Category Archives: Gene Medicine

GCC governments have invested significantly in healthcare infrastructure in the past couple of decades. Evidently, healthcare cities and luxurious health complexes are mushrooming, raising the quality of medical services, and aspiring to establish the gulf region as a medical hub.

With a world leading infrastructure, the healthcare sector is ready for the future that will come with a new set of foreseen and unforeseen challenges, including a growing population and density, growing life expectancy, share of population over 65, changing lifestyle, and more.

Related: Clinical Communication & Collaboration Platforms: Foundational Technology for Smart Hospitals

Conversely, the upcoming trends in health-tech services illustrate a promising perspective, empowering patients to play an active role in health management at every stage of the treatment process. Meanwhile, technology advancements will have a-far-reaching impact that enables prevention, diagnostics, treatment, rehabilitation and care.

Related: Interconnected solutions can boost personalised healthcare systems

The changing demographics and epidemiological trends are the main factors contributing to the increasing healthcare demand in the gulf countries. However, transformation programs across larger economies, such as the Kingdom of Saudi Arabia and the United Arab Emirates, are driving a fundamental change that aims to leapfrog the healthcare industry from an emerging market status to a market of opportunity and transformation.

Jad Bitar, Managing Director and Senior Partner, Boston Consulting Group (BCG)

The expanding and ageing population, high prevalence of noncommunicable diseases (NCDs), rising cost of treatment and increasing penetration of health insurance are contributing to the higher costs in the industry. Navigating through these challenges requires a deeper look into personalised healthcare, developing a patient's "digital profile. Several technologies are pushing the boundaries of traditional medicine and enabling the transition to personalised medicine and more customised treatments, including targeted and gene medicine, bionics and robotics technology, tissue engineering and 3D printing. Overall, the wave of innovation is expected to offer more personalised services and treatment options especially coupled with Artificial Intelligence (AI), Machine Learning and massive data. This will usher the era of consumer-driven digital health that has been much talked about in the last decade.

We have recently conducted extensive research across 25 cities and identified trends that are shaping the future of healthcare. With digital and technology acting as fundamental shaping forces, five key trends stand out:

Patient empowerment: Patients will take an increasingly active role in the patient journey, leveraging access to their own health records and personal health analytics, home testing kits, personal electronic monitoring devices, etc. to shape their health and become a major component in the decision-making process.

Prevention: Health systems will benefit from predictive diagnosis of diseases, supported by technologies such as AI-enabled risk profiling, epidemiological understanding of different districts and cities, and data analytics for targeted health screenings. This will be the foundation of predictive-preventive systems. 2

Personalised treatment: New technologies (e.g., tissue engineering and 3D printers) will enable personalised and more accurate treatment allowing the arrival of bespoke medicine; health systems will build and leverage a patients 'digital profile' to enable targeted treatment for individuals as well as populations with similar profiles.

Integrated delivery models: Significant shifts are underway, from in-patient to out-patient care, and the emergence of alternative patient friendly formats and seamless virtual health delivery networks, to home care and malls care, as well as other formats.

Healthcare Professional 4.0: Machine enabled diagnosis will facilitate a symbiosis between health professionals and AI, where digital image diagnostics, virtual reality, digital twins, and routine surgery will deliver greater value and better health results. 4.0 health professionals will require increased digital, cognitive and behavioral skills.

Rapid health technological advancements in the GCC result from the regions agility to overcome the COVID-19 pandemic with an expedited rollout of technology-enabled solutions. Healthcare has witnessed a more prominent and disruptive change with a massive shift towards prevention rather than cure through Health-Tech solutions that are more data-driven and are creating efficiencies in operations, costs, and delivery of care. Moving forward, decision-makers may leverage these solutions at-scale by envisaging public-private partnerships with HealthTech start-ups, consequently driving growth across the servicing chain in the health sector.

By Jad Bitar, Managing Director and Senior Partner, Boston Consulting Group (BCG)

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The Future of Healthcare: Five Trends in HealthTech Services - Omnia Health Insights

NEW YORK & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apertura Gene Therapy, a biotechnology company opening opportunities for treating debilitating diseases with limited options for patients, today announced that it has launched with a Series A financing of up to $67M from Deerfield Management Company to develop genetic medicines using platform technologies that address key limitations of genetic medicine delivery and expression. Deerfield Management has also committed additional operational support to further strengthen the companys ability to advance gene therapy discoveries.

Apertura is founded on a pair of platform technologies developed in the labs of Ben Deverman, Ph.D., Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard, and Michael Greenberg, Ph.D., the Nathan Marsh Pusey Professor of Neurobiology and Chair of the Department of Neurobiology at Harvard Medical School (HMS). The companys platform leverages machine learning and high-throughput assays to engineer novel capsids, gene regulatory elements, such as promoters and enhancers, and payloads to simultaneously enhance multiple functions of gene therapies for greater translational potential.

With these platform technologies from the Broad Institute and Harvard University, Deerfield saw an opportunity to bring together and support a unique and comprehensive platform that could address technical challenges that have prevented gene therapy from reaching its full potential, said Dave Greenwald, Ph.D., Acting Chief Executive Officer of Apertura and Vice President, Business Development at Deerfield Management Company. While next-generation approaches to gene therapy have largely focused on the innovation of delivery vectors, Apertura has the potential to innovate simultaneously across delivery, expression, and payloads.

Ben Deverman, Scientific Founder of Apertura, said: When developing a gene therapy, it has been common to use naturally occurring serotype AAV capsids. The technology we have developed uses proprietary assays and machine learning to design custom AAV capsids that have the chosen characteristics for treating specific diseases, and we believe this approach will result in new and effective gene therapies.

Apertura has certain exclusive rights to AAV capsids developed in the Deverman Lab at the Broad Institute.

A separate sponsored research and licensing agreement with Harvard University, spearheaded by the Harvard Office of Technology Development, grants Apertura exclusive access to certain powerful methods of identifying cell type-specific genetic regulatory elements (GREs), including access to the Paralleled Enhancer Single-Cell Assay (PESCA) platform, developed in the Greenberg Lab at Harvard University.

A major challenge in developing effective gene therapies is having the payload of the therapy expressed at the correct level in target cells, said Greenberg. "The technology we have developed at Harvard Medical School overcomes this hurdle by targeting transgene expression to specific cell types, fine-tuning expression levels in these cells, and, at the same time, avoiding expression of the transgene in non-target cell types. The Greenberg labs PESCA platform was advanced to commercial readiness through the strategic support of the Q-FASTR program at HMS and the Blavatnik Biomedical Accelerator at Harvard University.

The two technology platforms have unique capabilities to simultaneously engineer AAV capsids to exhibit enhanced cellular tropism, evasion of pre-existing immunity, while maintaining and potentially improving manufacturability. The companys GRE platform focuses on GREs and enhancers that drive cell type-specific expression, disease state-specific expression, and tunable expression levels. These capabilities together are expected to enable Apertura to develop best-in-class gene therapies designed for specific indications.

Our platform has the potential to unlock many new indications for gene therapy, said Kristina Wang, Director of Corporate Development and Board Member of Apertura. We aim to maximize our impact through dedicated internal programs and meaningful partnerships with other biopharma companies and academic groups. Committed to advancing the field of gene therapy, Apertura seeks to collaborate broadly to accelerate impact to patients.

About Apertura Gene TherapyApertura is a biotechnology company opening opportunities for treating currently intractable diseases. We are uniquely positioned to develop genetic medicines by simultaneously engineering AAV capsids, genetic regulatory elements, and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity, and manufacturability. Apertura is committed to growing the field of gene therapy and believes that together we maximize our impact by working with corporate and academic partners, patients, and foundations. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, the company is based at the Cure, Deerfields innovation campus in New York City. For more information, please visit our website at http://www.aperturagtx.com and follow us on LinkedIn and Twitter.

About Deerfield ManagementDeerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, please visit https://deerfield.com/.

About Harvard Universitys Office of Technology DevelopmentHarvards Office of Technology Development (OTD) promotes the public good by fostering innovation and translating new inventions made at Harvard University into useful products that are available and beneficial to society. Our integrated approach to technology development comprises sponsored research and corporate alliances, intellectual property management, and technology commercialization through venture creation and licensing. More than 90 startups have launched to commercialize Harvard technologies in the past 5 years, collectively raising more than $4.5 billion in financing. To further bridge the academic-industry development gap, Harvard OTD manages the Blavatnik Biomedical Accelerator and the Physical Sciences & Engineering Accelerator. For more information, please visit https://otd.harvard.edu.

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Apertura Gene Therapy Launches with $67M Series A Financing from Deerfield and an Innovative Technology Platform to Develop Genetic Medicines -...

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Global Gene Therapy Medicine Market 2022 | Demand and Scope with Outlook, Business Strategies, Challenges and Forecasts to 2028 Ripon College Days -...

Precision medicine and genetic testing will reign supreme when it comes to the future of Alzheimer disease care and treatment advancement, according to Richard S. Isaacson, MD, director of the Alzheimer's Prevention Clinic and associate professor of Neurology at Weill Cornell Medicine and New York-Presbyterian.

Isaacsons session Treatment Updates in Alzheimer's Disease: Tailoring Management and Care Approaches to Improve Outcomes, was presented atthis year'sNational Association of Managed Care Physicians Spring Managed Care Forum, held in Orlando, Florida, April 21-22.

Kicking off his presentation, Isaacson remarked on the recent FDA approval and CMS coverage determination of Aduhelm (aducanumab), the first new treatment for the disease brought to market in nearly 2 decades. The treatment functions by targeting the buildup of amyloid beta plaques in the brain, which are thought to be a pathophysiological driver of the neurodegenerative disease.

Following its controversial launch, CMS announced it would only cover administration if the drug were delivered in a clinical trial setting.

Despite the attention that development garnered from the press and public alike, Isaacson stressed a lesser-known development in Alzheimer disease detectionthat a blood-based biomarker for amyloid exists, offering an alternative to testing of cerebral spinal fluid.

Most people are unaware of [the blood-based biomarker], he said, equating the advancement to a cholesterol test for the brain.

Diagnostic testing is important because even though individuals may exhibit symptoms of the disease at age 85, the disease first starts in the brain at age 55 or 60. It begins in the brain 20 to 30 years before the first symptom of memory loss begins, providing a wide window of potential intervention time, Isaacson explained.

The disease is also a spectrum, based on the presentation of certain clinical symptoms over decades, he stressed. Individuals can have amyloid present in the spinal fluid for years and not exhibit symptoms, with recent data showing the amyloid levels in the blood actually increase prioir to those in spinal fluid.

Alzheimers disease affects 46 million Americans, Isaacson said. Not all of those people are going to get dementia because they're going to die from something else.

With regard to genetic testing, studies have shown presence of the APOE4 gene may increase individuals risk of dementia. However, if individuals have multiple copies of the APOE variantand 10% of the Alzheimer disease population doesthey are at an increased risk of side effects for aducanumab, underscoring the importance of precision medicine.

Genetic testing is controversial, Isaacson said, but the practice opens up a realm of possibilities for precision medicine for those with the highly heterogenous disease.

Lots of different genes can contribute to development of Alzheimer disease, and based on these, patients can and should receive different treatments, he stressed. In a few years, Isaacson expects computer programs to be able to determine polygenetic risk for the disease, taking these genetic factors into account as opposed to singular variants in isolation. But just because an individual is at risk does not mean they will develop the disease.

Another aspect of developing personalized care for this patient population is tracking of bodily metrics like blood sugar, as research has proved poor blood sugar control is associated with cognitive problems.

Currently, no curative medications for Alzheimer disease exist, only disease modifying therapies which provide modest benefits to patients. For these, Isaacson stressed the notion of start low, go slow, to help mitigate or even avoid any potential adverse effects. However, as more and more therapeutic targets are identified in the research, the potential for new treatment grows.

With regard to preventive measures, Isaacson highlighted the protective effects of the Mediterranean diet, regular exercise and sleep schedules, and low levels of stress and blood pressure. Cognitive engagement activities like listening to and practicing music can also help prevent decline.

Although the metabolic benefits of medications like semaglutide have been proven, and this could in turn help reduce Alzheimer disease risk, Isaacson prefers to recommend his patients follow behavioral changes and use devices like continuous glucose monitors to help assess metabolic metrics.

Overall, precision medicine and vascular risk factor care will kind of turn Alzheimer's disease and cognitive decline on its head. I think were just a couple of years away from that, he said.

Based on modifiable risk factors, 4 out of every 10 cases of Alzheimer disease may be preventable, he concluded, but cautioned that even though someone may do everything right, they can still get the disease.

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The Promise of Precision Medicine and Genetic Testing in Alzheimer Disease - AJMC.com Managed Markets Network

RenaCARE study expected to be fully enrolled by end of Q2 of 2022

AUSTIN, Texas, April 26, 2022 /PRNewswire/ -- Natera, Inc.(NASDAQ: NTRA), a global leader in cell-free DNA testing, today announced the RenaCARE (Renasight Clinical Application, Review and Evaluation) study - a real world, prospective, multi-center clinical study to assess the clinical utility of the Renasight genetic testing panel. The study has already enrolled 1,600 patients across 25 sites, representing leading academic and private nephrology clinics in the U.S., and will enroll up to 2,000 patients. It is expected to complete enrollment in the second quarter of 2022 with a publication expected to be submitted in late 2022.

The study aims to demonstrate how genetic findings impact the management of patient care and examines diagnostic outcomes of patients tested with the Renasight genetic testing panel. In addition, the study will assess patient satisfaction, health knowledge and genetic literacy. This study follows a 2019 publication1 in The New England Journal of Medicine (NEJM) showing that 89% of patients who tested positive with a multi-gene genetic test had actionable clinical implications.

"Chronic kidney disease affects more than 10% of the global population, and our 2019 NEJM study showed roughly a 10% genetic yield among CKD patients," said Ali Gharavi, M.D., chief of the Division of Nephrology at New York-Presbyterian/Columbia University Irving Medical Center, director of the Center for Precision Medicine and Genomics in the Department of Medicine, interim director of the Institute of Genomic Medicine at Columbia University Vagelos College of Physicians and Surgeons, the study's principal investigator and a close collaborator with Kidney Disease: Improving Global Outcomes foundation (KDIGO) and the National Kidney Foundation (NKF). "We're optimistic that this study will show that next-generation sequencing (NGS) multi-gene assays can be used in a real world setting, to inform and guide disease management and help improve patient outcomes."

"We're confident that the RenaCARE study will confirm the high clinical utility shown in prior studies. This will be an important addition to the growing body of evidence showing the value of genetic testing to clarify an undifferentiated diagnosis, identify a genetic subtype within a diagnosis, reclassify a diagnosis, or provide insights for genetic counseling, family planning and clinical trial access," said Hossein Tabriziani, M.D., senior medical director of organ health for Natera.

All patients undergoing testing using the Renasight panel are offered optional pre- and post-test genetic information sessions with a genetic counselor in addition to their test results. Similarly, providers have access to Natera's genetic counselors for questions about the Renasight testing panel and review of test results.

Natera designed and launched the Renasight genetic testing panel with the feedback of general nephrologists, pediatric nephrologists, and transplant nephrologists. Natera has performed over 10,000 Renasight tests to date.

About Renasight

The Renasight test is a germline genetic test that screens for hereditary causes of kidney disease. It is indicated for patients with diagnosed kidney disease and is run from a patient's blood or saliva sample. Providers can use the Renasight test to identify a genetic predisposition, clarify a clinical diagnosis, or identify the etiology of an unknown kidney disease to help inform medical management. Additionally, genetic counseling and familial testing can be offered based on the test result. The test has been developed and its performance characteristics determined by the CLIA-certified laboratory performing the test. The test has not been cleared or approved by the U.S. Food and Drug Administration (FDA). CAP accredited, ISO 13485 certified, and CLIA certified.

About Natera

Natera is a global leader in cell-free DNA testing, dedicated to oncology, women's health, and organ health. Our aim is to make personalized genetic testing and diagnostics part of the standard of care to protect health and enable earlier and more targeted interventions that help lead to longer, healthier lives. Natera's tests are validated by more than 100 peer-reviewed publications that demonstrate high accuracy. Natera operates ISO 13485-certified and CAP-accredited laboratories certified under the Clinical Laboratory Improvement Amendments (CLIA) in Austin, Texas and San Carlos, California. For more information, visit http://www.natera.com.

Forward-Looking Statements

All statements other than statements of historical facts contained in this press release are forward-looking statements and are not a representation that Natera's plans, estimates, or expectations will be achieved. These forward-looking statements represent Natera's expectations as of the date of this press release, and Natera disclaims any obligation to update the forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual results to differ materially, including with respect to whether the results of clinical or other studies will support the use of our product offerings, our expectations of the reliability, accuracy and performance of our screening tests, or of the benefits of our screening tests and product offerings to patients, providers and payers. Additional risks and uncertainties are discussed in greater detail in "Risk Factors" in Natera's recent filings on Forms 10-K and 10-Q and in other filings Natera makes with the SEC from time to time. These documents are available atwww.natera.com/investorsandwww.sec.gov.

ContactsInvestor Relations:Mike Brophy, CFO, Natera, Inc., 510-826-2350Media:Kate Stabrawa, Communications, Natera, Inc., 720-318-4080pr@natera.com

References

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Natera Announces Definitive Study to Evaluate the Clinical Utility of Renasight in the Diagnosis and Management of Chronic Kidney Disease (CKD) -...

An agreed Paediatric Investigation Plan (PIP) is the regulatory pathway to market authorization for leniolisib as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in children

A Promising Innovative Medicine (PIM) designation is an early indication that leniolisib is a candidate for the MHRA's Early Access to Medicines Scheme

LEIDEN, the Netherlands, April 26, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or the "Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that it has received a positive decision from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) on a Paediatric Investigation Plan (PIP) submission for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3K) inhibitor, for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in patients from 1 year of age to less than 18 years of age. The Company also announces that the MHRA has granted Promising Innovative Medicine (PIM) designation to leniolisib for the treatment of APDS.

A PIP is a development plan aimed at ensuring that the necessary data are obtained to support the marketing authorization of a medicine in the pediatric population. All applications for marketing authorization for new medicines in children require the results of studies as described in an agreed PIP, unless the medicine is exempt due to a deferral or waiver. The leniolisib PIP includes two planned global clinical trials in pediatric patients with APDS, the first in children ages 4-11 years and the second in children ages 1-6 years. The Company expects to initiate recruitment for this pediatric program for leniolisib during the second half of 2022.

A PIM designation indicates that a medicinal product is a promising candidate for the MHRA's Early Access to Medicines Scheme (EAMS), which provides pre-market access to products that are intended for the treatment, diagnosis, or prevention of a life-threatening or seriously debilitating condition and have the potential to address an unmet medical need.

Anurag Relan MD, Pharming's Chief Medical Officer, commented:

"We are pleased to have received agreement of this PIP and the PIM designation from the MHRA, these represent important regulatory milestones as we continue to advance leniolisib for the treatment of APDS, a rare, often debilitating, and sometimes fatal condition. The agreed PIP and the PIM designation further support our confidence in the potential of leniolisib to address APDS, as they provide us with a pathway toward marketing authorization in the United Kingdom. Pharming looks forward to continuing to work with regulatory authorities globally to bring leniolisib to patients with this significant unmet medical need as expeditiously as possible."

Leniolisib, which has received Orphan Drug Designation in Europe, received the same designation from the US Food and Drug Administration on January 30, 2018.

As previously announced, Pharming plans to begin submitting global registration filings for leniolisib in the second quarter of 2022 and, subject to approval, launching the treatment in the US in the first quarter of 2023 and starting a series of European launches in the second half of 2023.

About Activated Phosphoinositide 3-Kinase Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately one to two people per million. Also known as PASLI, it is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3K (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3K pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.

About leniolisib

Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger specifically activating AKT and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3K and PI3K, which are ubiquitously expressed, PI3K and PI3K are expressed primarily in cells of hematopoietic origin. The central role of PI3K in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3K is a valid and potentially effective therapeutic target for several immune diseases.

To date, leniolisib has been well tolerated during both a Phase 1 first-in-human trial in healthy subjects and a Phase II/III registration-enabling study.

About Pharming Group N.V.

Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit http://www.pharming.com.

Forward-looking Statements

This press release contains forward-looking statements, including with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to the conduct of its business, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2021 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2021 filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release.

References:

1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.

2. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218.

3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol. 2019;143(5):1676-1687.

4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606.

5. Maccari ME, et al. Front Immunol. 2018;9:543.

6. Jamee M, et al. Clin Rev Allergy Immunol. 2019;May 21.

7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338

For further public information, contact:Pharming Group, Leiden, The NetherlandsSijmen de Vries, CEO: T: +31 71 524 7400 E: [emailprotected]

FTI Consulting, London, UK Victoria Foster Mitchell/Alex ShawT: +44 203 727 1000

FTI Consulting, USA Jim PolsonT: +1 (312) 553-6730

LifeSpring Life Sciences Communication, Amsterdam, The NetherlandsLeon MelensT: +31 6 53 81 64 27E: [emailprotected]

US PR:Emily VanLareE: [emailprotected]T: +1 (203) 985 5596

EU PR:Dan CaleyE: [emailprotected]T: +44 (0) 787 546 8942

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SOURCE Pharming Group N.V.

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Pharming receives agreement of Paediatric Investigation Plan and Promising Innovative Medicine designation for leniolisib from UK MHRA - PR Newswire