StemCell Therapy

Its been known for decades that astronauts immune systems become suppressed in space, leaving them vulnerable to disease, but the exact mechanisms of immune dysfunction have remained a mystery now a Cornell undergraduate has found a potential solution.

A biological and mechanical engineering double major in the College of Engineering, Rocky An 23 published his theory, MRTF May be the Missing Link in a Multiscale Mechanobiology Approach toward Macrophage Dysfunction in Space, Sept. 12 in Frontiers in Cell and Developmental Biology.

An reviewed the last 20 years of literature on the behavior of macrophages key cells in the bodys immune response in space and recent research about how macrophages respond to forces in normal gravity, identifying a transcription factor that could prove to be the missing piece of the puzzle.

I just kept asking questions about how the data is presented, An said. There are these two really important papers, in particular, one a review of how macrophages are suppressed in microgravity, and another about the mechanobiology of macrophages. I was able to connect these two papers, and that's when the idea came to me. I was really excited, as it was kind of a eureka moment for me.

In space, the lack of gravity changes the shape of the immune cell, and scientists have suspected that changes to the cytoskeleton, the filamented infrastructure of the cell, were involved in immune dysfunction. Recent studies in normal gravity have shown that disturbing the cytoskeleton of macrophages reduces the transport of a particular protein, a transcription factor important for immune response, to the nucleus.

By comparing the studies of cells in microgravity and analyzing the modes of study and associated timescales whether macrophages were actually studied in space, or on a parabolic airplane, or in a simulation of microgravity in the lab An was able to point to this protein, Myocardin-Related Transcription Factor (MRTF), as a probable culprit in immune system dysfunction.

I think its a pretty convincing argument that MRTF is a big part of the problem, An said. I hope it will inspire future studies that really focus on that one protein and the cytoskeleton, and maybe it could be the first step towards a spaceflight immune treatment.

The paper suggests that MRTF could be implicated in the stress on the cardiovascular health of astronauts as well. An also points to other factors that may play a role in immune dysfunction and notes that further research is needed to understand how MRTF interacts with the macrophage nucleus in microgravity.

While An worked independently on the publication, he credits his Cornell professors (including Mingming Wu, professor, and Minglin Ma, associate professor, both of biological and environmental engineering in the College of Agriculture and Life Sciences; Brian J. Kirby, the Meinig Family Professor of Engineering; and Donna Cassidy Hanley, senior research associate in the College of Veterinary Medicine, among many other teachers) for modeling an interdisciplinary approach and encouraging inquiry.

Even before enrolling at Cornell, An had contact with the lab of Theodore Clark, professor of microbiology and immunology in the College of Veterinary Medicine, where An has engaged in research since his freshman year. He also credits his experience on the Cornell iGEM (Genetically Engineered Machines) Project Team, with advising from Jan Lammerding, professor in the Meinig School of Biomedical Engineering, as helping him develop as a scientist.

The biggest help has been the professors and the way theyve taught my classes, An said. And then with research in the lab and my project team, there have been a lot of opportunities to learn independently and ask our own questions.

An also gained valuable experience in summer internships. In 2021, he was chosen as a research associate for NASAs Space Life Sciences Training Program, where he studied the impact of microgravity on cells and co-authored his first paper, an optimization of a modeling framework for studying cells in microgravity.

An then spent the summer of 2022 as an Amgen Scholar at Harvard Universitys Wyss Institute, where he worked in the area of mechanical immunotherapy, exploring therapies that work by manipulating cells structure.

Ive always been interested in cells, but also the mechanics, how cells react to forces, An said. I like this approach because its somewhat new, and I think it's very different from what you generally learn in biology, where everything's a series of chemical reactions. I really enjoy the interaction between the two fields.

Support for Ans publication came from the Cornell Open Access Publication Fund.

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Undergrad publishes theory on immune dysfunction in space | Cornell Chronicle - Cornell Chronicle

September 19, 2022

Contact:Thonnia Lee, Office of Communications, Public Relations and Marketing

TUSKEGEE, Alabama-- Tuskegee University has been named a Fulbright Historically Black College and University (HBCU) Institutional Leader for 2022.

For the third consecutive year, the U.S. Department of States Bureau of Educational and Cultural Affairs (ECA) is recognizing selected HBCUs strong engagement with the Fulbright Program, the U.S. government's flagship international academic exchange program. Fulbright HBCU Institutional Leaders have demonstrated noteworthy support for Fulbright exchange participants during the 2020-2021 and 2021-2022 academic years and have promoted Fulbright program opportunities on campus. Tuskegee University is new to the list this year.

The announcement of the 19 Fulbright HBCU Institutional Leaders was made by the State Department as HBCU leaders prepare to gather in Washington, D.C., and virtually for the White House Initiative on HBCUs National HBCU Week Conference, and Fulbright opportunities will be highlighted in events such as the career and recruitment fair during this week.

Assistant Secretary of State for Educational and Cultural Affairs Lee Satterfield commended the HBCUs receiving the Fulbright HBCU Institutional Leader designation this year, noting that HBCUs are an important part of the American and global higher education communities, providing life-changing exchange opportunities for American and international students, faculty, and administrators alike. I hope that these institutions success encourages all HBCUs to engage further with Fulbright and with the State Department.

This is an exceptional note of distinction, said President Charlotte P. Morris. We have always known the caliber of students and faculty whose vigorous academic work influences our society in a variety of areas. This acknowledgment will help our students and the broader community see the enormous value our university brings.

On Nov. 3, a Fulbright HBCU Virtual Workshop will feature representatives of Fulbright HBCU Institutional Leaders sharing best practices for HBCUs to leverage Fulbright Program engagement to support students and faculty, increase campus internationalization, and build global networks. This event is open to the public and is specifically designed for HBCU faculty, staff, and stakeholders.

Our goals for academic excellence mapped out in our Strategic Plan are confirmed once again by this acknowledgment, said Provost Dr. S. Keith Hargrove. We are providing academic tools for scholars and researchers to develop and contribute their best work. By supporting this kind of academic brilliance, it confirms our commitment to remain an HBCU that attracts and maintains a strong community of global scholars.

The Fulbright Program is the U.S. government's flagship international academic exchange program. Since its inception over 75 years ago, the Fulbright Program has given over 400,000 talented and accomplished students, scholars, teachers, artists, and professionals of all backgrounds and fields the opportunity to study, teach, and conduct research abroad, exchanging ideas, and contributing to finding solutions to important international problems.

Each year, the U.S. Congress appropriates funds to the U.S. Department of State to sponsor the Fulbright Program. Many foreign governments contribute substantially as well. Additional funding and in-kind funding is provided by U.S. and foreign host institutions, non-governmental organizations, private organizations, corporate partnerships, and individual donors.

Over the years, we have had faculty and student Fulbright awardees representing Tuskegee around the globe, said Dr. Rhonda Collier, director of the TU Global Office, Fulbright Faculty Liaison and Fulbright Program Advisor. Im so proud of the work were doing to support their study and research.

Over many years the Fulbright Program has designed and implemented a wide range of initiatives to increase participant diversity and inclusion. The program strives to ensure that its participants reflect the diversity of U.S. society and societies abroad. Fulbrighters come from all backgrounds and are selected through an open, merit-based competition, regardless of their race, color, national origin, sex, age, religion, geographic location, socio-economic status, disability, sexual orientation, or gender identity. Information on the Fulbright Programs diversity and inclusion initiatives is detailed on the Fulbright U.S. Student Program website.

For more information on the Fulbright Program, visit http://eca.state.gov/fulbright or contact the Bureau of Educational and Cultural Affairs Press Office by e-mail ECA-Press@state.gov.

Stories about the positive impact of the Fulbright Program over its first 75 years can be found at: https://fulbright75.org

Follow the Fulbright Programs social media accounts and websites for highlights on HBCUs and Fulbright:

2022 Tuskegee University

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Tuskegee University is named HBCU Institutional Leader by Fulbright Program - Tuskegee University

News Release

Thursday, September 8, 2022

Gene augmentation rescues cilia defects in light-sensing cells derived from patients with blinding disease.

Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood. Using patient-derived retina organoids (also known as retinas-in-a-dish), the researchers discovered that a type of LCA caused by mutations in the NPHP5 (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body. The findings not only shed light on the function of NPHP5 protein in the primary cilium, but also led to a potential treatment for this blinding condition. NEI is part of the National Institutes of Health.

Its so sad to see little kids going blind from early onset LCA. NPHP5 deficiency causes early blindness in its milder form, and in more severe forms, many patients also exhibit kidney disease along with retinal degeneration, said the studys lead investigator, Anand Swaroop, Ph.D., senior investigator at the NEI Neurobiology Neurodegeneration and Repair Laboratory. Weve designed a gene therapy approach that could help prevent blindness in children with this disease and one that, with additional research, could perhaps even help treat other effects of the disease.

LCA is a rare genetic disease that leads to degeneration of the light-sensing retina at the back of the eye. Defects in at least 25 different genes can cause LCA. While there is an available gene therapy treatment for one form of LCA, all other forms of the disease have no treatment. The type of LCA caused by mutations in NPHP5 is relatively rare. It causes blindness in all cases, and in many cases it can also lead to failure of the kidneys, a condition called Senior-Lken Syndrome.

Three post-doctoral fellows, Kamil Kruczek, Ph.D., Zepeng Qu, Ph.D., and Emily Welby, Ph.D., together with other members in the research team collected stem cell samples from two patients with NPHP5 deficiency at the NIH Clinical Center. These stem cell samples were used to generate retinal organoids, cultured tissue clusters that possess many of the structural and functional features of actual, native retina. Patient-derived retinal organoids are particularly valuable because they closely mimic the genotype and retinal disease presentation in actual patients and provide a human-like tissue environment for testing therapeutic interventions, including gene therapies. As in the patients, these retinal organoids showed defects in the photoreceptors, including loss of the portion of the photoreceptor called outer segments.

In a healthy retina, photoreceptor outer segments contain light-sensing molecules called opsins. When the outer segment is exposed to light, the photoreceptor initiates a nerve signal that travels to the brain and mediates vision. The photoreceptor outer segment is a special type of primary cilium, an ancient structure found in nearly all animal cells.

In a healthy eye, NPHP5 protein is believed to sit at a gate-like structure at the base of the primary cilium that helps filter proteins that enter the cilium. Previous studies in mice have shown that NPHP5 is involved in the cilium, but researchers dont yet know the exact role of NPHP5 in the photoreceptor cilium, nor is it clear exactly how mutations affect the proteins function.

In the present study, researchers found reduced levels of NPHP5 protein within the patient-derived retinal organoid cells, as well as reduced levels of another protein called CEP-290, which interacts with NPHP5 and forms the primary cilium gate. (Mutations in CEP-290 constitute the most common cause of LCA.) In addition, photoreceptor outer segments in the retinal organoids were completely missing and the opsin protein that should have been localized to the outer segments was instead found elsewhere in the photoreceptor cell body.

When the researchers introduced an adeno-associated viral (AAV) vector containing a functional version of NPHP5 as a gene therapy vehicle, the retinal organoids showed a significant restoration of opsin protein concentrated in the proper location in outer segments. The findings also suggest that functional NPHP5 may have stabilized the primary cilium gate.

The study was funded by the NEI Intramural program. Patient samples were collected at the NIH Clinical Center.

NEI leads the federal governments efforts to eliminate vision loss and improve quality of life through vision researchdriving innovation, fostering collaboration, expanding the vision workforce, and educating the public and key stakeholders. NEI supports basic and clinical science programs to develop sight-saving treatments and to broaden opportunities for people with vision impairment. For more information, visit https://www.nei.nih.gov.

About the NIH Clinical Center:The NIH Clinical Center is the worlds largest hospital entirely devoted to clinical research. It is a national resource that makes it possible to rapidly translate scientific observations and laboratory discoveries into new approaches for diagnosing, treating, and preventing disease. Over 1,600 clinical research studies are conducted at the NIH Clinical Center, including those focused on cancer, infectious diseases, blood disorders, heart disease, lung disease, alcoholism and drug abuse. For more information about the Clinical Center, visit:https://www.cc.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Kruczek K, Qu Z, Welby E, et al. In vitro modeling and rescue of ciliopathy associated with IQCB1/NPHP5 mutations using patient-derived cells. Stem Cell Reports. Sept 8, 2022.

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NIH researchers develop gene therapy for rare ciliopathy - National Institutes of Health (.gov)

Repeated muscle injections with Engensis (VM202), Helixmiths investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial.

While the sample size was too small to determine the therapys efficacy, muscle biopsies were collected and will be examined to further evaluate the underlying mechanisms of Engensis.

These data suggest that high dose, repeated treatments of Engensis, were safe and well tolerated, providing a great deal of flexibility in designing dosing schemes for future clinical studies, Helixmith stated in a company press release.

Trial analysis will continue once the full dataset is available, and the company plans to present such findings at a future conference. The next steps for Engensis development will be determined at that time.

Engensis is a non-viral gene therapy that uses Helixmiths proprietary small circular DNA molecule to deliver the hepatocyte growth factor (HGF) gene to cells in the muscle environment.

HGF provides instructions to produce a protein of the same name that helps the body form new blood vessels, prevents muscle loss, and promotes the growth and survival of nerve cells. The therapyis delivered via intramuscular (into-the-muscle) injections.

Helixsmith believes that by increasing HGF production, Engensis has the potential to promote nerve cell and muscle regeneration, thereby countering the progressive loss of motor control that characterizes ALS.

The therapy has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration, both of which are intended to speed its clinical development and regulatory review.

A previous open-label Phase 1/2 trial (NCT02039401) found that four once-weekly intramuscular injections of Engensis (to a total dose of 64 mg) were safe and well-tolerated among 18 ALS patients. Signs that the therapy could slow disease progression were also observed.

These promising findings prompted the launch of a placebo-controlled Phase 2a trial, called REViVALS-1A (NCT04632225), which began patient enrollment last year. A total of 18 ALS patients experiencing motor symptoms in their limbs for four years or less were recruited at four sites in the U.S. and one in Korea.

Participants were randomized in a 2:1 ratio to receive three cycles of either Engensis or a placebo: at studys start, at two months, and at four months. Each cycle consisted of two days of injections to upper and lower limb target muscles, spaced two weeks apart (64 mg total of Engensis or a placebo).

This meant that Engensis-treated patients received a total of 192 mg of medication over the four-month period. All participants were monitored for six months from the studys start.

The trials main goal was to assess the safety and tolerability of Engensis, while efficacy measures were included as exploratory outcomes. These included changes in disability, muscle and lung function, survival, ALS-specific health-related quality of life, and the levels of muscle shrinkage biomarkers.

Top-line data showed that the investigational treatment was generally safe and well-tolerated, with no difference in the frequency of adverse events observed between the Engensis and placebo groups (83% for each).

One case of bronchitis a condition characterized by inflammation in the main airways of the lungs due to infection was observed in the Engensis group but was determined unrelated to treatment.

Injection site reactions were reported by 50% of Engensis-treated patients and 66.7% of those in the placebo group. Most of these reactions were mild or moderate in severity and temporary; no participant discontinued treatment due to the number of injections.

According to Helixmith, efficacy was unable to be evaluated due to the fact that four participants dropped out early from the small study.

Still, muscle tissue biopsies were obtained from injection sites to undergo analyses of muscle atrophy (shrinkage) biomarkers and others.

Since data on Engensis underlying mechanisms have been largely based on animal models, these results are expected to provide valuable information on the understanding of the mechanisms of actions of Engensis, and its effects on the [activity] of human genes, which will greatly help in the development of innovative medicines, the company stated in the release.

Helixmith greatly appreciates the generous and eager participation of the ALS patients, the company added.

Engensis is also being investigated across a range of conditions associated with deficits in circulation, and nerve and/or muscle damage, such as diabetic neuropathy, coronary artery disease, and Charcot-Marie-Tooth disease.

More than 500 patients have been treated with Engensis to date across 10 clinical trials and six different diseases, according to Helixmith. Data from these studies have also supported the therapys favorable safety profile and its ability to increase HGF production.

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Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |... - ALS News Today

DUBLIN--(BUSINESS WIRE)--The "Cancer Gene Therapy Market By Therapy, By End User: Global Opportunity Analysis and Industry Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Cancer Gene Therapy Market was valued at $1,389.42 million in 2020 and is estimated to reach $11,359.35 million by 2030, registering a CAGR of 23.3% from 2021 to 2030.

Cancer gene therapy is a technique used for the treatment of cancer where therapeutic DNA is being introduced into the gene of the patient with cancer. Owing to the high success rate during the preclinical and clinical trials, cancer gene therapy has gained popularity.

Many techniques are used for cancer gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of the gene whose function is abnormal. Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells.

The rise in the prevalence of cancer, the benefits of cancer gene therapy over conventional cancer therapies, and the advancement in this field are the major factors that drive the market growth.

In addition, the surge in government support, ethical acceptance of gene therapy for cancer treatment, and rise in biotechnological funding encouraging the R&D activities for cancer gene therapy and thus fuel the growth of the cancer gene therapy market.

In addition rise in awareness regarding cancer gene therapy is a major factor that drives the global cancer gene therapy market growth.

In addition, an increase in government support for research in gene therapy, ethical acceptance of gene therapy for cancer treatment, and a rise in the prevalence of cancer boost the growth of the cancer gene therapy market. However, the high cost associated with the treatment and unwanted immune responses is expected to restrain the market growth.

Key Benefits For Stakeholders

Key Market Segments

By Therapy

By End User

By Region

Key Market Players

Key findings of the Study

Key Topics Covered:

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: CANCER GENE THERAPY MARKET, BY THERAPY

CHAPTER 5: CANCER GENE THERAPY MARKET, BY END USER

CHAPTER 6: CANCER GENE THERAPY MARKET, BY REGION

CHAPTER 7: COMPANY LANDSCAPE

CHAPTER 8: COMPANY PROFILES

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

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Global Cancer Gene Therapy Market Report 2022: Benefits of Gene Therapy Over Conventional Therapies Driving Adoption - ResearchAndMarkets.com -...

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

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

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

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

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

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

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

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

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

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

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

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

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

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

The total budget for the training program was not disclosed.

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

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

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

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

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

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

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

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

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

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

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

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

ALDERLEY PARK, England--(BUSINESS WIRE)--Charles River Laboratories International, Inc. (NYSE: CRL) and Cure AP-4, a non-profit foundation dedicated to raising funds and awareness about Adapter-Protein 4 Hereditary Spastic Paraplegia (AP-4 HSP), today announced a manufacturing collaboration. Charles River, a contract research and development manufacturing organization (CRO/CDMO), will provide High Quality (HQ) plasmid DNA for Cure AP-4s Phase I/II gene therapy trials against AP-4 HSP.

Founded in 2016 by the families of two newly diagnosed AP-4 HSP (SPG47) patients, Molly Duffy and Robbie Edwards, Cure AP-4s gene therapy treatment will look to address the root cause of AP-4 HSP, a rare neurodegenerative disorder, and is intended as a one-time, curative treatment for the patient.

What is AP-4 HSP? AP-4 HSP, also known as AP-4 Deficiency Syndrome, includes four sub-types of HSP: SPG47, SPG50, SPG51 and SPG52. Each of these HSP sub-types is associated with a defective autosomal recessive gene which causes a failure in the AP-4 Adaptor Complex. The phenotype and prognosis for each sub-type is extremely similar. Patients afflicted with any of the AP-4 HSP genetic disorders generally present with symptoms including global developmental delay, microcephaly, seizures, brain malformation, and hypotonia (low-muscle tone). The few patients who learn to walk independently tend to lose that ability a few months or few years later as they develop hypertonia (high-muscle tone) and muscle spasticity. Of the 249 currently confirmed global AP-4 HSP cases, most patients experience mobility in some or all extremities as the disorder progresses and are severely intellectually challenged.

Plasmid DNA Manufacturing ServicesThe collaboration will leverage Charles Rivers market leading expertise in plasmid DNA production, specifically HQ plasmid, which combines key features of GMP manufacture with rapid turnaround times to accelerate the timeline to clinic. DNA plasmids are a critical starting material for many cell and gene therapy therapeutics and demand continues to outstrip supply. In response to this, Charles River recently announced the opening of a state-of-the-art HQ plasmid manufacturing center of excellence to address these supply shortages and support the growing needs of the cell and gene therapy field.

Charles River, with the acquisitions of Cognate BioServices, Cobra Biologics, and Vigene Biosciences in 2021, has extended its comprehensive cell and gene therapy portfolio to include CDMO capabilities spanning viral vector, plasmid DNA and cellular therapy production for clinical through to commercial supply.

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About Cure AP-4Cure AP-4, originally known as Cure SPG47, was founded in 2016 by the families of two newly diagnosed SPG47 patients, Molly Duffy and Robbie Edwards. At the time there were only nine other documented cases worldwide, and due to the extreme rarity of the disorder there are no known treatments or cures.

About Charles RiverCharles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit http://www.criver.com.

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Charles River and Cure AP-4 Announce Gene Therapy Manufacturing Collaboration - Business Wire

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

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

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

ABOUT MYRTELLE

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

ABOUT CANAVAN DISEASE

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

Forward-Looking Statements

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

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

IRVINE, Calif. & BASEL, Switzerland--(BUSINESS WIRE)--Urovant Sciences, a wholly owned subsidiary of Sumitovant Biopharma Ltd., receives coveted Best in Category award for an interim 12-week analysis from the ongoing Phase 2a trial of an investigational novel gene therapy product, URO-902 (plasmid human cDNA encoding maxi-K channel). The award-winning abstract was presented at the 2022 International Continence Society annual meeting on September 8, 2022. The 2022 ICS Annual meeting is being held September 7-10, 2022, in a hybrid format with both online and in person participation (Vienna, Austria).

According to ICS, this honor is awarded to the highest-scoring abstract in each category. Scores are awarded by the ICS scientific committee members, external reviewers, and scientific session chairs. Abstracts are judged based on criteria of scientific merit, originality/topicality, and clinical relevance. Review the full 2022 Abstract Awards List here.

The podium presentation at ICS 2022 took place on Thursday, September 8, at 10:20 Central European Time (CET). Presentation #6 in Scientific Podium Session S1, Best Urology, was titled, Efficacy and Safety of a Novel Gene Therapy (URO-902; PVAX/HSLO) in Female Patients with Overactive Bladder Syndrome and Urge Urinary Incontinence: Results from a Phase 2A Trial. The presentation described a prespecified, 12-week interim analysis of a 48-week multicenter, randomized, double-blind, placebo-controlled, dose-escalation study (NCT04211831). URO-902 was administered using direct intradetrusor injections via cystoscopy under local anesthesia. The presenting author was Kenneth Peters, M.D., Principal Investigator, and Chief of the Department of Urology at Beaumont Hospital, Royal Oak; Medical Director of the Beaumont Womens Urology and Pelvic Health Center; and Professor and Chair of Urology of the Oakland University William Beaumont School of Medicine in Rochester, Mich.

We are delighted that this presentation has received the Best in Category Prize: Overactive Bladder, reflecting the high-quality scientific research involved, said Dr. Peters. The promising interim safety and efficacy findings from this prespecified analysis indicate that URO-902 has potential as a therapeutic option for overactive bladder patients who have failed oral pharmacologic therapy.

At week 12, both URO-902 24 mg and 48 mg were associated with clinically relevant improvement in mean daily micturition (urination), urgency episodes, UUI episodes, OAB questionnaire symptom bother score, and proportion of patient global impression of change responders. Treatment-emergent adverse events occurred in 45.5% of patients receiving URO-902 24 mg, 46.2% receiving 48 mg, and 50.0% receiving placebo. The most commonly occurring adverse event was urinary tract infection (0% in individuals receiving the 24 mg dose of URO-902; 15.4% in those receiving the 48 mg dose; and 3.8% in those receiving placebo). One patient in the 48 mg arm of the study had asymptomatic elevated post-void residual urine volume at week 2; this resolved spontaneously and did not require catheterization.

URO-902 is a unique potential treatment for OAB. It brings together the accessibility of the anatomy of the condition with a new innovative approach to therapy, said Sef Kurstjens, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Urovant Sciences. Later this year, Urovant anticipates 48-week data from the Phase 2a trial, at that point, well have a greater sense of the durability of the therapy and our proposed next steps.

The data were first presented earlier this year at the 2022 annual meeting of the American Urological Association (AUA2022) in New Orleans, La., from May 13-16, 2022.

About Overactive Bladder

Overactive bladder (OAB) is a clinical condition that occurs when the bladder muscle contracts involuntarily. Symptoms may include urinary urgency (the sudden urge to urinate that is difficult to control), urgency incontinence (unintentional loss of urine immediately after an urgent need to urinate), frequent urination (usually eight or more times in 24 hours), and nocturia (waking up more than two times in the night to urinate).1

While 33 million US adults experience the bothersome symptoms of OAB, approximately 546 million people 20 years are affected by OAB worldwide. 1,2

About the Phase 2a Study of URO-902

The 48-week multicenter study was a randomized, double-blind, placebo-controlled trial to evaluate the efficacy, safety, and tolerability of a single physician administered dose of URO-902, a novel gene therapy being developed for patients with OAB who have not been adequately managed with oral or transdermal pharmacologic therapy. URO-902 is administered via direct intradetrusor injections into the bladder wall under local anesthesia in patients who are experiencing OAB symptoms and urge urinary incontinence (UUI).

The Phase 2a trial enrolled 80 female patients in two cohorts: the first cohort received either a single administration of 24 mg of URO-902 or matching placebo, and the second cohort received 48 mg of URO-902 or matching placebo into the bladder wall. Multiple outcome measures were explored, including the effect on the number of micturitions, urgency episodes, and quality-of-life indicators compared to placebo, 12 weeks post-administration, as well as an assessment of the safety and tolerability of this potential new therapy. Patients were followed for up to 48 weeks after initial administration.

About URO-902

URO-902 (plasmid human cDNA encoding maxi-K channel) has the potential to be the first gene therapy for patients with OAB. If approved, this innovative treatment has the potential to address an unmet need for patients who have failed oral pharmacologic therapies.

References: 1. Irwin DE, Kopp ZS, Agatep B, Milsom I, Abrams P. Worldwide prevalence estimates of lower urinary tract symptoms, overactive bladder, urinary incontinence and bladder outlet obstruction. BJU Int. 2011;108(7):1132-1138. doi:10.1111/j.1464-410X.2010.09993.x

2. Leron E, Weintraub AY, Mastrolia SA, Schwarzman P. Overactive bladder syndrome: evaluation and management. Curr Urol. 2017;11:117-125. doi:10.1159/000447205

About Urovant Sciences

Urovant Sciences is a biopharmaceutical company focused on developing and commercializing innovative therapies for areas of unmet need, with a dedicated focus in Urology. The Companys second product candidate, URO-902, is a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., intends to bring innovation to patients in need in urology and other areas of unmet need.

About Sumitovant Biopharma

Sumitovant is a technology-driven biopharmaceutical company accelerating development of new potential therapies for patients with high unmet medical need. Through our subsidiary portfolio and use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovants subsidiary portfolio includes wholly-owned Enzyvant, Urovant, Spirovant, and Altavant, and one majority-owned subsidiary that is publicly listed: Myovant (NYSE: MYOV). Sumitomo Pharma is Sumitovants parent company. For more information, please visit http://www.sumitovant.com.

UROVANT, UROVANT SCIENCES, the UROVANT SCIENCES logo are trademarks of Urovant Sciences GmbH, registered in the U.S. and in other countries. All other trademarks are the property of their respective owners. 2022 Urovant Sciences. All rights reserved.

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Urovant Sciences Receives Best in Category Award for Abstract Highlighting Investigational Novel Gene Therapy, URO-902, Presented at 2022...

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

At 44, Janet Waterhouse should have been the picture of health; a former Division I soccer player, she taught yoga, enjoyed running, and didnt drink alcohol. Despite her healthy and active lifestyle, over a span of decades she experienced a number of unexplained symptoms.

Her symptoms continued to worsen into her 20s when she began to sporadically lose function of her hands and experience severe bouts of vertigo. Most doctors attributed her symptoms to stress and anxiety. During this time, Waterhouse was seeing a pain management specialist, who was concerned enough about her worsening symptoms to run a blood test, where he found irregularly shaped blood cells, called acanthocytes.

A series of serendipitous referrals led Waterhouse to Ali Hamedani, an assistant professor of neurology and ophthalmology in the Perelman School of Medicine. Based on her symptoms and exam, he suspected a genetic condition called chronic progressive external ophthalmoplegia (CPEO) and referred her to Laynie Dratch, a certified genetic counselor in the Penn Neurogenetics Therapy Center, for genetic testing.

In May of 2022, Dratch gave Waterhouse what she had been chasing for decades: a diagnosis. When the genetic counselor told me they found the genetic mutation they were looking for, I cried for a solid five minutes out of relief, Waterhouse says.

Waterhouses case of CPEO was found to be caused by a variation on her RRM2B gene, which affects the mitochondria in her cells. While the condition is very rare and can sometimes take years to locate and diagnose, Hamedanis hunch about the gene mutation led them right to it.

Because little is known about CPEO, treatment options are limited. Most people would be discouraged by the uncertainty, she says, but it thrills me to get to be the blueprint. I get to show people how to live with this.

Launched in March 2020, the Penn Neurogenetics Therapy Center has a team of clinicians, nurses, genetic counselors, and clinical research staff who are devoted to the care of patients with inherited neurological disorders and to participating in clinical trials of novel gene and molecular therapies.

The programs mission is twofold: first, they utilize the expertise of clinicians and researchers throughout the department of Neurology and across Penn Medicine to achieve a genetic diagnosis for as many patients like Waterhouse as possible, creating a database of eligible patients for new treatments and clinical trials. Second, they work to establish clinical trials using novel gene and molecular therapies for patients with genetically-based neurological disorders.

Our genetics counselors are some of the best in the country, and are incredibly effective at diagnosing patients and matching them with effective treatments and clinical trials, says Steven Scherer, a professor of neurology and director of the Neurogenetics Therapy Center. Now we can utilize this expertise to design tomorrows therapies.

Read more at Penn Medicine News.

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Solving medical mysteries with genetics: The Penn Neurogenetics Therapy Center | Penn Today - Penn Today

BEIJING, Sept. 08, 2022 (GLOBE NEWSWIRE) -- George Clinical, a global clinical research organization with an extensive presence throughout the Asia-Pacific region, continues to expand the organizations team in China with the addition of Helen Xu as Project Director and Cell Gene Therapy Head. She will be based in Beijing and joins a rapidly growing team responsible for expanding clinical research activity in China with biopharmaceutical, medical device and diagnostic sponsors.

Dr. Xu obtained her MD in clinical medicine from Peking University Health Center. She is a licensed physician specialized in central nervous system (CNS) and has worked in the hospital setting for four years. Dr. Xu entered the pharmaceutical industry in 2007 starting as a clinical research associate (CRA) and has since accumulated 15 years of valuable clinical research experience in China.

I am sure Helen will make an incredibly valuable contribution to the growth and development of clinical research operations and cell gene therapy studies across China, said Zhenfei Yin, country head and regional head project operations, China.

Before joining George Clinical, Dr. Xu had served GSK, BI, Wuxiapp, a Chinese clinical research organization, CASI, and Carsgen, a CAR-T biotech firm. Her clinical trial experience covers the whole development lifecycle starting from phase I through PMS, the majority of the trial experiences being with pivotal trials. Therapeutic areas of expertise include cell therapy, immunotherapies, blood tumors (MM, AML, thalassemia), solid tumors (lymphoma, gastric and pancreatic cancer, prostate cancer, lung cancer), respiratory disease (IPF, asthma), SSc-ILD, CNS (stroke, schizophrenia, GAD), HCV, cirrhosis, psoriasis, and in vitro diagnostics (IVDs) devices. Her responsibilities spanned the full duration of studies from bid defense, strategic planning, feasibility, and start-up to project close-out. The majority of these pivotal global trials experienced global audits and authority inspections from entities such as FDA, EMA, and NMPA.

Additional new team members will be joining the organization to support the team in China with medical expertise

With a growing presence across the country, it is an honor to be part of global CRO able to bring further clinical research to China that can positively impact cancer care around the world, Xu said.

About George Clinical

George Clinical is a leading global clinical research organization founded in Asia-Pacific driven by scientific expertise and operational excellence. With over 20 years of experience and more than 400 people managing 39 geographical locations throughout the Asia-Pacific region, USA, and Europe, George Clinical provides the full range of clinical trial services to biopharmaceutical, medical device, and diagnostic customers, for all trial phases, registration and post-marketing trials.

LinkedIn: https://www.linkedin.com/company/george-clinical-pty-ltd

Twitter: https://twitter.com/george_clinical

Facebook: https://www.facebook.com/georgeclinical

Wechat: https://mp.weixin.qq.com/.

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George Clinical Expands China Team with New Project Director and Cell Gene Therapy Head Helen Xu - AsiaOne

Mumbai: Nearly 14 months after the clinical trials of the indigenously developed CAR-T technology for blood cancer treatment was kicked off at the Tata Memorial Centre (TMC), researchers have concluded the first phase of trials and called the results encouraging.

This is the first time that gene therapy indigenously developed by researchers at the Indian Institute of Technology (IIT), Bombay, was tested on patients in India.

Phase I clinical trial data demonstrates that Indias first indigenously developed novel CAR-T Cell therapy is safe and shows promising early sign of efficacy in treating Lymphoma, a type of blood cancer, said a statement released by IIT B, late on Sunday evening.

In June 2020, the central governments National Biopharma Mission (NBM) -Biotechnology Industry Research Assistance Council (BIRAC) had approved 18.96 crore to the team for conducting a first-in-human phase-I/II clinical trial of the CAR-T cells. The drug has the potential to benefit cancer patients, who currently are forced to opt for only palliative care.

While existing treatments work towards increasing the life of patients by a few years or months, CAR-T technology holds the promise of curing certain types of cancers. Unlike chemotherapy, this drug is administered only once to a patient.

Dr Gaurav Narula, principal investigator of the paediatric-Acute Lymphocytic Leukemia (ALL), TMC and Dr Hasmukh Jain, principal investigator of the adult B-cell lymphoma study, started recruiting patients in early 2021. So far, six patients in pediatric-ALL and 10 patients in adult lymphoma studies were treated with indigenous HCAR19.

The trials were conducted at the Advanced Centre for Treatment, Research and Education in Cancer (ACTREC), the research and development wing of TMC.

The participants received autologous HCAR19 therapy. None of the participants had immune effector cell-associated neurotoxicity syndrome. Three out of ten participants had a complete response post CAR-T cell therapy and none of the participants required ICU admission. There was no CAR-T treatment related death. Overall, the novel humanized HCAR19 tested in Phase I clinical trials for adult lymphoma was found to be safe and has shown promising early signs of activity, said Dr Hasmukh Jain, who presented the data in the Annual Symposium of Cell and Gene Therapy, CMC Vellore.

Chimeric Antigen Receptor T (CAR-T) cells are genetically engineered to produce an artificial T-cell receptor, which is widely used in developed nations for immunotherapy during treatment for cancer. As part of gene therapy, these cells are used with an intent to cure certain types of blood cancers. However, the technology is still unavailable in India.

IIT Bombay and Tata Memorial Hospital (TMH) started their R&D collaboration in 2015 to develop the novel CAR-T cell therapy platform for cancers and immune-disorders. Dr Rahul Purwar, Associate Professor, IIT Bombay (on-lien) and currently appointed as CEO of ImmunoACT, designed and developed the indigenous CAR-T platform and patented anti-CD19 CAR-T product (HCAR19). In early 2021, HCAR19 product entered into two Phase 1 clinical trials at TMH, Mumbai.

Dr Gaurav Narula will present the results of Phase I trial of paediatric B-ALL in the Asia Pacific Blood and Marrow Transplantation (APBMT) 2022 meeting soon. The clinical trials will now enter Phase-II, post approvals from the Central Drug Standard Control Organisation (CDSCO) and is expected to be available for commercial clinical usage in 2024.

Points for graphic:

IIT Bombay and Tata Memorial Hospital (TMH) started their R&D collaboration in 2015 to develop the novel CAR-T cell therapy platform for cancers and immune-disorders

This is the first time that gene therapy indigenously developed by researchers at the Indian Institute of Technology (IIT), Bombay, was tested on patients in India

CAR-T cells are genetically engineered to produce an artificial T-cell receptor, which is widely used in developed nations for immunotherapy during treatment for cancer

While existing treatments work towards increasing the life of patients by a few years or months, CAR-T technology holds the promise of curing certain types of cancers. Unlike chemotherapy, this drug is administered only once to a patient.

Shreya Bhandary is a Special Correspondent covering higher education for Hindustan Times, Mumbai. Her work revolves around finding loopholes in the current education system and highlighting the good and the bad in higher education institutes in and around Mumbai....view detail

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IIT-B-Tata hosp cancer therapy trials show promising results - Hindustan Times

This renewed international congress brings together around 800 experts in the field of Myology and neuromuscular diseases from all over the world (35 countries). More than 70 international speakers will take the floor during 18 plenary and parallel sessions and more than 400 scientific posters will be discussed during these four days.

Among these, 60 researchers and clinicians from the Institute of Myology, Centre of expertise on muscle and its diseases, will be there to highlight their work and last scientific results in the field of neuromuscular diseases. From fundamental research to therapeutic advances no less than 59 communications (6 oral presentations & 53 posters) will be presented by our neuromuscular scientific experts.

This congress is also the opportunity for Institute of Myology to present its complete environment and activities. From its Center of Research in Myology, its unique Neuromuscular Investigation Center (including 4 labs of excellence) and its clinical activities with two clinical trial platforms I-Motion - for neuromuscular patients and a dedicated clinical hospital unit, to its ambition to create a future Foundation of Myology, the Institute of Myology is the first Center of this kind for Muscle in its pathologies in France.

>> Tuesday, September 13th Motor neuron diseases (13 PM Parallel 1)

4.30 PM - Piera Smeriglio: The role of DNA epigenetics in modulating Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a motor neuron disorder caused by mutation in the SMN1 gene. In human, the presence of the SMN2 paralog can partially compensate for the SMN1 loss and the copy number of SMN2 inversely correlates to disease severity. We uncovered that DNA epigenetic regulation is altered in human and mouse SMA models, leading to abnormal profile of methylation and hydroxymethylation on the SMN2 gene and genome-wide. These defects promote the aberrant expression of SMN2 and of genes acting on the inflammatory response pathways, suggesting a direct epigenetics role in SMA pathogenesis.

5.20 PM - Giorgia Querin: Spinal cord MRI for early detection of presymptomatic pathology in C9orf72-mutation carriers: a longitudinal neuroimaging study

Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal dementia (FTD) share genetic susceptibility and a large portion of familial cases are due to C9orf72 gene mutations. Brain and spinal cord (SC) imaging studies in asymptomatic C9orf72 carriers have demonstrated white (WM) and grey matter (GM) degeneration up to 20 years before the expected symptom onset.

Objective of this study is to longitudinally analyze cervical spinal degeneration in asymptomatic carriers of the C9Orf72 mutation using different multimodal MRI sequences with the aim of tracking longitudinal degeneration at the spinal level and of identifying possible prognostic factors of disease evolution.

>> Tuesday, September 13th Development, regeneration & ageing - part 3 (13 PM Parallel 2)

5.00 PM - Alfredo Lopez Kolkovsky: Preliminary results of a multiparametric quantitative NMR ageing study at rest and during exercise in the lower leg in healthy subjects between 20 and 65 years of age

The progressive decline in muscle strength and performance during ageing negatively affects the quality of life in elderly subjects and increases the risk of falls, disability and frailty. The age-related loss of muscle mass, strength and quality is a complex multifactorial process whose mechanisms are incompletely understood.

Nuclear magnetic resonance (NMR) allows evaluating anatomical, structural and physiological aspects of muscle tissue non-invasively in vivo. Functional NMR also enables, for instance, to image during an exercise paradigm the tissue blood flow (BF) or energy metabolism using P MR spectroscopy (MRS).

We designed a protocol where multiple quantitative and complementary measures were performed at rest and during a plantar flexion exercise in 26 subjects. Age-related changes were observed for muscle water T1 relaxation times and muscle fat fraction as well as cellular membrane turnover and mitochondrial stress biomarkers. This study demonstrates the interest of a multiparametric NMR approach in aging studies.

5.45 PM - Massire Traore: Therapeutic approach based on GDF5 to counteract age-related muscle wasting

Sarcopenia is a disease defined as progressive age-related loss of muscle strength, function and mass, which results in increased mortality. Several mechanisms have been proposed to explain the onset and progression of sarcopenia, however, some pathophysiological aspects are still not very well understood and no cure has been established to date.

Our previous work demonstrated that GDF5 (Growth Differentiation Factor 5) overexpression in old mouse prevented muscle mass decline, although a deeper report on the mechanisms and consequences of GDF5 implement on aged muscle was missing. Here, we demonstrate that GDF5 overexpression in muscle during aging induces muscle mass gain and improves neuromuscular connectivity and endplate morphology. In addition, we present the characterization of the cellular and molecular effects of GDF5 in muscle during aging and show its rejuvenating signature. Based on this proof of concept, we defined a cutting-edge therapeutic approach describing how the treatment with the recombinant GDF5 protein is able to counteract the age-related skeletal muscle wasting in mice and might have a strong curative potential on humans.

>> Wednesday, September 14th Myotonic syndromes (14 AM Parallel 1)

10.30 AM - Denis Furling: Decoy gene therapy for Myotonic Dystrophy

Myotonic Dystrophy type 1 (DM1), one of the most common neuromuscular disorders in adults, is characterized by progressive muscle weakness and wasting, myotonia, cardiac defects, endocrine troubles and cognitive impairments. This autosomal dominant disease is caused by an expanded tract of CTG repeats within the 3 non-coding region of the DMPK gene. Expression of mutant transcripts containing expanded CUG repeats (CUGexp) leads to a toxic RNA gain-of-function mechanism affecting functions of specific RNA binding proteins (RBPs) and consequently, RNA metabolism. To date, there is no cure for DM1 but several therapeutic strategies including small molecule and antisense oligonucleotide approaches are under development.

Here we assessed a gene therapy approach for DM1 using a modified RBP with a high affinity for CUGexp that aims at acting as a decoy and displaces sequestered endogenous MBNL proteins from

RNA foci to reverse RNA toxicity. For this purpose, we engineered a truncated MBNL1 protein that keeps its zing finger domains required for the binding to CUGexp but lacks the C-terminal domain involved in splicing activity and homodimerization. Our decoy has a reduced splicing activity but can still compete with MBNL1 for CUGexp-binding. Effect of this decoy was next assessed in both human DM1 muscle cells and HSA-LR mouse model. We showed that the binding of the decoy to CUGexp in DM1 muscle cells allows the release of sequestered endogenous MBNL1 from nuclear foci, restores MBNL1 activity and corrects the transcriptomic signature of DM1. In vivo, local or systemic delivery of the decoy into skeletal muscles of DM1 mice using AAV9 vectors leads to long-lasting correction, up to one year, of both splicing defects and myotonia, hallmarks of DM1. This proof-of-concept study (Arandel et al., Nature Biomedical Engineering, 2022) supports the development of decoy-RBPs with high binding affinities for CUGexp as a therapeutic strategy for DM1.

11.45 AM - Mona Bensalah: Muscle fibrosis: a vicious circle between human fibroadipogenic progenitors and muscle fibers

Fibrosis is described in many organs as an excessive accumulation of extracellular matrix (ECM) proteins that replace tissue and alter its function. In skeletal muscle, fibrosis is a pathological feature common to many dystrophies including Oculopharyngeal Muscular Dystrophy (OPMD), a late-onset disorder, where only a small group of muscles are primarily affected and characterized by an exacerbated fibrosis, fiber atrophy and inflammation. While the cellular and molecular mechanisms regulating muscle fibrosis has been extensively studied in mouse, our understanding of the exact nature and role of mesenchymal cells involved in fibrosisis limited in human and theirimplication in dystrophic muscle progression remains to be clarified.

We investigated the role and nature of nonmyogenic cells (fibro/adipogenic progenitors, FAPs) from human fibrotic muscles of healthy individuals and OPMD patients, and compared them to nonmyogenic cells from human nonfibrotic muscle. Our data underline the key role of FAPs and their cross-talk with muscle cells through a paracrine signaling pathway in fibrosis of human skeletal muscle, and identify endothelin as a new druggable target to counteract fibrosis.

Beside these six oral presentations, no less than 53 posters will be presented by researchers from the Institute of Myology during break sessions in Rhodes exhibition hall:

The Institute of Myology, a unique centre of expertise on muscle

The Institute of Myology was created in 1996 by AFM-Telethon to diagnose and treat patients, and to study diseased muscle, in partnership with five public bodies (the AP-HP, the CEA, Inserm, Sorbonne Universit and the CNRS). This centre of expertise is globally unique and promotes the existence and recognition of the field of myology by bringing together, in a single location, fundamental and applied research, clinical research, physiological assessment, care and teaching. Eight centres, bringing together 250 doctors and researchers, are dedicated to the muscle in all its states, from the national reference centre for the diagnosis, management and monitoring of neuromuscular diseases, to the research centre, a clinical research platform with innovative investigational and measurement tools, and a centre for training and dissemination of knowledge in the field of myology. The Institute is patient-centred and brings together diagnosis, clinical care, assessment and research.

http://www.institut-myologie.org/en

Press contact:

Stphanie Bardon presse@afm-telethon.fr Tel.: +33 (0)1 69 47 29 01

See the article here:
7th International Congress of Myology Nice - EurekAlert

AI-generated image using Midjourney depicting scientists inventing new medicines

In 1998 I was exposed to the term disruptive innovation for the first time. I read a wonderful book, The Innovators Dilemma by Clayton Christensen, where I learned the difference between incremental innovation and disruptive innovation. He analyzed the hard drive industry and showed that while many companies were trying to increase the capacity of the drives, other companies changed the form factor and made the drives smaller. This resulted in disruptive progress in the industry. We also recently witnessed dramatic advances in artificial intelligence (AI), where in 2013/2014 AI systems started outperforming humans in image recognition. This was made possible by taking several existing technologies, deep neural networks (DNNs) and GPU computing, and training DNN on really big data sets. In fact, disruptive innovation often results from the combination of already existing technologies to make them work to address a massive unmet need.

Over past decades, the field of nanomedicine, mostly characterized by the targeted delivery of drugs for numerous types of diseases, has gained a special attention in oncology research. While some anti-cancer drugs work well when administered to the right patient and in the right dose, no drug is perfect, and no dose fits all. One of the most frequently used classes of anti-cancer drugs is platinum-based compounds, such as carboplatin, oxaliplatin, and cisplatin (one of the most effective anti-cancer drugs for the treatment of solid malignancies). Chemotherapeutic agents are necessarily toxic, as from a therapeutic stance, we need to kill the rapidly dividing cancer cells. However, as these drugs are nonselective (targeting both healthy and malignant tissues), patients often suffer the unfortunate combination of substantial side effects and low efficacy at the target site when these compounds are administered systemically via traditional routes (orally or intravenously).

Enhancing the accumulation of these drugs locally at the tumor site may significantly reduce the systemic toxicities and adverse side effects, while simultaneously improving treatment efficacy substantially. The major limitation is that these drugs do not penetrate very well, and so enhancing penetration has become a necessary goal in the optimization of delivering these agents. One technology that can help with this task is encapsulating the chemotherapeutic agents in nanoparticles (NPs). During the last decade, a wide range of nano-based drug delivery systems has been explored as alternative cisplatin delivery methods that may promote its accumulation and retention in cancer cells. While some of these NP formulations have demonstrated promising preclinical results and a few have entered clinical trials, none have been approved for the treatment of human cancers. Targeted accumulation of the drug can be further optimized through topical delivery of NPs in the form of gels and film composites, which may also increase the local and precise administration of chemotherapeutic drugs to accessible tumors (such as cancers arising in the oral cavity). Therefore, disruptive innovation has originated from merging these approaches and generating topical NP-based drug delivery platforms that primarily intend to ameliorate the adverse effects of systemically administered treatment and maximize the total dose and retention of the carried therapeutic agent at the local site, improving treatment efficacy.

Manijeh Goldberg, PhD, MBA, CEO of Privo Technologies

A multidisciplinary group led by Dr. Manijeh Goldberg, founder and CEO of Privo Technologies, together with Dr. Nishant Agrawal and Dr. Evgeny Izumchenko, Chief of Head and Neck Surgery and Professor of Hematology and Oncology at the University of Chicago, respectively, recently published the results of Privos preclinical and clinical studies using PRV111 treatment. This treatment is a topical mucoadhesive cisplatin delivery system that has the potential to revolutionize the field.

A screen capture of the header of the Nature Communications paper titled "A nanoengineered topical ... [+] transmucosal cisplatin delivery system induces anti-tumor response in animal models and patients with oral cancer" https://doi.org/10.1038/s41467-022-31859-3

In the recent Nature Communications paper titled A nanoengineered topical transmucosal cisplatin delivery system induces anti-tumor response in animal models and patients with oral cancer, the scientists describe a nanotechnology-based patch system for non-invasive, local delivery of cisplatin-loaded chitosan particles that penetrate tumor tissue and lymphatic channels while avoiding systemic circulation and toxicity. The system was used in both animal models (mice and hamsters) and patients with oral cancer and demonstrated promising, potentially disruptive, results.

Professor Nishant Agrawal, MD, Director, Head and Neck Surgical Oncology Chief, Section of ... [+] Otolaryngology-Head and Neck Surgery, University of Chicago

The hamster cheek model is a well-characterized system for studying oral cancer because of the similarities between the tissue in the pouch and the tissue in the human mouth. In the clinical trial, patients were treated with PRV111 as long as a week before undergoing surgery (surgery is standard for all patients with oral cancer). 87% of patients responded to treatment and showed decreased tumor volume, with an average decrease of 70% of tumor volume across all patients. As further confirmation of PRV111s efficacy, examination of the tumor tissue after surgery demonstrated that treatment also stimulated the patients innate immune system. Thus, the results from PRV111 use in humans suggest that PRV111 has shown a one-two punch therapeutic effect, simultaneously killing tumor cells and recruiting immune cells to attack the tumor site.

Briefly, in the PRV111 platform, chitosan (a non-toxic, biocompatible, and biodegradable polysaccharide derived from natural chitin), is used as a polymer for both the NPs and the porous matrix. As such, matrix-based water-soluble chitosan acts as a bioadhesive since the positively-charged chitosan can bind to negatively-charged mucoproteins, allowing the electrostatic interaction with mucin proteins in the oral cavity. Each cisplatin-loaded chitosan NPs containing patch covers a tumor region of 4 cm2, and incorporates a permeation enhancer that allows optimal penetration and absorption of the NPs released from the patch. When exposed to moisture, the NPs swell, allowing them to diffuse across the porous matrix and into the tumor tissue. However, these particles are too large to penetrate into the vasculature, and therefore prevent systemic cisplatin exposure.

Professor Evgeny Izumchenko, PhD

Since I made a tiny and insignificant contribution to the paper, and I am familiar with the leading authors. I got a chance to ask them a few questions about this possibly groundbreaking work.

Alex: I consider cisplatin to be one of the ancient uber-toxic cancer drugs that are responsible for the many misconceptions about modern cancer treatments. Why did you choose cisplatin for this study?

Cisplatin is one of the most potent anti-cancer agents. It has been studied and used in the field of oncology for decades. However, its dose-limiting systemic toxicities such as nephrotoxicity, ototoxicity and neurotoxicity can be severe and many times irreversible. In its early days, Privo discussed the best choice for its active ingredient with its advisor at the time, Dr. Jos Baselga at Bostons Mass General Hospital (MGH). Dr. Baselga, a renowned cancer researcher and the chief of hematology/oncology at MGH, recommended using cisplatin. He noted that cisplatin is a beast of a cancer drug and if tamed, it can be extremely effective in destroying cancer cells. Privo optimized the use of cisplatin to reduce its nasty systemic side effects while significantly improving its efficacy by its ability to directly deliver high concentrations of cisplatin to the tumor. This is like an atomic bomb applied specifically to the tumor, sparing the patients healthy tissues.

Alex: Nanomedicine is a frequently overused word, and it defines a very broad field. How do your group and your consortium of collaborators fit into this field?

We use both nano- and micro-particles in our matrix as part of our two-stage release platform technology. We design the particles with specific properties such as surface chemistry and size. The nano- and micro-particles can be programmed to control the release of the active ingredient based on the treatment requirements. In addition, the particle-containing polymeric matrix further protects the particles, allowing for longer lasting drugs following administration. For example, cisplatin is a very volatile drug which rapidly binds to proteins in the body, causing its deactivation before it can even reach the tumor site and have the chance to destroy cancer cells. Privos PRV111 is designed to protect and effectively insulate the drug for much longer, serving to decrease side effects and increase cellular uptake of the drug. The surface chemistry of the particles can also be optimized to increase cellular uptake further.

Alex: Can you tell me the story behind this paper - how did it come together?

Privo has been collaborating with Dr. Nishant Agrawal from Chicago Medicine since its early development days. I was introduced to Dr. Agrawal via a mutual friend, who was an oncologist that had lost a friend to oral cancer. He saw the potential of our research helping patients suffering from head and neck cancers. Dr. Agrawal is the chief of head and neck surgical oncology team at UChicago Medicine and has been a great mentor to our Privo team in addition to being a champion of our platform technology. Once Privo had successful preclinical data followed by phase 1 / 2 clinical study, the team agreed to publish the results. During the several years of research, Dr. Agrawal has made several introductions to other key opinion leaders such as Dr. Evgeny Izumchenko, an expert head and neck researcher at the University of Chicago who has been instrumental in compiling and publishing the data.

Alex: How much impact will this study have on patient lives once the technology reaches the market?

I think Privos technology has the potential to disrupt the treatment paradigm for several cancers starting with mucosal cancers such as oral, lung, cervical, and even brain tumors.

In todays world, social media has provided an unbiased, uncomfortable, and often raw insight into what a cancer patients journey is like. Over the past several years, we have followed a few oral cancer patients on their journey from initial diagnosis, treatment, surgery, physical therapy in addition to their mental health journey at each step of the road. Oral cancer has one of the highest suicide rates among cancers, and is second only to pancreatic cancer in terms of quality of life. Considering the accessibility of oral cancer, we aim to provide patients with a better alternative that offers them a better quality of life. Our data collected to date has shown that PRV111 has significantly reduced tumor volume for patients with T1/T2 stage tumors. In our Phase 3 design, we aim to target an early form of oral cancer, carcinoma in situ, where our goal is to eliminate the need for surgery indefinitely.

Alex: Are you planning to commercialize the technology and what are your plans to take it to market?

We are planning to commercialize the technology, especially after the successful first-in-human trial, which showed that PRV111 treatment performed better than expected, leading to early completion of the study. On average, PRV111 treatment decreased tumor volume by about 70% in just one week. Privo has successfully received support and collaboration from the FDA-OOPD, the NIH-NCI, the NIH-NIDCR, and the NSF. Their guidance and support is helping us be well on our way toward market approval.

In principle, this novel mucoadhesive system can be engineered to deliver virtually any chemotherapy and non-chemotherapy agent with a particular drug release profile, making it customizable for specific clinical applications. The unique properties of this carefully designed nanomedicine provide a promising framework and holds potential for the improved treatment of not only oral accessible cancers but also other solid malignancies.

Professor Alexander T Pearson, MD, PhD, Professor Evgeny Izumchenko, PhD, Alex Zhavoronkov, PhD at ... [+] the University of Chicago

Link:
Going Beyond Target Or Mechanism Of Disease: Disruptive Innovation In Drug Delivery Systems - Forbes

Noninvasive ventilation is possible in infants at limits of viability. But unlike in slightly older preterm infants, noninvasive ventilation did not show an advantage in infants of 22 weeks-0 days to 23 weeks-6 days gestational age.

Vivek Shukla, right, and Brian Sims, left, help hold Guinness World Records Certificate for Most Premature Baby to Survive, awarded in honor of mother Michelle Butler and child Curtis Means, born at 21 weeks gestation (center). Adults are masked for COVID precautions in 2021. Photography: Andrea MabryExtremely premature infants still face daunting risks of sickness or death, even though advances in neonatal-perinatal care have improved infant survival at progressively lower gestational ages. Bronchopulmonary dysplasia a serious condition of undeveloped lungs is a leading morbidity in these tiny infants.

Studies have shown that noninvasive respiratory support at birth rather than immediate intubation and delivery of lung surfactant improves short-term respiratory outcomes in premature infants born at gestational age 24 weeks-0 days to 27 weeks-6 days.

So, clinicians at the University of Alabama at Birmingham led by Charitharth Vivek Lal, M.D., and Vivek Shukla, M.D., asked whether the same was true for the tiny newborns at the limits of viability, whom they categorize as nano-preterm infants those born at gestational age 22 weeks-0 days to 23 weeks-6 days. These nano-preterms compose a highly specialized niche subgroup that is considerably more immature and has much higher risks of mortality and morbidity than the 24- through 27-week gestational age preterms, Lal says.

A full-term pregnancy is 39 to 40 weeks.

In one of the largest studies of this population, UAB researchers did a retrospective analysis of 230 consecutively born, eligible nano-preterm infants born from January 2014 through June 2021 at the UAB level IV neonatal intensive care unit. Eighty-eight infants in the noninvasive group were those whose first intubation attempt was more than 10 minutes after birth, and 142 infants in the invasive respiratory support at birth were those intubated within 10 minutes after birth. Unlike in several previous studies of slightly older pre-term infants, Lal and colleagues found no benefits for the noninvasive respiratory support of those nano-preterm infants, as measured by the composite outcome of bronchopulmonary dysplasia or death by 36 weeks postmenstrual age.

Vivek Lal, M.D.Some 94.3 percent of the noninvasive group and 90.9 percent of the invasive group had bronchopulmonary dysplasia or death by 36 weeks, which was not a significant difference. The clinicians did see that severe intraventricular hemorrhage or death by 36 weeks was lower in the invasive respiratory support group, a trend that will require a larger number of infants to confirm.

This cohort studys findings suggest that noninvasive respiratory support in the first 10 minutes after birth is feasible but may not be associated with a decrease in the risk of bronchopulmonary dysplasia or death compared with intubation and early surfactant delivery in nano-preterm infants, said Lal, an associate professor in the UAB Department of Pediatrics, Division of Neonatology. Shukla is an assistant professor in the Division of Neonatology.

The average weight of the noninvasive nano-preterm infants was 1 pound 4.4 ounces, and the average weight of the invasive preterm infants was 1 pound 2.4 ounces.

The study, Hospital and neurodevelopmental outcomes in nano-preterm infants receiving invasive vs noninvasive ventilation at birth, is published in the journal JAMA Network Open.

Co-authors with Lal and Shukla are Grant Imbrock, Colm P. Travers, Namasivayam Ambalavanan and Waldemar A. Carlo, UAB Department of Pediatrics, Division of Neonatology; and J. Paige Souder, Muhan Hu and A.K.M. Fazlur Rahman, Department of Biostatistics, UAB School of Public Health.

Support came from National Institutes of Health grant HL141652, UAB and Childrens of Alabama.

Pediatrics is a department in the Marnix E. Heersink School of Medicine at UAB.

Read the rest here:
Nano-preterm infants may not benefit from noninvasive versus invasive ventilation at birth - University of Alabama at Birmingham

The Global Nanomedicine Market 2022 research report presents an in-depth analysis of the Nanomedicine Market size, growth, share, segments, manufacturers, and forecast, competition landscape and growth opportunity. The researchs goal is to provide market data and strategic insights to help decision-makers to make educated investment decisions while also identifying potential gaps and development possibilities.We also analysed the impact of COVID-19 (Corona Virus) on the product industry chain based on the upstream and downstream markets, on various regions and major countries and on the future development of the industry are pointed out.

The market size was determined by estimating the market through a top-down and bottom-up approach, which was further validated with industry interviews. Considering the nature of the market we derived it by segment aggregation, the contribution of the materials and vendor share.

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Companies involved in theNanomedicine Market research report are:

What exactly is included in the Report?

Industry Trends and Developments:In this section, the authors of the research discuss the significant trends and developments that are occurring in the Nanomedicine Market place, as well as their expected impact on the overall growth.

Analysis of the industrys size and forecast:The industry analysts have provided information on the size of the industry from both a value and volume standpoint, including historical, present and projected figures.

Future Prospects:In this portion of the study, Nanomedicine Market participants are presented with information about the prospects that the Nanomedicine Market industry is likely to supply them with.

The Competitive Landscape:This section of the study sheds light on the competitive landscape of the Nanomedicine Market by examining the important strategies implemented by vendors to strengthen their position in the Nanomedicine Market.

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Nanomedicine Market Size, Share, Types, Products, Trends, Growth, Applications and Forecast 2022 to 2028 - Digital Journal

Spanish-American clinical research executive Juan de Borbn resides in the United States. He has vast experience running nearly 2000 clinical trials in various therapeutic fields. These include cancer, nephrology, hepatology, infectious illness (Covid-19, Ebola, Marburg, Influenza, HIV, Hepatitis), and neurology. Juan de Borbn has been inspiring and motivating the clinical drug development sector for more than 20 years by utilizing his extensive industry knowledge.

Alfonso XIII of Spains senior great-grandson Juan de Borbn is his matrilineal direct descendant. King Alfonso XIII of Spain and his wife, Princess Victoria Eugenie of Battenberg, had a son, Alfonso, the oldest of five siblings. Alfonso, Prince of Asturias, who lived from 10 May 1907 to 6 September 1938, was the presumptive heir to the Spanish crown and grandfather to Juan. At the time, Alfonsos renunciation and untimely death as the heir to Spains monarchy sparked debate. King Felipe VI of Spain, his cousin, now sits the Spanish throne.

At the age of two months, Juan de Borbn immigrated to the US and spent his formative years in Los Angeles. Growing up, he practiced martial arts, surfed, skateboarded, and shared a deep passion for baseball with his father. His parents wished for him to have a wider variety of experiences and a deeper appreciation of his roots. Today, he is a top-ranking CEO honored for his accomplishments and model leadership by the business, his employers, and coworkers; Juan now holds the positions of Principal and President of Global Strategy at Global Earth USA, Executive Chairman of Borbon dAnjou Holdings, and Principal of the Borbon Family Office.

He is a compassionate leader who upholds the principles of integrity, honesty, and creativity, collaborating directly with Nano Cures Pharma in 2021 to provide Covid-19 vaccinations to underserved countriesworking now with Thailand and the Central African Republic to obtain Emergency Use Authorization for the vaccination in those nations.

Juan de Borbn established the first American Heart Training Center in a Clinical pharmacology facility in 2017, guaranteeing the best level of staff training in acute care for research participants. He combined WCCT with Medelis, an institution that conducts oncology clinical research, in 2016. The deal focused on the complex and quickly expanding oncology drug development industry in the United States, Europe, and Asia. This requires a unique answer with knowledge, insight, and a focus on the future.

With a focus on oncology, ophthalmology, gastroenterology, gastrointestinal, renal, hepatic, and virology, Juan de Borbn created a multisite management service offering CRO Services for WCCT in 2014. This service provided data processing, statistics, medical monitoring, drug safety, tracking, site selection, and site management. He established the early clinical research facilities for cosmetology and ophthalmology in 2013 and combined OC Clinical Trials & Consulting with WCCT Global.

Juan believes that the era of doing regular activities in healthcare is finished. Despite the tireless efforts of well-meaning, well-trained professionals, every health care system in the world is battling escalating prices and unequal quality. Health care leaders and policymakers have attempted to combat fraud, reduce errors, enforcing practice guidelines, improve patient consumers, and deploy electronic medical records. Still, none of them have had much of an impact. It is time for a whole different approach.

He is focused on shifting from a supply-driven, physician-centered healthcare system to one that is patient-centered, patient-centered, and organized around patient needs. A system where services for specific medical diseases are focused in health-delivery organizations and in the appropriate places to deliver high-value care must be put in place to replace the fragmented system of today, in which every local provider offers a broad range of services. The volume and financial success of the services providedphysician visits, hospital stays, operations, and testsmust be shifted to the success of the patient outcomes.

Juan aims to launch the worlds first medical metropolis to promote lifespan and good health while utilizing information and communications technology for the exchange of reliable data for the assessment, care, and prevention of illness and injuries, research, and evaluation, as well as for the continuing education of health care professionals, all to improve the health of people and their communities. Additionally, it is intended to assist the patient in maintaining their performance level throughout their entire lifespan. The main risk factor for most diseases is advancing age, particularly of cellular structures, and life expectancy in the population is still rising.

Juan emphasizes that the cutting-edge longevity center offers tailored preventative medicine by implementing methods to promote healthy aging, delay diseases primarily related to aging, and lengthen a persons active time of life. Providers who continue to use the outdated methods of today will go extinct. Reputations that are built on perception rather than actual results will deteriorate. In the face of increased openness and declining reimbursement levels, maintaining present cost structures and prices will be impossible. The only reputation that should matter in health care is assisting people in leading the healthiest and longest-lasting lives possible.

Have you read?The Worlds Largest Economies, 2022.International Financial Centers Ranking, 2022.These are the countries with the Highest Average Salaries, 2022.CEO Spotlight: For Plaintree Systems CEO David Watson, the Future is Taking Its Cues From the Past.How to Build a Winning Sales Team That Gets your Business Soaring by Jakob Wissel.When Culture Matters by Bill Yeargin.

More here:
Juan De Borbon - Introducing Cutting-Edge Techniques To The Healthcare Industry - CEOWORLD magazine

Sep 05, 2022(Nanowerk News) In a recent publication in the scientific journal Advanced Materials ("Accurate Wavelength Tracking by Exciton Spin Mixing"), a team of physicists and chemists from TU Dresden presents an organic thin-film sensor that describes a completely new way of identifying the wavelength of light and achieves a spectral resolution below one nanometer.As integrated components, the thin-film sensors could eliminate the need for external spectrometers in the future. A patent application has already been filed for the novel technology.The active film for the novel sensor concept is only as thick as a human hair, here processed on thin glass substrates, and exhibits a wavelength-dependent luminescence. (Image: Anton Kirch)Spectroscopy comprises a group of experimental methods that decompose radiation according to a specific property, e.g. wavelength or mass. It is considered one of the most important analytical methods in research and industry.Spectrometers can determine colors (wavelengths) of light sources and are used as sensors in various applications, such as medicine, engineering, food industry and many more. Commercially available instruments are usually relatively large and very expensive. They are mostly based on the principle of the prism or grating: light is refracted and the wavelength is assigned according to the angle of refraction.At the Institute for Applied Physics (IAP) and the Dresden Integrated Center for Applied Physics and Photonic Materials (IAPP) of the TU Dresden, such sensor components based on organic semiconductors have been researched for years. With the spin-offs Senorics and PRUUVE, two technologies have already been developed towards market maturity.Now, researchers at the IAP and IAPP, in cooperation with the Institute of Physical Chemistry, have developed a thin-film sensor that describes a completely new way of identifying the wavelength of light and, due to its small size and cost, has clear advantages over commercially available spectrometers.The principle of operation of the novel sensors is as follows: Light of unknown wavelength excites luminescent materials in a hair-thin film. The film consists of a mixture of long-glowing (phosphorescent) and short-glowing (fluorescent) entities, which absorb the light under investigation in different ways. The intensity of the afterglow, can be used to infer the wavelength of the unknown input light."We exploit the fundamental physics of excited states in luminescent materials," explains Anton Kirch, doctoral student at the IAP. "Light of different wavelengths excites in such a system, when properly composed, certain proportions of long-lived triplet and short-lived singlet spin states. And we reverse that dependence. By identifying the spin fractions using a photodetector, we can identify light wavelengths.""The great strength of our research alliance here in Dresden is our partners," says Prof. Sebastian Reineke, who coordinated the project. "Together with the groups of Prof. Alexander Eychmller from Physical Chemistry and Karl Leo, professor of Optoelectronics, we can carry out all the fabrication and analysis steps ourselves, starting with material synthesis and film processing and ending with the fabrication of the organic detector."Dr. Johannes Benduhn is group leader for Organic Sensors and Solar Cells at the IAP: "I was honestly very impressed that a simple photoactive film combined with a photodetector can form such a high-resolution device."Using this strategy, the scientists have achieved sub-nanometer spectral resolution and have successfully tracked minor wavelength changes of light sources. In addition to characterizing light sources, the novel sensors can also be used in counterfeit protection: "The small and inexpensive sensors could be used, for example, to quickly and reliably check banknotes or documents for certain security features and thus determine their authenticity, without any need for expensive laboratory technology," explains Anton Kirch.

Read more:
Organic thin-film sensors for light-source analysis and anti-counterfeiting applications - Nanowerk