals. This SAP system can help to find shops/ offices/ restaurants/ car parking spaces inside a multi-storey building accurately while providing location-based services and management for precise target marketing. It is highly scalable, connecting shops/rooms on a floor to a building, then to a district and to the whole city through a Smart Address Plate Management System (SAP-MS).

The Smart Address Plates and Location Confirmation System were developed by Professor Anthony Yeh, Dr Zhong Teng and Dr Run Shi from the Department of Urban Planning and Design, The University of Hong Kong.

2. A Novel Stem Cell-based Platform for Antiviral Discovery, Vaccine and Healthy Natural ProductsThere is an urgent need for the vaccine industry to establish a new type of human cell model for anti-virus drug development in order to enhance sensitivity and efficiency. The human Expanded Potential Stem Cells (hEPSCs) developed under this project help generate trophoblast stem cells (TSCs) and syncytiotrophoblasts (STBs), which express high levels of SARS-CoV-2 host factors and are highly susceptible to the viral infection. Remarkably, the infected eSTBs are 1,000 times more sensitive to antiviral drugs, making the TSC-STB platform a more ideal human cell source for viral production and performing antiviral discovery, including SARS-CoV-2, MERS, their variants, and the FDA approved anti-virus drugs and natural products.

The platform was developed by Professor Pengtao Liu from the Li Ka Shing Faculty of Medicine, and Dr Degong Ruan and Professor Fang Liu from the Centre for Translational Stem Cell Biology, The University of Hong Kong.

3. Ultra-thin Flexible Robotic Instruments for Endoluminal SurgeryUnder this project, a robotic system with highly miniaturised and flexible instruments has been developed to address challenges in the endoluminal treatment of early-stage gastrointestinal and urinary tract cancers. In current practice, there is a steep learning curve for resecting tumours in their entirety due to a lack of instrument dexterity and poor tissue retraction. The developed system provides two robotic instruments as small as 2.5 mm with 5 degrees of freedom each, enabling enhanced endoluminal surgery with conventional rigid and flexible endoscopes. Overall, the system will help to flatten the learning curve so that more clinicians can provide patients with effective early-stage care.

The system was developed by the research team of Dr Ka-Wai Kwok from the Department of Mechanical Engineering, The University of Hong Kong.

4. Automatic Alignment of MicroparticlesThis groundbreaking chip-size invention revolutionises the way to align micro-particles in liquids. Using a microchannel with specifically arranged roadblocks that effortlessly guides disordered particles into an ultrafast-flowing chain, it provides outstanding performance at a low cost. As compact as a credit card and priced at less than USD10, each device can align up to one million particles per minute with precision down to 0.001 mm. This game-changer bypasses the limitations of current methods, thereby enhancing the efficiency of applications that involve microparticle analysis and processing, particularly blood cell screening and water filtration.

The invention was developed by Professor Kevin Tsia and Dr Kelvin Lee from the Department of Electrical and Electronic Engineering, The University of Hong Kong.

5. Interferon-integrated coronavirus vaccine: a next-generation universal live vaccine protects against pan beta-coronavirus infectionIn the wake of the COVID-19 pandemic, it is imperative to develop a next-generation coronavirus vaccine that can offer protection against current and potential future beta-coronavirus infections. However, there are several technical hurdles that must be overcome in order to create an ideal vaccine that has three key properties: 1) universal protection against a broad range of beta-coronaviruses, 2) enhanced mucosal T cell immunity, and 3) activated innate anti-viral mechanisms to ensure safety. The team has developed an innovative immunological design and genetic engineering technology that has resulted in the creation of a universal beta-coronavirus mucosal vaccine and a streamlined production pipeline. Preclinical studies in two animal models have demonstrated the efficacy of the vaccine in terms of the three key properties.

The technology was developed by Dr KH Kok and the team from the Department of Microbiology of The University of Hong Kong and the Centre for Virology, Vaccinology and Therapeutics.

6.NanoComplexNanoComplex is a next-generation vaccine technology designed for nasal use and carries the potential to target all kinds of viruses. Viruses have proteins as antigens and nucleic acids as genomes, and both are critical components to alert our immune system and generate strong protection. The NanoComplex technology takes full advantage of these components, combines viral proteins and nucleic acids with innovative methods, and creates a nano-sized complex that can be used as a nasal vaccine. Since all viruses have the same components, the NanoComplex can switch its internals for different viruses, and therefore can potentially defend against all known viruses.

The technology was developed by a research team lead by Dr Kin Hang Kok from the Department of Microbiology of The University of Hong Kong, and the Centre for Virology, Vaccinology and Therapeutics.

BRONZE MEDALS

1. Remote e-Inspection System for the Manufacturing and Delivery of Offsite Modular ConstructionThis e-Inspection System is a Modular Construction Supply Chain Quality Assurance system that includes i-Core (an loT device attached to each MC module to monitor the position, humidity, temperature and collision data), e-InStar (an App for uploading the checking result of each production step in a remote factory to the block chain), e-TranStar (an App for monitoring the location and condition of the MC module in the transport process) and a blockchain-based backend. Designed for offsite modular construction use to help solve housing problems, it reduces resources required for supervision and paperwork while ensuring tamper-proof data, helping overcome the current difficulties in monitoring the quality of production and transportation from remote sites. The system has been piloted in an HKU project in Hong Kong with two 17-storey buildings using 952 MiC modules.

The system was developed by Prof Wilson Lu, Director of the iLab of the Faculty of Architecture, together with Professor Anthony Yeh, Chair Professor of the Department of Urban Planning and Design, and Mr KL Tam, Former Director of the Estates Office of The University of Hong Kong.

2. On-site Fertilizer Production for Perpetual FarmingThe EC Flow device developed under this project is an energy-neutral system that fully integrates with solar panels and battery storage systems to detect and remove numerous toxic residual nitrates and nitrites (NOx) in soil using renewable energy. It can further upcycle NOx and feed fertilizer back into urban farming sites, achieving waste remediation, pollution mitigation and resource recovery to reach nitrogen neutrality. This solution can be applied to plantation areas of any scale and type, including farms, lawns, roof gardens and balconies. The goals are lowering toxin levels in municipal waste and alleviating ecological strains on urban sewage systems, ultimately improving the urban municipal discharge situation and local marine environment.

The device was innovated by Dr Edmund Tse, Wanying Wang from the Department of Chemistry, The University of Hong Kong, and commercialised with the help of Yip Jackson Ho So.

3. Smart Elderly WalkerThis is a smart and handy device pairing advanced Artificial Intelligence with a home-friendly design, helping the elderly stay healthy, active and independent while making their lives easier and more secure. It brings together several advanced features, including a stable mechanical structure with smooth omnidirectional mobility, dual-mode actuation and control for simultaneous walking/standing support and fall prevention/recovery, soft robotic handles and privacy-friendly sensors for event detection and control and fall detection, as well as specialised sound-source localisation to locate the user quickly and effectively, providing enhanced safety protection and superior walking assistance for older adults.

The device was developed by the research team of Professor Chuan Wu, Dr Hao Luo, Dr Zhao Chongyu and Dr Wen Rongwei from the Department of Computer Science of The University of Hong Kong; and Professor Wang Zheng from the Department of Mechanical and Energy Engineering of The Southern University of Science and Technology in Shenzhen.

4. Aptasensor for Sepsis DiagnosticDiagnosing sepsis in a timely manner to ensure optimal patient survival with prompt treatment remains a major challenge. This invention utilises a novel DNA-based biosensor that integrates aptamers into a point-of-care platform to advance sepsis diagnosis. Aptamers recognise biomarkers by specifically binding to unique structures on their surface. Unlike antibody-based tools, aptamers provide multiple advantages in terms of stability, production cost, reproducibility and flexibility that can readily be integrated into various platforms, such as electrochemical platforms (quantitative device) and/or colorimetric assays (rapid test). The aptasensor provides a quick and highly sensitive solution for sepsis detection that can help revolutionise point-of-care sepsis detection.

The invention was developed by Dr. Louisa Hoi-Ying Lo, Dr. William Whitehouse, supervised by Professor Julian Tanner, from the Advanced Biomedical Instrumentation Centre.

The Universitys participation in this years competition was coordinated by the Technology Transfer Office of HKU.

For details about the International Exhibition of Inventions of Geneva, please visit:https://inventions-geneva.ch/en/home/.

About Technology Transfer Office, HKUThe Technology Transfer Office (TTO) manages the use of HKUs intellectual property assets by providing patenting, licensing and other commercialisation support to the Universitys researchers and inventors. Acting as the bridge linking HKU to society in the area of technology commercialisation, TTO helps industries and businesses to access HKUs powerhouse of knowledge, innovation and expertise through close collaboration. Website:http://www.tto.hku.hk.

For media enquiries, please contact:Communications and Public Affairs Office, HKUMs Melanie Wan (Email: melwkwan@hku.hk)Ms Jaymee Ng (Email:ngjaymee@hku.hk)Mr Kenneth Choi (Email:khkchoi@hku.hk)

Technology Transfer Office, HKUMs Joy Ma (Email: joy@tto.hku.hk)

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HKUs innovative research novelties excel at - EurekAlert

The Skin’s Secret Weapon: Researchers Uncover an Ancient … – SciTechDaily

A variety of cells (white) proliferate at the ragged edge of a five-day-old wound, including epidermal stem cells (basal layer of epithelium in green), which secrete IL24. Credit: Laboratory of Elaine Fuchs

The world can be a hazardous place, with various dangers lurking around us such as bacteria, viruses, accidents, and injuries. Our skin acts as the ultimate shield, providing a steadfast defense against these threats. It serves as the boundary between the internal and external environment and is the largest organ in the body, functioning nearly seamlessly to protect us.

Still, the skin is not immune to harm. It endures daily assaults and still tries to keep us safe by detecting and responding to these dangers. One method is the detection of pathogens, which activates the immune system. However, recent research conducted by Elaine Fuchs at Rockefeller University and published in the journal Cell, has uncovered a new protection mechanism that responds to injury signals in damaged tissue, such as low oxygen levels caused by blood vessel disruption and scab formation. This mechanism is activated without the need for an infection.

The study is the first to identify a damage response pathway that is distinct from but parallel to the classical pathway triggered by pathogens.

At the helm of the response is interleukin-24 (IL24), whose gene is induced in skin epithelial stem cells at the wound edge. Once unleashed, this secreted protein begins to marshal a variety of different cells to begin the complex process of healing.

IL24 is predominately made by the wound-edge epidermal stem cells, but many cells of the skinthe epithelial cells, the fibroblasts, and the endothelial cellsexpress the IL24 receptor and respond to the signal. IL24 becomes an orchestrator that coordinates tissue repair, says Fuchs, head of the Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Development.

Scientists have long understood how the host responses protect our body from pathogen-induced threats: somatic cells recognize invading bacteria or viruses as foreign entities and induce a number of defense mechanisms with the help of signaling proteins such as type 1 interferons.

But how does the body respond to an injury that may or may not involve foreign invaders? If we cut a finger while slicing a cucumber, for example, we know it instantlytheres blood and pain. And yet how the detection of injury leads to healing is poorly understood on a molecular basis.

While type 1 interferons rely on the signaling factors STAT1 and STAT2 to regulate the defense against pathogens, previous research by the Fuchs lab had shown that a similar transcription factor known as STAT3 makes its appearance during wound repair. Siqi Liu, a co-first author in both studies, wanted to trace STAT3s pathway back to its origin.

IL24 stood out as a major upstream cytokine that induces STAT3 activation in the wounds.

In collaboration with Daniel Mucidas lab at Rockefeller, the researchers worked with mice under germ-free conditions and found that the wound-induced IL24 signaling cascade is independent of germs.

But what injury signals induced the cascade? Wounds often extend into the skin dermis, where capillaries and blood vessels are located.

We learned that the epidermal stem cells sense the hypoxic environment of the wound, says Yun Ha Hur, a research fellow in the lab and a co-first author on the paper.

When the blood vessels are severed and a scab forms, epidermal stem cells at the edge of the wound are starved of oxygen. This state of hypoxia is an alarm bell for cell health and induced a positive feedback loop involving transcription factors HIF1a and STAT3 to amplify IL24 production at the wound edge. The result was a coordinated effort by a variety of cell types expressing the IL24 receptor to repair the wound by replacing damaged epithelial cells, healing broken capillaries, and generating fibroblasts for new skin cells.

Collaborating with Craig Thompsons group at Memorial Sloan Kettering Cancer Center, the researchers showed that they could regulate Il24 gene expression by changing oxygen levels.

Once the researchers pinpointed the origin of the tissue-repair pathway in epidermal stem cells, they studied the wound repair process in mice that had been genetically modified to lack IL24 functionality. Without this key protein, the healing process was sluggish and delayed, taking days longer than in normal mice to completely restore the skin.

They speculate that IL24 might be involved in the injury response in other body organs featuring epithelial layers, which act as a protective sheath. In recent studies, elevated IL24 activity has been spotted in epithelial lung tissue of patients with severe COVID-19 and in colonic tissue in patients with ulcerative colitis, a chronic inflammatory bowel disease.

IL24 could be working as a cue to signal the need for injury repair in many organs, Hur says.

Our findings provide insights into an important tissue damage sensing and repair signaling pathway that is independent of infections, explains Fuchs.

An analysis with evolutionary biologist Qian Cong at UT Southwestern Medical Center revealed that IL24 and its receptors share close sequence and structure homology with the interferon family. Though they may not always be working in coordination at every moment, IL24 and interferons are evolutionarily related and bind to receptors sitting near each other on the surface of cells. The researchers suspect that these signaling molecules derive from a common molecular pathway dating far back in our past.

We think that hundreds of millions of years ago, this ancestor might have diverged into two pathwaysone being pathogen defense and the other being tissue injury, Liu says.

Perhaps the split occurred to cope with an explosion of pathogens and injuries that caused a sea of troubles for life on Earth.

Reference: A tissue injury sensing and repair pathway distinct from host pathogen defense by Siqi Liu, Yun Ha Hur, Xin Cai, Qian Cong, Yihao Yang, Chiwei Xu, Angelina M. Bilate, Kevin Andrew Uy Gonzales, S. Martina Parigi, Christopher J. Cowley, Brian Hurwitz, Ji-Dung Luo, Tiffany Tseng, Shiri Gur-Cohen, Megan Sribour, Tatiana Omelchenko, John Levorse, Hilda Amalia Pasolli, Craig B. Thompson, Daniel Mucida and Elaine Fuchs, 24 April 2023, Cell.DOI: 10.1016/j.cell.2023.03.031

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The Skin's Secret Weapon: Researchers Uncover an Ancient ... - SciTechDaily

Childrens Hospital Los Angeles Researchers Uncover New Clues to Origins of the Most Common Pediatric Kidney Cancer – Newswise

Newswise While Wilms tumoralso known as nephroblastoma-- is rare, it is the most prevalent childhood kidney cancer. Researchers at Childrens Hospital Los Angeles have now pinpointed a disruption in early kidney progenitor cell development that can be linked to the formation of Wilms tumor.

In a study published in Advanced Science, researchers at the GOFARR Laboratory in Urology compared kidney progenitor cells from a tumor with precursor cells from a healthy kidney. Normally, these precursor cells mature into kidney cells, but when their early development is dysregulated, they behave like cancer stem cells.

While most children with Wilms tumor are successfully treated, current therapies are aggressive. A minority of these patients have unfavorable prognoses or relapses; for these children, there is no existing therapy. By achieving a more precise understanding of how Wilms tumors develop, our goal is to find new treatments for all types of Wilms tumor, says Laura Perin, PhD, Co-Director of the GOFARR laboratory and senior study co-author with Stefano Da Sacco, PhD, another researcher at the GOFARR Laboratory.

Pediatric Wilms tumor can be considered a developmental cancer, says Dr. Perin, who is also Associate Professor at the Keck School of Medicine of USC. The normal adult kidney lacks kidney precursor cells, as they are exhausted before birth. But in Wilms tumors, instead of giving rise to a functional kidney, these precursor cells persist and form the tumor mass.

The researchers characterized these Wilms tumor kidney precursor cells, finding that these cells can reproduce the original tumor. They are aggressive, theyre drug-resistant, they metastasize like cancer cells, and they are able to create the full tumor that we see in patients, says Astgik Petrosyan, PhD, researcher at the GOFARR Lab and first author of the study.

The kidney precursor cells that generate Wilms tumors also abnormally expressed ITG1 and ITG4, proteins that help cells communicate with their microenvironment. This abnormal attachment to their microenvironment favors the uncontrolled replication of these cells and guides the formation of the tumor mass, says Dr. Da Sacco.

"Our findings provide a more accurate understanding of the different stages of both normal and abnormal kidney development, says Dr. Perin. This can possibly help the diagnosis of Wilms tumor, leading to more effective treatments for these patients.

Other study co-authors include: Valentina Villania, PhD, of CHLA; Paola Aguiari, PhD, of CHLA and David Geffen School of Medicine at UCLA - VA Healthcare System; Matthew E. Thornton, MS, of the Keck School of Medicine of USC; Yizhou

Wang, PhD, and Alex Rajewski, PhD, of Cedars-Sinai Medical Center; Shengmei Zhou, MD, of CHLA, Paolo Cravedi, MD, PhD of the Icahn School of Medicine at Mount Sinai; Brendan H. Grubbs, MD, of the Keck School of Medicine of USC; Roger E. De Filippo, MD, Sargis Sedrakyan, PhD and Kevin V. Lemley, MD, PhD, of CHLA and the Keck School of Medicine at USC; and Marie Csete, MD, PhD of USC.

About Childrens Hospital Los Angeles

Childrens Hospital Los Angeles is at the forefront of pediatric medicine, offering acclaimed care to children from across the world, the country and the greater Southern California region. Founded in 1901, Childrens Hospital Los Angeles is the largest provider of care for children in Los Angeles County, the No. 1 pediatric hospital in the Pacific region and California, and among the top 10 in the nation on U.S. News & World Reports Honor Roll of Best Childrens Hospitals. Clinical expertise spans the pediatric care continuum for newborns to young adults, from everyday preventive medicine to the most advanced cases. Inclusive, kid- and family-friendly clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC, and proven discoveries reach patients faster through The Saban Research Institute of Childrens Hospital Los Angelesamong the top 10 childrens hospitals for National Institutes of Health funding. The hospital also is home to the largest pediatric residency training program at a freestanding childrens hospital in the western United States. To learn more, follow us onFacebook,Instagram,LinkedIn,YouTubeandTwitter, and visit our blog atCHLA.org/blog.

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Childrens Hospital Los Angeles Researchers Uncover New Clues to Origins of the Most Common Pediatric Kidney Cancer - Newswise

Global Breast Cancer Market Size Projected To Hit $70 Billion in … – StreetInsider.com

News and research before you hear about it on CNBC and others. Claim your 1-week free trial to StreetInsider Premium here.

Palm Beach, FL - May 5, 2023 FinancialNewsMedia.com News Commentary Breast cancer is the most commonly occurring disorder among the population majorly seen in females with rising disorder. It is a disorder that spreads the disease with increasing symptoms such as pain in the bones swelling of lymph nodes, breathing problems, and many more. Breast cancer development takes place with the presence of genes like Human Epidermal growth factor receptor 2 (HER2). Stimulation of cancer in the body with the presence of this protein extends the proliferation of the cells. Positive HER2 receptor patients having the most aggression in breast cancer than other types of breast cancer. Numerous treatments are available which are used to treat and eradicate the tumor and increase the target on HER2. Which enlarges the market of Breast cancer with an increasing number of patients every year with an increase in the recovery rate of breast cancer. A report from Precedence Research projects that the global breast cancer market size was estimated at USD 31.89 billion in 2022 and is projected to hit around USD 70.51 billion by 2030 with a registered CAGR of 10.43% during the forecast period 2022 to 2030. The report said: "Enhance research and development with increased new technologies and therapies developed in breast cancer which is prominently contributing to enhancing the market growth with increased demands from the medical sector for treating the patients with new treatments and medications. Government support with increasing providence for new developments in medications and increased efficiency and efficacy of the treatment and integration of newly developed medications in the medical sector." Active biotech and pharma companies in the markets this week include Oncolytics Biotech(R) Inc. (NASDAQ: ONCY) (TSX: ONC), Bristol Myers Squibb (NYSE: BMY), NANO-X IMAGING LTD (NASDAQ: NNOX), Soligenix, Inc. (NASDAQ: SNGX), Cardiff Oncology, Inc. (NASDAQ: CRDF).

Precedence Research said that key insights were: "By therapy, the targeted therapy segments involved the largest market; By cancer type, the hormone receptor segments extend the market share at the largest rate; By distribution channel, the hospital pharmacies segments developed their own pharmacies in the hospital with the highest market share; and By geography, The North American region holds the highest position in the market with increased market share. On the basis of geography, North America holds the highest position in the market with an increased market share in breast cancer. Advanced developed technologies in the market with an increased number of treatments available for treating breast cancer with an increasing number of patients helps to propel the market growth with increased government initiative in developing new drug developments and increased efficacy of the medications with increased results help to enhance the market growth."

Oncolytics Biotech(R) Inc. (NASDAQ: ONCY) (TSX: ONC) BREAKING NEWS: Oncolytics Biotech(R) Reports First Quarter 2023 Financial Results and Operational Highlights -

Oncolytics Biotech(R) today announced recent operational highlights and financial results for the first quarter ended March 31, 2023. All dollar amounts are expressed in Canadian currency unless otherwise noted.

"Our core programs in breast and pancreatic cancer are moving towards registrational studies with compelling clinical data and important milestones expected this year," said Dr. Matt Coffey, President and Chief Executive Officer. "The first of these milestones will come later this month when we report data from BRACELET-1, a randomized phase 2 trial expected to inform the design of a subsequent pivotal registrational study in HR+/HER2- metastatic breast cancer. Stakeholders across the breast cancer community have demonstrated a keen interest in this readout, which will add to a dataset that already includes statistically significant phase 2 results showing pelareorep's ability to drive a robust overall survival benefit in this indication. Notably, the BRACELET-1 abstract was selected for an oral presentation at the upcoming ASCO meeting, providing a highly prestigious venue to discuss the results with potential partners and the clinical community."

Dr. Coffey continued, "Looking into the second half of 2023, we expect to report updated results from GOBLET's first-line pancreatic cancer cohort and provide guidance on the optimal registration paths for our breast and pancreatic cancer programs. With these programs both targeting large markets with significant clinical unmet needs and supported by robust clinical proof-of-concept data, we believe confirming their paths towards approval will represent important value-creating events and substantially de-risk and maintain pelareorep's development pathway." CONTINUED... Read this full press release and more news for ONCY at: https://www.financialnewsmedia.com/news-oncy/

Other recent developments in the biotech industry of note include:

NANO-X IMAGING LTD (NASDAQ: NNOX) recently announced that it has received a 510(k) clearance from the U.S. Food and Drug Administration (FDA) to market the multi-source Nanox.ARC, including the Nanox.CLOUD, its accompanying cloud-based infrastructure. Nanox.ARC is a stationary X-ray system intended to produce tomographic images of the human musculoskeletal system adjunctive to conventional radiography on adult patients. Representing a major advancement in X-ray technology, Nanox.ARC is a multi-source digital 3D tomosynthesis system that utilizes novel, cold cathode X-ray tubes, which the Company intends to offer using an innovative pay-per-scan business model.

The FDA cleared Nanox.ARC for use in professional healthcare ??facilities or ??radiological ??environments, such as ??hospitals, clinics, imaging ??centers, and ??other medical practices?? by trained radiographers, ??radiologists, and physicians, and has the potential to increase availability to medical imaging around the world, once approved by local regulatory authorities and deployed at scale.

Soligenix, Inc. (NASDAQ: SNGX) recently announced positive clinical results from a compatibility study evaluating HyBryte (synthetic hypericin sodium) in the treatment of cutaneous T-cell lymphoma (CTCL) using the commercially ready Daavlin Series 7 visible light device, which recently received 510(k) clearance from U.S. Food and Drug Administration (FDA). The open-label study (protocol HPN-CTCL-02) enrolled 9 patients to receive 8 weeks of HyBryte treatment of their cancerous lesions, with an assessment of treatment response conducted at week 10 using the Composite Assessment of Index Lesion Severity (CAILS) score. All subjects were enrolled by Brian Poligone, MD, PhD, at the Rochester Skin Lymphoma Medical Group.

We were excited for the opportunity to work with Soligenix and make HyBryte available to our patients, stated Brian Poligone, MD, PhD, Director of the Rochester Skin Lymphoma Medical Group, Fairport, NY, and Principal Investigator for the compatibility study and Leading Enrolling Investigator in the FLASH study. Since the completion of the Phase 3 FLASH study, I have had a number of patients asking about possible access to this promising therapy. Fortunately, we were chosen to conduct this study and the patients enthusiasm for the product was evident by their willingness to participate in the trial, allowing for its rapid completion. I look forward to continuing to work with Soligenix to further advance the HyBryte program so my patients can have this much needed treatment option available to them.

Cardiff Oncology, Inc. (NASDAQ: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition, a well-validated oncology drug target, to develop novel therapies across a range of cancers, recently announced that the first patient was dosed this month with its investigational drug onvansertib in its Phase 2 ONSEMBLE trial (NCT05593328). The trial is designed to demonstrate a clinically meaningful difference in response and onvansertibs contribution to standard of care (SoC) FOLFIRI/bevacizumab for the second line treatment of patients with KRAS/NRAS-mutated metastatic colorectal cancer (mCRC).

We are excited to be underway with our ONSEMBLE trial that builds on the promising efficacy and tolerability results demonstrated in our phase 1b/2 trial in mCRC, said Fairooz Kabbinavar, MD, Chief Medical Officer of Cardiff Oncology. mCRC is a difficult-to-treat cancer and patients in the second line setting need novel therapeutic options to improve clinical outcomes. Based on our open-label phase 1b/2 trial, we believe the combination of onvansertib with FOLFIRI/bevacizumab could positively impact patients responses to treatment and the durability of the responses. Nearly half of our planned 40 sites in the US are open to enroll patients and weve seen great enthusiasm from participating investigators.

Bristol Myers Squibb (NYSE: BMY) recently announced that the European Commission (EC) has granted approval for Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adult patients with diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma (HGBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B), who relapsed within 12 months from completion of, or are refractory to, first-line chemoimmunotherapy. This approval covers all European Union (EU) member states.

The approval is based on results from the pivotal Phase 3 TRANSFORM trial in which Breyanzi demonstrated statistically significant and clinically meaningful improvements in the study's primary endpoint of event-free survival (EFS), and key secondary endpoints of complete responses (CR) and progression-free survival (PFS) compared to standard therapy (consisting of salvage immunochemotherapy followed by high-dose chemotherapy and hematopoietic stem cell transplant [HSCT]), along with a manageable and well-established safety profile.

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates FinancialNewsMedia.com and MarketNewsUpdates.com, is a third party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNMs market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM was compensated forty nine hundred dollars for news coverage of the current press releases issued by Oncolytics Biotech(R) Inc. by a non-affiliated third party.

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The post Global Breast Cancer Market Size Projected To Hit $70 Billion in Revenues By 2030 appeared first on Financial News Media.

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Uncovering the transcriptional regulatory network involved in … – Nature.com

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Oncopeptides AB – Invitation to presentation of the Q1 report 2023 – Marketscreener.com

Oncopeptides AB (publ) a biotech company focused on research, development, and commercialization of therapies for difficult-to-treat hematological diseases, will publish the report for the first quarter 2023 at 08:00 (CET) on May 4.

Investors, financial analysts, and media are invited to participate in a webcast and a following QnA session on May 4 at 09:00 (CET). The presentation will be hosted by Monica Shaw, CEO, together with Holger Lembrer, CFO, and will be held in English. It will be published on the website of Oncopeptides in conjunction with the start of the presentation.

Information for participants

If you wish to participate in the webcast, please use the link below. Through the webcast you will have opportunities to ask written questions to the company's leadership.

If you wish to participate via teleconference, please register on the link below. After the registration you will be provided with a phone number and a conference ID to access the conference. You can ask questions to the leadership verbally via the teleconference.

Contact:

Rolf Gulliksen

Email: rolf.gulliksen@oncopeptides.com

Tel: + 46 70 262 96 28

About Oncopeptides

Oncopeptides is a biotech company focused on research, development, and commercialization of therapies for difficult-to-treat hematological diseases. The company uses its proprietary Peptide Drug Candidate platform (PDC) to develop compounds that rapidly and selectively deliver cytotoxic agents into cancer cells.

Pepaxti (melphalan flufenamide, also called melflufen) has been granted Marketing Authorization, in the European Union, the EEA-countries Iceland, Lichtenstein and Norway, as well as in the UK. Pepaxti is indicated in combination with dexamethasone for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation. Melflufen has been granted accelerated approval in the US under the trade name Pepaxto. The drug is currently not marketed in the US.

Oncopeptides is developing several new compounds based on its proprietary technology platforms and is listed on the Small Cap segment on Nasdaq Stockholm with the ticker ONCO.

(C) 2023 Electronic News Publishing, source ENP Newswire

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Join the Autism Summit For Parents Free Virtual Talks to Cover … – GlobeNewswire

London, May 01, 2023 (GLOBE NEWSWIRE) -- London, England -

Autism Parenting Magazine Ltd. will be hosting its Autism Conference in May, continuing to bridge the ever-growing gap between autism experts and special needs families.

Autism Parenting Magazine (APM) is an award-winning publication aimed at improving the quality of life for families affected by autism. For over a decade, APM has been at the forefront of bringing special needs families from around the world together and disseminating invaluable information on the latest interventions and treatments for autism. Today, it has become an essential resource for parents around the world.

APM will soon be hosting its annual Autism Summit For Parents featuring some of the leading experts in the study and management of autism. At least 30 keynote sponsors and speakers will be presenting valuable insights on treatments, interventions, and support strategies.

The event will take place between 12 15 May 2023 and will be hosted online, thus saving families the time and costs related to travel and accommodation. Parents and family members are encouraged to join the event's global audience of some 25,000 attendees and be part of the growing community.

The Autism Conference will offer parents caring for children on the spectrum the opportunity to meet experts in different areas of the autism world. The Summit will benefit the entire autism communityparents, family members, allies, caregivers, and people on the spectrum will find inspiration and gain knowledge from this event.

Interested parties only need to visit APMs website and register for the free pass.

For more information, visit https://autismparentingsummit.com/

The Autism Summit 2023 will have much to offer parents and caregivers as a panel of notable keynote speakers will be explaining cutting-edge concepts based on the latest technologies science has to offer.

Dr. Neil Riordan is one of the early pioneers and experts in applied stem cell research and the founder and chairman of Medistem Panama, Inc., a leading stem cell laboratory and research facility. He is also the founder, chairman, and chief science officer of the Stem Cell Institute in Panama, specializing in the treatment of human diseases and conditions with adult stem cells.

Dr. Gabriel Belfort is the senior vice president of Clinical Development Sciences and Operations at Axial Therapeutics. A physician-scientist focusing on exploring molecular, cellular, and pre-clinical pharmacological aspects of central nervous system function, he serves as the medical director accelerating the clinical development of high-quality novel therapeutics.

Carmellina Stetson is the clinical director of Blue Balloon ABA, North Carolina with a masters degree in Applied Behavior Analysis with a concentration in Autism Studies. Stetson has extensive experience developing programs for individuals with ASD, valuing all individuals learning styles and preferences in order to help each individual reach the highest potential.

Dr. Erik Won is the president & chief medical officer of Wave Neuroscience, a biotechnology company developing innovative solutions to optimize brain function, including autism spectrum disorder. Wave technologies utilize computational neuroanalytics and brain imaging to customize treatment protocols with the aim of restoring optimal neurological function.

Dr. Theoharis Theoharides, a Professor of Pharmacology for 40 years and presently a professor at an institute of neuroImmune medicine, has received multiple awards for his work as well as his humanitarian efforts. He has published myriad scientific papers on neural conditions, including autism spectrum disorder, and has helped formulate unique dietary supplements with over 37 patents and trademarks, including some covering the use of luteolin in autism.

Over the four-day Autism Summit For Parents, these highly acclaimed individuals will be joined by 25 other experts who will speak on a wide range of topics that matter most to special needs families.

About the Publication:

Founded by Mark Blakey, Autism Parenting Magazine is the leading publication for parents of autistic children with an international following. The online magazine has expanded and now offers virtual summits and courses for parents that struggle with special needs children. Daily, the people at Autism Parenting Magazine inspire and entertain its global audience through social media posts. By providing informative content and autism resources, its goal is to improve the quality of life of families affected by autism.

###

For more information about Autism Parenting Magazine, contact the company here:

Autism Parenting MagazineMark Blakey+44(0)203 290 9176press@autismparentingmagazine.comKemp House, 160 City Road, London, EC1V 2NX, United Kingdom

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Join the Autism Summit For Parents Free Virtual Talks to Cover ... - GlobeNewswire

Single-Use Bioreactors Market is Expected to Reach $10.0 billion … – GlobeNewswire

Chicago, April 28, 2023 (GLOBE NEWSWIRE) -- The single-use bioreactor industry is expected to experience tremendous growth in the near future due to increased demand for more cost-effective and efficient production of biopharmaceuticals. The use of single-use bioreactors has the potential to reduce capital investment and operational costs, as well as increase production flexibility. This is expected to lead to a significant increase in the number of companies investing in and utilizing single-use bioreactors. Additionally, ongoing research and development of new technologies and systems, such as advanced sensors and advanced analytics, are expected to further drive growth in the near future. Finally, the growing global demand for biopharmaceuticals is expected to increase the demand for single-use bioreactors, further driving growth in the industry.

Single-Use Bioreactors market in terms of revenue was estimated to be worth $4.2 billion in 2023 and is poised to reach $10.0 billion by 2028, growing at a CAGR of 19.0% from 2023 to 2028 according to a latest report published by MarketsandMarkets. The factors driving the growth of this market include the increasing adoption of single-use bioreactors among startups and SMEs, lower operational complexity of single-use bioreactors compared to conventional stainless-steel bioreactors, reduced energy and water consumption, growing size of the biologics and biosimilars market, and technologically advanced offerings by players in single-use bioreactors. However, extractability and leachability issues regarding disposable of single-use components used in bioreactors and regulatory concerns related to single-use bioreactors are the major factors restraining the growth of this market to certain extent.

Download an Illustrative overview: https://www.marketsandmarkets.com/pdfdownloadNew.asp?id=49113750

Single-Use Bioreactors Market Scope:

Based on product, the single-use bioreactors market is segmented into single-use bioreactor systems, single-use media bags, single-use filtration assemblies, and other products. The single-use bioreactor systems segment dominated the single-use bioreactors market in 2022. Single-use bioreactor systems offer advantages such as low capital investment, low operating expenses, and lower environmental footprint.

Based on type, the single-use bioreactors market is segmented into stirred-tank single-use bioreactors, wave-induced single-use bioreactors, bubble-column single-use bioreactors, and other single-use bioreactors such as hybrid bioreactors and single-use bioreactors with vertically perforated discs. The stirred-tank single-use bioreactors segment dominated the market in 2022 owing to stirred-tank single-use bioreactors in the culturing of aerobic microbial cell cultures.

Based on the type of molecule, the global single-use bioreactors market is segmented into monoclonal antibodies (mAbs), vaccines, gene-modified cells, stem cells, and other molecules. In 2022, the monoclonal antibodies segment accounted for the largest share of the global single-use bioreactors market owing to the increasing demand for single-use bioreactors in the manufacturing of mAbs, owing to low investment costs and a reduction in time-intensive changeover procedures.

Based on the type of cell, the global single-use bioreactors market is segmented into mammalian cells, bacterial cells, yeast cells, and other cells (insect and plant cells). In 2022, the mammalian cells segment accounted for the largest share of the market. The increasing adoption of mammalian cells due to their post-translational modification capacity and molecular structure assembly that closely resembles proteins in humans are the major factors driving the growth of this segment.Based on application, the single-use bioreactors market is segmented into research & development, process development, and bioproduction. The bioproduction segment accounted for the largest share of the market in 2022 and is projected to register the highest CAGR during the forecast period owing to the increasing use of single-use bioreactors in biomanufacturing and the increasing demand for single-use bioreactors in CMOs due to the advantages it offers, such as flexibility and easy scalability.

Based on end users, the single-use bioreactors market is segmented into pharmaceutical & biotechnology companies, CROs & CMOs, and academic & research institutes. The pharmaceutical & biotechnology companies segment accounted for the largest share of the single-use bioreactors market in 2022 owing to the increasing R&D initiatives by pharmaceutical, biopharmaceutical, and biotechnology companies and growing production of biologics & biosimilars.

Based on region, single-use bioreactors market is segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. In 2022, Asia Pacific has the fastest growth rate owing to The factors such as the growing biopharmaceutical industry, growing investments by pharmaceutical & biotechnology companies in the Asia Pacific region, and the growing number of CROs & CMOs in different countries in the region are supporting the growth of the market in the region.

Key Market Players:

Major players operating in the single-use bioreactors market include Sartorius AG (Germany), Thermo Fisher Scientific (US), Danaher Corporation (US), and Merck Millipore (Germany). These companies have manufacturing units as well as strong distribution networks across key regions, such as North America, Europe, Asia Pacific, Latin America and the Middle East & Africa. They have an established portfolio of reputable services, a robust market presence, and strong business strategies. Furthermore, these companies have a significant market share, and vast service portfolio.

Enquiry Before Buying: https://www.marketsandmarkets.com/Enquiry_Before_BuyingNew.asp?id=49113750

Hypothetic Challenges of Single-Use Bioreactors Market in Near Future:

Top 3 Use Cases of Single-Use Bioreactors Market:

Recent Developments:

Get 10% Free Customization on this Report: https://www.marketsandmarkets.com/requestCustomizationNew.asp?id=49113750

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Single-Use Bioreactors Market is Expected to Reach $10.0 billion ... - GlobeNewswire

Fort Worth Biotech Innovator Honored With Lifetime Achievement … – dallasinnovates.com

Stella Robertson [Image: Courtesy photo, iamguru/istockphoto, DI]

Longtime Fort Worth biotech innovator and investor Stella Robertson has been honored with a 2023 ARVO Foundation award recognizing her lifetime of work in vision research and philanthropy.

Robertsons expertise and guidance has helped countless entrepreneurs and start-ups, making her a true inspiration in biotech and the field of ophthalmology. The award was given in recognition for her long-standing support of the Women in Eye and Vision Research (WEAVR) initiative and her generous philanthropic support of the foundation.

The ARVO Foundation is the philanthropic arm of ARVO (The Association of Research in Vision and Ophthalmology). WEAVR is the Foundation initiative supporting women in vision research.

Robertson and Suchi Acharya, founder and CEO of Ayuvis Research in Fort Worth, were speakers at ARVOs B2B education course this year. The course, Bench to Bedside, is a translational research and pitch workshop for ARVO members.

I have always wanted my research to make a difference in peoples lives, to solve problems and help them have a better life, Robertson said in an Arvo Q&A in 2019.

The scientist is known for her work at Fort Worth-based Alcon where she launched roughly 17 products, and is co-founder at Bios Partners, a venture capital firm focusing on life sciences based in the city. Bios Partners was founded in 2015 by Robertson, along with managing partners Aaron G.L. Fletcher and Les Kreis.

With over 25 years of experience in pharmaceutical research and development, she has a wealth of knowledge to share.

During her time at Alcon, where she was vice president in R&D at Alcon Laboratories, Inc., a division of Novartis, she grew and led organizations responsible for the ophthalmic pipeline, including pharmaceuticals and medical devices. Robertson developed some of the first human ocular cell lines used for drug discovery and successfully launched sixteen ophthalmic medications to treat ocular allergy, pain, inflammation, glaucoma, uveitis, and infection.

Her research interests are diverse, ranging from local immune and inflammatory mechanisms to diagnostics and drug delivery. Robertson is a published author and also holds several patents.

The scientist received a Ph.D. in biology-immunology from Johns Hopkins University, was an Arthritis Foundation postdoctoral research fellow at UTHSC Dallas, and completed the Program for Management Development at Harvard Business School.

Today, as the founder of Arrochar Consulting, Robertson specializes in due diligence, translational research, product development and life sciences, providing support to entrepreneurs and start ups in emerging technology.

Robertsons passion for helping others extends beyond her consulting work. She volunteers and mentors with TECH Fort Worth, a local non-profit incubator/accelerator, and local university entrepreneurial program. She also serves as a corporate board member, board observer, and scientific advisor for early-stage companies. In addition, Robertson is a member and investor with Cowtown Angels, an angel investment network based in Fort Worth.

My focus now is on giving back, according to the ARVO Q&A. Her dedication to the field is apparent through her involvement in various organizations such as Women in Ophthalmology and ARVO (IM section). Shes served on multiple committees and sits on the ARVO Foundation Board.

Robertson is credited with expanding the research and entrepreneurial community in Fort Worth and encouraging students to stay in STEM education. Her advice to young women scientists is to get the best training in their chosen field, find something that makes them feel fulfilled, and persevere through many nos in their careers, she said in the Q&A.

A career in research is a life choice, Robertson has advised. But, she adds, dont forget to take time for yourself and family. Together you will delight in and discover the world, nature, and research again through their eyes.

Quincy Preston contributed to this report.

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Slated to be built in Fort Worth's Historic Southside neighborhood, the planned $70 million museum will get the city funding once the balance for the project has been raised. Designed by the New York office of Denmark-based Bjarke Ingels Group, the building will house the museum on its second level, with a business incubator, restaurant, 250-seat amphitheater, and storefronts at ground level. Literally and figuratively, it was designed to be a beacon of light in an area that has been dark for a very long time, says Jarred Howard, principal of the project's developer.

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Bold new therapy delivery method shows initial promise as treatment for Duchenne muscular dystrophy – Medical Xpress

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Graphical abstract. Credit: Cell (2023). DOI: 10.1016/j.cell.2023.03.033

Doug Millay, Ph.D., a scientist with the Division of Molecular Cardiovascular Biology at Cincinnati Children's has dedicated his career to revealing the most fundamental mechanisms of skeletal muscle development. He has been a leader in characterizing how two "fusogens" called Myomaker and Myomerger mediate the entry of stem cells into mature muscle cells to build the tissue that humans depend upon for movement, breathing, and survival.

Now, some of the basic discoveries made by Millay and colleagues are translating into a potential treatment for people living with Duchenne muscular dystrophy (DMD). Their latest research, published April 12, 2023, in the journal Cell, reveals that in mice, modified viruses, engineered with Myomaker and Myomerger, result in specific fusion with muscle cells. These viruses can therefore be used as a vector to deliver a vital gene needed for muscle function that is mutated in people with DMD.

A key unknown prior to this work was whether proteins like Myomaker and Myomerger, which mainly function on cells, could even work on viruses. First author Sajedah Hindi, Ph.D., also with the Division of Molecular Cardiovascular Biology at Cincinnati Children's and a leading member of the research team, took on the challenge to test this idea.

Hindi first designed a strategy to place Myomaker and Myomerger on the surface of viruses and showed that they were functional in cultured cells. She went on to leverage her extensive experience in skeletal muscle biology to test the efficacy of these novel vectors in mice.

"This modified viral vector appears to be a promising tool for delivering a potential lifelong supply of the gene that is absent in people with DMD," Millay says. "The unique advantages of this vector provide an opportunity to significantly impact gene therapy for a myriad of muscle diseases."

DMD is a rare and fatal genetic muscle disease characterized by the lack of a critical membrane-stabilizing protein called dystrophin, which results in progressive muscle degeneration and weakness. DMD primarily strikes boys, occurring in about 1 of every 3,500 male births worldwide.

Doctors often diagnose the disease between ages 3 and 6 when children show early signs of significant muscle weakness, such as delayed ability to sit, stand, or walk and difficulties learning to speak. Over time, DMD becomes fatal as muscle degeneration disrupts lung and heart function.

There is no cure. However, lifespans have been extended and quality of life has been improved for many through physical therapy and medications to address certain symptoms. Some gene therapy clinical trials are evaluating the use of adeno-associated virus (AAV) as the delivery vector, and there is hope that these strategies work. However, novel vectors, such as the lentiviruses described by Millay and colleagues, have the potential to improve long-term delivery of therapeutic material for muscular dystrophies.

Conducting the numerous experiments involved in this study took Hindi and collaborators about four years to complete. A significant collaborator who helped initiate the project was Benjamin Podbilewicz, from the Technion-Israel Institute of Technology, Haifa, Israel. Their findings include:

Much more research will be needed to further develop this discovery into a treatment that could someday benefit people with DMD. Even more work will be needed to determine which other muscle diseases might be treated with this lentivirus vector.

"We envision that this concept, transferring a naturally occurring process within muscle to membrane vehicles, could revolutionize delivery of therapeutic material to skeletal muscle to improve genetic conditions such as muscular dystrophy and conditions associated with muscle loss and weakness," Millay says.

More information: Sajedah M. Hindi et al, Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery, Cell (2023). DOI: 10.1016/j.cell.2023.03.033

Journal information: Cell

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Bold new therapy delivery method shows initial promise as treatment for Duchenne muscular dystrophy - Medical Xpress