Advanced Therapy Medicinal Products Market Research Report by Therapy Type (CAR-T Therapy, Cell Therapy, Gene Therapy, and Tissue Engineered Product) - Global Forecast to 2025 - Cumulative Impact of COVID-19

New York, Sept. 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Advanced Therapy Medicinal Products Market Research Report by Therapy Type - Global Forecast to 2025 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p05953100/?utm_source=GNW

The Global Advanced Therapy Medicinal Products Market is expected to grow from USD 2,946.38 Million in 2019 to USD 6,524.94 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 14.16%.

Market Segmentation & Coverage:This research report categorizes the Advanced Therapy Medicinal Products to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Therapy Type , the Advanced Therapy Medicinal Products Market studied across CAR-T Therapy, Cell Therapy, Gene Therapy, and Tissue Engineered Product. The Cell Therapy further studied across Non-stem Cell Therapy and Stem Cell Therapy.

Based on Geography, the Advanced Therapy Medicinal Products Market studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas region surveyed across Argentina, Brazil, Canada, Mexico, and United States. The Asia-Pacific region surveyed across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, South Korea, and Thailand. The Europe, Middle East & Africa region surveyed across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Company Usability Profiles:The report deeply explores the recent significant developments by the leading vendors and innovation profiles in the Global Advanced Therapy Medicinal Products Market including AveXis, Inc., Bluebird Bio, Inc., Celgene Corporation, Gilead Lifesciences, Inc., JCR Pharmaceuticals Co., Ltd., Kolon TissueGene, Inc., MEDIPOST, Novartis AG, Organogenesis Inc., PHARMICELL Co., Ltd, Spark Therapeutics, Inc., UniQure N.V., and Vericel Corporation.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Advanced Therapy Medicinal Products Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, so for and, the long-term effects projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlaying COVID-19 issues and potential paths forward. The report is delivering insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecast, considering the COVID-19 impact on the market.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:1. What is the market size and forecast of the Global Advanced Therapy Medicinal Products Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Advanced Therapy Medicinal Products Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Advanced Therapy Medicinal Products Market?4. What is the competitive strategic window for opportunities in the Global Advanced Therapy Medicinal Products Market?5. What are the technology trends and regulatory frameworks in the Global Advanced Therapy Medicinal Products Market?6. What are the modes and strategic moves considered suitable for entering the Global Advanced Therapy Medicinal Products Market?Read the full report: https://www.reportlinker.com/p05953100/?utm_source=GNW

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Advanced Therapy Medicinal Products Market Research Report by Therapy Type - Global Forecast to 2025 - Cumulative Impact of COVID-19 - Yahoo Finance...

The European Medicines Agency is on the verge of releasing revised guidance for advanced therapy medicinal products containing genetically modified cells, which includes chimeric antigen receptor (CAR)-T cell therapies.

The Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells was originally issued in 2012 but underwent revision and consultation from July 2018-July 2019. The revised version is expected to be adopted in October and published in November, according to Ana Hidalgo-Simon, MD, PhD, head of advanced therapies at EMA. She previewed the major changes at RAPS Convergence 2020.

There were an enormous number of comments on the document, Hidalgo-Simon said.The agency is also working on a Q&A document on principles of good manufacturing practices (GMP) for Advanced Therapy Medicinal Products (ATMP) starting material. There will likely be consultation on the document in 2021, she said. (RELATED: Regulation of advanced therapy medicinal products in the EU, Regulatory Focus, 16 July 2020.)

Major changesEMA chose to update the guidance to reflect the increase in clinical experience with these therapies, particularly chimeric antigen receptor-T (CAR-T) cells; to cover new categories of products, such as induced pluripotent stem (iPS) cells; and to allow for consideration of new tools for genetic modification of cells, such as genome editing technologies, she said.

The main quality updates are related to starting materials, the manufacturing process, and characterization and release. For example, the starting materials guidance will now include genome editing tools, while the manufacturing process includes a new section on comparability. The characterization and release portion of the guidance includes specific advice for CAR-T cells.

Additionally, the guidance calls for dose-finding studies to explore safety, toxicity, and anti-tumor activity at different dose levels, to define the threshold dose required for anti-tumor effect, and to define the recommended dose or range for Phase 2 studies. She said sponsors need to show a solid rationale for the criteria being used to find the dose.

The guidance also calls for Phase 3 confirmatory trials to follow a randomized controlled design, comparing the CAR-T cell therapy to a reference regimen, unless otherwise scientifically justified. Single-arm studies will continue to be allowed, but they will be the exception, Dr. Hidalgo-Simon said.

Be very careful with the design of the trials, she advised. The assumptions need to be really, very well backed.

When it comes to safety, the guidance calls for a 15-year follow period. While sponsors wont have all the answers at the time of submission, Hidalgo-Simon said they should have a plan that includes monitoring during the post-authorization period.

Hidalgo-Simon also advised sponsors to think beyond the approval process and consider what evidence will be needed to convince other stakeholders -- from patients to payers -- about the safety and efficacy of the therapy.

Avoiding development pitfallsRichard Dennett, PhD, the senior director of chemistry, manufacturing and controls regulatory affairs at PPD, also participated in the RAPS Convergence 2020 session on advanced therapies. He reviewed development points where companies can run into trouble with advanced therapies, particularly CAR-T cell products.Dennett recommended that product sponsors keep the end in mind when developing advanced therapies by focusing on the target product profile at the beginning of development. That profile includes the indication for which approval will be sought and the incidence of that indication; other considerations include mode of action, demographics, how much of the product needs to be produced, and market access and reimbursement considerations.

He also outlined several areas where developers should focus to create a watertight regulatory package, including sufficient product characterization, potency assay, impurities, formulation, stability, lack of sufficient development batches, and validation strategy.

Dennett urged developers to dive into the growing number of regulatory guidance documents for advanced therapies. In addition to the European guidance documents, developers should consultthe US Food and Drug Administrations Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), which was released in January 2020. (RELATED: Advanced therapies: Trip hazards on the development pathway, Regulatory Focus, 02 August 2020)

Live and breathe the guidances that are out there, Dennett advised. They allow us to understand what expectations we need to meet.

The key to success in advancing CAR-T cell therapies is the mitigation of risk, Dennett said: The biggest risk is the one that you havent thought of.RAPS 2020 Convergence

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Convergence: EMA close to finalizing guidance for advanced therapies - Regulatory Focus

BEDFORD, Mass., Sept. 18, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today participation and presentations at the following virtual conferences:

The live webcast presentations from the Oppenheimer and Chardan conferences will be accessible on Homologys website in the Investors section, and the webcast replays will be available on the website for 90 days following the presentations. For on-demand webcasts from the Cell & Gene Meeting on the Mesa conference, please visit http://www.meetingonthemesa.com for full information.

About Homology Medicines, Inc. Homology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

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Homology Medicines to Participate in Upcoming Conferences - GlobeNewswire

The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments. The sector can certainly point to the significant progress that has taken place during the past few years, with new medicines reaching the market and several novel therapeutics with new mechanisms of action advancing in the pipeline.

Ted Love, president and CEO of Global Blood Therapeutics Inc. (GBT), said 2019 was a landmark year with the FDA approval of two new novel therapies to treat sickle cell disease. He was speaking at the virtual annual Sickle Cell Disease (SCD) Therapeutics Conference this week. His company, together with the Sickle Cell Disease Association of America, was hosting the one-day event featuring discussions on the latest advances and future trends.

Approvals

The key manifestation of the inherited blood disorder is that red blood cells (RBCs) are abnormally shaped (crescent), which restricts their flow in blood vessels and limits oxygen delivery to the bodys tissues, leading to severe pain and organ damage. The condition is also characterized by severe chronic inflammation that results in vaso-occlusive crisis (VOC) where patients experience episodes of extreme pain and organ damage.

Late November, GBT gained accelerated approval for its Oxbryta (voxelotor) tablets for the treatment SCD in adults and pediatric patients 12 and older. The agencys green light came less than two weeks after it gave the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of VOCs in adult and pediatric patients ages 16 and older with SCD.

According to Love, Oxbryta is a new class of therapy binding to hemoglobin and stabilizing RBCs in an oxygenated state and inhibiting deoxygenated sickle hemoglobin polymerization, making cells less likely to bind together and form the distinctive sickle shape.

The launch of the drug has gone well since it was approved, he said in the companys second-quarter financial report and business update, despite the impact of COVID-19. Net sales in the period reached $31.5 million, well ahead of the Streets expectations. Going forward, the company is planning to expand the potential use of Oxbryta for the treatment of SCD in children as young as 4 years old and also seek marketing authorization in Europe for Oxbryta to treat hemolytic anemia in SCD patients ages 12 and older with a marketing authorization application being submitted to the EMA by the middle of next year.

Pipeline progress

Cambridge, Mass.-based Agios Pharmaceuticals Inc. is working on mitapivat (AG-348), an investigational, oral, small-molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes, in patients with SCD. The compound has been shown to decrease 2,3-diphosphoglycerate (2,3-DPG) and increase adenosine triphosphate (ATP), and through that mechanism, it may reduce hemoglobin (Hb) S polymerization and red blood cell sickling. In June, the company reported that clinical proof of concept had been established based on a preliminary analysis in a phase I trial being conducted in collaboration with the U.S. NIH as part of a cooperative research and development agreement.

The ongoing study had enrolled nine patients, with eight completing all planned dose levels of mitapivat. Seven of eight patients who completed all dose levels experienced a Hb increase, with five of eight patients (63%) achieving a hemoglobin increase of 1 g/dL from baseline (range 1-2.7 g/dL). All five patients who achieved a hemoglobin increase of 1 g/dL did so at doses of 50 mg BID or lower. Decreases in 2,3-DPG and increases in ATP levels were observed, consistent with the proposed mechanism of action and comparable to that observed in healthy volunteer studies with mitapivat.

The company said it expects to report data from ACTIVATE and ACTIVATE-T, its two global pivotal trials for mitapivat in adults with pyruvate kinase deficiency, between the end of 2020 and mid-2021.

Watertown, Mass.-based Forma Therapeutics Holdings Inc., which made its public debut this year, also has a selective RBC pyruvate kinase-R activator in its pipeline for treating SCD. FT-4202 is being evaluated in a phase I trial in SCD patients ages 12 and older and has been granted fast track, rare pediatric and orphan drug designations. The compound is a potent activator of pyruvate kinase-R designed to improve RBC metabolism, function and survival by decreasing 2,3 DPG and increasing ATP, potentially resulting in both increased hemoglobin levels and reduced VOCs.

Olinciguat, an oral guanylate cyclase (sGC) stimulator, being developed by Cyclerion Therapeutics Inc., has completed the treatment period in its STRONG-SCD study with a total of 70 patients randomized. The placebo-controlled, dose-ranging study is designed to evaluate safety, tolerability and pharmacokinetics, as well as to explore effects on daily symptoms and biomarkers of disease activity when dosed over a 12-week treatment period. Top-line results are expected this year. Olinciguat is a compound that aims to stimulate sGC production, leading to the production of a signaling molecule called cyclic guanosine monophosphate (cGMP). High levels of cGMP help reduce inflammation in blood vessels, decrease adhesion between RBCs, and allow for improved blood flow by increasing the availability of nitric oxide.

Boston-based Imara Inc. is developing IMR-687, a small-molecule inhibitor of PDE9 that degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. The company said that lower levels of cGMP are often found in people with SCD and beta-thalassemia and are associated with impaired blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide-mediated vasodilation. Blocking PDE9 acts to increase cGMP levels, which are associated with reactivation of fetal hemoglobin.

In August, the company dosed the first patient in its Ardent phase IIb trial of IMR-687 for adult patients with SCD. The planned primary efficacy objective is to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an increase of 3% in HbF from baseline to week 24, compared to placebo.

Gene therapy/gene editing

Since SCD is a monogenic disease condition, researchers believe that it would be a good candidate for gene therapy therapeutic approaches. For example, significant progress is being made by Cambridge, Mass.-based Bluebird Bio Inc. with lentiglobin, its lentiviral-based gene therapy that inserts an anti-sickling beta-globin variant into CD34-positive cells, progenitors of red blood cells.

At the virtual European Hematology Association (EHA) meeting in June, it reported new data from its ongoing phase I/II study involving adult and adolescent patients with SCD that showed a near-complete reduction of serious VOCs and acute chest syndrome. The company expects to submit a BLA to the FDA for the gene therapy next year.

Crispr Therapeutics AG and Vertex Pharmaceuticals Inc. are progressing CTX-001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies. At EHA, the companies reported that in the phase I/II Climb-121 study, at nine months after CTX-001 infusion, the first treated patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%.

Last month, Cambridge, Mass.-based Editas Medicine Inc., a genome editing company, reported that the FDA had granted rare pediatric disease designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for SCD. The company plans to file an investigational new drug application for EDIT-301 by the end of this year. EDIT-301 comprises sickle patient CD34+ cells genetically modified using a hCRISPR/Cas12a (also known as Cpf1) ribonucleoprotein to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production.

In its second-quarter financial report and business update, Beam Therapeutics Inc. announced the nomination of its first two adenine base editing development candidates, BEAM-101, targeting patients with hereditary persistence of fetal hemoglobin, and BEAM-102 (Makassar variant), both aimed at correcting SCD.

New Initiative

The NIH, which reports it spends approximately $100 million on sickle cell disease research, announced that is has launched The Cure Sickle Cell Initiative designed to speed the development of cures for the disease. It will take advantage of the latest genetic discoveries and technological advances to progress the most promising genetic-based curative therapies safely into clinical trials within five to 10 years.

Aided by research partners, the initiative will establish a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost effectiveness. National networks will also be created to make it easier for patients and providers to interact with the research, clinical trials, and other activities.

Excerpt from:
New medicines in the pipeline to treat sickle cell disease - BioWorld Online

Moderna has agreed to separate deals with Vertex and Chiesi for research and development spanning cystic fibrosis (CF) therapies and pulmonary arterial hypertension (PAH).

In its agreement with Vertex, Moderna will receive $75m to collaborate on the research and development into gene therapies for CF. As part of the deal, Moderna will discover and develop lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies for the treatment of CF.

Under the terms of the deal, Moderna will receive $75m upfront to conduct research activities to discover and develop LNPs for gene-editing CF therapies. The US-based biotech company will also be eligible to receive up to $380m in further development, regulatory and commercial milestone payments, as well as tiered royalties on any products that come out of the collaboration.

While Moderna will be responsible for the initial discovery and manufacturing of LNPs and mRNA constructs for the gene-editing treatments, Vertex will be responsible for providing other components of the therapies to be formulated into LNPs, as well as the preclinical and clinical development and commercialisation efforts of any potential candidates.

This is the second agreement reached between Moderna and Vertex, with the previous collaboration aimed at the discovery and development of mRNA therapeutics for CF having been recently extended.

Our first collaboration with Vertex to deliver mRNA coding for cystic fibrosis protein in lung cells is advancing well and this second collaboration aims at using Modernas technologies to explore the use of gene editing in lung cells, said Stphane Bancel, chief executive officer of Moderna.

Prior to announcing the Vertex deal, Moderna revealed that it had come to a separate agreement with Italian-domiciled pharma company Chiesi, for the development of mRNA treatments for PAH.

As part of that deal, Moderna will receive $25m upfront as well as a potential $400m in milestone payments to conduct research and development activities in this therapy area.

PAH is a rare and progressive disorder, which occurs when arteries in the lungs constrict, which forces the heart to work harder, often causing heart failure. The condition affects around two to five million adults across the globe, and remains an area of high unmet medical need.

Read more from the original source:
Moderna inks R&D agreements with Vertex and Chiesi - PMLiVE

When a researchers talk about gene editing, theyre usually thinking about several steps. First you need to zero in on the defective gene; then, depending on the need, youd want to knock out, replace or insert genetic material.

CRISPR/Cas9 technologies have transformed the field by making a breakthrough for the first problem. Inducing double-stranded DNA breaks, or achieving single-letter changes as base editing allows, have promising applications in multiple diseases that are starting to get tested in humans.

But Versant Ventures, one of the pioneering investors in the space, still sees a gap. And theyre teaming up with a group of prominent Stanford researchers plus a seasoned biotech exec to fill it.

Achieving high-efficiency targeted gene integration has been a critical objective of gene editing for more than 15 years, but only now is this technologically possible, Jerel Davis, Versants Vancouver-based managing director, said.

Graphite Bio has $45 million to start things off, but the money matters little when compared to the rich brain bank its drawing from.

Matthew Porteus, an academic founder of CRISPR Therapeutics, is lending the tech platform to create this next-gen play alongside gene therapy expert Maria Grazia Roncarolo.

Drawing from research work led by Danny Dever while a postdoc at Porteus lab, Graphites big promise is to increase integration efficiency from less than 1% to greater than 50% across diverse genetic lesions in a wide range of cell types.

Specifically, Dever and Porteus showed that they could correct the sickle globin gene in patient-derived hematopoietic stem cells ex vivo by combining Cas9 ribonucleoproteins with a donor molecule that serves as a template, delivered in recombinant adeno-associated viral vectors of serotype 6 (rAAV6).

That could make for a much more scalable replacement for transplants, they wrote in a 2017 paper describing mouse studies.

Notably, we devise an enrichment paradigm to purify a population of HSPCs with >90% targeted integration, they wrote. We also show efficient correction of the SCD-causing E6V mutation in patient-derived HSPCs that after differentiation into erythrocytes, express adult -globin (HbA) mRNA, confirming intact transcriptional regulation of edited HBB alleles.

Chief executive Josh Lehrer is leading the charge to start Phase I for this program in early 2021, bringing all the sickle cell knowledge and experience from a six-year run as Global Blood Therapeutics, most recently as CMO. More preclinical therapies are in the works for unnamed serious diseases. Samsara BioCapital is joining Versant for the launch round and sending Abe Bassan to the board, which also features Davis and Carlo Rizzuto from Versant.

More:
Versant teams up with Stanford gene editing experts on a $45M next-gen play marrying CRISPR and AAV to fix sickle cell - Endpoints News

Technology plays a key role in the bleeding disorders community and will dictate where we will go with treatments and care from gene therapy to novel technologies and cell therapy on the horizon.

JUPITER, Fla. (PRWEB) September 18, 2020

An upcoming episode of Advancements with Ted Danson will focus on recent developments in health management as it relates to those affected with inheritable bleeding disorders. Check local listings for more info.

Bleeding disorders result when the bloods ability to form a clot at the site of blood vessel injury is impaired. Shining a light on the National Hemophilia Foundation (NHF), this segment will explore the latest information surrounding inheritable bleeding disorders, such as hemophilia, von Willebrand disease, and rare factor deficiencies.

Viewers will learn about NHFs programs and initiatives, which are dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy, and research.

The National Hemophilia Foundation is excited to participate in this unique opportunity, said Dr. Leonard Valentino, President and CEO at NHF. Technology plays a key role in the bleeding disorders community and will dictate where we will go with treatments and care from gene therapy to novel technologies and cell therapy on the horizon.

Audiences will hear from experts and will also be educated about experimental methods, such as gene therapy, that are currently being investigated as potential cures for bleeding disorders.

Bleeding disorders have been documented throughout history and can be found in writings as far back as the second century AD, said Richard Lubin, senior producer for Advancements. We look forward to exploring how technology, research, and NHFs dedication is opening doors for breakthrough treatment options for hemophilia and similar bleeding disorders.

About the National Hemophilia Foundation (NHF)The National Hemophilia Foundation (NHF) is a 501(c)(3) non-profit organization dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research. NHFs programs and initiatives are made possible through the generosity of individuals, corporations and foundations, as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC). For more information, visit: http://www.hemophilia.org.

About Advancements and DMG Productions:The Advancements series is an information-based educational show targeting recent advances across a number of industries and economies. Featuring state-of-the-art solutions and important issues facing todays consumers and business professionals, Advancements focuses on cutting-edge developments, and brings this information to the public with the vision to enlighten about how technology and innovation continue to transform our world.

Backed by experts in various fields, DMG Productions is dedicated to education and advancement, and to consistently producing commercial-free, educational programming on which both viewers and networks depend.

For more information, please visit http://www.AdvancementsTV.com or call Richard Lubin at 866-496-4065.

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Advancements Television Series to Explore Breakthroughs in Research and Treatment for Inheritable Bleeding Disorders - PR Web

With September being Sickle Cell Disease Awareness Month, Loma Linda University Childrens Health wants to help educate the community about SCD one of the most common yet overlooked genetic disorders in the world.

Each year, approximately 1,000 babies in the U.S. and 500,000 worldwide are born with the disease, according to the Sickle Cell Disease Association of America.

Akshat Jain, MD, MPH, a global sickle cell disease expert at Childrens Hospital, is passionate about establishing awareness and proper care for children suffering from SCD and Sickle Cell Trait, especially the diverse patient population in San Bernardino County.

There are many barriers to receiving care for those with SCD in our community, Jain says. One barrier specifically is lack of awareness surrounding the disease coupled with lack of awareness surrounding the treatment options available at Childrens Hospital.

In sickle cell disease, a persons red blood cells have an irregular cell shape, Jain says. Instead of round discs, theyre in a crescent or sickle shape.

Due to their shape, texture and inflexibility, the cells become clumped together. This grouping causes a blockage in a childs blood vessels, hindering blood-flow. This blockage may cause varying levels of pain and potentially organ damage long-term.

Jain says some of the signs and symptoms of SCD include:

Jain says that many children with SCD develop symptoms in their first year of life. SCD is commonly diagnosed during newborn screening tests, which check for the abnormal hemoglobin found in SCD. Additionally, if both parents of a child are known carriers of a SCD trait, their child will have a 25% chance of having the disease, Jain says.

Some of the emergent issues needing immediate medical care in kids with SCD disease include:

Treatments for SCD include pain medicines for pain management, adequate hydration, blood transfusions, vaccines and antibiotics, and some medicines. Currently, stem cell transplant from bone marrow is the recognized cure for SCD.

Childrens Hospital, with Jain working as a lead on the team, performed the institutions first stem cell transplant in 2019, curing a then 11-year-old girl who had suffered from SCD since birth. Since then, the team has successfully performed the transplant on several pediatric patients.

Patients with SCD at Childrens Hospital are placed into a treatment and care program where Jain and his team offer non-traditional services such as individualized patient treatment plans and direct access to the care team in case of an emergent event. Additionally, the program is working toward offering curative gene therapy for both sickle cell and hemophilia patients.

The bottom line is children and families suffering from this disease need to know that theyre not alone, Jain says. Here at Childrens Hospital, we are here to manage and fight this disease alongside of you.

Learn more about our treatments for sickle cell disease at our Specialty Team Centers.

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Here's what to know about Sickle Cell Disease in kids - Loma Linda University Health

Many patients with non-muscle-invasive bladder cancer (NMIBC) failed intravesical BCG therapy. Currently, radical cystectomy is the recommended standard of care for those patients. There is unfortunately no effective other second-line therapy recommended.

In this review, we present the topics of BCG unresponsive NMIBC; definition, prognosis, and further treatment options: immunotherapy, intravesical chemotherapy, gene therapy, and targeted individualized therapy.

There are major challenges of the management of NMIBC who failed BCG therapy as many patients refuse or are unfit for radical cystectomy. Multiple new modalities currently under investigation in ongoing clinical trials to better treat this category of patients. Immunotherapy, especially PD-1/PD-L1 inhibitors, offers exciting and potentially effective strategies for the treatment of BCG unresponsive NMIBC. As the data expands, it is sure that soon there will be established new guidelines for NMIBC.

Expert review of anticancer therapy. 2020 Sep 11 [Epub ahead of print]

Mohamad Moussa, Athanasios G Papatsoris, Athanasios Dellis, Mohamed Abou Chakra, Wajih Saad

Head of Urology department, Zahraa Hospital, University Medical Center, Lebanese University , Beirut, Lebanon., 2nd Department of Urology, School of Medicine, Sismanoglio Hospital, National and Kapodistrian University of Athens , Athens, Greece., Department of Surgery, School of Medicine, Aretaieion Hospital, National and Kapodistrian University of Athens , Athens, Greece., Faculty of Medical Sciences, department of Urology, Lebanese University , Beirut,Lebanon., Head of Oncology department, Zahraa Hospital, University Medical Center, Lebanese University , Beirut, Lebanon.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/32915676

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Novel anticancer therapy in BCG unresponsive non-muscle-invasive bladder cancer. - UroToday

CAMBRIDGE, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced the addition of Nancy Vitale as an independent director to its Board of Directors, effective as of September 15, 2020. Ms. Vitale brings more than 25 years of experience to Voyagers Board, with deep expertise in human resources. She is a former Senior Vice President and Chief Human Resource Officer at Genentech, a member of the Roche Group.

We are thrilled to welcome Nancy to our Board, said Andre Turenne, President and CEO of Voyager. Nancys accomplished background in helping organizations thrive by focusing on a strong patient-centric culture and employee wellbeing will be enormously valuable as we continue to grow Voyager.

Since leaving Genentech, in 2019, Ms. Vitale co-founded and continues to co-manage Partners for Wellbeing, LLC., a boutique human resources consulting firm. Prior to her 13-year tenure at Genentech, Ms. Vitale held senior human resources roles at Procter & Gamble Company and CIGNA. Ms. Vitale earned a Bachelor of Business Administration from the University of Michigan and an MBA from the Goizueta Business School of Emory University.

Im delighted to join Voyagers Board of Directors, Ms. Vitale commented. The company is led by an outstanding team and Im highly compelled by Voyagers mission to deliver life-changing medicines for patients suffering from severe neurological diseases. I look forward to working with other members of the Board and contributing to this important mission.

Ms. Vitale will also serve as a member of the Boards Compensation Committee.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information onVoyager Therapeutics, please visit the companys website atwww.voyagertherapeutics.com or follow@VoyagerTxon Twitter andLinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities law. The use of words such as may, might, will, should, expect, plan, anticipate, believe, estimate, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding Ms. Vitales participation as a member of Voyagers Board of Directors and her ability to help Voyager grow and develop a strong patient-centric culture and focus on employee wellbeing are forward-looking. All forward-looking statements are based on assumptions by Voyagers management that, although Voyager believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected, including the ability of Ms. Vitale to quickly integrate onto the Voyager Board of Directors, to make contributions as a member of the Voyager Board of Directors and to contribute to Voyagers culture. These statements are also subject to a number of material risks and uncertainties that are described in Voyagers most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, as updated by its future filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investors:Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media: Sheryl Seapy W2Opure949-903-4750sseapy@purecommunications.com

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Voyager Therapeutics Appoints Nancy Vitale to its Board of Directors - GlobeNewswire