Daily Archives: April 20, 2022

(The Conversation is an independent and nonprofit source of news, analysis and commentary from academic experts.)

Steven Walkley, Albert Einstein College of Medicine and Melissa Wasserstein, Albert Einstein College of Medicine

(THE CONVERSATION) Mr. and Mrs. Smith, we finally have an answer for you. The couple, whose real names we are protecting for privacy, looked at me anxiously. I had been evaluating their young daughter, Sally, in my role as a medical geneticist at the Childrens Hospital at Montefiore in the Bronx, a borough of New York City. For years, the Smiths had been searching to learn why Sally was suffering from epilepsy, why she didnt seem to understand them and why she wasnt speaking at 6 years of age. In 2021, they ended up in my clinic.

I decided to send a sample of Sallys blood for whole-exome sequencing, a test that could identify a change in one of her genes that might be responsible for her symptoms. A few weeks later I had the answer.

Sally has an extremely rare disorder that youve probably never heard of, I told them. Its so rare that it doesnt even have a real name yet. Its called NAA10-related disorder. The family looked at me with blank stares. I took a deep breath and continued.

The NAA10 gene codes for an enzyme that modifies critical proteins, enabling them to function properly. A single change in Sallys NAA10 gene would cause the enzyme to be made incorrectly, resulting in intellectual disability and seizures. The NAA10 gene is located on the X chromosome, which is one of two sex chromosomes in humans.

Males typically carry an X and a Y chromosome, while females usually have two X chromosomes; as a result, boys are usually more severely affected and girls have a less predictable course. I explained to the family that only about 50 other people with NAA10-related disorder have been reported across the globe. They then asked me about treatment. I replied sadly, none. I could see them struggling to wrap their heads around this.

They asked further questions about what might happen to Sally: Will she learn to speak? Will she be able to learn? Will she grow old? I told them that there is not enough experience to accurately predict what Sallys future will look like. Feeling useless, I said, Here is a patient support group that might be helpful. And with nothing more to offer, I added: Ill see you in a year.

Moments like this a long-awaited answer that is met with more bewilderment than relief are not uncommon in the practice of medical genetics. Most people expect that after a long, frustrating search, finding the underlying diagnosis will provide answers and a path forward. But sometimes, in cases like Sallys, the answer simply begets more questions.

Weve faced these difficult questions as two researchers with decades of experience in rare genetic diseases. One of us is a medical geneticist whose clinical work focuses on the diagnosis and management of individuals with rare genetic disorders; the other is a neuroscientist working to determine how rare genetic diseases impact brain function and possible ways to correct them.

Putting rare disease into context

Most so-called rare diseases are poorly understood and have no treatment. The National Institutes of Health has estimated that there are about 7,000 rare diseases, defined as ones affecting fewer than 200,000 Americans. Many rare diseases, however, are like NAA10-related disorders and affect only a handful of individuals.

Major advances in the precision and speed of gene sequencing technology followed by dramatic reductions in the costs of testing have radically changed how medical genetics clinics function. Next-generation genetic sequencing, which was so expensive just a decade ago that it was used only after all other testing options had been exhausted, is now the go-to test in most clinics.

But while sequencing can provide confirmation of a suspected, well-understood condition, it frequently results in a situation like that faced by the Smiths, where the testing result shows an incredibly rare disorder with little known about it.

Putting the puzzle pieces together

The speed and ease with which modern gene sequencing can generate a diagnosis stand in sharp contrast to the prolonged effort required to understand how the genetic variant causes disease. Humans all have the same 20,000-plus genes, which govern the traits that make us characteristically human, such as a large brain, 10 fingers and round pupils. Changes, or variants, in these genes determine our uniqueness. So while we all have genes that tell our bodies to make hair, variants in these genes can make hair that is straight or curly, brown or red. Some genetic variants, however, change the gene product so significantly that they result in disease.

Unraveling the natural history of a rare condition requires years of focused attention by clinicians and scientists. Researchers like us also work to piece together the complex puzzle of how a rare genetic difference can alter metabolic pathways in the brain, as well as other organs that might be affected.

Over time, a fuller picture of the rare disorder begins to emerge. The role of the gene in normal cells or commoner diseases unfolds, as well as possible therapies. For instance, potential treatments might involve replacing or modifying a gene that isnt properly functioning, infusing a vital enzyme that an individuals body isnt making or prescribing a specialized diet or medications. But before one can determine how to treat a genetic disease, researchers first need to determine what is altered and not working normally. Only after this is understood can we begin to envision treatment.

Personalized medicine offers a way forward

To provide our patients and their families with more answers, we here at the Albert Einstein College of Medicine have begun a program in which we build what we call Gene Teams. These consist of parents or caregivers, their childs physician and interested scientists and their trainees. These researchers are typically working on deciphering the genes function, its encoded protein or the role the gene or protein plays inside of cells.

We bring all the team members together, and the childs physician outlines what is known about the clinical condition, followed by the parents sharing their childs story. The scientists and their trainees then provide an accessible tutorial to the families about what the gene and its associated protein do in cells. Whenever possible our team also discusses ways by which the condition could be treated.

These tutorials are the first encounters in ongoing relationships. Remarkably, three different families who were empowered by their Gene Team experience have gone on to establish foundations focused on their childs disease, and they have built networks to other families affected by the same rare condition worldwide. These are the STAR Foundation for SLC17A5, the KARES Foundation for KDM5C and the CACNA1A Foundation. The scientists, too, after the team meetings, have often gone on to build major research projects, some focused on the exact variant observed in the affected child.

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We as scientists and our trainees have also been transformed by our involvement with the Gene Teams. Working directly with the families brings real-life experience to our laboratory work and inspires us and other researchers to remember that our work matters not only for expanding scientific knowledge but also for helping families in need.

We have learned that the blank stare experienced in the doctors office following diagnosis of a rare disease can be transformed by empowering families not only with greater knowledge of the involved gene, but also with an understanding that they are not alone and that there can be a more hopeful path forward.

This article is republished from The Conversation under a Creative Commons license. Read the original article here: https://theconversation.com/when-it-comes-to-the-rarest-of-diseases-the-diagnosis-isnt-the-answer-its-just-the-starting-point-177424.

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When it comes to the rarest of diseases, the diagnosis isn't the answer it's just the starting point - Jacksonville Journal-Courier

The major pharma and biotech money winners this week were tech-heavy, including a DNA editing platform, a machine-learning platform that creates digital patient twins and wearable temperature-monitoring patches. Continue reading for that and more.

Tessera Therapeutics

Genetic medicine pioneer Tessera Therapeutics raked in a huge Series C financing worth more than $300 million. The funding was led by Flagship Pioneering, a subsidiary of the Abu Dhabi Investment Authority, Alaska Permanent Fund Corporation and Altitude Life Science Ventures, among others.

The Series C attracted many investors because of Tesseras unique genetic gene writing technology. Unlike CRISPR gene editing, which destroys damaged DNA, Tesseras gene writing platform writes new sequences of DNA into the genome. This gives the unique platform the potential to both cure and prevent nearly any disease at a scalable level.

We are thankful for the support from our new partners and existing investors alike in this latest funding round. It is our belief that genetic medicine will be the most important next epoch in medicine, said Geoffrey von Maltzahn, Ph.D., CEO of Tessera Therapeutics.

Unlearn.AI

Unlearn is a machine-learning technology that creates a digital twin of patients in clinical trials to enable smaller, faster studies. The unique company closed a $50 million Series B round this week, led by Radical Ventures, Insight Partners, DCVC Bio and Mubadala Capital Ventures.

The funding will help advance Unlearns TwinRCT platform feature, which uses a patients medical history, disease information and AI to essentially create an extra patient that functions as though it were receiving a placebo treatment in a trial. This allows the real patients to receive the trial medication and potentially benefit from it. The funding will also help expand Unlearns goal of expanding into clinical trials.

Blue Spark Technologies

As a leader in wearable remote patient monitoring solutions, Blue Spark Technologieswas able to raise a $40 million growth fund. The funding is an intellectual property-based debt solution led by Ghost Tree Partners and Aon. With the monetary support, Blue Spark hopes to create more patient monitoring solutions. Currently, its only product on the market is TempTraq, a Bluetooth-enabled disposable patch that patients wear to monitor fever spikes for up to 72 hours. TempTraq is an FDA-cleared Class II medical device, and Blue Spark says it has more products in the pipeline.

Having Ghost Trees support and expertise will be invaluable as we continue to expand our remote patient monitoring solutions to the market, said John Gannon, president and CEO of Blue Spark.

Alzheon

Massachusetts-based Alzheon, a clinical-stage biopharma company developing therapies to treat and diagnose Alzheimers disease, closed an oversubscribed Series D round of financing worth $50 million. The press release said that the funding came from private and institutional investors, but did not name anyone specifically.

The funding will largely go toward Alzheons ALZ-801 drug, an oral agent that blocks the formation of amyloid plaque in the brain, a significant contributor to Alzheimers Disease. ALZ-801 has shown positive results throughout a Phase II biomarker trial, and Alzheon hopes the Series D financing will help bring the product to market.

Trevena

Trevena, a biopharma company that develops medicines to treat Central Nervous System (CNS) diseases, received a $15 million tranche from its royalty-based financing agreement with an affiliate of R-Bridge Healthcare Fund. The original agreement totaled roughly $40 million and included an initial $15 million tranche, $15 million for the sale of opioid-based analgesic Olinvyk to China, and $10 million dependent on achieving milestones.

R-Bridge is an affiliate of CBC Group, one of Asias largest pharma and biotech groups. R-Bridge will receive royalties from Trevenas license with Jiangsu Nhwa Pharmaceutical and is hoping to have Olinvyk approved by China by the end of 2023.

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Money on the Move: Tessera, Unlearn.AI, Blue Spark, Alzheon and Trevana - BioSpace

By Ed Miseta, Chief Editor, Clinical LeaderFollow Me On Twitter @EdClinical

Recent years have seen unprecedented innovation in the clinical space. Precision medicine, cell and gene therapies, decentralized trials, real-world data, and the promise of artificial intelligence (AI) and machine learning (ML) are just a few of the reasons to be excited about the future of clinical research. But what can we expect to see in the next three years, and what are the challenges sponsor companies will need to overcome?

A webinar hosted by IBM Watson hoped to answer those questions. The discussion featured Lorraine Marchand, general manager of life sciences at IBM Watson Health; Nimita Limaye, research VP, Life Sciences R&D Strategy and Technology at IDC Health Insights; and Greg Cunningham, director of the RWE Center of Excellence at Eli Lilly and Company. The three shared insights into what we might expect to impact trials over the next three years.

In this article the panel discusses precision medicine and real-world data. In part 2 of this article the panel looks at the future of decentralized clinical trials.

The Growth Of Precision Medicine

The first game changer the panel discussed is the advancement of precision medicine. It has moved from exploring single gene mutations to performing research using combinations of genes. This change has the potential to bring better drug targets forward and get the best products to patients faster.

This has been playing out in the last decade in oncology real-world evidence, notes Cunningham. We've seen an evolution in precision medicine as we've built out the patient record. As we have done that, the marketplace has evolved rapidly, particularly for electronic medical record data and genomic data.

Pharma companies were happy to get their hands on electronic medical record data. When genetic test results were combined with that data, researchers gained the ability to look at a single mutation and develop better patient outcomes.

Where precision medicine will continue to evolve in 2022 and beyond is the growing use of genetic testing in oncology. This will provide the industry with more data about patients. With more genes at their disposal, researchers can look at groups of genes and the complex combinations of gene mutations. This has the potential to open the door for tools like artificial intelligence to help researchers analyze the complex number of permutations.

RWD Creates More Efficient Research

Next the panel discussed RWD and the ability to utilize it across several use cases from discovery and development to commercial. Limaye likes the prospect of being able to create a data exchange where researchers can bring together claims, clinical, EMR, and genomics data directly from patients to create an intelligent and digital patient health record. That record gives researchers the digital equivalent of a real-life patient which can be used as a natural history or synthetic control arm in randomized control clinical trials.

These data can allow drug developers to track patient response to drugs and look at outcomes after being exposed to new therapies. The promise of data and technology is using tools like AI to advance therapies and get them to patients faster. This will be done with better information and a much more efficient way to perform drug development and track and monitor outcomes in patients.

Big data has been a topic of discussion in pharma for years. The volume of clinical data is now growing exponentially. Approximately 30% of the world's datavolume is being generated by the healthcare industry and by 2025, the compound annual growth rate will hit 36%. That's 6% faster than manufacturing, 10% faster than financial services, and 11% faster than media & entertainment.

In addition to getting bigger, data is also getting broader. Researchers can not only look at a patients medical history but can now consider factors such as social determinants of health and behavioral data.

Since most EHRs do not include genomic data, researchers need the ability to look at patient data more holistically. Type 2 diabetes was one example discussed. Today, 40% to 70% of it is genetically inherited and there are over 500 different genetic loci which could be involved in causing the disease. The earlier strategy of looking at genetic risk scoring was single trait. That is now transitioning to multi-trait research with an integrated view that will drive a precision medicine strategy. This creates an interesting situation where drug discovery will continue to get more specific and focused towards an individual while also getting bigger and broader.

The Challenge Of RWD

With access to RWD, drug developers can benefit from data they may not have known existed. Although the data is rich and robust, it can be difficult to access. One of the biggest challenges the industry faces is data stored in silos. The panel notes data is stored in patient claims, electronic medical records, in lab apps, images, and genetic files on a smartphone. Having the technology to tap into those sources to identify quality data is the primary challenge.

The data must be de-identified for patient privacy, cleaned, curated to remove noise, and enriched, which means bringing together the various components that will be meaningful to drug development. That will allow researchers to have a patient record that is useful across pharma, from development through to commercial. An exchange would enable that exact process a platform where various entities can bring their data to have it linked, integrated, cleaned, and enriched, creating a data package that can be plugged into studies.

An important component of that exchange is the data being housed in a place where various third parties can feel comfortable bringing their data to match it with data from other third parties.

Cunningham cites lupus as an example of where pharma could benefit from such an exchange. I would like to have a complete data set of lupus, he says. Lupus is an autoimmune condition, and a quintessential data set could be used for a number of uses, such as preparing a Phase 1 trial, selecting patients, or understanding patient responses to different therapies when designing studies. Specific data sets could be created for each therapeutic area, and pharma companies need that hard work of bringing the data together removed.

Data Assembly And Analysis

Currently, drug developers spend 80% of their time assembling data and 20% of their time analyzing it. The situation must be flipped so that 80% of the time is spent performing analysis. The panel recommends rethinking how health records are created. The healthcare and life science industries require the ability to easily put data together. That comes back to investing in data standards everyone can agree upon. With the right standards and technology, the industry can spend its time improving lives as opposed to assembling data.

The FDA has indicated it is aware and supportive of the fact that pharma needs use RWD in drug discovery. The industry now needs to create the interoperability, standards, and methods to ensure that data can be included in regulatory submissions. This evolution may be akin to the critical path initiative. When the FDA embraced the idea of the critical path and allowing more in silico modeling of clinical trial design and development, it took the industry almost 10 years to adopt and apply the guidance.

The FDA has said it recognizes the importance of RWD, but that acknowledgement has resulted in few approvals. Looking at the use of synthetic control arms and RWD in regulatory submissions over the last five years shows just 10 submissions and all were in oncology. Only one was a successful submission, and the rest were rejected because of lack of completeness of the data.

Those numbers should tell the industry the FDA is not going to dictate how to get to approvals. The industry is going to have to figure out the interoperability and how to apply the standards. Regulators are always going to require quality data. Industry will need to enrich the data and create the cohort that is going to be equivalent to a patient in the real world.

In part 2 of this article, the panel discusses the role of technology in clinical trials, how decentralized trials will continue to evolve, what capabilities sponsor companies will need, and whether decentralized trials might offer cost benefits to companies.

Original post:
The Next Three Years Of Clinical Trials DCTs RWE And Beyond - Clinical Leader

Bordeaux, France / April 19th, 2022 TreeFrog Therapeutics, a biotechnology company aimed at making safer, more efficient and more affordable cell therapies based on induced pluripotent stem cells (iPSCs), today announced the launch of The Stem Cell SpaceShot Grant, a $100,000 research funding in the field of stem cell biology and regenerative medicine.

The Stem Cell Spaceshot Grant is open to PhD-level scientists and PhD students conducting research in stem cell biology, biophysics, gene editing, cell therapy, and bioproduction engineering. With this grant, TreeFrog Therapeutics aims at supporting scientific discoveries with therapeutic impact in 4 areas:

1. Progressing stem cell culture and cell therapy products through biomimetic strategies

2. Upgrading stem cell-products and manufacturing processes using new technologies and scientific approaches

3. Improving the quality control and safety profile of stem cells and stem cell-derived transplants

4. Enhancing the engraftment, integration and long-term survival of stem-cell derived transplants

To apply, researchers only need to submit an email, a one-page research project description and a graphical abstract through an online form available on the TreeFrog Therapeutics website. Applications will be opened from May 1st to June 30th 2022. 10 nominees will be selected in August 2022. The winner will be awarded in October 2022 following interviews with an interdisciplinary jury composed of world-class experts in stem cell biology, gene editing and biophysics:

Emeritus Prof. Peter ANDREWS, University of Sheffield, UK STEM CELL GENOMIC INTEGRITY

Prof. L. MAHADEVAN, Harvard University, USA BIOPHYSICS

Marta SHAHBAZI, PhD, MRC Laboratory of Molecular Biology, Cambridge, UK DEVELOPMENTAL BIOLOGY

Justin EYQUEM, PhD, University of California San Francisco, USA GENE EDITING

Shin KAWAMATA, MD, PhD, R&D Center for Cell Therapy, FBRI, Kobe, Japan CELL THERAPY MANUFACTURING & QUALITY CONTROL

Pierre NASSOY, PhD, CNRS, France BIOIMAGING & OPTOFLUIDICS

The technology of TreeFrog Therapeutics emerged at the cross-roads of stem cell biology and biophysics after I met my co-founder, Kevin Alessandri, in an atomic bunker of the Geneva University while we were post-docs. Today, our C-StemTM technology demonstrated its capacity to mass-produce stem cells and stem cell-derived products with unprecedented scalability and quality, and TreeFrog Therapeutics raised over $82M to advance a pipeline of cell therapies to the clinic and finance the global deployment of C-StemTM. I believe its now our role to encourage the next generation of scientists to make discoveries that will shape the future of regenerative medicine. We designed the grant that we would have liked to apply to: the application process is simple, fast and anonymous - so that it is ideas, rather that resumes that are awarded , no cumbersome reporting is required, and the intellectual property of scientists is preserved. I cant wait to read the first projects!

Maxime Feyeux, PhD, co-founder and Chief Scientific Officer, TreeFrog Therapeutics

About TreeFrog Therapeutics

TreeFrog Therapeutics is a French-based biotech company aiming to unlock access to cell therapies for millions of patients. TreeFrog Therapeutics is developing a pipeline of therapeutic candidates using proprietary C-StemTM technology, allowing for the mass production of induced pluripotent stem cells and their differentiation into ready-to-transplant microtissues with unprecedented scalability and cell quality. Bringing together over 70 biophysicists, cell biologists and bioproduction engineers, TreeFrog Therapeutics raised $82M over the past 3 years to advance its pipeline of stem cell-based therapies in the field of neurodegeneration, cardio-metabolic disorders, and immuno-oncology. In 2022, the company will open technological hubs in Boston, USA, and Kobe, Japan, to drive the adoption of C- StemTM and initiate co-development partnerships with leading academic, biotech and industry players in the field of cell therapy.

Media Contact

Pierre-Emmanuel Gaultier

TreeFrog Therapeutics

+ 33 6 45 77 42 58

pierre@treefrog.fr

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TREEFROG THERAPEUTICS LAUNCHES A $100,000 RESEARCH GRANT IN REGENERATIVE MEDICINE - BioSpace

Posoleucels third RMAT designation marks an unprecedented regulatory distinction among cell and gene therapies

Global Phase 3 multi-virus prevention trial initiated in March 2022 and is enrolling patients

WALTHAM, Mass., April 20, 2022--(BUSINESS WIRE)--AlloVir (Nasdaq: ALVR), a late clinical-stage allogeneic T cell immunotherapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead investigational multi-virus-specific T cell therapy, posoleucel, for the prevention of clinically significant infections and disease from six devastating viruses that commonly impact high-risk adult and pediatric patients following allogeneic hematopoietic cell transplant (allo-HCT) adenovirus (AdV), BK virus (BKV), cytomegalovirus (CMV), Epstein-Barr virus (EBV), human herpes virus-6 (HHV-6) and JC virus (JCV). This is the third RMAT designation that FDA has granted to posoleucel, in recognition of the therapys transformative potential to address significant unmet medical needs facing immunocompromised allo-HCT patients.

The FDA previously granted RMAT designation to posoleucel for the treatment of hemorrhagic cystitis (HC) caused by BKV in adults and children following allo-HCT and for the treatment of adenovirus infection following allo-HCT. RMAT designation enables early interactions with the FDA to discuss clinical trial design and other actions to expedite development and review. Outside of the United States, the European Medicines Agency has granted posoleucel PRIority Medicines (PRIME) designation for the treatment of serious infections with AdV, BKV, CMV, EBV and HHV-6.

"The receipt of three RMAT designations for a single therapy is unprecedented. Posoleucels three RMAT designations reflect the strength of AlloVirs multi-virus platform and its potential both to deliver an important treatment option for immunocompromised patients who currently have none, and to transform the management of allo-HCT patients with a multi-virus prevention approach," said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer, AlloVir.

Story continues

Posoleucel has the potential to fundamentally transform the landscape for allo-HCT by preventing life-threatening viral diseases and infections, either as a prophylactic therapy in high-risk patients or as a preemptive therapy in patients who have already reactivated one or more of the six viruses targeted by posoleucel. As 90% of allo-HCT patients reactivate at least one of these viruses, there is a large global market opportunity for the prevention of devastating viral diseases, with an estimated addressable patient population of 40,000 allo-HCT patients annually.

The new RMAT designation was based on initial data from an open-label Phase 2 study evaluating the potential for posoleucel to prevent life-threatening infections from six common viruses following allo-HCT. Initial data from this study were most recently presented at the 48th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) in March 2022. Out of 26 patients who received at least one dose of posoleucel in the ongoing Phase 2 trial, and including those who completed, discontinued or are continuing posoleucel, only three clinically significant infections were observed through Week 14, as of the data cut-off for this analysis. Of the 24 patients who had reached the Week 14 primary endpoint, 21 remained free of clinically significant infections. Repeat dosing was generally well-tolerated. Final results of the Phase 2 study are expected to be available at the end of this year.

About Posoleucel

AlloVirs lead product, posoleucel, is in late-stage clinical development as an allogeneic, off-the-shelf, multi-virus specific T cell therapy targeting six viral pathogens in immunocompromised individuals: adenovirus (AdV), BK virus (BKV), cytomegalovirus (CMV), Epstein-Barr virus (EBV), human herpes virus-6 (HHV-6) and JC virus (JCV). In the positive Phase 2, proof-of-concept CHARMS study, more than 90% of patients who failed conventional treatment and received posoleucel, demonstrated a complete or partial clinical response based on predefined criteria, most with complete elimination of detectable virus in the blood and resolution of major clinical symptoms.

Posoleucel is being studied in three Phase 3 clinical trials for three distinct indications - the treatment of virus-associated HC, the treatment of AdV infection, and the prevention of infections and disease caused by posoleucels six target viruses. A Phase 2 proof-of-concept trial with posoleucel for the preemptive treatment of BKV in adult kidney transplant recipients is also ongoing.

In addition to the RMAT designations for multi-virus prevention and for the treatment of AdV and virus-associated HC, the FDA has also granted posoleucel Orphan Drug Designation for the treatment of virus-associated HC. The European Medicines Agency has granted posoleucel PRIority Medicines (PRIME) designation for the treatment of serious infections with AdV, BKV, CMV, EBV and HHV-6, and Orphan Medicinal Product designation as a potential treatment of viral diseases and infections in patients undergoing HCT.

About AlloVir

AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in pediatric and adult patients with weakened immune systems. The companys innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, single- and multi-virus-specific T cells for patients with T cell deficiencies who are at risk from the life-threatening consequences of viral diseases. AlloVirs technology and manufacturing process enable the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio. For more information, visit http://www.allovir.com or follow us on Twitter or LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding AlloVirs development and regulatory status of our product candidates, the planned conduct of its preclinical studies, and clinical trials and its prospects for success in those studies and trials, and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties, and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to AlloVirs financial results, the timing for the initiation and successful completion of AlloVirs clinical trials of its product candidates, whether and when, if at all, AlloVirs product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, the impact of the COVID-19 pandemic on AlloVirs product development plans, supply chain, and business operations and other risks identified in AlloVirs SEC filings. AlloVir cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. AlloVir disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent AlloVirs views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220420005069/en/

Contacts

Media and Investor Contact: Sonia ChoiAlloVirschoi@allovir.com

Excerpt from:
FDA Grants Regenerative Medicine Advanced Therapy (RMAT) Designation to AlloVirs Posoleucel for Prevention of Multiple Life-Threatening Infections...

EDEN PRAIRIE, Minn., April 19, 2022 (GLOBE NEWSWIRE) -- Miromatrix Medical Inc. (NASDAQ: MIRO), a life sciences company pioneering a novel technology for bioengineering fully transplantable organs to help save and improve patients lives, announced that CEO Jeff Ross will present at the 2022 Cell & Gene Meeting on the Mediterranean. The conference, held this year on April 20 22 in Barcelona, Spain, brings together the leading public and private sector companies in the field of advanced therapies.

Mr. Ross will deliver his remarks at 3:45 pm CET on April 21st live from the Gaudi 3 Ballroom, which will be simultaneously streamed for virtual attendees. The recordings will also be uploaded into the virtual platform within 24 hours for further on-demand viewing. A short Q&A will follow his presentation.

I am honored to have been invited to speak at this years Meeting on the Med amongst the top luminaries in advanced therapies, said Jeff Ross, Miromatrix CEO. Transplant medicine is seeing advancements and evolution like never before, and we find ourselves on the precipice of the most important, transformative moment in our field. Miromatrixs proprietary perfusion technology platform for bioengineering organs is a vital part of this moment, and we believe our approach can help address the shortage of available human organs and bring hope to millions of people.

The Cell & Gene Meeting on the Mediterranean is the leading conference bringing together the ATMP community from Europe and beyond. It features in-person and pre-recorded virtual presentations by leading public and private companies highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and regenerative medicine. Over 650 attendees are expected to take part in the 2022 hybrid conference.

Registration for the conference is available here.

About MiromatrixMiromatrix Medical Inc. is a life sciences company pioneering a novel technology for bioengineering fully transplantable human organs to help save and improve patients lives. The Company has developed a proprietary perfusion technology platform for bioengineering organs that it believes will efficiently scale to address the shortage of available human organs. The Companys initial development focus is on human livers and kidneys. For more information, visit miromatrix.com.

Investor Contact:Greg Chodaczek332-895-3230ir@miromatrix.com

Media Contact:press@miromatrix.com

Link:
Miromatrix CEO Jeff Ross to Present at the 2022 Cell & Gene Meeting on the Med - GlobeNewswire

High blood pressure means the force of blood flowing through your arteries is greater than it should be. If not controlled, it could damage your blood vessels and cause other health problems.

High blood pressure (hypertension) tends to be a condition we associate with being too sedentary or getting older. But high blood pressure can also be a genetic condition, affecting people who are otherwise fit and healthy.

A parent with high blood pressure can pass along a gene to a child, raising that persons risk of developing hypertension one day. Familial hypertension may also result from a family lifestyle that includes high blood pressure risk factors, such as smoking or an unhealthy diet.

Blood pressure is the force of circulating blood against the inner wall of your arteries. Its measured in millimeters of mercury (mm Hg) and is presented as two numbers:

According to the American Heart Association, healthy blood pressure is a systolic pressure of less than 120 mm Hg and a diastolic pressure of less than 80 mm Hg. This is a blood pressure of less than 120/80 mm Hg.

If your blood pressure is higher than that, doctors consider you to have elevated blood pressure or stage 1 or 2 hypertension.

Risk factors for high blood pressure include a family history of hypertension, as well as:

What makes high blood pressure so dangerous is that it can exist for a long time without presenting any obvious symptoms. Measuring your blood pressure is the only way to know if you have hypertension.

In extreme cases, when blood pressure exceeds 180/120 mm Hg, you have a medical emergency known as a hypertensive crisis. Symptoms can include:

Research from 2017 suggests that high blood pressure results from a combination of factors, including genetic, environmental, and behavioral components.

Unlike some diseases with only one or a few genes as risk factors, familial hypertension can result from variations in hundreds of different genes, according to a 2019 study of more than 750,000 individuals. This makes it difficult to pinpoint specific genes that could be treatment targets.

The Centers for Disease Control and Prevention (CDC) also notes that families may affect a persons hypertension risk because of the home environment.

Smoking or even breathing in secondhand smoke can raise blood pressure risks. A diet high in sodium and saturated fat may also cause a blood pressure increase. If physical activity and good sleeping habits arent part of a family dynamic, blood pressure can also be negatively affected.

Monogenic hypertension refers to blood pressure caused by one genetic variant inherited from a parent. Monogenic hypertension accounts for about 30 percent of hypertension cases. Most of those are associated with imbalances of electrolytes, such as potassium.

There are several types of monogenic hypertension syndromes, each with a unique set of origins and symptoms. These include:

Knowing about your family medical history is important for many reasons. A history of certain cancers, for example, may determine when you get screened for those cancers. If high blood pressure runs in your family, its important to share this information with your doctor and regularly monitor your blood pressure.

One way to organize information about your family health history, as well as your own, is to use My Family Health Portrait, an online tool created by the National Institutes of Health. You can gather your family medical history, share it with other relatives, and learn about your risk levels for conditions that tend to run in families.

If your blood pressure is currently at a healthy level, you can make several key lifestyle adjustments to lower the odds of it rising too much. If your blood pressure is higher than usual, these steps, along with medications, may help you bring it back down to a healthy range:

The National Heart, Lung, and Blood Institute developed the Dietary Approaches to Stop Hypertension (DASH) eating plan as a heart-healthy eating strategy.

This plan focuses on managing blood pressure by emphasizing fruits, vegetables, whole grains, lean proteins, and sodium reduction. Its also flexible enough to let people enjoy many of their favorite foods.

Sufficient sleep is essential to good overall health, especially for brain and heart function. Blood pressure is especially susceptible to problems related to poor sleep.

A 2022 study suggests that frequent sleep disturbances and short sleep, or less than 5, 6, or 7 hours, can contribute to hypertension.

Taking steps to improve sleep duration and quality may improve more than just your cardiovascular health. It can also improve your mood, concentration, energy, metabolism, and more.

Hypertension is a major risk factor for cardiovascular disease, the leading cause of death in the United States. High blood pressure is also a leading cause of stroke and a risk factor for chronic kidney disease and other health problems.

If your family medical history includes high blood pressure, start taking steps to lower your risk through heart-healthy behaviors. Even if you dont know your family history or dont have a close relative with hypertension, its still important to take steps to keep your blood pressure under control.

Theres a variety of anti-hypertensive medications that can help. But these medications dont take the place of a healthy diet, exercise, and getting plenty of sleep to help maintain a healthy blood pressure.

Originally posted here:
Familial Hypertension: The Genetics of High Blood Pressure - Healthline

This document has been translated from the Japanese original for reference purposes only.

In the event of any discrepancy between this translated document and the Japanese original, the original shall prevail. The Company assumes no responsibility for this translation or for direct, indirect or any other forms of damages arising from the translation.

April 20, 2022

Company name: Modalis Therapeutics Corporation

Stock exchange listing: Tokyo Stock Exchange

Code number: 4883

URL:https://www.modalistx.com/en/

Representative: Haruhiko Morita

Modalis Therapeutics to Present Data Supporting of Development of Transformative Epigenetic Modulating Medicines for the Treatment of a Type of Muscular Dystrophy and the Other Genetic Disorders at the ASGCT Annual Meeting

MDL-101 preclinical data support durability and efficacy of a differentiated precision medicine approach for Congenital Muscular Dystrophy type 1a

Preclinical data demonstrating our proprietary CRISPR based epigenetic modulating technology regulates target gene expression, demonstrating its potential as a therapeutic approach for serious genetic disorders.

20-Apr-2022 TOKYO & Waltham, Mass - Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM epigenetic modulating technology, today announced that six scientific abstracts have been accepted for presentation at the 25th Annual Meeting of the The American Society of Gene & Cell Therapy (ASGCT), being held in Washington D.C. and virtually, May 16-19, 2022. The abstracts present preclinical data from the Company's Congenital Muscular Dystrophy type 1a (CMD1A) and the other rare disease programs in cardiovascular and neuroscience indications, as well as validation of our technology.

Modalis presentations at ASGCT will include preclinical data demonstrating that:

Our single AAV vector system coding dCas9-trans activating domain fusion protein and gRNA targeting LAMA-1 gene (AAV9-CRISPR-GNDM-LAMA1) upregulate LAMA-1 protein to compensate LAMA-2 function in LAMA-2 knock out mice that improved survival, supporting continued development of MDL-101 for the treatment of CMD1A; and

"At ASGCT, we will present preclinical data on MDL-101 that have validate our differentiated therapeutic strategy leveraging our CRISPR-GNDM (guide nucleotide directed modulation) technology for CMD1A which has been an undruggable target. We believe that MDL-101 has the potential to be a life changing gene modulation therapy for CMD1A, and Modalis remains on track to file an IND by end 2023," said Tetsuya Yamagata, M.D. Ph.D., Chief Scientific Officer, Modalis Therapeutics. "We will also share exciting preclinical data showcasing use of our proprietary epigenetic technology to modulate genes to restore expression levels of disease-causing genes. These data reinforce our view that we have a unique epigenetic modulating approach with the potential to treat a wide range of serious genetic disorders that have been unapproachable with other platforms."

The complete list of Modalis Therapeutic presentations is below. Abstracts can be accessed on the ASGCT website and the presentations will be posted on the Modalis website during the conference.

Oral Presentations:

Title: NGS based evaluation of AAV genome integrity for improved production and function Date and Time: 5/16/2022 11:45AM

Session Name: Vector Manufacturing and Engineering 1

Title: Novel single AAV vector treatment for Congenital Muscular Dystrophy type 1A (MDC1A) using CRISPR-GNDMtechnology

Date and Time: 5/18/2022 10:30AM

Session Name: Musculo-skeletal Diseases

Poster Presentations:

Title: Robust suppression of Tau by CRISPR-GNDM system for treatment of Tauopathies Date and Time: 5/16/2022 5:30 PM

Session Name and poster board#: Neurologic Diseases I /M-141

Title: Utilizing CRISPR-GNDM mediated gene activation of the extra-large gene titin for the treatment of dilated cardiomyopathy and other titinopathies

Date and Time: 5/17/2022 5:30 PM

Session Name and poster board#: Cardiovascular and Pulmonary Diseases/Tu-124

Title: Blocking SNHG14/UBE3A-ATS lncRNA Transcription with Dead Cas9 (CRISPR-GNDM) Can Un-Silence Paternal UBE3A in an Angelman Syndrome Mouse Model Date and Time: 5/18/2022 5:30 PM

Session Name and poster board#: Neurologic Diseases III/W-153

Title: Evaluation of Cas9 mediated immune response effect on long term transgene expression in WT mice and NHPs without immune suppression

Date and Time: 5/18/2022 5:30 PM

Session Name and poster board#: Immunological Aspect of Gene Therapy and Vaccine II /W-252

About MDL-101

MDL-101 is an experimental, epigenetic modulation therapy under investigation for the treatment of Congenital Muscular Dystrophy type 1A (CMD1A). MDL-101. MDL-101 is comprised of guide nucleotide targeting LAMA-1 gene, a highly homologous sister gene of the disease-causing gene LAMA-2, enzyme-null Cas9 (dCas9) fused with trans-activating domain driven by a muscle specific promoter and coded in an AAV vector. MDL-101 upregulates LAMA-1 gene products in patient's muscle tissue to compensate loss of function caused by mutation of LAMA-2, and therefore has the potential to provide a one-time, durable treatment benefit for people living with CMD1A.

About Modalis:

Modalis Therapeutics develops precision genetic medicines using epigenetic gene modulation. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or epigenetic editing without the need for double-stranded DNA cleavage, gene editing or base editing. Modalis is initially focusing on genetic disorders caused by loss of gene regulation - resulting in excess or insufficient protein production - by targeting more than 660 genes that are thought to cause human disease as a result of haploinsufficiency. Headquartered in Tokyo with laboratories and facilities in Waltham Massachusetts, the company is listed on Tokyo Stock Exchange's Growth market. For additional information, visitwww.modalistx.com.

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Modalis Therapeutics : to Present Data Supporting of Development of Transformative Epigenetic Modulating Medicines at the ASGCT Annual Meeting -...

Pune, India, April 20, 2022 (GLOBE NEWSWIRE) -- The global gene therapy market size is set to gain momentum from the rising incidence of different types of cancer. The field of this therapy is undergoing several technological advancements that would help in treating cancer in those patients who are at high risks of getting affected by this disease through genetic mutations. The report further mentions that the market size was USD 3.61 billion in 2019 and is projected to reach USD 35.67 billion by 2027, exhibiting a CAGR of 33.6% during the forecast period.

Fortune Business Insights provided this information in a new report, titled, Gene Therapy Market Size, Share & COVID-19 Impact Analysis, By Application (Oncology, Neurology, and Others), By Vector Type (Viral and Non-viral), By Distribution Channel (Hospitals, Clinics, and Others), and Regional Forecast, 2020-2027.

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Industry Developments:

Report Scope & Segmentation

Drivers & Restraints-

Increasing Innovations & Research Activities to Boost Growth

The U.S Food and Drug Administration (FDA) stated that it is expecting to receive more than 200 applications of this therapy by the end of 2020. This showcases that the rising number of research studies and innovations in this field would affect the gene therapy market growth positively in the near future. In North America, almost 208 companies are currently operating in this market. In addition to this, the Alliance for Regenerative Medicine declared that as of 2018, approximately 259 potential drug candidates are under Phase I clinical trials across the globe.

For more information in the analysis of this report, visit: https://www.fortunebusinessinsights.com/industry-reports/gene-therapy-market-100243

However, the outbreak of the COVID-19 pandemic is presently impacting the field of research. According to the director of the Office of Tissues and Advanced Therapy (FDA) named Wilson Brayan, nowadays the officials are prioritizing only those drugs that are associated with coronavirus. Hence, there hasnt been a surge in the application of potential drugs for gene therapy. This factor may hamper the gene therapy market growth in the forthcoming years.

Highlights of This Report:

Segment-

Neurology Segment to Earn High Share Fueled by High Cost of Drugs

Based on product type, the market is divided into neurology, oncology, and others. Out of these, the neurology segment earned 78.2% in terms of gene therapy market share in 2019. This growth is attributable to the increasing usage of this therapy for treating patients living with spinal muscular atrophy.

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The U.S. to Dominate Owing to Presence of Favorable Policies

In 2019, the U.S. generated USD 2.16 billion in terms of revenue. The country is expected to dominate throughout the coming years stoked by the increasing usage of advanced gene therapies for the treatment of rare conditions. Besides, the presence of favorable reimbursement policies and guidelines would also help in propelling the market growth here. As this type of treatment is not legal in several developing nations, industry giants are emphasizing on the U.S. for launching their products.

Europe, on the other hand, is anticipated to grow significantly backed by the adoption of unique treatment options. Asia Pacific is set to hold a comparatively lower share on account of the decreasing usage of gene therapy because of its expensive nature.

Fortune Business Insights lists out the names of all the gene therapy providers present in the global market. They are as follows:

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With 33.6% CAGR, Gene Therapy Market Size worth USD 35.67 Billion in 2027 - GlobeNewswire

The UAB Cardiogenomics Clinic provides genetic testing and counseling for a gene variant associated with a risk of heart failure and death.

Researchers believe that the presence of the Val122Ile genetic variant in African Americans is believed to predispose them to the development of transthyretin amyloidosis, which can lead to higher risk of heart failure.A new study published in the Journal of the American Medical Association led by researchers from the University of Alabama at Birmingham Marnix E. Heersink School of Medicine found that being a carrier of a genetic variation known as Val122Ile in the transthyretin, or TTR gene, was significantly associated with an increased risk of heart failure and death. Research shows that this Val122Ile variation is more commonly seen among individuals of African ancestry.

Transthyretin protein is produced by the liver and helps circulate vitamin A and thyroxine through the body. This genetic variation causes misfolding of the transthyretin protein leading to hereditary transthyretin amyloidosis, a condition characterized by the buildup of abnormal deposits of a protein in the bodys organs and tissues. As buildup increases over time, the heart may become stiff, leading to cardiomyopathy, a disease of the heart muscle that makes it difficult to pump blood through the heart.

For this study, UAB researchers Vibhu Parcha, M.D., and Pankaj Arora, M.D., looked at this genetic variation in a cohort of 7,500 Black individuals living in the United States.

The TTR Val122Ile genetic variant is unfortunately more common among those of African ancestry with nearly three out of 100 individuals carrying the genetic variation, said Parcha, a clinical research fellow in the UAB Cardiogenomics Clinic and the UAB Division of Cardiovascular Disease.

Parcha says the presence of the Val122Ile genetic variant in African Americans is believed to predispose them to the development of transthyretin amyloidosis.

We wanted to examine whether carrying this genetic variant will lead to a higher risk of new-onset heart failure, death due to heart failure, cardiovascular causes or any other causes, Parcha said.

(Left) Vibhu Parcha, M.D., clinical research fellow in the UAB Cardiogenomics Clinic and the UAB Division of Cardiovascular Disease. (Right) Pankaj Arora, M.D., an associate professor in the Division of Cardiovascular Disease and director of the UAB Cardiogenomics Clinic.In this study, researchers analyzed participants from the REasons for Geographic and Racial Differences in Stroke study living in the United States without baseline heart failure. Among 7,514 Black participants, the population frequency of the TTR Val122Ile variant was 3.1 percent. Over a median follow-up of 10.9 years, Val122Ile variant carriers had a higher risk of incident heart failure compared with non-carriers. Over a median follow-up of 11.6 years, Val122Ile variant carriers had a higher risk of mortality compared with non-carriers. Overall researchers found that those with the TTR Val122Ile variant had a 2.5-fold higher risk of heart failure and a 40 percent higher risk of death from any reason.

Among those with the pathogenic TTR Val122Ile genetic variation, the heart may gradually become unable to function correctly, which will lead to heart failure and ultimately death, said Arora, an associate professor in the Division of Cardiovascular Disease and director of the UAB Cardiogenomics Clinic. However, the true probability of genetic variation being expressed in all those with the variant is not known, and further work is needed to understand this. The good news is that there are several new treatments approved or awaiting approval for this hereditary heart disease.

Medical facilities like the UAB Cardiogenomics Clinic provide genetic testing for this variant. At the clinic, those who carry this variant will have access to comprehensive genetic counseling and assessment of their heart structure and function.

Those with the variant may be eligible for getting access to evidence-based therapies that improve their heart health and improve their long-term outcomes, Arora said. It is also important to identify any family members who may have the genetic variation as they will benefit from early diagnosis and access to medical therapies that improve their health.

Learn more about the UAB Cardiogenomics Clinic here.

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Genetic variation common among Black individuals is associated with higher risk of heart failure and death - University of Alabama at Birmingham