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More than 800 medicines are in development for diseases that disproportionately affect racial and ethnic communities – PRNewswire

By adminJune 25, 2021Stell Cell Research

WASHINGTON, June 22, 2021 /PRNewswire/ -- We are in a new era of medicine where groundbreaking biopharmaceutical research and development is transforming medicine, but these innovations are meaningless if they don't reach patients, including those in underserved communities. Health disparities are not new, but the COVID-19 pandemic put a spotlight on long-standing health inequities that affect diverse racial and ethnic communities in America. Data shows these populations have been disproportionately impacted by COVID-19. In fact, American Indian/Alaskan Native, Hispanic, and Black populations are approximately twice as likely to die from COVID-19, as compared to non-Hispanic whites.

Researchers have found that people with certain health conditions, including chronic conditions such as Alzheimer's disease, certain cancers, chronic kidney disease, chronic lung diseases, type 2 diabetes, heart conditions, HIV infection, liver disease, obesity, sickle cell disease and stroke, are at higher risk of severe illness or death from COVID-19. Many of these conditions are tied to health disparities that disproportionality affect racial and ethnic communities for genetic and environmental reasons, or due to inequities in social and economic conditions.

Today, PhRMA released a new report exploring the 829 medicines in development that aim to address the diseases and conditions that affect racial and ethnic communities at a higher rate and are also associated with worse COVID-19 outcomes.

Among the medicines in development to improve management of these diseases are:

It is critical that all patients, including historically underserved racial and ethnic communities, have access to medicines. One way to reduce barriers to health care access and enable everyone to benefit from new medicines is to ensure that clinical trials are diverse and inclusive and include participants representative of the population the medicine intends (or aims) to treat. The biopharmaceutical industry has been working with patients, communities, regulatory authorities, health care practitioners, academics and policymakers to enhance diversity in clinical trials, so the clinical trial population testing medicines better reflect the patients that will use the new therapies and medicines should they are approved by the U.S. Food and Drug Administration.

To this end, PhRMA and its member companies have voluntarily adopted first-ever industry-wide principles on clinical trials diversity, adding a new chapter to the already existing Principles on Conduct Clinical Trials & Communication of Clinical Trial Results.The new clinical trial diversity principles are designed to build trust, reduce barriers to clinical trial access, enhance an understanding of drug effects in diverse patient populations, and promote the sharing of information on policies and practices to increase clinical trial diversity.

Equity is critical to the health and well-being of diverse racial and ethnic communities, and it remains essential to a robust ecosystem of innovation. America's biopharmaceutical companies are pushing for necessary systemic and long-term change to better meet the needs of underserved communities in America.

To learn more about the PhRMA Equity Initiative and PhRMA's commitment to inclusion, visit https://phrma.org/Equity and tune in to The Atlantic's Health Equity Summit where PhRMA's Chief Operating Officer, Lori Reilly, and Genentech's Chief Diversity Officer, Quita Highsmith, will have a conversation about building trust in clinical trials.

Learn more about the medicines in development to address health equity here.

This post originally appeared on the Catalyst blog.

CONTACT:Andrew Powaleny,[emailprotected], 202-835-3460

SOURCE Pharmaceutical Research and Manufacturers of America (PhRMA)

https://phrma.org/

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More than 800 medicines are in development for diseases that disproportionately affect racial and ethnic communities - PRNewswire

Andrs Garca Receives Distinguished Professor Award | Research – Research Horizons

By adminJune 25, 2021Stell Cell Research

Vision. Collaboration. Innovation. The qualities for which Georgia Tech has become so well-known were embodied in people like Bob Nerem, founding director of the Parker H. Petit Institute for Bioengineering and Bioscience (IBB) from 1995 to 2009, Parker H. Petit Distinguished Chair for Engineering in Medicine, and Institute professor emeritus until his death in March 2020.

In 1997 Nerem recruited Andrs Garca and his wife, Michelle LaPlaca, to join the pioneering IBB program at Tech after they completed their work as postdoctoral fellows at the University of Pennsylvania his in cell and molecular biology, hers in neurotrauma.

In 1998 when Garca and LaPlaca joined Tech, IBB launched its National Science Foundation Engineering Research Center in Tissue Engineering with Emory University, making it a strategic community for Garca to join to start his research program in an emerging field. Now as executive director of IBB and a Regents Professor in the Woodruff School of Mechanical Engineering, Garca is continuing Nerems legacy of vision, collaboration, and innovation in everything he does. In recognition of his work, he is the 2021 recipient of the Class of 1934 Distinguished Professor Award, the highest honor given to a Georgia Tech professor. The award is presented to a professor who has made significant, long-term contributions to teaching, research, and public service.

Known as a global pioneer in developing biomaterials systems for translational applications in regenerative medicine, Garca holds more than a dozen U.S. patents. Discoveries include the development of hydrogels for protein and cell delivery in regenerative medicine, engineering biofunctional materials to improve islet survival, and the design of infection-fighting materials. His research focuses on creating an engineered class of materials that can be used for applications to transplant a graft without immune-suppressive drugs. Human studies are planned to start next year. Researchers in his lab are developing new ways to treat Type 1 diabetes, eventually working with adult stem cells to reprogram them into insulin-producing cells. Future applications include addressing kidney failure and other diseases.

Creating Opportunities for Collisions

Garca is enthusiastic about his research, as well as all of the collaborative research in IBB. IBB is a fantastic community of faculty, trainees, and staff who come together in making discoveries and developing the technologies in bioengineering and bioscience that will change the world, he said. His goal is that IBB will continue to expand research and integrative opportunities to have a major economic impact, creating an environment to translate research into commercial products and therapies. With IBB we want to provide opportunities for collisions, unexpected interactions that lead to the discoveries. It was Bob Nerems vision to drive that sort of collaboration, he said.

Garca shared an example of one such collision: As part of a grant from the Juvenile Diabetes Research Foundation (JDRF), I was required to present unpublished research progress at a meeting with other researchers from throughout the country. After I made my presentation that morning, a JDRF director announced that for the next three-year cycle of funding we would need to collaborate with someone in the room on research. We went to lunch, and as I was building my sandwich, an immunologist introduced himself to me, complimented me on my presentation, and asked me if I thought I could develop a biomaterial to deliver the particular protein he was working with. You never ask an engineer if they think they can do something. Theyll find a way. I said I could, and we started working together.

An elected member of both the National Academy of Inventors and the National Academy of Engineering, Garca has established three startup companies in the past seven years. He has received numerous awards for his teaching and research and has published more than 230 peer-reviewed papers in prestigious journals.

Mentoring Students

Garca has supervised 15 postdoctoral researchers and advised/co-advised 37 Ph.D. students. He is known for his long-term commitment to his trainees, as well as mentoring students outside of his laboratory and classroom. While he has not taught for the past three years because of his responsibilities as IBB executive director, he still mentors students in his lab.

I take my responsibility as a mentor and supervisor seriously. It is important to have one-to-one interactions, Garca said. I take a practical approach and feel it is critical to explain why learning a topic is important, sharing practical applications, and offering experiential hands-on learning. I have had very supportive and engaged mentors and would like to pass that on to others.

Background

A native of Puerto Rico, Garca originally came to the states to study at Cornell University. He was very interested in the emerging field of biomedical engineering, but his father, an industrial engineer, advised him to major in another engineering discipline as a backup in case the biomedical field didnt develop as anticipated. Garca took his fathers advice, earning his bachelors degree in mechanical engineering and also taking biology and bioengineering classes.

During his senior year Garca participated in a project to design a structure to support fractured legs for horses. He worked to optimize the way a boot attached to the bone so that it wouldnt fracture again. He became interested in research, and his professors recommended that he go to graduate school. He earned his masters and Ph.D. in bioengineering from the University of Pennsylvania. Garca was the first person in his immediate family to earn a doctoral degree.

Garca and his family have embraced all things Georgia Tech. He and LaPlaca have two sons, Rafael, a Tech mechanical engineering (ME 2018) graduate working at GTRI, and Andrs, a fourth-year mechanical engineering student at Tech. They hold season basketball and football tickets. One of their dogs is named Buzz.

Garca said he was deeply honored, humbled, and shocked when Georgia Tech President ngel Cabrera called and told him he had been selected for this years Distinguished Professor Award. The award is special to me because it reflects the great contributions my friends, family, and peers have made in my life to get me to this point. I am grateful for my trainees, my collaborators, and colleagues, and for the support that Georgia Tech has provided in giving me the tools to succeed. Georgia Tech is the best, Garca said.

Quotes From Colleagues and Former Students

Professor Garca has been an integral part of growth of the international reputation of our bioengineering program and the Institute for Bioengineering and Bioscience. Having seen the sustained impact that he has had on students from K-12 (Project Engages) through graduate students, he is a remarkable educator who I feel is well deserving of this award.

Sam Graham Eugene C. Gwaltney Jr. School Chair in Mechanical Engineering Georgia Tech

He remains on my short list of speakers because I resonate so strongly with his approach very deep technical skills, outstanding problem definition, and tremendous colleague in service and collegiality. He is also a terrific mentor, and his former lab members are stars. He cares about doing great science and teaching people what he learned. Andrs Garca is a gem at Georgia Tech, and as an alum I hope you can keep him there he is doing some of the best biology on campus and is a superb attractor of the best students from MIT.

Linda G. Griffith S.E.T.I. Professor of Mechanical and Biological Engineering Director, MIT Center for Gynepathology Research Chair, MIT Biological Engineering Undergraduate Programs Committee

The lab around Professor Garca performs research at a unique broadness and depth. His remarkable combination of professional and personal skills is the key for his success and makes him a highly estimated collaboration partner for other scientists across disciplines and continents. He is the most invited American scientist at plenary lectures in European conferences on biomaterials. This is not only due to the high quality of his work, but also to his ability as a communicator and active discussion partner, his openness to address new topics in collaboration, and his passion for science and education that truly inspires and motivates young researchers.

Arnzazu del Campo Director INM-Leibniz Institute for New Materials Professor, Materials Synthesis, Saarland University

The five years that I spent in Andrs lab were transformative for me, and the influence of that experience is difficult to put into words. Andrs taught me many things how to be a scientist; how to develop creative and impactful ideas; how to execute on those ideas; how to write; how to present, etc. But more important than all the technical aspects of what I learned from Andrs, I learned from him who I wanted to be. Most of my professional life, and much of my personal life, is modeled after what I have learned from watching Andrs as a professor, colleague, friend, father, and husband.

Charles Gersbach Professor, Department of Biomedical Engineering Director, Center for Biomolecular and Tissue Engineering Director, Center for Advanced Genomic Technologies Duke University

Local foundation awards $1.25 million to MIND Institute to study rare genetic condition – UC Davis Health

By adminJune 25, 2021Stell Cell Research

The RDM Positive Impact Foundation is funding an ambitious $1.25 million research project at the UC Davis MIND Institute to study SYNGAP1. The rare genetic condition causes seizures (epilepsy), intellectual disability and developmental delays. It is also highly associated with autism; about half of all SYNGAP1 patients have an autism diagnosis.

A staff member conducts research in the Segal Lab.

Ron Mittelstaedt and his wife, Darin, who live in El Dorado Hills, run the foundation. Hes the executive chairman of Waste Connections, a solid waste and recycling company with 20,000 employees in North America, and his family operates Toogood Estate Winery in Somerset. The Mittelstaedts have donated millions of dollars to organizations that help children over the past 15 years.

This time, its personal.

About three and-a-half years ago, Ron Mittelstaedts best friend died, leaving behind three sons and their families. Ive become sort of a surrogate dad, and now a surrogate grandfather, explained Mittelstaedt. One of those grandsons was diagnosed with a SYNGAP1 mutation a year and-a-half ago. With the familys support, Mittelstaedt is providing meaningful funding to the MIND Institute to advance research about the syndrome.

The reality is, like many rare conditions, there arent a lot of great options. So, were trying to find potentially life-changing treatment that hopefully may impact the lives of people with SYNGAP1, he said.

Mittelstaedt was previously on the MIND Institutes inaugural National Council of Visitors (then called the MIND Institute Advisory Council), and funded a successful research project that developed a blood test for Tourette syndrome.

A staff member in the Silverman lab conducts behavioral neuroscience research.

We are grateful to the Mittelstaedts for their generosity, said MIND Institute Director Leonard Abbeduto. As a collaborative hub for preclinical and clinical research on neurodevelopmental disability, the MIND Institute is uniquely suited to build on past successes and tackle the complexities of SYNGAP1 to provide help for families.

The funding also supports UC Davis' $2 billion fundraising campaign, Expect Greater: From UC Davis, For the World, the largest philanthropic endeavor in university history. Together, donors and UC Davis are advancing work to prepare future leaders, sustain healthier communities, and bring innovative solutions to today's most urgent challenges.

SYNGAP1-related non-syndromic intellectual disability is a rare neurodevelopmental condition caused by a variation in one gene. The gene, SYNGAP1, contains instructions for making a protein (SynGAP). This protein is located at the junctions between nerve cells, called synapses, and helps regulate changes important for memory and learning. The protein also helps regulate communication between neurons.

When the variation is present, the SYNGAP1 protein in cells is reduced which causes an increase in the excitability in the synapses. This makes it difficult for neurons to communicate and increases the likelihood of seizure events. This can lead to a variety of symptoms:

Jill Silverman

SYNGAP1 syndrome affects 1-4 out of 10,000 people. The first patient was identified in 2009.

The MIND Institutes interventional genetics team includes faculty who specialize in multiple research areas.

Ron Mittelstaedt

Each of us is a world expert in our particular discipline, so bringing us all together means the chances of success are much more likely, said Jill Silverman, associate professor in the Department of Psychiatry and Behavioral Sciences and an internationally recognized expert in the use of rodent models for therapeutic development. Silvermans Lab is known for its expertise in behavioral neuroscience research.

In addition to Silverman, the SYNGAP1 team includes three other MIND Institute faculty members:

The sum of the group is going to be much greater than anything we could have done alone, said Fink, whose lab focuses on therapeutic development for neurodevelopmental conditions and neurodegenerative diseases. The fact that the foundation has funded us as a team, across multiple centers and programs is really unique. This funding brings us all together for an important project.

Kyle Fink in his lab, which focuses on therapeutic development for neurodevelopmental conditions and neurodegenerative diseases.

The researchers will work on parallel tracks, each contributing a piece of the puzzle.

Silverman will conduct specialized behavioral tests on mouse models of SYNGAP1, using tools with corresponding metrics in humans, such as EEGs (a type of brain scan) to determine clinically relevant outcomes.

Nord and Fink will create a new mouse model that contains the mutated human SYNGAP1 gene, while Segal and Fink will create new molecular therapies to counter that mutated gene. Theyll also figure out how to deliver those therapies to the brain.

Were not just trying to treat the symptoms of the disease with a drug, explained Segal, whose lab specializes in molecular analysis. We are trying to change the underlying genetic condition, and our particular approach is to do that in a way that does not change the DNA sequence. We use tools to change the gene expression instead, which we think will make safer therapies. Its really a state-of-the-art approach. Its molecular therapy.

David Segal working in his lab, which specializes in molecular analysis.

The collaborative approach, often called team science, coupled with the RDM Positive Impact Foundations support, allows for an ambitious, fast-tracked research program. The $1.25 million frees the researchers from the need to apply for multiple federal grants and enables them to focus immediately on SYNGAP1.

The team excels in whats often called bench to bedside research, translating results from the lab directly into therapies for patients.

We see these patients, we meet with them, were on Zoom calls with them and I want to find something that works for them. I want to change their lives. Thats what Im driven by, Silverman said.

Silverman, Fink and Segal have had previous success with their work on another rare genetic condition, Angelman syndrome, which causes developmental delay, speech and balance challenges and intellectual disability.

David Segal

Their labs helped to create and characterize the first rat model of Angelman syndrome last year. The Segal lab also created a protein therapeutic that could increase the level of the affected gene in mouse models of Angelman syndrome, a major discovery.

All three labs are still working on a wide range of therapeutics for Angelman, including molecular therapies delivered with viruses or stem cells and novel small molecule compounds.

Ron Mittelstaedt is hoping for another success story, this time with SYNGAP1, but hes also realistic about the research process.

We are all very aware that going down this path doesnt guarantee anything except the ability to get up to bat, and we could get a hit or strike out. But doing nothing guarantees you dont get a hit, so its important for us to take action, and were hopeful well hit a home run.

UC Davis researchers get $3 million FAST grant to find treatment for Angelman syndrome

The UC Davis MIND Institute in Sacramento, Calif. was founded in 1998 as a unique interdisciplinary research center where families, community leaders, researchers, clinicians and volunteers work together toward a common goal: researching causes, treatments and potential prevention of neurodevelopmental disabilities. The institute has major research efforts in autism, fragile X syndrome, chromosome 22q11.2 deletion syndrome, attention-deficit/hyperactivity disorder (ADHD) and Down syndrome. More information about the institute and its Distinguished Lecturer Series, including previous presentations in this series, is available on the Web at mindinstitute.ucdavis.edu.

MeCP2 is a microsatellite binding protein that protects CA repeats from nucleosome invasion – Science Magazine

By adminJune 25, 2021Stell Cell Research

MeCP2 binds hydroxymethylated CA repeats

Despites of decades of research on the Rett syndrome protein MeCP2, its function remains unclear. Ibrahim et al. show that MeCP2 is a hydroxymethylated cytosine-adenosine (CA) repeat-binding protein that modulates chromatin architecture at a distance from the transcription start site (see the Perspective by Zhou and Zoghbi). MeCP2 accumulates and spreads around modified CA repeats and competes for nucleosome occupancy. Loss of MeCP2 results in a widespread increase in nucleosome density inside lamina-associated domains and transcriptional dysregulation of genes enriched in CA repeats. These results shed light on the underlying molecular mechanism of Rett syndrome, a severe disease associated with mutations in MeCP2.

Science, abd5581, this issue p. eabd5581; see also abj5027, p. 1390

Rett syndrome is a severe neurodevelopmental disorder that is mainly caused by mutations in the methyl-CpG-binding protein 2 gene (MeCP2). Initially, MeCP2 was identified as an essential brain protein that binds to methylated CpG (mCG) via its methyl-binding domain (MBD) and acts as transcriptional repressor. However, during early brain development, the postnatal accumulation of MeCP2 parallels the genome-wide high-level accumulation of hydroxymethylcytosine (hmC) and methylated CpA (mCA), suggesting that MeCP2 may also recognize and bind to DNA sequences that contain these modified nucleotides.

The ability of MeCP2 to recognize both mCA and hmC as well as mCG has led to conflicting conclusions regarding its function in transcriptional regulation, because these cytosine modifications are associated with either repression (mCA and mCG) or activation (hmC) of transcription. The unambiguous identification of the MeCP2 target sequence(s) would help to clarify this issue.

CA repeats (CAn) represent ~1% of the mouse genome and belong to the microsatellite family. They are widely distributed throughout the genome and have been shown to affect transcription of nearby genes. Our recent data reveal that CAn are methylated (mCAn) or hydroxymethylated (hmCAn) in various cell types. In a search for proteins that could specifically recognize and bind these CA repeats, we identified MeCP2 as a specific reader of CA repeats. We hypothesized that the methylation status of CAn is essential for the recognition and binding of MeCP2, possibly through recognition of the modified nucleotides in CA repeats with distinct affinities, relevant for its neuronal function in transcriptional regulation.

Here we show that within the MBD family, MeCP2 is the only protein that specifically recognizes and binds to CA repeats, with much stronger affinity than mCG and mCA. MeCP2 selectively recognizes CA repeat DNA in a strand-specific manner and requires at least five consecutive CA dinucleotides to optimally bind DNA. While MeCP2 can bind in vitro to modified and nonmodified CA repeats, it exhibits impressive selectivity toward hydroxymethylated CA repeats, which are modified by DNA (cytosine-5)-methyltransferase 3A. The modified cytosine, only when located within a CA repeat, serves as a nucleation point for both MeCP2 accumulation and spreading around the repeat, which, in turn, correlates with nucleosome exclusion. In addition, loss of MeCP2 results in widespread increase in nucleosome density within lamina-associated domains (LADs) and transcriptional dysregulation of CA repeatenriched genes located outside LADs.

We have also dissected the molecular basis of the MeCP2 hydroxymethylated CA repeat recognition by solving the crystal structure of MeCP2 in complex with hmCAn. The CA repeat creates a well-defined DNA shape, with a considerably modified geometry, including a widened major groove and negative roll parameters, located precisely at the modification site. We show that the molecular recognition of the hydroxymethylated CA repeat specifically occurs through Arg133, a key MeCP2 residue whose mutation causes Rett syndrome.

Our work identifies MeCP2 as a hydroxymethylated CA repeat DNA binding protein that targets the 5hmC-CA-rich sequence, which are specifically located on one strand. Our data provide insights into the origin of Rett syndrome at the molecular level and suggest that this neurodevelopmental disorder could be viewed as a chromatin disease, originating from the inability of mutant MeCP2 to bind and protect the CA repeats from nucleosome invasion. Our results open a previously unexplored area of research focused on understanding the role of specific protein binding to microsatellites and other repeats in neurological diseases of unknown etiology.

MeCP2 is a microsatellite binding protein that specifically recognizes hydroxymethylated CA repeats. Depletion of MeCP2 alters the chromatin organization of CA repeats and LADs and results in nucleosome accumulation on CA repeats and genome-wide transcriptional dysregulation. WT, wild-type; KO, knockout.

The Rett syndrome protein MeCP2 was described as a methyl-CpG-binding protein, but its exact function remains unknown. Here we show that mouse MeCP2 is a microsatellite binding protein that specifically recognizes hydroxymethylated CA repeats. Depletion of MeCP2 alters chromatin organization of CA repeats and lamina-associated domains and results in nucleosome accumulation on CA repeats and genome-wide transcriptional dysregulation. The structure of MeCP2 in complex with a hydroxymethylated CA repeat reveals a characteristic DNA shape, with considerably modified geometry at the 5-hydroxymethylcytosine, which is recognized specifically by Arg133, a key residue whose mutation causes Rett syndrome. Our work identifies MeCP2 as a microsatellite DNA binding protein that targets the 5hmC-modified CA-rich strand and maintains genome regions nucleosome-free, suggesting a role for MeCP2 dysfunction in Rett syndrome.

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MeCP2 is a microsatellite binding protein that protects CA repeats from nucleosome invasion - Science Magazine

Bedford Biotech Restores "Meaningful Vision" in Blind Patients With Gene Therapyand May Soon Go Public Dallas Innovates -…

By adminJune 25, 2021Gene Therapy Clinics

Update 6/23/21: Nanoscope Therapeutics announced today that it has received FDA approval of its Investigational New Drug application for its Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with advanced retinitis pigmentosa (RP). Its randomized, double-blind, sham-controlled Phase 2b trial will begin this month in locations across the U.S.

When you have retinitis pigmentosa, the world slowly goes dark. Most patients with RP lose most of their sight by young adulthood and are often legally blind by age 40. The genetic disorder affects around one in 4,000 people, causing the retinas photoreceptive cells to degrade over time. But a new gene therapy is giving hope and producing life-changing resultsand it could lead to treatments for far more common retinal diseases.

Bedford-based Nanoscope Therapeutics is trying to turn the light back on for RP patientsby developing gene therapies using light-sensitive molecules that could re-sensitize the retina to detect low light levels. That could restore vision in millions of visually impaired people who suffer from RP and other retinal degenerative diseases, like Stargardt disease and dry age-related macular degeneration (AMD). The startups gene therapies, called optogenetics, aim to bring sight to the blind. Nanoscope is one of a number of teams and companies exploring optogenetics as a treatment for RP.

There is no treatment for the diseases that were working on, Nanoscopesco-founder, president, and chief scientific officer, SamarendraMohanty, told Dallas Innovates. So theres a real unmet need that we are trying to fill.

Samarendra Mohanty [Photo: Nanoscope Therapeutics]

Nanoscope, a clinical-stage biotech company, announced earlier this month that its Phase 1/2a clinical study using Multi-Characteristic Opsin (MCO) on RP patients had restored clinically meaningful vision. Significant dose-dependent improvement of visual acuity was demonstrated at 16 weeks, and continued through one year in patients suffering from severe RP, the company said.

The study included 11 patients with advanced RP who had either no light perception or just perception of light in the study eye and no better ability than counting fingers in their other eye. The studys initial positive results were reported at the American Academy of Ophthalmologys 2020 annual meeting last November.

After MCO treatment, the patients reported long-lasting improvements in outdoor light sensitivity and daily activities, saidthe principal investigator, Dr. Santosh Mahapatra, an ophthalmologist and eye surgeon, in a statement. We were pleasantly surprised that after eight weeks of treatment, some subjects could attend their follow-up visits during the study without the assistance of a chaperone. Some of the patients even gained the ability to read letters on a wall or even the large text in a newspaper, use a cell phone, watch television, and could even thread a needle.

Another benefit of the treatment: Vision restoration was produced without the need of stimulating retinal implants or explants (goggles).

Nanoscope co-founder and CEO Sulagna Bhattacharya said the trial studys impact on patients lives has been powerful.

Their quality of life improves significantly, she told us. This is a relief to patients family members, healthcare systems, and society as a whole.

Sulagna Bhattacharya [Photo: Nanoscope Therapeutics]

We expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the U.S. this summer to further validate our gene therapys ability to improve clinically meaningful vision in RP patients, Bhattacharya said in the statement. If successful, it will be the first-ever restorative drug for millions of RP patients worldwide.

On June 23 the company announced it has received FDA approval of its application for the 2b trial, and specified that the trial would begin this month. The trial will involve eight to 10 centers in the U.S., from Beverly Hills to Florida to other locations on the east coast.

[Image: Ivan-balvan/istockphoto]

Nanoscopes RP gene therapy has received orphan drug designation from the FDA. It uses a proprietary AAV2 vector to deliver MCO genes into the retina. This mutation-independent gene therapy is delivered via a single injection through the eye administered in a doctors office.

All 11 subjects participating in the trial had objective and subjective improvement in functional vision, Nanoscope reports. Shape discrimination accuracy improved more than 90% in all the subjects compared to baseline. Further, the performance in two different mobility tests improved by 50%, including the reduction in time to touch a lighted panel. Nanoscope says the test outcomes were highly correlated with improved patient-reported outcomes.

Nanoscopes co-founder, Mohanty, is the inventor of the technology used in the trial.

Optogenetics is a powerful research tool, he said in a statement, but had limited scope of clinical benefit because the opsins had a narrow band of activation, unlike natural light environment. MCO is sensitive to broadband light and activatable by ambient light, thus eliminating the risk of photo-toxicity from long-term continuous use of external intense light stimulation devices.

Nanoscope Therapeutics got a $2 million grant from the National Eye Institute in June 2020 and closed anoversubscribed Series A funding roundin July 2020 to help fund its clinical trial.The startup, a TechFW client, is a spinout from Nanoscope Technologies, serving as a commercialization partner for the R&D enginesMCO vision restoration work. It has 12 employees and a group of consultants and advisors.

Nanoscope Technologies, meanwhile, has received around$10 million in grants from theNational Institutes of Health to help fund its R&D,Bhattacharya said.

The CEO added that the RP study could lead to pivotal results.

Were very excited about our Phase 1/2a results, Bhattacharya said. This trial has the potential to become pivotal, which will allow our product to be available in the clinics to treat millions of blind individuals for whom there is no treatment so far.

Beyond the RP trials, Mohanty says his company placing a big focus on dry age-related macular degeneration (AMD),

Thats a big indication that we are targeting, Mohanty said of AMD, since unlike RP, AMD is super prevalent. According to the National Eye Institute, 11 million Americans have AMD.

Mohanty said that AMD is very rapidly progressing as we are aging and worldwide there are major concerns.

Nanoscope plans to initiatemultiple trials to treat both dry AMD and Stargardt disease, another inherited retina disorder.

Nanoscope Technologies was founded in 2009 by Mohanty. Bhattacharya joined the company as co-founder in 2013. Mohanty and CFO Anthony Togba told Dallas Innovates they are open-minded to the option of going public with their company, and doing due diligence to prepare for that potentiality.

CFO Anthony Togba [Photo: Nanoscope Therapeutics]

Togba says the company is performing readiness activities, undertaking internal processes, and putting in place the structures to be ready to go public.

The timeline? By late Q4 or before, well have a better idea about where were headed, Togba said. The upcoming clinical trial is the preoccupation now to make sure that we have a flawless execution to obtain those results that we expect.

Quincy Preston contributed to this report. It was updated on 6/23/21 to reflect FDA approval of Nanoscopes Phase 2b trial application.

The investment was led by Advantech Capital, a PE fund based in China that focuses on TMT, pharmaceuticals, and healthcare. This combined with the support from the Cancer Prevention and Research Institute of Texas (CPRIT), which granted OncoNano $9.97 million last year, will support Phase 3 clinical trials for the biotech's technology that can diagnose and treat cancer with high specificity.

Taysha Gene Therapies, which rocketed from a UTSW spinout to a $157 million IPO in under six months last year, has gone global with rights to TSHA-120, apromising AAV9 clinical-stage genetherapy. It's a historic announcement. There are no current treatments for giant axonal neuropathy, or GAN,a severe, progressive disease that affects the central and peripheral nervous systems.TSHA-120 is the first-ever successful in-human intrathecal (spinal) gene transfer.

In this weekly roundup of executive moves in North Texas, you'll also find news from Liberty Capital Bank, Krista Software, Tuesday Morning, Trive Capital, Cantey Hanger, UNT, JUNO, NuZee, Jaunt Air Mobility, Korbyt, and ID90 Travel.

The Series C funding brings Allied BioSciences total to more than $80 million, the team told us. It will be used to grow the biotech's flagship product, SurfaceWise2, which is an active surface coating that can continuously destroy 99.9 percent of viruses on surfaces. Last year, SurfaceWise2 was the first and only surface coating that the EPA approved for continuous protection against COVID-19 with a single application.

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‘Like turning back the clock’: Windsor dad with cystic fibrosis among patients seeking access to new therapy – CBC.ca

By adminJune 25, 2021Gene Therapy Clinics

Windsor, Ont., dad Rian Murphy has had cystic fibrosis since childhood, and wants to live to see his year-old son grow up. Murphy has hope he and other people with the respiratory disease will get access to a new drug, Trikafta, which was approved Friday by Health Canada.

Rian Murphy was diagnosed with cystic fibrosis as a child andnever expected to live into his 30s, but Health Canada's recent approval of a new breakthrough drug treatmenthas theWindsor, Ont., dad hopefulhe'll be spending many more years with hisson.

"It's a massive step going forward for cystic fibrosis patients such as myself," said Murphy about the triple-combination therapy Trikafta. "At the end of the day, it's a big, big window of opportunity for us to look down the road, future-wise."

On Friday, Trikaftawasapproved for use in patientsage 12 and over who have aminimum of one of the CF F508del gene mutations.

Cystic Fibrosis Canada (CFC) calls Trikaftaa "transformational" therapy that couldtreat up to 90 per centof Canadianswith theprogressive, genetic disease, whichaffects the lungs and digestive system, and is the most common fatal genetic disease in children. TheCFCestimates one in every 3,600 children is born with the disease, and over4,370 Canadiansattend specialized clinics.

Over time, the CFC says, Trikafta could reduce severe lung disease by 60 per cent andthe number of deaths by 15 per cent,and increase life expectancy by several years, the CFC says in quoting research.Clearing the airways from mucus buildup is important in CF care.

In the last three years, half of Canadians who died of cystic fibrosis were under age 34.

"I never thought about retirement. Inever thought about those things because my whole life I was told you're never going to make it until you're 20, you're 30," said Murphy, who with wife Diane are parents to their year-old son Logan.

Three years ago, Murphy lost significant lung function andwas hospitalized for threeweeks at St. Michael's Hospital in Toronto. He hadbeen on and off intravenousantibiotics for months.

"On an average day, I'm doing about two to three hours of masks, and vests and physiotherapy, not including all the pills I take," he said.

"I'm 34 years old. If I can obtain this drug [Trikafta] and take it for the recommended period of time to get the results, it would be like me turning back the clock."

There's no cure for CF. While other therapies work to address the symptoms, Trikafta helps the defective protein function more effectively.

With Health Canada's approval, doctors can now prescribe Trikafta.

But aswith a couple of other drugs for CF, provincial insurance coverage for Trikaftaremains a concern for patients, Kelly Grover, president and chief executive officer of CFC, saidin a release.

"We turn to the provinces next. They must immediately fund Kalydeco and Orkambi, which have been in negotiations for more than a year, and fund Trikafta as soon as possible. Provincesend the wait and save lives."

The pan-Canadian Pharmaceutical Alliance (pCPA) isa regulatory body that negotiates drug prices on behalf of the provinces.

In astatement released shortly after Health Canada announcedapproval of Trikafta, thepCPAsaid it has agreed to negotiate prices for the CF drugsOrkambi and Kalydeco, and Trikafta might be added to the agreements, pending a positive regulatory and health technology assessment recommendation.

CBC reached out to the Ontario government for comment, and in an email, the Ministry of Health said the province "recognizes that the cystic fibrosis community is anxious for access to new and effective treatments such as Trikafta," and "will continue to work productively through the established review and pCPA processes."

In the meantime, Murphy and his wife have started a fundraiser, hoping to raise enough money so hecan eventually access the drug.

"If I can get a couple months, that's huge," he said.

His wife Diane, who'sactively involved with CFC, as well as a petition and Instagram groups calling for the Ontario government to fund Trikafta, encourages the public to send letters to the province.

Shehas hopes of her husband "watching our child grow up."

Link:
'Like turning back the clock': Windsor dad with cystic fibrosis among patients seeking access to new therapy - CBC.ca

5 potential benefits of exosome treatment – AZ Big Media

By adminJune 8, 2021Stem Cell Treatment

For the better part of the 2000s, stem cell therapy ruled the public health conversation in the United States. The only thing that came close to supplanting it as the most controversial science and health topic was cloning.

These days, its normalized enough that people line up for treatments involving stem cells without giving it a second thought. Exosome treatment is one of the more popular varieties, and theres no wonder why. It has a broad range of benefits, many of which youll learn about if you read on.

Before COVID-19, the opioid epidemic was the biggest public health issue in the United States. As important as solving that issue is, it cut the number of options available to chronic pain patients.

Without effective treatment and accommodation, chronic pain affects mobility, mood, and relationships. It makes daily life and employment difficult. Suffering from it and the ensuing struggles can even lead to suicide.

The good news is that exosome therapy and other stem cell treatments lend some hope.

Arthritis is a common immune condition that causes great pain for many. Immune system disorders often involve miscommunication between cells. Exosomes primary function is communication, solving that issue, and boosting the immune system.

Joint inflammation is a key symptom of arthritis but exists in other forms, as well. Inflamed joints after injuries can end athletes seasons without proper treatment. Exosome therapy treats joint inflammation and pain, whatever the cause.

Surgery solves an endless range of ailments and helps achieve appearance goals. In terms of risk, theres never been a better time to get surgery. Laparoscopy, lasers, and robots are a few of many tools that reduce tissue damage.

Todays post-surgery therapies have folks back to regular activity faster than we imagined possible even a decade ago. Exosome treatment and other stem cell therapies are one way to restore function sooner than later.

No matter how advanced surgery gets or how effective rehab becomes, there are always risks. Issues with anesthesia, infections, and even freak accidents like surgeons sewing their equipment into patients bodies are all too common. The only way to remove these concerns is by avoiding surgery.

Exosome therapy is a non-invasive substitute for some operations. It doesnt come with the same risks or recovery period. Its also a great option for elderly people who cant risk surgery and folks with conditions that make it impossible.

Exosomes can turn around someones quality of life by solving a painful condition or restoring mobility. Theyre also useful for less pressing matters, such as restoring youthful looks.

Treatments like Botox and collagen injections arent long-lasting and can lead to adverse reactions. Because exosome therapy stimulates cell production, the body fills in wrinkles and restores skin elasticity. It doesnt come with the infamous stiffness of Botox and wont droop as dermal fillers can.

Anti-aging therapies arent a must for everyone, but they are for some, making this extra important.

Whether you think its right or not, we have high expectations for entertainers and models. Showing your age in some professions can push you out of your field. Using exosomes to reverse the aging process has a less artificial look than some other procedures and lasts longer, extending careers.

Medication is the most popular treatment for erectile dysfunction (ED), to the point that solutions have nicknames like the little blue pill. Despite pills popularity, they have several downsides.

The most popular ED meds have no long-term benefits: You rely on them for each sexual encounter. They can interact with other drugs and arent recommended for patients with certain conditions, such as heart disease and both high and low blood pressure.

ED pills also come with ugly side effects, including headaches and gastrointestinal distress.

Exosomes, on the other hand, have long-lasting results and no major side effects. Rather than providing a temporary fix, they help heal damaged nerves and tissues. This can increase how long erections last. For some, the method also boosts penile length and girth.

The treatment also helps people with conditions such as Peyronies disease, also known as PD. The main symptom is built-up scar tissue that results in a curved penis. Some PD patients cant have sex due to erectile dysfunction and/or pain.

That all can change for PD patients who undergo exosome therapy. The healing process awakens dormant cells and improves blood flow. It makes enjoyable sex possible again.

Bald is beautiful, but its not everyones cup of tea. Those who have a lot of pride in their hair may see their self-confidence tank when they go bald. It affects some folks sex lives, whether thats because their significant others dislike it or because they dont feel attractive and struggle to get in the mood.

For all of these reasons, theres an infinite range of treatments and has been pretty much since the beginning of recorded history. The grand majority of them never amounted to much, and some were downright nasty!

If youve tried everything from hair plugs to superstitious treatments without success, dont despair.

Exosome treatment is a modern solution for hair restoration, and its effective. Its not like treatments that try to mask hair loss or graft hairs from one part of the head to another. Instead, exosomes restore follicles so hair can grow again.

Expect to hear more and more about exosome treatment in the coming years. Its one of the most modern medical treatments available and continues growing due to its wide range of benefits.

If you want to learn about more of the latest and greatest science to make your life better and info to propel you to success, youre on the right website. Our articles are sure to inform and entertain, so click on another one and pick up new knowledge today.

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5 potential benefits of exosome treatment - AZ Big Media

Orion and the Blood Service launch collaboration to develop new CAR T-cell cancer therapy – Yahoo Finance Australia

By adminJune 8, 2021Stem Cell Treatment

ORION CORPORATION PRESS RELEASE 8 JUNE 2021 AT 8.00 a.m. EEST

Orion and the Blood Service launch collaboration to develop new CAR T-cell cancer therapy

Orion Corporation and the Finnish Red Cross Blood Service have concluded an agreement on research collaboration with the aim of developing chimeric antigen receptor (CAR) T-cell therapy.

Cancer immunotherapies, with CAR T-cell therapy as one form of therapy, have introduced entirely new possibilities for cancer treatment. In CAR T-cell therapy, the patient's own white blood cells are genetically modified to attack the cancer and kill it. CAR T-cells are currently used to treat certain haematological cancers.

The first T-cell therapies received marketing authorisation in the USA in 2017 and in Finland in 2019 for the treatment of recurrent acute lymphocytic leukaemia and B-cell lymphoma. Despite the good treatment results, development needs have been identified in CAR T-cell therapies, which is why research related to CAR-T cell therapy is being pursued.

The Blood Service has considerable experience in cell research, in the supply of cord blood and stem cell grafts classified as tissue products, and in the preparation of ATMP cell products classified as medicinal products. The Blood Service also has expertise and the necessary clean rooms for the research and high-quality production of therapeutic cells.

Orion has a solid research infrastructure and has introduced several proprietary drugs to the market in different therapy areas. Orion's oncology therapy area researches and develops novel proprietary drugs for the treatment of cancer. In recent years, Orion has expanded its research activities to include immuno-oncology therapies.

New possibilities for cancer treatment

The research collaboration is based on new innovations to improve the structure of the CAR T-cell product. Matti Korhonen, Senior Medical Officer, who leads the research at the Blood Service, believes that the collaboration offers a great opportunity to develop new cell therapy products for patient care. According to Oliver Cooper, Ph.D., director of Discovery Sciences at Orions R&D, Orions drug development expertise will provide the necessary momentum for this project.

Story continues

Targeted immunotherapy has opened up entirely new possibilities for the treatment of cancer, and it is important to advance this development in Finland, too. I also believe that as a result of the development work, more and more patients will be able to benefit from this effective treatment, says Matti Korhonen

Project managers Satu Juhila from Orion and Jan Koski from the Blood Service say that the collaboration has got off to a good start: Both parties are very enthusiastic regarding the opportunities of the collaboration and about advancing the development of cell therapy in Finland. We believe that the collaboration will provide plenty of new opportunities for the development of cell therapy products.

Finnish Red Cross Blood Service The Finnish Red Cross Blood Service is the nationwide blood service provider in Finland. Its services comprise recruiting blood donors, organising blood donation, collecting blood and testing the donated blood. It is also responsible for producing and storing blood products and delivering them to hospitals. The Finnish Red Cross Blood Service is also responsible for supplying stem cell transplants, the HLA typing of organ and stem cell transplants and various laboratory testing related to blood. The Blood Services Cell Production Centre develops and produces experimental cell therapy products for the treatment of patients. The Finnish Red Cross Blood Service conducts scientific research related to its field. More detailed information is available in the Blood Service annual report 2020.

Further information:

Orion Corporation: Outi Vaarala Senior Vice President, Research and Development, Orion Corporation Tel. +358 10 426 3472 outi.vaarala@orionpharma.com

Contact person for the media:

Terhi Ormio Vice President, Communications, Orion Corporation Tel. +358 50 966 4646 terhi.ormio@orion.fi

Finnish Red Cross, Blood Service: Matti Korhonen, Senior Medical Officer Finnish Red Cross, Blood Service Tel. +358 50 396 9450 matti.korhonen@veripalvelu.fi

Contact person for the media: Willy Toiviainen, Director of Communications and Human Resources Finnish Red Cross, Blood Service Tel. +358 40 523 7451 willy.toiviainen@veripalvelu.fi

Publisher: Orion Corporation Communications Orionintie 1A, FI-02200 Espoo, Finland Homepage: http://www.orion.fi/en

Orion is a globally operating Finnish pharmaceutical company a builder of well-being. Orion develops, manufactures and markets human and veterinary pharmaceuticals and active pharmaceutical ingredients. The company is continuously developing new drugs and treatment methods. The core therapy areas of Orions pharmaceutical R&D are neurological disorders, oncology and respiratory diseases for which Orion develops inhaled pulmonary medication. Orions net sales in 2020 amounted to EUR 1,078 million, and the company had about 3,300 employees at the end of the year. Orions A and B shares are listed on Nasdaq Helsinki.

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Orion and the Blood Service launch collaboration to develop new CAR T-cell cancer therapy - Yahoo Finance Australia

Improvements in the Treatment of Patients With MCL Over the Past Decade – Oncology Learning Network

By adminJune 8, 2021Stem Cell Treatment

Transcript

Hi, I'm Peter Martin. I'm the Chief of the Lymphoma Program at Weill Cornell Medicine in New York. My colleagues and I were interested in how our patients with mantle cell lymphoma (are) actually treated in the United States. We have seen over the past decade a lot of significant improvements in the outcomes of patients with mantle cell lymphoma in clinical trials.

The real question that comes up though is, How do the results of those clinical trials translate into practices that are using the therapies that are defined in clinical trials as they are defined. What are the outcomes that we're getting in those patients who are being treated outside of clinical trials, mostly in community practices in the United States?

We used the deidentified database derived from electronic medical records in the Flatiron database with a statistician, who did an excellent job. We came up with a series of questions.

One, what are the patterns of care that are being used in the United States in these practices? Two, what are the outcomes of patients being treated in these practices? Three, how is stem cell transplantation being used, and what are the outcomes of patients who receive stem cell transplantation compared to patients who do not receive stem cell transplantation?

Separately, Dr. Gilles A. Salles evaluated the role of rituximab maintenance using the same deidentified Flatiron electronic medical record database, and those data are being presented at EHA.

What we found was that, in general, there was less use of standard treatment regimens that you might think would be a case. This could be a variety of reasons. One is there may be insufficient communication regarding what standard treatments should be used. That's one possibility. Another possibility is that the treatment regimens that are being developed may not apply to the broad set of people who develop mantle cell lymphoma in the United States, either related to comorbidy conditions, age, geography, or other socioeconomic factors.

As a researcher who spends a lot of time thinking about how to treat people with mantle cell lymphoma better, one of the takehome messages that I've learned from this study is that it doesn't make sense to develop a regimen that can't be administered broadly in the United States. There are a lot of good regimens.

If they can only be administered in academic settings, then we're really ignoring the majority of people with mantle cell lymphoma. As researchers, we can do a better job developing regimens that are applicable more broadly to people with mantle cell lymphoma. Those regimens should focus on practicability in addition to efficacy and safety.

Whenever you look at deidentified health record data, there's always a chance that there are some biases. For example, we don't have response rates. A lot of these factors could impact the results in one way or another, overcome that to some degree by having a large number, in this case, over 4,000 patients to start with.

But really, the key when you're looking at data like this is to trying to focusing on certain key questions and then to validate those questions in other databases, ideally, separate databases for different countries or different types of medical records. That's what we're hoping to do in the next step.

One of the findings that is most relevant from my perspective was we are finding that amongst patients who we defined, in this case, a stem cell transplant eligible and there are debates about how that should be done. In those patients, we did not find a significant advantage through the use of stem cell transplantation.

Now, there are at least 2 ongoing large randomized clinical trials in the United States and in Europe that are evaluating stem cell transplant. Ultimately, if we are going to rule out stem cell transplantation in the world, we need to have the results of those clinical trials.

As we increasingly find large datasets like this one, Flatiron dataset, showing that there may not be an advantage to stem cell transplantation, I think as researchers, we need to start coming up with treatments that might be applicable to younger patients that don't use stem cell transplantation.

We need to be prepared to move forward with regimens that are reasonable, effective, and welltolerated that maybe don't use stem cell transplantation so that when those large clinical trials read out, we're ready to take those trials into the next phase of large randomized trials.

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Improvements in the Treatment of Patients With MCL Over the Past Decade - Oncology Learning Network

Novo Nordisk partners with Heartseed on heart failure cell therapy – PMLiVE

By adminJune 8, 2021Stem Cell Treatment

Danish pharma company Novo Nordisk has announced a new collaboration and licence agreement with Japans Heartseed to develop the companys investigational cell therapy HS-001 for heart failure.

HS-001, Heartseeds lead asset, is an investigational cell therapy using purified cardiomyocytes derived from induced pluripotent stem cells (iPSC). The therapy is currently being developed as a treatment for heart failure.

Heartseed is already planning to launch a phase 1/2 study of HS-001 in Japan in the second half of 2021, which will evaluate the safety and efficacy of the therapy for the treatment of heart failure caused by ischaemic heart disease.

Under the terms of their agreement, Novo Nordisk will gain exclusive rights to develop, manufacture and commercialise HS-001 globally, excluding Japan where Heartseed will retain the rights to solely develop the therapy.

However, Novo Nordisk has the rights to co-commercialise HS-001 with Heartseed in Japan, with equal profit and cost sharing.

In return, Heartseed is eligible to receive up to a total $598m, with $55m earmarked in upfront and near-term milestone payments.

The Japanese biotech company is also eligible to receive tiered high single-digit to low double-digit royalties of annual net sales on the product outside Japan.

"We are delighted to have a company with the expertise and resources of Novo Nordisk as our partner for development and commercialisation of HS-001, and are also honoured that Novo Nordisk has recognised the innovativeness and high potential of our technology," said Keiichi Fukuda, chief executive officer of Heartseed.

"We believe that the partnership with Novo Nordisk is very valuable as we seek to disseminate our Japan-origin innovation globally as early as possible, he added.

Through this important collaboration with Heartseed, we aim to pioneer novel treatment solutions for people with cardiovascular disease, said Marcus Schindler, chief scientific officer, EVP research and early development at Novo Nordisk.

We [will] gain access to an innovative clinical asset, underlying technology and deep expertise within the field of iPSC biology and cardiac cell transplantation, which can be combined with our knowledge and capabilities in stem cell biology and manufacturing, he added.

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Novo Nordisk partners with Heartseed on heart failure cell therapy - PMLiVE