Xerostomia, a lack of saliva production leading to dry mouth, has a variety of causes, including radiotherapy and chemotherapy, the chronic use of drugs and rheumatic and dysmetabolic diseases. It is also a common side effect of ionizing radiation used to treat head and neck cancer.1

A proof of concept Phase 1 clinical trial sponsored by a third party showed that Aquaporin-1 (AQP1), a trans-membrane protein that facilitates water movement across lipid layers, restored saliva flow in a human population using an adenovirus-based vector encoding AQP1 to a single previously irradiated parotid gland.2 The results from this study led AHN to research using UAGT technology targeting the salivary gland, which combines the use of nonviral DNA vector and lipid microbubbles with a low-frequency acoustic field to create a sonoporation effect allowing gene transfer to the cells of the salivary gland without the introduction of viral antigens.3

According to Mark Trombetta, MD, Director of Clinical Program Development for the AHN Cancer Institute, a radiation oncologist and co-investigator in the networks study of UAGT, thousands of cancer patients suffer from radiation-induced xerostomia, which can cause severe tooth decay, chronic oral pain, loss of taste, inability to eat properly and increased oral infections. Once xerostomia begins, it is a permanent condition.

We believe that UAGT may provide long-term relief of radiation-induced xerostomia, with adjustable dosing and potential for booster doses over time, said Warren Swegal, MD, an AHN head and neck surgeon and clinical lead of the networks UAGT program. No other existing treatment offers a long-lasting solution for this life-altering condition, and we are thrilled to be able to further develop and improve the therapy by leveraging Lantheus proven microbubble technology.

We are excited to support AHN in its efforts to progress this innovative development program. Xerostomia is a chronic and debilitating condition with limited treatment options for patients, said Mary Anne Heino, President and Chief Executive Officer of Lantheus. We believe our microbubble combined with AHNs UAGT technology have the potential to make a difference in the lives of these patients.

Lantheus will supply its microbubbles and activation devices to AHN. AHN will be solely responsible for all development work, future regulatory submissions and commercialization. The deal terms include a transfer price and royalties.

About Lantheus Holdings, Inc.

Lantheus Holdings, Inc. is the parent company of Lantheus Medical Imaging, Inc., Progenics Pharmaceuticals, Inc. and EXINI Diagnostics AB and an established leader and fully integrated provider of innovative imaging diagnostics, targeted therapeutics and artificial intelligence solutions to Find Fight and Follow serious medical conditions. Lantheus provides a broad portfolio of products, including the echocardiography agent DEFINITY Vial for (Perflutren Lipid Microsphere) Injectable Suspension; TechneLite (Technetium Tc99m Generator), a technetium-based generator that provides the essential medical isotope used in nuclear medicine procedures; AZEDRA for the treatment of certain rare neuroendocrine tumors; and RELISTOR for the treatment of opioid-induced constipation, which is partnered with Bausch Health Companies, Inc. The Company is headquartered in North Billerica, Massachusetts with offices in New York, New Jersey, Canada and Sweden. For more information, please visit http://www.lantheus.com.

About Allegheny Health Network

Allegheny Health Network (AHN.org) is an integrated healthcare delivery system serving the greater Western Pennsylvania region. The Network is composed of 13 hospitals, ambulatory surgery centers, Health + Wellness Pavilions, an employed physician organization, home and community-based health services, a research institute and a group purchasing organization. The Network provides patients with access to a complete spectrum of advanced medical services, including nationally recognized programs for primary and emergency care, cardiovascular disease, cancer care, orthopedic surgery, neurology and neurosurgery, womens health, autoimmune diseases, diabetes and more. AHN employs approximately 21,000 people, has more than 2,600 physicians on its medical staff and serves as a clinical campus for Drexel University College of Medicine and the Lake Erie College of Osteopathic Medicine.

Safe Harbor for Forward-Looking and Cautionary Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, that are subject to risks and uncertainties and are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by their use of terms such as anticipate, believe, confident, could, estimate, expect, intend, may, plan, predict, potential, project, target, will and other similar terms. Such forward-looking statements are based upon current plans, estimates and expectations that are subject to risks and uncertainties that could cause actual results to materially differ from those described in the forward-looking statements. The inclusion of forward-looking statements should not be regarded as a representation that such plans, estimates and expectations will be achieved. Readers are cautioned not to place undue reliance on the forward-looking statements contained herein, which speak only as of the date hereof. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law. Risks and uncertainties that could cause our actual results to materially differ from those described in the forward-looking statements include (i) expectations for future clinical trials, the timing and potential outcomes of clinical studies and filings and other interactions with regulatory authorities; (ii) the impact of legislative, regulatory, competitive and technological changes; (iii) AHNs ability to successfully launch its UAGT technology with our microbubble as a commercial product; and (iv) the risk and uncertainties discussed in our filings with the Securities and Exchange Commission (including those described in the Risk Factors section in our Annual Reports on Form 10-K and our Quarterly Reports on Form 10-Q).

1Pinna R, Campus G, Cumbo E, Mura I, Milia E. Xerostomia induced by radiotherapy: an overview of the physiopathology, clinical evidence, and management of the oral damage. Ther Clin Risk Manag. 2015; 11:171-188.2https://clinicaltrials.gov/ct2/show/NCT02446249 3Wang Z, Zourelias L, Wu C, Edwards PC, Trombetta M, Passineau MJ. Ultrasound-assisted nonviral gene transfer of AQP1 to the irradiated minipig parotid gland restores fluid secretion. Gene Therapy. 2015; 22:739-749.

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Lantheus Grants Allegheny Health Network Exclusive Rights for the Use of its Microbubbles in Combination with Ultrasound Assisted Gene Therapy for the...

Recommendation and review posted by Bethany Smith

New fund to support regenerative medicine discoveries with high potential impact for patients

TORONTO and MISSISSAUGA, ON, April 29, 2021 /CNW/ - New regenerative medicine discoveries, including cell and gene therapies, will never reach patients without access to the funding, expertise and other specialized support that are required to move along the commercialization pathway. To address this need, CCRM, a leader in developing and commercializing regenerative medicine-based technologies and cell and gene therapies, and Amgen, a global leader in the biotechnology industry, are announcing a multi-year fund, for early-stage regenerative medicine-based technologies and therapies to benefit patients and the health-care system. The collaboration is made up of equivalent investments from CCRM and Amgen.

To bridge the gap from bench to bedside, the program will identify, develop and commercialize promising technologies and therapies arising from research conducted in institutions that form CCRM's global network. CCRM and Amgen's contributions will range from financial support to in-kind technical services and expertise.

"CCRM's collaboration with Amgen to create this fund is a perfect example of how public-private partnerships can leverage resources and expertise to support development and commercialization, and change patients' lives," said Michael May, President and CEO, CCRM. "The regenerative medicineresearch ecosystemacross Canada offers a rich discovery pipeline and it is ready for such a program."

"There are few places in the world that have clustered all the necessary resources and talent to drive regenerative medicine from the bench to the bedside. Canada has consistently led the way for decades," said Alan Russell, Vice-President, Research, Amgen Inc."Amgen is delighted to have the opportunity to partner with CCRM and leverage an extraordinary platform to benefit patients."

To identify opportunities, a Joint Steering Committee, made up of representatives from both CCRM and Amgen, will assess proposals from CCRM's Canadian and international member institutions. Selected projects will possess high scientific merit, be developed by scientists and academics with established credentials and expertise, and demonstrate the greatest potential to have an impact in the industry.

Regenerative medicine, including cell and gene therapy, harnesses the power of (stem) cells, biomaterials, molecules and genetic modification to repair, regenerate or replace diseased cells, tissues and organs. It has the promise of creating revolutionary new treatments for devastating and costly conditions such as heart disease, diabetes and cancer.

About CCRM CCRM is a global, public-private partnership headquartered in Canada. It receives funding from the Government of Canada, the Province of Ontario, and leading academic and industry partners. CCRM supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding, and infrastructure. CCRM is the commercialization partner of the University of Toronto's Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About Amgen in CanadaAs a leader in innovation, Amgen Canada understands the value of science. With main operations located in Mississauga, Ontario's vibrant biomedical cluster, and its research facility in Burnaby, B.C., Amgen Canada has been an important contributor to advancements in science and innovation in Canada since 1991. The company contributes to the development of new therapies and new uses for existing medicines in partnership with many of Canada's leading healthcare, academic, research, government and patient organizations. To learn more about Amgen Canada, visitwww.amgen.ca.

SOURCE Amgen Canada

For further information: CCRM: Stacey Johnson, Director, Communications and Marketing, 647-309-1830, [emailprotected]; Amgen Canada: Natasha Bond, Head of Corporate Affairs, 905-285-3007, [emailprotected]

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CCRM and Amgen partner to advance emerging medical innovations - Canada NewsWire

Recommendation and review posted by Bethany Smith

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data presentations at the upcoming 24thAmerican Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 11-14, 2021. Investigators will review new data from Rockets Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Fanconi Anemia (FA) gene therapy programs in oral and poster presentations.

Details for oral presentations are as follows:

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim ResultsSession: Genetic Blood and Immune DisordersPresenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesDate: Tuesday May 11, 2021Time: 6:15-6:30 p.m. EDTLocation: Room 7Abstract number: 39

Title: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric PatientsSession: Gene Therapies for HemoglobinopathiesPresenter: Jos Luis Lpez Lorenzo, M.D., Hospital Universitario Fundacin Jimnez Daz, Madrid, SpainDate: Wednesday May 12, 2021Time: 6:45-7:00 p.m. EDTLocation: Room 7Abstract number: 83

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene Therapy Presenter: Juan A. Bueren, Ph.D., Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT) in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday, May 13, 2021Time: 10:00-10:45 a.m. EDTLocation: Room 7Abstract number: 36

Select results from Dr. Buerens presentation will also be highlighted by Paula Rio, Ph.D. Details for this Invited Presentation are as follows:

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene TherapyPresenter: Paula Ro, Ph.D., Senior Researcher, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday May 13, 2021Time: 10:00-11:45 a.m. EDT

Details for poster presentation are as follows:

Title: Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical TrialsSession: Hematologic and Immunologic DiseasesPresenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineDate: Tuesday, May 11, 2021Time: 8:00-10:00 a.m. EDTLocation: Digital GalleryAbstract number: 697

Abstracts for the presentations can be found online at: https://annualmeeting.asgct.org/

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Upcoming Clinical Data Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -...

Recommendation and review posted by Bethany Smith

LONDON, April 27, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline), a clinical-stage biotechnology company developing transformative gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that the Company will give six poster presentations at the American Society of Gene and Cell Therapy Annual Meeting 2021, taking place May 11 14.

We are excited to be presenting six posters at the upcoming ASGCT conference, which together highlight the scientific foundation that underlies our gene therapy programs and broader platform technology, said Theresa Heggie, CEO of Freeline. These posters are reflective of steady progress at Freeline, which we expect will include three gene therapies in the clinic by year end.

Poster Presentation Details

#329:FLT201, a Novel Investigational AAV-Mediated Gene Therapy Candidate for Gaucher Disease Type 1Presenter: RomualdCorbau, PhD, Chief Scientific Officer

#509:GLA Uptake and Metabolic Cross Correction in Fabry Disease Relevant Cell Lines: A Rationale for Liver-Directed AAV Gene TherapyPresenter:Jey Jeyakumar, PhD, Scientific Director

#821:Development of a 96-Well Plate-Based High-Throughput System forrAAVManufacturing Platform Optimization and Candidate SelectionPresenter: BettinaPrieler, PhD Student Technology Development

#843:Development and Scale Up of a Suspension Cell-Based AAV Manufacturing ProcessPresenter:Ahmed Youssef, Team Leader USP Development

#878:Defining a Reliable Quantification Assay Strategy for Adeno-Associated Virus (AAV)-Based Gene TherapiesPresenter:FeliciaThoennissen, PhD, Scientist Analytical Development

#894:Development of an Assay to Measure Transduction Efficiency of Adeno-Associated Virus (AAV)-Based Gene TherapiesPresenter:Anita Heinlein, Scientist Analytical Development

Abstracts will be available on the ASGCT website starting today.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally-designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing protein into the patients bloodstream. The Companys integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in Hemophilia B and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia A. Freeline is headquartered in the UK and has operations in Germany and the US.

Forward-Looking Statements

This press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Companys opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include discussion of the Companys research, pipeline and clinical trial plans. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review; the Companys ability to design and implement successful clinical trials for its product candidates; the potential for a pandemic, epidemic or outbreak of infectious diseases in the US, UK or EU, including the COVID-19 pandemic, to disrupt the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with the U.S. Securities and Exchange Commission. You may get these documents by visiting EDGAR on the SEC website at http://www.sec.gov.

Contact

David S. ArringtonVice President Investor Relations & Corporate CommunicationsFreeline Therapeuticsdavid.arrington@freeline.life+1 (646) 668 6947

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Freeline to Present Data at the American Society of Gene and Cell Therapy Annual Meeting 2021 - GlobeNewswire

Recommendation and review posted by Bethany Smith

CRISPR Technology Market: Introduction

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CRISPR Technology Market: Rise in focus on gene therapeutics is projected to contribute to the growth of the market - BioSpace

Recommendation and review posted by Bethany Smith

The exciting potential of immunotherapy for cancer treatment continues its exploration and here, Drug Target Review investigates three of the latest pre-clinical developments in immuno-oncology research.

Scientists at McMaster University, Canada, have shown in new research that changing the metabolism of natural killer (NK) immune cells allows them to overcome the hostile conditions found inside tumours and destroy advanced ovarian and lung cancer.

In this study, we discovered that the metabolism, or energy hub, of NK cells is paralysed by tumours, causing the NK cells to undergo an energy crisis and lose their tumour-killing functions, said Sophie Poznanski, a PhD student and lead author of the study. With that understanding, we were able to reverse the dysfunction of NK cells by repurposing a pre-existing metabolism drug that restored their energy production.

Published in Cell Metabolism, the study shows that NK cells can also be modified to mimic the metabolism of tumours. These modified NK cells proved to be far better adapted for the hostile tumour environment, the team found.

We were just hoping that the modified NK cells would better resist suppression in tumours. We were astounded to see that not only did they show no suppression, but they paradoxically functioned better inside of the tumour than outside of it, said Poznanski.

This is the first report of an antitumour immune cell that exploits the hostility of tumours for their own advantage, said senior author Professor Ali Ashkar. Generating cytotoxic immune cells to have tumour-like metabolism is key for their antitumour functions in a very hostile environment of a solid tumour. This could be a paradigm shift for immune cell-based cancer immunotherapy.

So far, NK cells have only proven effective against blood cancers. However, re-programmed and trained NK cells could afford patients with otherwise terminal cancers with a safe and effective treatment option. Furthermore, immunotherapy with NK cells has already proven safe with few, if any, side effects.

This research revealed a novel targeted immunotherapy approach that employs new antibodies against genetically altered proteins to target cancers. The technique was developed at the Johns Hopkins Kimmel Cancer Center, US.

The researchers targeted their immunotherapy approach to alterations in the common cancer-related p53 tumour suppressor gene, the RAS tumour-promoting oncogene or T-cell receptor genes. Their findings are reported in three related studies published inScience Immunology, Science and Science Translational Medicine.

Although common across cancer types, p53 mutations have thus far not been successfully targeted with drugs. Genetic alterations in tumour suppressor genes often result in their functional inactivation.

Traditional drugs are aimed at inhibiting proteins. Inhibiting an already inactivated tumour suppressor gene protein in cancer cells, therefore, is not a feasible approach, said Dr Emily Han-Chung Hsiue, lead author on theSciencepaper.

Instead of drugs, the researchers set out to target these gene alterations with antibodies. Conventional antibodies require an antigen target on the cell surface most commonly a protein that looks like a foreign invader to the immune system. However, the proteins produced by mutant oncogenes and tumour suppressor genes are inside the cells, out of reach from conventional antibodies. However, proteins are routinely degraded within cells, generating protein fragments called peptides.

These peptides can be presented on the cell surface when complexed with the human leukocyte antigen (HLA) proteins, saidKatharine Wright, postdoctoral fellow and a lead author on theScience Immunologypaper. Mutated proteins in cancer cells can also be degraded and generate mutant peptides presented by the HLA molecules. These mutant peptide HLA complexes serve as antigens and mark cancer cells as foreign to the immune system.

They developed an approach in the form of bispecific antibodies, comprising one component that specifically recognises cancer cells and another component that recognises immune cells and brings both cells together. In laboratory and animal tumour cell models, it resulted in the destruction of tumour cells.

This therapeutic strategy is dependent on a cancer containing at least one p53 or RAS alteration and the patient having an HLA type that will bind to the mutant peptide to present it on the cell surface, said senior authorAssociate Professor Shibin Zhou, a study leader.

In theScience Translational Medicinepaper, the researchers report that the powerful bispecific antibody approach they developed could also be used for the treatment of T-cell cancers. In animal models, the researchers showed that their approach selectively killed the cancerous T cells while sparing the majority of healthy T cells.

The scientists say the next research steps are to assess whether the strategy can be applied to other gene alterations in p53, KRAS and other cancer driver genes.

We intend to develop a large number of bispecific antibodies that would target such genes, said Alex Pearlman, PhD student and co-author of the three studies. Although any individual bispecific antibody would target a small fraction of cancer patients, a suite of antibodies would allow for the treatment of many patients.

Researchers at Moffitt Cancer Center, US, have investigated how to combine and sequence new therapies to improve survival of patients with advanced melanoma. In a new article published inCancer Immunology Research, the team demonstrated that sequential administration of immunotherapy followed by targeted therapy prolongs antitumour responses in pre-clinical models and may be a potential treatment option for patients.

One of the most common genetic alterations in melanoma are mutations of the BRAF gene, which affect approximately 50 percent of patients. These alterations result in downstream signalling through the protein MEK and stimulation of cell growth, invasion and survival. Several therapies that target both BRAF or MEK have been approved and standard treatment for patients who have BRAF mutations is now combination therapy with BRAF and MEK inhibitors.

Given the success of both BRAF/MEK inhibitors and immunotherapies, researchers have attempted to combine these two classes of anticancer therapies. However, some of these combinations have not always been successful.

Initial attempts to develop targeted therapy/immunotherapy combinations clinically were not successful due to severe toxicity, said Dr Keiran Smalley, lead researcher.

These toxicities led the research team to determine whether using a sequential treatment approach rather than a simultaneous combination approach would lead to durable antitumour activity in BRAF and NRAS-mutated melanoma.

The researchers analysed sequential combinations of immunotherapy followed by either BRAF/MEK-targeted therapy or another targeted therapy combination (ceritinib/trametinib) and vice versa in pre-clinical mouse models. They discovered that immunotherapy followed by targeted therapy was more effective than either treatment approach alone. While using targeted therapy followed by immunotherapy was better than targeted therapy alone, it was not as effective as the immunotherapy-targeted therapy sequence.

The team discovered the immunotherapy-targeted therapy sequence modulated the environment to promote immune cell functions resulting in antitumour activity while simultaneously blocking activity that permitted tumour cell escape from immune detection. They also showed that the immunotherapy-targeted therapy sequence enriched the melanoma cells for proteins that promoted immune cell detection and suppressed signalling pathways associated with drug resistance.

sequential administration of immunotherapy followed by targeted therapy prolongs antitumour responses

Results from this research suggest that sequential treatment approaches with anti-PD-1 agents followed by targeted therapy may be clinically beneficial and several ongoing trials are currently investigating this hypothesis.

Together, our data demonstrates that sustained antitumour responses to targeted therapy are dependent upon a vigorous, sustained immune response and that upfront use of immunotherapy can augment this, said Smalley.

Read more:
New developments in the research field of immuno-oncology - Drug Target Review

Recommendation and review posted by Bethany Smith

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, today announced that the following poster, highlighting a preclinical research collaboration using its ARCUS genome editing platform for treatment of transthyretin amyloidosis (ATTR), will be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, scheduled for May 11-14, 2021.

Title: Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis in animal modelsPoster Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases, Abstract 497Date/Time: Tuesday May 11, 2021 8:00 AM - 10:00 AMPresenting Author: Jenny A. Greig, Ph.D., Senior Director, Gene Therapy Program, Perelman School of Medicine, University of PennsylvaniaCo-Authors: Cassandra Gorsuch2, Joanna K. Chorazeczewski1, Melanie K. Smith1, Thomas Furmanak1, Alexa N. Avitto1, Scott N. Ashley1, Wendy Sharer2, Hui Li2, Jeff Smith2, Peter Clark1, Camilo Breton1, Derek Jantz2, and James M. Wilson1

Transthyretin amyloidosis is a rare disease caused by the progressive accumulation of misfolded transthyretin (TTR) protein into amyloid fibrils, which leads to peripheral neuropathy and/or cardiomyopathy. Research to be presented at the annual ASGCT meeting, led by Dr. Jenny A. Greig at the Perelman School of Medicine, University of Pennsylvania, used an AAV vector for in vivo delivery of ARCUS gene editing nucleases to knock out the TTR gene, which is responsible for ATTR.

With this program, we are excited to continue building a dataset demonstrating in vivo gene editing in large animal models using ARCUS nucleases, said Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of Precision BioSciences. In this study, use of an optimized ARCUS nuclease to knock out the TTR gene was found to be effective in both mice and nonhuman primates, where we observed a good correlation between TTR gene editing in the liver and reductions of TTR in the serum. This approach addresses the root cause of the disease and results in genomic edits that are expected to be permanent. These results continue to demonstrate the power and versatility of ARCUS nucleases, particularly for in vivo editing.

Abstracts for the ASGCT 2021 Meeting are available on the meeting website.

About ARCUSARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision's platform and products are protected by a comprehensive portfolio including more than 75 patents to date.

About Precision BioSciences, Inc.Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Companys pipeline consists of multiple off-the-shelf CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit http://www.precisionbiosciences.com.

Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding statements regarding the potential results, uses and advancement of our in vivo gene editing programs and ARCUS-based gene editing technology, including, without limitation, its attributes and effects upon the transthyretin gene, transthyretin serum levels and transthyretin amyloidosis. In some cases, you can identify forward-looking statements by terms such as aim, anticipate, believe, could, eligible, expect, expected, should, plan, intend, estimate, target, mission, goal, may, will, would, should, could, target, potential, potentially, promising, project, predict, contemplate, potential, or the negative thereof and similar words and expressions.

Forward-looking statements are based on managements current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators development of product candidates; our or our collaborators ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators ability to enroll patients; changes in interim top-line and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

1 Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA2 Precision BioSciences, Inc., Durham, NC, USA

Excerpt from:
Precision BioSciences Announces Poster Presentation at the Upcoming American Society of Gene & Cell Therapy Annual Meeting - Business Wire

Recommendation and review posted by Bethany Smith

As per the research report, Global Gene Therapy Market is expected to reach the market valuation of US$ 20.9 billion by 2027 expanding at a reasonable CAGR of 29.7% during the forecast period (2021-2027) from US$ 3.5 billion in 2019.

Gene therapy is a technique that involves inserting genetic material into cells in order to correct for abnormal genes or produce a desirable protein. The growing number of ongoing clinical trials, as well as significant mergers, acquisitions, and venture capital investments in the gene-therapy field, suggest that the future of such therapies is promising. About 2,600 gene therapy clinical trials have been completed, are in progress, or have been accepted around the world to date. Gene therapy researchers are working more than ever to find a path to the clinic and the market. About two thousand clinical trials in human gene therapy have been published worldwide, and about 20 gene therapies have been approved. Such progress raises the prospect of treating devastating rare and hereditary illnesses, as well as incurable diseases.

For complete research, request for Sample of the report browse through https://univdatos.com/request_form/form/352

As of the end of the first quarter of 2019, 372 gene therapy clinical trials were in development, according to the Alliance for Regenerative Medicines (ARM) Quarterly Regenerative Medicine Global Data Study. Surprisingly, trials in Phase II had the largest margin (217 or 58 percent), followed by Phase I (123 or 33 percent), and Phase III (123 or 33 percent) (32 or 9 percent). The number of gene therapy clinical trials increased by ten from the previous years total of 362 trials. In fact, from 319 trials in progress in Q1 2018, the number of clinical tests has increased by 17% year over year. Many gene therapy clinical trials have focused on treating cancers such as breast, gynaecological, skin, urological, neurological, and gastrointestinal tumours, as well as haematological malignancies and paediatric tumours. According to the International Agency for Research on Cancer, 1 in 5 people will grow cancer over their lifetime, with 1 in 8 men and 1 in 11 women dying from it. According to these recent estimates, nearly 50 million people are living within five years of a previous cancer diagnosis. Globally, ageing demographics and socioeconomic risk factors continue to be major factors driving this increase.

For a detailed analysis of the applications in the Gene Therapy Market browse through https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

Insights Presented in the Report

Amongst vectors, Viral vectors segment holds the major share

The market is divided into viral and non-viral vectors based on the form of vector. Due to major advances in vector engineering, distribution, and protection, viral vectors dominated the industry in 2019 with an 88.6 percent share and are projected to continue their dominance over the forecast era.

Amongst viral vectors, adeno-associated virus vectors are anticipated to dominate the market during the analyzed period

Lentivirus, adeno-associated virus, retrovirus & gammaretrovirus, transformed herpes simplex virus, and adenovirus are the major segments of the viral vector market. Because of the fast penetration into the host genome, no viral genes, ability to transduce cells that are not actively dividing, a wide variety of host cells, and being non-inflammatory and non-pathogenic, the adeno-associated virus sub-segment accounted for a maximum market sales share of 34% in 2019 and is projected to remain dominant throughout the studied timeframe. The non-viral vector, on the other hand, is expected to rise at the fastest rate over the projected period.

Amongst gene type, antigen segment dominated the market during the forecast period

The market is divided into antigen, cytokine, tumour suppressor, suicide, deficiency, antibody, and others based on gene form. The antigen category has the highest market sales share of 19.2 percent in 2019 and is projected to remain dominant over the forecast period.

Amongst indication, oncology segment holds the major share

The industry is divided into oncology, rare disorders, respiratory, neurology, infectious diseases, among others based on indication. Thanks to the prevalence of cancer cases, the oncology segment accounted for the largest market share of 48.6% in 2019 and is projected to be the leading segment of the gene therapy market over the forecast period.

Amongst the delivery method, in-vivo segment dominated the market during the forecast period

The market is primarily divided into in-vivo and ex-vivo delivery methods, depending on the method of delivery. Due to the direct transfer of genes into patients, the in-vivo segment accounted for a maximum market revenue share of 87.5 percent in 2019 and is projected to remain dominant throughout the studied timeframe.

North America represents one of the largest markets of Gene Therapy market

A systematic study of the consumer dynamics of the gene therapy market was performed for various regions around the world, including North America (the United States, Canada, and the Rest of North America), Europe (Germany, France, Italy, Spain, United Kingdom and Rest of Europe), Asia-Pacific (China, Japan, Australia, South Korea, and the Rest of APAC), and the Rest of the World. Because of the high incidence of cancer, the presence of high disposable income, and a rise in support for R&D initiatives associated with gene therapy, North America led the industry and reported sales of US$ 1.7 billion in 2019.

Customization Options:

The Gene TherapyMarket can further be customized as per the requirement or any other market segment. Besides this, UMI understands that you may have your own business needs, hence feel free to connect with us to get a report that completely suits your requirements.

Table of Contents

1 Market Introduction

2 Research Methodology or Assumption

3 Industry Performance

4 Executive Summary

5 Top Start-Ups Under Gene Therapy Sector

6 COVID-19 Impact

7 Market Insights by Vector

8 Market Insights by Gene Type

9 Market Insights by Indication

10 Market Insights by Delivery Method

11 Market Insights by Region

12 Gene Therapy Market Dynamics

13 Legal & Regulatory Framework

14 Demand and Supply Side Analysis

15 Value Chain Analysis

16 Gene Therapy Market Opportunities

17 Gene Therapy Market Trends & Insights

18 Competitive Scenario

19 Company Profiled

20 Disclaimer

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Gene Therapy Market Worth US$ 20.9 billion UnivDatos Industry Analysis- by Size, Share, Growth, Trends, and Forecast 2021-2027 The Courier - The...

Recommendation and review posted by Bethany Smith

The US Food and Drug Administration (FDA) has removed a clinical hold on Dutch biotech uniQures haemophilia B gene therapy, following concerns over a case of cancer in a patient in a pivotal trial.

In December 2020, the FDA placed a hold on uniQures haemophilia B clinical programme following the diagnosis of hepatocellular carcinoma (HCC) a type of liver cancer in a patient in the HOPE-B trial evaluating AMT-061 (etranacogene dezaparvovec).

In a statement, uniQure said that the patient diagnosed with HCC had multiple risk factors associated with this type of cancer. This included a 25-year history of hepatitis C (HCV) as well as a history of hepatitis B (HBV). Chronic infections with both HCV and HBV are associated with around 80% of HCC cases.

Multiple analyses, following a surgical resection of both the patients tumour and adjacent liver tissue, showed that AAV vector integration in the tissue sample was extremely rare, accounting for 0.027% of the cells in the sample.

The integration events that were present were randomly distributed, the company added, with no signs of clonal expansion or any dominant integration event.

Whole genome sequencing of the tumour also confirmed that it had genetic mutations characteristic of HCC, independent of vector integration.

UniQure noted that a gene expression analysis of the tumour and adjacent tissue suggested a precancerous state in the liver that could have predisposed the patient to developing HCC.

Patient safety is our top priority, and we are grateful to our advisors and the FDA for their help in resolving this clinical hold, said Ricardo Dolmetsch, president of research and development at uniQure.

Our comprehensive investigation showed that AMT-061 is very unlikely to have contributed to the HCC in our patient. We look forward to announcing top-line 52-week data from the HOPE-B pivotal trial later this quarter, he added.

UniQure added that all patients in its haemophilia clinical programme have abdominal ultrasounds performed one year after dosing, with patients continuing to receive this test every six months.

AMT-061 has been granted breakthrough therapy designation by the FDA and a priority medicine (PRIME) designation by the European Medicines Agency (EMA).

Visit link:
FDA removes clinical hold on uniQure's haemophilia gene therapy - PMLiVE

Recommendation and review posted by Bethany Smith

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

Sensorion (FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, announced today that it will host a key opinion leader (KOL) webinar on GJB2-Related Genetic Hearing Loss on Monday, May 10, 2021 at 10am Eastern Time.

The event will feature a presentation by KOL Thomas Lenarz, M.D. Ph.D., Medical University of Hannover, who will discuss the clinical aspects, current treatments landscape and unmet medical needs in treating patients with a pediatric onset of GJB2related hearing loss as well as the role of the GJB2 gene. Dr. Lenarz will be available to answer questions following the formal presentations.

Sensorion's management team will also discuss their internal capabilities dedicated to gene therapy development as well as their GJB2-GT gene therapy program, which aims to restore hearing in people living with hearing loss due to mutations in the GJB2 gene in both children and adult populations.

To register for the call, please click here.

Thomas Lenarz, M.D. Ph.D. is Professor of Otorhinolaryngology and Chair of the Department of Otorhinolaryngology at the Medical University of Hannover, Germany, and a pioneer in the development of surgical and pharmacological interventions for hearing loss. Under his leadership, the department has become the center of an internationally recognized world-leading network for diagnosis, treatment, and translational research on hearing loss.

Professor Lenarz and his team have developed the largest international cochlear implantation program in the world. His research activity on surgical approaches to hearing loss extends beyond cochlear implants and includes central auditory implants in the midbrain and brain stem for neural deafness and implantable hearing aids for middle ear and inner ear hearing loss.

Professor Lenarz received his Ph.D. in pharmacology of the auditory system in 1987 before undertaking postdoctoral research at the University of California in San Francisco in 1989. He is currently vice president of the German Society of Biomedical Technology and speaker of the expert group on health care technologies at Acatech (German National Academy of Technology).

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) for sudden sensorineural hearing loss (SSNHL). Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. Sensorion has launched three gene therapy programs, currently at preclinical stage, aimed at correcting hereditary monogenic forms of deafness including deafness caused by a mutation of the gene encoding for Otoferlin, hearing loss related to gene target GJB2 as well as Usher Syndrome Type 1 to potentially address important hearing loss segments in adults and children. The Company is potentially uniquely placed, through its platforms and pipeline of potential therapeutics, to make a lasting positive impact on hundreds of thousands of people with inner ear related disorders, a significant global unmet medical need.

http://www.sensorion.com

Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2020 Half-Year financial report published on October 21, 2020 and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

Read the original post:
Sensorion Hosting Key Opinion Leader Webinar with Dr. Thomas Lenarz on the GJB2 Gene Related Hearing Loss on May 10, 2021 - Business Wire

Recommendation and review posted by Bethany Smith

TheHemophilia Gene Therapy Marketis expected to have a highly positive outlook for the next eight years 2020-2027. This Research Reports emphasizes on key industry analysis, market size, Share, growth and extensive industry dynamics with respect to with respect to drivers, opportunities, pricing details and latest trends in the industry.

The global Hemophilia Gene Therapy Market analysis further provides pioneering landscape of market along with market augmentation history and key development involved in the industry. The report also features comprehensive research study for high growth potential industries professional survey with market analysis. Hemophilia Gene Therapy Market report helps the companies to understand the market trends and future market prospective,opportunities and articulate the critical business strategies.

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The Hemophilia Gene Therapy market analysis report speaks about the growth rate till 2027 manufacturing process, Growth, key factors driving this market with sales, revenue, and price analysis of lead manufacturers of Digital Mapping Cameras Market, distributors, traders and dealers of Hemophilia Gene Therapy.

The comprehensive value chain analysis of the Hemophilia Gene Therapy market will assist in attaining better product differentiation, along with detailed understanding of the core competency of each activity involved. The market attractiveness analysis provided in the report measures the potential value of the Hemophilia Gene Therapy providing business strategists with the latest growth opportunities.

The report studies the historical and present performance of the market to project future growth. For this market report, 2019 is considered as a base year. The historical data was studied for year 2020 to 2021.

Geographically Regions Covered:

Table of Content:

Chapter 1 Research Objective and Assumptions

Research ObjectivesAssumptionsAbbreviations

Chapter 2 Market Purview

Report DescriptionExecutive SummaryCoherent Opportunity Map (COM)

Chapter 3 Global Market Demand Productions Supply Sales Demand Market Status and Forecast

Chapter 4 Market Dynamics, Regulations, and Trends Analysis

RestraintsMarket OpportunitiesRegulatory ScenarioIndustry TrendMerger and AcquisitionsNew Product Approvals/LaunchPromotion and Marketing Initiatives

Chapter 5 Competitive Landscape

Heat Map AnalysisMarket Share AnalysisCompany Profiles

Some Major Players are BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics

Chapter 6 Conclusion

Key Features of the Hemophilia Gene Therapy Market Report:

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Hemophilia Gene Therapy reportcover complete modest view with the market stake and company profiles of the important contestants working in the market. Also it offers a summary of product specification, production analysis, technology, product type, considering key features such as gross, gross margin, revenue & cost structure. The report helps the user to strengthen decisive power to plan their strategic moves to launch or expand their businesses by offering them a clear picture of this market.

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Hemophilia Gene Therapy Market: Global Industry Insights, Growth Prospects, Business Overview, Growth Rate and Forecasts 2027 KSU | The Sentinel...

Recommendation and review posted by Bethany Smith

- CANbridge to lead development and commercialization in China; expands rare disease pipeline

- Mirum is entitled to receive up to an aggregate of $120.0 million in upfront and milestone payments, and significant double-digit tiered royalties

FOSTER CITY, Calif. & BEIJING--(BUSINESS WIRE)-- Mirum Pharmaceuticals (Nasdaq: MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan). Maralixibat, an investigational, orally administered medication, is being evaluated in Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210429005614/en/

Maralixibat targets the apical sodium dependent bile acid transporter (ASBT), ultimately resulting in lower levels of bile acid systemically, which could mediate liver damage. The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for maralixibat for the treatment of cholestatic pruritus in patients with ALGS under priority review. The European Medicines Agency is reviewing maralixibat for the treatment of PFIC2. Mirum has commenced a global Phase 2b maralixibat study (EMBARK) for the treatment of BA.

Under the terms of the licensing agreement, CANbridge has obtained the exclusive right to develop and commercialize maralixibat within the Greater China regions for ALGS, PFIC, and BA. In exchange, Mirum is entitled to receive an $11.0 million upfront payment, R&D funding, and up to $109.0 million for the achievement of future regulatory and commercial maralixibat milestones, with significant double-digit tiered royalties based on product net sales.

In collaboration with Mirum, CANbridge has agreed to oversee Mirums clinical study sites in China, with the goal of accelerating enrollment of the global Phase 2b EMBARK study, which was recently initiated for patients with BA. CANbridge would also have the right to manufacture maralixibat in Greater China under certain conditions.

Maralixibat has the potential to be a transformative medication for certain cholestatic liver diseases and our goal is to ensure its availability to patients globally, said Chris Peetz, president and chief executive officer at Mirum. CANbridge is a leading rare disease company in China and with their track record of commercial success, we believe they will be a strategic partner to accelerate the global launch of maralixibat, if approved.

We are thrilled to add maralixibat to our rare disease portfolio, as we expand into liver diseases and strengthen our pipeline with a late-stage asset in indications for which there are no approved treatments, said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc. We are looking forward to participating in the global development of maralixibat in BA by supporting the China sites for the global Phase 2b EMBARK study, in collaboration with Mirum, and to working closely with Mirum to bring this treatment to patients and families in Greater China, where the need is great.

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirums lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirums marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

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About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of treatments for orphan diseases and rare cancers to address unmet medical needs. CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in rare diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase, an enzyme replacement therapy for the treatment of Hunter syndrome (also known as mucopolysaccharidosis type II), developed by GC Pharma and marketed in more than 12 countries worldwide. CANbridge also has entered into a strategic collaboration and licensing agreement with LogicBio Therapeutics to develop, manufacture and commercialize gene therapy candidates for treatments for Fabry and Pompe diseases. CANbridge also has a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases.

For more on CANbridge Pharmaceuticals Inc., please go to: http://www.canbridgepharma.com.

About Maralixibat

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. Food and Drug Administration has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Until maralixibat is approved and available for prescribing, the medication is available to patients with ALGS through Mirums expanded access program. For more information, please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential development and commercialization by CANbridge of maralixibat in Greater China, Mirums receipt of milestone and royalty revenue in connection with the license agreement with CANbridge, and the regulatory approval pathway for maralixibat. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as will, could, would, potential and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirums current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirums business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirums filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on managements assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210429005614/en/

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Mirum Pharmaceuticals and CANbridge Pharmaceuticals Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat in Greater China...

Recommendation and review posted by Bethany Smith

The emergence of efficacious human gene therapy for life-limiting genetic diseases such as spinal muscular atrophy type 11 has raised hopes among families of children with previously incurable genetic disorders. However, a number of concerns have surfaced regarding the gene therapy trial process. Two bereaved parents, both deeply engaged in their disease communities, collaborated with an experienced gene therapy researcher to create patient education materials to aid parents in critical decision-making. In this Family Partnerships article, we present a summary of the module, as well the reflections of each of the 3 contributors describing the impetus for the Courageous Parents Network (CPN) unit Evaluating the Clinical Trial Option, and their hopes for its impact, as captured in an interview conducted after completion of the educational module.

The Evaluating the Clinical Trial Option unit (https://courageousparentsnetwork.org/topics/clinical-trial) offers an overview of the fundamental elements of clinical trial participation, written for a nonmedical audience to help families understand and evaluate their options. It is disease and therapy agnostic and does not advocate for any particular path or decision. Content areas include a glossary of terms; informed consent; clinical trial basics, such as efficacy; trial design, including the phases; participation criteria; and the emotional impact of decision-making. Its elements include a variety of media types: 85 short videos (average 24 minutes each, a total of 240 minutes); downloadable guides in English and Spanish; 3 podcasts; 4 blog posts written by parents; and a curated educational digital experience (Guided Pathway) that, through videos and text, walks parents

Address correspondence to Terence R. Flotte, MD, Medical School, University of Massachusetts and Deans Office, 55 Lake Ave North, Worcester, MA 01655. E-mail: terry.flotte{at}umassmed.edu

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Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives - American Academy of Pediatrics

Recommendation and review posted by Bethany Smith

The global gene therapy market is estimated to grow from USD 3.8 billion in 2021 to USD 13.0 billion by 2027, at a CAGR of 27.8%.

TheGene Therapy Marketresearch report thoroughly explains each and every aspect related to the Global Gene Therapy Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business.

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Scroll down 100s of data Tables, charts and graphs spread through Pages and in-depth Table of Content on Gene Therapy Market, By Vector (Non-Viral Vectors, Viral Vectors), Indication (Neurological Diseases, Cancer, Duchenne Muscular Dystrophy, Hepatological Diseases, Other Indications), Delivery Method ( In Vivo, Ex Vivo) and Geography Global Forecast to 2026. Early buyers will get 10% customization on study.

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The global gene therapy market is estimated to grow from USD 3.8 billion in 2021 to USD 13.0 billion by 2027, at a CAGR of 27.8%. The growth of this market is majorly driven by the high incidence of cancer and other target diseases, availability of reimbursements, and the increasing funding for gene therapy research. However, the high cost of gene therapies is expected to hamper market growth to a certain extent during the forecast period.

The demand for gene therapies for the treatment of cancer is estimated to grow at a high rate during the forecast period

Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The cancer segment is estimated to grow at the highest CAGR during the forecast period owing to the approval of a growing number of gene therapies for the treatment of cancer and the rising incidence of hematologic cancers.

Non-viral vectors segment accounted for the largest share of the gene therapy market, by vector, in 2018

The market, by vector, has been segmented into viral and non-viral vectors. The non-viral vectors segment accounted for the largest share of the market in 2018. This can be attributed to the high market penetration of oligonucleotide-based non-viral vector gene therapies.

North America is the largest regional market for gene therapies

The global market is segmented into four major regions, namely, North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the market, followed by Europe. The rising prevalence of chronic diseases, high and growing healthcare expenditure, presence of advanced healthcare infrastructure, availability of reimbursements, and the presence of major market players in the region are the major factors driving the growth of the gene therapy market in North America.

Key Market Players

The prominent players operating in the gene therapy market include Biogen (US), Novartis AG (Switzerland), Gilead Sciences, Inc. (US), Spark Therapeutics, Inc. (US), MolMed S.p.A. (Italy), Orchard Therapeutics plc. (UK), SIBIONO (China), Shanghai Sunway Biotech Co., Ltd. (China), bluebird bio, Inc. (US), Human Stem Cells Institute (Russia), AnGes, Inc. (Japan), Alnylam Pharmaceuticals, Inc. (US), Sarepta Therapeutics (US), Jazz Pharmaceuticals, Inc. (Ireland), Akcea Therapeutics (US), and Dynavax Technologies (US).

Biogen was the leading player in the market in 2018. Strong business operations across all key regions, coupled with a strong brand image, have made Biogen a leading player in this market. Biogen adopted organic and inorganic growth strategies, such as agreements & collaborations and acquisitions, to maintain its leading position in the market. For instance, in 2017, Biogen entered into a collaboration with Ionis Pharmaceuticals to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy. In 2019, the company received the NMPA (National Medical Products Association) approval in China for SPINRAZA, which is used for treating 5q spinal muscular atrophy.

Recent Developments

What Porters Five Forces of Competitive Analysis Provides?

Competitive rivalry:-The main driver is the number and capability of competitors in the market. Many competitors, offering undifferentiated products and services, will reduce market attractiveness.

Threat of substitution:-Where close substitute products exist in a market, it increases the likelihood of customers switching to alternatives in response to price increases. This reduces both the power of suppliers and the attractiveness of the market.

Threat of new entry:-Profitable markets attract new entrants, which erodes profitability. Unless incumbents have strong and durable barriers to entry, for example, patents, economies of scale, capital requirements or government policies, then profitability will decline to a competitive rate.

Supplier power:-An assessment of how easy it is for suppliers to drive up prices. This is driven by the: number of suppliers of each essential input; uniqueness of their product or service; relative size and strength of the supplier; and cost of switching from one supplier to another.

Buyer power:-An assessment of how easy it is for buyers to drive prices down. This is driven by the: number of buyers in the market; importance of each individual buyer to the organisation; and cost to the buyer of switching from one supplier to another. If a business has just a few powerful buyers, they are often able to dictate terms.

Five forces analysis helps organizations to understand the factors affecting profitability in a specific industry, and can help to inform decisions relating to: whether to enter a specific industry; whether to increase capacity in a specific industry; and developing competitive strategies.

The country section of the report also provides individual market impacting factors & changes in regulation in the market domestically that impacts the current & future trends of the market. Data points such as consumption volumes, production sites & volumes, import export analysis, price trend analysis, cost of raw materials, down-stream & upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence & availability of global brands & their challenges faced due to large or scarce competition from local & domestic brands, impact of domestic tariffs & trade routes are considered while providing forecast analysis of the country data.

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We offer services that are most relevant and beneficial to the users, which help businesses to sustain in this competitive market. Our detailed and in-depth analysis of the markets catering to strategic, tactical, and operational data analysis & reporting needs of various industries utilize advanced technology so that our clients get better insights into the markets and identify lucrative opportunities and areas of incremental revenues.

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Gene Therapy Market 2021-2026 to Witness High Growth in Near Future and Competitive Analysis | Leading Players Abeona Therapeutics, Audentes...

Recommendation and review posted by Bethany Smith

ALISO VIEJO, Calif.--(BUSINESS WIRE)--The international rare disease patient advocacy organization, Global Genes, is pleased to announce the 6th Annual RARE Drug Development Symposium (RDDS) in partnership with the Orphan Disease Center of the University of Pennsylvania. The June 9-11 virtual event will connect and educate hundreds of advocates, clinicians, and academic and industry researchers to explore the latest science, opportunities, and challenges to the advancement of therapies for more than 7,000 rare diseases.

New to this year's event is an optional preconference workshop targeted to attendees who are in the earlier stages of their research efforts or careers and looking to build knowledge in core competencies that will be expanded upon in the main RDDS program. The main program will address the current landscape of rare drug development and allow attendees to interact with subject matter experts and other rare disease stakeholders to help them better understand and develop their impact and role in advancing potential treatments.

This year, the RDDS keynote speaker will be David Fajgenbaum, M.D., MBA, MSc, co-founder and executive director of the Castleman Disease Collaborative Network (CDCN), assistant professor of medicine in Translational Medicine & Human Genetics at the University of Pennsylvania, associate director, Patient Impact for the Orphan Disease Center of the University of Pennsylvania, and author of the national bestselling book, Chasing My Cure: A Doctors Race to Turn Hope Into Action.

Dr. Fajgenbaum has been a leader in helping researchers to prioritize treatments for COVID-19 clinical trials and inform patient care through the CORONA (COvid19 Registry of Off-label & New Agents) Project. Were thrilled to have him share his insights during this critical time in health care and understand how we can apply these approaches to accelerate progress in rare disease research and treatments, said Craig Martin, CEO at Global Genes. The rare disease leaders who will be featured during the RDDS have tremendous depth of knowledge to share, and we look forward to sharing it with members of the rare community during this event.

RDDS will continue to host the CureAccelerator Live! For Rare Diseases 2021 event on June 10 in partnership with Cures Within Reach, a not-for-profit organization exclusively dedicated to using the speed, safety, and cost-effectiveness of already approved drugs, devices, diagnostics, nutraceuticals, and combination products to impact patients with unmet medical needs driving more treatments to more patients more quickly.

The emergence of therapeutic platforms creates unprecedented opportunities for treatments to improve the lives of those living with rare diseases, said Jim Wilson, M.D., Ph.D., director, Gene Therapy Program, and Rose H. Weiss, professor and director at the Orphan Disease Center of the University of Pennsylvania. We are delighted to collaborate with Global Genes to educate the rare disease community on research directed to these treatments.

Thank you to our gold sponsors, Horizon Therapeutics and Greenwich Biosciences, and silver sponsor, Pfizer, Inc., for their generous support of this important event.

For more information, visit http://www.globalgenes.org/rdds.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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Global Genes and Orphan Disease Center of the University of Pennsylvania to Convene Patient Group Leaders, Researchers, Clinicians and Industry for...

Recommendation and review posted by Bethany Smith

Professor Naoto Hirano of the Temerty Faculty of Medicine and collaborators have developed a new technology that rigorously and robustly identifies the immune cells that are capable of recognizing and eliminating cancer cells.

The findings, published in Nature Biotechnology, pave the way for novel immunotherapies to help more patients, regardless of their genetic ancestry, live longer and healthier lives.

Adoptive cell therapy, a promising cancer treatment that uses our immune system to eliminate cancer cells, is effective only for a small subset of individuals with specific types of cancer and specific inherited genes. The new technology developed by Hirano, a professor in the department of immunology, allows researchers to develop new immunotherapies for cancer patients that are not limited by the differences or heterogeneity of tumour cells, expanding the potential impact of immunotherapy for patients around the world.

The technology applies to an immunotherapy approach called T cell receptor (TCR) gene therapy that is based on genetically-engineered immune cells (T cells) recognizing and binding to specific molecules, called peptide-loaded human leukocyte antigens (HLA), on the surface of cancer cells. Although there has been progress in TCR therapy, there are more than 28,000 different variations of HLA found in humans and current TCR therapies only work for a few of these variations.

Historically, TCR treatments have been developed for those who had the most common and well-studied HLA alleles, which often meant that these immunotherapies only worked for people from Caucasian ancestry, says Hirano, who is also a senior scientist at the Princess Margaret Cancer Centre and Ontario Institute for Cancer Research clinician scientist.

It was an important goal for us to develop a technology that could work for a broad range of HLA alleles. Were proud of what we developed because it could help many more cancer patients in the future.

The technology presented in this study involves a methodology that can in a single step, at a low expense form a functional protein structure, called a dimer, that is composed of any peptide and HLA molecule, regardless of type, and can bind to and identify a variety of T cells. The method improves the binding affinity between T cells and HLA molecules nearly 200-fold relative to prior methods, which could allow researchers to better identify and engineer the T cells for novel immunotherapies.

The technology has been licensed to TCRyption Inc., a company co-founded by Hirano,for further development, translationand large-scale implementation. In the future, it may be applied to fields other than cancer research and care, including autoimmune diseases such as rheumatoid arthritis and type 1 diabetes.

Im grateful for the cancer research communitys support over the years, which has enabled me to focus on important and challenging issues, says Hirano, who was named the University Health Networks Inventor of the Year last year for developing these analysis techniques. Only with the support for rigorous experimentation, deep expertise, and innovative thinking, were we able to make this breakthrough.

This article was originally published by the Ontario Institute for Cancer Research.

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New technology could allow more cancer patients to benefit from immunotherapy: U of T researchers - News@UofT

Recommendation and review posted by Bethany Smith

VANCOUVER, Wash.--(BUSINESS WIRE)--Wisdom Health Genetics, the world leader in pet genetics and maker of the WISDOM PANEL dog DNA test, announced the publication of two studies in Human Genetics conducted in partnership with the University of Helsinki.

Both studies discovered genetic variants related to disease in dog breeds of Finnish origin: the first study identifies a variant associated with an endocrine disease in Karelian Bear Dogs, and the second identifies a variant associated with an eye disease in Lapponian Herders.

By analyzing genetic samples including those from the Wisdom Health Genetics database, our researchers and Professor Hannes Lohis team at the University of Helsinki were able to identify genetic variants associated with two inherited diseases found in Finnish dog breeds, said Jonas Donner, Ph.D., Discovery Manager at Wisdom Health Genetics. We are thrilled that we could aid in these discoveries, both of which have practical implications for humans and dogs. These findings will inform the development of genetic tests for the conditions, thus improving veterinary diagnostics and breeding plans, and also help further our understanding of similar conditions in humans.

The first study, Intronic variant in POU1F1 associated with canine pituitary dwarfism, established a novel canine model for endocrine dysfunction known as combined pituitary hormone deficiency (CPHD). Through a combination of genome-wide association and next-generation sequencing studies, researchers identified a candidate gene (POU1F1) containing a disease-predisposing genetic variant present in the dogs with CPHD.

Thirty genes, many of which are regulatory genes affecting pituitary development, have been linked to human hereditary hypopituitarism. Impairment occurs not only in humans but also in mice and dogs. This is only the second and thus very important new dog model for human pituitary disease, said Professor Lohi about the discovery in the Universitys news release about the CPHD publication.

In a second study, A missense variant in IFT122 associated with a canine model of retinitis pigmentosa, researchers identified a recessive genetic variant (IFT122) as the likely cause of progressive retinal atrophy (PRA) in Lapponian Herders. The IFT122 gene has not previously been linked to retinal degeneration in mammals, thus establishing a new model to examine the role of IFT122 in degenerative eye diseases in both humans and dogs alike.

Gene discovery is important because genetic testing can now easily distinguish retinal degenerations associated with different genes in breeds, which is important for disease monitoring, prognosis, and the development of new therapies, said Maria Kaukonen, DVM, in the Universitys news release announcing the PRA publication. Diagnostics will improve and it will make the work of veterinarians easier.

The discoveries of both studies will contribute to the development of targeted genetic screening for dogs like those found in Wisdom Panel tests to improve breeding practices and personalized veterinary care.

Additionally, both discoveries hold promise for advancing human medicine thanks to the novel clues about disease gene biology they provide, and the newly characterized canine models enabling further studies of the conditions.

About the combined pituitary hormone deficiency (CPHD) study:

About the canine model of retinitis pigmentosa (RP) study:

About Wisdom Health Genetics

The mission of the Wisdom Health business, a division of Kinship Partners, Inc, is to strengthen the bond between pets and their people through world-leading insights powered by DNA. Wisdom Panel dog DNA testsbacked by WISDOM HEALTH scientific researchcan help pet parents plan better, care smarter, and love longer. For more than a decade, Wisdom Health scientific research has contributed to state-of-the-art genetic tests for companion animals, revolutionizing personalized pet care. By unlocking the secrets of their dog or cat's DNA, pet parents and veterinarians can work together to tailor wellness programs that fit the one-of-a-kind needs of their pets. More than 7,000 veterinarians worldwide recommend and offer Wisdom Panel products. For more information, visit http://www.wisdompanel.com, and follow the Wisdom Panel brand on Facebook and Instagram.

About Kinship Partners, Inc

Kinship is here to help everyone pet parent like a pro. Why? Because our pets make us better humans, and we owe them the best possible care. As allies to pet parents learning on the job, we use our data, products, and services to help people be the best pet parents they can be. We unite changemakers in pet care to break down barriers, open new doors, share insights, and advance our collective knowledge. By reimagining the pet parenting experience and upping peoples confidence, were helping the world find better ways to care.

Our coalition includes our world-leading Wisdom Panel genetic health screening and DNA testing for dogs, the award-winning WHISTLE GPS dog tracker and health monitor, Pet Insight Project, our ground-breaking science stream that uses AI to turn billions of data points into actionable insights, and partnerships like our Leap Venture Studio accelerator that supports innovators and start-ups, to bring new solutions to pet parents. Kinship is a division of Mars Petcare. Learn more at http://www.kinship.co.

About Mars Petcare

Part of Mars, Incorporateda family-owned business with more than a century of history-making diverse products and offering services for people and the pets people lovethe 85,000 Associates across 50+ countries in Mars Petcare are dedicated to one purpose: A BETTER WORLD FOR PETS. With 85 years of experience, our portfolio of almost 50 brands serves the health and nutrition needs of the worlds petsincluding brands PEDIGREE, WHISKAS, ROYAL CANIN, NUTRO, GREENIES, SHEBA, CESAR, IAMS, and EUKANUBA, as well as the Waltham Petcare Science Institute, which has advanced research in the nutrition and health of pets for over 50 years. Mars Petcare is also a leading veterinary health provider through an international network of over 2,000 pet hospitals and diagnostic services including BANFIELD, BLUEPEARL, VCA, Linnaeus, AniCura, and Antech. Were also active in innovation and technology for pets, with Wisdom Panel genetic health screening and DNA testing for dogs, the WHISTLE GPS dog tracker, LEAP VENTURE STUDIO accelerator, and COMPANION FUND programs that drive innovation and disruption in the pet care industry. As a family business guided by our principles, we are privileged with the flexibility to fight for what we believe inand we choose to fight for our purpose: A BETTER WORLD FOR PETS.

Excerpt from:
Novel Genetic Variants Discovered in Two Studies of Inherited Disease in Finnish Dog Breeds - Business Wire

Recommendation and review posted by Bethany Smith

In examining the proportion of patients in the Caribbean with breast or ovarian cancers who carry actionable pathogenic variants, a unique landscape of genetic mutations was uncovered across 7 island nations, according to Sophia George, PhD, and Judith E. Hurley, MD.

We started off in the Bahamas, but we ended up in Haiti, Jamaica, Barbados, Trinidad and Tobago, Dominica, and the Cayman Islands, Hurley said. What we realized was that each island is very unique and very individual, even in the types of breast cancer and the genetic mutations that are found. This is important in the Caribbean, but it is also really important here, in the United States. When you emigrate to another country, you bring your genes with you; [approximately] 1 in 10 immigrants in the United States are from the Caribbean, so this is a big issue that needs to be recognized.

In an interview with OncLive, George, a research assistant professor at Sylvester Comprehensive Cancer Center of the University of Miami Health System, and Hurley, an oncologist at Jackson Memorial Hospital, discussed the importance of the genetic association study, challenges faced with launching such an effort, the key findings from the research, and the clinical implications of the data in the Caribbean as well as the United States.

Hurley: We both live and work in Miami, [which] is really, geographically, part of the Caribbean.It all started with a clinical observation that the Bahamian patients with breast cancer that I saw in my clinic at the University of Miami in the Jackson Memorial Hospital were very young. This led me to ask, 'Well, why is that?'

We discovered that Bahamian women had a very high incidence of inherited breast cancer. When we started to speak with other oncologists in the Caribbean, they said, 'The same thing that is happening in the Bahamas, is happening on my island. Why don't you come to my island? We basically [focused on] 7 islands in the Caribbean and spent many years accruing patients to the study.

George: For me, as a person from the Caribbean, clearly my interest was a bit self-serving. Also, at the time, I was studying hereditary ovarian cancer. I learned about the initial BRCA testing in the Bahamas through a colleague [who was also] in the Bahamas while I was there. This led to me reaching out to Dr Hurley, where I asked her about the other islands, and [she told me] that we were actually going to [look at] the others.

Hurley: The black woman that you are seeing in your practice with a new diagnosis of breast cancer could easily be from the Caribbean, and you do not even know it or have not [considered] it. [Also, perhaps] you do not realize that she [may have] a very high rate of inherited breast cancer. For example, we found that 24% of women in the Bahamas who had breast cancer had a deleterious mutation, which is the highest rate in the world. Before that, it was the Ashkenazi Jewish women, at 12%. This changed the way that Bahamian women are treated in the Bahamas and how they are screened. It should change the way that your patients who are Bahamian are screened for breast cancer.

George: The objective was really straightforward. [We wanted to identify] the prevalence of inherited breast cancer and ovarian cancer in the Caribbean. It really stemmed from observations in the clinic that young black woman from the Caribbean, at that point, were being diagnosed with triple-negative breast cancer, advanced stage [disease], and often with a family history, when probed. [Due to the fact that] we knew about what was happening in the Bahamas, we wanted to know [whether we would see] the same [thing across] all the islands. That is why we included different populations of Caribbean women in this particular study.

Hurley: We ended up gathering a lot of collateral information, like their fertility factors, family histories, how their breast cancer was diagnosed, and what stage their breast cancer was. Although our primary goal was to look at inherited risk, we collected a lot of other data that showed us that over 90% of the women in the Caribbean who were diagnosed with breast cancer were diagnosed because they felt the mass themselves.

[Knowing this] really changes the way that you need to deal with the epidemic of breast cancer in the Caribbean. Breast cancer is a leading cause of cancer death in Caribbean women. [About] 40 million people [reside] in the Caribbean, so you are talking about a population of [approximately] 20 million women; it is a significant population.

George: [Due to the fact] we were looking at germline DNA, we decided to collect saliva DNA from consenting study participants instead of blood because it was easier to manage, easier to transport, and also was more acceptable for the population. Initially, in the study, we looked at BRCA1 and BRCA2 [mutations] across the participants. As the study progressed, it became cheaper to do more panel testing. Initially, it was BRCA1/2 next-generation sequencing with multiplex ligation-dependent probe amplification; then, we included PALB2 and RAD51C. We then did the panel test in a larger cohort of women.

Hurley: The technology really evolved over the course of the study. When we first started, the BRCA1 and BRCA2 tests costed $3500 to test 1 person. By the time we were done, we could do 40 genes for $199. The improvement in technology over the course of this study did not change results that much. Once we went back, and panel tested everyone, we did not find a single Lynch mutation in over 1000 patients; that was kind of surprising. Presumably, there is Lynch syndrome in the Caribbean, but it does not appear to manifest as breast cancer. We were expecting to pick up a few [cases of] Lynch [syndrome] that we had not known about, but we did not.

Hurley: Although many countries in the Caribbean do not have tumor registries, the countries that do, [such as] Trinidad and Tobago and Jamaica, will show you that the average age of their patients is relatively young compared with the United States population. The average patient from the United States who has breast cancer is a white woman who is 62 years of age; in Trinidad, it is a 50-year-old black woman; and in Jamaica, it is a 49-year-old black woman. All very different pictures. Breast cancer is postmenopausal in the United States, and it is premenopausal in the Caribbean.

We also looked at the body mass index of our patients, which was high. We looked at parity, age of menopause and menarche, and other fertility factors. Interestingly, [when looking at] known people who were alive [over the course of the study], the grandmothers vs the mothers vs the daughters, [we saw that] family size markedly decreased and the age of first pregnancy markedly increasedall of this epidemiologically impacts breast cancer risk.

[We have seen] a transition from a third-world fertility pattern to a first-world fertility pattern during the course of our lives, which is fascinating. There has also been a major change in diet patterns during that exact same period of time. All these [factors] impact breast cancer risk, so it may be that as the Caribbean has gone from being a relatively third-world area to being a first-world area, [some good has come out of it] but also some bad, like an increased risk of breast cancer, which is impacting their survivorship and their health systems.

George: About 14% to 14.2% of participants who were on the study had a germline pathogenic mutation variant in BRCA1/2, PALB2, and, very rarely, in RAD51C, NBN, STK11, TP53, and CHEK2. One in 7 people who were in our study had a germline variant, [which] is very high. Secondly, the majority of people in the Caribbean are of African descent, but we also have a mixed population of Middle Eastern, European, Chinese, and Southeast Asian [individuals]. As a result, we also have a very mixed population that we saw play out in the types of mutations that we [observed].

We did not see the [same] mutations across the entire region. What we saw was that each country essentially had their own spectrum of mutations. This led to us believe that unlike with the Ashkenazi Jewish population where you have 3 [kinds] of mutations, for the purposes of doing genetic testing in a Caribbean population, you would want to offer panel testing across genes and for multiple geneseven the rare [ones].

Hurley: [To reiterate what Dr George said], each island has a very individual profile. We started at the Bahamas and were nave enough to think thatback before panel testing was [accessible]we could just get the mutations from the Bahamas and make a panel from that. [We felt] it would be cheaper than $3500, and [that we could] apply it across the Caribbean. [However], that was a complete failure because each island has different mutations.

Fortunately, technology rescued us: Broad panel testing became available and financially feasible. You could not extrapolate from 1 island to the other [to determine what] the mutations were going to be. You could not extrapolate what the frequency [of those mutations] was going to [to look like]; it was completely unique. For example, we saw a very high incidence of PALB2 in Barbados and Jamaica, but nowhere else. In Barbados, [the incidence of this mutation] was [approximately] 4.4%, while in Jamaica it was 2.2%. These are very high rates of PALB2; we do not see those kinds of rates in any population. In the Bahamas, 28% [of individuals] had a germline mutation vs 11.7% in Trinidad and Tobago, and [approximately] 17.4% in Barbados.

Each island was totally unique because of the mix of people who [live there]. At the beginning of the study, we used to ask people about their racial and ethnic background. We would [ask], How do you describe yourself? Often, in the Bahamas, they would say, It is a conch salad. You take a little of this, a little of that, and throw it all in. That is what I am.

That is kind of what the Caribbean is. You have this very broad, diverse mixture of backgrounds with the [indigenous] population, Southeast Asia, China, Middle East, Northern Europe, Southern Europe, Western Africa, and a little bit Eastern Africa. A lot of different contributions [to make up an individual profile].

Read more:
Unique Landscape of Actionable Pathogenic Variants Unfolds in Breast and Ovarian Cancers in the Caribbean - OncLive

Recommendation and review posted by Bethany Smith

As the COVID-19 outbreak is that, producers are rapidly adjusting their business and buying strategies to meet the demands of a epidemic that has created market-based need for Direct-to-Consumer Genetic Testing. A sequence of both pros and cons shocks will occur over a few months when producers and their suppliers react to increasing customer demands. Many countries look susceptible to export-dependent markets, with an unfavorable global environment. Any factories either close down or decrease their efficiency because of a shortage of downstream demand; the effects of this pandemic would reshape the global Direct-to-Consumer Genetic Testing industry.

To get leading market solutions, visit the link below: https://www.emergenresearch.com/industry-report/direct-to-consumer-genetic-testing-market

Global Direct-to-Consumer Genetic Testing Market COVID-19 Analysis

The report offers an extensive analysis of the factors that are likely to influence the growth of the industry. The research study considers the COVID-19 pandemic a key growth influencing factor. The pandemic has negatively impacted the Direct-to-Consumer Genetic Testing industry through disruptions in global supply chains and grueling economic conditions. The report extensively analyzes the impact of the pandemic on the Direct-to-Consumer Genetic Testing market on a global as well as regional scale. To be updated, the report is furnished with the latest impact of the currently unfolding coronavirus pandemic on the Direct-to-Consumer Genetic Testing industry. The pandemic began in late 2019 and hit the world in early 2020, disrupting the normal lives and affecting every segment of the world. The report covers the impact of the pandemic on the Direct-to-Consumer Genetic Testing market and its key segment. The report also offers an initial and future assessment of the impact of the pandemic on the Direct-to-Consumer Genetic Testing market and offers revenue estimations in a post-COVID-19 scenario.

Key participants include 23andMe, Full Genome Corporation, Color Genomics, Genesis Healthcare, Helix OpCo LLC, FamilyTreeDNA, MyHeritage, Identigene, Pathway genomics, and Living DNA, among others.

The segmentation of the market is done on the basis of product types, application spectrum, and the key regions of the Direct-to-Consumer Genetic Testing market. Along with this, the report also provides extensive coverage of the key companies and their strategic schemes to gain a substantial market position.

The report offers a comprehensive overview of the global Direct-to-Consumer Genetic Testing market scenario pertaining to market value, volume, production and consumption rates, and market segmentation based on types, regions, and applications.

Continued here:
Global Direct-to-Consumer Genetic Testing Market Forecast region |Market Scenario, Opportunity, and Industry Expansion Strategies to 2027 KSU | The...

Recommendation and review posted by Bethany Smith

GRAND RAPIDS, Mich., April 12, 2021 /PRNewswire/ --Today, NxGen MDx announced a partnership with Concentric by Ginkgoto provide COVID-19 testing to congregate settings such as schools operating in-person or hybrid learning environments using a scalable and simple testing modality - pooled testing.

As more and more schools begin to support in-person learning environments, communities across the country have turned to routine COVID-19 testing to monitor the spread of the virus. Even as teachers and guardians become eligible for vaccines, clinical vaccine trials for individuals under 16 are just beginning, and efforts to track and mitigate viral spread remain critical.

Concentric by Ginkgo's goal is to provide easy, affordable pooled testing to every school in America. Pooled classroom testing, which combines swabs from all consenting individuals in a classroom and runs them as a single test, can significantly increase testing capacity and lower the cost of testing programs. In partnership with labs like NxGen MDx, Concentric by Ginkgo currently operates testing for hundreds of schools across America.

"It's our mission to ensure that cost andlack of access are never a factor when it comes to COVID-19 testing," said Alan Mack, CEO of NxGen MDx. "That's why it made perfect sense to partner with Ginkgo."

"As communities consider the many COVID-19 testing models available, pooled testing stands out as an affordable option to identify pockets of prevalence as schools look to reopen," said Jason Kelly, CEO and co-founder of Ginkgo Bioworks. "We started Concentric because everyone's health is connected, and giving schools the capacity to test every student, every week provides the critical information and confidence communities need."

To learn more about Concentric by Ginkgo or to get your school district involved, head over to concentricbyginkgo.com.

About NxGen MDxNxGen MDx LLC is a leading women's health company delivering highly accurate and precise genetic testing. NxGen MDx's history of whole-gene sequencing combined with advanced technology allows us to provide accessible, high-quality testing options to families as they plan for the future. NxGen MDx employs state-of-the-art technology, including rapid molecular diagnostics for infectious disease and genetic screening technology that examines the entire gene rather than parts of the gene, giving women and families a comprehensive assessment of their health. NxGen MDx is based in Grand Rapids, Michigan. To learn more, visit the company's website at http://www.nxgenmdx.com.

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About Ginkgo BioworksConcentric by Ginkgo is Ginkgo Bioworks' public health and biosecurity effort. Headquartered in Boston, Ginkgo uses the most advanced technology on the planetbiologyto grow better products. The company's cell programming platform is enabling the growth of biotechnology across diverse markets, from food to fragrance to pharmaceuticals. Ginkgo is also actively supporting a number of COVID-19 response efforts, including community testing, epidemiological tracing, vaccine development, and therapeutics discovery. For more information, visit http://www.ginkgobioworks.com.http://www.ginkgobioworks.com

SOURCE NxGen MDx

Genetic Carrier Screening

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NxGen MDx Announces Partnership with Ginkgo Bioworks to Scale COVID Response through Pooled Testing in K-12 Schools - PRNewswire

Recommendation and review posted by Bethany Smith

14April 2021,A leading market research company, Facts and Factors recently published a research report onDirect to Consumer Genetic Testing Market: By Types, Segments, Size, Share, Top Players, COVID-19 Impact Analysis and Forecast to 2020-2026. This report is a broad review that includes a detailed overview of the Direct to Consumer Genetic Testing industry. The report explains the type of Direct to Consumer Genetic Testing and its application in different verticals of the market with regard to various countries and key regions. The analysis has listed and evaluated all the key players in theDirect to Consumer Genetic Testing marketand compared them on the basis of different metrics such as annual sales shipments volume, historical growth rates, market revenue, and marketing strategies. On the basis of all these findings, the Direct to Consumer Genetic Testing industry study report proposes strategic plans to improve market positions for existing market participants.

According to the Facts and Factors research analysis,Global Direct to Consumer Genetic Testing market expected a CAGR of 19% and is Garner Revenue Worth USD 2400 million by 2026. DTC testing is a means of marketing genetic testing to consumers without the direct involvement of a health professional..

This Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution, etc., these data help the consumer know about the competitors better. This report also covers all the regions and countries of the world, which shows a regional development status, including market size, volume, and value, as well as price data.

The report provides a cross-sectional analysis of the Direct to Consumer Genetic Testing market in terms of market estimates and forecasts for all the segments across different geographic regions. The report covers all the prevalent trends and technologies playing a major role in the growth of the Direct to Consumer Genetic Testing market over the forecast period. It also highlights various drivers, restraints, and opportunities expected to influence the market growth during the said period.

Request to Download Your Updated Free Sample Research Report on Direct to Consumer Genetic Testing Market:https://www.fnfresearch.com/sample/direct-to-consumer-genetic-testing-market-by-test-718

(The free sample of this report is readily available on request and updated with new research additions).

The Updated FREE Sample Report Includes:

Key Questions Answered in this Report

1) What was the impact of COVID-19 on the Direct to Consumer Genetic Testing market?

2) What is the market size, share of the Direct to Consumer Genetic Testing market?

3) Who are the top market players in the Direct to Consumer Genetic Testing market?

4) What are the major regions in the Direct to Consumer Genetic Testing market?

Top Market Players in the Direct to Consumer Genetic Testing Market:

In finalizing their position in the Direct to Consumer Genetic Testing market player positioning, recent events for these firms, such as new solution/product releases, marketing projects, R&D, partnerships, mergers & acquisitions (M&A), regional expansions, and technical innovations, are considered. For all the key stakeholders of the Direct to Consumer Genetic Testing market value chain and technology ecosystem, the information provided in theDirect to Consumer Genetic Testing market research reportis expected to be beneficial.

This report also provides valuable suggestions for established and new players around the world. Furthermore, the research report includes a precise analysis of business strategy for the growth of the key market players.

For Additional List of Revised Market Players, Request Free Sample Report Here:https://www.fnfresearch.com/sample/direct-to-consumer-genetic-testing-market-by-test-718

Short Term and Long Term Impact of COVID-19 Pandemic on Businesses

We at Facts and Factors (www.fnfresearch.com)understand how difficult it is for you to plan, strategize, or make business decisions, and as such, we have your back to support you in these uncertain times with our research insights. Our team of consultants, analysts, and experts has developed an analytical model tool for markets that helps us to assess the impact of the virus more effectively on the industrial markets. We are further implementing these insights into our reports for a better understanding of our clients.

Download Report PDF Brochure Here to Know the COVID-19 Pandemic Business Impact Analysis on Direct to Consumer Genetic Testing Market:https://www.fnfresearch.com/sample/direct-to-consumer-genetic-testing-market-by-test-718

In addition, the study also recommends business penetration plans for potential entrants to the business. Furthermore, the Direct to Consumer Genetic Testing industry study report has listed the main manufacturers and distributors operating in all the major regions. It is expected that this research and data will enable industry players to improve their networks of market penetration and broaden their geographical scope.

Browse detail report with in-depth TOC @https://www.fnfresearch.com/direct-to-consumer-genetic-testing-market-by-test-718

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Direct to Consumer Genetic Testing Market Size, Share, COVID Impact Analysis and Forecast to (2020-2026) KSU | The Sentinel Newspaper - KSU | The...

Recommendation and review posted by Bethany Smith

Mr Justice Henshaw handed down judgment on 14 April 2021 in Toucan Energy Holdings Ltd v Wirsol Energy Ltd [2021] EWHC 895 (Comm), a substantive dispute begun in 2018 regarding the construction and sale of 19 solar parks located throughout Great Britain and Northern Ireland.

Wirsol has very successfully defended allegations that it designed and constructed the solar parks defectively, which was said to have caused Toucan significant losses and to require effectively a total re-construction of many of the sites due to allegedly fundamental faults risking catastrophic failures.

Wirsol contended that the solar parks were properly built (save for minor issues) and that Toucans losses were minimal. Wirsol also counterclaimed for approximately 6.5 million in unpaid fees arising out of a contract to procure asset life extensions for the solar parks. Toucan denied that the asset life extension invoice was payable, including on the basis that a necessary condition subsequent to payment had not been satisfied or waived.

Toucans total claim amounted to some 30 million in damages, including a significant sum for diminution in value of the solar parks due to ongoing blight in addition to the remedial/replacement costs it said would be incurred, along with lost revenue from under-performance of the solar parks and claims for liquidated damages.

A five-week trial took place in person at the Rolls Building through October and November 2020, with experts in the fields of electrical engineering, solar asset valuation, quantity surveying and transformer design.

Mr Justice Henshaw dismissed the vast majority of Toucans 30 million claim, finding that only a fraction of the losses relating to alleged defects were made out. Toucans claims for the alleged blight and consequential losses arising from refinancing of its debts were entirely dismissed.

The Judge upheld Wirsols counterclaim on almost all issues, finding that c.6.25m was due for the asset life extensions. The judgment contains a detailed discussion of the circumstances in which a waiver can be given under a commercial contract. The Judge ultimately concluded that Toucan was obliged to give a waiver under the contract in certain circumstances, and that it had done so. In any event, Toucans conduct was such that (if required) it would have been found to have given an equitable waiver.

The case also attracted national press attention after it emerged that Toucan had raised very substantial sums from a local authority to refinance its investments (see, for example, here).

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The sun shines for Wirsol Energy: guidance on the circumstances in which waiver can be given in a commercial contract - Lexology

Recommendation and review posted by Bethany Smith

Solid State Disks specialises in the design and manufacture of advanced flash data storage systems

In a Q&A session, Solid State Disks sales director, James Hilken looks at the latest trends and demands surrounding the areas of memory and storage and what the sector needs to consider when designing and developing new systems.

As part of the Reactive Group, Solid State Disks (SSD) delivers innovative system solutions to defence, commercial and industrial customers worldwide, and specialises in the design, development and integration of advanced flash data storage systems. The company is also UK flash memory distributors of SanDisk, Innodisk, APRO, Transcend and Smart Modular.

Q) Why is it important to record vast amounts of data and what advantages does it offer to sub-system manufacturers and technology users on the front line?

James Hilken, sales director of Solid State Disks

Operators use such systems to plan and measure mission success, acquire data, develop pilot training, and sadly to investigate accidents or record system failures. Recordings will be from instrumentation and from audio and video sources. Maintenance and configuration data is also stored for future use. Recent successes have been for legacy avionics, simulation and training systems as well as automatic test equipment.

Q) What in your mind is the correct storage technology?

Solid state flash memory is the preferred choice, but often backwards compatibility with the legacy system is the key, as new interfaces may require mechanical or software changes and/or system/aircraft requalification. The benefits of solid state in harsh operating conditions are well-established.

Q) What are the main customer storage demands?

Meeting performance and environmental specification is key, and price needs to take into account alongside long-term servicing costs and contracts that require contracted levels of availability. Aerospace customers need to meet performance requirements in challenging environments and to manage the consequences of any failure in the drive selection criteria.

Q) How are you assisting the OEMs and Primes with solutions that can be found embedded within systems connected with C4ISR technologies, mission, flight, simulation, training, and condition-based monitoring activities around the world?

Both military and civil customers need supporting where fleets require ongoing maintenance and life extension, but a computer subsystem refresh cannot be afforded. Often the legacy data storage is the weak link. When this can be replaced by new technology the legacy system and its existing software can be given a mid-life extension. If a firm out-of-service date for the platform is known, longer-term decisions can be made, but often that is not the case.

Q) Is there the perception that storage is generally perceived as an afterthought, especially in embedded design?

In some applications, data recording and storage is seen as an extra to the design as a reporting feature, although it is of course, an integral part of any IT product. In the aerospace world this has never been true, but engineers can only select the best technology available at the design stage, which may lead manufacture and product end of life (EOL) by many years. Avionics, simulation and training and automatic test equipment (ATE) is a case in point. Even the best conventional disk and tape drives require reasonable environmental conditions to perform well and can never match the Mean Time Between Failure (MTBF) of solid state.

Q) Is it your experience that technology moves on so fast and aerospace so slow that in some cases the initial requirement has become obsolete?

Yes, this is certainly true of data storage, and often true of the initial aerospace requirement. However, not only is it possible to deliver fit, form and function replacements for legacy storage devices more than thirty years old, it is also possible to provide features never conceived of at the original design point, but regarded as essential today. For example, off- and on-line backup via Ethernet connection or rugged removable flash media, duplicate media for additional data security, and encryption or other protection against an illegally acquired asset.

Q) Why is obsolescence in memory and storage increasingly more important when designing and developing systems especially those that need to run for many years without fault?

SSDs SCSIFlash (CF) network drive

Memory technology has historically been driven firstly by desktop computing, then gaming and mobile communications, but is now driven by data servers and cloud computing. The interface standards used to interconnect storage products with the host are also subject to rapid change driven by the need for performance. Product form factors are also driven by the need for small size, weight and low power consumption. Aerospace customers and designers need to be aware of the latest technology and plan to future proof their designs, bearing in mind that often the latest parts take some time to pass reliability and qualification testing for use in harsh environments.

Q) Do you provide a complete service that covers potential obsolescence issues? For example, what happens if a manufacturer issues an end of life notification?

Yes, in general. We are governed by our suppliers product planning, but work closely with them to ensure our products remain supportable. This includes early EOL warnings. To some extent we are buffered from the memory device level interface specification by the legacy interface we are required to emulate.

Q) Would you say that COTS still isnt fit for purpose, i.e. specialist kit requires specialist storage?

It is recognised that commercial systems and storage products may not work well or at all for long periods in hot, humid, high vibration, and extreme temperature cycling applications. Equally important is that commercial systems often do not have change control and configuration discipline to ensure subsequent purchases are made to the same specification. For COTS or MOTS (modified COTS) solutions, industrial solid state flash memory is usually suitable for the majority of applications. It all depends on the specification.

Q) Are manufacturers of storage upping their game?

SSD has worked with leading customers in key market sectors to provide our networked Touchless Removable drive solutions. Customers benefit from access to systems via our Direct Ethernet port from a host system. This allows data to be kept in a digital library located anywhere within the organisation without the need for physical media.

Many of the flash lines SSD support are fully compliant with aerospace and defence standards

Direct Ethernet is also available on our hard disk emulations, which allows a full copy to be remotely taken of a system drive and restored as a bootable image. This dramatically reduces the time taken to rebuild systems in the event of data corruption. SMART is used as an excellent means of monitoring the remaining life of Flash media.

Q) What kind of industry landscape will we be faced with when everything gets back to a new normal?

Businesses have become more cost-conscious, with reserves and working capital hard hit despite support from the Government for some companies. It is anticipated that life extensions and out-of-service dates will be extended for reliable systems. Sustainability and energy consumption in developing new products is also driving new product investment decisions.

Q) Where to next for your company?

Were looking at the development of alternative interface replacement devices to supplement the existing SCSI and IDE ranges. Were also investigating offering system level support to engineers using our proprietary technology and experience.

https://solidstatedisks.co.uk

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Recommendation and review posted by Bethany Smith

JK Group, part of Dover Digital Printing and Dover, announced several new initiatives that support improved sustainability.

JK Group said it developed a new way of packaging its reactive inks to reduce its environmental footprint, while also releasing an extended shelf life across all of its ink ranges.

The company's Kiian brand recently launched a new reactive ink series called Digistar Bellagio, with one of the new departures for this range being its Bag-in-Box (BiB) packaging.

Through this environmentally-friendly initiative, all reactive inks will now be packed in flexible, multilayered plastic bags, which are incorporated into a cardboard outer shell.

The system has several advantages and represents an 80% reduction in the use of plastic compared to traditional high-density polyethylene containers of a similar capacity, according to JK Group.

Additionally, the reduced packaging creates a reduction in shipping and warehouse space, leading to lower transport and storage costs and less environmental impact across the supply chain, according to JK Group.

"We are thrilled about these initiatives because not only are they better for the planet, but also for our customers, who are looking for cost-efficient ways to reduce their environmental impact. This represents a continued effort by all of our companies to improve the sustainability of our products," said Abhi Agrawal, president of Dover Digital Printing.

JK Group also announced a shelf life extension of all of its ink series. Harnessing continuous improvements in performance brought about by research and development activities, the company has conducted an in-depth analysis of its ink formulations, including stress tests, to determine more precisely how long they will last on the shelf.

The resulting extension of shelf life means improvements for stock management and order rationalization, enabling efficiency and cost savings.

See the original post here:
JK Group Unveils Sustainability Initiatives - Covering the Printing Inks, Coatings and Allied Industries - Ink World - Ink World Magazine

Recommendation and review posted by Bethany Smith

Pyrroloquinoline Quinone Market Latest Research Report 2021- 2026 covers a complete market scenario across the globe with a detailed industry analysis of major key players like Haotian Pharm, Doctors Best, Health Thru Nutrition, Life Extension, NOW Foods, Swanson, etc. This report provides strategic recommendations consulted by the industrial experts including market forecasts, profit, supply, raw materials, cost structures, investment landscape, latest market trends, demands, and much more.

The report begins from overview of the Industry Chain structure, and describes the industry environment, then analyses the market size and forecast of Pyrroloquinoline Quinone by product, region, and application, in addition, this report introduces the market competition situation among the vendors, market price analysis, and value chain features are covered in this report.

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Pyrroloquinoline Quinone Market Segmentation:

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Market Assessment of Pyrroloquinoline Quinone Industry: Growth Drivers by Top Players like Haotian Pharm, Doctor's Best, Health Thru Nutrition, Life...

Recommendation and review posted by Bethany Smith