The publication of proposals for new European Union rules on the use of substances of human origin (SoHO) has elicited largely positive responses from industryso far. But it is early days yet. The draft text, released in mid-July, will now enter that curious black box that is the EUs legislative machinery, and exactly when and how it emerges is, at the moment, anyones guess. What can be said at this stage is that there is wide relief right across the healthcare community in Europe that these proposals allow the chance of at last dragging the current legal frameworkcreated in the hugely different scientific and technological circumstances of 20 years agokicking and screaming into the second quarter of the 21st century (which it will be by the time any new rule proposed comes into force).

Expectations are highand diverse. The scope of the products covered by the legislation is wide, from blood transfusions to plasma collection, from IVF to plasma-derived medicines (PDMPs) and to transplants of bone marrow or stem cells or corneas, and extending as far as breast milk and fecal microbiota. The proposed new rules imply big changes for companies working on advanced therapy medicinal products (ATMPs) and on PDMPsand are accordingly provoking anxieties as well as hopes, not least because an EU-wide authorization procedure is foreseen, with upfront risk assessment and clinical outcome data collection requirements. Even if the proposals insist these requirements will be proportionate to the identified risks, these are the sort of suggestions that cause company bosses to lose sleep over yet another round of new regulation.

EuropaBio, representing many European biotech companies, welcomed the proposal as an avenue that can support the growth of ATMPs and allow Europe to ensure it reclaims its title as a global leader in ATMP innovation. According to Claire Skentelbery, its director general, establishing a predictable, future-proof, and fair SoHO framework is critical for the sector, as requirements for donation, procurement, and testing apply to blood, tissues, and cells used in the production of ATMPs.

The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), with many member companies involved in ATMP development, was also glad to see the proposal emerge. But its early reaction highlighted concerns over the risk that the new rules could unhelpfully spill over into regulation of ATMPs, where, for instance, borderline issues frequently emerge when blood cells are used as starting materials for ATMP manufacture. As starting material for ATMPs, maintaining the clear classification between blood, tissues and cells, and ATMPs is crucial to provide clarity and appropriate regulatory standards when developing these transformative treatments, said EUCOPE. And in a clear hint of more reserved position, it added: We will stay active around the new SoHO regulation discussion.

The industry-backed European Alliance for Transformative Therapies (TRANSFORM) says it expects the outcome to reflect exigence of high quality standards, and, like EUCOPE, insists on retaining a clear distinction between advanced therapies and blood, tissues, and cells.The US-based Alliance for Regenerative Medicine greeted the proposal as holding the promise of improving patient safety while establishing greater legal and regulatory certainty for patients and developersagain with an emphasis on maintaining clear regulatory distinctions between starting materials and ATMPs. The EUs ATMP classification has established the region as a global leader in the regulation of cell and gene therapies, it saysurging that determining the classification of borderline cases between SoHOs and ATMPs should be based on the advice of the European Medicines Agency.

Another industry grouping engaged in PDMPs also took a conspicuously conditional stance on the proposals. The Plasma Protein Therapeutics Association said it welcomes positive developments but regrets missed opportunities to support both donors and patients. In particular, it fears not enough will be done to increase supplies of the plasma at the heart of the crucial and often irreplaceable rare disease treatments its members manufacture.

The declared aims of the EUs proposal are to increase the safety and quality of the processes in which these substances are donated and used, and to boost their supply and to ease their availability. The EU says it wants to offer support for innovation. But how far any such support will turn out to be balanced with new obstacles is what is behind the industry caution.

Reflector is Pharmaceutical Executives correspondent in Brussels

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Optimism Abounds On New EU Blood And Tissue Rules - Pharmaceutical Executive

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HAEMOGLOBIN IS a protein in your red blood cells. Your red blood cells carry oxygen throughout your body. If you have a condition that affects your bodys ability to make red blood cells, your haemoglobin levels may drop. Low haemoglobin levels may be a symptom of several conditions, including different kinds of anaemia and cancer.

If a disease or condition affects your bodys ability to produce red blood cells, your haemoglobin levels may drop. When your haemoglobin level is low, it means your body is not getting enough oxygen, making you feel very tired and weak.

Normal haemoglobin levels are different for men and women. For men, a normal level ranges between 14.0 grams per decilitre (gm/dL) and 17.5 gm/dL. For women, a normal level ranges between 12.3 gm/dL and 15.3 gm/dL. A severe low-haemoglobin level for men is 13.5 gm/dL or lower. For women, a severe low haemoglobin level is 12 gm/dL.

Your doctor diagnoses low haemoglobin by taking samples of your blood and measuring the amount of haemoglobin in it. This is a haemoglobin test. They may also analyse different types of haemoglobin in your red blood cells, or haemoglobin electrophoresis.

Several factors affect haemoglobin levels and the following situations may be among them:

Your body produces red blood cells and white blood cells in your bone marrow. Sometimes, conditions and diseases affect your bone marrows ability to produce or support enough red blood cells.

Your body produces enough red blood cells, but the cells are dying faster than your body can replace them.

You are losing blood from injury or illness. You lose iron any time you lose blood. Sometimes, women have low haemoglobin levels when they have their periods. You may also lose blood if you have internal bleeding, such as a bleeding ulcer.

Your body cannot absorb iron, which affects your bodys ability to develop red blood cells.

You are not getting enough essential nutrients like iron and vitamins B12 and B9.

Your bone marrow produces red blood cells. Diseases, conditions and other factors that affect red blood cell production include:

Lymphoma: This is a term for cancers in your lymphatic system. If you have lymphoma cells in your bone marrow, those cells can crowd out red blood cells, reducing the number of red blood cells.

Leukaemia: This is cancer of your blood and bone marrow. Leukaemia cells in your bone marrow can limit the number of red blood cells your bone marrow produces.

Anaemia: There are many kinds of anaemias involving low-haemoglobin levels. For example, if you have aplastic anaemia, the stem cells in your bone marrow dont create enough blood cells. In pernicious anaemia, an autoimmune disorder keeps your body from absorbing vitamin B12. Without enough B12, your body produces fewer red blood cells.

Multiple Myeloma: This causes your body to develop abnormal plasma cells that may displace red blood cells.

Chronic Kidney Disease: Your kidneys dont produce the hormone that signals to your bone marrow to make red blood cells. Chronic kidney disease affects this process.

Antiretroviral medications: These medications treat certain viruses. Sometimes these medications damage your bone marrow, affecting its ability to make enough red blood cells.

Chemotherapy: Chemotherapy may affect bone marrow cells, reducing the number of red blood cells your bone marrow produces.

Doctors treat low haemoglobin by diagnosing the underlying cause. For example, if your haemoglobin levels are low, your healthcare provider may do tests that reveal you have iron-deficiency anaemia. If that is your situation, they will treat your anaemia with supplements. They may recommend that you try to follow an iron-rich diet. In most cases, treating the underlying cause of anaemia will bring the haemoglobin level up.

Many things can cause low haemoglobin, and most of the time you cannot manage low haemoglobin on your own. But eating a vitamin-rich diet can help maintain your red blood cells. Generally, a balanced diet with a focus on important nutrients is the best way to maintain healthy red blood cells and haemoglobin.

keisha.hill@gleanerjm.comSOURCE: Centres for Disease Control and Prevention

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Factors that affect haemoglobin levels and how to detect when it's low - Jamaica Gleaner

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Country

United States of AmericaUS Virgin IslandsUnited States Minor Outlying IslandsCanadaMexico, United Mexican StatesBahamas, Commonwealth of theCuba, Republic ofDominican RepublicHaiti, Republic ofJamaicaAfghanistanAlbania, People's Socialist Republic ofAlgeria, People's Democratic Republic ofAmerican SamoaAndorra, Principality ofAngola, Republic ofAnguillaAntarctica (the territory South of 60 deg S)Antigua and BarbudaArgentina, Argentine RepublicArmeniaArubaAustralia, Commonwealth ofAustria, Republic ofAzerbaijan, Republic ofBahrain, Kingdom ofBangladesh, People's Republic ofBarbadosBelarusBelgium, Kingdom ofBelizeBenin, People's Republic ofBermudaBhutan, Kingdom ofBolivia, Republic ofBosnia and HerzegovinaBotswana, Republic ofBouvet Island (Bouvetoya)Brazil, Federative Republic ofBritish Indian Ocean Territory (Chagos Archipelago)British Virgin IslandsBrunei DarussalamBulgaria, People's Republic ofBurkina FasoBurundi, Republic ofCambodia, Kingdom ofCameroon, United Republic ofCape Verde, Republic ofCayman IslandsCentral African RepublicChad, Republic ofChile, Republic ofChina, People's Republic ofChristmas IslandCocos (Keeling) IslandsColombia, Republic ofComoros, Union of theCongo, Democratic Republic ofCongo, People's Republic ofCook IslandsCosta Rica, Republic ofCote D'Ivoire, Ivory Coast, Republic of theCyprus, Republic ofCzech RepublicDenmark, Kingdom ofDjibouti, Republic ofDominica, Commonwealth ofEcuador, Republic ofEgypt, Arab Republic ofEl Salvador, Republic ofEquatorial Guinea, Republic ofEritreaEstoniaEthiopiaFaeroe IslandsFalkland Islands (Malvinas)Fiji, Republic of the Fiji IslandsFinland, Republic ofFrance, French RepublicFrench GuianaFrench PolynesiaFrench Southern TerritoriesGabon, Gabonese RepublicGambia, Republic of theGeorgiaGermanyGhana, Republic ofGibraltarGreece, Hellenic RepublicGreenlandGrenadaGuadaloupeGuamGuatemala, Republic ofGuinea, RevolutionaryPeople's Rep'c ofGuinea-Bissau, Republic ofGuyana, Republic ofHeard and McDonald IslandsHoly See (Vatican City State)Honduras, Republic ofHong Kong, Special Administrative Region of ChinaHrvatska (Croatia)Hungary, Hungarian People's RepublicIceland, Republic ofIndia, Republic ofIndonesia, Republic ofIran, Islamic Republic ofIraq, Republic ofIrelandIsrael, State ofItaly, Italian RepublicJapanJordan, Hashemite Kingdom ofKazakhstan, Republic ofKenya, Republic ofKiribati, Republic ofKorea, Democratic People's Republic ofKorea, Republic ofKuwait, State ofKyrgyz RepublicLao People's Democratic RepublicLatviaLebanon, Lebanese RepublicLesotho, Kingdom ofLiberia, Republic ofLibyan Arab JamahiriyaLiechtenstein, Principality ofLithuaniaLuxembourg, Grand Duchy ofMacao, Special Administrative Region of ChinaMacedonia, the former Yugoslav Republic ofMadagascar, Republic ofMalawi, Republic ofMalaysiaMaldives, Republic ofMali, Republic ofMalta, Republic ofMarshall IslandsMartiniqueMauritania, Islamic Republic ofMauritiusMayotteMicronesia, Federated States ofMoldova, Republic ofMonaco, Principality ofMongolia, Mongolian People's RepublicMontserratMorocco, Kingdom ofMozambique, People's Republic ofMyanmarNamibiaNauru, Republic ofNepal, Kingdom ofNetherlands AntillesNetherlands, Kingdom of theNew CaledoniaNew ZealandNicaragua, Republic ofNiger, Republic of theNigeria, Federal Republic ofNiue, Republic ofNorfolk IslandNorthern Mariana IslandsNorway, Kingdom ofOman, Sultanate ofPakistan, Islamic Republic ofPalauPalestinian Territory, OccupiedPanama, Republic ofPapua New GuineaParaguay, Republic ofPeru, Republic ofPhilippines, Republic of thePitcairn IslandPoland, Polish People's RepublicPortugal, Portuguese RepublicPuerto RicoQatar, State ofReunionRomania, Socialist Republic ofRussian FederationRwanda, Rwandese RepublicSamoa, Independent State ofSan Marino, Republic ofSao Tome and Principe, Democratic Republic ofSaudi Arabia, Kingdom ofSenegal, Republic ofSerbia and MontenegroSeychelles, Republic ofSierra Leone, Republic ofSingapore, Republic ofSlovakia (Slovak Republic)SloveniaSolomon IslandsSomalia, Somali RepublicSouth Africa, Republic ofSouth Georgia and the South Sandwich IslandsSpain, Spanish StateSri Lanka, Democratic Socialist Republic ofSt. HelenaSt. Kitts and NevisSt. LuciaSt. Pierre and MiquelonSt. Vincent and the GrenadinesSudan, Democratic Republic of theSuriname, Republic ofSvalbard & Jan Mayen IslandsSwaziland, Kingdom ofSweden, Kingdom ofSwitzerland, Swiss ConfederationSyrian Arab RepublicTaiwan, Province of ChinaTajikistanTanzania, United Republic ofThailand, Kingdom ofTimor-Leste, Democratic Republic ofTogo, Togolese RepublicTokelau (Tokelau Islands)Tonga, Kingdom ofTrinidad and Tobago, Republic ofTunisia, Republic ofTurkey, Republic ofTurkmenistanTurks and Caicos IslandsTuvaluUganda, Republic ofUkraineUnited Arab EmiratesUnited Kingdom of Great Britain & N. IrelandUruguay, Eastern Republic ofUzbekistanVanuatuVenezuela, Bolivarian Republic ofViet Nam, Socialist Republic ofWallis and Futuna IslandsWestern SaharaYemenZambia, Republic ofZimbabwe

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BUDDY SCOTT: Love stems from the Father | Brazos Living | thefacts.com - Brazosport Facts

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The media continues the one-handed count of patients that seem to be cured of HIV as a man who has lived with the disease since the 1980s has been in remission for 17 months.

The story is always the samethey seem to be cured, and they get a cool nicknamein this case the City of Hope Patient, after Duarte, California, where he was treated.

The difference in this case was the treatmenta bone marrow transplant to treat blood cancer leukemia from a donor who was naturally resistant to the virus.

The most remarkable difference however, is that he is only patient cured of HIV by coincidence.

The man had developed leukemia, and took the bone marrow transplant for that reason. As it happened, the donor was resistant to HIV, and taught the mans body to create an immune response against the virus.

RELATED: Worlds Second Person Cured of HIV: 40-Year-old Man is Confirmed to Be 30 Months Virus-Free

This is also the first one who got it during the epidemic of HIV/AIDS that took so many lives.

When I was diagnosed with HIV in 1988, like many others, I thought it was a death sentence, said the City of Hope Patient. I never thought I would live to see the day that I no longer have HIV.

SIMILAR: Two Patients Make History After Essentially Being Cured of HIV Using Stem Cell Transplant

So far, only three people have been seemingly cured of human immunodeficiency virus (HIV) which weakens the bodys immune system and leads to the more severe AIDS (autoimmune deficiency syndrome) which can be lethal.

The man no longer takes antiretroviral drugs, the only treatment for HIV. A bone marrow transplant is not a likely future cure, do to it being a tricky and side-effectual procedure.

Nevertheless, all cure cases have been those where a patient is given a transplant of some kind, mostly stem cells, that contain the very rarely occurring natural immunity to the virus.

The case was reported at the AIDS 2022 conference in Montreal, Canada.

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Fourth Patient Seemingly Cured of HIV Through Wild Coincidence - Good News Network

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BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, announces dosing of the first patient in a company-sponsored Phase 1/2 study evaluating a cryopreserved, readily available formulation of GDA-201 for the treatment of follicular and diffuse large B cell lymphomas (NCT05296525).

We are excited to further advance the development of GDA-201, a NAM-enabled natural killer (NK) cell therapy candidate which we believe has the potential to be a new readily available, cryopreserved treatment option for cancer patients with relapsed/refractory lymphoma, said Ronit Simantov, M.D., chief medical and scientific officer of Gamida Cell. Our NK cells elicited an adaptive immune response in patients in the previous investigator-sponsored study with the fresh formulation of GDA-201, potentially leading to durable remissions. We are truly grateful for the contribution of all the participants and clinical collaborators who will allow us to continue studying GDA-201 in this multi-center open label trial.

The Phase 1 portion of the study is a dose escalation phase, designed to evaluate the safety of GDA-201, and will include patients with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma, marginal zone lymphoma or mantle cell lymphoma. The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in 63 patients comprised of two cohorts of patients with either FL or DLBCL. The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments, which may include CAR-T or stem cell transplant.

Interest in NK cell therapies has increased in recent years as a potential alternative to current cell therapies, as NK cells have the potential to be effective in hematological and solid tumors while avoiding common safety issues, said Veronika Bachanova, M.D., Ph.D., University of Minnesota. We are particularly excited about Gamidas cryopreserved formulation of GDA-201, which has potential as a new treatment option for patients.

GDA-201 leverages Gamida Cells proprietary NAM (nicotinamide) technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity (ADCC). In an investigator-sponsored Phase 1/2 study in patients with relapsed or refractory lymphoma, treatment with the fresh formulation of GDA-201 with rituximab demonstrated significant clinical activity. Of the 19 patients with non-Hodgkin lymphoma (NHL), 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%. Two-year data on outcomes and cytokine biomarkers associated with survival data demonstrated a median duration of response of 16 months (range 5-36 months) and an overall survival at two years of 78% (95% CI, 51%91%). In this study, GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 19 patients with NHL and 16 patients with multiple myeloma. The most common Grade 3/4 adverse events were thrombocytopenia, hypertension, neutropenia, febrile neutropenia, and anemia. There were no incidents of cytokine release syndrome, neurotoxic events, graft versus host disease or marrow aplasia.

About NAM Technology

Our NAM-enabled technology, supported by positive Phase 3 data for omidubicel, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM, we can expand and metabolically modulate multiple cell types including stem cells and NK cells with appropriate growth factors to maintain the cells active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About GDA-201

Gamida Cell applied the capabilities of its NAM-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves ADCC and tumor targeting of NK cells. There are approximately 40,000 patients with relapsed/refractory lymphoma in the US and EU, which is the patient population that will be studied in the currently ongoing GDA-201 Phase 1/2 clinical trial.

For more information about GDA-201, please visit https://www.gamida-cell.com. For more information on the Phase 1/2 clinical trial of GDA-201, please visit http://www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapy candidates with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to: the timing of initiation of the expansion portion of the currently ongoing Phase 1/2 clinical trial of GDA-201, as well as the progress of, and data reported from, this clinical trial; the potentially life-saving or curative therapeutic and commercial potential of Gamida Cells product candidates (including omidubicel and GDA-201); and Gamida Cells expectations for the expected clinical development milestones set forth herein. Any statement describing Gamida Cells goals, expectations, or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including statements related to: the impact that the COVID-19 pandemic could have on our business; the scope, progress and expansion of Gamida Cells clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics; and the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Quarterly Report on Form 10-Q, filed with the Securities and Exchange Commission (SEC) on May 12, 2022, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cells forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.

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Gamida Cell Announces Dosing of First Patient in Company-Sponsored Phase 1/2 Study of NK Cell Therapy Candidate GDA-201 - Business Wire

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Forward-Looking Statements

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Forward-looking statements are subject to significant risks and uncertainties,including those discussed in Part I Item 1A, "Risk Factors," of our AnnualReport on Form 10-K for the year ended December 31, 2021, as supplemented by thesection entitled "Risk Factors" in this Quarterly Report on Form 10-Q, thatcould cause actual results to differ materially from those projected in suchforward-looking statements due to various factors, including our ability tocontinue as a going concern; our ability to comply with Nasdaq's continuedlisting requirements; impacts resulting from or relating to the COVID-19pandemic and actions taken to contain it; anticipated use of capital and itseffects; our ability to increase the volume of our product sales; failures bythird-party payers to reimburse for our products and services or changes toreimbursement rates; our ability to continue developing existing technologiesand to develop, protect and promote our proprietary technologies; plans todevelop and perform LDTs; our ability to comply with Food and DrugAdministration ("FDA") regulations that relate to our products and to obtain anyFDA clearance or approval required to develop and commercialize medical devices;our ability to develop and commercialize additional diagnostic products andachieve market acceptance with respect to these products; our ability to competesuccessfully; our ability to obtain any regulatory approval required for ourfuture diagnostic products; or our suppliers' ability to comply with FDArequirements

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for production, marketing and post-market monitoring of our products; ourability to maintain sufficient or acceptable supplies of immunoassay kits fromour suppliers; in the event that we succeed in commercializing our productsoutside the United States, the political, economic and other conditionsaffecting other countries; changes in healthcare policy; our ability to complywith environmental laws; our ability to comply with the additional laws andregulations that apply to us in connection with the operation of ASPiRA LABS;our ability to use our net operating loss carryforwards; our ability to useintellectual property; our ability to successfully defend our proprietarytechnology against third parties; our ability to obtain licenses in the event athird party successfully asserts proprietary rights; the liquidity and tradingvolume of our common stock; the concentration of ownership of our common stock;our ability to retain key employees; our ability to secure additional capital onacceptable terms to execute our business plan; business interruptions; theeffectiveness and availability of our information systems; our ability tointegrate and achieve anticipated results from any acquisitions or strategicalliances; future litigation against us, including infringement of intellectualproperty and product liability exposure; and additional costs that may berequired to make further improvements to our laboratory operations.Company Overview

?Growth. In 2022, we have continued to grow the top line in terms of bothproduct volume and revenue. Our focus has been on OVA1plus, and, in the secondhalf of 2022, we plan to drive OVA1plus sales volume not only through our owncommercial team but also through our collaboration with BioReference Health,LLC, formerly known as BioReference Laboratories, Inc. ("BRL"). We believeAspira GenetiX and Aspira Synergy should also contribute to increased revenue.In addition, positive trends in the tenure of our sales professionals shouldlead to volume growth. As of June 30, 2022, 67% of our sales professionals hadbeen with us for more than three months and 58% had been with us for more thansix months. We aim not only to increase the number of physicians ordering forthe first time but also to increase repeat orders from existing physiciancustomers.

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In parallel to building our OVA platform offering and our commercial deployment,we have been working on several key publications and product extensions.The OVAWatch manuscript, "Analytical Validation of a Deep Neural NetworkAlgorithm for the Detection of Ovarian Cancer," has been published online in theJournal of Clinical Oncology Clinical Cancer Informatics. The Company hasprepared an application for a Proprietary Laboratory Analyses code with the

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American Medical Association for the OVAWatch test to distinguish it fromOVA1plus with an expectation that Novitas and other payers will apply theOVA1plus Centers for Medicare & Medicaid Services fee to OVAWatch, ensuringconsistent coverage and pricing for both OVA products.COVID-19 Pandemic

In December 2019, a novel strain of coronavirus was reported to have surfaced inWuhan, China. The novel coronavirus has since spread to over 100 countries,including every state in the United States. In March 2020, the World HealthOrganization declared COVID-19, the disease caused by the novel coronavirus, apandemic, and the United States declared a national emergency with respect tothe coronavirus outbreak. This outbreak has severely impacted global economicactivity, and many countries and many states in the United States have reactedto the outbreak by instituting quarantines, mandating business and schoolclosures and restricting travel periodically throughout the pandemic. In orderto reduce the impact of limitations on visiting physician offices due toclosures and quarantines, we implemented other mechanisms for reachingphysicians such as virtual sales representative meetings, Key Opinion Leaderpresentations, and increased digital sales and marketing. Patient enrollment forour planned clinical research studies has been slower than originally planneddue to the impact of clinic closures and patients not seeking medical care insome states, which has led to delays in the completion of such studies.Given the potential for future resurgences of COVID-19 cases and the variety offederal and state actions taken to contain them, we are unable to estimate thepotential future impact of the COVID-19 pandemic on our business, results ofoperations or cash flows as of the date of the filing of this Form 10-Q.In addition, as of the date of the filing of this Form 10-Q, we haveapproximately four months of reagents, one of our key testing supplies, instock, depending on volume of tests performed, and we are working with themanufacturer to ensure a consistent supply over the next six months. Aspreviously disclosed, we have put in place staffing and reagent contingencyplans to ensure there is no down time at our lab. We believe the lab couldcontinue to operate in the event any isolated infection were to impact a portionof the workforce. The full impact of the COVID-19 pandemic continues to evolveas of the date of the filing of this Form 10-QCritical Accounting Policies and Estimates

Our product revenue is generated by performing diagnostic services using ourOVA1, OVERA, OVA1plus or Aspira GenetiX tests, and the service is completed uponthe delivery of the test result to the prescribing physician. The entiretransaction price is allocated to the single performance obligation contained ina contract with a patient. Under ASC Topic 606, Revenue from Contracts withCustomers, all revenue is recognized upon completion of the OVA1, OVERA,OVA1plus or Aspira GenetiX test and delivery of test results to the physicianbased on estimates of amounts that will ultimately be realized. In determiningthe amount of revenue to be recognized for a delivered test result, we considerfactors such as payment history and amount, payer coverage, whether there is areimbursement contract between the payer and us, and any developments or changesthat could impact reimbursement. These estimates require significant judgment bymanagement. For OVA1, OVERA, OVA1plus and Aspira GenetiX tests, we also reviewour patient account population and determine an appropriate distribution ofpatient accounts by payer (i.e., Medicare, patient pay, other third-party payer,etc.) into portfolios with similar collection experience. When evaluated forcollectability, this results in a materially consistent revenue amount for suchportfolios as if each patient account were evaluated on an individual contractbasis.

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Results of Operations - Three Months Ended June 30, 2022 Compared to ThreeMonths Ended June 30, 2021The selected summary financial and operating data of the Company for the threemonths ended June 30, 2022 and 2021 were as follows:

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Results of Operations - Six Months Ended June 30, 2022 Compared to Six MonthsEnded June 30, 2021The selected summary financial and operating data of the Company for the sixmonths ended June 30, 2022 and 2021 were as follows:

Product Revenue. Product revenue was $3,853,000 for the six months ended June30, 2022, compared to $3,136,000 for the same period in 2021. Revenue for ASPiRALABS is recognized when the OVA1, OVERA, or OVA1plus test is completed based onestimates of what we expect to ultimately realize. The 23% product revenueincrease is primarily due to an increase in OVA1 test volume compared to theprior year, partially offset by a modest decrease in AUP, which decreased from$377 in the first half of 2021 to $376 in the first half of 2022.Medicaid represents approximately 11.8% of volume in the six months ended June30, 2022, at an AUP of $90. This is compared to 11.7% of volume in the firsthalf of 2021, at an AUP of $90. Our OVA1plus AUP without Medicaid was $415 forthe six months ended June 30, 2022, compared to $417 for the same period in2021.The number of Product tests performed increased 23% to 10,257 during the sixmonths ended June 30, 2022, compared to 8,328 Product tests for the same periodin 2021. This increase was due to increased access to provider offices andincreased investment in our current commercial channel.Genetics Revenue. Genetics revenue was $106,000 for the six months ended June30, 2022, compared to $159,000 for the same period in 2021. Revenue for AspiraGenetiX is recognized when the Aspira GenetiX test is completed based onestimates of what we expect to ultimately realize. The 33% genetics revenuedecrease is primarily due to decreased volumes as compared to the same period in2021, in addition to the AUP decreasing to $424 from $483 from the same periodin 2021.

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Liquidity and Capital ResourcesWe plan to continue to expend resources selling and marketing OVA1, OVERA,OVA1plus and Aspira GenetiX and developing additional diagnostic tests andservice capabilities. We plan to launch our next generation ovarian cancer riskassessment test, OVAWatch, in the second half of 2022.We have incurred significant net losses and negative cash flows from operationssince inception, and as a result have an accumulated deficit of approximately$489,239,000 as of June 30, 2022. We also expect to incur a net loss andnegative cash flows from operations for 2022. Working capital levels may not besufficient to fund operations as currently planned through the next twelvemonths, absent a significant increase in revenue over historicrevenue or additional financing. Given the above conditions, there issubstantial doubt about our ability to continue as a going concern.We expect to raise capital through sources that may include public or privateequity offerings, debt financings, collaborations, licensing arrangements,grants and government funding and strategic alliances. However, additionalfunding may not be available when needed or on terms acceptable to us. If we areunable to obtain additional capital, we may not be able to continue sales andmarketing, research and development, or other operations on the scope or scaleof current activity, and that could have a material adverse effect on ourbusiness, results of operations and financial condition.As discussed in Note 2 to the condensed consolidated financial statements, inMarch 2016, we entered into a loan agreement (as amended on March 7, 2018 andApril 3, 2020, the "DECD Loan Agreement") with the State of ConnecticutDepartment of Economic and Community Development (the "DECD"), pursuant to whichwe may borrow up to $4,000,000 from the DECD.

As discussed in Note 3 to the condensed consolidated financial statements, onFebruary 8, 2021, the Company completed a public offering (the "2021 Offering")resulting in net proceeds of approximately $47,720,000, after deductingunderwriting discounts and offering expenses. There was a change in estimate inthe third quarter of 2021 in the amount of $138,000 relating to an expensereversal of offering costs.

In connection with a private placement offering of common stock and warrants wecompleted in May 2013, we entered into a stockholders agreement which, amongother things, gives two of the primary investors in that offering the right toparticipate in any future equity offerings by the Company on the same price andterms as

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Off-Balance Sheet ArrangementsAs of June 30, 2022, we had no off-balance sheet arrangements that arereasonably likely to have a current or future material effect on our condensedconsolidated financial condition, results of operations, liquidity, capitalexpenditures or capital resources.

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Although once ridiculed, Lovelock's theory that the earth's natural cycles are living, self-regulating organisms underpins much of climate science

Although most of the world knew James Lovelock as an independent scientist and originator of the Gaia hypothesis, he had a slightly different take. Im not a scientist really. Im an inventor or a mechanic. Its a different thing. The Gaia theory is just engineering written very large indeed, hetold theGuardianin 2020.

Regardless of labels, theres no denying the significant influence of Lovelock, whodied July 26on his 103rdbirthday. Although many of his discoveries and ideas on subjects ranging from cryonics to chlorofluorocarbons, and climate to nuclear power were controversial, most gained acceptance as the world caught up.

Named for the Greek Earth goddess, hisGaia theory developed with evolutionary biologist Lynn Margulis during the 1960s when he was working for NASAs moon and Mars programs saw the world with its natural cycles as a living, self-regulating organism. When one cycle is knocked out of equilibrium, others work to restore balance.

At the time, many prominent scientists ridiculed the hypothesis, but its continued to gain acceptance because it helps to explain the chemical and physical balances in air, land and water that make life possible. It underpins much of climate science. The idea isnt that Earth is conscious of these processes; just that the cycles work together to keep the planet healthy and able to support life.

Its similar to the ways in which many Indigenous Peoples worldwide view the living Earth. Everything is interconnected. He understood that human activities that destroy rainforests and reduce biodiversity, for example, hinder Gaias ability to minimize the impacts of runaway greenhouse gases in the atmosphere.

Lovelock wasnt afraid to change his views in the face of evolving evidence, but he also refused to ever soften his message, something I learned from interviewing him several times.

His research revealed the effects of CFCs on the ozone layer, and he warned that burning fossil fuels was changing the climate before these issues were on most peoples radar. His electron capture device, invented in the late 1960s, detected rising CFC levels in the atmosphere as well as pollutants like PCBs in air, soil and water and led to the discovery that this was causing ozone depletion. That eventually resulted in theMontreal Protocol on Substances that Deplete the Ozone Layer, adopted in 1987 by all countries helping the ozone layer to recover and preventing millions of cases of skin and other cancers and eye cataracts.

Like many who clearly see the environmental predicaments weve created, Lovelock wasnt always optimistic, despite his knowledge of the many available and emerging solutions. I would say the biosphere and I are both in the last 1% or our lives, he told theGuardiantwo years ago.

Lovelock, who started out in medicine, even thought pandemics such as COVID-19 could be related to planetary self-regulation: I could easily make you a model and demonstrate that as the human population on the planet grew larger and larger, the probability of a virus evolving that would cut back the population is quite marked.

He said opposition to the Gaia hypothesis surprised him: Im wondering to what extent you can put that down to the coal and oil industries who fought against any kind of message that would be bad for them.

As for solutions to the climate crisis, he advocated for technologies that havent always been popular, including nuclear energy and Edward Tellers suggestion of a sunshade in a heliocentric orbit that would diffuse a few percent of sunlight from the Earth. However, he cautioned, I dont think we should start messing about with the Gaia system until we know a hell of a lot more about it. It is beginning to look as if renewable energy wind and solar if properly used, may be the answer to the energy problems of humanity.

James Lovelock continued to work, write and speak until his final days. My main reason for not relaxing into contented retirement is that like most of you I am deeply concerned about the probability of massively harmful climate change and the need to do something about it now, he said.

Lovelock may have left Gaia, but the knowledge he left endures and is essential to understanding our place, predicament and future.

David Suzuki is a scientist, broadcaster, author and co-founder of the David Suzuki Foundation. Written with contributions from David Suzuki Foundation Senior Writer and Editor Ian Hanington.

@nowtoronto

David Suzuki

David Suzuki is a scientist, broadcaster, author and cofounder of the David Suzuki Foundation.

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David Suzuki: Gaia theorist James Lovelock was always ahead of the times - NOW Toronto

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Immediate cord clamping (ICC), within a few seconds after birth, became routine in the latter half of the 20th century, as part of a tranche of medical birth-related interventions that collectively, undoubtedly improved maternal and neonatal survival and outcomes [1]. The trend to ICC (within 15-20 seconds after birth) was partly driven by some early studies suggesting that the most benefit in terms of blood volume is achieved within this time frame [2], and that deferred cord clamping (DCC) increased rates of polycythemia and jaundice [1]. It may also have been partly driven by increased rates of operative deliveries and consequent pressure to minimize surgical times, as well as the increased availability and effectiveness of neonatal resuscitation. Furthermore, ICC was proposed as a means to reduce the risk of maternal exposure to fetal blood group antigens at a time (before RhD immunoprophylaxis) when hemolytic disease of the fetus and newborn was far more common than it is now.

Formal evidence that ICC was beneficial was never sought, and recent research summarized in systematic reviews [3-6] has suggested that it may be harmful when compared with DCC for various intervals from 30 seconds until when the cord stops pulsating (defined in some studies as physiological cord clamping). ICC before the onset of breathing exposes the newborn baby to a period of significantly restricted cardiac function, whereas DCC until after the onset of breathing (which often does not occur until late in the first minute after birth) may mean that the expanding pulmonary circulation is able to fill with blood from the placenta, rather than by reverse flow across the ductus arteriosus [7]. This may improve left ventricular preload and stabilize pressures and flows in major vessels [7].

In addition, when cord clamping is deferred, babies may receive a transfusion of blood from the umbilical cord and placenta. A recent systematic review demonstrated that DCC in preterm babies improves peak hematocrit in the first week by 2.7% (95% confidence intervals (CI) 1.88-3.52) and reduced the proportion of babies receiving any subsequent blood transfusion (RD: -0.07, 95%CI -0.11 to -0.04) [6]. Some studies have found a weight increase in the first two minutes after birth when the cord is not clamped, supporting the hypothesis of placental transfusion [8]. Yet, recent evidence shows that placental transfusion may not always occur (Conference abstract: Vijayaselvi R, Abraham A, Kumar M, Kuruvilla A, Mathews J, Duley L. Measuring Umbilical Flow and Placental Transfusion for Preterm Births: Weighing Babies at 33-36 Weeks Gestation with Cord Intact. 1st Congress of Joint European Neonatal Societies; 2015).

The relative roles of cardiovascular stabilization at birth versus placental transfusion in improving outcomes have not been established. Understanding the contributions of these two mechanisms has significant implications for research and practice: for example, if the size of placental transfusion is more important, then prescribing a top-up transfusion soon after birth for babies with lower than average hemoglobin (who are known to be at higher risk of various adverse outcomes) [9] may be justified, especially for the babies for whom DCC has been precluded by maternal or fetal conditions. These include significant maternal bleeding, and monochorionic twins, where deferred cord clamping in the first twin could lead to one twin losing blood to the other. However, if it is the effects on improving cardiovascular stability in the first minutes (with consequential benefits for cardiorespiratory function and reducing severity of illness during the subsequent neonatal intensive care unit (NICU) stay), regardless of the magnitude of transfusion, then early top-up transfusion is unlikely to be helpful.

Observational studies suggest that exposure to blood transfusion itself is harmful to preterm babies, increasing the risk of adverse outcomes [10]. However, this suggestion has not been supported by the small number (to date) of randomized controlled trials of blood (red cell) transfusion thresholds [11-14]. It is unlikely to be the means by which DCC reduced deaths in the largest trial to date of deferred cord clamping in preterm babies, the Australian Placental Transfusion Study (APTS), and in the most recent systematic review on this, because neither showed a difference in rates of other adverse outcomes [6,15].

Another possibility is that it is the umbilical cord blood stem cells received by the baby are the main reason for the observed benefits to both survival and reduced requirement for later blood transfusion [16]. Umbilical cord blood has been demonstrated to be such a good contributor to hematopoiesis that it is a recognized stem cell resource for pediatric and adult hematopoietic stem cell transplant [17]. In addition, umbilical cord blood is a potential regenerative and immunomodulatory agent for a variety of clinical conditions [18], so in this case, the extent of placental transfusion would be critical to the improvement of outcomes, and transfusion with adult red cells would not suffice. There are no established methods to quantify the contribution of umbilical cord stem cells to placental transfusion. However, a larger volume of placental transfusion results in the baby receiving more nucleated cells [19], including more umbilical cord stem cells.

Discerning whether these effects (initial enhanced cardiovascular stability leading to early and sustained reduction in severity of illness or volume of placental transfusion) appear to be the main driver of improved outcomes is likely to contribute to practice change, as well as to informing the design of future research studies into methods to improve outcomes of high-risk newborn babies and reduce their transfusion dependence.

The causal mechanisms of reduced transfusion requirements found in DCC relative to ICC are yet to be resolved. The aim of the study is to address the question; In preterm infants (P) does DCC (I) compared to ICC (C) reduce dependence on red cell transfusion via enhanced cardiovascular stability (mediator 1, M1) or via an increased volume of placental transfusion (M2).

The study is a nested retrospective study, called the Transfusions in the APTS Newborns Study (TITANS) (study registration: ACTRN12620000195954), of the cohort of babies who were enrolled and randomly assigned to ICC or DCC in the Australian and New Zealand (NZ) sites for APTS (study registration: ACTRN12610000633088). This design has been developed to take advantage of the comprehensive dataset already collected for APTS, and because there is currently no suitable prospective study that could address the same research questions in such a large group of participants.

Babies had been considered eligible for APTS if obstetricians or maternal-fetal medicine specialists anticipated that delivery would occur before 30 weeks of gestation. Exclusion criteria included fetal hemolytic disease, hydrops fetalis, twin-twin transfusion, genetic syndromes, and potentially lethal malformations. Further details are available in the original APTS publication [15]. In the present TITANS analysis, we will also exclude any baby with a diagnosis of hemolytic anemia or aplastic/hypoplastic anemia.

There were 1401 babies enrolled for APTS from the 13 Australian and 5 NZ hospital sites [15]. APTS data was provided to the TITANS team on 31 July, 2020. It is planned to collect additional data from Australian and NZ APTS sites using a customised, secure web-based database application (REDCap) [20], which is maintained by the University of Sydney, Sydney, Australia. Data will be obtained from source documents (patient hospital records and laboratory reports) using the electronic data collection application from each study site. The individual participant data collected will correspond to the minimum data required to answer the research questions. Baby identification (ID) and other babies details from APTS will be used to re-identify participants and link them to hospital records. Identified data will be collected, in order to allow linkage between the data newly collected from patient records and hospital laboratories and the existing APTS dataset. The data will be checked with respect to range, internal consistency, consistency with published reports and missing items. After data cleaning and analysis, data will be stored in re-identifiable form, with each participants data being identified with the same study numbering system as used for the APTS study.

We will combine the data already extracted, stored and cleaned from APTS with the additional data obtained from study sites for each participating baby, to determine which factors are most influential in reducing transfusion requirements. The specific objectives are, after adjustment for prior risk factors (listed below), to determine:

1.Whether the effect of the intervention (cord clamping) on the outcome (blood transfusions) is mediated by placental transfusion (measured by hematocrit (Hct)) as seen in Figure 1 (a, c) following the causal path X M1 Y, where X is the intervention, ICC or DCC, Y is the outcome, mediator M1 is placental transfusion, and M2 is initial severity of illness stability

2.Whether the effect of the intervention (cord clamping) on the outcome (blood transfusions) is mediated by initial severity of illness (respiratory support, sampling line yes/no and total duration number, blood pressure, cumulative blood sample volume) as seen in Figure 1 (b, c) following the causal path X M2 Y

3.Whether the effect of cord clamping intervention on the outcome (blood transfusions) is driven by multiple mediators (placental transfusion and initial severity of illness) as seen in Figure 1 (c)

4.Whether cording clamping intervention (ICC or DCC) has a direct effect on the outcome after accounting for the mediators as seen in all panels of Figure 1: X Y.

The protocol was approved by the Northern Sydney Local Health District Human Research Ethics Committee in November 2019 (Version 3.0, Reference 2019/ETH12819), the Mater Misericordiae Ltd Human Research Ethics Committee (Version 1.0, Reference HREC/MML/56247), the Mercy Health Human Research Ethics Committee (Version 2.0, Reference 2020-078), and the Southern Health and Disability Ethics Committee (Version 1.0, Reference 19/STH/195). The ethics committees have granted a waiver of consent. The study is conducted in accordance with the National Health and Medical Research Council Statement on Ethical Conduct in Research Involving Humans.

Intervention

The intervention consisted of either immediate or delayed cord clamping (as assigned in APTS). Immediate clamping was defined as clamping the cord within 10 seconds of delivery. Delayed clamping was defined as clamping the cord at least 60 seconds after delivery, with the infant held as low as possible, below the introitus or placenta, and with no palpation of the cord. Variations in the protocol were allowed if they would aid the mother, baby, or both. If the baby was non-vigorous (heart rate <100 beats per minute, low muscle tone, or lack of breathing, or crying), clinicians were allowed to break protocol using their discretion. Cord milking was not part of the protocol for either intervention. Further details may be sourced from the original APTS publication [15].

Outcomes

The primary outcome is the proportion of babies receiving red cell transfusion (for restoration of hemoglobin or blood volume). The secondary outcomes are number of transfusions per baby, cumulative transfusion volume (mL/kg) per baby, and primary reasons for each transfusion.

Putative Mediators

M1: Indicators of placental transfusion to be assessed will be hematocrit (on admission, highest on the first day, highest in the first week collected before any postnatal transfusion).

M2: Indicators of initial severity of illness to be assessed will be cumulative blood sample volume collected throughout hospital stay (number of blood tests multiplied by hospitals usual sample volume for each type of test), sampling line (umbilical arterial line or peripheral arterial line) - yes/no and total duration, mechanical ventilation or inspired O2, and blood pressure.

Sensitivity Analyses (For the Primary Outcome Analysis Only)

Sensitivity analyses will adjust for the following variables: gender, birth <27 weeks vs. 27 weeks, method of delivery (vaginal versus cesarean), intraventricular hemorrhage (IVH) (yes/no and grade III/IV yes/no), surgery for patent ductus arteriosus (PDA), necrotizing enterocolitis (NEC), and sodium in the first 24 hours of life. We will also test model assumptions relating to sequential ignorability and post-randomization confounding (discussed further in the data analysis plan).

Potential Confounders (Covariates)

The following covariates may be used for adjustment in the analysis: gestational age at randomization before birth and any oral iron supplement pre-transfusion.

Timing of Assessments

Putative mediating variables will only be analyzed if they have been measured before the outcome and will be excluded if there is not adequate time and date information available. If the multiple mediator model is applied, careful consideration of timing information will be evaluated. If there is insufficient empirical information to conclude the causal ordering of mediators (M1 causes M2), we will adjust our analytic approach (as discussed in the analysis plan) and discuss any limitations.

Data Analysis Plan

The analysis will include all babies who were initially randomized in the APTS trial for whom we were able to obtain the relevant data and be based on intention-to-treat. All statistical analyses will be conducted in R version 4.1.3 (2022-03-10; R Foundation for Statistical Computing, Vienna, Austria). Descriptive characteristics for continuous data will be presented as means or medians, as appropriate, and categorical data will be presented as frequencies and percentages.

A model-based inference approach will be applied to estimate the average causal mediation effect (ACME), average direct effect (ADE), and the average total effect as recommended [23-25]. This approach will be applied with the R mediation package [26]. We will initially fit two models, one model with mediation as the dependent variable and intervention as the independent variable (mediator model), and a second model with the outcome as the dependent variable, and both mediation and intervention as independent variables (outcome model). To account for the clustering of multiples, estimates will be calculated with generalized estimating equations with a compound symmetric correlation structure to account for within subject correlations. Depending on the outcome (binary, count, skew) these will be modelled with the appropriate family and link functions.

A counterfactual framework will be applied to the mediator and outcome models to simulate the values of the mediator and outcome to estimate the potential values of the mediator. This process is used to estimate the ACME, ADE, and average total effects; 95%CI will be estimated with 1000 bootstrap simulations.

We will apply single mediator models on both placental transfusion variables and initial severity of illness variables if mediators are statistically independent, as seen in Table 1. Independence will be tested using linear regression and any appropriate link functions. If both mediators are not statistically independent, we will investigate the possibility of multiple mediator models, which require an expanded framework for analysis [21]. Here we assume that initial severity of illness is causally related to placental transfusion. For this process, we will use the method developed by Imai and Yamamoto [21] to estimate the ACME and ADE. Following this, 95%CI will be estimated with 1000 bootstrap simulations. If theoretical and empirical timing data and sensitivity analyses suggest that M1 and M2 have non-causal correlation and may be affected by an unmeasured latent mediator, we will adjust our approach to estimate interventional direct and path-specific indirect effects [27,28].

Sensitivity analyses have been limited to a set of biologically plausible and clinically meaningful groups that will be explored by including them for adjustment with covariates, and with the introduction of interaction terms if appropriate. Missing data will be described, reasons for missing data will be explored, and the impact of missing data on conclusions about the treatment effect on the primary outcome will also be explored where possible (e.g., using sensitivity analyses and multiple imputation techniques).

Methodological Assumptions

The causal mediation approach assumes sequential ignorability: that the treatment effect on the outcome is not confounding and that the mediator effect on the outcome is not confounded. As treatment was randomly allocated to neonates, we will assume that the treatment-mediator relationship is not confounded. However, the mediator itself has not been randomized. Thus, unknown confounders may be driving a spurious effect in the mediator-outcome relationship. We will employ additional sensitivity analyses to estimate whether any mediation effects are sensitive to the violation of the assumption of sequential ignorability. To test the possibility of unmeasured confounders we will examine the correlation between residuals in the mediator model and the outcome model. If there is no correlation this would suggest there is no unmeasured confounding, if there is correlation between the residuals, an unmeasured mediator may be affecting both the measured mediator and the outcome. We will apply the method developed by Imai et al. andTingley et al. [23,26] that uses sensitivity analyses to evaluate if the ACME estimate is sensitive to unmeasured confounding.

Post-randomization confounders are dependent on the treatment allocated, affect both mediator and outcome, and can corrupt the mediation estimate. In the context of the present trial, it is possible that non-adherence to the intervention is a post-randomization confounder. We are analyzing our data based on intention to treat principles; however, a sensitivity analysis based on the actual time of cord clamping to assess the influence of non-adherence with the treatment protocol on our estimates may be performed.

Blood transfusions of neonates have been associated with a number of serious adverse outcomes [29]. Nevertheless, there are few evidence-based methods to reduce transfusion exposure [30]. The APTS study found that DCC was associated with a statistically significant reduced need for red cell transfusions by about 10% compared to ICC [15]. However, the mechanism remains unclear.

The study will, at a minimum, provide further information that should increase clinicians understanding of the pathways by which DCC (or other methods to accomplish placental transfusion) results in beneficial patient outcomes. Since one of the main barriers to implementation is lack of understanding about the mechanisms by which such a simple practice change should have such dramatic effects, this should improve adherence to recommendations to defer cord clamping for most babies, thereby reducing mortality and transfusion incidence.

By elaborating on the mechanisms, it may also provide good evidence for how other routine neonatal intensive care practices and interventions affect likelihood of needing to transfuse. Better understanding of these effects may lead to other testable hypotheses or improvements in other aspects of practice, further reducing transfusion exposure and improving other outcomes.

Potential limitations of the study include the dependence on some routinely collected clinical data, which were not collected at the time by the original study according to predefined research definitions. However, we have no reason to think that potential problems of data quality would have been influenced by study group allocation and so do not anticipate that this will be a source of bias.

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Protocol for a Nested, Retrospective Study of the Australian Placental Transfusion Study Cohort - Cureus

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Market (5th Edition) report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035.

Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, LibmeldyT, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors).

Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain.

In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods.

For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics.

Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation.

Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

3.1. Context and Background

3.2. Evolution of Gene Therapies

3.3. Classification of Gene Therapies

3.3.1. Somatic and Germline Gene Therapies

3.3.2. Ex Vivo and In Vivo Gene Therapies

3.4. Routes of Administration

3.5. Mechanism of Action

3.6. Overview of Gene Editing

3.6.1. Evolution of Genome Editing

3.6.2. Applications of Genome Editing

3.6.3. Available Genome Editing Techniques

3.7. Advantages and Disadvantages of Gene Therapies

3.7.1 Ethical and Social Concerns Related to Gene Therapies

3.7.2. Constraints and Challenges Related to Gene Therapies

3.7.3. Therapy Development Concerns

3.7.4. Manufacturing Concerns

3.7.5. Commercial Viability Concerns

4. GENE DELIVERY VECTORS

4.1. Chapter Overview

4.2. Viral and Non-Viral Methods of Gene Transfer

4.3. Viral Vectors for Genetically Modified Therapies

4.4. Types of Viral Vectors

4.5. Types of Non-Viral Vectors

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

5.1. Chapter Overview

5.2. Regulatory Guidelines in North America

5.3. Regulatory Guidelines in Europe

5.4. Regulatory Guidelines in Asia-Pacific

5.5. Reimbursement Scenario

5.6. Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW

6.1. Chapter Overview

6.2. Gene Therapy Market: Clinical and Commercial Pipeline

6.3. Gene Therapy Market: Early-Stage Pipeline

6.4. Gene Therapy Market: Special Drug Designations

6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)

7. COMPETITIVE LANDSCAPE

7.1. Chapter Overview

7.2. Gene Therapy Market: List of Developers

7.3. Key Players: Analysis by Number of Pipeline Candidates

8. MARKETED GENE THERAPIES

8.1. Chapter Overview

8.2. Gendicine (Shenzhen Sibiono GeneTech)

8.3. Oncorine (Shanghai Sunway Biotech)

8.4. Rexin-G (Epeius Biotechnologies)

8.5. Neovasculgen (Human Stem Cells Institute)

8.6. Imlygic (Amgen)

8.7. Strimvelis (Orchard Therapeutics)

8.8. LuxturnaT (Spark Therapeutics)

8.9. ZolgensmaT (Novartis)

8.10. Collategene (AnGes)

8.11. ZyntelgoT (bluebird bio)

8.12. LibmeldyT (Orchard Therapeutics)

9. KEY COMMERCIALIZATION STRATEGIES

9.1. Chapter Overview

9.2. Successful Drug Launch Strategy: ROOTS Framework

9.3. Successful Drug Launch Strategy: Product Differentiation

9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product

9.5. List of Currently Approved Gene Therapies

9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers

9.6.1. Strategies Adopted Before Therapy Approval

9.6.1.1. Participation in Global Events

9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms

9.6.1.3. Indication Expansion

9.6.2. Strategies Adopted During/Post Therapy Approval

9.6.2.1. Geographical Expansion

9.6.2.2. Participation in Global Events

9.6.2.3. Patience Assistance Programs

9.6.2.4. Awareness through Product Websites

9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms

9.7. Concluding Remarks

10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES

10.1. Chapter Overview

10.2. Lumevoq (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

10.3. OTL-103

10.4. PTC-AADC

10.5. BMN 270

10.6. rAd-IFN/Syn3

10.7. beti-cel

10.8. eli-cel

10.9. lovo-cel

10.10. SRP-9001

10.11. EB-101

10.12. ProstAtak

10.13. D-Fi

Continued here:
Gene Therapy Market Research Report 2022 - ResearchAndMarkets.com - Business Wire

Recommendation and review posted by Bethany Smith

Published: Published Date - 09:21 PM, Wed - 10 August 22

Hyderabad: This concept may seem quite fictional and even futuristic. However, this is what geneticists worldwide through gene therapy are pursuing, while trying to find cure for a wide range of diseases that challenge modern medicine including cancers, heart diseases, diabetes, haemophilia, AIDS, genetic disorders, among others.

Gene therapy involves altering the genes inside the cells of the human body, in order to treat or prevent the disease progression. Essentially, geneticists worldwide are exploring ways to utilise gene therapy to alter genetic composition of cells that are responsible for causing diseases and in the process find a long term cure for diseases. The potential to unlock the cure for a wide range of diseases has become a major driving force for researchers and pharma giants worldwide to focus their energies and resources on gene therapy.

So what exactly is gene and gene therapy?

The Gene Therapy Advisory and Evaluation Committee (GTAEC), which monitors clinical trials across India on gene therapies, defines Gene is the most basic and functional unit of heredity and inheritance and consists of a specific sequence of nucleotides in DNA or RNA located on chromosomes that encodes for specific proteins. The human genome comprises more than 20,000 genes. Gene therapy refers to the process of introduction, removal or change in content of an individuals genetic material with the goal of treating the disease and a possibility of achieving long term cure.

The genetic material that has to be introduced to the diseased cell is done through a vector, whch is usually a virus. Viruses are the preferred vectors or vehicles as they are adaptable and efficient in delivering genetic material, the GTAEC, said.

While worldwide major pharmaceutical companies are developing gene therapies for treatment of single gene defects like haemophilia and muscular dystrophy, the Department of Biotechnology (DBT), Government of India, Tata Memorial Hospital, Mumbai and IIT-Mumbai have collaborated to start clinical trials of gene therapy on cancer in India.

Gene therapy in cancer:

In the last few years, CAR- (Chimeric Antigen Receptor) T therapy, a form of gene therapy has emerged as a breakthrough treatment for cancer, especially for leukemia, lymphoma (cancer of the lymphatic system) and multiple myeloma or the cancer of the plasma cells.

The CAR-T cells are genetically engineered in a laboratory and they bind with the cancer cells and kill them. The therapy is available in a few cancer research centres (on clinical trials basis) in US and cost of treatment ranges anywhere from Rs 3 crore to Rs. 4 crore.

To reduce treatment costs, promote and support development CAR-T cell technology against cancers, for the first time in India, Biotechnology Industry Research Assistance Council (BIRAC), established by DBT, Tata Memorial Hospital and IIT Bombay, have launched clincal trials of CAR-T gene therapy to treat cancers. The CAR-T cells were designed and manufactured at Bioscience and Bioengineering (BSBE) department of IIT Bombay. The gene therapy study on cancers is in early phase clinical trials at Tata Memorial in Mumbai.

Regulation of gene therapy:

Realising the potential of gene therapies in treating complex diseases, the GOI is providing financial and even technical guidance to researchers through ICMR, DBT and DST. To ensure gene therapies are introduced in India and clinical trials for gene therapies are performed in an ethical, scientific and safe manner, the ICMR has also framed National Guidelines for Gene Therapy Product Development and Clinical Trials document.

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Health and Tech: The promise of gene therapy to cure cancers - Telangana Today

Recommendation and review posted by Bethany Smith

DUBLIN, Aug. 10, 2022 /PRNewswire/ -- The "Stem Cell Therapy Global Market Opportunities And Strategies To 2031" report has been added to ResearchAndMarkets.com's offering.

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The global stem cell therapy market reached a value of nearly $4,019.6 million in 2021, having increased at a compound annual growth rate (CAGR) of 70.9% since 2016. The market is expected to grow from $4,019.6 million in 2021 to $10,600.2 million in 2026 at a rate of 21.4%. The market is then expected to grow at a CAGR of 11.4% from 2026 and reach $18,175.4 million in 2031.

Growth in the historic period in the stem cell therapy market resulted from rising prevalence of chronic diseases, a rise in funding from governments and private organizations, rapid growth in emerging markets, an increase in investments in cell and gene therapies, surge in healthcare expenditure, and an increase in pharmaceutical R&D expenditure. The market was restrained by low healthcare access in developing countries, limited reimbursements, and ethical concerns related to the use of embryonic stem cells in the research and development.

Going forward, increasing government support, rapid increase in the aging population, rising research and development spending, and increasing healthcare expenditure will drive market growth. Factors that could hinder the growth of the market in the future include high cost of stem cell therapy, stringent regulations imposed by regulators, and high cost of storage of stem cells.

The stem cell therapy market is segmented by type into allogeneic stem cell therapy and autologous stem cell therapy. The autologous stem cell therapy segment was the largest segment of the stem cell therapy market segmented by type, accounting for 100% of the total in 2021.

The stem cell therapy market is also segmented by cell source into adult stem cells, induced pluripotent stem cells, and embryonic stem cells. The induced pluripotent stem cells was the largest segment of the stem cell therapy market segmented by cell source, accounting for 77.2% of the total in 2021. Going forward, the adult stem cells segment is expected to be the fastest growing segment in the stem cell therapy market segmented by cell source, at a CAGR of 21.7% during 2021-2026.

Story continues

The stem cell therapy market is also segmented by application into musculoskeletal disorders and wounds & injuries, cancer, autoimmune disorders, and others. The cancer segment was the largest segment of the stem cell therapy market segmented by application, accounting for 49.7% of the total in 2021. Going forward, musculoskeletal disorders and wounds & injuries segment is expected to be the fastest growing segment in the stem cell therapy market segmented by application, at a CAGR of 22.1% during 2021-2026.

The stem cell therapy market is also segmented by end-users into hospitals and clinics, research centers, and others. The hospitals and clinics segment was the largest segment of the stem cell therapy market segmented by end-users, accounting for 66.0% of the total in 2021. Going forward, hospitals and clinics segment is expected to be the fastest growing segment in the stem cell therapy market segmented by end-users, at a CAGR of 22.0% during 2021-2026.

Scope:

Markets Covered:

By Type: Allogeneic Stem Cell Therapy; Autologous Stem Cell Therapy

By Cell Source: Adult Stem Cells; Induced Pluripotent Stem Cells; Embryonic Stem Cells

By Application: Musculoskeletal Disorders and Wounds & Injuries; Cancer; Autoimmune Disorders; Others

By End-Users: Hospitals And Clinics; Research Centers; Others

Key Topics Covered:

1. Stem Cell Therapy Market Executive Summary

2. Table of Contents

3. List of Figures

4. List of Tables

5. Report Structure

6. Introduction

7. Stem Cell Therapy Market Characteristics

8. Stem Cell Therapy Trends And Strategies

9. Impact Of Covid-19 On Stem Cell Therapy Market

10. Global Stem Cell Therapy Market Size And Growth

11. Global Stem Cell Therapy Market Segmentation

12. Stem Cell Therapy Market, Regional And Country Analysis

13. Asia-Pacific Stem Cell Therapy Market

14. Western Europe Stem Cell Therapy Market

15. Eastern Europe Stem Cell Therapy Market

16. North America Stem Cell Therapy Market

17. South America Stem Cell Therapy Market

18. Middle East Stem Cell Therapy Market

19. Africa Stem Cell Therapy Market

20. Stem Cell Therapy Global Market Competitive Landscape

21. Stem Cell Therapy Market Pipeline Analysis

22. Key Mergers And Acquisitions In The Stem Cell Therapy Market

23. Stem Cell Therapy Market Opportunities And Strategies

24. Stem Cell Therapy Market, Conclusions And Recommendations

25. Appendix

Companies Mentioned

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Stem Cell Therapy Global Market Report 2022: Rising Research and Development Spending, and Increasing Healthcare Expenditure Present Future...

Recommendation and review posted by Bethany Smith

When coronary arteries are blocked, starving the heart of blood, there are good medications and treatments to deploy, from statins to stents. Not so for heart failure, the leading factor involved in heart disease, the top cause of death worldwide.

Its whats on death certificates, said cardiologist Christine Seidman.

Seidman has long been interested in heart muscle disorders and their genetic drivers. She studies heart failure and other conditions that affect the myocardium the muscular tissue of the heart not the blood vessels where atherosclerosis and heart attacks come from, although their consequences are also felt in the myocardium, including heart failure.

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With her colleagues at Brigham and Womens Hospital and Harvard Medical School, she and a long list of international collaborators have been exploring the genetic underpinnings of heart failure. Based on experiments deploying a new technique called single-nucleus RNA sequencing on samples from heart patients, on Thursday they reported in Science their discovery of how genotypes change the way the heart functions.

Their work raises the possibility that some of the molecular pathways that lead to heart failure could be precisely targeted, in contrast to treating heart failure as a disease with only one final outcome.

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Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting, she said. To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

Seidman talked with STAT about the research, including how snRNAseq solves the smoothie problem, and what it might mean for patients. The conversation has been edited for clarity and brevity.

What happens in heart failure?

The heart becomes misshapen in one of two ways. It either becomes hypertrophied, where the walls of heart muscle become thickened and the volume within the heart is diminished, in what we call hypertrophic cardiomyopathy. Or it becomes dilated, when the volume in the heart is expanded and the walls become stretched. I think of it as an overinflated balloon, and that is called dilated cardiomyopathy.

Hypertrophy and dilatation are known to cause the heart over time to have profoundly diminished functional capacity. And clinically, we call that heart failure, much more commonly arising from dilated cardiomyopathy.

What does it feel like to patients?

When we see patients clinically, theyre short of breath, they have fluid retention. When we look at their hearts, we see that the pump function is diminished. That has led to a hypothesis of heart failure as sort of the end stage of many different disorders, but eventually the heart walks down a final common pathway. Then you need a transplant or a left ventricular assist device, or youre going to die prematurely.

What can be done?

Heart failure is a truly devastating condition, and it can arise early in life, in middle age, and in older people. There is no treatment for it, no cure for it, except cardiac transplantation, of course, which provides a whole host of other problems.

How did you approach this problem?

One of the questions we wanted to answer is, are there signals that we can discern that say there are different pathways and there are molecules that are functioning in those pathways that ultimately converge for failure, but through different strategies of your heart?

We treat every patient with heart failure with diuretics. We give them a series of different medications to reduce the pressure against which the heart has to contract. Im clinically a cardiologist, but molecularly Im a geneticist, so it doesnt make sense. If your house is falling down because the bricks are sticking together or if its falling down because the roof leaks and the water is pooling, you do things differently.

Tell me how you used single-cell RNA sequencing to learn more.

Looking at RNA molecules gives us a snapshot of how much a gene is active or inactive at a particular time point. Until recently, we couldnt do that in the heart because the approach had been to take heart tissue, grind it all up, and look at the RNAs that are up or down. But that gives you what we call a smoothie: Its all the different component cells those strawberries, blueberries, bananas mixed together.

But theres a technology now called single-cell RNA sequencing. And that says, what are the RNAs that are up or down in the cardiomyocytes as compared to the smooth muscle cells, as compared to the fibroblasts, all of which are in the cells? You get a much more precise look at whats changing in a different cell type. And thats the approach that we use, because cardiomyocytes [the cells in the heart that make it contract] are very large. Theyre at least three times bigger than other cells. We cant capture the single cell it literally does not fit through the microfluidic device. And so we sequenced the nuclei, which is where the RNA emanates from.

What did you find?

There were some similarities, but what was remarkable was the degree of differences that we saw in cardiomyocytes, in endothelial cells, in fibroblasts. Theres a signature thats telling us I walked down this pathway as compared to a different one that caused the heart to fail, but through activation or lack of activation of different signals along the way.

And that to me is the excitement, because if we can say that, we can then go back and say, OK, what happens if we were to have tweaked the pathway in this genotype and a different pathway in a different genotype? Thats really what precision therapy could be about, and thats where we aim to get to.

Whats the next step?

It may be that several genotypes will have more similarities as compared to other genotypes. But understanding that, I think, will allow us to test in experimental models, largely in mice, but increasingly in cellular models of disease, in iPS [induced pluripotent stem] cells that we can now begin to use molecular technologies to silence a pathway and see what that does to the cardiomyocytes, or silence the fibroblast molecule and see what that does in that particular genotype.

To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

What might this mean for patients?

If we knew that an intervention would make a difference thats where the experiments are we would intervene when we saw manifestations of disease. So the reason I can tell you with confidence that certain genes cause dilated cardiomyopathy is theres a long time between the onset of that expansion of the ventricle until you develop heart failure. So theres years for us to be able to stop it in its tracks or potentially revert the pathology, if we can do that.

What else can you say?

I would be foolish not to mention the genetic cause of dilated cardiomyopathy. Ultimately, if you know the genetic cause of dilated cardiomyopathy, this is where gene therapy may be the ultimate cure. Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting.

My colleagues have estimated that approximately 1 in 250 to 1 in 500 people may have an important genetic driver of heart muscle disease, cardiomyopathy. Thats a huge number, but not all of them will progress to heart failure, thank goodness. Around the world, there are 23 million people with heart failure. Its what ends up on most peoples death certificate. It is the most common cause of death.

Its a huge, huge burden. And there really is no cure for it except transplantation. We dont have a reparative capacity, so were going to have to know a cause and be able to intervene precisely for that cause.

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New research digs into the genetic drivers of heart failure, with an eye to precision treatments - STAT

Recommendation and review posted by Bethany Smith

New York, Aug. 08, 2022 (GLOBE NEWSWIRE) -- The global intracranial therapeutic delivery market is currently valued at around US$ 1.6 Bn and is anticipated to progress at an impressive CAGR of 7.9% over the 2022-2032 study period.

Cell and gene therapies are at the forefront of innovation in treating severe diseases, such as cancer, as well as rare diseases, accounting for around 12 percent of the pharmaceutical industrys clinical pipeline. However, the growing focus on effective therapy has impacted positive financial grades for cell and gene therapy throughout the clinical and social spectrum; intracranial therapeutic administration has been gaining favor in the biopharma industry.

The progressive development of CRISPR and next-generation sequencing has led to a surge in the interest in gene therapy and cell treatment in the past few years. The manufacturing community for cell and gene therapies, including pharmaceutical companies, contract development and manufacturing organizations (CDMOs), and suppliers of lab supplies and equipment, are looking into ways to strengthen supply chains and address process bottlenecks.

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Sales footprint expansion, which has been gaining more and more traction among key participants, calls for the desired assistance, based on financial approvals and consolidated activities. Additionally, several clinical trials have been carried out in association with research institutes.

Key Takeaways from Market Study

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Rising prevalence of neurological disorders and increasing research activities for the development of regenerative medicine to drive market growth over the coming years, says an analyst of Persistence Market Research.

Market Competition

The therapeutic delivery for intracranial is a highly consolidated market with limited key manufacturers operating in the industry. A majority of market players are focused on offering a limited range of cell, gene, and enzyme replacement therapy used for neurological disorder indications.

To strengthen their position in the global market, key players are focusing on strategic approaches such as mergers and collaborations to improve their production capabilities and expand their portfolios in various clinical and research fields.

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What Does the Report Cover?

Persistence Market Research offers a unique perspective and actionable insights on the intracranial therapeutic delivery market in its latest study, presenting a historical demand assessment of 2017 2021 and projections for 2022 2032.

The research study is based on the therapy (cell-based therapy, gene therapy, and enzyme replacement therapy) and indication (spinal muscular atrophy (SMA), multiple sclerosis, batten disease), and amyotrophic lateral sclerosis, across three key regions of the world considered in the taxonomy.

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Intracranial Therapeutic Delivery Market revenue will climb to US$ 4.2 Bn by the end of 2032 Persistence Market Research - GlobeNewswire

Recommendation and review posted by Bethany Smith

The Alliance for Regenerative Medicine Announces the Appointment of Timothy D. Hunt as Chief Executive Officer

WASHINGTON, D.C. August 10, 2022

The Alliance for Regenerative Medicine (ARM), the leading international advocacy organization representing the cell and gene therapy sector, today formally announced that its Board of Directors has appointed Timothy D. Hunt as the organizations next Chief Executive Officer. Hunt will succeed Janet Lynch Lambert, who announced in April her plan to step down as CEO and who served on the Boards Search Committee. Hunt will start at ARM on September 6.

We are excited to welcome Tim to the ARM team at such a pivotal moment for our sector, said Emile Nuwaysir, Chair of the ARM Board and Search Committee,and President and Chief Executive Officer of Ensoma, an in vivo genomic medicines company. Tims two decades of experience advocating for biotechnology companies, knowledge of the key issues facing the cell and gene therapy field, and expertise in leading teams make him the ideal choice to guide ARM in building the future of medicine. Tim has a deep philosophy of engagement with major stakeholders that will support ARM members and help bring cell and gene therapies into mainstream medical practice.

Hunt was most recently the Chief Culture and Corporate Affairs Officer at Xilio Therapeutics, a biotechnology company developing tumor-selective immuno-oncology therapies for patients with cancer. Prior to that, he was the Chief Corporate Affairs Officer at CRISPR gene-editing pioneer Editas Medicine, where he led the companys global policy and government affairs, bioethics, communications, market development and human resources initiatives. He also served in executive public affairs roles at Cubist Pharmaceuticals and Biogen.

Hunt was an Advisory Group member of the Value-Based Payments for Medical Products consortium at the Duke-Margolis Center for Health Policy. He also has been a member of the Board of Directors of the non-profit organization Life Science Cares and has chaired the Ethics Committee of the American Society of Gene and Cell Therapy (ASGCT). Hunt previously served as a member of ARMs Gene Editing Task Force and on the Biotechnology Innovation Organizations Gene Editing Working Group. He received a B.A. in history and philosophy from Boston College and a J.D. from the Columbus School of Law at the Catholic University of America.

I am honored to succeed Janet as Chief Executive Officer of the Alliance for Regenerative Medicine and for the tremendous opportunity to build upon her legacy of developing ARM into the leading sector advocate and resource for the industry, said Hunt. Cell and gene therapies are already transforming patients lives, and we are on the cusp of even more breakthroughs in both rare and prevalent diseases. Our mission is both urgent and clear: to engage all our major stakeholders to ensure the patients we serve have access to the durable and potentially curative therapies of the present and future.

Tim is an excellent choice to continue to grow and strengthen this amazing organization and help realize the potential of regenerative medicine, said Lambert, whose tenure includes doubling ARMs global membership to 425 members, strengthening the organizations advocacy in the US and Europe, and building the ARM team.

Cell and gene therapies to treat blood cancers, spinal muscular atrophy, and an inherited form of blindness are approved in the US and Europe. 2022 could be a record year for new gene therapy approvals for rare disease, and regulators in the US and Europe could approve the first such therapies for hemophilia and sickle cell disease in late 2022 and 2023. More than 2,400 regenerative medicine clinical trials 60% of which targeted prevalent diseases including diabetes and cardiovascular disease were active globally at the end of 2021. ARM is committed to working with stakeholders to ensure that patients benefit from this rapidly advancing pipeline of transformative therapies.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organisation dedicated to realizing the promise of advanced therapy medicinal products (ATMPs).ARMpromotes legislative, regulatory, reimbursement and manufacturing initiativesin Europe and internationally to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-engineered therapies.Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing ATMPs.In its 12-year history,ARMhas become the global voice of the sector, representing the interests of 425+ members worldwide and 80+ members across 15 Europeancountries, including small and large companies, academic research institutions, major medical centres and patient groups.To learn more aboutARMor to become a member, visithttp://www.alliancerm.org.

Media inquiries

For more information or for media requests, please contact Stephen Majors, Senior Director of Public Affairs for ARM, atsmajors@alliancerm.org.

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The Alliance for Regenerative Medicine Announces the Appointment of Timothy D. Hunt as Chief Executive Officer - GlobeNewswire

Recommendation and review posted by Bethany Smith

COVID-19 came with plenty of mysteries, some of which have been resolved, and many that haven't. One mystery since the beginning of the pandemic was why children seemed much less susceptible to the virus than adults. For that and more research news, continue reading.

Why Kids May Have More Protection Against COVID-19

Generally, children are less at risk of COVID-19 infection than adults - not immune, but less likely to be infected and less likely to have severe disease. But why?

Researchers at theUniversity of Queensland, Australia,suggestthat children's nasal lining, or the nasal epithelium, inhibits infection and replication, particularly of the original Wuhan wild-type strain of SARS-CoV-2 and the Delta variant. However, the Omicron variant and its subvariants do not seem to have the same issue. They published their research inPLOS Biology.

"We have provided the first experimental evidence that the pediatric nasal epithelium may play an important role in reducing the susceptibility of children to SARS-CoV-2," the authors wrote. "The data strongly suggest that the nasal epithelium of children is distinct and that it may afford children some level of protection from ancestral SARS-CoV-2."

They found that the wild-type virus replicated less efficiently and was linked to an increased antiviral response in the nasal epithelial cells of children. The effect was also seen with the Delta variant, but not with the Omicron. They also found that Omicron replicated better in pediatric nasal epithelial cells than the wildtype or Delta, although they caution that this was a small study and may reflect how the virus is evolving.

New Molecules Discovered for Treating Age-Related Diseases

Researchers at theHebrew University of Jerusalemhaveidentifieda group of molecules that help cells repair damaged components. Impaired mitophagy is associated with various aging-associated diseases, such as Alzheimer's and Parkinson's, as well as sarcopenia.

Augmenting mitophagy, where defective mitochondria are removed and replaced by new ones, is an emerging strategy for treating the elderly. The researchers designed and tested a family of compounds related to spermidine, a known mitophagy-promoting agent. A prototype, 1,8-diaminooctane (VL-004), was better than spermidine in its ability to induce mitophagy and protect against oxidative stress.

The researchwas publishedin the journalAutophagy. The researchers founded a biotech company, Vitalunga, that is working to develop the drug.

Unique Subset of Immune Cells Protects Against Stroke

A study from theUniversity of Pittsburgfounda subset of white blood cells, a novel subset of CD8+ regulatory-like T cells (CD8+TRLs), that appear to confer fast-acting and lasting protection against ischemic stroke in mice.

These cells were attracted to the site of ischemic injury by a unique "homing" signal produced by the damaged brain cells. The CD8+TRLs reached the brain 24 hours after the stroke, then released molecules that provide direct neuroprotective effects and knock down inflammation and secondary brain damage.

In the mice studies, the animals who received a transfusion of purified CD8+TRLs did better and recovered faster than the untreated mice over five weeks. The CD8+TRLs appear to act as early responders to alert and recruit defenses after a stroke.

Gene Therapy Shows Promise in ALS

Researchers at theUniversity of California San DiegoSchool of Medicinereportthat gene therapy in animal models of ALS measurably delayed disease onset. Although most ALS cases are of unknown cause, the mouse and rat models were of an inherited form of ALS.

The therapy involved an AAV-vector carrying synapsin-Caveolin-1 cDNA into the spinal cords of the mice. The SynCav1 protected and preserved spinal cord motor neurons and added to the mice's longevity. They speculate that SynCav1 might serve as novel gene therapy for ALS and other forms of CNS disease.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease marked by progressive loss of motor neurons in the brain and spinal cord. It is fatal, and there are no cures.

New Therapeutic Target for Colorectal Tumors

Researchers at theTokyo University of Scienceidentifieda potential new therapeutic target for colorectal tumors.

Inflammatory bowel disease (IBD), which includes Crohn's disease and ulcerative colitis, can often lead to colorectal tumors. Innate immune receptors, such as C-type lectin receptors (CLRs), are responsible for the development of IBD. But they also play a key role in regulating gut microbiota and defending against pathogens.

One of those CLRs is a dendritic cell immunoreceptor (DCIR), responsible for maintaining the homeostasis of the immune and skeletal systems. Working with mice, the researchers found that mice without DCIR had less colitis severity and AOM-DSS-induced colorectal tumor growth. They found that using an antibody called anti-NA2 against asialo-biantennary-N-glycans (NA2), which binds to DCIR, decreased colitis symptoms and prevented colorectal tumor growth.

Two Common Viruses Associated with Alzheimer's Disease

Astudyout ofTufts Universityand theUniversity of Oxfordstrengthened the association between specific viral infections and Alzheimer's disease. Two common viruses can lie dormant in brain cells herpes simplex virus (HSV) and varicella-zoster virus (VZV); the latter is associated with shingles and chickenpox.

Researchers leveraged 3D human tissue culture models to mimic the brain and demonstrated that VZV may activate HSV to lay the groundwork for the early stages of Alzheimer's disease. HSV-1, the primary variant of HSV, when activated, leads to the accumulation of tau and amyloid-beta proteins, which are implicated in Alzheimer's disease. They published their research in theJournal of Alzheimer's Disease.

"Our results suggest one pathway to Alzheimer's disease, caused by a VZV infection which creates inflammatory triggers that awaken HSV in the brain," Dana Cairns, Ph.D., GBS12, a research associate in the Biomedical Engineering Department at Tufts, said. "While we demonstrated a link between VZV and HSV-1 activation, it's possible that other inflammatory events in the brain could also awaken HSV-1 and lead to Alzheimer's Disease."

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According toGrand View Research, the global cell therapy market was valued at $7.8 billion in 2020 and is expected to expand at a compound annual growth rate (CAGR) of 14.5% between 2021 and 2028.

The rising number of clinical studies for cell-based therapies and investments in the industry may have a symbiotic relationship. The industry is seeing a snowballing number of ongoingclinical trialswith funding from governments and private agencies.

Theres an arguably thin line between cell and gene therapy. Cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease, according to theAmerican Society of Gene and Cell Therapy (ASGCT). The cells may originate from the patient (autologous cells) or a donor (allogeneic cells).

Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. Some protocols use both gene therapy and cell therapy.

Companies are using thebuilding blocks of lifeand advanced technologies to improve the treatment of human diseases and disorders such as cancer, providing an alternative to traditionally relied-on drugs and surgical treatments.

Cell therapy companies like Longeveron Inc. LGVN, Biogen Inc. BIIB, Alzamend Neuro Inc. ALZN and Solid Biosciences Inc. SLDB, as a result, have gained attention for their progress in using living cells to treat previously incurable diseases and disorders.

COVID-19 has reportedly causedsignificant disruptionto the cell and gene therapy industry. The pandemic has exacerbated the woes of an industry thats had its fair share of challenges with the supply of materials and the manufacturing and logistics processes.

General investments also slowed for the industry as governments shifted focus to saving lives and reviving economies. But things are starting to pick up now that the pandemic is on a downward trend.

Regulatory bodies like the Food and Drug Administration (FDA) have been urged to be more flexible in their approval timelines to make therapies affordable. Discussions continue around access and ensuring these therapies are affordable, reimbursable and profitable for the biopharmaceutical companies that develop them.

Academic and industry collaborations are expected to continue to expand and grow with noticeable impacts on the approval of products. Partnerships among academia, global pharmaceutical companies and small biotechs are expected to continue to shape the cell and gene therapy industry.

Longeveron, a clinical-stage biotechnology company, is one example of a company in the industry that has seemingly done well even during the pandemic. The company reports developing cellular therapies for investigation in chronic aging-related and certain life-threatening conditions.

The companys lead investigational product is Lomecel-B, a cell-based therapy product, derived from culture-expanded medicinal signaling cells sourced from the bone marrow of young, healthy adult donors.

Longeveron believes using the same cells that promote formation of new blood vessels, enhance cell survival and proliferation, inhibit cell death, and modulate immune system function may result in safe and effective therapies for some of the most difficult disorders associated with aging and some medical disorders.

Longeveron is sponsoring Phase 1 and 2 clinical trials in the following indications: Aging frailty, Alzheimers disease, metabolic syndrome, acute respiratory distress syndrome and hypoplastic left heart syndrome.

The companys mission is to advance Lomecel-B and other cell-based product candidates into pivotal Phase 3 trials to achieve regulatory approvals, subsequent commercialization and broad use by the healthcare community.

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OHSU researchers will receive a grant to helpadvance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos. (OHSU/Christine Torres Hicks)

Scientists at Oregon Health & Science University have received significant philanthropic support to advance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos.

The technique, initially demonstrated in mice, could eventually provide a new avenue for child-bearing among couples unable to produce viable eggs of their own.

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, andShoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. (OHSU/Christine Torres Hicks)

Even though the proof of concept in mice shows promise, significant challenges remain to be resolved before the technique could be ready for clinical trials under strict ethical and scientific oversight. Even then, Congress currently precludes the Food and Drug Administration from providing oversight for clinical trials involving genetic modification of human embryos.

Shoukhrat Mitalipov, Ph.D., (OHSU)

It will take probably a decade before we can say were ready, said Shoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. The science behind it is complex, but we think were on the right path.

This type of research is not funded by the National Institutes of Health, so it depends on philanthropic support. For this project, Open Philanthropy awarded $4 million over three years through the OHSU Foundation.

Paula Amato, M.D. (OHSU)

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, sees the potential for an enormous benefit to families struggling to have children if the technique proves successful.

Age-related decline in fertility remains an intractable problem in our field, especially as women are delaying childbearing, said Amato, who is the principal investigator for the grant award.

The technique holds promise for helping families to have genetically related children, a cohort that includes women unable to produce viable eggs because of age or other causes, including previous treatment for cancer. It also raises the possibility of men in same-sex relationships having children genetically related to both partners.

The skin cell can come from somebody who doesnt have any eggs themselves, Amato said. The biggest implication is for female, age-related infertility. It can also come from women with premature ovarian insufficiency due to cancer treatment or genetic conditions, or from men who would be able to produce a genetically related child with a male partner.

The award from Open Philanthropy will enable OHSU researchers to develop the technique in early human embryos using eggs and sperm from research donors. As with other groundbreaking research at OHSU including a gene-editing discovery that generated worldwide attention in 2017 none of the early embryos will be allowed to develop past the early blastocyst stage.

Researchers will build on a study in mice published this January in the journal Communications Biology.

The study demonstrated that it is possible to produce normal eggs by transplanting skin-cell nuclei into donor eggs from which the nuclei have been removed. Known as somatic cell nuclear transfer, the technique was famously used in 1997 to clone a sheep in Scotland named Dolly. In contrast to a direct clone of one parent, the mouse study published earlier this year required OHSU and collaborating scientists to cut the donor DNA in half and then fertilize the resulting egg with sperm to generate a viable embryo with chromosomes from both parents.

The process involves implanting the skin cell nuclei into a donor egg, and then allowing the egg to discard half its skin cell chromosomes a process similar to meiosis, when cells divide to produce sperm or egg cells. This results in a haploid egg with a single set of chromosomes with precisely half the chromosomes of the diploid skin cell with two sets of chromosomes. At just the right phase of the cell cycle, the new egg is combined with sperm chromosomes through in vitro fertilization.

An embryo then develops with the correct diploid number of chromosomes from each parent.

We had to show in the mouse that this hypothesis works, Mitalipov said. Open Philanthropy saw the implications for fertility with a new way of looking into this. The key is inducing haploidy.

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The new report titled Hemophilia Gene Therapy Market offer by Key Players, Types, Applications, Countries, Market Size, Forecast to 2030offered by Market Research, Inc. includes a comprehensive analysis of the market size, geographical landscape along with the revenue estimation of the industry. In addition, the report also highlights the challenges impeding market growth and expansion strategies employed by leading companies in the Hemophilia Gene Therapy Market.

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia A, B, and C. Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage.

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This market study covers and analyzes the potential of the global Hemophilia Gene Therapy industry, providing geometric information about market dynamics, growth factors, major challenges, PEST analysis and market entry strategy analysis, opportunities and forecasts. One of the major highpoints of the report is to provide companies in the industry with a strategic analysis of the impact of COVID-19 on Hemophilia Gene Therapy market.

Hemophilia Gene Therapy Market: Competition Landscape

The Hemophilia Gene Therapy market report includes information on the product presentations, sustainability and prospects of leading player including: BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo Therapeutics, Inc., and Freeline Therapeutics

Hemophilia Gene Therapy Market: Segmentation

By Type:

By Application:

Hemophilia Gene Therapy Market: Regional Analysis

All the regional segmentation has been studied based on recent and future trends and the market is forecasted throughout the prediction period. The countries covered in the regional analysis of the Global Hemophilia Gene Therapy market report are North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and Latin America.

Years Considered for the Hemophilia Gene Therapy Market Size:

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Market Status and Outlook by Region: In this segment, the report examines the net deals, income, creation and portion of the overall industry, CAGR and market size by locale. The global Hemophilia Gene Therapy Market is profoundly examined based on areas and nations like North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

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Scope of Hormone Replacement Therapy Market

The main objective of thisHormone Replacement TherapyMarket research analysis is to discover the right opportunities, provide the right investment options and evaluate some actions to be taken for business growth. It forecasts the competition level in the market for the period 2022-2030. There are a few crucial factors covered in this market study report such as identification of potential sales, risks involved in the business, and pointing out exact problems in the market. It further goes on to depict how the dangerous virus COVID-19 affected human health and the world economy and how to reduce its effects on society.

Hormone Replacement Therapy market report covers the customary market situation, effective market strategies, innovative breakthroughs, and key developments. It not only focuses on factors that enhance the growth of the market but also focuses on factors that can hinder the market growth. Under the competitive analysis section, It estimates the market growth for the estimation period 2022-2030. This Hormone Replacement Therapy statistical survey report depicts the acquisitions, collaborations, and mergers adopted by the market players to leverage their place and the expansion of their business in the market. Some crucial market tactics provided here help organizations to grow hugely.

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Key Players Mentioned in the Hormone Replacement Therapy Market Research Report:Abbott, Bayer AG, Merck KGaA, Novartis AG, and Pfizer Inc.

Hormone Replacement Therapy Market Segmentation:

Product Type SegmentationEstrogen HormoneGrowth HormoneThyroid HormoneTestosterone Hormone

Application SegmentationMenopauseHypothyroidismGrowth Hormone DeficiencyMale HypogonadismOther Diseases

The global Hormone Replacement Therapy market, based on different geographic regions, is divided as follows:

North America (the United States, Canada, and Mexico)Europe (Germany, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)South America (Brazil, Argentina, Colombia, etc.)The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

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Analysis supports positive lifestyle habits, such as exercising and quitting smoking, investigators say.

Menopause before aged 40 years is associated with elevated risks of atrial fibrillation (AF) and heart failure (HF), according to the results of a study published in the European Heart Journal.

In the study of more than 1.4 million women, results showed that the younger the individual is at menopause, the higher the risk of new-onset HF and AF.

Women with premature menopause should be aware that they may be more likely to develop heart failure or atrial fibrillation than their peers, Ga Eun Nam of Korea University College of Medicine, Seoul, Republic of Korea, said in a statement. This may be good motivation to improve lifestyle habits known to be linked with heart disease, such as quitting smoking and exercising.

Investigators included postmenopausal women who were aged 30 years or older and completed a National Health Interview Survey health check-up in 2009. Investigators followed up with individuals until the end of 2018 for new-onset HF and AF.

Information on demographics, health behaviors, and reproductive factors, including age at menopause and use of hormone replacement therapy, were collected.

Investigators categorized age at menopause as below aged 40, 40 to 44, 45 to 49, and 50 years or older. Premature menopause was defined as the individuals final menstrual period before the aged 40 years.

Approximately 2% of individuals had a history of premature menopause. Of these women, the average age at menopause was 36.7 years. The average age at study enrollment for women with and without a history of premature menopause was 60 and 61.5 years, respectively.

During an average follow up of 9.1 years, 3.2% developed AF and 3% of individuals developed HF.

Investigators analyzed the association between history of premature menopause and incident HF and AF after adjusting for age, smoking, alcohol, physical activity, income, body mass index, and other factors that could have affected the incidence.

They found that women who experienced premature menopause had a 33% higher risk of HF and a 9% higher risk of AF compared with those who did not.

The misconception that heart disease primarily affects men has meant that sex-specific risk factors have been largely ignored. Evidence is accumulating that undergoing menopause before the age of 40 may increase the likelihood of heart disease later in life, Nam said in the statement

Our study indicates that reproductive history should be routinely considered in addition to traditional risk factors such as smoking when evaluating the future likelihood of heart failure and atrial fibrillation, she said.

Investigators also analyzed the association between age at menopause and the incidence of AF and HF after adjusting for the same factors as in the previous analyses.

The risk of HF increased as the age at menopause decreased.

Compared with those aged 50 years and older at menopause, those aged 45 to 49, 40 to 44, and below 40 years had an 11%, 23%, and 39% greater risk of incidence HF, respectively.

Additionally, the risk of AF increased as age decreased with 4%, 10%, and 11% higher, respectively, compared with women aged 50 years and above at menopause.

Several factors could explain the associations, including changes in body fat distribution and a drop in estrogen levels, investigators said.

Reference

Premature menopause is associated with increased risk of heart problems. News release. EurekAlert. August 3, 2022. Accessed August 5, 2022. https://www.eurekalert.org/news-releases/960487

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Study Results Link Menopause Before 40 to Increase in Atrial Fibrillation, Heart Failure - Pharmacy Times

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More than 37 million people are estimated to have chronic kidney disease (CKD), according to the CDCand 9 out of 10 people don't even know they have it. "There are a number of physical signs of kidney disease, but sometimes people attribute them to other conditions," says Dr. Joseph Vassalotti, Chief Medical Officer at the National Kidney Foundation. "Also, those with kidney disease tend not to experience symptoms until the very late stages, when the kidneys are failing or when there are large amounts of protein in the urine. This is one of the reasons why only 10% of people with chronic kidney disease know that they have it." Here are five sure symptoms of kidney disease, according to doctors. Read onand to ensure your health and the health of others, don't miss these Sure Signs You've Already Had COVID.

Unexplained fatigue could be a sign of kidney disease, doctors warn. "In addition to filtering waste, healthy kidneys make a hormone called erythropoietin (EPO), which tells your bone marrow to make red blood cells. If your kidneys aren't working as well as they should, they won't make enough of this important hormone," according to Staci Leisman, MD, kidney specialist at Mount Sinai Hospital in New York City. "As a result, you produce fewer red blood cells, which could lead to a condition called anemia."

Foamy, discolored, bloody urine could be a sign of kidney disease, doctors warn. Changes in urinationfor example, urinating more or less than usualcould also be a red flag. "Even a little blood can change the color of urine dramatically," says nephrologist Juan Calle, MD.6254a4d1642c605c54bf1cab17d50f1e

Nausea and vomiting are often reported as particularly unpleasant symptoms of kidney disease. "The earliest signs are you may get nauseated especially in the morning, or you have an appetite and you smell food and then it just turns your stomach," says nephrologist James Simon, MD. "You are actually nauseated all day, a bitter and metallic taste in your mouth. Those are the earliest signs."

High blood pressure could be a sign of kidney disease, but physicians are unsure how they are connected. "High blood pressure may be a sign of kidney disease, or it may cause it. It's the chicken-or-egg dilemma," says Dr. Calle. "Anyone who has high blood pressure and diabetes needs to be screened for kidney diseases."

Knowing the risk factors for chronic kidney disease is key for prevention. "Primary risk factors include diabetes, high blood pressure, cardiovascular disease, a family history of kidney failure and age over 60," says Leslie Spry, MD, FACP. "Secondary risk factors include obesity, autoimmune diseases, urinary tract infections, systemic infections, and kidney loss, damage, injury or infection. Taking care of overall health helps protect kidney health. Wise practices include exercising regularly, low salt diet, controlling weight, monitoring blood pressure, cholesterol and glucose levels, not smoking, drinking moderately, avoiding non-steroidal anti-inflammatory drugs (NSAIDs) and getting an annual physical."

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Sure Signs You Have Kidney Disease, Say Physicians Eat This Not That - Eat This, Not That

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According to the CDC, 37.3 million people in the U.S. have diabetes (11.3% of the population) and 96 million people aged 18 years or older have prediabetes (38.0% of the adult population).

"As recently as 50 years ago, type 2 diabetes was extremely rare. Now, around the world, the number of people with diabetes is increasing rapidly and is heading towards 643 million by 2030. This is a worldwide epidemic," says Adele Hite, PhD, MPH, RD. "In the past, type 2 diabetes was thought to be a progressive disease with no hope for reversal or remission. People were and sometimes still are taught to 'manage' type 2 diabetes, rather than to try to reverse the underlying process. But now people with type 2 diabetes can hope to regain their health." Here are five habits increasing your prediabetes risk. Read onand to ensure your health and the health of others, don't miss these Sure Signs You've Already Had COVID.

Lack of sleep is strongly linked to both diabetes and prediabetes, doctors warn. "How does poor sleep contribute to obesity, diabetes, and metabolic issues? It's known that lack of sleep raises the stress hormone cortisol and increases glucose production, which raises your blood sugar," says Tony Hampton, MD. "Studies have shown that markers of increased insulin resistance appear after just one night of partial sleep deprivation. Symptoms of prediabetes can arise with as little as five days of sleep disturbances. Blood pressure also increases with sleep deprivation."

Excessive belly fat is particularly dangerous in raising the risk of prediabetes, studies show. "Being overweight is known to increase the risk of type 2 diabetes, but this study shows that not all fat is equal: where any excess fat is stored in the body has a big impact on disease risk," says Professor Mark McCarthy, University of Oxford.

If you're overweight or obese, even minor weight loss can help lower the risk of getting prediabetes, experts say. "If you have prediabetes, losing a small amount of weight if you're overweight and getting regular physical activity can lower your risk for developing type 2 diabetes," the CDC advises. "A small amount of weight loss means around 5% to 7% of your body weight, just 10 to 14 pounds for a 200-pound person."6254a4d1642c605c54bf1cab17d50f1e

Regular exercise is important for lowering the risk of prediabetes. "Even if you don't lose weight, exercise will make you stronger and healthier," says endocrinologist Douglas Zlock, MD, medical director of the diabetes center at John Muir Health. "Healthy habits can definitely postpone the onset of diabetes even if they don't prevent it."

There is evidence that stress can impact blood sugar control, helping raise the risk of prediabetes. "Most people with type 2 diabetes know the importance of exercising regularly, eating a healthy diet, and getting plenty of rest," says Dr. Joshua J. Joseph, endocrinologist at The Ohio State Wexner Medical Center. "But stress relief is a crucial and often forgotten component of diabetes management. Whether it's a yoga class, taking a walk or reading a book, finding ways to lower your stress level is important to everyone's overall health, especially for those with type 2 diabetes."

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At a time when our bodily autonomy is under attack, women have become more vigilant about their basic human rights to equality, information, and the highest attainable standard of healthincluding sexual and reproductive health without discrimination. But the traditional healthcare system tends to treat the complex needs of women all wrong. It takes an acute approach rather than preventative. Disparate versus integrative. As a result, American women are suffering through a national infertility crisis and high maternal mortality (a rate of 23.8 to be exact, the worst among industrialized nations). The Covid-19 pandemic meanwhile has had lasting effects on the state of mental health for women, who are already twice as likely to experience depression and anxiety than men.

In an effort to change this, numerous founders are disrupting the wellness industry as we know it. We spoke with three who were unwilling to wait for the massive ship that is the American healthcare system to course correctone a former investment banker, another a former journalist, and the third a doctor-turned-serial entrepreneur. They recognize that, for women, innovation is a matter of life and death.

Kimberly Seals Allers, founder of Irth

(Image credit: Future)

With her first pregnancy, Kimberly Seals Allers, a former reporter at Fortune and editor at Essence, deployed her journalistic skills to research the best hospitals. But when she gave birth, her experience didnt match the glowing reviews.

I walked [into] a highly rated and best of hospital in New York City, having done months of due diligence and reading reviews, says Allers. But I left feeling disrespected, traumatized, and unseen.

This impacted her earliest memories of motherhood. I blamed myself completely, unaware that people are not being treated the same way even in the same place.

According to a White House statement issued on its first Maternal Health Day of Action in 2021, the U.S. has the highest maternal mortality rate of any wealthy nation. For women of color, the reality is stark. According to the CDC, Black, American Indian, and Alaska Native women are two to three times more likely to die from pregnancy-related complications than white women. The pregnancy-related mortality rate for Black women with at least a college degree was 5.2 times that of their white counterparts.

So in February 2021, Allers launched Irth, an app that seeks to equalize the experience of delivering a child by giving women a central platform for rating and reviewing hospitals, OB-GYNs, and other pre- and post-natal care providers. Think of it like Yelp, but for childbirth, aimed at helping Black and Brown women access the best prenatal, birthing, postpartum, and pediatric services.

It started as a mommy-son project. Her youngest child, Michael, then a 13-year-old coding enthusiast, created the apps wireframes and soon, the duo started going to pitch competitions. I had this idea for an app and thought this would be a great way to bridge our worlds, says Allers. In 2018, they went to an MIT hackathon related to birth and breastfeeding and won the Media for Change award. There, they met an MIT engineer who went on to build Irths prototype, which enabled Allers to get more grant funding. To date, Allers has raised more than $850,000 in grants. I'm really proud of our origin story, she says.

Were turning our anonymized reviews into qualitative data...to give every Black birthing person a five-star experience while bringing life into the worldwhich is what we all deserve.

Earlier this year, she was invited to meet with Tim Cook during Apples Worldwide Developers Conference, where Apple recognizes trailblazing creators uplifting their communities through technology. Apple also featured Irth in a spotlight (opens in new tab) on the App Store to grow awareness. It was an incredible platform to share about Irth and our mission to remove the bias from birth, says Allers.

To date, Irth has more than 20,000 users and reviews from 48 states. Allers is most alarmed by the frequency at which women of color are going through similarly poor experiences, such as hearing negative comments from care providers about family size or marital status. There were too many reviews from fathers saying they were referred to as Mr. Baby Daddy while supporting their partner; Black women being coerced and admonished about birth control while in active labor, says Allers.

Right now, according to Allers, the number-one negative experience being reported on the Irth app nationwide is: "My requests for help were ignored or refused."Such medical racism has resulted in disrespect and death. A common thread in every maternal death story is that too many hospitals want to dismiss or explain away these reports of pain, instead of taking them seriously.

Irth has its sights on reform. The team of just 15 employees recently launched pilot programs with five hospitals to improve patient care for Black mothers. Allers says the hardest work is being done at the community level, encouraging more women of color to submit reviews. After all, it will take sheer volume to sound the alarm bells on one of the biggest failures of the countrys healthcare system.

Were turning our anonymized reviews into qualitative data to teach providers, payers, and hospitals how to give every Black birthing person a five-star experience while bringing life into the worldwhich is what we all deserve.

Rebecca Parekh, ceo and cofounder of The Well.

(Image credit: Future)

Step into The Well's New York City flagship, and one might wonder whether theyve entered a luxurious retreat in Montauk or Malibu. But smack in the middle of Manhattan, visitors to The Well can book meditation classes in a glowing white dome, access body treatments rooted in Traditional Chinese Medicine and Ayurveda, or try their hand at hydrotherapy, aromatherapy, meditation, and more.

Rebecca Parekh, cofounder and CEO, says the very premise of her business is to integrate holistic health into the urbanite's day-to-dayand that meant building a geographically accessible hub to promote such a lifestyle.

Our original business was a physical business and were remaining true to that because were passionate about the in-person experience and offering integrated wellness in big, busy cities, she says. Its not a common mindset in this Web3, Metaverse-obsessed startup world.

Burnout in her previous career led Parekh to rediscover the importance of holistic health. "I was an investment banker and was not modeling a wellness lifestyle; when it comes to getting swept into hustle-culture, I got disconnected," says Parekh, who worked at Deutsche Bank for 10 years. Her mom encouraged her to simply cook more and practice yoga daily. Parekh ultimately left banking, transitioning to work as Executive Director of the Global Foundation for Eating Disorders, and then COO for Deepak Chopra Radical Wellbeing.

She began envisioning a business that would promote health on a grander scale and on a daily basis. In 2016 she teamed up with cofounders Sarrah Hallock and Kane Sarhan to begin fundraising for The Well. It opened its nearly 15,000-square-foot Manhattan flagship, complete with a full-service restaurant and retail shop in September 2019just six months before the pandemic made in-person experiences practically obsolete.

Parekh and cofounders began offering digital wellness classes and services, such as a three-part webinar focused on how to ease the anxiety of returning to office work and the outside world after months in lockdown. It also focused on e-commerce for its line of vitamins, supplements, and personal care goods.

Although The Well nimbly pivoted to digital during the pandemic, its cofounders never abandoned their commitment to physical locations and products. Through a partnership with Auberge Resorts Collection, the company continued to invest in its second location at The Mayflower Inn & Spa in Connecticut, which opened in November 2020, then its third spot, Hacienda AltaGracia, in Costa Rica in 2021.

To date, The Well employs about 100 people and has raised roughly $50 million in venture capital. After surviving Covid-19 closures, the brand is set for expansion: Parekh says The Well will likely open two more locations by the end of the year, and confirmed the company is working on projects in Mexico, Miami, London, and Aspen.

While it grows via brick-and-mortar, the company is staying grounded in an industry driven by fads. Parekh credits The Wells medical and nutrition teams with vetting everything, from services offered to ingredients served in the restaurant. The rigorous standards are set forth by Frank Lipman, M.D., Chief Medical Officer and a renowned voice in integrative and functional medicine. He went from quack to guru, as [mainstream] folks are now believing in what hes been talking about for quite some time, says Parekh.

And in an industrywellnessthat can mean many things and nothing at once, The Well filters out fads by making sure each service fits within the brands key pillars: nutrition, body work, skincare, lifestyle, community, and, a newly added category: emotional wellbeing.

We focus on meeting people where they are at in their wellness journeys, says Parekh. Whether youre sick or not we believe yoga is medicine, along with the food we eat.

Parsley Health founder and CEO, Dr. Robin Berzin.

(Image credit: Future)

Just 13 percent of healthcare CEOs are femaleand even fewer are M.D.s. But Dr. Robin Berzin has defied these odds. In 2011, the graduate of Columbia Universitys College of Physicians and Surgeons cofounded Cureatr, an app that streamlines communication with physicians. That experience with health-tech inspired her to launch Parsley Health in 2016this time as a solo female founder.

Parsley Health is a functional medicine company that takes a preventive approach to chronic disease management. It uses a holistic snapshot of a patients sleep, fitness, nutrition, family history, and mental health to determine the best regimens and health protocols.

When we look at the body as an integrated systemrather than isolated partswere able to implement the right solution at the right time.

It has served tens of thousands of patients nationwide via telehealth, in addition to in-person clinics in New York City and Los Angeles. We have independent data showing that Parsley's approach to care improves health while reducing specialist referrals and prescription drug use, Dr. Berzin says. According to company data, by year two, patients have reduced their referrals to specialists by 77 percent. Those previously on chronic medications see a 65 percent reduction in prescription drug use.

We closely monitor their health to ensure that their concerns were heard, their treatments were working, and, ultimately, that we were measurably putting them on a path to feeling better, says Dr. Berzin. She points to the initial visitwhich takes 75 minutes, five times longer than the average appointment with a primary care physicianas the real changemaker.

Last year was a big one for Dr. Berzin. In response to the wellness wave sparked by Covid-19, she grew telehealth from seven states in 2020 to a nationwide operation in early 2021. She also raised a Series C round of venture capital, bringing the total raised to date to more than $100 million. She finished writing her first book, State Change (opens in new tab).

Last but not least, she welcomed her third child. In August 2021, she posted a beaming Instagram photo of herself just three weeks before giving birth, and wrote I convinced myself at 39 that I was too old to get pregnant easily, even though of course I know better. I help women beat the fertility odds every freaking day at work!

With a female physician and a mother of three at the helm, Parsley Health is a family practice with an emphasis on transforming womens healthcare. Its programs are designed to support women throughout their reproductive lifecycle: pre-conception, fertility, pregnancy, and postpartum. Last month, the company announced the launch of comprehensive menopause care.

Menopause affects more than 55 million American women and yet 73 percent of these women will suffer in silence without treatment. Perimenopause symptoms can start as early as age 45 while menopause can last for a decade. And, too often, according to Darcy McConnell, M.D., Director of Medical Affairs at Parsley Health, older women experience age-related conditions that are misattributed to menopause. For example, bloating and weight gain are often blamed on hormones, but can actually be due to high cortisol levels, leading to incorrect treatments.

Parsley Healths menopause protocol will deviate from the traditional practice of defaulting to hormone replacement therapy (HRT), which, according to Cancer.gov, is linked to numerous side effects including vaginal bleeding, dementia, and breast cancer. Parsley Healths holistic model instead takes a closer look at a womans metabolic health and mental health to offer a comprehensive system for monitoring multiple causes and conditions.

Dr. Berzin says Parsley Health aims to connect the dots, which traditional, patriarchal medicine has failed to do: When we look at the body as an integrated systemrather than isolated partswere able to implement the right solution at the right time, which halts the cycle of frustration women experience when theyre stuck in the revolving door of specialist referrals and prescription drugs.

(Image credit: Kanya Iwana / Brittany Holloway-Brown)

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Excerpt from:
The CEOs Disrupting and Democratizing Women's Health - Marie Claire

Recommendation and review posted by Bethany Smith

Data from the Phase 3 SPIRIT program showed MYFEMBREE reduced menstrual pain and non-menstrual pelvic pain in premenopausal women with endometriosis, and a loss of mean bone mineral density of less than 1% from baseline through one year of treatment Myovant and Pfizer will continue to jointly commercialize MYFEMBREE, with product available immediately Myovant to host conference call and webcast on Monday, August 8, 2022, at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time

BASEL, Switzerland and NEW YORK, [August 5] (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV) and Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has approved MYFEMBREE (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) as a one-pill, once-a-day therapy for the management of moderate to severe pain associated with endometriosis in pre-menopausal women, with a treatment duration of up to 24 months. The approval is supported by one-year efficacy and safety data, including 24-week data from the Phase 3 SPIRIT 1 and SPIRIT 2 trials, which were published in The Lancet, and the first 28 weeks of an open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2. MYFEMBREE also is approved for heavy menstrual bleeding associated with uterine fibroids in pre-menopausal women. Myovant and Pfizer will continue to jointly commercialize MYFEMBREE in the U.S. and product is available immediately.

Endometriosis is a painful, chronic disease with limited therapies to manage symptoms, said Juan Camilo Arjona Ferreira, M.D., Chief Medical Officer of Myovant Sciences, Inc. The new MYFEMBREE indication helps advance our mission to redefine care for women by helping address a disease with high unmet need, giving women and physicians a new meaningful treatment option to manage moderate to severe pain associated with endometriosis.

This approval is an important milestone reflecting Pfizer and Myovants commitment to womens health in areas of significant unmet need, said James Rusnak, M.D., Ph.D., Senior Vice President, Chief Development Officer, Internal Medicine and Hospital, Global Product Development at Pfizer. We look forward to making MYFEMBREE available to women with endometriosis and broadening their options in managing this complex disorder.

MYFEMBREE offers an effective, once-daily treatment option for the management of moderate to severe pain associated with endometriosis, with a treatment duration of up to 24 months. Endometriosis is a serious chronic condition that requires long-term interventions. Optimization of medical therapies is the recommended treatment paradigm. 1,2,3 MYFEMBREE introduces an option for up to two years of pharmacological management of moderate to severe pain associated with endometriosis in pre-menopausal women.

The data from the SPIRIT studies showed the clinical benefit that relugolix combination therapy can have on moderate to severe pain associated with endometriosis and how it can impact patients, said Linda Giudice, M.D., Ph.D., Distinguished Professor at the University of California, San Francisco (UCSF), and Chair, SPIRIT Program Steering Committee. This newly approved option for patients with pain from endometriosis offers the convenience of one pill taken once daily with a mean change in bone mineral density of <1% that did not appear to worsen at 12 months of treatment; however, monitoring is recommended.

This approval is supported by one-year data from the Phase 3 SPIRIT program, which included two 24-week multi-national clinical studies (SPIRIT 1 and SPIRIT 2) in more than 1,200 women with pain associated with endometriosis, as well as the first 28 weeks of an open-label extension study to assess its longer-term use. Overall, these studies showed MYFEMBREE reduced menstrual pain and non-menstrual pelvic pain with a loss of mean bone mineral density of less than 1% from baseline through one year of treatment.4

SPIRIT 1 and 2 each met their co-primary endpoints with 75% of women in the MYFEMBREE group in both studies achieving a clinically meaningful reduction in dysmenorrhea compared with 27% and 30% of women in the placebo groups at Week 24, respectively (both p <0.0001). For non-menstrual pelvic pain, treatment with MYFEMBREE demonstrated a clinically meaningful reduction in pain in 59% and 66% of women, compared with 40% and 43% of women in the placebo groups (p < 0.0001). Adverse reactions occurring in at least 3% of women treated with MYFEMBREE and greater than placebo were: headache, vasomotor symptoms, mood disorders, abnormal uterine bleeding, nausea, toothache, back pain, decreased sexual desire and arousal, arthralgia, fatigue, and dizziness.The open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2 showed mean bone mineral density loss of less than 1% from baseline through one year of treatment; some patients (19.7%) had losses >3%. Annual bone density measurement is recommended while treating women for endometriosis.

MYFEMBREE is available immediately to patients with moderate to severe pain associated with endometriosis with a prescription from their healthcare provider. Myovant and Pfizer also are committed to supporting women in the U.S. who are prescribed MYFEMBREE throughout their treatment journeys. The MYFEMBREE Support Program provides access support services, including insurance benefits checks, prior authorization support, co-pay support for commercially insured patients, and patient assistance for qualifying uninsured patients. Program terms and conditions apply. For more information and additional resources, please contact 833-MYFEMBREE (833-693-3627), 8 a.m. 8 p.m. Eastern Time, Monday Friday.

Myovant Conference CallMyovant will hold a conference call on Monday, August 8, 2022, at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time to discuss the FDA approval of MYFEMBREE for the management of moderate to severe pain associated with endometriosis. Investors and the general public may access the live webcast here. The live webcast can also be accessed by visiting the companys investor relations page of Myovants website at: https://investors.myovant.com/.

Endometriosis can also impact general physical, mental, and social well-being, requiring a multi-disciplinary approach to care. Approximately 190 million women suffer from symptoms of endometriosis globally.5 In the U.S., there are approximately 7.5 million premenopausal women with endometriosis and approximately 75-80 percent of them are symptomatic.6,7,8,9 Many women with pain associated with endometriosis are not able to manage their pain symptoms with current treatment options, underscoring the high unmet need for this disease.10 It can take between four and eleven years to get an endometriosis diagnosis11,12,13 and for some women, current treatment options do not provide relief.14

About MYFEMBREEMYFEMBREE (relugolix, estradiol, and norethindrone acetate) is a once-daily oral treatment approved by the U.S. Food and Drug Administration for the management of moderate to severe pain associated with endometriosis, with a treatment duration of up to 24 months. It is also currently available in the U.S. for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months. MYFEMBREE contains relugolix, which reduces the amount of estrogen (and other hormones) produced by ovaries, estradiol (an estrogen) which may reduce the risk of bone loss, and norethindrone acetate (a progestin) which is necessary when women with a uterus (womb) take estrogen.

For full prescribing information including Boxed Warning and patient information, please click here.Indications and UsageMYFEMBREE is indicated in premenopausal women for the management of: Heavy menstrual bleeding associated with uterine leiomyomas (fibroids) Moderate to severe pain associated with endometriosisLimitations of Use: Use of MYFEMBREE should be limited to 24 months due to the risk of continued bone loss which may not be reversible.

IMPORTANT SAFETY INFORMATION

BOXED WARNING: THROMBOEMBOLIC DISORDERS AND VASCULAR EVENTS Estrogen and progestin combination products, including MYFEMBREE, increase the risk of thrombotic or thromboembolic disorders including pulmonary embolism, deep vein thrombosis, stroke and myocardial infarction, especially in women at increased risk for these events. MYFEMBREE is contraindicated in women with current or a history of thrombotic or thromboembolic disorders and in women at increased risk for these events, including women over 35 years of age who smoke or women with uncontrolled hypertension.

CONTRAINDICATIONSMYFEMBREE is contraindicated in women with any of the following: high risk of arterial, venous thrombotic, or thromboembolic disorder; pregnancy; known osteoporosis; current or history of breast cancer or other hormone-sensitive malignancies; known hepatic impairment or disease; undiagnosed abnormal uterine bleeding; known hypersensitivity to components of MYFEMBREE.

WARNINGS AND PRECAUTIONSThromboembolic Disorders: Discontinue immediately if an arterial or venous thrombotic, cardiovascular, or cerebrovascular event occurs or is suspected. Discontinue at least 4 to 6 weeks before surgery associated with an increased risk of thromboembolism, or during periods of prolonged immobilization, if feasible. Discontinue immediately if there is sudden unexplained partial or complete loss of vision, proptosis, diplopia, papilledema, or retinal vascular lesions and evaluate for retinal vein thrombosis as these have been reported with estrogens and progestins.

Bone Loss: MYFEMBREE may cause a decrease in bone mineral density (BMD) in some patients, which may be greater with increasing duration of use and may not be completely reversible after stopping treatment. Consider the benefits and risks in patients with a history of low trauma fracture or risk factors for osteoporosis or bone loss, including medications that may decrease BMD. Assessment of BMD by dual-energy X-ray absorptiometry (DXA) is recommended at baseline in all women. During treatment, periodic DXA is recommended for women with heavy menstrual bleeding due to uterine fibroids; in those with moderate to severe endometriosis pain, annual DXA is recommended. Consider discontinuing MYFEMBREE if the risk of bone loss exceeds the potential benefit.Hormone-Sensitive Malignancies: Discontinue MYFEMBREE if a hormone-sensitive malignancy is diagnosed. Surveillance measures in accordance with standard of care, such as breast examinations and mammography are recommended. Use of estrogen alone or estrogen plus progestin has resulted in abnormal mammograms requiring further evaluation.

Suicidal Ideation and Mood Disorders (Including Depression): Evaluate patients with a history of suicidal ideation, depression, and mood disorders prior to initiating treatment. Monitor patients for mood changes and depressive symptoms including shortly after initiating treatment, to determine whether the risks of continuing therapy with MYFEMBREE outweigh the benefits. Patients with new or worsening depression, anxiety, or other mood changes should be referred to a mental health professional, as appropriate. Advise patients to seek immediate medical attention for suicidal ideation and behavior and reevaluate the benefits and risks of continuing MYFEMBREE. Gonadotropin-releasing hormone receptor antagonists, including MYFEMBREE, have been associated with mood disorders (including depression) and suicidal ideation. Hepatic Impairment and Transaminase Elevations: Steroid hormones may be poorly metabolized in these patients. Instruct women to promptly seek medical attention for symptoms or signs that may reflect liver injury, such as jaundice or right upper abdominal pain. Acute liver test abnormalities may necessitate the discontinuation of MYFEMBREE use until the liver tests return to normal and MYFEMBREE causation has been excluded.

Gallbladder Disease or History of Cholestatic Jaundice: Discontinue MYFEMBREE if signs or symptoms of gallbladder disease or jaundice occur. For women with a history of cholestatic jaundice associated with past estrogen use or with pregnancy, assess the risk-benefit of continuing therapy. Studies among estrogen users suggest a small increased relative risk of developing gallbladder disease.

Elevated Blood Pressure: For women with well-controlled hypertension, monitor blood pressure and stop MYFEMBREE if blood pressure rises significantly.

Change in Menstrual Bleeding Pattern and Reduced Ability to Recognize Pregnancy: Advise women to use non-hormonal contraception during treatment and for one week after discontinuing MYFEMBREE. Avoid concomitant use of hormonal contraceptives. MYFEMBREE may delay the ability to recognize pregnancy because it alters menstrual bleeding. Perform testing if pregnancy is suspected and discontinue MYFEMBREE if pregnancy is confirmed.

Risk of Early Pregnancy Loss: MYFEMBREE can cause early pregnancy loss. Exclude pregnancy before initiating and advise women to use effective non-hormonal contraception.

Uterine Fibroid Prolapse or Expulsion: Advise women with known or suspected submucosal uterine fibroids about the possibility of uterine fibroid prolapse or expulsion and instruct them to contact their physician if severe bleeding and/or cramping occurs.

Alopecia: Alopecia, hair loss, and hair thinning were reported in phase 3 trials with MYFEMBREE. Consider discontinuing MYFEMBREE if hair loss becomes a concern. Whether the hair loss is reversible is unknown.

Effects on Carbohydrate and Lipid Metabolism: More frequent monitoring in MYFEMBREE-treated women with prediabetes and diabetes may be necessary. MYFEMBREE may decrease glucose tolerance and result in increased blood glucose concentrations. Monitor lipid levels and consider discontinuing if hypercholesterolemia or hypertriglyceridemia worsens. In women with pre-existing hypertriglyceridemia, estrogen therapy may be associated with elevations in triglycerides levels leading to pancreatitis. Use of MYFEMBREE is associated with increases in total cholesterol and LDL-C.

Effect on Other Laboratory Results: Patients with hypothyroidism and hypoadrenalism may require higher doses of thyroid hormone or cortisol replacement therapy. Use of estrogen and progestin combinations may raise serum concentrations of binding proteins (e.g., thyroid-binding globulin, corticosteroid-binding globulin), which may reduce free thyroid or corticosteroid hormone levels. Use of estrogen and progestin may also affect the levels of sex hormone-binding globulin, and coagulation factors.

Hypersensitivity Reactions: Immediately discontinue MYFEMBREE if a hypersensitivity reaction occurs.

ADVERSE REACTIONS: Most common adverse reactions for MYFEMBREE (incidence 3% and greater than placebo) were: Heavy menstrual bleeding associated with uterine fibroids: vasomotor symptoms, abnormal uterine bleeding, alopecia, and decreased libido. Moderate to severe pain associated with endometriosis: headache, vasomotor symptoms, mood disorders, abnormal uterine bleeding, nausea, toothache, back pain, decreased sexual desire and arousal, arthralgia, fatigue, and dizziness.

These are not all the possible side effects of MYFEMBREE.

DRUG INTERACTIONS: P-gp Inhibitors: Avoid use of MYFEMBREE with oral P-gp inhibitors. If use is unavoidable, take MYFEMBREE first, separate dosing by at least 6 hours, and monitor patients for adverse reactions. Combined P-gp and Strong CYP3A Inducers: Avoid use of MYFEMBREE with combined P-gp and strong CYP3A inducers.

LACTATION: Advise women not to breastfeed while taking MYFEMBREE.

About Myovant SciencesMyovant Sciences aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. Founded in 2016, Myovant has executed five successful Phase 3 clinical trials across oncology and womens health leading to three regulatory approvals by the U.S. Food and Drug Administration (FDA) for men with advanced prostate cancer, women with heavy menstrual bleeding associated with uterine fibroids, and pre-menopausal women with moderate to severe pain associated with endometriosis, respectively. Myovant also has received regulatory approvals by the European Commission (EC) and the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) for women with symptomatic uterine fibroids and for men with advanced hormone-sensitive prostate cancer. Myovant has a supplemental New Drug Application under review with the FDA for updates to the United States Prescribing Information (USPI) based on safety and efficacy data from the Phase 3 LIBERTY randomized withdrawal study (RWS) of MYFEMBREE in premenopausal women with heavy menstrual bleeding due to uterine fibroids for up to two years. Myovant also is conducting a Phase 3 study to evaluate the prevention of pregnancy in women with uterine fibroids or endometriosis. Myovant also is developing MVT-602, an investigational oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Pharma Co., Ltd., is Myovants majority shareholder. For more information, please visit http://www.myovant.com. Follow @Myovant on Twitter and LinkedIn.

About Pfizer: Breakthroughs That Change Patients LivesAt Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Myovant Sciences Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In this press release, forward-looking statements include, but are not limited to, all statements reflecting Myovant Sciences expectations, including: statements regarding Myovants aspiration to redefine care for women and for men; the expectations regarding the continued commercialization of MYFEMBREE by Myovant and Pfizer jointly in the U.S. and the timeline of product availability; the expectations that MYFEMBREEs indication helps advance Myovants mission to redefine care for women by helping address a disease with high unmet need, giving women and physicians a new meaningful treatment option to manage moderate to severe pain associated with endometriosis in Dr. Arjona Ferreiras quote; the expectation of making MYFEMBREE available to women with endometriosis and broadening their options in managing this complex disorder in Dr. Rusnaks quote; and the expectations of the MYFEMBREE Support Program for patients and the features of such program.Myovant Sciences forward-looking statements are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties, assumptions, and other factors known and unknown that could cause actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements, including unforeseen circumstances or other disruptions to normal business operations arising from or related to the COVID-19 pandemic and the conflict in Ukraine. Myovant Sciences cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur, and actual results could differ materially from those expressed or implied by these forward-looking statements. Factors that could materially affect Myovant Sciences operations and future prospects or which could cause actual results to differ materially from expectations include, but are not limited to, the risks and uncertainties listed in Myovant Sciences filings with the United States Securities and Exchange Commission (SEC), including under the heading Risk Factors in Myovant Sciences Quarterly Report on Form 10-Q filed on July 27, 2022, as such risk factors may be amended, supplemented, or superseded from time to time. These risks are not exhaustive. New risk factors emerge from time to time, and it is not possible for Myovant Sciences management to predict all risk factors, nor can Myovant Sciences assess the impact of all factors on its business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not place undue reliance on the forward- looking statements in this press release, which speak only as of the date hereof, and, except as required by law, Myovant Sciences undertakes no obligation to update these forward-looking statements to reflect events or circumstances after the date of such statements.

Pfizer Disclosure NoticeThe information contained in this release is as of August 5, 2022. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about MYFEMBREE (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg), a new indication in the U.S. for the management of moderate to severe pain associated with endometriosis in pre-menopausal women, and a collaboration between Pfizer and Myovant Sciences to develop and commercialize relugolix in advanced prostate cancer and womens health, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of MYFEMBREE; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when applications may be filed for any other potential indications for MYFEMBREE; whether and when regulatory authorities may approve any such applications for MYFEMBREE that may be pending or filed, which will depend on myriad factors, including making a determination as to whether the products benefits outweigh its known risks and determination of the products efficacy and, if approved, whether MYFEMBREE will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of MYFEMBREE; whether our collaboration with Myovant Sciences will be successful; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

1American Society for Reproductive Medicine (ASRM), Treatment of pelvic pain associated with endometriosis: a committee opinion. Fertil Steril. 2014;101(4):927-35.2Becker CM et al. ESHRE guideline: endometriosis, Human Reproduction Open. 2022 Feb 26;2022(2): hoac009. 3Taylor HS et al. Endometriosis is a chronic systemic disease: clinical challenges and novel innovations. Lancet 2021;397(10276):839-52 4Giudice LC, et al. Lancet. 2022 Jun; 399(10343): 2267-2279. 5Adamson, G. et al. Journal Endometriosis. 2010; 2:3-66US census 2019 (table 1; approx. 75 million women in the US ages 15-49). Available online at https://data.census.gov/cedsci/table?q=United%20States&t=Age%20and%20Sex7Shafrir. Best Pract Res Clin Obstet Gynaecol. 2018 Aug; 51:1-15 8Fuldeore Gynecol Obstet Invest. 2017;82:453-461 9Bulletti J Asist Reprod Genet 201010Becker CM, et al. Fertil Steril. 2017 Jul;108(1):125-136. 11Zondervan KT, et al. NEJM. 2020;382(13):12441256 12Nnoaham KE et al. Fertil Steril. 2011;96(2):366.e8373.e8 13Ballard K et al. Fertil Steril. 2006;86:1296301 14Soliman et al. J Womens Health. 2017. 26(7): 788-797

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Myovant Sciences and Pfizer Receive U.S. FDA Approval of MYFEMBREE, a Once-Daily Treatment for the Management of Moderate to Severe Pain Associated...

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